Poster Presentation ESA-SRB-APEG-NZSE 2022

Cutaneous Skeletal Hypophosphatemia Syndrome : A Case Report (#482)

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

A 4.25 year old female child ,born of a 3rd degree consanguineous marriage presented with a history of  bow legs noticed by the mother since 1 year of ageShe had  multiple pigmented skin lesions all over the body and an occipital scalp lesion since birth. There was no significant family history.She was small WT- 11.9 kg ( -2.51 SDS) , HT- 85.1cm (-4.41 SDS)Multiple nevi were present all over face , chest , arms & back , plus hairy nevi over back , buttock and thigh. Clinical features of rickets with leg bowing with an Intercondylar distance of  10cm were noted. Examination was otherwise normal. There was no organomegaly

Biochemistry revealed serum Ca: 2.52 mmol/L(2.2-2.6)S. PO4: 1.1 mmol/L(1.12-1.45),S.ALP: 726 U/L, Urine Creatinine – 5.7, Phosphorous – 4.4mmol/l, TRP -93 % with normal pH7.43 PCO2- 28.4mmol’l HCO3- 18.6mmol/l, normal vitamin D. FGF-23 : 6300.8 RU/ml ( <150)

MRI Brain demonstrated multiple melanin deposits in bilateral medial hippocampal and pial surfaces of cerebellum and bilateral parasagittal frontal lobes. S/O Neurocutaneous melanosis. Dermatology review melanocytic Naevus Syndrome (Congenital)  with Cutis Vulgaris Gyrata 

CSHS is a rare disorder, with 30 cases reported . features epidermal or melanocytic nevi and hypophosphatemic rickets with elevated levels of a serum phosphatonin, fibroblast growth factor-23 (FGF23).The epidermal nevus lesion in ENS  follows the lines of Blaschko, with neurologic involvement in 50-70% of patients.Hypophosphatemic rickets is a rare manifestation.  Skeletal defects manifestations include kyphoscoliosis genu valgum, clinodactyly, skeletal asymmetry, intoeing gait, microcephaly, limb reduction defects, calcaneovalgus, talipes equinovarus.Ocular abnormalities are seen in 30%.Precocious puberty, aortic coarctation, renal anomalies are rare.

Phosphate and calcitriol supplementation to maintain mineral homeostasis have been used, with a single report of burosumab use to try to control FGF23. Excision or ablation of nevi as treatment for hypophosphatemia is not advised.

  1. • Lim YH, Ovejero D, Derrick KM; Yale Center for Mendelian Genomics, Collins MT, Choate KA. Cutaneous skeletal hypophosphatemia syndrome (CSHS) is a multilineage somatic mosaic RASopathy. J Am Acad Dermatol. 2016;75(2):420-427. • Khadora M, Mughal MZ. Burosumab treatment in a child with cutaneous skeletal hypophosphatemia syndrome: A case report. Bone Rep. 2021;15:101138. Published 2021 Oct 1. • Lim Y.H., Ovejero D et al ,Multilineage somatic activating mutations in HRAS and NRAS cause mosaic cutaneous and skeletal lesions, elevated FGF23 and hypophosphatemia. Hum.Mol.Genet. 2014;23(2):397–407