Drug intelligence / Profile preview

Allogeneic Hematopoietic Stem Cell Transplantation (M.D. Anderson Regimen)

Development stage
Unknown
Modality
Stem Cell Therapies → Cell Therapies, Small Molecules, Antibody-Based Therapeutics
01

Overview

This therapeutic regimen involves allogeneic hematopoietic stem cell transplantation (HSCT) preceded by a multi-agent intensive conditioning protocol. Developed by the M.D. Anderson Cancer Center, it is specifically designed for pediatric and adolescent-young adult (AYA) patients with high-risk, recurrent, or refractory solid tumors, including Ewing sarcoma, neuroblastoma, and rhabdomyosarcoma. The conditioning phase utilizes chemotherapy agents such as fludarabine, thiotepa, etoposide, and melphalan to eradicate malignant cells and suppress the host immune system, alongside rabbit anti-thymocyte globulin (ATG) for T-cell depletion. Following the infusion of donor stem cells, immunosuppressants like tacrolimus, cyclosporine, and mycophenolate mofetil are employed to manage graft-versus-host disease (GVHD). The treatment aims to leverage the graft-versus-tumor effect to achieve long-term remission in aggressive solid malignancies.

Other names
Allogeneic HSCT for Pediatric and AYA High-Risk Solid Tumors

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