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.