systemic chemotherapy + antibodies-St. Jude Children's Research Hospital-malignant infantile osteopetrosis

This refers to a specific multi-agent conditioning regimen utilized by St. Jude Children's Research Hospital in clinical trials for Malignant Infantile Osteopetrosis (MIOP). The regimen is designed for patients undergoing allogeneic hematopoietic stem cell transplantation (HSCT), particularly those receiving haploidentical (partially matched) donor cells. It combines systemic chemotherapy agents (fludarabine, thiotepa, and melphalan) with monoclonal antibodies (muromonab-CD3/OKT-3 for T-cell depletion and rituximab for B-cell depletion). The goal of this reduced-intensity conditioning (RIC) protocol is to achieve stable donor engraftment while minimizing the severe regimen-related toxicities often seen with myeloablative conditioning in MIOP patients. Cyclosporine is typically administered alongside this regimen for graft-versus-host disease (GVHD) prophylaxis.

Other names

OKT-3 + fludarabine + thiotepa + melphalan + rituximab + cyclosporineSt. Jude MIOP RIC regimenReduced intensity conditioning for MIOP