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Status quo of allogeneic stem cell transplantation for patients with sickle cell disease using matched unrelated
1Department of Pediatric Oncology & Hematology, Charité University Medicine, Berlin, Germany.
This mini review is based on an oral presentation reflecting the current status quo of allogeneic hematopoietic stem cell transplantation (HSCT) for patients with sickle cell disease (SCD) using matched unrelated donors (MUDs) presented at the EBMT Sickle Disease Meeting held in Regensburg, Germany, in May 2019. Although the clinical trial landscape for MUD HSCT in patients with SCD is limited to date, some attempts to improve patient outcome in terms of overall survival and event-free survival have been made recently, including optimization of conditioning regimens and prevention of engraftment failure as well as graft-versus-host disease. The results achieved by these approaches are summarized in this review and are still unsatisfactory. Whether new haploidentical transplantation protocols will achieve superior results and are able to replace MUD HSCT for patients with SCD remains to be elucidated.
This mini review is based on an oral presentation reflecting the current status quo of allogeneic hematopoietic stem cell transplantation (HSCT) for patients with sickle cell disease (SCD) using matched unrelated donors (MUDs) presented at the EBMT Sickle Disease Meeting held in Regensburg, Germany, in May 2019. Although the clinical trial landscape for MUD HSCT in patients with SCD is limited to date, some attempts to improve patient outcome in terms of overall survival and event-free survival have been made recently, including optimization of conditioning regimens and prevention of engraftment failure as well as graft-versus-host disease. The results achieved by these approaches are summarized in this review and are still unsatisfactory. Whether new haploidentical transplantation protocols will achieve superior results and are able to replace MUD HSCT for patients with SCD remains to be elucidated.
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