title: Long-term outcome and evaluation of organ function in pediatric patients undergoing haploidentical and matched related hematopoietic cell transplantation for sickle cell disease.
abstract: HLA-matched related donor (MRD) hematopoietic stem cell transplantation (HSCT) is a well-established therapy for patients with sickle cell disease (SCD); however, experience using alternative donors, including haploidentical donors, in HSCT for SCD is limited. We report the long-term outcomes of 22 pediatric patients who underwent related donor HSCT for SCD at St. Jude Children's Research Hospital, either a myeloablative sibling MRD HSCT (n = 14) or reduced-intensity parental haploidentical donor HSCT (n = 8). The median patient age was 11.0 ± 3.9 years in the MRD graft recipients and 9.0 ± 5.0 years in the haploidentical donor graft recipients. The median follow-up was 9.0 ± 2.3 years, with an overall survival (OS) of 93% and a recurrence/graft failure rate of 0%, for the MRD cohort and 7.4 ± 2.4 years, with an OS of 75%, disease-free survival of 38%, and disease recurrence of 38%, for the haploidentical donor cohort. We report the long-term hematologic response and organ function in patients undergoing MRD or haploidentical donor HSCT for severe SCD. Our data demonstrate long-term hematologic improvements after HSCT with sustained engraftment, and confirm that HSCT offers long-term protection from common complications of SCD, including stroke, pulmonary hypertension, acute chest, and nephropathy, regardless of donor source.
authors: Department of Bone Marrow Transplantation and Cellular Therapy, St. Jude Children's Research Hospital, Memphis, TN, USA. mari.dallas@stjude.org
authors: Mari H
authors: MH
authors: Dallas
authors: Brandon
authors: B
authors: Triplett
authors: David R
authors: DR
authors: Shook
authors: Christine
authors: C
authors: Hartford
authors: Ashok
authors: A
authors: Srinivasan
authors: Joseph
authors: J
authors: Laver
authors: Russell
authors: R
authors: Ware
authors: Wing
authors: W
authors: Leung
mesh_terms: D000293:Adolescent; D000755:Anemia, Sickle Cell / Q000188:drug therapy / Q000601:surgery*; D002648:Child; D002675:Child, Preschool; D005260:Female; D006238:Haploidy; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods*; D006801:Humans; D008297:Male; D015996:Survival Rate; D014184:Transplantation, Homologous; D016896:Treatment Outcome