Hematopoietic Stem Cell Transplantation Outcomes in Diamond–Blackfan Anemia Patients Based on Myeloablative Conditioning Regimen With or Without Total Body Irradiation: A Systematic Review and Meta-Analysis

Abstract

Diamond–Blackfan anemia (DBA) is a rare, congenital bone marrow failure syndrome characterized by hypoplastic anemia. Earliest descriptions of this disease date back to 1936, and since then, a plethora of treatment strategies have been used to control or treat the disease. In recent decades, hematopoietic stem cell transplantation (HSCT) has been declared the only curative treatment. Despite the time elapsing from the first time HSCT has been used in this setting, no unified standard preparative and prophylactic protocol has been established. In this article, for the first time, the published articles concerning the efficacy of the most verified conditioning regimens established for these patients, the myeloablative conditioning regimen (MAC), were systematically reviewed. A comparison of two groups, based on the presence or absence of radiation in their protocol, was performed. Electronic and manual searches were conducted on PubMed, Scopus, and Web of Science. The primary study domains, selection, and outcome were assessed using the JBI Scale quality assessment for cohort and case series studies. Cohorts were categorized into treatment groups, and the characteristics of patients and donors, in addition to intervention characteristics and outcomes, were synthesized. Among a total of 196 studies reviewed, we included five cohorts in our systematic review. The studies were heterogeneous in various aspects. In conclusion, our analysis suggests that DBA patients who underwent a MAC non-total body irradiation (TBI) conditioning regimen may experience better post-HSCT outcomes; however, the findings are inconclusive.