Rickets in Tumor-Induced Osteomalacia: Treatment When a Resectable FGF23-Producing Tumor Is Identified

This protocol targets rickets and osteomalacia caused by a tumor that secretes excess FGF23, in cases where the tumor has been localized and is surgically removable. The clinical approach spans a pre-operative medical phase and definitive surgical treatment.

Clinical Scenario

Tumor-induced osteomalacia (TIO) with a confirmed, resectable FGF23-producing tumor. Complete tumor resection is the treatment of choice in both pediatric and adult TIO patients. Until surgery is undertaken, medical therapy is started to improve skeletal mineralization and relieve clinical symptoms.

Treatment Approach (Partial Overview)

Management combines phosphate supplementation and active vitamin D therapy as a medical bridge prior to surgery, followed by complete surgical removal of the FGF23-producing tumor. The full structured regimen — specific agents, dosing, intervals, and sequencing — is available in the complete protocol.

Treatment Goals

Normalization of serum FGF23 within 1 hour following tumor resection
Normalization of serum phosphate within 5 days post-resection
Complete bone healing within 12 months

Instant Access to Structured Evidence-Based Regimens

References

DOI: 10.1007/s00467-022-05505-5

Complete tumor resection is the treatment of choice in pediatric and adult TIO patients.

Until surgery, patients should be started on treatment with phosphate salts and active vitamin D, which was shown to improve skeletal mineralization and ameliorate clinical symptoms.

Serum FGF23 and phosphate levels were shown to normalize after tumor removal within 1 h and 5 days, respectively, but complete bone healing may take up to 12 months.

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