Chapter 17. Prenatal Diagnosis. Will Obs

 Chapter 17. Prenatal Diagnosis

BS. Nguyễn Hồng Anh

Prenatal diagnosis is the science o identiying congenital abnormalities and genetic conditions in the etus. It encompasses the

diagnosis o structural malormations with specialized ultrasound, screening tests or aneuploidy, carrier screening or

genetic diseases, and diagnostic tests perormed on chorionic

villi and amnionic uid.

Based on population-based registry data including births,

etal deaths, and pregnancy terminations, 4 per 1000 pregnancies have a chromosomal abnormality (Wellesley, 2012).

I chromosomal microarray analysis (CMA) is perormed on

chorionic villi or amnionic uid, an additional 4 per 1000 are

ound to have a pathogenic copy number variant, such as a

microdeletion or microduplication (Srebniak, 2017). It is

important or all pregnant women to be oered both screening

and diagnostic tests.

Te goal o prenatal diagnosis is to provide accurate inormation about short- and long-term prognosis, recurrence risk,

and potential therapy. Nondirective counseling and provision

o unbiased knowledge are paramount. Management o an

aected pregnancy that includes diagnostic testing, discussion

o potential etal therapy options and postnatal care, and decisions related to expectant management or pregnancy termination are all incorporated into counseling (Flessel, 2011). Fetal

imaging o congenital anomalies is discussed in Chapter 15,

etal therapy in Chapter 19, and pregnancy termination in

Chapter 11.

HISTORICAL PERSPECTIVE

Nearly 50 years ago, Brock (1972, 1973) observed that pregnancies complicated by neural-tube deects had higher levels

o alpha-etoprotein (AFP) in maternal serum and amnionic

uid. Widespread serum screening began in 1977, ater a collaborative trial rom the United Kingdom established the association between elevated maternal serum AFP levels (MSAFP)

and etal open neural-tube deects (Wald, 1977). Screening at

16 to 18 weeks’ gestation detected 90 percent o etuses with

anencephaly and 80 percent o those with open spina bida,

similar to current screening perormance (American College o

Obstetricians and Gynecologists, 2019c).

Te terms level I and level II ultrasound were coined in this

context. In the Caliornia MSAFP Screening Program o the

1980s and early 1990s, the rst step in evaluation o an abnormally elevated MSAFP was a level I ultrasound examination.

Tis screening examination demonstrated that one third o

pregnancies with MSAFP elevation had incorrect gestational

age, multietal gestation, or etal demise as the etiology (Filly,

1993). Amniocentesis was then oered to the remaining two

thirds. I the amnionic uid AFP concentration was elevated,

a level II ultrasound examination was perormed to detect and

characterize a etal abnormality.

Additionally, an elevated amnionic uid AFP level prompted

a concurrent assay or amnionic uid acetylcholinesterase.

Because acetylcholinesterase leaks rom the exposed neural

tissue into the amnionic uid, presence o both analytes was

used to conrm the diagnosis. However, other etal abnormalities are associated with elevated amnionic uid AFP and

positive assay results or acetylcholinesterase. Tese include

ventral wall deects, esophageal atresia, etal teratoma, cloacal exstrophy, and skin abnormalities such as epidermolysis

bullosa.

Most neural-tube deects are now detected with a standard

ultrasound examination, and the detailed ultrasound examination

is the preerred diagnostic test (American College o Obstetricians

and Gynecologists, 2019c; Dashe, 2006). Level II ultrasound is

no longer an appropriate synonym or detailed ultrasound examination, because the study now includes a much more comprehensive evaluation o etal anatomy (Chap. 14, p. 251).

As MSAFP screening was being adopted, the designation

“advanced maternal age” (AMA) became popular. A 1979

National Institutes o Health Consensus Development Con-

erence recommended advising pregnant women who were 35

years and older about the possibility o amniocentesis or etal

karyotyping. Te threshold was based on the greater risk or

selected etal chromosomal abnormalities with increasing maternal age (Table 17-1). Tere was also an assumption that the

loss rate attributable to amniocentesis was equivalent to the etal

Down syndrome risk at maternal age 35. Tis is no longer the

case.

In 1984, Merkatz and colleagues reported that midtrimester MSAFP levels were lower in pregnancies with trisomies

21 and 18 than in unaected pregnancies. Maternal age was

incorporated into the calculation, which enabled assignment o

a specic risk (DiMaio, 1987; New England Regional Genetics Group, 1989). Te MSAFP screen detected approximately

25 percent o cases o etal trisomy 21 when the threshold ratio

or a positive result was set at 1:270—the approximate secondtrimester risk at maternal age 35. Tis trisomy 21 risk threshold

and the associated 5-percent alse-positive rate became standards that remain in use in some laboratories.

ANEUPLOIDY SCREENING

Aneuploidy is the presence o one or more extra chromosomes,

usually resulting in trisomy, or loss o a chromosome—monosomy

(Chap. 16, p. 309). O recognized pregnancies with chromosomal abnormalities, trisomy 21 accounts or approximately hal

o cases; trisomy 18 accounts or 15 percent; trisomy 13, or

5 percent; and the sex chromosomal abnormalities—45,X,

47,XXX, 47,XXY, and 47,XYY—or approximately 12 percent.

Screening tests are available or some or all o these aneuploidies.

Te risk or etal trisomy increases with maternal age, particularly ater age 35 (Fig. 16-2, p. 310). Te positive predictive value o all aneuploidy screening tests is higher or

women aged 35 years and older. Such women account or

18 percent o deliveries in the United States (Fig. 17-1). At

Parkland Hospital, the majority o Down syndrome births

occur in this age group (Hussamy, 2019). Other important

aneuploidy risk actors include a numerical chromosomal

abnormality or structural chromosomal rearrangement in the

woman or her partner—such as a balanced translocation—or a

prior pregnancy with autosomal trisomy or triploidy.

TABLE 17-1. Estimated Risks for Fetal Trisomy 21 and Any Aneuploidy

According to Maternal Age and Timing of Diagnosis

Trisomy 21 Any Aneuploidy

Age

Chorionic Villus

Sampling Amniocentesis Delivery Amniocentesis Delivery

35 1/249 1/280 1/356 1/132 1/204

36 1/196 1/220 1/280 1/105 1/167

37 1/152 1/171 1/218 1/83 1/130

38 1/117 1/131 1/167 1/65 1/103

39 1/89 1/100 1/128 1/53 1/81

40 1/68 1/76 1/97 1/40 1/63

41 1/51 1/57 1/73 1/31 1/50

42 1/38 1/43 1/55 1/25 1/39

43 1/29 1/32 1/41 1/19 1/30

44 1/21 1/24 1/30 1/15 1/24

45 1/16 1/18 1/23 1/12 1/19

Adapted from Hook, 1983; Snidjers, 1999.

0

Percent

2

10

8 6 4

12

14

16

18

Year

2018

18.0

15.5

2010

14.2 14.3

2002

13.7

12.8

1994

11

8.8

1986

6.9

FIGURE 17-1 Trends in the percentage of births to women aged

35 to 44 years. (Data from the Centers for Disease Control and

Prevention, 2015, 2019).334 The Fetal Patient

Section 6

Broadly speaking, there are two types o aneuploidy screening tests, those that are analyte-based and those that are cell-

ree DNA-based. Cell-ree DNA (cDNA) screening is more

sensitive and specic than analyte-based screening, and it has a

much higher positive predictive value. One prospective screening study or trisomy 21 in 15,000 pregnancies ound that the

average positive predictive value was 80 percent or cDNA but

only 3 percent or rst-trimester screening (Norton, 2015).

However, analyte-based screening has the unusual benet that

an abnormal result may indicate a genetic abnormality or

which screening was not specically perormed (p. 337). Te

position o the American College o Obstetricians and Gynecologists (2020e) is that no one screening test is superior in all

circumstances. No aneuploidy screening test is diagnostic, and

prenatal diagnosis is strongly recommended beore acting upon

a result.

Tere are selected circumstances in which aneuploidy screening is not recommended. Following diagnosis o a major etal

abnormality, prenatal diagnosis with amniocentesis or chorionic villus sampling is advised. Te etal risk cannot be normalized with a negative aneuploidy screening result. Screening

results can be alsely negative, and major abnormalities coner

risk or genetic syndromes not identied through screening

tests. Analyte-based screening tests are not valid in the setting o an abnormality that aects the AFP component, such

as a neural-tube deect or ventral-wall deect. Additionally, all

aneuploidy screening methods are less accurate in twin gestations and are invalid in triplet gestations and higher-order

multiples.

Aneuploidy screening should be an inormed patient choice

that incorporates the patient’s clinical circumstances, values,

interests, and goals. Counseling should be individualized, with

the understanding that at least 20 percent o women preer

not to receive aneuploidy screening, even when nancial barriers are removed (Kuppermann, 2014). Elements o counseling prior to aneuploidy screening are listed in Table 17-2. Te

screening test(s) oered and elected may depend on actors

such as gestational age, provider location or practice setting,

and the patient’s out-o-pocket costs.

