| Literature DB >> 32949751 |
Leonardo Javier Arcuri1, Samir Kanaan Nabhan2, Renato Cunha3, Samantha Nichele2, Andreza Alice Feitosa Ribeiro4, Juliana Folloni Fernandes5, Liane Esteves Daudt6, Ana Luiza Melo Rodrigues7, Celso Arrais-Rodrigues8, Adriana Seber9, Elias Hallack Atta10, Jose Salvador Rodrigues de Oliveira8, Vaneuza Araujo Moreira Funke11, Gisele Loth2, Luiz Guilherme Darrigo Junior3, Alessandra Paz6, Rodolfo Froes Calixto12, Alessandra Araujo Gomes13, Carlos Eduardo Sa Araujo14, Vergilio Colturato15, Belinda Pinto Simoes3, Nelson Hamerschlak4, Mary Evelyn Flowers16, Ricardo Pasquini2, Vanderson Rocha17, Carmem Bonfim2.
Abstract
Severe aplastic anemia (SAA) is a life-threatening disease that can be cured with allogeneic cell transplantation (HCT). Haploidentical donor transplantation with post-transplantation cyclophosphamide (haplo-PTCy) is an option for patients lacking an HLA-matched donor. We analyzed 87 patients who underwent haplo-PTCy between 2010 and 2019. The median patient age was 14 years (range, 1 to 69 years), most were heavily transfused, and all received previous immunosuppression (25% without antithymocyte globulin). Almost two-thirds (63%) received standard fludarabine (Flu)/cyclophosphamide (Cy) 29/total body irradiation (TBI) 200 cGy conditioning, and the remaining patients received an augmented conditioning: Flu/Cy29/TBI 300-400 (16%), Flu/Cy50/TBI 200 (10%), or Flu/Cy50/TBI 400 (10%). All patients received PTCy-based graft-versus-host disease (GVHD) prophylaxis. Most grafts (93%) were bone marrow (BM). The median duration of follow-up was 2 years and 2 months. The median time to neutrophil recovery was 17 days. Primary graft failure occurred in 15% of the patients, and secondary or poor graft function occurred in 5%. The incidences of grade II-IV acute GVHD was 14%, and that of chronic GVHD was 9%. Two-year overall survival and event-free survival (EFS) were 79% and 70%, respectively. EFS was higher for patients who received augmented Flu/Cy/TBI (hazard ratio [HR], .28; P = .02), and those who received higher BM CD34 cell doses (>3.2 × 10E6/kg) (HR, .29; P = .004). The presence of donor-specific antibodies before HSCT was associated with lower EFS (HR, 3.92; P = .01). Graft failure (HR, 7.20; P < .0001) was associated with an elevated risk of death. Cytomegalovirus reactivation was frequent (62%). Haploidentical HCT for SAA is a feasible procedure; outcomes are improved with augmented conditioning regimens and BM grafts with higher CD34 cell doses.Entities:
Keywords: Alternativo donors; Aplastic anemia; Haploidentical
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Year: 2020 PMID： 32949751 DOI： 10.1016/j.bbmt.2020.09.007
Source DB: PubMed Journal: Biol Blood Marrow Transplant ISSN： 1083-8791 Impact factor: 5.742