Poster Presentation ESA-SRB-APEG-NZSE 2022

A  curious case of a boy with repeated fractures (#481)

Margaret Zacharin 1 , Rachna Keshwani 2 , Sudha Rao 2 , Rajesh Joshi 2 , a Mehta 2 , a Patil 2
  1. royal children's Hospital, Parkville , VIC, Australia
  2. paediatric endocrinology, Bai Jerbai Wadia Hospital for Children, Mumbai, India

A 5 years 3 months old boy born of a third degree consanguineous union sustained multiple fragility fractures involving the forearm and legs since age 3 years. Deformities of the lower limbs due to repeated fractures were present. He had normal hearing, development and dentition and no family history of repeated fractures, deformities, hearing or dental problems. On examination dentition was normal, sclerae white, no birth marks, pallor or organomegaly.

Bone turnover, Vitamin D, PTH, coeliac screen urine Calcium/creatinine, pH and electrolytes were all normal. Skeletal survey demonstrated normal bone density,Z score : AP spine: z+0.1 Total body: +0.7. Long bone fracture sites demonstrated poor callus formation, normal vertebrae, no bone dysplasia.

Clinical exome revealed a homozygous missense pathogenic mutation in Exon 1 of the Bone Morphogenetic Protein 1 c.34G>V (p.Gly12Arg) causing Osteogenesis Imperfecta type 13.The BMP1 gene on chromosome 8p21.3 is a multifunctional protein acting as a procollagen type 1 C-terminal propeptide endopeptidase. Inactivation of BMP1 impairs proteolytic removal of the carboxyl terminal propeptide from procollagen type 1 and the normal assembly of mature collagen type 1 fibrils
Autosomal recessive mutations result in clinical type 3 (moderate to severe) and genetic type 13 OI.

  1. Asharani PV, Keupp K, Seemler O, et al. Identification of a mutation causing deficient BMP1/attenuation of BMP1 function compromises osteogenesis leading to bone fragility in humans and zebrafish. Am J Med Genet. 2012: 90:661-674 Martinez-Glen V, Valencia M, Caparros-Martin JA, et al. Identification of a mutation causing deficient BMP/mTLD proteolytic activity in autosomal recessive osteogenesis imperfecta. Hum Mutat. 2012; 33:343-350 Fahiminiya S, Al-Jahhad H, Majewski J, et al. A polyadenylation site variant causes transcript –specific BMP1 deficiency and frequent fractures in children. Hum Mol Genet. 2015; 24(2):516-24