title: Unrelated Cord Blood Transplantation for Acute Leukemia Diagnosed in the First Year of Life: Outcomes and Risk Factor Analysis.
abstract: Infant acute leukemia still has a poor prognosis, and allogeneic hematopoietic stem cell transplantation is indicated in selected patients. Umbilical cord blood (UCB) is an attractive cell source for this population because of the low risk of chronic graft-versus-host disease (GVHD), the strong graft-versus-leukemia effect, and prompt donor availability. This retrospective, registry-based study reported UCB transplantation (UCBT) outcomes in 252 children with acute lymphoblastic leukemia (ALL; n = 157) or acute myelogenous leukemia (AML; n = 95) diagnosed before 1 year of age who received a single-unit UCBT after myeloablative conditioning between 1996 and 2012 in European Society for Blood and Marrow Transplantation centers. Median age at UCBT was 1.1 years, and median follow-up was 42 months. Most patients (57%) received a graft with 1 HLA disparity and were transplanted in first complete remission (CR; 55%). Cumulative incidence function (CIF) of day 100 acute GVHD (grades II to IV) was 40% ± 3% and of 4-year chronic GVHD was 13% ± 2%. CIF of 1-year transplant-related mortality was 23% ± 3% and of 4-year relapse was 27% ± 3%. Leukemia-free-survival (LFS) at 4 years was 50% ± 3%; it was 40% and 66% for those transplanted for ALL and AML, respectively (P = .001). LFS was better for patients transplanted in first CR, regardless of diagnosis. In multivariate model, diagnosis of ALL (P = .001), advanced disease status at UCBT (<.001), age at diagnosis younger than 3 months (P = .012), and date of transplant before 2004 were independently associated with worse LFS. UCBT is a suitable option for patients diagnosed with infant acute leukemia who achieve CR. In this cohort, patients with AML had better survival than those with ALL.
authors: Service d'Hématologie et Therapie cellulaire, Hôpital Saint Antoine, Paris, France; Eurocord, University Paris VII IUH Paris, France. Electronic address: annalisa.ruggeri@aphp.fr.
authors: Annalisa
authors: A
authors: Ruggeri
authors: Eurocord, University Paris VII IUH Paris, France.
authors: Fernanda
authors: F
authors: Volt
authors: Dipartimento di Oncoematologia Pediatrica, Ospedale Bambino Gesù, IRCSS, Roma, Università di Pavia, Italy.
authors: Franco
authors: F
authors: Locatelli
authors: Hôpital d'Enfants de la Timone, Marseille, France.
authors: Gerard
authors: G
authors: Michel
authors: Servicio de Hematologia y Oncologia Pediátrica, Hospital Vall d'Hebron, Barcelona, Spain.
authors: Cristina
authors: C
authors: Diaz de Heredia
authors: BMT Unit, Inst. Portugues Oncologia, Porto, Portugal.
authors: Manuel
authors: M
authors: Abecasis
authors: Unità di Onco-Ematologia, IRCCS Policlinico San Matteo, Pavia, Italy.
authors: Marco
authors: M
authors: Zecca
authors: Department of Haematology, Sheffield Children's Hospital and University of Sheffield, Sheffield, United Kingdom.
authors: Ajay
authors: A
authors: Vora
authors: Service d'Hématologie, Hôpital Robert Debré, APHP, Paris, France.
authors: Karima
authors: K
authors: Yakouben
authors: Sydney Children's Hospital, Kids Cancer Centre, Sydney, Australia.
authors: Tracey A
authors: TA
authors: O'Brien
authors: UOSD Trapianto di Midollo, Istituto G. Gaslini, Largo G. Gaslini, Genova, Italy.
authors: Stefano
authors: S
authors: Giardino
authors: Bristol Royal Hospital for Children, Bristol, United Kingdom.
authors: Jacqueline
authors: J
authors: Cornish
authors: Eurocord, University Paris VII IUH Paris, France; Sirio Libanes Hospital and University of Sao Paulo, Sao Paulo, Brazil.
authors: Vanderson
authors: V
authors: Rocha
authors: BMT Unit, St. Anna Kinderspital, Vienna, Austria.
authors: Christina
authors: C
authors: Peters
authors: Universitätsklinikum Frankfurt, Goethe-Universität, Frankfurt, Germany.
authors: Peter
authors: P
authors: Bader
authors: Eurocord, University Paris VII IUH Paris, France; Monacord Centre Scientifique de Monaco, Monaco.
authors: Eliane
authors: E
authors: Gluckman
authors: Service d'Hématologie, Hôpital Robert Debré, APHP, Paris, France.
authors: Jean Hugues
authors: JH
authors: Dalle
mesh_terms: D000208:Acute Disease; D036101:Cord Blood Stem Cell Transplantation / Q000009:adverse effects / Q000379:methods* / Q000401:mortality; D006086:Graft vs Host Disease / Q000209:etiology; D006801:Humans; D007223:Infant; D007231:Infant, Newborn; D007938:Leukemia / Q000150:complications / Q000401:mortality / Q000628:therapy*; D015470:Leukemia, Myeloid, Acute / Q000150:complications / Q000401:mortality / Q000628:therapy; D019653:Myeloablative Agonists / Q000627:therapeutic use; D054198:Precursor Cell Lymphoblastic Leukemia-Lymphoma / Q000150:complications / Q000401:mortality / Q000628:therapy; D012042:Registries; D012074:Remission Induction; D012189:Retrospective Studies; D012307:Risk Factors; D019172:Transplantation Conditioning / Q000379:methods; D016896:Treatment Outcome; D061349:Unrelated Donors
chemical_list: D019653:Myeloablative Agonists
keywords: Acute leukemia; Cord blood transplantation; Infant; MLL-rearranged leukemia