Allogeneic stem cell transplantation for severe acquired aplastic anaemia using a fludarabine‐based preparative regimen

• Published in: British journal of haematology Vol. 133; no. 6; pp. 649 - 654
• Main Authors: Resnick, Igor B., Aker, Memet, Shapira, Michael Y., Tsirigotis, Panagiotis D., Bitan, Menachem, Abdul‐Hai, Ali, Samuel, Simcha, Ackerstein, Aliza, Gesundheit, Benjamin, Zilberman, Irina, Miron, Svetlana, Yoffe, Luba, Lvovich, Alex, Slavin, Shimon, Or, Reuven
• Format: Journal Article
• Language: English
• Published: Oxford, UK Blackwell Publishing Ltd 01.06.2006; Blackwell
• Subjects: Adolescent; Adult; Anemia, Aplastic - therapy; Antilymphocyte Serum - therapeutic use; Biological and medical sciences; Child; Cyclophosphamide - therapeutic use; Female; fludarabine; Graft vs Host Disease - prevention & control; Hematologic and hematopoietic diseases; Hematology; Hematopoietic Stem Cell Transplantation - methods; Humans; Immunosuppressive Agents - adverse effects; Immunosuppressive Agents - therapeutic use; Leukemias. Malignant lymphomas. Malignant reticulosis. Myelofibrosis; Male; Medical sciences; Middle Aged; Mucositis - chemically induced; non‐myeloablative HSCT; Opportunistic Infections - etiology; Pilot Projects; Quality of Life; severe aplastic anaemia; Transplantation Chimera; Transplantation Conditioning - methods; Treatment Outcome; Vidarabine - analogs & derivatives; Vidarabine - therapeutic use; Antineoplastic agent; non-myeloablative HSCT; Purine nucleotide; Hematology; Acquired; Stem cell; Aplastic anemia; Hematopoietic cell; Homograft; Hemopathy; Non myeloablative treatment; Fludarabine; Antimetabolic; Bone marrow failure; severe aplastic anaemia; Graft; Fluorine Organic compounds
• ISSN: 0007-1048; 1365-2141
• DOI: 10.1111/j.1365-2141.2006.06084.x

Summary We reviewed our experience in the treatment of 13 patients with severe acquired aplastic anaemia, using a newly developed non‐myeloablative regimen consisting of fludarabine (total dose 180 mg/m2), cyclophosphamide (total dose 120 mg/kg), and antithymocyte globulin (total dose 40 mg/kg). All except one patient received multiple transfusions and had failed prior immunosuppressive treatment. Twelve out of 13 patients achieved sustained engraftment. One patient was not evaluable for engraftment because of early death on day +10. None of the patients developed graft failure. Mucositis of mild‐to‐moderate severity was the only observed regimen‐related toxicity. The cumulative incidence of acute graft‐versus‐host disease (GvHD) grade II–IV and III–IV was 8·3% and 0%, respectively. With a median follow‐up period of 45 months, the 5‐year overall survival probability was 84%. Eight out of 11 surviving patients have been followed for more than 1 year and only one developed limited chronic GvHD. All patients enjoy a normal life style, with a Karnofsky score of 100%, and all except three, followed for 3, 5 and 6 months respectively, are free of any immunosuppressive medication. The results of this study look promising, while prospective clinical trials may be required to confirm the benefits of this regimen as an alternative to existing protocols.