■ Test Characteristics

Validity characteristics convey inormation about how well an

aneuploidy screening test dierentiates aected rom unaected

pregnancies. Te sensitivity, also known as the detection rate, is the

proportion o etuses with a particular aneuploidy who are detected

by the screening test. Te converse is the alse-negative rate. I a

woman elects a rst-trimester aneuploidy screen with a sensitivity

o 80 percent or trisomy 21, the alse-negative rate is 20 percent,

so the test is anticipated to miss 1 in 5 aected pregnancies.

Specicity is the proportion o unaected pregnancies with

a negative screening result. Te converse o specicity is the

TABLE 17-2. Aneuploidy Screening Counseling Elements

All pregnant women have 3 options: screening, diagnostic testing, and no screening or testing.

The purpose of a screening test is to provide information, not to dictate a course of action.

Diagnostic testing is safe and effective and provides information that screening does not.

There are differences between a screening test and a diagnostic test.

Screening evaluates whether the pregnancy is at increased risk for specific conditions and estimates the degree of risk.

Screening does not provide information regarding all genetic abnormalities.

With a negative screening test result, the risk is decreased but not eliminated.

With a positive screening result, a diagnostic test is recommended if the patient wants to know whether the fetus is

affected.

Irreversible management decisions should not be based on screening test results.

Cell-free DNA screening does not always provide a result, and the aneuploidy risk is increased in such cases.

Basic information is provided regarding the conditions for which screening is performed and the limitations of the

screening test.

Information may include prevalence of a genetic condition, associated abnormalities, and prognosis.

Diagnosis may aid earlier identification of associated abnormalities.

In the case of trisomy 18 or 13, diagnosis may affect pregnancy management if complications arise such as growth

restriction or nonreassuring fetal heart rate.

All screening tests are less effective in multifetal gestations.

Phenotypic expression of sex chromosome aneuploidies varies widely.

Apriori risk for fetal aneuploidy may affect whether a woman elects a screening test or diagnostic test.

Age-related risk information may be found in reference tables.

If a patient has had a prior fetus with autosomal trisomy, robertsonian translocation, or other chromosomal abnormality,

additional evaluation and counseling are recommended.

If a major fetal abnormality has been identified with ultrasound, a diagnostic test is preferred, and aneuploidy screening

is not recommended.

American College of Obstetricians and Gynecologists, 2020e; Dashe, 2016.Prenatal Diagnosis 335

CHAPTER 17

alse-positive rate, those without aneuploidy who nonetheless

screen positive. Because aneuploidies are individually uncommon, the screen-positive rate, which is the overall proportion o

tests with a positive result, is usually similar to the alse-positive

rate. Aneuploidy screening tests generally have screen-positive

rates no higher than 5 percent.

Sensitivity and specicity do not convey any inormation

regarding individual risk. Te positive predictive value (PPV)

is the proportion with a positive screening result who have

an aneuploid etus. PPV may be viewed as the test result. It

is usually expressed as a 1:X ratio or as a percentage. Because

PPV is directly aected by disease prevalence, it is higher

in women aged 35 years and older than in younger women

(Table 17-3). Negative predictive value is the proportion o

those with a negative screening test result who have unaected

(euploid) etuses. Because the prevalence o aneuploidy is low,

the negative predictive value o all aneuploidy screening tests

generally exceeds 99 percent (Gil, 2017; Norton, 2015).

■ Cellfree DNA Screening

Tis screening test identies DNA ragments derived primarily

rom apoptotic trophoblasts, which are placental cells undergoing programmed cell death. Tus, the term cell-ree etal DNA is

a misnomer. Dierences between placental and etal DNA may

cause alse-positive and alse-negative results. Tere are three types

o assays: whole-genome sequencing, chromosome selective or

targeted sequencing, and single nucleotide polymorphism analysis. CDNA screening can be perormed at any time ater 9 to

10 weeks’ gestation. When it was rst introduced, cDNA screening was oered primarily to pregnancies with increased aneuploidy risk, but it has since become widely available (American

College o Obstetricians and Gynecologists, 2020e).

CDNA screening is most commonly used to screen or

autosomal trisomies—trisomy 21, trisomy 18, and trisomy

13. It may also be used to screen or 45,X (urner syndrome),

47,XXX, 47,XXY, and 47,XYY. A recent metaanalysis concluded that the number o reported sex aneuploidy cases is too

small or accurate assessment o screening perormance (Gil,

2017). Te prognosis or these sex chromosomal aneuploidies

diers considerably rom that o the autosomal trisomies (Chap.

16, p. 313). I screening or sex chromosomal abnormalities is

elected, counseling should include this inormation.

CDNA has the highest sensitivity and specicity o any aneuploidy screening test. In a metaanalysis o 35 studies o largely

high-risk pregnancies, the pooled sensitivity to detect trisomy 21

was 99.7 percent, and or trisomies 18 and 13, 98 percent and

99 percent, respectively (Gil, 2017). For each o these autosomal

trisomies, the specicity is 99.9 percent, and the combined alsepositive rate is less than 0.2 percent. I the screening platorm

includes other genetic conditions, each additional condition

increases the overall alse-positive rate or the test.

Te PPV o a positive cDNA result is provided in the report

or may be estimated rom an online calculator or reerence table

(see able 17-3). Because the risk estimate or each aneuploidy

depends on maternal age, such inormation may be included as

part o pretest counseling. Tis can help to avoid the misconception that a positive screen indicates aneuploidy—it does not.

Because cDNA screening has a PPV ar higher than analyte

screening, it may be oered as a secondary screening test or

women who wish to avoid amniocentesis. Such an approach

has important caveats. I cDNA is used as a secondary screening test and yields a negative result, the residual risk or a chromosomal abnormality may be as high as 2 percent (Norton,

2014). And, i the cDNA result is positive, the delay in denitive diagnosis may aect management options.

In addition to the aorementioned aneuploidies, cDNA

screening is available or other genetic conditions. Tese

include aneuploidies such as trisomies 16 and 22, specic

microdeletion syndromes, and large copy number changes

throughout the genome. Currently, the American College

o Obstetricians and Gynecologists (2020e) does not recommend cDNA screening or these other conditions. Sensitivity and specicity o cDNA screening have not been

established or them, and screening accuracy has not been

validated clinically.

Limitations of cfDNA Screening

In 2 to 4 percent o pregnancies screened with cDNA, no result

is obtained. Tese cases are termed “no-call,” or indeterminate.

TABLE 17-3. Positive Predictive Value of Cell-Free DNA Screening for Autosomal

Trisomies and Sex Chromosome Abnormalities, According to Maternal

Age at Term

Maternal Age Trisomy 21 Trisomy 18 Trisomy 13 45,X 47,XXX 47,XXY 47,XYY

20 48% 14% 6% 41% 27% 29% 25%

25 51% 15% 7% 41% 27% 29% 25%

30 61% 21% 10% 41% 27% 29% 25%

35 79% 39% 21% 41% 28% 30% 25%

40 93% 69% 50% 41% 45% 52% 25%

45 98% 90% NA 41% 73% 77% NA

NA = not available.

Positive predictive values were obtained using the Cell Free DNA Screening Predictive

Value Calculator from the Perinatal Quality Foundation, 2020.

Calculations based on prevalence at 16 weeks’ gestation using sensitivities and specificities

from Gil, 2015.336 The Fetal Patient

Section 6

With a no-call result, the etal aneuploidy risk is as high as

4 percent (Norton, 2015). As shown in Table 17-4, this result

is similar to the average risk conerred by a positive analyte

screening test result.

Low etal raction is a common reason or no-call results.

Te etal raction is the proportion o cDNA in the maternal

circulation that is placental rather than maternal in origin.

Te etal raction approximates 10 percent in the late rst

trimester, increasing slightly thereater (Ashoor, 2013; Scott,

2018). Low etal raction is usually dened as <4 percent

o the total, and it is more prevalent beore 10 weeks’ gestation and in the setting o maternal obesity (Ashoor, 2013;

Juul, 2020; Scott, 2018). Te no-call rate may exceed 10 to

20 percent in women whose body mass index is 40 kg/m2 or

greater (Juul, 2020). Low etal raction is also more common

in the setting o aneuploidy, particularly trisomies 18 and 13

(Norton, 2015; Pergament, 2014). Te American College

o Medical Genetics and Genomics recommends that the

etal raction be included in cDNA reports because it assists

with interpretation o results (Gregg, 2016). Pretest counseling should include the possibility o a no-call result and its

clinical signicance (see able 17-2).

I the cDNA screening test is positive or yields a no-call

result, additional genetic counseling is indicated, and amniocentesis should be oered. I cDNA screening is repeated,

the risk or repeat screen ailure approximates 40 percent

(Dar, 2014; Quezada, 2015; Rolnik, 2018). Detailed ultrasound examination may be oered, but it is not a substitute

or amniocentesis, and the residual aneuploidy risk ollowing

normal detailed ultrasound examination is unknown.

It is important to remember that cDNA results may not

reect the etal DNA complement. Rather, a positive result

may be caused by conned placental mosaicism or early demise

o an aneuploid co-twin. Alternately, it may indicate maternal

aneuploidy, mosaicism, or even occult maternal malignancy

(Bianchi, 2015; Curnow, 2015; Grati, 2014; Wang, 2014). I

ultrasound identies an empty second gestational sac, cDNA

screening is not recommended. Te patient should be inormed

o such limitations prior to screening.

