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. 2015 May;6(3):449-58.
doi: 10.1016/j.jare.2014.11.001. Epub 2014 Nov 7.

Allogeneic hematopoietic stem cell transplantation for non-malignant hematological disorders

Hossam K Mahmoud  1 , Alaa M Elhaddad  2 , Omar A Fahmy  3 , Mohamed A Samra  1 , Raafat M Abdelfattah  1 , Yasser H El-Nahass  4 , Gamal M Fathy  5 , Mohamed S Abdelhady  5
Affiliations

Allogeneic hematopoietic stem cell transplantation for non-malignant hematological disorders

Hossam K Mahmoud et al. J Adv Res. 2015 May.

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.

Keywords: B-thalassemia major; Fanconi’s anemia; Hematopoietic stem cell transplantation; Immunodeficiency diseases; Inherited metabolic disorders.

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Figures

Fig. 1
Fig. 1
Overall survival of severe aplastic anemia patients after allogeneic hematopoietic stem cell transplantation: Flu/Cy group compared with Cy/ATG group.
Fig. 2
Fig. 2
Overall survival of the whole group of B-thalassemia patients after allogeneic hematopoietic stem cell transplantation.
Fig. 3
Fig. 3
Disease-free survival of B-thalassemia in PBSC transplantation group compared with BM stem cell transplantation group.
Fig. 4
Fig. 4
Overall survival of Fanconi’s anemia patients after allogeneic hematopoietic stem cell transplantation.
Fig. 5
Fig. 5
Overall survival of patients with immunodeficiency diseases after allogeneic hematopoietic stem cell transplantation.
Fig. 6
Fig. 6
Overall survival of patients with inherited metabolic disorders after allogeneic hematopoietic stem cell transplantation.
None
See this image and copyright information in PMC

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