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Burosumab improved clinical and biochemical outcomes in patients with tumor-induced osteomalaciaCan a new drug help patients with tumor-induced bone loss before surgery?

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Key Takeaway
Consider burosumab for TIO management while awaiting surgery or if inoperable, noting small sample sizes and lack of statistical certainty.

This hybrid study, comprising a local clinical survey and a systematic review, assessed the efficacy of burosumab in patients affected by tumor-induced osteomalacia (TIO). The investigation included 10 patients from the local survey and 49 cases from the systematic review, sourced from Federico II University and literature databases including Medline and the Cochrane Library. The intervention involved burosumab administration, compared against no treatment.

Regarding primary outcomes, clinical and biochemical improvement was observed in 2 of 2 patients receiving burosumab. In the comparator group, death was observed in 2 of 8 patients not receiving treatment. Secondary outcomes included safety, tolerability, normalization of phosphate serum levels, and reduction of clinical symptoms. The study did not report specific adverse events, serious adverse events, discontinuations, or statistical measures such as p-values or confidence intervals.

Safety and tolerability were described as well tolerated, though specific adverse event data were not reported. The study lacked reported follow-up duration and formal causality assessments. Key limitations include the small sample size of the local survey and the absence of statistical reporting for the primary efficacy outcome.

The practice relevance suggests burosumab may be administered in cases of tumor identification while awaiting surgery or when surgical inoperability exists to manage hormonal FGF23 effects. Clinicians should interpret these results with caution due to the observational nature of the data and the lack of rigorous statistical analysis.

Imagine waking up with bones that feel too weak to hold you up. This is the reality for people with tumor-induced osteomalacia, a condition where a tumor disrupts the body's ability to keep minerals in the blood. Without treatment, these patients struggle with debilitating symptoms. A recent hybrid study looked at whether a specific medicine called burosumab could help them before or instead of surgery.

The researchers combined data from a local survey and a systematic review. They found that among the very few patients who received burosumab, two out of two showed clear improvement in their health and blood chemistry. In contrast, among those who did not receive the drug, two out of eight experienced death. The study noted that the drug was well tolerated, with no reported serious side effects or reasons for stopping the treatment.

However, the numbers are small. The local survey involved only ten patients, and the broader review looked at forty-nine cases total. Because the group is so tiny, we cannot yet say for sure if this drug works for everyone or if the results will hold up in larger groups. The study suggests the drug might be useful while waiting for surgery or if surgery is not an option, but more evidence is needed to be certain.

What this means for you:
Small study shows drug helped two patients, but results need more proof.

Study Details

Study typeMeta analysis
EvidenceLevel 1
PublishedApr 2026
View Original Abstract ↓
IntroductionBurosumab, a recombinant antibody against fibroblast growth factor 23 (FGF23), is a new therapeutical option for Tumor induced osteomalacia (TIO). To estimate the clinical need and to evaluate the efficacy and safety of burosumab, we conducted a hybrid study composed by a clinical survey and a systematic review.MethodsAll patients referring for TIO to the Federico II University were enrolled in our clinical survey. A comprehensive literature search on a Medline, Google Scholar, Google Books, and the Cochrane Library was conducted for the Systematic Review. Data were extracted on 12/31/2025.ResultsFor the local survey, we collected 10 patients affected by TIO [M: F = 5 (50.0%): 5 (50.0%); mean age at symptom onset 57.6 ± 14.3 years]. Two (20.0%) patients received burosumab, obtaining clinical and biochemical improvement. Two who didn’t receive the treatment died. For the systematic review, we collected 42 studies and 49 cases [M: F = 19 (44.2%): 24 (55.8%); mean age at onset 36.8 ± 21.3 years; mean age at diagnosis 44.5 ± 22.9 years]. Burosumab reduced clinical symptoms and ameliorates biochemical investigations by normalizing phosphate serum levels. It is also safe and well tolerated.ConclusionBurosumab represents a safe and efficient treatment in TIO patients, to be administered in case of tumor identification while waiting for surgery, and in case of surgical inoperability to manage hormonal FGF23 effect.
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