A pilot study combining primary busulfan-based haploidentical stem cell transplantation with GD2 antibody to treat high-risk neuroblastoma - PubMed
4 hours ago
- #Neuroblastoma
- #GD2 Antibody
- #Haploidentical Stem Cell Transplantation
- High-risk neuroblastoma with poor prognosis (event-free survival <20%) was targeted in a pilot study.
- A combination of busulfan-based haploidentical stem cell transplantation (haplo-SCT) and GD2 antibody (dinutuximab beta) therapy was tested.
- The approach aimed to utilize graft-versus-tumor effects and enhanced antibody-dependent cellular cytotoxicity.
- Five patients underwent conditioning with busulfan, melphalan, fludarabine, and antithymocyte globulin before T/B-cell-depleted haplo-SCT, followed by six cycles of dinutuximab beta.
- All patients achieved successful engraftment; three remained alive in first complete remission for up to 7.3 years post-transplant.
- Two patients experienced adverse events: one relapse and one non-relapse death.
- Early results indicate feasibility and potential survival benefits, warranting further controlled trials.