■ Analytebased Aneuploidy Screening

Te three categories o multiple marker-serum analyte screening are rst-trimester screens, second-trimester screens, and

combined rst- and second-trimester screens. Te serum concentration o each analyte is converted to a multiple o the

median (MoM) o the unaected population by adjusting or

maternal age, maternal weight, and gestational age. Te serum

AFP concentration is urther adjusted or maternal race and

or presence o diabetes, which aect calculation o neural-tube

deect risk (Greene, 1988; Huttly, 2004).

Te analyte-based screening result is based on a composite

likelihood ratio, and the maternal age-related risk is multiplied

by this ratio. Adjustment normalizes the distribution o analyte

levels and permits comparison o results rom dierent laboratories and populations. Each woman is provided with a specic

risk or trisomy 21 and or trisomy 18—or in the rst trimester,

or trisomy 18 or 13 in some cases. Each screening test has

a predetermined value at or above which it is deemed “positive” or abnormal. For second-trimester tests, this threshold

has traditionally been set at the risk or etal Down syndrome

at a maternal age o 35 years—approximately 1 in 270 in the

second trimester. Te trisomy 21 risk may be urther modied

based on selected ultrasound markers (p. 340).

TABLE 17-4. Characteristics of Screening Tests for Trisomy 21 in Singletons

Screening Test

Detection

Rate

FalsePositive

Rate

Positive Predictive

Valuea

Quadruple screen:

AFP, hCG, estriol, inhibin 80–82% 5% 3%

Firsttrimester screen:

NT, hCG, PAPP-A

First-trimester NT alone

80–84%

64–70%

5%

5%

3–4%

Integrated screening 94–96% 5% 5%

Sequential screening:

Stepwise

Contingent

92–97%

91–95%

5.1%

4.5%

5%

5%

Cellfree DNA screening:

Positive result

Low fetal fraction or no result

99%

0.1%

2–4%

Table 17-3

3–4%

aThe positive predictive value represents the overall population studied and cannot

be applied to any individual patient.

AFP = alpha-fetoprotein; hCG = human chorionic gonadotropin; NT = nuchal

translucency; PAPP-A = pregnancy-associated plasma protein A.

Data from Aldred, 2017; Baer, 2015; Dashe, 2016; Gil, 2015; Malone, 2005b; Norton,

2015; Pergament, 2014; Quezada, 2015.Prenatal Diagnosis 337

CHAPTER 17

A benet o analyte screening is that pregnancies with

abnormal results may have genetic abnormalities other than

those or which screening was perormed. For example, in a

review o more than 2500 pregnancies with genetic abnormalities rom the Caliornia Prenatal Screening Program, combined

rst- and second-trimester screening results were abnormal in

93 percent with trisomies 21 and 18 (Norton, 2016). However,

results were also abnormal in 80 percent with trisomy 13, in

80 percent with 45,X, in nearly 60 percent with other sex

chromosomal abnormalities, and in more than 50 percent with

other genetic abnormalities. Alamillo and associates (2013)

similarly identied an abnormal karyotype other than trisomies

21, 18, or 13 in 2 percent o pregnancies with abnormal rsttrimester screening results, accounting or 30 percent o abnormal karyotypes in the series.

Women younger than 35 are at lower risk or the specic

autosomal trisomies or which cDNA screening is typically

perormed. Tus, i the goal is to select a screening test that

will identiy the highest proportion o etuses with any chromosomal abnormality, the yield may be comparable or even

slightly higher with integrated or sequential screening than

with current cDNA screening (Baer, 2015; Norton, 2014).

First-trimester Aneuploidy Screening

Tis test includes human chorionic gonadotropin (hCG),

pregnancy-associated plasma protein A (PAPP-A), and ultrasound measurement o the nuchal translucency (N). It is per-

ormed between 11 and 14 weeks’ gestation. Pregnancies with

etal trisomy 21 are characterized by higher levels o hCG and

lower PAPP-A. With trisomy 18 and trisomy 13, levels o both

analytes are lower (Cuckle, 2000; Malone, 2005b).

Nuchal Translucency. Tis is the maximum thickness o the

subcutaneous translucent area between the skin and sot tissue

overlying the etal spine at the back o the neck (Fig. 17-2). It is

measured in the sagittal plane and is valid when the crown-rump

length (CRL) measurement is between 38 to 45 mm and 84 mm,

with the lower limit varying according to the laboratory. Measurement criteria are listed in able 14–4 (p. 250). Te N

should be imaged and measured with a high degree o precision

or aneuploidy detection to be accurate. Tis has led to standardized training, certication, and ongoing quality review programs.

An increased N is not a etal abnormality but rather a

marker that coners increased risk. It is helpul to dierentiate rom cystic hygroma, which appears sonographically as

a septated hypoechoic space behind the neck that extends

down the back (Fig. 15-30, p. 286). Te etal aneuploidy risk

is ve times greater with rst-trimester cystic hygroma than

with increased N (Malone, 2005a). An increased N measurement is associated with other genetic syndromes and various birth deects, especially etal cardiac abnormalities (Clur

2008; Simpson, 2007). I the N measurement is at least 3

mm or reaches the 99th percentile or CRL, a detailed ultrasound examination should be oered. Similarly, etal echocardiography may be considered i the N measurement is

at least 3 mm and is recommended i the N measurement

is 3.5 mm or greater (American College o Obstetricians and

Gynecologists, 2020e; American Institute o Ultrasound in

Medicine, 2019).

As an isolated marker, N detects only 70 percent o trisomy 21 etuses, and or this reason it is not used to screen singleton pregnancies (Alldred, 2017; Malone, 2005b). I cDNA

screening has already been perormed, the N measurement

does not improve etal aneuploidy detection and is not recommended or this purpose (American College o Obstetricians

and Gynecologists, 2020e; Rei, 2016). N evaluation may be

helpul in multietal gestations because serum screening is less

accurate, may not be available, and cannot provide inormation

specic to each etus. Te N distribution is similar in twins

and singletons (Cleary-Goldman, 2005).

Efficacy of First-trimester Screening. Beore rst-trimester

screening became widely adopted, our large prospective trials

were conducted, together including more than 100,000 pregnancies (Reddy, 2006). When the screen-positive rate was set

at 5 percent, trisomy 21 detection was 84 percent. A recent

metaanalysis also reported detection o 87 percent o trisomy

21 etuses (Alldred, 2017). First-trimester screening similarly

detects approximately 80 percent o etuses with trisomy 18

and 50 percent with trisomy 13 (Norton, 2015).

Maternal age aects the perormance o rst-trimester

aneuploidy screening. In women younger than age 35 at delivery, etal trisomy 21 detection ranges rom 67 to 75 percent

(Malone, 2005b; Wapner, 2003). Among women older than

35 at delivery, detection is as high as 90 to 95 percent, albeit at

a higher alse-positive rate o 15 to 22 percent.

In twin pregnancies, serum ree β-hCG and PAPP-A levels

are approximately double the singleton values (Vink, 2012).

Even with specic curves, a normal dichorionic co-twin will

tend to normalize screening results. Tus the aneuploidy detection rate is at least 15-percent lower (Bush, 2005).

Analyte abnormalities are termed unexplained i prenatal

diagnostic testing results are normal. Tere is a signicant

FIGURE 17-2 Sagittal image of a normal, 12-week fetus shows

correct caliper placement (+) for nuchal translucency measurement. The fetal nasal bone and overlying skin are indicated. The

nasal tip and the 3rd and 4th ventricles (asterisk), which are other

landmarks that should be visible in the nasal bone image, also are

shown. (Reproduced with permission from Dr. Michael Zaretsky.)338 The Fetal Patient

Section 6

association between serum PAPP-A levels below the 5th percentile and preterm birth, growth restriction, preeclampsia, and

etal demise (Cignini, 2016; Dugo, 2004; Jellie-Pawlowski,

2015). Similarly, low levels o ree β-hCG have been associated

with etal demise (Goetzl, 2004). Te sensitivity and PPVs o

these markers are too low to make them clinically useul as

screening tests (American College o Obstetricians and Gynecologists, 2020c).

Second-trimester Aneuploidy Screening

Te quadruple marker or “quad” screening test is perormed

rom 15 through 20 or 21 weeks’ gestation depending on the

laboratory. Pregnancies with etal trisomy 21 are characterized

by lower MSAFP, higher hCG, lower unconjugated estriol,

and higher dimeric inhibin levels. In large prospective trials, trisomy 21 detection was 81 to 83 percent at a 5-percent

screen-positive rate (Malone, 2005b; Wald, 1996, 2003). In

cases o trisomy 18, levels o MSAFP, hCG, and unconjugated

estriol are all decreased, and inhibin is not part o the calculation. risomy 18 detection is similar to that or trisomy 21, but

the alse-positive rate is just 0.5 percent (Benn, 1999). As with

rst-trimester screening, aneuploidy detection rates are lower

in younger women and higher in women older than 35 years

at delivery. I second-trimester serum screening is used in twin

pregnancies, aneuploidy detection rates are signicantly lower

(Vink, 2012).

