title: Allogeneic hematopoietic stem cell transplantation for non-malignant hematological disorders.
abstract: Allogeneic hematopoietic stem cell transplantation (allo-HSCT) from a geno-identical matched sibling (MSD) is one of the most successful therapies in patients with non-malignant hematological disorders. This study included 273 patients with severe aplastic anemia (SAA), 152 patients with B-Thalassemia major (BTM), 31 patients with Fanconi's anemia (FA), 20 patients with congenital immunodeficiency diseases (ID), and 13 patients with inherited metabolic disorders (IMD) allografted from a MSD. In SAA, the 8-year overall survival (OS) of the whole group patients was 74%. OS was significantly better in patients conditioned with fludarabine and cyclophosphamide (Flu/Cy) than in those who received cyclophosphamide and antithymocyte globulin (Cy/ATG) (p = 0.021). Acute graft-versus-host disease (aGVHD) grade II-IV occurred in 15% while chronic GVHD (cGVHD) occurred in 28%. In BTM, the 12-year disease-free survival (DFS) of the whole group of BTM patients was 72.4%. DFS was 74% for peripheral blood stem cell (PBSC) group compared to 64% in the BM stem cell group. The incidence of graft rejection was significantly lower in patients who received PBSC than in those who received BM (9% vs 25%) (p = 0.036). AGVHD grade II-IV and cGVHD occurred in 15% and 12% of the whole group of BTM patients respectively. In FA, the 5-year OS was 64.5%. Graft rejection occurred in 10% of patients. Grade II-IV aGVHD occurred in 16% while cGVHD occurred in 4%. In ID, the 5-year OS was 62%. Graft rejection occurred in two (10%) patients. Three patients (15%) developed grade II-IV aGVHD, 2 of them progressed to secondary cGVHD. In IMD, OS was 46% at 5 years. Graft rejection occurred in 8% of patients. AGVHD grade II-IV occurred in 15% while cGVHD occurred in 14%. In conclusion, Allo-HSCT provides a higher DFS rate over conventional therapies for patients with non-malignant hematological disorders with prolonged survival.
authors.affiliation: Department of Medical Oncology, National Cancer Institute, Cairo University, Egypt.
authors.forename: Hossam K
authors.initials: HK
authors.lastname: Mahmoud
authors.affiliation: Department of Pediatric Oncology, National Cancer Institute, Cairo University, Egypt.
authors.forename: Alaa M
authors.initials: AM
authors.lastname: Elhaddad
authors.affiliation: Department of Internal Medicine, Faculty of Medicine, Cairo University, Egypt.
authors.forename: Omar A
authors.initials: OA
authors.lastname: Fahmy
authors.affiliation: Department of Medical Oncology, National Cancer Institute, Cairo University, Egypt.
authors.forename: Mohamed A
authors.initials: MA
authors.lastname: Samra
authors.affiliation: Department of Medical Oncology, National Cancer Institute, Cairo University, Egypt.
authors.forename: Raafat M
authors.initials: RM
authors.lastname: Abdelfattah
authors.affiliation: Department of Clinical Pathology, National Cancer Institute, Cairo University, Egypt.
authors.forename: Yasser H
authors.initials: YH
authors.lastname: El-Nahass
authors.affiliation: Department of Hematology and BMT, Nasser Institute for Research and Treatment, Ministry of Health, Egypt.
authors.forename: Gamal M
authors.initials: GM
authors.lastname: Fathy
authors.affiliation: Department of Hematology and BMT, Nasser Institute for Research and Treatment, Ministry of Health, Egypt.
authors.forename: Mohamed S
authors.initials: MS
authors.lastname: Abdelhady
keywords: B-thalassemia major; Fanconi’s anemia; Hematopoietic stem cell transplantation; Immunodeficiency diseases; Inherited metabolic disorders