title: A single-center analysis of chronic graft-versus-host disease-free, relapse-free survival after alternative donor stem cell transplantation in children with hematological malignancies.
abstract: We assessed the clinical outcomes of allogeneic hematopoietic stem cell transplantation (SCT) from alternative donors for pediatric patients with hematological malignancies, defining graft-versus-host disease (GVHD)-free, relapse-free survival (GRFS) as a composite endpoint. We also defined chronic GVHD-free, relapse-free survival (cGRFS) as survival without severe chronic GVHD, relapse, or death. The probabilities of 2-year disease-free survival from a human leukocyte antigen (HLA) matched unrelated donor (n = 57), related donor with HLA-1 antigen mismatch in the graft-versus-host direction (1Ag-GvH-MMRD, n = 28), and unrelated umbilical cord blood (n = 35) were 52.2, 38.5, and 40.4%, respectively (P = 0.14), and for 2-year GRFS were 26.2, 13.4, and 30.4%, respectively (P = 0.089), and for 2-year cGRFS were 36.2, 16.7, and 40.4%, respectively (P = 0.015). Of the three groups, the 1Ag-GvH-MMRD group showed a significantly higher cumulative incidence of severe cGVHD, and was identified as a significant risk factor for worse cGRFS. These results suggest that intensification of GVHD prophylaxis may be needed for SCT from 1Ag-GvH-MMRD. As with GRFS, cGRFS should be used as an endpoint of the clinical study to predict long-term morbidity and mortality for patients who need longer follow-up such as pediatric SCT recipients.
authors.affiliation: Department of Pediatrics, National Kyushu Cancer Center, 3-1-1, Notame, Minami-ku, Fukuoka, 811-1395, Japan. inagakij@nk-cc.go.jp.
authors.forename: Jiro
authors.initials: J
authors.lastname: Inagaki
authors.affiliation: Department of Pediatrics, National Kyushu Cancer Center, 3-1-1, Notame, Minami-ku, Fukuoka, 811-1395, Japan.
authors.forename: Reiji
authors.initials: R
authors.lastname: Fukano
authors.affiliation: Department of Pediatrics, National Kyushu Cancer Center, 3-1-1, Notame, Minami-ku, Fukuoka, 811-1395, Japan.
authors.forename: Maiko
authors.initials: M
authors.lastname: Noguchi
authors.affiliation: Department of Pediatrics, National Kyushu Cancer Center, 3-1-1, Notame, Minami-ku, Fukuoka, 811-1395, Japan.
authors.forename: Jun
authors.initials: J
authors.lastname: Okamura
mesh_terms: D000293:Adolescent; D064591:Allografts; D002648:Child; D002675:Child, Preschool; D018572:Disease-Free Survival; D005260:Female; D005500:Follow-Up Studies; D006086:Graft vs Host Disease / Q000453:epidemiology / Q000401:mortality* / Q000517:prevention & control; D006680:HLA Antigens; D019337:Hematologic Neoplasms / Q000401:mortality* / Q000628:therapy*; D018380:Hematopoietic Stem Cell Transplantation / Q000401:mortality*; D006648:Histocompatibility; D006801:Humans; D015994:Incidence; D007223:Infant; D008297:Male; D013997:Time Factors; D014019:Tissue Donors; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D006680:HLA Antigens
keywords: Alternative donor; Chronic graft-versus-host disease; Graft-versus-host disease-free, relapse-free survival; Pediatrics