Quadruple-marker screening oers no benet over rsttrimester screening rom the standpoint o trisomy 21 or trisomy

18 detection. As a stand-alone test, it is generally used i women

do not begin care until the second trimester or i rst-trimester

screening and cDNA screening are not available. In 2019, women

who initiated prenatal care beyond the rst trimester made up

more than 20 percent o pregnancies in the United States (Martin, 2020). As subsequently discussed, combining rst- and second-trimester screening yields greater aneuploidy detection.

Maternal Serum AFP Elevation: Neural-tube Defect Screening. AFP is the major protein in etal serum, analogous to

albumin in a child or adult. Deects in etal integument, such

as neural-tube and ventral-wall deects, permit AFP to leak

into the amnionic uid and result in dramatically increased

maternal serum levels. Accurate gestational age assessment

is imperative because the AFP value rises by approximately

15 percent per week during the screening window (Knight,

1992).

Using an MSAFP level o 2.5 MoM as the upper limit o

normal, the neural-tube deect detection rate approximates

95 percent or anencephaly and 80 percent or spina bida,

with a screen-positive rate o 1 to 3 percent (American College

o Obstetricians and Gynecologists, 2019c; Palomaki, 2019).

Te overall PPV approximates just 2 percent. Higher screening threshold values are used in twin pregnancies, which have

twice the AFP level o singletons (Cuckle, 1990). Importantly,

because nearly all cases o anencephaly and most with spina

bida are detected during the standard second-trimester ultrasound examination, MSAFP screening is considered optional

(American College o Obstetricians and Gynecologists, 2019c;

Dashe, 2006; Norem, 2005).

I the serum AFP is elevated, a detailed ultrasound examination is recommended, and characteristic sonographic ndings

are diagnostic (Chap. 15, p. 276). Other abnormalities and

conditions also may result in MSAFP elevation (Table 17-5).

Amniocentesis or measurement o amnionic uid AFP and acetylcholinesterase levels can help to diagnose whether a myelomeningocele is open or closed, which is relevant when considering

etal surgical repair (Chap. 19, p. 372).

Most pregnancies with MSAFP elevation have no etal

abnormality. Te patient should receive counseling about the

benets and limitations o detailed ultrasound examination or

the diagnosis o neural-tube deects and other potential etiologies. Tese include other etal abnormalities, placental abnormalities, and selected adverse pregnancy outcomes (able 17-5).

Te likelihood o any abnormality or adverse outcome increases

in proportion to the AFP level.

Tere are similarly signicant associations between secondtrimester elevation o either hCG or dimeric inhibin alpha

levels and adverse pregnancy outcomes. Te likelihood o

adverse outcome is greater i multiple analytes are elevated

(Dugo, 2005). Te sensitivity and positive predictive values

o these markers are considered too low to be useul or screening or management. No specic program o maternal or etal

surveillance has been ound to avorably aect pregnancy outcomes (Dugo, 2010).

Low Maternal Serum Estriol Level. A maternal serum estriol

level less than 0.25 MoM has been associated with two uncommon but important conditions. Smith-Lemli-Opitz syndrome is

TABLE 17-5. Conditions Associated with MSAFP Level

Elevation

Underestimated gestational age

Multifetal gestation

Fetal death

Neural-tube defect

Gastroschisis

Omphalocele

Cystic hygroma

Esophageal or intestinal obstruction

Liver necrosis

Renal anomalies—polycystic kidneys, renal agenesis,

congenital nephrosis, urinary tract obstruction

Cloacal exstrophy

Osteogenesis imperfecta

Sacrococcygeal teratoma

Congenital skin abnormality

Pilonidal cyst

Chorioangioma of placenta

Placenta intervillous thrombosis

Placental abruption

Oligohydramnios

Preeclampsia

Fetal-growth restriction

Maternal hepatoma or teratoma

MSAFP = maternal serum alpha-fetoprotein.Prenatal Diagnosis 339

CHAPTER 17

an autosomal recessive condition resulting rom mutations in

the 7-dehydrocholesterol reductase gene. It is characterized by

abnormalities o the central nervous system, heart, kidney, and

extremities; with ambiguous genitalia; and with etal-growth

restriction. Detailed sonography should be oered i an unconjugated estriol level is <0.25 MoM (American Institute o

Ultrasound in Medicine, 2019; Dugo, 2010). An elevated

amnionic uid 7-dehydrocholesterol level can conrm the

diagnosis.

Te second condition is steroid sulatase defciency, also known

as X-linked ichthyosis. It is typically an isolated condition, but it

may also occur in the setting o a contiguous gene deletion syndrome (Chap. 16, p. 315). In such cases, it may be associated

with Kallmann syndrome, chondrodysplasia punctata, and/or

mental retardation (Langlois, 2009). I the estriol level is <0.25

MoM and the etus appears to be male, chromosomal microarray analysis or uorescence in situ hybridization to assess the

steroid sulatase locus on the X-chromosome may be considered.

Integrated and Sequential Screening

I rst-trimester screening is combined with second-trimester

screening, aneuploidy detection is signicantly improved.

Combined screening test options require that specimens are

obtained at the correct gestational age and sent to the same

laboratory. Te rst- and second-trimester components cannot be perormed independently, because the alse-positive rate

would be higher and providing accurate risk assessment would

be problematic.

Tree types o screening strategies are available:

1. Integrated screening combines results o rst- and secondtrimester tests. Tis includes a combined measurement o

etal N and serum analyte levels at 11 to 14 weeks’ gestation

plus quadruple markers at approximately 15 to 21 weeks. risomy 21 detection is 94 to 96 percent (see able 17-4).

2. Sequential screening involves inorming the patient o the

results ater rst-trimester screening, with plan to oer prenatal

diagnostic testing i the calculated risk value lies above a speci-

ed threshold. Tere are two testing strategies in this category:

• With stepwise sequential screening, women whose rsttrimester risk is at or below the threshold receive second-trimester screening. Using data rom the First- and

Second-rimester Evaluation o Risk trial, when the

rst-trimester threshold is set at approximately 1:30, and

the overall threshold is set at 1:270, stepwise sequential

screening resulted in a 92-percent trisomy 21 detection

rate (Cuckle, 2008). Te alse-positive rate remained 5

percent (see able 17-4).

• With contingent sequential screening, women are divided

into high-, moderate-, and low-risk groups. Tose at highest risk or trisomy 21—or example, risk >1:30, are counseled and oered diagnostic testing. Women at moderate

risk, between 1:30 and 1:1500, undergo second-trimester

screening, whereas those at lowest risk o <1:1500 receive

negative screening test results and have no urther testing

(Cuckle, 2008). Using this strategy, more than 75 percent

o those screened are provided with reassuring results almost

immediately. Assuming that women accept diagnostic

testing when inormed that the risk is elevated, detection

approaches 91 percent.

■ Ultrasound Screening

Ultrasound can improve aneuploidy screening by providing

accurate gestational age assessment, by detecting multietal gestations, and by identiying major structural abnormalities and

minor aneuploidy markers. With rare exception, the aneuploidy

risk associated with any major abnormality is high enough to

recommend prenatal diagnosis with chromosomal microarray

analysis. We do not recommend aneuploidy screening ater

TABLE 17-6. Aneuploidy Risk Associated with Selected Major Fetal Anomalies

Abnormality Birth Prevalence

Aneuploidy

Risk (%) Common Aneuploidiesa

Cystic hygroma 1/5000 50–70 45,X; 21; 18; 13; triploidy

Nonimmune hydrops 1/1500–4000 10–20 21; 18; 13; 45,X, triploidy

Ventriculomegaly 1/1000–2000 5–25 13; 18; 21; triploidy

Holoprosencephaly 1/10,000–15,000 30–40 13; 18; 22; triploidy

Dandy-Walker malformation 1/12,000 40 18; 13; 21; triploidy

Cleft lip/palate 1/1000 5–15 18; 13

Cardiac defects 5–8/1000 10–30 21; 18; 13; 45,X; 22q11.2 microdeletion

Diaphragmatic hernia 1/3000–5000 5–15 18; 13; 21

Esophageal atresia 1/4000 10 18; 21

Duodenal atresia 1/10,000 30 21

Gastroschisis 1/2000 No increase

Omphalocele 1/3000–5000 30–50 18; 13; 21; triploidy

Clubfoot 1/1000 5–30 18; 13

aNumbers indicate autosomal trisomies except where indicated. For example, 45,X indicates Turner syndrome.

Data from Best, 2012; Canfield, 2006; Colvin, 2005; Cragan, 2009; Dolk, 2010; Ecker, 2000; Gallot, 2007;

Long, 2006; Orioli, 2010; Pavone, 2018; Pedersen, 2012; Sharma, 2011; Solomon, 2010; Walker, 2001.340 The Fetal Patient

Section 6

detection o a major abnormality, because o the many genetic

conditions are not detectable with screening tests. Counseling

should include the association o the abnormality with aneuploidy (Table 17-6). A caveat is that etal abnormalities are

requently not isolated, and associated-but-undetectable abnormalities may greatly aect prognosis.

