Systematic Review: Efficacy of Medical Therapy on Outcomes Important to Pediatric Patients With X-Linked Hypophosphatemia - Summary - MDSpire

Systematic Review: Efficacy of Medical Therapy on Outcomes Important to Pediatric Patients With X-Linked Hypophosphatemia

  • By

  • Dalal S Ali

  • Reza D Mirza

  • Salma Hussein

  • Farah Alsarraf

  • R Todd Alexander

  • Hajar Abu Alrob

  • Natasha M Appelman-Dijkstra

  • Martin Biosse-Duplan

  • Maria Luisa Brandi

  • Thomas O Carpenter

  • Catherine Chaussain

  • Karel Dandurand

  • Guido Filler

  • Pablo Florenzano

  • Seiji Fukumoto

  • Corinna Grasemann

  • Erik A Imel

  • Suzanne M Jan de Beur

  • Emmett Morgante

  • Leanne M Ward

  • Aliya A Khan

  • Gordon Guyatt

  • January 9, 2025

  • 0 min

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Objective:

To examine the evidence addressing the management of X-linked hypophosphatemia (XLH) in children to inform treatment recommendations, particularly regarding the efficacy and safety of burosumab compared to conventional therapies.

Key Findings:
  • Burosumab likely prevents lower limb deformity and improves physical health quality of life (QoL) with moderate certainty.
  • Burosumab may increase height and enhance the burden of symptoms related to chronic hypophosphatemia with low certainty.
  • Burosumab probably increases treatment-emergent adverse events (moderate certainty) and may increase dental abscesses (low certainty).
Interpretation:

Burosumab shows potential benefits for children with XLH, particularly in preventing deformities and improving QoL, but may also lead to increased adverse events, necessitating careful monitoring.

Limitations:
  • The open-label RCT was at high risk of bias, which may affect the reliability of the findings.
  • Evidence regarding the impact of conventional therapy compared to no treatment on final height was of very low certainty, limiting conclusions.
Conclusion:

Further research is required to understand the long-term effects of medical therapy in children with XLH, particularly studies that assess the durability of benefits and the safety profile of burosumab.

Original Source(s)

Systematic Review: Efficacy of Medical Therapy on Outcomes Important to Pediatric Patients With X-Linked Hypophosphatemia

  • by Dalal S Ali, Reza D Mirza, Salma Hussein, Farah Alsarraf, R Todd Alexander, Hajar Abu Alrob, Natasha M Appelman-Dijkstra, Martin Biosse-Duplan, Maria Luisa Brandi, Thomas O Carpenter, Catherine Chaussain, Karel Dandurand, Guido Filler, Pablo Florenzano, Seiji Fukumoto, Corinna Grasemann, Erik A Imel, Suzanne M Jan de Beur, Emmett Morgante, Leanne M Ward, Aliya A Khan, Gordon Guyatt

  • January 9, 2025

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