Ilan Uri, Brivio Erica, Algeri Mattia, Balduzzi Adriana, Gonzalez-Vincent Marta, Locatelli Franco, Zwaan Christian Michel, Baruchel Andre, Lindemans Caroline, Bautista Francisco
Princess Máxima Center for Pediatric Oncology, 3584 CS Utrecht, The Netherlands.
Department of Hematology/Oncology and Cell and Gene Therapy, Bambino Gesù Children Hospital, 00165 Rome, Italy.
J Clin Med. 2023 Mar 9;12(6):2149. doi: 10.3390/jcm12062149.
Hematopoietic stem cell transplantation (HSCT) is often the only curative treatment option for patients suffering from various types of malignant diseases and some non-cancerous conditions. Nevertheless, it is associated with a high risk of complications leading to transplant-related mortality and long-term morbidity. An increasing number of therapeutic and prevention strategies have been developed over the last few years to tackle the complications arising in patients receiving an HSCT. These strategies have been mainly carried out in adults and some are now being translated into children. In this manuscript, we review the recent advancements in the development and implementation of treatment options for post-HSCT non-infectious complications in pediatric patients with leukemia and other non-malignant conditions, with a special attention on the new agents available within clinical trials. We focused on the following conditions: graft failure, prevention of relapse and early interventions after detection of minimal residual disease positivity following HSCT in acute lymphoblastic and myeloid leukemia, chronic graft versus host disease, non-infectious pulmonary complications, and complications of endothelial origin.
造血干细胞移植(HSCT)通常是患有各种恶性疾病和一些非癌性疾病患者的唯一治愈性治疗选择。然而,它与导致移植相关死亡率和长期发病率的高并发症风险相关。在过去几年中,已经开发出越来越多的治疗和预防策略来应对接受HSCT患者出现的并发症。这些策略主要在成人中实施,现在有些正在应用于儿童。在本手稿中,我们回顾了白血病和其他非恶性疾病的儿科患者HSCT后非感染性并发症治疗方案开发与实施的最新进展,特别关注临床试验中可用的新药物。我们重点关注以下情况:移植物失败、预防复发以及在急性淋巴细胞白血病和髓细胞白血病HSCT后检测到微小残留病阳性后的早期干预、慢性移植物抗宿主病、非感染性肺部并发症以及内皮源性并发症。