Protection against chemotherapy induced mucositis by TGF-β₂ in childhood cancer patients: Results from a randomized cross-over study

Background. Mucositis is one of the most frequent and severe side-effect of chemotherapy in childhood-cancer patients for which there is no prophylaxis available. The efficacy and feasibility of a TGF-β₂-enriched feeding for preventing oral and gastro-intestinal-mucositis in childhood-cancer patients were studied. Procedure. The study was designed as a two-period cross-over, randomized, double-blinded, placebo, controlled trial. Patients who had a high risk for developing mucositis and who would receive two comparable cycles of chemotherapy were eligible for the study. During one cycle of chemotherapy, TGF-β₂-enriched feeding was administered; during the other, a 'placebo' (not enriched) feeding was used. WHO toxicity scales of diarrhea, oral mucositis, fever, anal lesions and nausea/vomiting were scored daily. In addition, the incidence of occurrence of blood cultures, antibiotic therapy, and interventions or diagnostics related to mucositis were measured. Results. The feasibility of the study was good: 83% of the patients completed two cycles and 86% of the study-feeding was effectively consumed. Administration of TGF-β₂ was safe as serum TGF-β₂ did not increase, and renal and liver function were not affected during TGF-β₂ consumption compared to normal feeding. Differences in toxicity, scored during the whole observation period and the number of days with WHO 3/4 toxicity, were not significantly different between cycles with TGF-β₂ enriched and normal feeding. Conclusions. TGF-β₂ administration via feeding is well tolerated and safe. Although this study might have had limitations to show potential benefit of TGF-β₂, it does not provide evidence that TGF-β₂ decreases the incidence or degree of mucositis induced by combination chemotherapy in childhood-cancer patients.