Friday, 24 April 2020

Skeltal dysplasia


which dysplasia are lethal??Ans;- Short   Femur  length   and prediction   of lethality   in skeletal   dysplasias . Fetuses with skeletal dysplasias in which    all lethal cases were associated with a femur length   abdominal   circumference ratio   of 0.16  . Although   the test detected   lethal cases   with 100 % sensitivity   few cases   of achondroplasia   were  erroneously  identified   as lethal using this method. A different   approach   has been proposed   which mentions   lethality    in 23 out of 25 cases of skeletal dysplasias with a femur length below the 1st percentile for gestational   age and   presence of bell shaped  thorax  or decreased  bone echogenicity.  If one cannot estimate many important diameters  necessary to confirm the diagnosis of skeletal dysplasia / due possibly to time constraints then a femur/  abdominal   circumference ratio  less than 0.16  have to be measured as a minimum. In fact  F/ AC  is the  main  discriminator  among fetuses  with lethal skeletal   dysplasias and   that this   measurement   has  still a  better  performance  than a)  femur  shortening b)  thoracic circumference and c) thoracic circumference/ abdominal circumference  ratio.

Which  gene has undergone mutation?? :-- Till date it is known that about    25%   of all bone   disorders  the mutated gene   has not been  yet identified . What is the classification of constitutional disorders of bone ?? Ans:- Skeletal dysplasias   are a heterogeneous group  of disorders affecting the development   of chondro-osseous tissues leading  to abnormalities in the size ,   mineralization  and   shape of various  segments of  the skeleton. Despite recent advances in   imaging and molecular genetics accurate   prenatal diagnosis of  skeletal   dysplasias remains a clinical   challenge. In the most recent   revision   of the International Nosology and Classification of Constitutional disorders of bones , it  is mentioned that in approximately   25%   of all bone   disorders  the mutated gene   has not been  yet identified.

What does  International  skeletal  Dysplasia   Registry tells us??  :--It is   important  to acknowledge  the contribution  of the International  skeletal  Dysplasia   Registry in the  identification   and study  of skeletal   anomalies   assisting  providers  and  patients  in the diagnosis   and clinical  management  of skeletal  disorders. This registry informs us that a-twenty three percent   of the affected infants  were stillborn  whereas 32%  died during the first  week of life. The overall   frequency  of skeletal  dysplasias among perinatal  deaths was 9.1/1000.

 c) How significant is  incidental  solo findings of “short femoral length at late trimester”  ?? Will  we be  worried?? Concern to the Obstetrician is “Am I   dealing with normal biological variant/ real pathology of bone so called –“skeltal dysplasia” ?? Ans:- Significance of short   femoral length if noticed at  mid to late  third trimester “” This is  not unusual  for the femur  to be significantly shorter than the other  measurements   used to estimate   gestational age. In many cases  the femur is approximately 2 weeks   less than   the head circumference and menstrual age. The cause of this is often uncertain   and the short femur is often   attributed to biologic variation or ethnicity.  If the bones appear normal   morphologically and no other anomalies   are seen it is likely a normal variant.

One week less is permissible. Short F L . But my dear members this even 1 week lag may rarely be due to 1)  a sign  of trisomy   21 or 2) an  early manifestation of a short    limb   skeletal   dysplasia.  :-- One should acknowledge that a fetus from white  parents of normal height   and middle   socioeconomic   status will have a standard FL which one should not compare with that of FL of Indian parents of low socioeconomic  status .  Although   the femur can be   smaller than other   measurements in the third trimester   it is not acceptable for it to be greater    than 1 week less than the other measurements in the late first   and early to mid second   trimesters. Although   this deviation  may be nothing  more than   a temporary  growth lag or  normal   variation   at this stage , it may also be  1)  a sign  of trisomy   21 or 2) an  early manifestation of a short    limb   skeletal   dysplasia. This  may be  a case  of trisomy 21 as well . One should    not take   comfort   in comparing the femur measurement to the estimated   gestational   age by ultrasound imaging   . Remember that   unless excluded the femur measurement    is included   in the sonographic estimation of   gestational  age.
Classification  of Genetic    skeletal disorders—As many as   364        genes may be involved in the etiology of skeletal dysplasia !!! :-   . Most  skeletal    anomalies  are a phenotypic  manifestation of  a   mutation in a gene   and altered  protein expression therefore they can be  grouped according  to the   affected   genes as they share    similar clinical characteristics. The International skeletal   dysplasia Society   periodically   reviews this.  But with the advent of   “sequencing   technologies    and the  increasing  availability of whole   exome sequencing”--   allowing the discovery  of more gene   related skeletal anomalies. With these technologies as many as  436   clinical conditions were classified into   42 groups involving 364    affected    genes . The classification provides the A) group/ name of the skeletal disorder B)  type of   inheritance; C) MIM number   ; D)  locus  of  the mutation in the  gene   , F) affected protein  and F) associations/ difference with other skeletal   anomalies   . International Nosology and Classification of Constitutional disorders of bones  &  International  skeletal  Dysplasia   Registry:-- Also tell us  classification includes:-Skeletal   dysplasias , metabolic bone   disorders,   dysostosis  ,  skeletal malformations and reduction  syndromes are included   in this   classification . However   the authors also   clarify that in approximately 25% of skeletal   disorders   the mutated gene has  not yet been  identified . The genetic   basis   for classification of very  rare   diseases   was done  by   family pedigree or was based   on homogeneity  or phenotype  in unrelated families. The classification aims to provide  more complete   information   for prenatal  counseling   and clinical   management  . The full   document  can be consulted   ate the ISDS website.
Shortening of the extremities can involve   the entire limb , or   the proximal segment  only, or   the intermediate  segment  or only to the distal segment   . What is meant by   rhizomelia  ?? Ans:-The diagnosis of rhizomelia  or mesomelia requires comparing   the bony dimensions of   the lower legs   and forearms with those  of the thighs and arms . The relationships between the humerus and ulna as well as    the femur   and tibia which    can be used for the objective    assessment   of rhizomelia   and mesomelia   presents  a list of   skeletal   dysplasias  characterized by  rhizomelia,    mesomelia and micromelia.
There are several  skeletal dysplasias which feature    alternation of the   hands and feet. The term A)  polydactyly refers  to the presence of   more than  five digits. It is classified   as postaxial  if the extra digits  are on the ulnar    or fibular  side and preaxial if they  are located   on the radial or tibial  side  . B) By contrast Syndactyly refers    to soft   tissue or bony   fusion of adjacent digits. C) Clinodactyly  consists   of deviation of a finger  or fingers.

