Bone marrow transplantation for transfused patients with severe aplastic anemia using cyclophosphamide and total lymphoid irradiation as conditioning therapy : long-term follow-up from a single center

• Published in: Bone marrow transplantation (Basingstoke) Vol. 24; no. 3; pp. 253 - 257
• Main Authors: GAZIEV, D, GIARDINI, C, GALIMBERTI, M, LUCARELLI, G, ANGELUCCI, E, POLCHI, P, BARONCIANI, D, ERER, B, SOTTI, G
• Format: Journal Article
• Language: English
• Published: Basingstoke Nature Publishing Group 01.08.1999
• Subjects: Adolescent; Adult; Anemia; Anemia, Aplastic - mortality; Anemia, Aplastic - therapy; Anesthesia. Intensive care medicine. Transfusions. Cell therapy and gene therapy; Aplastic anemia; Biological and medical sciences; Bone marrow; Bone Marrow Transplantation; Bone marrow, stem cells transplantation. Graft versus host reaction; Child; Child, Preschool; Conditioning; Cyclophosphamide; Cyclophosphamide - therapeutic use; Female; Follow-Up Studies; Graft rejection; Graft Survival; Graft versus host disease; Graft vs Host Disease - etiology; Graft-versus-host reaction; Histocompatibility antigen HLA; Humans; Irradiation; Lymphatic Irradiation; Male; Malignancy; Medical sciences; Methotrexate; Methylprednisolone; Middle Aged; Neoplasms; Patients; Prophylaxis; Radiation; Retrospective Studies; Stem cell transplantation; Survival; Transfusions. Complications. Transfusion reactions. Cell and gene therapy; Transplantation; Transplantation Conditioning; Human; HLA-System; Major histocompatibility system; Aplastic anemia; Related donor; Homograft; Hemopathy; Lymphoid tissue; Chemotherapy; Cyclophosphamide; Oxazaphosphinane derivatives; Treatment; Follow up study; Nitrogen mustard; Irradiation; Bone marrow; Graft; Immunosuppressive agent; Pretreatment; Histocompatibility
• ISSN: 0268-3369; 1476-5365
• DOI: 10.1038/sj.bmt.1701887

In transfused patients with aplastic anemia, incidence of graft rejection remains significant. Seventeen transfused patients with severe aplastic anemia received BMT from HLA-identical sibling donors after conditioning with cyclophosphamide (CY, 50 mg/kg/day for 4 days) plus total lymphoid irradiation (TLI, 750 cGy in a single dose). For graft-versus-host disease (GVHD) prophylaxis one patient received methotrexate, five patients received CsA and 11 received CsA in association with methylprednisolone. All patients had sustained engraftment. The actuarial survival of patients was 76% with a median follow-up for surviving patients of 11 years (range 0.3-14.5 years). The incidence of grade II-III acute GVHD was 24%, and chronic GVHD 35%. Median Karnofsky score of surviving patients is 100 (range 90-100). Only one patient developed interstitial pneumonia. None of the patients has developed a malignancy after BMT. The role of limited field irradiation in development of malignant neoplasms after BMT for aplastic anemia is discussed. We conclude that a conditioning regimen using CY + TLI in sensitized aplastic anemia patients results in a high survival rate on long-term follow-up.