Conclusion
Our single institutional case series experience with bortezomib for SR and SD GVHD after pediatric HSCT, serves as proof of principle for tolerability, potential efficacy and development of future clinical trials. Among dose-limiting toxicities, pancytopenia was observed in almost all patients to varying degrees. No patients experienced infections or prolonged complications, and all had resolution of pancytopenia following dose reduction of bortezomib. Most patients tolerated bortezomib well, without serious adverse events. One patient experience peripheral neuropathy.
Though limited by sample size and single-center non-controlled anecdotal experience, we believe that this series is supportive of the possibility that bortezomib’s use in pediatric patients could be effective as second-line, steroid-weaning therapy in SD or SR GVHD. Bortezomib appears to be more effective in skin GVHD, and some response with liver GVHD. Our goal in the future is to create a clinical trial to evaluate the tolerability and efficacy of bortezomib in SR and SD GVHD and to standardize a dosing regimen for pediatric patients.