{"entity": "publication", "iuid": "268f8cd7903748f5b5851102da869fc4", "timestamp": "2026-09-26T23:47:32.715Z", "links": {"self": {"href": "https://publications-affiliated.scilifelab.se/publication/268f8cd7903748f5b5851102da869fc4.json"}, "display": {"href": "https://publications-affiliated.scilifelab.se/publication/268f8cd7903748f5b5851102da869fc4"}}, "title": "Survival and Late Effects after Allogeneic Hematopoietic Cell Transplantation for Hematologic Malignancy at Less than Three Years of Age.", "authors": [{"family": "Vrooman", "given": "Lynda M", "initials": "LM"}, {"family": "Millard", "given": "Heather R", "initials": "HR"}, {"family": "Brazauskas", "given": "Ruta", "initials": "R"}, {"family": "Majhail", "given": "Navneet S", "initials": "NS"}, {"family": "Battiwalla", "given": "Minoo", "initials": "M"}, {"family": "Flowers", "given": "Mary E", "initials": "ME"}, {"family": "Savani", "given": "Bipin N", "initials": "BN"}, {"family": "Akpek", "given": "G\u00f6rg\u00fcn", "initials": "G"}, {"family": "Aljurf", "given": "Mahmoud", "initials": "M"}, {"family": "Bajwa", "given": "Rajinder", "initials": "R"}, {"family": "Baker", "given": "K Scott", "initials": "KS"}, {"family": "Beitinjaneh", "given": "Amer", "initials": "A"}, {"family": "Bitan", "given": "Menachem", "initials": "M"}, {"family": "Buchbinder", "given": "David", "initials": "D"}, {"family": "Chow", "given": "Eric", "initials": "E"}, {"family": "Dandoy", "given": "Christopher", "initials": "C"}, {"family": "Dietz", "given": "Andrew C", "initials": "AC"}, {"family": "Diller", "given": "Lisa", "initials": "L"}, {"family": "Gale", "given": "Robert Peter", "initials": "RP"}, {"family": "Hashmi", "given": "Shahrukh K", "initials": "SK"}, {"family": "Hayashi", "given": "Robert J", "initials": "RJ"}, {"family": "Hematti", "given": "Peiman", "initials": "P"}, {"family": "Kamble", "given": "Rammurti T", "initials": "RT"}, {"family": "Kasow", "given": "Kimberly A", "initials": "KA"}, {"family": "Kletzel", "given": "Morris", "initials": "M"}, {"family": "Lazarus", "given": "Hillard M", "initials": "HM"}, {"family": "Malone", "given": "Adriana K", "initials": "AK"}, {"family": "Marks", "given": "David I", "initials": "DI"}, {"family": "O'Brien", "given": "Tracey A", "initials": "TA"}, {"family": "Olsson", "given": "Richard F", "initials": "RF"}, {"family": "Ringden", "given": "Olle", "initials": "O"}, {"family": "Seo", "given": "Sachiko", "initials": "S"}, {"family": "Steinberg", "given": "Amir", "initials": "A"}, {"family": "Yu", "given": "Lolie C", "initials": "LC"}, {"family": "Warwick", "given": "Anne", "initials": "A"}, {"family": "Shaw", "given": "Bronwen", "initials": "B"}, {"family": "Duncan", "given": "Christine", "initials": "C"}], "type": "journal article", "published": "2017-08-00", "journal": {"title": "Biol Blood Marrow Transplant", "issn": "1523-6536", "volume": "23", "issue": "8", "pages": "1327-1334", "issn-l": null}, "abstract": "Very young children undergoing hematopoietic cell transplantation (HCT) are a unique and vulnerable population. We analyzed outcomes of 717 patients from 117 centers who survived relapse free for \u22651 year after allogeneic myeloablative HCT for hematologic malignancy at <3 years of age, between 1987 and 2012. The median follow-up was 8.3 years (range, 1.0 to 26.4 years); median age at follow-up was 9 years (range, 2 to 29 years). Ten-year overall and relapse-free survival were 87% (95% confidence interval [CI], 85% to 90%) and 84% (95% CI, 81% to 87%). Ten-year cumulative incidence of relapse was 11% (95% CI, 9% to 13%). Of 84 deaths, relapse was the leading cause (43%). Chronic graft-versus-host-disease 1 year after HCT was associated with increased risk of mortality (hazard ratio [HR], 2.1; 95% CI, 1.3 to 3.3; P = .0018). Thirty percent of patients experienced \u22651 organ toxicity/late effect >1 year after HCT. The most frequent late effects included growth hormone deficiency/growth disturbance (10-year cumulative incidence, 23%; 95% CI, 19% to 28%), cataracts (18%; 95% CI, 15% to 22%), hypothyroidism (13%; 95% CI, 10% to 16%), gonadal dysfunction/infertility requiring hormone replacement (3%; 95% CI, 2% to 5%), and stroke/seizure (3%; 95% CI, 2% to 5%). Subsequent malignancy was reported in 3.6%. In multivariable analysis, total body irradiation (TBI) was predictive of increased risk of cataracts (HR, 17.2; 95% CI, 7.4 to 39.8; P < .001), growth deficiency (HR, 3.5; 95% CI, 2.2 to 5.5; P < .001), and hypothyroidism (HR, 5.3; 95% CI, 3.0 to 9.4; P < .001). In summary, those who survived relapse free \u22651 year after HCT for hematologic malignancy at <3 years of age had favorable overall survival. Chronic graft-versus-host-disease and TBI were associated with adverse outcomes. Future efforts should focus on reducing the risk of relapse and late effects after HCT at early age.", "doi": "10.1016/j.bbmt.2017.04.017", "pmid": "28461213", "labels": [], "xrefs": [{"db": "mid", "key": "NIHMS888867"}, {"db": "pmc", "key": "PMC5666571"}, {"db": "pii", "key": "S1083-8791(17)30426-3"}], "notes": [], "created": "2026-09-23T09:18:36.867Z", "modified": "2026-09-23T09:18:36.882Z"}