Importantly, etuses with trisomy 21 and other genetic syndromes may not have obvious ultrasound abnormalities. Just

one third o trisomy 21 etuses have a major abnormality identied with second-trimester ultrasound evaluations (Bromley,

2002; Hussamy, 2019; Vintileos, 1995). I major anomalies

and minor aneuploidy markers are considered, 50 to 75 percent o pregnancies aected by Down syndrome are ound

to have a sonographic abnormality. Tis is not unexpected,

because nearly 40 percent o inants with trisomy 21 do not

have major organ system abnormalities identied in the newborn period (Hussamy, 2019). In contrast, the vast majority

o etuses with trisomies 18 and 13 and with triploidy do have

ultrasound abnormalities detectable in the second trimester.

Aneuploidy Markers

For more than three decades, investigators have recognized

that ultrasound detection o aneuploidy, particularly trisomy

21, may be improved by minor markers that are collectively

reerred to as “sot signs.” Minor markers are normal variants

rather than etal abnormalities, and in the absence o aneuploidy or an associated abnormality, they do not signicantly

aect prognosis. One or more markers are present in at least

10 percent o pregnancies (Bromley, 2002; Nyberg, 2003).

Examples are listed in Table 17-7 and depicted in Figure 17-3.

TABLE 17-7. First- and Second-Trimester

Ultrasound Markers Associated with Fetal

Trisomy 21

First trimester

Ductus venosus with A-wave absence or flow reversal

Nasal bone absence or hypoplasia

Nuchal translucency enlargement

Tricuspid valve regurgitation

Second trimester

Brachycephaly or shortened frontal lobes

Clinodactyly (hypoplastic middle phalanx of fifth digit)

Echogenic bowel

Flat-appearing facies

Echogenic intracardiac focus

Nasal bone absence or hypoplasia

Nuchal fold thickening

Renal pelvis dilatation

“Sandal gap” between first and second toes

Short ear length

Single transverse palmar crease

Single umbilical artery

Short femur

Short humerus

Subclavian artery aberrant (right side)

Widened iliac angle

Markers listed alphabetically.

B C

D E F

A

FIGURE 17-3 Minor sonographic markers that are associated with increased risk for fetal Down syndrome. A. Nuchal skinfold thickening

(bracket). B. Echogenic intracardiac focus (arrow). C. Mild renal pelvis dilatation (pyelectasis) (arrows). D. Echogenic bowel (arrow). E. Clinodactyly—hypoplasia of the 5th finger middle phalanx creates an inward curvature (arrow). F. “Sandal-gap” (arrow).Prenatal Diagnosis 341

CHAPTER 17

A minor marker is an indication or a detailed survey o etal

anatomy to determine i it is isolated (American Institute o

Ultrasound in Medicine, 2019).

I an isolated marker is identied and aneuploidy screening

has not yet been perormed, it should be oered. A benet o

cDNA screening is that isolated aneuploidy markers no longer

actor into the aneuploidy risk, because the negative predictive value o a normal cDNA test is so high. Conversely, i a

cDNA result is abnormal, the absence o minor markers is not

considered reassuring.

First-trimester Markers. Detection o rst-trimester ndings

associated with aneuploidy requires specialized imaging. N

evaluation is discussed earlier (p. 337). Other rst-trimester

markers are not routinely used in the United States but may

be available at specialized centers. Te Perinatal Quality Foundation’s Nuchal ranslucency Quality Review Program oers

an education program in rst-trimester nasal bone assessment.

Te Fetal Medicine Foundation also provides online instruction and certication in rst-trimester assessment o nasal bone,

ductus venosus ow, and tricuspid ow.

Tere are additional benets o rst-trimester ultrasound

evaluation in women who elect aneuploidy screening, including accurate assessment o gestational age and early detection o multietal gestation or etal demise. As discussed in

Chapter 14 (p. 249), standard rst-trimester sonography may

identiy selected major anomalies associated with aneuploidy,

such as cystic hygroma.

Second-trimester Markers. I analyte-based aneuploidy screening has been perormed, minor markers may be used to modiy

the risk using likelihood ratios (Table 17-8). Te risk increases

with the number o markers identied. Risk modication

should ollow a protocol that species criteria or each marker

(Reddy, 2014).

Te nuchal skinold is imaged in the transcerebellar view o

the head and is measured rom the outer edge o the skull to

the outer border o the skin (see Fig. 17-3A). Te skinold is

considered thickened i it measures ≥6 mm at 15 to 20 weeks

(Benacerra, 1985). Tis nding is seen in approximately 1 per

200 pregnancies and coners more than tenold risk or trisomy

21 (Bromley, 2002; Nyberg, 2001; Smith-Bindman, 2001).

An echogenic intracardiac ocus is a ocal papillary muscle

calcication that is neither a structural nor unctional cardiac

abnormality (see Fig. 17-3B). It is present in 4 to 7 percent

o normal etuses. Te etal trisomy 21 risk is approximately

doubled. Te nding is more commonly identied in Asian

individuals (Shipp, 2000). Fetuses with trisomy 13 may have

bilateral echogenic oci (Nyberg, 2001).

Mild renal pelvis dilatation is usually transient or physiologic

(Chap. 15, p. 298). Te renal pelves are measured anteriorto-posterior in a transverse image o the kidneys, with calipers

placed at the inner borders o the uid collection (see Fig.

17-3C). A measurement ≥4 mm is ound in about 2 percent o etuses and approximately doubles the risk or trisomy

21. Te degree o pelvic dilatation beyond 4 mm correlates

with the likelihood o an underlying renal abnormality, and

third-trimester sonography is recommended at approximately

32 weeks.

Echogenic etal bowel is dened as bowel that is sonographically as bright as etal bone (see Fig. 17-3D). It is identied in

approximately 0.5 percent o pregnancies and oten represents

a small amount o swallowed blood, not inrequently in the setting o MSAFP elevation. Te etal trisomy 21 risk is increased

approximately sixold. Echogenic bowel is also associated with

etal cytomegalovirus inection and cystic brosis—representing inspissated meconium in the latter. Follow-up ultrasound

evaluation is suggested at 32 weeks’ gestation to evaluate etal

growth (American College o Obstetricians and Gynecologists,

2020e).

For the purpose o Down syndrome screening, the etal

emur and humerus are considered short i below the 2.5th percentile or gestational age (American College o Obstetricians

and Gynecologists, 2020e). Tird-trimester ultrasound evaluation is suggested to assess etal growth. Fetuses with trisomy

21 and short emur are not more likely to be growth restricted,

however (Herrera, 2019).

Te etal nasal bone is absent in 30 percent o second-trimester etuses with trisomy 21 and hypoplastic in more than

50 percent (Moreno-Cid, 2014). Absent nasal bone occurs in

approximately 1 in 200 euploid etuses (American College o

Obstetricians and Gynecologists, 2020e; Viora, 2005). Te

nding represents delayed ossication rather than true absence.

Te nasal bone is measured between 15 and 22 weeks’ gestation. Hypoplasia may be dened as shorter than 2.5 mm, as >2

standard deviations below the mean, or based on a ratio to the

biparietal diameter (Cicero, 2003). Te etal prole and nasal

bone are components o the detailed etal anatomic survey,

but they are not assessed as part o the standard examination

and thus not used or routine screening (American Institute o

Ultrasound in Medicine, 2019).

One or more choroid plexus cysts are seen in 1 to 2 percent o

euploid pregnancies (Fig. 16-5, p. 312). Te cyst is a benign,

TABLE 17-8. Likelihood Ratios and False-Positive Rates

for Isolated Second-Trimester Markers

Used in Trisomy 21 Screening Protocols

Sonographic Marker

Likelihood

Ratio

Prevalence in

Unaffected

Fetuses (%)

Nuchal skinfold thickening 11–17 0.5

Renal pelvis dilation 1.5–1.9 2.0–2.2

Echogenic intracardiac

focus

1.4–2.8 3.8–3.9a

Echogenic bowel 6.1–6.7 0.5–0.7

Short femur 1.2–2.7 3.7–3.9

Short humerus 5.1–7.5 0.4

Any one marker 1.9–2.0 10.0–11.3

Two markers 6.2–9.7 1.6–2.0

Three or more 80–115 0.1–0.3

aHigher in Asian individuals.

Data from Bromley, 2002; Nyberg, 2001; Smith-Bindman,

2001.342 The Fetal Patient

Section 6

2. Te condition should have a well-dened phenotype, lead to a

detrimental eect on quality o lie, cause cognitive or physical

impairment, or require surgical or medical intervention.

3. Conditions with adult onset are not recommended or

inclusion.

Tere is a long-held principle that genetic carrier screening

or a particular condition not be repeated in a subsequent pregnancy. Te number o mutations or which testing is available

has greatly increased in recent years, but the additional yield

o rescreening is usually low. Consultation with a provider

with genetic expertise should be considered beore screening

is repeated (American College o Obstetricians and Gynecologists, 2020a).

■ Cystic Fibrosis

Tis disorder is caused by a mutation in the cystic fbrosis conductance transmembrane regulator (CFR) gene, which is located

on the long arm o chromosome 7 and encodes a chloridechannel protein. Although the most common CFR mutation

associated with classic cystic brosis (CF) is the ΔF508 mutation, more than 2100 mutations have been identied (Cystic

Fibrosis Mutation Database, 2020). CF may develop rom

either homozygosity or compound heterozygosity or mutations in

the CFR gene. One mutation must be present in each copy o

the gene, but they need not be the same mutation. Tere is a

wide range o clinical disease severity. Median survival length is

42 years, but approximately 15 percent have milder disease and

can survive or decades longer. Care o the pregnant woman

with CF is discussed in Chapter 54 (p. 968).

