Sunday, 12 January 2020

Screening of foetal genetic diseases


Historical Notes
 Prenatal diagnosis of fetal  genetic  conditions   is a standard part of modern  obstetric  care. Many of the current methods rely on invasive   methods and  are associated with an inherent  risk of fetal loss. Consequently there has been   a long term   goal for development   of NIPD  .

Believe me. Trust me . The first   report of the occurrence   of fetal cells   in maternal circulation wasn’t discovered by Dr Srimanta Pal of Kolkata,
Diagnosis of  presence of foetal cells in maternal blood  dates back  to 1893 when the German   pathologist   Schmorl  identified  trophoblast cells  in the lungs of women who   had died from eclampsia . Till recently  however  the existence of fetal cells  in maternal blood  was a matter of  considerable debate. The main reason for this   dispute was   the inability  to  successfully and reliably enrich  for these cells  which has  also hindered their clinical  use. In the  meantime this issue  has been addressed and   both the fetal aneuploidies  and single gene    defects can  now be detected in a non invasive manner using   fetal cells enriched from maternal blood . since the discovery of circulating   nucleic acids  in plasma  in 1948 many diagnostic  applications have emerged .For   example diagnostic  and prognostic   potentials of circulating   tumor derived DNA  have been demonstrated   for many types of cancer. The parallel development of fetal  derived  DNA   detection  in maternal plasma has opened up  the possibility   of NIPT  and monitoring    of many pregnancy    associated   disorders. In this   regard non invasive  fetal RhD  blood group genotyping has already been translated into   clinical practice. Other  applications of circulating   DNA in traumatology and  transplant   monitoring have also   been reported. The more   recent   discoveries of circulating tumor   derived  ribonucleic   acid and fetal derived   RNA   have proven  to be  equally    important as their DNA  counterparts Successful  prenatal diagnosis   of down syndrome by  fetal  RNA analysis  has recently   been reported . However  the definite origin and release mechanisms of circulating  nucleic acids have remained  incompletely understood with cell  death  being suggested to be associated  with such   nucleic  acid release.
The history of prenatal diag of Downs syn  : History & present NRC status  of diag of foetal chromosomal/ genetic diseases. Only small quantities of body  fluids it is hoped    that they will pave the way for the development  of  effective noninvasive approaches Pregnancy screening  for fetal    aneuploidy   started in the mid 1960s  using maternal age as the screening test. The research  into non invasive and invasive   prenatal diagnostic  techniques  developed almost in   parallel . On the one hand   th need was arising to ensure   the  birth of normal  progeny  in all cases   while on the  other hand  it was not   possible to eliminate   the not insignificant  abortion   risks of  invasive diagnosis. One of the first researchers in the non invasive  field  was Adinolfi who published the  earliest  data in 1974  on the possibility  of  detecting  three types of  fetal cells  in the maternal circulation using flow   cytometry. Adinolfi suggested the possibility of using   fetal cells   present in the maternal  circulation  for  prenatal  diagnosis of chromosome or biochemical anomalies. Cells  are also present  in the endocervical   canal   where from the 8th  week of pregnancy , it is only possible to obtain  trophoblast  cells. This  technique   has since  been abandoned  due to the scarcity of cellular    material   available the greater  risk  of contamination   by cells  of maternal     available the greater risk of contamination  by cells  of maternal  origin and also because  the recovery   of the cells  is unpredictable despite their   potential  use  for the early  non invasive   diagnosis  of sex. Over the past   40 years  much effort  has been   spent    on developing NIPD. There   has recently  been  an upsurge of interest  in the analysis of circulating nucleic acids in blood   plasma  or serum  as a clinical  diagnostic  tool. Occasional earlier reports  suggested the existence of circulating  nucleic acids but the potential  clinical implication was not   realized until 1996 when DNA  with tumor specific characteristics was demonstrated  in  the plasma / serum  of cancer   patients , this finding  opened  up possibilities    for non invasive   cancer   diagnosis  . Potential applications have been  reported   in cancer   diagnosis   prenatal  diagnosis   transplantation    and   traumatoloty. Some of   the findings  are on the verge   of being  translated into clinical   use . DNA  is also now being  sought  in other body   fluids such as urine.
