Treatment of Rickets in Tumor-Induced Osteomalacia When the FGF23-Producing Tumor Is Unresectable or Cannot Be Located

Tumor-induced osteomalacia (TIO) causes rickets through excess FGF23 secreted by a causative tumor. When that tumor cannot be located, cannot be completely removed, or when severe comorbidities preclude surgery, sustained medical therapy becomes the primary treatment strategy.

Clinical scenario: Rickets in the setting of tumor-induced osteomalacia where the FGF23-producing tumor is unresectable or unlocatable, or where severe comorbidities make surgical intervention unsafe. In this population, medical management is indicated as the definitive approach rather than curative resection.

Treatment approach

Management in this setting centres on oral phosphate supplementation combined with an active vitamin D analogue. A targeted biologic agent, approved for eligible paediatric patients based on clinical trials in both children and adults, is also established as an effective alternative. The complete regimen — including agent selection, dosing algorithm, monitoring schedule, and escalation criteria — is available in the structured protocol.

Clinical goals

Serum phosphate within age-related normal range Improvement of skeletal mineralisation Amelioration of clinical symptoms

Instant Access to Structured Evidence-Based Regimens

References

DOI: 10.1007/s00467-022-05505-5

This treatment is also indicated in cases where the FGF23 producing tumor cannot be located, complete tumor removal is not possible or in patients with severe comorbidities.

As an alternative to conventional treatment, patients can also be treated very effectively with burosumab which was recently approved for treatment of TIO in children and adults by the FDA and the Japanese health authorities based on clinical trials in adult patients.

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