Allogeneic hematopoietic stem cell transplantation in 24 patients with β－thalassemia major

Liu Rongrong(劉容容，Depy Hematol，1st Hosp，Guangxi Med Univ，Nanning 530021)…∥Chin J Organ Transplant.－2011，32(3).－144 ～147

Allogeneic hematopoietic stem cell transplantation in 24 patients with β－thalassemia major

Liu Rongrong(劉容容，Depy Hematol，1st Hosp，Guangxi Med Univ，Nanning 530021)…∥Chin J Organ Transplant.－2011，32(3).－144 ～147

ObjectiveTo investigate the effect of allgeneic hematopoietic stem cell transplantation(allo－HSCT)for β － thalassemia major.MethodsTwenty － four β －thalassemia major patients with median age of 4 years(range:2－15 years)，18 boys and 6 girls，received allo－HSCT.They were classified into classⅡ －Ⅲ according to Pesaro thalassemia classification.Twentythree transplantations were from sibling donor and 1 was from mother，either HLA －identical(n=23)or HLA －mismatched(5/6)(n=1).Fifteen patients received bone marrow transplantation(BMT)plus peripheral blood stem cell transplantation(PBSCT)，and 9 were subjected to umbilical cord blood transplantation(UCBT).The conditioning regimen consisted of busalphan，cyclophosphamide，fludarabine，plus hydroxyurea before transplantation.Graft－versus－h(huán)ost disease(GVHD)prophylaxis included CsA，methotrexate，antilymphpcute globulin，and mycophenolate mofetil.The median follow－up period was 13 months(range:3 －69).ResultsOf 24 patients，there were 21 cases(87.5%)of disease－free survival，1(4.2%)transplantation － related death，and 2 cases(8.3%)of rejection.Three－year overall survival and disease－free survival rate was 91.7%and 87.5%，respectively.The cumulative incidence of gradeⅡ－Ⅳacute GVHD and chronic GVHD was 16.7 and 20.3%，particularly cumulative extensive chronic GVHD was 5.0%.ConclusionThe sibling donor BMT plus PBSCT is an effective and safe way to treat β － thalassemia major.Cord blood is an important source of hematopoietic stem cells for HSCT.The protocol GVHD prophylaxis of CsA，MTX，ATG with a low－dose and short course of MMF can effectively reduce the incidence of severe acute GVHD，improve the outcome of thalassemia transplantation.12 refs，1 fig，1 tab.

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