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Article: Favorable preliminary results using TLI/ATG-based immunomodulatory conditioning for matched unrelated donor allogeneic hematopoietic stem cell transplantation in pediatric severe aplastic anemia

TitleFavorable preliminary results using TLI/ATG-based immunomodulatory conditioning for matched unrelated donor allogeneic hematopoietic stem cell transplantation in pediatric severe aplastic anemia
Authors
Keywordspediatrics
hematopoietic stem cell transplantation
aplastic anemia engraftment
transplant tolerance
graft-versus-host disease
non-myeloablative
Issue Date2011
Citation
Pediatric Transplantation, 2011, v. 15, n. 6, p. 628-634 How to Cite?
AbstractTo assess whether a tolerance-induction regimen could be applied for unrelated (MUD) HCT in SAA, we retrospectively reviewed our HCT experience using unmanipulated 10/10 HLA-matched bone marrow grafts from MSD vs. MUD donors. Conditioning was CTX 200 mg/kg (CTX) + rabbit ATG 10 mg/kg (ATG) for MSD (n = 9) and TLI (800 cGy) + CTX/ATG for MUD HCT (n = 5). Immunoprophylaxis was CSA and short-course MTX. Median patient age was 14.7 yr, median time to HCT 1.5 yr, and median follow-up 3 yr. Outcome measures included EFS, time to engraftment, and cumulative incidence of GVHD (CIN of GVHD) for MSD and MUD cohorts. EFS and stable engraftment rate were 100%. CIN of acute GVHD was: MSD, Grade I-II: 1 (11%), Grade III-IV: 0%; MUD, Grade I-II: 1 (20%), Grade III-IV: 1 (20%). CIN of chronic GVHD was: MSD, limited: 1 (11%), extensive: 0%; MUD, limited: 0%, extensive: 0%. All immunosuppressive-compliant patients successfully weaned immunosuppression. Although in limited patients, our results suggest that immunomodulatory TLI added to backbone CTX/ATG conditioning is a promising option for MUD HCT in SAA patients, which we will examine in a prospective clinical trial. © 2011 John Wiley & Sons A/S.
Persistent Identifierhttp://hdl.handle.net/10722/294443
ISSN
2023 Impact Factor: 1.2
2023 SCImago Journal Rankings: 0.494
PubMed Central ID
ISI Accession Number ID

 

DC FieldValueLanguage
dc.contributor.authorPillai, Asha-
dc.contributor.authorHartford, Christine-
dc.contributor.authorWang, Chong-
dc.contributor.authorPei, Deqing-
dc.contributor.authorYang, Jie-
dc.contributor.authorSrinivasan, Ashok-
dc.contributor.authorTriplett, Brandon-
dc.contributor.authorDallas, Mari-
dc.contributor.authorLeung, Wing-
dc.date.accessioned2020-12-03T08:22:45Z-
dc.date.available2020-12-03T08:22:45Z-
dc.date.issued2011-
dc.identifier.citationPediatric Transplantation, 2011, v. 15, n. 6, p. 628-634-
dc.identifier.issn1397-3142-
dc.identifier.urihttp://hdl.handle.net/10722/294443-
dc.description.abstractTo assess whether a tolerance-induction regimen could be applied for unrelated (MUD) HCT in SAA, we retrospectively reviewed our HCT experience using unmanipulated 10/10 HLA-matched bone marrow grafts from MSD vs. MUD donors. Conditioning was CTX 200 mg/kg (CTX) + rabbit ATG 10 mg/kg (ATG) for MSD (n = 9) and TLI (800 cGy) + CTX/ATG for MUD HCT (n = 5). Immunoprophylaxis was CSA and short-course MTX. Median patient age was 14.7 yr, median time to HCT 1.5 yr, and median follow-up 3 yr. Outcome measures included EFS, time to engraftment, and cumulative incidence of GVHD (CIN of GVHD) for MSD and MUD cohorts. EFS and stable engraftment rate were 100%. CIN of acute GVHD was: MSD, Grade I-II: 1 (11%), Grade III-IV: 0%; MUD, Grade I-II: 1 (20%), Grade III-IV: 1 (20%). CIN of chronic GVHD was: MSD, limited: 1 (11%), extensive: 0%; MUD, limited: 0%, extensive: 0%. All immunosuppressive-compliant patients successfully weaned immunosuppression. Although in limited patients, our results suggest that immunomodulatory TLI added to backbone CTX/ATG conditioning is a promising option for MUD HCT in SAA patients, which we will examine in a prospective clinical trial. © 2011 John Wiley & Sons A/S.-
dc.languageeng-
dc.relation.ispartofPediatric Transplantation-
dc.subjectpediatrics-
dc.subjecthematopoietic stem cell transplantation-
dc.subjectaplastic anemia engraftment-
dc.subjecttransplant tolerance-
dc.subjectgraft-versus-host disease-
dc.subjectnon-myeloablative-
dc.titleFavorable preliminary results using TLI/ATG-based immunomodulatory conditioning for matched unrelated donor allogeneic hematopoietic stem cell transplantation in pediatric severe aplastic anemia-
dc.typeArticle-
dc.description.naturelink_to_OA_fulltext-
dc.identifier.doi10.1111/j.1399-3046.2011.01542.x-
dc.identifier.pmid21762328-
dc.identifier.pmcidPMC3538876-
dc.identifier.scopuseid_2-s2.0-80052097795-
dc.identifier.volume15-
dc.identifier.issue6-
dc.identifier.spage628-
dc.identifier.epage634-
dc.identifier.eissn1399-3046-
dc.identifier.isiWOS:000294170200019-
dc.identifier.issnl1397-3142-

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