Protection against chemotherapy induced mucositis by TGF-beta(2) in childhood cancer patients: results from a randomized cross-over study

Pediatr Blood Cancer. 2007 May;48(5):532-9. doi: 10.1002/pbc.20910.


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-beta(2)-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-beta(2)-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-beta(2) was safe as serum TGF-beta(2) did not increase, and renal and liver function were not affected during TGF-beta(2) 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-beta(2) enriched and normal feeding.

Conclusions: TGF-beta(2) administration via feeding is well tolerated and safe. Although this study might have had limitations to show potential benefit of TGF-beta(2), it does not provide evidence that TGF-beta(2) decreases the incidence or degree of mucositis induced by combination chemotherapy in childhood-cancer patients.

Publication types

  • Randomized Controlled Trial
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Antineoplastic Agents / adverse effects
  • Child
  • Child, Preschool
  • Cross-Over Studies
  • Double-Blind Method
  • Female
  • Food
  • Gastrointestinal Diseases / chemically induced
  • Gastrointestinal Diseases / prevention & control
  • Humans
  • Infant
  • Male
  • Mouth Diseases / chemically induced
  • Mouth Diseases / prevention & control
  • Mouth Mucosa
  • Mucositis / chemically induced*
  • Mucositis / prevention & control*
  • Neoplasms / drug therapy
  • Transforming Growth Factor beta2 / administration & dosage*
  • Transforming Growth Factor beta2 / blood


  • Antineoplastic Agents
  • Transforming Growth Factor beta2