Iqbal, Madiha and Reljic, Tea and Corbacioglu, Selim and de la Fuente, Josu and Gluckman, Eliane and Kumar, Ambuj and Yassine, Farah and Ayala, Ernesto and El-Jawahri, Areej and Murthy, Hemant and Almohareb, Fahad and Hashmi, Shahrukh K. and Cappelli, Barbara and Alahmari, Ali and Scigliuolo, Graziana Maria and Kassim, Adetola and Aljurf, Mahmoud and Kharfan-Dabaja, Mohamed A. (2021) Systematic Review/Meta-Analysis on Efficacy of Allogeneic Hematopoietic Cell Transplantation in Sickle Cell Disease: An International Effort on Behalf of the Pediatric Diseases Working Party of European Society for Blood and Marrow Transplantation and the Sickle Cell Transplantation International Consortium. TRANSPLANTATION AND CELLULAR THERAPY, 27 (2). 167.e1-167.e12. ISSN 2666-6375, 2666-6367
Full text not available from this repository. (Request a copy)Abstract
Sickle cell disease (SCD) affects more than 300,000 children annually worldwide. Despite improved supportive care, long-term prognosis remains poor. Allogeneic hematopoietic cell transplantation (allo-HCT) is the sole validated curative option, resulting in sustained resolution of the clinical phenotype. The medical literature on allo-HCT for SCD is largely limited to children. Recent studies have evaluated allo-HCT efficacy in adults. Here, we conducted a systematic review/meta-analysis to assess the totality of evidence on the efficacy, or lack thereof, of allo-HCT in treating SCD. We performed a comprehensive literature search using PubMed/Medline, Embase, and Cochrane library databases on November 13, 2019. Four authors independently extracted data on clinical outcomes related to benefits (overall survival [OS] and disease-free survival [DFS]) and harms (acute graft-versus-host disease [aGVHD], chronic graft-versushost disease [cGVHD], nonrelapse mortality [NRM], and graft failure [GF]). Our search identified a total of 1906 references. Only 33 studies (n= 2853 patients) met our inclusion criteria. We also performed a subset analysis by age. Analyses of all-age groups showed pooled rates of 96% for OS, 90% for DFS, 20% for aGVHD, 10% for cGVHD, 4% for NRM, and 5% for GF. In the pediatric population, pooled rates for OS, DFS, aGVHD, cGVHD, NRM, and GF were 97%, 91%, 26%, 11%, 5%, and 3%, respectively. In adults, pooled rates for OS, DFS, aGVHD, cGVHD, NRM, and GF were 98%, 90%, 7%, 1%, 0%, and 14%, respectively. Our data show that allo-HCT is safe and effective, yielding pooled OS rates exceeding 90%. The high GF rate of 14% in adults is concerning and emphasizes the need to evaluate new strategies. (C) 2020 The American Society for Transplantation and Cellular Therapy. Published by Elsevier Inc. All rights reserved.
| Item Type: | Article |
|---|---|
| Uncontrolled Keywords: | MATCHED RELATED DONORS; EVENT-FREE SURVIVAL; REDUCED INTENSITY; CHILDREN; ANEMIA; METAANALYSIS; OUTCOMES; THERAPY; STROKE; RISK; Sickle cell disease; Allogeneic hematopoietic cell transplantation; Graft failure; Overall survival |
| Subjects: | 600 Technology > 610 Medical sciences Medicine |
| Divisions: | Medicine > Abteilung für Pädiatrische Hämatologie, Onkologie und Stammzelltransplantation |
| Depositing User: | Petra Gürster |
| Date Deposited: | 20 Dec 2022 10:49 |
| Last Modified: | 20 Dec 2022 10:49 |
| URI: | https://pred.uni-regensburg.de/id/eprint/47935 |
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