Salamonowicz-Bodzioch Małgorzata, Rosa Monika, Frączkiewicz Jowita, Gorczyńska Ewa, Gul Katarzyna, Janeczko-Czarnecka Małgorzata, Jarmoliński Tomasz, Kałwak Krzysztof, Mielcarek-Siedziuk Monika, Olejnik Igor, Owoc-Lempach Joanna, Panasiuk Anna, Gajek Kornelia, Rybka Blanka, Ryczan-Krawczyk Renata, Ussowicz Marek
Department of Pediatric Oncology, Haematology and Bone Marrow Transplantation, Wroclaw Medical University, Borowska 213, 50-556 Wroclaw, Poland.
Department of Oncology, Hematolgy and Transplantology, University Hospital USK in Wroclaw, 50-556 Wroclaw, Poland.
J Clin Med. 2021 Sep 26;10(19):4416. doi: 10.3390/jcm10194416.
Severe aplastic anemia (SAA) is a bone marrow failure syndrome that can be treated with hematopoietic cell transplantation (HCT) or immunosuppressive (IS) therapy. A retrospective cohort of 56 children with SAA undergoing transplantation with fludarabine-cyclophosphamide-ATG-based conditioning (FluCyATG) was analyzed. The endpoints were overall survival (OS), event-free survival (EFS), cumulative incidence (CI) of graft versus host disease (GVHD) and CI of viral replication. Engraftment was achieved in 53/56 patients, and four patients died (two due to fungal infection, and two of neuroinfection). The median time to neutrophil engraftment was 14 days and to platelet engraftment was 16 days, and median donor chimerism was above 98%. The overall incidence of acute GVHD was 41.5%, and that of grade III-IV acute GVHD was 14.3%. Chronic GVHD was diagnosed in 14.2% of children. The probability of 2-year GVHD-free survival was 76.1%. In the univariate analysis, a higher dose of cyclophosphamide and previous IS therapy were significant risk factors for worse overall survival. Episodes of viral replication occurred in 33/56 (58.9%) patients, but did not influence OS. The main advantages of FluCyATG include early engraftment with a very high level of donor chimerism, high overall survival and a low risk of viral replication after HCT.
重型再生障碍性贫血（SAA）是一种骨髓衰竭综合征，可通过造血细胞移植（HCT）或免疫抑制（IS）治疗。对56例接受基于氟达拉滨 - 环磷酰胺 - 抗胸腺细胞球蛋白预处理（FluCyATG）进行移植的SAA儿童进行了回顾性队列分析。终点指标为总生存期（OS）、无事件生存期（EFS）、移植物抗宿主病（GVHD）的累积发生率（CI）和病毒复制的CI。53/56例患者实现了造血重建，4例患者死亡（2例死于真菌感染，2例死于神经感染）。中性粒细胞植入的中位时间为14天，血小板植入的中位时间为16天，供体嵌合率中位数高于98%。急性GVHD的总发生率为41.5%，III-IV级急性GVHD的发生率为14.3%。14.2%的儿童被诊断为慢性GVHD。2年无GVHD生存期的概率为76.1%。单因素分析中，较高剂量的环磷酰胺和既往IS治疗是总生存期较差的显著危险因素。33/56（58.9%）例患者发生病毒复制事件，但未影响OS。FluCyATG的主要优点包括早期植入且供体嵌合率非常高、总生存期高以及HCT后病毒复制风险低。