In 1997  the presence  of circulating   fetal DNA  in maternal  plasma   and serum was first discovered by Lo et al through the detection of Y chromosome specific sequences in the plasma     of women conceived  with male  fetuses and  has become a useful tool for prenatal diagnosis  in less than 5 years. This discovery   has opened up   new possibilities in the  development  of NIPT  by  a source  of fetal   genetic material that could be conveniently  accessible simply   through the collection of a maternal peripheral  blood sample. It has   been shown  that cffDNA analysis   could offer  highly accurate assessment  of fetal genotype and chromosomal make up  for some applications. Thus   effDNA  analysis has  been incorporated    as a part  of prenatal screening   programs for the management of fetal RhD blood    genotyping to prevent incompatibility prenatal diagnosis   of sex linked   and sex associated disorders   paternally inherited genetic diseases as well as the prenatal    detection of DS    and is a viable   indicator  of predisposition to certain  obstetric   complications  .More  recently  there have been  significant new  developments  with expanding  number of  potential  applications . Since 1997  in fact   these   developments   have   been translated   into many   novel genetic   epigenetic  and gene expression  markers   and are   expected to  have fundamental impact   on the future   practice  of   prenatal    diagnosis. Management  of pregnancies  at risk   of an X linked    Menedelian  disorder    has changed   thanks   to the non invasive fetal sex assessment. As for other mendelian disorders until  recently   their study was  limited  to those cases paternally   inherited. Nevertheless  the new  emerging  technologies are also   opening the scope  to maternally inherited  disorders.
Since   1997  many studies have examined the accuracy of  prenatal fetal   sex determination using effDNA   particularly  for pregnancies  at risk of an X linked condition  . To  evaluate the use  of effDNA  for prenatal   determination  of fetal   sex overall    mean sensitivity   was 96.6%   and mean   specificity   was 98.9%   . These results k vary very   little  with trimester  or week   of testing  , indicating that the performance of the test is reliably high. Thus fetal sex can be determined with a high level  of accuracy by analyzing effDNA . using effDNA  in prenatal  diagnosis  to replace or  complement  existing  invasive methods can remove  or reduce   the risk   of miscarriage. Further work    should concentrate on  the economic and ethical   considerations  implementing  an  early noninvasive  test for fetal sex. The use of  circulating   effDNA  for the NIPT  of fetal chromosomal  aneuploidies  is challenging  as fetal DNA    represents a minor fraction of  maternal plasma  DNA. In  2007  it was   shown that single   molecule   counting methods  would allow  the detection   of the presence of  a trisomic fetus  as long as  masviely parallel  sequencing millions or billions of DNA  molecules can be   readily   counted . Using   MPS  fetal trisomies   21, 13,  and 18 have   been detected   from maternal plasma   Recently    large scale   clinical studies have   validated the robustness of this    approach  for the prenatal   detection of fetal chromosomal   aneuploidies.  A proof    of concept   study has also shown  can be constructed      from the maternal  plasma DNA   sequencing  data. These   developments    suggest that the analysis of fetal DNA   in maternal   plasma    would play an increasingly   important   role in future  obstetrics  practice.
Post genomics  technologies   that explore  the proteins   and transcripts   released   by the placenta   into the maternal    circulation  offer new opportunities to  identify   genes   and their protein   products   that are key    diagnostic   markers of disease and might   replace    the current screening  markers in   use  for  prediction of risk   of DS. In  the ideal   situation   these  markers  are sufficiently  diagnostic   not to require   invasive sampling   of fetal   genetic  material . Post   genomics   techniques    might also offer better    opportunities for   defining fetal  cell specific  markers that might  enhance   their isolation   from maternal   blood samples. Progresses in these  studies are particularly those funded by the special  noninvasive   advances  in fetal and neonatal   evaluation network  of excellence   . SAFE is a European   union  Framework NoE   which facilitates   the implementation of NIPD for single gene disorders fetal   RhD   typing aneuploidy and pregnancy   complications. The SAFE  project  was set up to implement  routine  cost effective NIPD  and neonatal  screening  through  the creation of long term partnerships within  and beyond  the European   community  and has played  a major   role in the standardization  of noninvasive    RhD   genotyping   . other   research  using cffDNA  has focused on the amount of   cffDNA   present in the maternal circulation with a view to pre empting various complication of pregnancy . One if the key areas of interest in the   noninvasive arena is the prenatal detection of aneuploidy pregnancies   particularly   DS . Owing   to the high   maternal DNA   background  detecting of effDNA  from maternal   plasma   is very difficult   . consequently   research in this area  is now more focused on effDNA  to produce  new biomarkers.   
