ABSTRACT
Objective@#To observe the efficacy and safety of intensive cladribine-based conditioning regimen for allogeneic hematopoietic stem cell transplantation (allo-HSCT) in children with major thalassemia.@*Methods@#Retrospective analysis was performed for the clinical data of 12 children with major thalassemia undergoing allo-HSCT from March 2017 to July 2018. All of them were diagnosed definitely and the median age at transplantation was 5 years (range: 2-13 years), including HSCT from HLA-matched unrelated donor (n=8), HLA8/10-matched unrelated donor (n=1), HLA-matched sibing donor (n=2) and haploidentical donor (n=1). They received a new intensive conditioning regimen of cyclophosphamide (CTX), cladribine, busulfan (Bu) and antithymocytic globulin. The median doses of mononuclear cell (MNC) and CD34 positive cell were 10.97×108/kg (range: 5.72-12.49×108/kg) and 12.2×106/kg (range: 6.7-22×106/kg). Graft-versus-host disease (GVHD) was prevented by cyclosporine A (CSA), methotrexate (MTX) and mycophenolate mofetil (MMF).@*Results@#Engraftment succeeded (n=11) and failed (n=1). The median time of neutrophil and platelet engraftment was 11 days (range: 8-17 days) and 13 days (range: 8-37 days) respectively. There were grade II acute GVHD (n=6) and grade IV intestinal acute GVHD (n=1) at 35 days post-transplantation. The latter one finally died of severe infection at 70 days post-transplantation. Two recipients of DLI developed limited chronic GVHD. Three cases (25%) developed cytomegaloviremia. None suffered from severe transplantation-related complications, such as cytomegalovirus diseases, hepatic veno-occlusive disease (HVOD), hemorrhagic cystitis or septicemia, etc. The median follow-up time was 15(8-18) months. Among 11 survivors, ten became transfusion-independent.@*Conclusions@#Cladribine-based conditioning regimen is both safe and effective for allo-HSCT in children with major thalassemia. However, vigorous immunosuppression may increase the risks of infection and viral activation after transplantation.
ABSTRACT
Objective To observe the efficacy and safety of intensive cladribine-based conditioning regimen for allogeneic hematopoietic stem cell transplantation (allo-HSCT ) in children with major thalassemia .Methods Retrospective analysis was performed for the clinical data of 12 children with major thalassemia undergoing allo-HSCT from March 2017 to July 2018 .All of them were diagnosed definitely and the median age at transplantation was 5 years (range:2-13 years ) , including HSCT from HLA-matched unrelated donor (n= 8 ) , HLA8/10-matched unrelated donor (n=1) ,HLA-matched sibing donor (n=2) and haploidentical donor (n=1) .They received a new intensive conditioning regimen of cyclophosphamide (CTX ) , cladribine , busulfan (Bu ) and antithymocytic globulin .The median doses of mononuclear cell (MNC) and CD34 positive cell were 10 .97 × 108/kg (range:5 .72-12 .49 × 108/kg) and 12 .2 × 106/kg (range:6 .7-22 × 106/kg) .Graft-versus-host disease (GVHD) was prevented by cyclosporine A (CSA) ,methotrexate (MTX) and mycophenolate mofetil (MMF) .Results Engraftment succeeded (n= 11) and failed (n= 1) .The median time of neutrophil and platelet engraftment was 11 days (range:8-17 days) and 13 days (range:8-37 days) respectively .There were grade II acute GVHD (n= 6) and grade IV intestinal acute GVHD (n=1) at 35 days post-transplantation .The latter one finally died of severe infection at 70 days post-transplantation .Two recipients of DLI developed limited chronic GVHD .Three cases ( 25％ ) developed cytomegaloviremia . None suffered from severe transplantation-related complications , such as cytomegalovirus diseases , hepatic veno-occlusive disease ( HVOD ) , hemorrhagic cystitis or septicemia ,etc .The median follow-up time was 15(8-18) months .Among 11 survivors ,ten became transfusion-independent .Conclusions Cladribine-based conditioning regimen is both safe and effective for allo-HSCT in children with major thalassemia . However , vigorous immunosuppression may increase the risks of infection and viral activation after transplantation .