Darrigo Luiz Guilherme, Colturato Vergilio, de Souza Mair Pedro, Loth Gisele, Calixto Rodolfo, Seber Adriana, Zecchin Victor Gottardello, Esteves Daudt Liane, Tavares Rita Barbosa, Arcuri Leonardo, de Macedo Antonio Vaz, Vieira Ana Karine, Kuwahara Cilmara, Ribeiro Lisandro, Fernandes Juliana Folloni, Flowers Mary E, Pasquini Ricardo, Bonfim Carmem
Ribeirão Preto Medical School, University of São Paulo, Ribeirão Preto, Brazil.
Hospital Amaral de Carvalho, Jau, Brazil.
Pediatr Transplant. 2019 Nov;23(7):e13552. doi: 10.1111/petr.13552. Epub 2019 Jul 11.
In this study, we report on major MRD or URD BMT outcomes in pediatric patients with SAA in Brazil. This was a retrospective study, which included 106 patients ≤18 years old who received a first BMT for SAA. All patients received bone marrow as graft source from an MRD (n = 69) or a URD (n = 37). Conditioning regimen was non-myeloablative in 73.6% of cases, and GVHD prophylaxis comprised a calcineurin inhibitor plus methotrexate in 89.6% of patients. After a median follow-up of 4.5 years after BMT, 81 patients are alive, with a 4-year OS of 77% and no statistically significant difference between the MRD and URD groups (82% vs. 69%, respectively; P = .08). Grade III-IV aGVHD at 6 months and cGVHD at 2 years were observed in 8% and 14% of cases, respectively, and were not statistically different between the groups. Twenty-five (23%) patients died at a median of 2.9 months after BMT. Our study showed that 4-year OS after BMT was not statistically different between MRD and URD recipients. This study shows that the outcomes of pediatric patients transplanted for SAA with a URD in Brazil are approaching those of MRD transplants. In contrast, OS after MRD BMT was lower than we would expect based on previous reports. The wide range of preparatory regimens used by the study centers highlights the need for standardized protocols for these children. Our findings provide a benchmark for future studies focused on improving BMT outcomes in this setting in Brazil.
在本研究中，我们报告了巴西儿童重型再生障碍性贫血（SAA）患者接受主要的单倍体相合造血干细胞移植（MRD）或非血缘关系造血干细胞移植（URD）后的结局。这是一项回顾性研究，纳入了106例年龄≤18岁、因SAA接受首次造血干细胞移植（BMT）的患者。所有患者均接受来自MRD供者（n = 69）或URD供者（n = 37）的骨髓作为移植物来源。73.6%的病例采用非清髓性预处理方案，89.6%的患者采用钙调神经磷酸酶抑制剂加甲氨蝶呤预防移植物抗宿主病（GVHD）。BMT后中位随访4.5年，81例患者存活，4年总生存率（OS）为77%，MRD组和URD组之间无统计学显著差异（分别为82%和69%；P = 0.08）。6个月时III-IV级急性移植物抗宿主病（aGVHD）和2年时慢性移植物抗宿主病（cGVHD）的发生率分别为8%和14%，两组之间无统计学差异。25例（23%）患者在BMT后中位2.9个月死亡。我们的研究表明，MRD和URD受者BMT后的4年OS无统计学差异。本研究表明，巴西接受URD移植治疗SAA的儿童患者的结局正在接近MRD移植的结局。相比之下，MRD BMT后的OS低于我们基于既往报告的预期。研究中心使用的预处理方案范围广泛，凸显了为这些儿童制定标准化方案的必要性。我们的研究结果为未来旨在改善巴西这种情况下BMT结局的研究提供了一个基准。
