Positive Response to One-Year Treatment With Burosumab in Pediatric Patients With X-Linked Hypophosphatemia
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 a...
| Main Authors: | , , , , |
|---|---|
| Format: | Article |
| Language: | English |
| Published: |
Frontiers Media S.A.
2020-02-01
|
| Series: | Frontiers in Pediatrics |
| Subjects: | |
| Online Access: | https://www.frontiersin.org/article/10.3389/fped.2020.00048/full |
| _version_ | 1811212810312482816 |
|---|---|
| author | Silvia Martín Ramos Marta Gil-Calvo Virginia Roldán Ana Castellano Martínez Fernando Santos Fernando Santos Fernando Santos |
| author_facet | Silvia Martín Ramos Marta Gil-Calvo Virginia Roldán Ana Castellano Martínez Fernando Santos Fernando Santos Fernando Santos |
| author_sort | Silvia Martín Ramos |
| collection | DOAJ |
| description | 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. |
| first_indexed | 2024-04-12T05:35:29Z |
| format | Article |
| id | doaj.art-5160c395c6f449eb81d2ae7bf346db49 |
| institution | Directory Open Access Journal |
| issn | 2296-2360 |
| language | English |
| last_indexed | 2024-04-12T05:35:29Z |
| publishDate | 2020-02-01 |
| publisher | Frontiers Media S.A. |
| record_format | Article |
| series | Frontiers in Pediatrics |
| spelling | doaj.art-5160c395c6f449eb81d2ae7bf346db492022-12-22T03:45:53ZengFrontiers Media S.A.Frontiers in Pediatrics2296-23602020-02-01810.3389/fped.2020.00048503914Positive Response to One-Year Treatment With Burosumab in Pediatric Patients With X-Linked HypophosphatemiaSilvia Martín Ramos0Marta Gil-Calvo1Virginia Roldán2Ana Castellano Martínez3Fernando Santos4Fernando Santos5Fernando Santos6Hospital Universitario Central de Asturias, Oviedo, SpainHospital Clínico Universitario de Santiago, Santiago de Compostela, SpainHospital Universitario Puerta del Mar, Cádiz, SpainHospital Universitario Puerta del Mar, Cádiz, SpainHospital Universitario Central de Asturias, Oviedo, SpainInstituto de Investigación Sanitaria del Principado de Asturias, Oviedo, SpainUniversidad de Oviedo, Oviedo, SpainX-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.https://www.frontiersin.org/article/10.3389/fped.2020.00048/fullX-linked hypophosphatemia (XLH)ricketsburosumabFGF23hypophosphatemiavitamin D |
| spellingShingle | Silvia Martín Ramos Marta Gil-Calvo Virginia Roldán Ana Castellano Martínez Fernando Santos Fernando Santos Fernando Santos Positive Response to One-Year Treatment With Burosumab in Pediatric Patients With X-Linked Hypophosphatemia Frontiers in Pediatrics X-linked hypophosphatemia (XLH) rickets burosumab FGF23 hypophosphatemia vitamin D |
| title | Positive Response to One-Year Treatment With Burosumab in Pediatric Patients With X-Linked Hypophosphatemia |
| title_full | Positive Response to One-Year Treatment With Burosumab in Pediatric Patients With X-Linked Hypophosphatemia |
| title_fullStr | Positive Response to One-Year Treatment With Burosumab in Pediatric Patients With X-Linked Hypophosphatemia |
| title_full_unstemmed | Positive Response to One-Year Treatment With Burosumab in Pediatric Patients With X-Linked Hypophosphatemia |
| title_short | Positive Response to One-Year Treatment With Burosumab in Pediatric Patients With X-Linked Hypophosphatemia |
| title_sort | positive response to one year treatment with burosumab in pediatric patients with x linked hypophosphatemia |
| topic | X-linked hypophosphatemia (XLH) rickets burosumab FGF23 hypophosphatemia vitamin D |
| url | https://www.frontiersin.org/article/10.3389/fped.2020.00048/full |
| work_keys_str_mv | AT silviamartinramos positiveresponsetooneyeartreatmentwithburosumabinpediatricpatientswithxlinkedhypophosphatemia AT martagilcalvo positiveresponsetooneyeartreatmentwithburosumabinpediatricpatientswithxlinkedhypophosphatemia AT virginiaroldan positiveresponsetooneyeartreatmentwithburosumabinpediatricpatientswithxlinkedhypophosphatemia AT anacastellanomartinez positiveresponsetooneyeartreatmentwithburosumabinpediatricpatientswithxlinkedhypophosphatemia AT fernandosantos positiveresponsetooneyeartreatmentwithburosumabinpediatricpatientswithxlinkedhypophosphatemia AT fernandosantos positiveresponsetooneyeartreatmentwithburosumabinpediatricpatientswithxlinkedhypophosphatemia AT fernandosantos positiveresponsetooneyeartreatmentwithburosumabinpediatricpatientswithxlinkedhypophosphatemia |