An Iranian Patient with Maroteaux Type Acromesomelic Dysplasia, Showing no Involvement of Distal Lower Limbs
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Letter to the Editor
P: 122-123
March 2020

An Iranian Patient with Maroteaux Type Acromesomelic Dysplasia, Showing no Involvement of Distal Lower Limbs

J Clin Res Pediatr Endocrinol 2020;12(1):122-123
1. Neonatal research center, Shiraz University of Medical Sciences, Shiraz, Iran
2. Department of Pediatric Endocrinology, Shiraz University of Medical Sciences, Shiraz, Iran
3. Student Research Committee, Shiraz University of Medical Sciences, Shiraz, Iran
No information available.
No information available
Received Date: 21.06.2019
Accepted Date: 13.07.2019
Publish Date: 19.03.2020
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Dear Editor,

Acromesomelic dysplasia, Maroteaux type (AMDM) is an autosomal recessive form of acromesomelic dysplasia characterized by disproportionately short stature, shortening of the middle and distal segments of the limbs as well as vertebral involvement. AMDM is the result of a mutation in the natriuretic peptide receptor 2 (NPR2) genes which impairs skeletal growth (1,2,3).

A 2- years old boy, offspring of non-consanguineous parents and of a 2nd pregnancy, was referred to the endocrine and metabolic center of the Nemazee Hospital, located in southwestern Iran, for evaluation of short stature. The patient was born at 38 weeks of gestation by cesarean section and was healthy by Apgar scoring. Birth weight was 3100 g, length 45 cm and head circumference 35 cm. He had no dysmorphic features and general physical examination revealed no pathology. There was no satisfactory length gain after birth, as noticed by his parents. At the referral time at age 2 years the patient had a weight of 8200 g (-4 SD), a length of 71 cm [-4 standard deviation (SD)]. Head circumference was 48 cm (0.3 SD). Fingers of the hand were extremely short and broad with small nails; there was no redundant skin on the fingers (Figure 1). His feet and toes were normal. Frontal bossing, low set ears and wrist joint hyperflexibility were prominent features. All developmental milestones were within normal limits. His older sibling was of normal stature. Matental height was 156 cm (-1.6 SD) and the father was 163 cm (-1.9 SD) tall. His older sibling was of normal stature. None of the other family members were affected.

Figure 1

Radiographic findings showed curved radius, relatively short ulna, and broad metacarps with wide phalanges. The vertebrae were of normal size and showed no beaking. Iliac wings and metatarsal bones were normal (Figure 1). DNA was extracted from the peripheral blood by standard techniques and microsatellite analyses were performed. Whole exon sequencing test and mutation confirmation by direct Sanger screening were performed and evaluated by reference sequence, AMDM maps to 9p13.3. Cytogenic evaluation could not be performed in the parents and in the older sibling. Informed consent was obtained from his parents for this report.

Figure 1

The mutation of the case was displayed in NPR2 with cytogenic location of 9p13.3. This mutation overlaps with two diseases: firstly, autosomal dominant epiphyseal chondrodysplasia, miura type which is characterized by tall stature, long hands and feet with arachnodactyly, and secondly, short-rib thoracic dysplasia 5 with or without polydactyly (4,5). Both diseases have completely different clinical patterns and radiographic manifestations from AMDM.

In summary, considering the skeletal changes, radiological findings and sequence analysis of the mutation, this patient is the first AMDM case reported from Iran. The patient had severe short stature, but no obvious abnormality in the distal segment of his lower limb. We suggest that this patient may represent a new variant form of AMDM.

References

1
Irfanullah, Umair M, Khan S, Ahmad W. Homozygous sequence variants in the NPR2 gene underlying Acromesomelic dysplasia Maroteaux type (AMDM) in consanguineous families. Ann Hum Genet 2015;79:238-244. Epub 2015 May 11
2
Vasques GA, Amano N, Docko AJ, Funari MF, Quedas EP, Nishi MY, Arnhold IJ, Hasegawa T, Jorge AA. Heterozygous mutations in natriuretic peptide receptor-B (NPR2) gene as a cause of short stature in patients initially classified as idiopathic short stature. J Clin Endocrinol Metab 2013;98:1636-1644. Epub 2013 Sep 3
3
Tran TH, Cao MH, Luong LH, Le PT, Vu DC, Ta TD, Bui TH, Nguyen DH, Van Ta T, Tran VK. Acromesomelic dysplasia Maroteaux-type in patients from Vietnam. Am J Med Genet A 2019;179:1420-1422. Epub 2019 May 11
4
Yamamoto K, Kawai M, Yamazaki M, Tachikawa K, Kubota T, Ozono K, Michigami T. CREB activation in hypertrophic chondrocytes is involved in the skeletal overgrowth in epiphyseal chondrodysplasia Miura type caused by activating mutations of natriuretic peptide receptor B. Hum Mol Genet 2019;28:1183-1198.
5
Naki MM, Gür D, Zemheri E, Tekcan C, Kanadikirik F, Has R. Short rib-polydactyly syndrome. Arch Gynecol Obstet 2005;272:173-175. Epub 2004 Dec 17