For more than two decades, the American College o Obstetricians and Gynecologists (2020a) has recommended that all

women who are pregnant or considering pregnancy be oered

CF carrier screening. Such screening should include, at minimum, a panel o 23 panethnic gene mutations, selected because

they are present in at least 0.1 percent o patients with classic CF. Te CF-mutation carrier requency approximates 1:25

in Ashkenazi Jews and those o non-Hispanic white ethnicity,

1:60 in Arican Americans and those o Hispanic white ethnicity, and 1:95 Asian Americans. Using the 23-mutation panel,

the residual carrier risk ater a negative test result was roughly

1:380 or Ashkenazi Jews and 1:170 to 1:200 or other ethnicities (American College o Obstetricians and Gynecologists,

2020a).

More recently however, expanded panels that use gene

sequencing have been reported to improve detection o at-risk

couples by 30 percent compared with the 23-mutation panel

(Beauchamp, 2019). Te American College o Medical Genetics now recommends either a targeted approach to carrier

screening, in which the standard mutation panel may be used,

or a more comprehensive approach in which specic variant

classications may be reported (Deignan, 2020).

CF is included in all newborn screening panels but is not a

substitute or carrier screening because it only identies aected

individuals. I the patient carries a mutation, her partner should

be oered screening. I both parents are carriers, genetic counseling is indicated, and prenatal diagnostic testing should be

oered.

normal variant o no clinical consequence. I associated with

structural abnormalities however, the trisomy 18 risk is

increased (Reddy, 2014).

CARRIER SCREENING FOR GENETIC

DISORDERS

Te goal o genetic carrier screening is to provide individuals with meaningul inormation to guide pregnancy planning

according to their personal values (American College o Obstetricians and Gynecologists, 2020b). Tere are three approaches,

each o which is an acceptable strategy: ethnicity-based carrier

screening, panethnic screening (perormed regardless o ethnicity), and expanded carrier screening—which is a type o panethnic screening perormed or a large number o conditions. All

carrier screening is optional and should be an inormed choice.

Carrier screening is ideally perormed prior to conception

so that both partners can have the opportunity to consider the

results and pursue options such as preimplantation genetic testing (p. 348) or donor gametes. I the screen detects a genetic

condition, partner screening should be oered. No screening

test will identiy all carriers o a condition. Genetic counseling

should include the residual risk or the condition i the partner’s screen is negative, as well as prenatal diagnosis options

(American College o Obstetricians and Gynecologists, 2020b).

Prenatal diagnosis is increasingly available i disease-causing

mutation or mutations are known. Te Genetic esting Registry o the National Institutes o Health contains detailed inormation regarding more than 18,000 genetic conditions and

78,000 genetic tests (www.ncbi.nlm.nih.gov/gtr/). In the setting o consanguinity, additional genetic counseling is indicated.

Ethnicity-based carrier screening is oered or selected autosomal recessive conditions that are ound in greater requency in

specic racial or ethnic groups. Te ounder eect occurs when

an otherwise rare gene is ound with greater requency in a

certain population and can be traced back to a single amily

member or small group o ancestors. It may develop when generations o individuals procreate only within their own groups

because o religious or ethnic prohibitions or geographical isolation. Because assignment o a single ethnicity is oten difcult, panethnic screening is increasingly preerred.

Expanded carrier screening has the advantage o screening or many conditions simultaneously, ranging rom a ew

dozen to more than 1000 (Chokshvilli, 2018). An unortunate

result is that much o the population—more than 50 percent

o those screened–may be identied to be carriers or at least 1

condition. Some conditions are so rare that inormation about

detection and residual risk are limited. Counseling regarding

phenotypic expression o dierent mutations and determination o whether variants have clinical signicance has become

increasingly complex. Tus, expanded carrier screening can

cause anxiety or amilies and may pose challenges to alreadystrained genetic counseling resources. Te American College

o Obstetricians and Gynecologists (2020b) has the ollowing

guidelines or conditions included in expanded panels:

1. Te carrier requency should be at least 1:100, which corresponds to a population prevalence o 1:40,000.Prenatal Diagnosis 343

CHAPTER 17

or β-thalassemia. Hemoglobin S is also more common among

individuals o Mediterranean, Middle Eastern, and Asian

Indian descent (Davies, 2000). I a couple is at risk to have

a child with a sickle hemoglobinopathy, genetic counseling

should be oered. Prenatal diagnosis can be perormed with

either chorionic villus sampling or amniocentesis.

Thalassemias

Tese are the most common single-gene disorders worldwide.

Talassemias are characterized as alpha (α) or beta (β) depending on whether α- or β-hemoglobin chains are aected. In general, α-thalassemia is more likely to be caused by deletions o

α-globin chains, whereas β-thalassemia more oten stems rom

mutations in β-globin chains.

In α-thalassemia, the number o α-globin genes that are

deleted may range rom one to all our. I two α-globin genes

are deleted, both may be deleted rom the same chromosome—

cis conguration (αα/--), or one may be deleted rom each

chromosome—trans conguration (α–/α–). Te cis conguration is more prevalent among Southeast Asians, whereas those

o Arican descent are more likely to inherit the trans conguration. Alpha-thalassemia trait results in mild anemia. However,

i the patient and her partner both carry cis deletions, ospring

are at risk or an absence o α-hemoglobin, called Hb Barts

disease. Tis can lead to hydrops and etal loss (Chap. 18, p.

360). Hemoglobin electrophoresis cannot detect α-thalassemia

or α-thalassemia trait. Molecular genetic testing should be

considered i there is microcytic anemia in the absence o iron

deciency and i the hemoglobin electrophoresis is normal,

particularly among individuals o Southeast Asian descent

(American College o Obstetricians and Gynecologists, 2019a).

In β-thalassemia, β-globin genes may cause reduced or

absent production o β-globin chains. I the mutation aects

one gene, it results in β-thalassemia minor. I both copies

are aected, the result is either β-thalassemia major—termed

Cooley anemia—or β-thalassemia intermedia. Hemoglobin

electrophoresis demonstrates elevated levels o hemoglobins

that do not contain β-chains, which are hemoglobins F and A2.

β-Talassemia minor should be considered i an individual o

one or more o the aorementioned ethnicities is ound to have

microcytic anemia in the absence o iron deciency. A hemoglobin A2 level exceeding 3.5 percent conrms the diagnosis.

■ Recessive Diseases in Ashkenazi

Jewish Individuals

Among Jewish individuals o Eastern and Central European

(Ashkenazi) descent, there are three severe diseases or which

the carrier requency and detection rate are high enough that

screening should be oered: ay-Sachs disease, Canavan disease,

and amilial dysautonomia (American College o Obstetricians

and Gynecologists 2020a). Te carrier rate approximates 1 in

30 or ay-Sachs disease, 1 in 40 or Canavan disease, and 1

in 32 or amilial dysautonomia. For each, the detection rate is

at least 98 percent. As or other genetic conditions, screening is

ideally perormed prior to conception or during early pregnancy.

Tere are several other autosomal recessive conditions

or which the College also recommends that screening be

■ Spinal Muscular Atrophy

Tis autosomal recessive disorder results in spinal cord motor

neuron degeneration that leads to skeletal muscle atrophy and

generalized weakness. Tere is currently no eective treatment.

Te prevalence o spinal muscular atrophy (SMA) is 1 in 6000 to

10,000 live births. ypes I, II, III, and IV are caused by mutations

in the survival motor neuron (SMN1) gene, which is located on

the long arm o chromosome 5 (5q13.2) and encodes the SMN

protein. ypes I and II account or 80 percent o cases and are

both lethal (American College o Obstetricians and Gynecologists, 2020a). SMA type I, known as Werdnig-Homann disease,

is the most severe. Disease onset is within the rst 6 months, and

aected children die o respiratory ailure by age 2 years. ype

II generally has onset beore age 2 years, and the age at death

can range rom 2 years to the third decade o lie. ype III also

presents beore age 2 years, with disease severity that is milder and

more variable. ype IV does not present until adulthood.

Te American College o Obstetricians and Gynecologists

(2020a) recommends that carrier screening or SMA be oered

to all women who are considering pregnancy or are currently

pregnant. Prior to screening, the potential spectrum o severity, carrier requency, and detection rate should be provided.

Posttest counseling should include the residual risk ater a

negative result, which diers according to ethnicity and also

according to the number o SMN1 copies detected. Te SMA

carrier requency approximates 1:35 in those o non-Hispanic

white ethnicity, 1:41 in Ashkenazi Jews, 1:53 in Asians, 1:66 in

Arican Americans, and 1:117 in those o Hispanic white ethnicity (Hendrickson, 2009). Carrier detection rates range rom

90 to 95 percent or each ethnicity except Arican Americans,

in whom it approximates 70 percent (MacDonald, 2014).

