A Early Report of Bone Marrow Transplantation from HLA Mismatched Mother for β-Thalassemia Major.

Objective: To study the possibility of Bone Marrow Transplantation for β-thalassemia major using an alternative donor from HLA mismatch mother.

Methods: Three patients withβ-thalassemia major were given a BMT from HLA one-antigen mismatched mothers. The patient-1 was 7 year old, patient-2 was 1.5 and patient-3 was 4, respectively. All of patients were boys The oldest was class III in the Pesano grouping and others were classI-II. Patient-1 and patient-3 were given the conditioning of TBI 300 rad, Bu 14mg/kg, Cy 160mg/kg and ATG-F (patient-1 was 90mg/kg, patient-3 was given 30mg/kg). Patient-2 was given Fudalabine 150mg/kg instead of TBI and ATG-F 30mg/kg. Infused nuclear cells were 5.3x10e8/kg for patient-1, 6x10e8/kg for patient-2 and 19.4x10e8/kg for patient-3, respectively. Donor of patient-3 received G-CSF from day −2 to day 0. The prophylaxis of GVHD included CsA+methylpregnisolone but patient-3 received additionally MMF and anti-CD25 monoclonal antibody (Daclizumab).

Results: Time of follow-up was 4 years for patient-1, 3.2 years for patient-2 and 10 months for patient-3. Patient-1 and patient-3 are EFS and with only mild acute GVHD. Patient-2 was no engraftment.

Conclusions: We conclude that BMT from HLA one-antigen mismatched mother may be used for patients withβ-thalassemia major in some developing countries, such as China, in where the convention treatment of thalassemia was not well performed, whereas there are only three cases in our experiment.