94 Hematopoietic Stem Cell Transplantation in Primary Immunodeficiencies in Brazil - a Survey of the Working Group on Paediatric Transplantation of the Brazilian Society of Bone Marrow Transplantation

Track: BMT Tandem "Scientific" Meeting
Wednesday, February 11, 2015, 4:45 PM-6:45 PM
Harbor Ballroom ABC (Manchester Grand Hyatt)
Juliana Folloni Fernandes , Hospital Israelita Albert Einstein, Sao Paulo, Brazil
Adriana Seber, MD , Instituto de Oncologia Pediatrica, Sao Paulo, Brazil
Liane Esteves Daudt , Hospital das Clínicas de Porto Alegre, Porto Alegre, Brazil
Lisandro Ribeiro, MD , Bone Marrow Transplantation Unit, Federal University of Paraná, Curitiba, Brazil
Victor Zecchin , Instituto de Oncologia Pediátrica, Sao Paulo, Brazil
Marcos A. Mauad, MD , Bone Marrow Transplantation, Centro de Oncologia e Hematologia, Jau, Brazil
Luiz Guilherme Darrigo Jr. , Hospital das Clínicas – USP – Ribeirão Preto, Ribeirão Preto, Brazil
Ana Karine Vieira , Universidade Federal de Minas Gerais, Belo Horizonte, Brazil
Lauro Gregianin, MD , Hospital de Clinicas de Porto Alegre, Porto Alegre, RS, Brazil
Adriana Martins Sousa , Universidade Federal do Rio de Janeiro, Rio de Janeiro, Brazil
Adriana Santini , Hospital das Clínicas de Porto Alegre, Porto Alegre, Brazil
Rita Tavares, MD , Instituto Nacional de Cancer, Centro Nacional de Transplate de Medula Ossea - CEMO, Rio de Janeiro, Brazil
Samantha Nichele, MD , Bone Marrow Transplantation Unit, Federal University of Paraná, Curitiba, Brazil
Adriana Koliski, MD , Paediatric Intensive Care Unit, Federal University of Parana, Curitiba, Brazil
Luis Fernando S. Bouzas, MD, PhD , National Institute of Cancer INCA, Rio De Janeiro, Brazil
Nelson Hamerschlak, MD, PhD , Hematology and Bone Marrow Transplantation Dept, Hospital Israelita Albert Einstein, Sao Paulo, Brazil
Carmem Bonfim, MD , Bone Marrow Transplantation Unit, Federal University of Paraná, Curitiba, Brazil
Hematopoietic Stem Cell Transplantation (HSCT) is curative in a large number of primary immunodeficiencies (PID). The first transplant in PID in Brazil was held in 1992 at the Hospital de Clínicas da Universidade Federal do Paraná. Since then, in Brazil, only few centers have performed transplants in these pathologies. Objective: Retrospective analysis of outcomes of HSCT performed in patients with PID in 10 Brazilian centers participating in the working group of pediatric transplantation of the Brazilian Society of BMT (SBTMO). From 1992 to April 2014, 166 patients underwent HSCT on PID in 10 different centers in Brazil, mostly in the Hospital of UFPR (n = 90), followed by the Hospital Israelita Albert Einstein (n = 27 ) and the other centers with 10 or fewer. Most patients were male (M = 130, F = 36), and most patients had age lower than 3 years (n = 119). The diagnoses were distributed among: Wiskott-Aldrich syndrome (n = 56); Severe Combined Immunodeficiency (n = 52); Chronic granulomatous disease (n = 14); Familiar Hemophagocytic lymphohistiocytosis (n = 13); Chediak-Higashi Syndrome (n = 12); Hyper-IgM syndrome (n = 5); Severe congenital neutropenia (n = 5); IPEX syndrome (n = 3); combined immunodeficiency disease (n = 2); Leukocyte Adhesion Deficiency (n = 2); Deficiency of IFN-gamma (n = 1) and Griscelli syndrome (n = 1). Thirty-two patients received transplants from identical sibling donors, 112 unrelated donors, and 22 other family donors (10/10 compatible or haploidentical). Graft was bone marrow in 84 cases, umbilical cord blood in 78 cases and 4 patients received stem cells from peripheral blood. Ninety-nine patients received myeloablative transplants (including busulfan> 8mg / Kg), 55 patients received reduced toxicity conditioning and 12 patients received no conditioning. The prophylaxis of graft-versus-host disease was performed with cyclosporine A in most cases, associated with mycophenolate mofetil, methotrexate or steroids. Overall survival was 68.7% at 3 years. In univariate analysis, there was no statistical difference between gender, diagnosis, age at HSCT, year of transplant, source of cells, or donor type. Among the most frequent pathologies groups, analysis was done in 2 groups, the severe combined immunodeficiency (n = 52), and the Wiskott-Aldrich syndrome (n = 56) Syndrome. In these subgroups, the overall survival at 3 years was 60% and 79%, respectively. Conclusion: This is the first survey made on tranplants on primary immunodeficiencies in Brazil. Despite the difficulties in diagnosing and referral of these children for special treatment in our country, we have seen that the overall survival of our patients is not different than those reported in international studies in Europe or North America. This survey is extremely important to have the results of these cases in Brazil, and identify areas to improve the outcomes of transplants in lower income countries.
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