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Article: Long-term outcome and evaluation of organ function in pediatric patients undergoing haploidentical and matched related hematopoietic cell transplantation for sickle cell disease

TitleLong-term outcome and evaluation of organ function in pediatric patients undergoing haploidentical and matched related hematopoietic cell transplantation for sickle cell disease
Authors
KeywordsReduced-intensity regimen
Matched sibling donor
Sickle cell disease
Haploidentical donor
Long-term follow-up
Issue Date2013
Citation
Biology of Blood and Marrow Transplantation, 2013, v. 19, n. 5, p. 820-830 How to Cite?
AbstractHLA-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. © 2013 American Society for Blood and Marrow Transplantation.
Persistent Identifierhttp://hdl.handle.net/10722/294463
ISSN
2020 Impact Factor: 5.742
2015 SCImago Journal Rankings: 2.094
PubMed Central ID
ISI Accession Number ID

 

DC FieldValueLanguage
dc.contributor.authorDallas, Mari H.-
dc.contributor.authorTriplett, Brandon-
dc.contributor.authorShook, David R.-
dc.contributor.authorHartford, Christine-
dc.contributor.authorSrinivasan, Ashok-
dc.contributor.authorLaver, Joseph-
dc.contributor.authorWare, Russell-
dc.contributor.authorLeung, Wing-
dc.date.accessioned2020-12-03T08:22:48Z-
dc.date.available2020-12-03T08:22:48Z-
dc.date.issued2013-
dc.identifier.citationBiology of Blood and Marrow Transplantation, 2013, v. 19, n. 5, p. 820-830-
dc.identifier.issn1083-8791-
dc.identifier.urihttp://hdl.handle.net/10722/294463-
dc.description.abstractHLA-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. © 2013 American Society for Blood and Marrow Transplantation.-
dc.languageeng-
dc.relation.ispartofBiology of Blood and Marrow Transplantation-
dc.subjectReduced-intensity regimen-
dc.subjectMatched sibling donor-
dc.subjectSickle cell disease-
dc.subjectHaploidentical donor-
dc.subjectLong-term follow-up-
dc.titleLong-term outcome and evaluation of organ function in pediatric patients undergoing haploidentical and matched related hematopoietic cell transplantation for sickle cell disease-
dc.typeArticle-
dc.description.naturelink_to_OA_fulltext-
dc.identifier.doi10.1016/j.bbmt.2013.02.010-
dc.identifier.pmid23416852-
dc.identifier.pmcidPMC4712646-
dc.identifier.scopuseid_2-s2.0-84876333389-
dc.identifier.volume19-
dc.identifier.issue5-
dc.identifier.spage820-
dc.identifier.epage830-
dc.identifier.eissn1523-6536-
dc.identifier.isiWOS:000318132500021-
dc.identifier.issnl1083-8791-

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