In this prospective longitudinal cohort study we studied the efficacy and safety of burosumab in real-clinical practice for \<13- and \>13-years old children affected with X-linked hypophosphatemia. 57 children with XLH were switched from conventional treatment to burosumab. After 12 months we assessed the efficacy and safety of treatment with burosumab on the whole cohort and separately on the cohort of \>13-years old adolescents.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
57
Children affected with X-linked hypophosphatemia were switched from conventional therapy to burosumab
Hospital Bicetre
Le Kremlin-Bicêtre, France
RECRUITINGRadiological changes in rachitic lesions evaluated with knee MRI
maximum width of the physis and transverse extent of widening
Time frame: 12 months
serum phosphate
mmol/l
Time frame: 12 months
renal phosphate reabsorption
mmol/l
Time frame: 12 months
alkaline phosphatase
U/l
Time frame: 12 months
1,25(OH)vitaminD
pg/ml
Time frame: 12 months
parathyroid hormone
ng/l
Time frame: 12 months
height
standard deviation score
Time frame: 12 months
functional capacity
6-minute walk test, standard deviation score
Time frame: 12 months
incidence of dental abcesses
dental examination
Time frame: 12 months
incidence of hearing problems
ORL examination, audiogramm
Time frame: 12 months
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incidence of neurological problems (craniosynostosis, Chiari I malformation)
neurosurgical examination and brain MRI
Time frame: 12 months
incidence of nephrocalcinosis
renal ultrasound
Time frame: 12 months
incidence of hyperparathyroidism
blood levels of parathyroid hormone
Time frame: 12 months
incidence of any side effects
registration of any side effects during the treatment by telephone call
Time frame: 12 months