title: Feasibility and outcome of haploidentical SCT in pediatric high-risk hematologic malignancies and Fanconi anemia in Uruguay.
abstract: In total, 17 pediatric patients with hematologic malignancies (n=14) and Fanconi anemia (FA) (n=3) underwent haploidentical SCT with T-cell depletion. The patients were conditioned with reduced-intensity regimens, and CYA was used for GVHD prophylaxis. Successful engraftment occurred in 16 patients (94%). One patient failed to achieve a primary engraftment. Another patient rejected the first SCT after 10 weeks and had a successful second transplant. Of all engrafted patients, only one developed severe acute GVHD. Ten patients were alive at a median follow-up of 18 months (range, 5-62 months). The 5-years' OS was 53.8%. The three patients with FA are currently well with full-donor chimerism at 16, 6 and 5 months post transplant, respectively. The OS of 14 patients with high-risk hematologic malignancies was 47.6%. Three patients died as a result of post transplant leukemia relapse. CMV infection, GVHD and organ injury were other causes of mortality. Haploidentical SCT was found to be an alternative feasible treatment in Uruguay for patients who need allogenic transplantation but lack an HLA-identical family donor. It should be considered as an early option in FA patients before transformation or significant exposure to blood products.
authors.affiliation: Pediatric Hemato-Oncology Department, Centro Hospitalario Pereira Rossell, Montevideo, Uruguay. gdufort@chasque.net
authors.forename: G
authors.initials: G
authors.lastname: Dufort
authors.forename: S
authors.initials: S
authors.lastname: Pisano
authors.forename: A
authors.initials: A
authors.lastname: Incoronato
authors.forename: M
authors.initials: M
authors.lastname: Castiglioni
authors.forename: M
authors.initials: M
authors.lastname: Carracedo
authors.forename: C
authors.initials: C
authors.lastname: Pagés
authors.forename: E
authors.initials: E
authors.lastname: Simon
authors.forename: S
authors.initials: S
authors.lastname: Zuccolo
authors.forename: R
authors.initials: R
authors.lastname: Barcelona
authors.forename: R
authors.initials: R
authors.lastname: Mezzano
authors.forename: A
authors.initials: A
authors.lastname: Tiscornia
authors.forename: F
authors.initials: F
authors.lastname: Lemos
authors.forename: F
authors.initials: F
authors.lastname: Morosini
authors.forename: M
authors.initials: M
authors.lastname: Schelotto
authors.forename: H
authors.initials: H
authors.lastname: Giordano
authors.forename: E
authors.initials: E
authors.lastname: Carreto
authors.forename: M
authors.initials: M
authors.lastname: Bengoechea
authors.forename: B
authors.initials: B
authors.lastname: Boggia
authors.forename: I
authors.initials: I
authors.lastname: Rodriguez
authors.forename: L
authors.initials: L
authors.lastname: Guerrero
authors.forename: A
authors.initials: A
authors.lastname: Dabezies
authors.forename: L
authors.initials: L
authors.lastname: Castillo
mesh_terms: D000293:Adolescent; D000328:Adult; D000741:Anemia, Aplastic / Q000628:therapy; D002648:Child; D002675:Child, Preschool; D003586:Cytomegalovirus Infections / Q000209:etiology; D005199:Fanconi Anemia / Q000150:complications / Q000628:therapy*; D005260:Female; D005500:Follow-Up Studies; D006086:Graft vs Host Disease / Q000517:prevention & control; D006680:HLA Antigens / Q000276:immunology; D019337:Hematologic Neoplasms / Q000401:mortality / Q000628:therapy*; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects; D006801:Humans; D007223:Infant; D008297:Male; D008875:Middle Aged; D018183:Transplantation Chimera; D019172:Transplantation Conditioning / Q000009:adverse effects; D014184:Transplantation, Homologous; D016896:Treatment Outcome; D014583:Uruguay / Q000453:epidemiology
chemical_list: D006680:HLA Antigens