Key result
Etiology-specific therapy for refractory rickets is linked to universal radiological healing but variable biochemical recovery.
Why the study?
Data from Eastern India on refractory rickets were limited, prompting a characterization of its etiological spectrum, clinical features, and six-month treatment outcomes.
Does etiology-specific treatment improve radiological healing, biochemical response, and height gain in children with refractory rickets?
Cohort (n=32)
No
Does etiology-specific treatment improve radiological healing, biochemical response, and height gain in children with refractory rickets?
p-value: p=<0.05
Etiology-specific therapy for refractory rickets in children leads to universal radiological healing but variable biochemical recovery at six months.
May support radiological healing in refractory rickets; leaves open optimal biochemical targets and height outcomes.
BACKGROUND: Refractory rickets comprises a heterogeneous group of inherited and acquired disorders characterized by persistent clinical, biochemical, and radiological evidence of rickets despite adequate vitamin D and calcium therapy. Owing to limited data from Eastern India, this study aimed to characterize the etiological spectrum of refractory rickets, compare the clinical, biochemical, and radiological characteristics across different etiologies, and evaluate treatment outcomes following six months of etiology-specific therapy. METHODS: This hospital-based prospective observational cohort study included 32 consecutive children with refractory rickets attending a tertiary pediatric endocrine centre in Eastern India. Demographic, clinical, biochemical, radiological, and genetic data (where available) were collected. Children were classified into distal renal tubular acidosis (DRTA), proximal renal tubular acidosis (PRTA), hypophosphatemic rickets (HPR), and vitamin D-dependent rickets (VDDR). All participants received etiology-specific treatment and were followed for six months. Treatment outcomes included radiological healing, biochemical response, and height gain. RESULTS: <0.05). CONCLUSIONS: DRTA was the predominant cause of refractory rickets in this cohort. Although clinical manifestations overlapped, characteristic biochemical abnormalities enabled accurate etiological differentiation. Etiology-specific therapy resulted in universal radiological healing but variable biochemical recovery, highlighting the importance of early diagnosis and individualized management.
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Mukhopadhyay et al. (2026) conducted a cohort in Refractory rickets (n=32). Etiology-specific therapy was evaluated on Radiological healing, biochemical response, and height gain (p=<0.05). Etiology-specific therapy for refractory rickets resulted in universal radiological healing but variable biochemical recovery (p<0.05).
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