title: Composite end point of graft-versus-host disease-free, relapse-free survival after allogeneic hematopoietic cell transplantation.
abstract: The success of allogeneic hematopoietic cell transplantation (HCT) is typically assessed as individual complications, including graft-versus-host disease (GVHD), relapse, or death, yet no one factor can completely characterize cure without ongoing morbidity. We examined a novel composite end point of GVHD-free/relapse-free survival (GRFS) in which events include grade 3-4 acute GVHD, systemic therapy-requiring chronic GVHD, relapse, or death in the first post-HCT year. In 907 consecutive University of Minnesota allogeneic HCT recipients (2000-2012), 1-year GRFS was 31% (95% confidence interval [CI] 28-34). Regression analyses showed age, disease risk, and donor type significantly influencing GRFS. Adults age 21+ had 2-fold worse GRFS vs children; GRFS did not differ beyond age 21. Adjusted for conditioning intensity, stem cell source, disease risk, age, and transplant year, HLA-matched sibling donor marrow resulted in the best GRFS (51%, 95% CI 46-66), whereas HLA-matched sibling donor peripheral blood stem cells were significantly worse (25%, 95% CI 20-30, P = .01). GRFS after umbilical cord blood transplants and marrow from matched unrelated donors were similar (31%, 95% CI 27-35 and 32%, 95% CI 22-42, respectively). Because GRFS measures freedom from ongoing morbidity and represents ideal HCT recovery, GRFS has value as a novel end point for benchmarking new therapies.
authors.affiliation: Blood and Marrow Transplant Program, and.
authors.forename: Shernan G
authors.initials: SG
authors.lastname: Holtan
authors.affiliation: Biostatistics and Informatics Core, Masonic Cancer Center, University of Minnesota, Minneapolis, MN.
authors.forename: Todd E
authors.initials: TE
authors.lastname: DeFor
authors.affiliation: Blood and Marrow Transplant Program, and.
authors.forename: Aleksandr
authors.initials: A
authors.lastname: Lazaryan
authors.affiliation: Blood and Marrow Transplant Program, and.
authors.forename: Nelli
authors.initials: N
authors.lastname: Bejanyan
authors.affiliation: Blood and Marrow Transplant Program, and.
authors.forename: Mukta
authors.initials: M
authors.lastname: Arora
authors.affiliation: Blood and Marrow Transplant Program, and.
authors.forename: Claudio G
authors.initials: CG
authors.lastname: Brunstein
authors.affiliation: Blood and Marrow Transplant Program, and.
authors.forename: Bruce R
authors.initials: BR
authors.lastname: Blazar
authors.affiliation: Blood and Marrow Transplant Program, and.
authors.forename: Margaret L
authors.initials: ML
authors.lastname: MacMillan
authors.affiliation: Blood and Marrow Transplant Program, and.
authors.forename: Daniel J
authors.initials: DJ
authors.lastname: Weisdorf
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D015415:Biomarkers / Q000032:analysis; D002648:Child; D002675:Child, Preschool; D018572:Disease-Free Survival; D005260:Female; D006086:Graft vs Host Disease / Q000453:epidemiology / Q000209:etiology / Q000517:prevention & control; D019337:Hematologic Neoplasms / Q000175:diagnosis* / Q000453:epidemiology / Q000628:therapy*; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects; D006801:Humans; D007223:Infant; D008297:Male; D008875:Middle Aged; D012008:Recurrence; D012189:Retrospective Studies; D019172:Transplantation Conditioning / Q000379:methods; D014184:Transplantation, Homologous; D061349:Unrelated Donors; D055815:Young Adult
chemical_list: D015415:Biomarkers