Clinical Report: Impact of Burosumab and Conventional Therapy on Pediatric XLH Outcomes
Overview
This review evaluates the efficacy and safety of burosumab compared to conventional therapy or no treatment in children with X-linked hypophosphatemia (XLH). Burosumab likely prevents lower limb deformities and improves physical health quality of life but may increase adverse events. Evidence on conventional therapy versus no treatment remains limited and uncertain.
Background
X-linked hypophosphatemia (XLH) is a rare genetic disorder caused by PHEX gene mutations leading to elevated FGF23 levels, resulting in renal phosphate wasting and impaired vitamin D metabolism. Children with XLH commonly present with rickets, bone deformities, and dental issues. Conventional treatment involves phosphate salts and active vitamin D, while burosumab, a monoclonal antibody targeting FGF23, represents a newer therapeutic option. Early diagnosis and treatment are critical to optimizing growth and preventing complications.
Data Highlights
Outcome
Burosumab vs Conventional Therapy
Certainty of Evidence
Lower limb deformity prevention
Burosumab probably prevents deformities
Moderate
Physical health quality of life
Improved with burosumab
Moderate
Height increase
Burosumab may increase height
Low
Symptom burden of hypophosphatemia
Burosumab may enhance symptom burden
Low
Treatment-emergent adverse events
Probably increased with burosumab
Moderate
Dental abscesses
May be increased with burosumab
Low
Conventional therapy vs no treatment on final height
Very low certainty evidence, impact unclear
Very Low
Key Findings
Burosumab probably prevents lower limb deformities in children with XLH (moderate certainty).
Burosumab improves physical health-related quality of life compared to conventional therapy (moderate certainty).
Burosumab may increase final height and reduce symptom burden related to chronic hypophosphatemia (low certainty).
Burosumab probably increases treatment-emergent adverse events and may increase dental abscesses (moderate to low certainty).
Evidence comparing conventional therapy to no treatment is limited and of very low certainty, particularly regarding effects on final height.
Further research is needed to clarify long-term outcomes of medical therapies in pediatric XLH.
Clinical Implications
Burosumab offers a promising treatment option for pediatric XLH by reducing skeletal deformities and improving quality of life, but clinicians should monitor for increased adverse events including dental complications. Conventional therapy remains standard but lacks robust evidence for long-term benefits compared to no treatment. Individualized treatment decisions should consider potential benefits and risks, with ongoing assessment of growth and symptom control.
Conclusion
Burosumab likely provides meaningful clinical benefits in children with XLH by preventing deformities and enhancing quality of life, albeit with an increased risk of adverse events. Current evidence on conventional therapy is insufficient, underscoring the need for further high-quality studies to inform optimal management strategies.
References
Comprehensive Review 2023 -- Impact of Medical Interventions on Pediatric Outcomes in 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
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