G-CSF-Mobilized Blood and Bone Marrow Grafts as the Source of Stem Cells for HLA-Identical Sibling Transplantation in Patients with Thalassemia Major

Abstract

As an inherited anemia, thalassemia major (TM) is currently only curable with allogeneic hematopoietic stem cell transplantation (allo-HSCT). Here we report an allo-HSCT protocol for patients with TM who received a combination of granulocyte colony-stimulating factor-primed bone marrow and peripheral blood stem cells (G-BM & PBSCs) from a matched sibling donor (MSD). The conditioning regimen consisted of i.v. busulfan, cyclophosphamide, fludarabine, and antithymocyte globulin. Chimerism analysis was performed for all patients. Immunosuppressive treatment was terminated if rejection was suspected, and donor lymphocyte infusion was administered once no response was observed. A total of 184 patients with TM were enrolled in the study between July 2007 and July 2018. The cumulative incidence of grade II-IV acute graft-versus-host disease (GVHD) was 13.1%, and that of moderate or severe chronic GVHD was 5.7%. The cumulative incidence of graft rejection was .6%. In the total cohort, the 3-year overall survival, thalassemia-free survival, and GVHD-free, relapse-free survival were 97.8%, 97.3%, and 89.5%, respectively. Collectively, our results indicate that G-BM & PBSCs from an MSD is be a good stem cell source for patients with TM undergoing allo-HSCT.

The consensus from The Chinese Society of Hematology on indications, conditioning regimens and donor selection for allogeneic hematopoietic stem cell transplantation: 2021 update

“…The most commonly used regimen in China is the BuCyATG protocol, followed by T-replicate HID-HSCT (Table 6 ). Thalassemia major: Intensified conditioning regimens instead of standard conditioning regimens should generally be used in patients with thalassemia [ 57 , 85 ]. Fanconi anemia: The FluCy-ATG regimen (Flu 150 mg/m2, Cy 5–20 mg/kg/day × 4 days, and rabbit ATG 10 mg/kg) with or without low-dose TBI may be used for alternative donor transplantation.…”

“…Thalassemia major: Intensified conditioning regimens instead of standard conditioning regimens should generally be used in patients with thalassemia [ 57 , 85 ].…”