pentostatin + cyclophosphamide + busulfan

This entry refers to a specific multi-drug conditioning and graft-versus-host disease (GVHD) prophylaxis regimen developed by the National Cancer Institute (NCI) for patients with primary immunodeficiencies undergoing allogeneic blood or marrow transplantation (Allo BMT). The regimen is evaluated in a Phase 2 pilot trial (NCT03547258) and utilizes varying intensities of conditioning to facilitate donor stem cell engraftment. The primary conditioning agents include **pentostatin** (a purine antimetabolite that inhibits adenosine deaminase), **cyclophosphamide** (an alkylating agent), and **busulfan** (a cell-cycle non-specific alkylating agent). The protocol also incorporates post-transplantation cyclophosphamide (PTCy), **sirolimus** (an mTOR inhibitor), and **mycophenolate mofetil** (an IMPDH inhibitor) for GVHD prophylaxis. This approach aims to provide a safe and effective transplant platform for a broad age range (4-75 years) of patients with diverse primary immune system disorders.

Other names

NCI conditioning regimen for primary immunodeficiencyimmunosuppression-National Cancer Institute (NCI)-primary immunodeficiency