Although there is usually one copy o the SMN1 gene on

each chromosome, approximately 3 to 4 percent o carriers have no copies o this gene on one chromosome and two

copies on the other. Because such individuals have two copies in total, they are not detected i a quantitative polymerase

chain reaction test is used. Carrier screening is less sensitive in

Arican American individuals, because they are more likely to

have this genetic variation. Additionally, some individuals have

three copies o the gene and are at even lower risk. I the patient

or her partner has a amily history o SMA, or i carrier screening is positive, genetic counseling is indicated.

■ Hemoglobinopathies

Tese include the sickle hemoglobinopathies—sickle cell anemia and sickle cell hemoglobin C disease, thalassemias, and

sickle cell–β-thalassemia. Teir pathophysiology and management are discussed in Chapter 59 (p. 1053). Te risk is

increased in women who are o Arican or Arican-American,

Mediterranean, Middle Eastern, or Southeast Asian ethnicity. In such individuals, hemoglobin electrophoresis should be

oered prenatally or prior to conception (American College o

Obstetricians and Gynecologists, 2019a).

Sickle Hemoglobinopathies

Approximately 1 in 12 Arican Americans has sickle-cell trait,

1 in 40 carries hemoglobin C, and 1 in 40 carries the trait344 The Fetal Patient

Section 6

considered. Tese include Bloom syndrome, amilial hyperinsulinism, Fanconi anemia, Gaucher disease, glycogen storage

disease type I (von Gierke disease), Joubert syndrome, maple

syrup urine disease, mucolipidosis type IV, Niemann-Pick disease, and Usher syndrome. Gaucher disease diers rom the

other conditions listed because it has a wide range in phenotype

and because treatment is available in the orm o enzyme repletion and substrate reduction therapy.

Tay-Sachs Disease

Tis lysosomal-storage disease causes progressive neurodegeneration and death in early childhood. It is characterized by

deciency o the enzyme hexosaminidase A, which results in

accumulation o GM2 gangliosides in the central nervous system. Aected individuals have almost complete absence o the

enzyme, whereas carriers are asymptomatic but have less than

55-percent hexosaminidase A activity. An international aySachs carrier screening campaign was initiated in the 1970s

and met with unprecedented success in the Ashkenazi Jewish

population. Te incidence o ay-Sachs disease subsequently

declined more than 90 percent (Kaback, 1993). Other groups

at increased risk or ay-Sachs disease include those o FrenchCanadian and Cajun descent.

Carrier screening or ay-Sachs disease is perormed with

DNA-based mutation analysis or hexosaminidase activity testing. DNA-based mutation analysis is the preerred test in Ashkenazi Jewish individuals and other high-risk groups but has

a lower detection rate in the general population. Tereore,

hexosaminidase activity testing is recommended or screening

in individuals rom lower-risk ethnicities. I a woman is pregnant or taking oral contraceptives, a hexosaminidase activity

test should be perormed on leukocytes to avoid a high alsepositive rate (American College o Obstetricians and Gynecologists, 2020a). I both partners are ound to be carriers, prenatal

diagnostic testing should be oered. Hexosaminidase activity

may be measured rom chorionic villi or amnionic uid.

PRENATAL AND PREIMPLANTATION

DIAGNOSTIC TESTS

Diagnostic procedures used in prenatal diagnosis include

amniocentesis and chorionic villus sampling (CVS). Fetal

blood sampling is rarely perormed solely or genetic diagnosis

but may be used in conjunction with intrauterine transusion.

Preimplantation genetic tests or aneuploidies, structural chromosomal rearrangements, and monogenic disorders are available or couples undergoing in vitro ertilization (IVF).

In the setting o a etal structural abnormality, chromosomal

microarray analysis (CMA) is recommended. CMA is preerred

to cytogenetic analysis (karyotyping) because it can detect clinically signicant chromosomal abnormalities in approximately

6.5 percent o etuses with normal karyotype (Callaway, 2013;

de Wit, 2014, Wapner, 2012). And, in the absence o a etal

structural abnormality, CMA has detected additional chromosomal abnormalities (pathogenic copy number variants) in up

to 1 percent o those with normal karyotype. CMA is thereore

made available whenever a prenatal diagnostic procedure is per-

ormed (American College o Obstetricians and Gynecologists,

2018; Callaway, 2013). ypes o CMA platorms and their

benets and limitations are reviewed in Chapter 16 (p. 326).

Karyotyping identies aneuploidy and polyploidy. Additionally, it can identiy balanced chromosomal rearrangements that

are currently undetectable with CMA. For example, karyotyping

perormed when a couple has experienced recurrent pregnancy

loss may reveal that one parent carries a balanced robertsonian

translocation (Chap. 16, p. 315). An additional indication is

the etus with a structural abnormality that strongly suggests

a particular aneuploidy—such as endocardial cushion deect in

trisomy 21 or holoprosencephaly in trisomy 13.

I rapid identication o a specic chromosomal abnormality, such as trisomy 21, 18, or 13, is needed, uorescence in

situ hybridization (FISH) may be used in conjunction with

CMA or karyotyping. Decision-making based on FISH should

incorporate clinical inormation consistent with the suspected

diagnosis, such as a screening result or ultrasound nding

(American College o Obstetricians and Gynecologists, 2018).

Because o improvements in aneuploidy screening tests, there

has been a dramatic drop in the number o prenatal diagnostic

procedures. Larion and coworkers (2014) reported a 70-percent

decline in CVS and a nearly 50-percent drop in amniocentesis procedures since the introduction o cDNA screening in

2012. Tis urther amplied the decrease in amniocentesis procedures that occurred with the advent o rst-trimester screening (Warso, 2015). Unortunately, ewer than 40 percent o

women with a positive screening result elect prenatal diagnosis

(Dar, 2014). I prenatal diagnosis is elected, however, the

percent with an abnormal result has increased signicantly

(Awomolo, 2018).

■ Amniocentesis

Because o its established saety and efcacy, amniocentesis is

oered to all pregnant women (American College o Obstetricians and Gynecologists, 2020e). Amnionic uid is withdrawn

transabdominally under direct ultrasound guidance. Although

typically perormed between 15 and 20 weeks’ gestation,

amniocentesis may be done at any point later in gestation. ests

include those or genetic conditions, congenital inections, and

alloimmunization. Fetal lung maturity is no longer routinely

assessed, based on recommendations against using it to assist

with delivery timing (American College o Obstetricians and

Gynecologists, 2019b).

Technique

Amniocentesis is perormed with a 20- or 22-gauge needle

using aseptic technique (Fig. 17-4). A standard spinal needle is

9 cm long. A longer needle may be required or obese patients.

o aid in needle selection, we nd it helpul to measure the

distance rom skin to amnionic uid with ultrasound prior to

the procedure. Ater the patient empties her bladder, the skin is

prepared with an antiseptic such as povidone-iodine. Shellsh

allergy is not a contraindication to the povidone-iodine antiseptic (Westermann-Clark, 2015).

All aspects o the procedure are perormed under direct

ultrasound guidance. A pocket o amnionic uid close to the

midline is selected, avoiding etal parts and being cognizantPrenatal Diagnosis 345

CHAPTER 17

o uterine size and shape. Te needle is inserted perpendicular

to the skin, gently puncturing the chorioamnion rather than

pushing or “tenting” it away rom the uterine wall. Te amnion

usually uses with the underlying chorion by 16 weeks’ gestation, and the procedure is generally deerred until ater chorioamnion usion. Discomort rom the procedure is considered

minor and is related more to ocal myometrial contraction than

needle discomort at the skin. Local anesthetic has not been

ound to be benecial (Mujezinovic, 2011).

Te amnionic uid should be clear and colorless or pale

yellow. Blood-tinged uid is more requent with transplacental

passage o the needle and generally clears with continued aspiration. Needle passage through the placenta occurs in approximately 60 percent o cases with anterior placentation and is

not associated with pregnancy loss (Bombard, 1995, Marthin,

1997). Dark brown or greenish uid may represent a past episode o intraamnionic bleeding.

Te volume o uid generally needed or commonly per-

ormed analyses is shown in Table 17-9. Because the initial 1 to

2 mL o uid aspirate may be contaminated with maternal cells,

it is oten discarded. Approximately 20 to 30 mL o uid is then

collected or either etal CMA or karyotyping beore the needle

is withdrawn. Te uterine puncture site is observed or bleeding,

and etal cardiac motion is documented ollowing the procedure.

I the patient is Rh D-negative and unsensitized, anti-D immune

globulin is administered ater the procedure (Chap. 18, p. 356).

Multifetal Pregnancy. When perorming the procedure in a

dichorionic or monochorionic diamnionic twin gestation, care-

ul attention is paid to the location o each sac and the dividing

membrane. Until recently, a small quantity o dilute indigo carmine dye was oten injected beore removing the needle rom

the rst sac, with return o clear amnionic uid anticipated ollowing needle placement into the second sac. Because o widespread shortages o indigo carmine dye, experienced providers

typically oer amniocentesis without dye injection. Methylene

blue dye is contraindicated because it has been associated with

jejunal atresia and neonatal methemoglobinemia (van der Pol,

1992).