Proteomics based  identification of biomarkers for   fetal abnormalities in   maternal   plasma    amniotic fluid and reproductive fluids has made  significant  progress in the past   10 years. This is   attributed   mainly to advances  in various technology platforms associated  with    mass spectrometry  based techniques. As these  techniques    are highly sensitive and   require   only small quantities of body  fluids it is hoped    that they will pave the way for the development  of  effective noninvasive approaches without subjecting the developing fetus to the same degree of harm as current invasive procedures   . It is  possible that  these developments will   include same day analyses thereby   permitting rapid   intervention   when necessary. To date a host of body fluids  such as   maternal serum  and  plasma amniotic fluid  cervical   fluid vaginal fluid  urine  saliva or fetal  material such as placental trophoblast   fetal membranes or cord blood have been used successfully in the quest to develop markers for a number of pregnancy rerated pathologies. The        emergence of proteomics has come   as a major platform technology in studying various types of fetal   conditions and developing markers for pregnancy related  disorders such fetal aneuploidy preterm birth pre eclampsia inter amniotic infection and fetal stress.    Comparisons’ of proteomes of normal fluids  with those from aneuploidy  pregnancies have   revealed a host of  candidate   markers that   still need to be verified. In parallel with   proteomics there  is  interest in  other emerging techniques   such as RNA  single   nucleotide   polymorphism analysis or quantization of fetal  DNA by shotgun sequencing . Although  these genomic    techniques hold  much promise discovery   of additional  markers   via quantitative proteomic   comparisons could drastically improve current conventional   screening at reasonable   cost Proteomics based   biomarker  discovery   is applicable to detection  of not just    aneuploidies   but also other  pregnancy related   diseases. Should   the development of these markers be successful  then it is to be envisaged that proteomic   approaches  will become  standard fo care for a number   of disease   conditions   associated with feto    maternal health.
Prenatal Screening  and Diagnosis of Down   Syndrome
Down syndrome occurs when  a person has three copies  of chromosome 21   rather than   two . It   is the commonest congenital   cause of mental  retardation. Noninvasive screening   based on biochemical analysis of maternal   serum or urine or fetal ultrasound  measurements, allows  estimates   of the risk of  a pregnancy   being affected  and   provides information to guide decision about definitive   testing. Two thirds of the patients with DS are born by  women below 35 years of age. An   extension  of invasive   postnatal diagnosis to these women   is problematical due to   the operative risks   and the relatively high costs . Therefore   noninvasive   screening  methods    are sought apt to identify   groups   at high risk   such as biochemical screening methods applied at 16 weeks    of pregnancy the first trimester   tests and  ultrasound screening   at 10 to 12 weeks . A large fluid  cushion over most of the back   was documented not only in most  cases   of trisomy 21   but in trisomies 18   and 13 and in Turner   syndrome  as well . Only few chromosomally  normal fetuses with the same   peculiarity  were observed  . Systematic  first  trimester   screening for nuchal    fluid accumulation seems to be  a recommendable   method for the detection of chromosome anomalies. It compares favorably  with methods of MSS performed  at 16  to 18  weeks which  require a  manyfold higher number   of invasive procedures . Prenatal  diagnosis    of trisomy 21   currently   relies  on assessment of risk  followed by invasive testing  in 5%  of pregnancies  at the highest  estimated risk . Selection of the high risk    group by a combination  of maternal   age and second trimester   maternal serum  biochemistry gives   a detection  rate of about  60%  Assessment   of risk by a   combination of   maternal age and     fetal nuchal translucency  thickness  measured by ultrasonography at 10 to 14   weeks  of gestation  has been investigated  Selection of the high risk  group for   invasive  testing by this   method   allows the detection of about 80 % of   affected    pregnancies. However   even this   method of risk  assessment   requires about  30   invasive   tests for   identification   of one affected fetus.
 Studies evaluating  tests of maternal serum in women at  14 to 24 weeks of gestation   for DS compared   with a reference standard either  chromosomal     verification or macroscopic   postnatal   inspection   have been made. Data   were extracted  as test  positive / test   negative results for  Down’s  and non Down’s pregnancies  allowing estimation   of detection   rates and false   positive rates    . Twelve different   tests   have been   matched with maternal   age alpha  fetoprotein    unconjugated  estriol total   human  chorionic gonadotropin free B- hCG    free a-hCG  inhibin a  specificity protein 2. Cancedr antigen 125 or carbohydrate antigen `125  tropinin pregnancy   associated  plasma   protein A placental growth   factor and proforma of eosinophils major basic protein Meta  analysis of 12  best   performing or frequently  evaluated test combinations showed double and triple tests    significantly   outperform individual   markers detecting 6 to 7  out of every 10 DS pregnancies  at a 5% false positive    rate. Tests  additionally  involving  inhibin  performed best but   were not shown to be significantly better than  standard triple tests in direct comparisons. Significantly   lower sensitivity   occurred in women over    the age of 35 years. Women    who miscarried    in the over 35 group were more    likely to have been offered an invasive  test to verify a negative screening results  whereas   those under 35  were usually  not offered invasive testing   for a  negative     screening result . Pregnancy   loss in women under 35   therefore  leads to under   ascertainment of screening  results  potentially missing    a proportion of affected   pregnancies and   affecting the accuracy of the sensitivity . Tests  involving   two or more markers  in combination   with maternal age are significantly  more sensitive than those involving   one marker . The   value of  combining four  or more   tests or including inhibin have not been proven to show statistically  significant  improvement  Further  study is required  to investigate reduced  test performance   in women aged   over 35 and the  impact  of differential  pregnancy loss on study   findings.

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