defect in a given case  is not that easy: Despite   increasing   availability of    molecular   testing in about one third of skeletal dysplasias their molecular basis   has not been   defined. The role of diagnostic imaging in the prenatal investigation   of skeletal dysplasia  to narrow the   differential diagnosis of  skeletal dysplasias   so that appropriate confirmatory molecular tests can be done to predict  lethality and to identify   the fetus   with a skeletal dysplasia  early enough in gestation so that   the diagnostic   workup   can be completed before   the limit of fetal   viability.
Lung  volumetry by three   dimensional sonography: can it help to identify the lethality of skeletal dysplasia ?? Ans:- . Fetal  lung volumetry by 3D  sonography  has been performed   using two techniques    multiplanar  and VOCAL  . Nomograms for lung volumetry   using 3D  sonography  are available  in the literature  . Both  the 3D   multiplanar   and 3D VOCAL modes    can be used  to measure   fetal lung volumes  an observation   that was  subsequently   . A potential    advantage   of the VOCAL  technique  is the possibility  of obtaining   fine contours of the lungs   which may be   particularly   valuable   when the  outline   of the organ is irregular such as in cases of congenital diaphragmatic   hernia.  In contrast obtaining   lung volume  measurements using the 3D multiplanar technique is faster   taking usually less  than 5 minutes  to perform . Volumes   are best estimated   when datasets are acquired   using a transverses view of the fetal   thorax.  compared volumetric measurements   of the fetal  lungs obtained using   the VOCAL   method with lung   volumes calculated at the time of   autopsy   in 8 cases   of congenital   diaphragmatic hernia   and in 25   control fetuses   without pulmonary   malformation. The mean   relative error   of 3D   sonography  to estimate the actual lung volume was -7.19 % in cases  of congenital diaphragmatic   hernia and -0.72 %  in normal   fetuses . Barros and colleagues   studied   24  fetuses   with skeletal   dysplasia and measured the total   lung volume   using VOCAL   . an   abnormally    reduced lung volume was defined   as below   the 5th  percentile   for gestational age   . From    18 fetuses diagnosed at birth   with lethal pulmonary    hypoplasia.  83%   had a total   lung volume    below the 5th  percentile for gestational age. Lung  volume  more accurately   predicted pulmonary   hypoplasia than thoracic circumference   thoracic  circumference / abdominal   circumference ratio   and thoracic area/ cardiac area   ratio .

.  Skeletal Dysplasias  characterized  by Rhizomelia , Mesomelia and Micromelia What is platyspondyly ?? Ans:-The most   common spinal    abnormality   seen in skeletal   dysplasias is  platyspondyly which      consists  of flattening  of the vertebrae . Xyphhosis   scoliosis  and coronal   clefting   of vertebral   bodies  have been   also reported.

Types of skeletal defects : Rhizomelia ,Thanatophoric dysplasia
Atelosteogenesis
Chondrodysplasia  punctata
Congenital short   femur
Achondroplasia
Hypochondroplasia
Mesomelia
Mesomelic  dysplasia
Ellis- van Creveld  syndrome
Acromesomelia
Ellis-  van Creveld syndrome
Micromelia
Achondrogenesis
Atelosteogenesis
Short rib-  polydactyly syndrome
Diastrophic  dysplasia
Fibrochondrogenesis
Osteogenesis   imperfect
Kniest  dysplasia
Dyssegmental dysplasia
Roberts syndrome

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