RT Journal Article
T1 Positive Response to One-Year Treatment With Burosumab in Pediatric Patients With X-Linked Hypophosphatemia.
A1 Martín Ramos, Silvia
A1 Gil-Calvo, Marta
A1 Roldán, Virginia
A1 Castellano Martínez, Ana
A1 Santos, Fernando
K1 FGF23
K1 X-linked hypophosphatemia (XLH)
K1 burosumab
K1 children
K1 hypophosphatemia
K1 rickets
K1 vitamin D
AB X-linked hypophosphatemia (XLH) causes significant burden in pediatric patients in spite of maintained treatment with phosphate supplements and vitamin D derivatives. Administration of burosumab has shown promising results in clinical trial but studies assessing its effect in the everyday practice are missing. With this aim, we analyzed the response to one-year treatment with burosumab, injected subcutaneously at 0.8 mg/kg every 2 weeks, in five children (three females) aged from 6 to 16 years, with genetically confirmed XLH. Patients were being treated with phosphate and vitamin D analogs until the beginning of burosumab treatment. In all children, burosumab administration led to normalization of serum phosphate in association with marked increase of tubular reabsorption of phosphate and reduction of elevated serum alkaline phosphatase levels. Baseline height of patients, from -3.56 to -0.46 SD, increased in the three prepubertal children (+0.84, +0.89, and +0.16 SD) during burosumab treatment. Growth improvement was associated with reduction in body mass index (-1.75, -1.47, and -0.17 SD, respectively), suggesting a salutary effect of burosumab on physical activity and body composition. Burosumab was well-tolerated, mild local pain at the injection site and transient and mild headache following the initial doses of burosumab being the only reported undesirable side effects. No patient exhibited hyperphosphatemia, progression of nephrocalcinosis, worsening of metabolic control or developed hyperparathyroidism. Mild elevation of serum PTH present at the beginning of treatment in one patient 4 was not modified by burosumab administration. These results indicate that in the clinical setting, beyond the strict conditions and follow-up of clinical trials, burosumab treatment for 1 year exerts positive effects in pediatric patients with XLH without major adverse events.
SN 2296-2360
YR 2020
FD 2020-02-18
LK http://hdl.handle.net/10668/15203
UL http://hdl.handle.net/10668/15203
LA en
DS RISalud
RD Apr 11, 2025