Favorable outcome of non‐myeloablative allogeneic transplantation in adult patients with severe sickle cell disease: A single center experience of 200 patients

Allogeneic hematopoietic stem cell transplant (HSCT) for adults with severe sickle cell disease (SCD) is potentially curative but not commonly utilized therapy due to complications such as graft failure (GF) and organ toxicity. Herein, we are reporting our long‐term outcome data of non‐myeloablative...

Full description

Saved in:

Bibliographic Details
Published in:	American journal of hematology Vol. 99; no. 6; pp. 1023 - 1030
Main Authors:	Damlaj, Moussab, Alahmari, Bader, Alaskar, Ahmed, Alhejazi, Ayman, Alsadi, Husam, Ahmed, Mazin, Alanazi, Tahani, Ahmed, Rasha, Alharbi, Amani, Shehabeddine, Inaam, Alzaidi, Afnan, Alkhuraisat, Suha, Mahassnah, Isam, Alquraan, Hamza, Ballili, Maybelle, Alzahrani, Mohsen
Format:	Journal Article
Language:	English
Published:	Hoboken, USA John Wiley & Sons, Inc 01-06-2024 Wiley Subscription Services, Inc
Subjects:	ABO system Allografts Antigens CD34 antigen Graft rejection Hematopoietic stem cells Leukocytes (neutrophilic) Malignancy Rapamycin Sickle cell disease Stem cell transplantation Toxicity Transplants & implants
Online Access:	Get full text
Tags:	Add Tag No Tags, Be the first to tag this record!

Description
Summary:	Allogeneic hematopoietic stem cell transplant (HSCT) for adults with severe sickle cell disease (SCD) is potentially curative but not commonly utilized therapy due to complications such as graft failure (GF) and organ toxicity. Herein, we are reporting our long‐term outcome data of non‐myeloablative (NMA) HSCT in adults with severe SCD with emphasis on factors predicting event free survival (EFS). Adults with severe SCD undergoing NMA match‐related donor allogeneic HSCT from 2015 to 2021 with at least 12 months of follow‐up were included. A total of 200 patients were included with a median age of 26 years (14–43) and 56% were male. The median infused CD34 dose was 13.7 (5.07–25.8), respectively. Median absolute neutrophil count engraftment was 19 (13–39) days with 51% of patients receiving GCSF to expedite recovery. A total of 17 patients experienced GF; 3 as primary and 14 as secondary within a median time of 204 days (40–905). A 76% successfully discontinued sirolimus at the last follow‐up. Median follow‐up for the cohort is 29.2 (2.1–71.4) months. Estimated 3‐year EFS and OS were 88.2% (81.9–92.5) and 94.6% (89.2–97.3). At multivariable analysis, minor ABC incompatibility hazard ratio (HR) 4 (1.3–12.1; 0.014) and allo‐antibody against non‐ABO donor antigens HR 4.3 (1.3–14.1; 0.016) were significant for EFS. No clonal evolution or myeloid malignancies were seen. This largest single‐center report of NMA HSCT in adults with severe SCD further delineated its feasibility, potential toxicities, and fertility outcomes. GF remains a major impediment and appears dependent on ABO matching and non‐ABO antibodies.
Bibliography:	ObjectType-Article-1 SourceType-Scholarly Journals-1 ObjectType-Feature-2 content type line 23
ISSN:	0361-8609 1096-8652 1096-8652
DOI:	10.1002/ajh.27295