busulfan + cyclophosphamide + fludarabine + antithymocyte globulin

This conditioning regimen is a non-total body irradiation (non-TBI) protocol developed by Tehran University of Medical Sciences for the treatment of children with Mucopolysaccharidosis (MPS) types 1, 6, and 7 undergoing hematopoietic stem cell transplantation (HSCT). The regimen is a combination therapy that typically includes the DNA alkylating agents busulfan and cyclophosphamide as a core myeloablative backbone. Depending on the donor source, the regimen is supplemented with antithymocyte globulin (ATG) for unrelated or mismatched donors to enhance immunosuppression, or fludarabine for haploidentical transplants to facilitate engraftment. By avoiding radiation, the protocol aims to reduce the risk of long-term growth and endocrine complications in young patients while ensuring stable donor chimerism, which is necessary for the continuous production of the lysosomal enzymes deficient in MPS patients.

Other names

Conditioning regimen-Tehran University of Medical Sciences-mucopolysaccharidosis type 1-mucopolysaccharidosis type 6-mucopolysaccharidosis type 7Conditioning regimen (Tehran University of Medical Sciences)Busulfan/Cyclophosphamide/Fludarabine/ATG regimen