Complications

Midtrimester losses attributable to amniocentesis have decreased

with improvements in imaging technology. When perormed

by an experienced provider, the procedure-related loss rate is

estimated to be 0.1 to 0.3 percent, or about 1 per 500 procedures (American College o Obstetricians and Gynecologists,

2018; Odibo, 2008, Salomon, 2019). Te loss rate may be

doubled in women whose body mass index exceeds 40 kg/m2

(Harper, 2012). In twin pregnancies, Cahill and coworkers

(2009) reported a loss rate o 1.8 percent attributable to amniocentesis.

A B

FIGURE 17-4 A. Amniocentesis. B. The amniocentesis needle

is seen in the upper right portion of this sonogram. (Figures 17-4,

17-5, and 17-7 are reproduced with permission from Mastrobattista

JM, Espinoza J: Invasive prenatal diagnostic procedures. In Yeomans

ER, Hoffman BL, Gilstrap LC III, et al (eds): Cunningham and Gilstrap’s

Operative Obstetrics, 3rd ed. New York, NY: McGraw Hill; 2017.)

TABLE 17-9. Selected Tests Performed on Amnionic

Fluid and Volume of Fluid Required

Test Volume (mL)a

Chromosomal microarray analysis 20

Fetal karyotype 20

FISHb 10

Genotype studies (alloimmunization) 20

PCR tests for cytomegalovirus,

toxoplasmosis, or parvovirus

1–2 each test

Cytomegalovirus culture 2–3

Tests no longer commonly used:

Fetal lung maturity tests 10

Delta OD 450 (bilirubin analysis) 2–3

Alpha-fetoprotein 2

aThe volume of fluid needed for each test may vary according to individual laboratory specifications.

bFluorescence in situ hybridization (FISH) is typically performed for chromosomes 21, 18, 13, X, and Y.

PCR = polymerase chain reaction.346 The Fetal Patient

Section 6

When counseling regarding etal loss ollowing a procedure, it can be helpul to explain the dierence between overall losses and procedure-related losses. In a recent metaanalysis

that included more than 60,000 amniocentesis procedures and

300,000 control pregnancies, the loss rate in the absence o

a procedure was 0.6 percent, approximately twice the rate o

losses attributable to procedures (Salomon, 2019). Te indication or the procedure is also relevant, because severe etal

abnormalities and hydrops can signicantly increase the loss

rate, regardless o a procedure.

Other complications o amniocentesis include amnionic

uid leakage or transient vaginal spotting in 1 to 2 percent.

Leakage o amnionic uid generally occurs within 48 hours o

the procedure. Leakage o uid typically resolves ater a ew

days, and etal survival exceeds 90 percent (Borgida, 2000).

Management recommendations include pelvic rest and avoidance o strenuous activity. Needle injuries to the etus are rare.

I karyotyping is perormed, amnionic uid culture is successul

in more than 99 percent o cases, although cells are less likely to

grow i the etus is abnormal (Persutte, 1995).

Early Amniocentesis

Amniocentesis was ormerly oered between 11 and 14 weeks’

gestation. Te technique is the same as or traditional amniocentesis, but the procedure may be more challenging because

o lack o membrane usion to the uterine wall. Approximately

1 mL is withdrawn or each week o gestation. Early amniocentesis is associated with signicantly higher rates o etal loss

and other procedure-related complications than other procedures. Complications include development o talipes equinovarus (cluboot) and amnionic uid leakage (Canadian Early

and Mid-rimester Amniocentesis rial, 1998; Philip, 2004).

For these reasons, early amniocentesis is not recommended

(American College o Obstetricians and Gynecologists, 2018).

■ Chorionic Villus Sampling

Biopsy o chorionic villi is typically perormed between 10

and 13 weeks’ gestation. As with amniocentesis, the specimen

is usually sent or CMA or karyotype analysis. Te primary

advantage o CVS is that results are available earlier in pregnancy, permitting more time or decision-making and saer

pregnancy termination, i desired.

Technique

CVS is perormed transcervically or transabdominally. Both

approaches are equally sae and eective (American College

o Obstetricians and Gynecologists, 2018). Te transcervical

approach uses a exible polyethylene catheter that contains

a blunt-tipped, malleable stylet. ransabdominal sampling is

perormed with an 18- or 20-gauge spinal needle. Both use

aseptic technique and are perormed under direct transabdominal ultrasound guidance. Te catheter or needle is inserted into

the early placenta—chorion rondosum, and villi are aspirated

into a syringe that contains tissue culture media (Fig. 17-5).

Relative contraindications include vaginal bleeding or spotting, active genital tract inection, extreme uterine ante- or

retroexion, or body habitus precluding adequate visualization. I the patient is Rh D-negative and unsensitized, anti-D

immune globulin is administered ater the procedure.

Complications

Te overall etal loss rate ollowing CVS is higher than that with

midtrimester amniocentesis. Tis is because o losses that would

have occurred between the rst and second trimester in the absence

o a procedure. Te procedure-related loss rate is comparable to

that with amniocentesis, approximately 0.1 to 0.3 percent (American College o Obstetricians and Gynecologists, 2018). In act, a

recent metaanalysis identied no signicant losses rom CVS when

controls with similar risk proles were selected (Salomon, 2019).

As with amniocentesis, CVS indication will aect the loss rate. For

example, etuses with increased N thickness have a greater likelihood o demise. Tere is also a learning curve associated with sae

perormance o CVS (Silver, 1990; Wijnberger, 2003).

An early problem with CVS was its association with limbreduction deects and oromandibular limb hypogenesis, shown

in Figure 17-6 (Firth, 1991, 1994; Hsieh, 1995). Tese were

A B

FIGURE 17-5 A. Transcervical chorionic villus sampling. B. Catheter entering the placenta is marked and labeled.Prenatal Diagnosis 347

CHAPTER 17

subsequently ound to be associated with procedures perormed

at 7 weeks’ gestation (Holmes, 1993). When perormed at or

ater 10 weeks’ gestation, the incidence o limb deects does not

exceed the background population rate o approximately 1 case

per 1000 (Evans, 2005; Kuliev, 1996).

Vaginal spotting is not uncommon ollowing transcervical

sampling, but it is sel-limited and not associated with pregnancy loss. Te incidence o inection or leakage o amnionic

uid is less than 0.5 percent (American College o Obstetricians and Gynecologists, 2018).

A limitation o CVS is that chromosomal mosaicism is identied in up to 2 percent o specimens (Malvestiti, 2015). In

most cases, the mosaicism reects con-

ned placental mosaicism rather than

a true second cell line within the etus.

Tis is discussed in Chapter 16 (p. 318).

Amniocentesis should be oered, and

i the result is normal, the mosaicism is

usually presumed to be conned to the

placenta. Conned placental mosaicism

is associated with etal-growth restriction

(Baero, 2012; outain, 2018).

■ Fetal Blood Sampling

Tis procedure is also called cordocentesis

or percutaneous umbilical blood sampling

(Fig. 17-7). It was initially described or

etal transusion o red blood cells in the

setting o anemia rom alloimmunization,

and etal anemia assessment remains the

most common indication (Chap. 18, p.

356). Other indications include assessment and treatment o platelet alloimmunization and or etal

karyotype determination, particularly in cases o mosaicism

identied ollowing amniocentesis or CVS. Improvements in

testing using an amniocentesis specimen have eliminated the

need or etal blood sampling in most cases (Society or Maternal-Fetal Medicine, 2013).

Technique

Using aseptic technique, a 22- or 23-gauge spinal needle is

introduced into the umbilical vein under direct ultrasound

guidance, and blood is slowly withdrawn into a heparinized

syringe. Precise visualization o the needle is essential. As with

A B

FIGURE 17-6 Oromandibular limb hypogenesis is characterized by transverse limb

deficiency and absence or hypoplasia of the tongue or mandible. This is hypothesized to

result from vascular disruption with subsequent loss of tissue. A. Sonogram obtained at

25 weeks’ gestation demonstrates a fetal limb reduction defect involving the right hand.

B. Photograph of the right extremity of the same newborn. Chorionic villus sampling was

not performed in this pregnancy. (Reproduced with permission from Dr. Jamie Morgan.)

Uterine

wall

Placenta

Umbilical

cord

Ultrasound

transducer

A

B

FIGURE 17-7 Fetal blood sampling. A. Access to the umbilical vein

varies depending on placental location and cord position. With an

anterior placenta, the needle may traverse the placenta. Inset: With

posterior placentation, the needle passes through amnionic fluid

before penetrating the umbilical vein. Alternatively, a free loop of

cord may be accessed. B. Sonogram shows an anterior placenta

with transplacental needle passage into the umbilical vein (UV).348 The Fetal Patient

Section 6

Te American Society or Reproductive Medicine (2018) has

concluded that the utility o routine perormance o PG-A or

all IVF pregnancies has yet to be determined.

PG-M may be perormed when there is increased risk or a

particular single-gene disorder or a hereditary cancer syndrome

(American College o Obstetricians and Gynecologists, 2020d).

It is a test or a specic condition rather than a panel. In selected

cases, this technique may also be used identiy a suitable uture

donor o umbilical cord blood or an aected amily member.

PG-SR is oered i one o the parents is a carrier o such

a rearrangement, such as a duplication, deletion, translocation,

insertion, or inversion. Te test will identiy an unbalanced

chromosomal rearrangement but will not detect a balanced carrier. Counseling should thereore include this limitation

Nhận xét