Department I - General Pediatrics, Hematology/Oncology, Children's Hospital, University Hospital Tübingen, Tübingen, Germany.
Department of Pediatrics, University Frankfurt, Frankfurt, Germany.
Eur J Haematol. 2021 Apr;106(4):473-483. doi: 10.1111/ejh.13569. Epub 2021 Jan 11.
Pediatric patients with relapsed or refractory acute lymphoblastic leukemia have a poor prognosis. We here assess the response rates, adverse events, and long-term follow-up of pediatric patients with relapsed/refractory acute lymphoblastic leukemia receiving blinatumomab.
Retrospective analysis of a single-center experience with blinatumomab in 38 patients over a period of 10 years.
The median age at onset of therapy was 10 years (1-21 years). Seventy-one percent of patients had undergone at least one hematopoietic stem cell transplantation (HSCT) prior to treatment with blinatumomab. We observed a response to blinatumomab in 13/38 patients (34%). The predominant side effect was febrile reactions, nearly half of the patients developed a cytokine release syndrome. Eight events of neurotoxicity were registered over the 78 cycles (15%). To date, nine patients (24%) are alive and in complete molecular remission. All survivors underwent haploidentical HSCT after treatment with blinatumomab.
Despite heavy pretreatment of most of our patients, severe adverse events were rare and response rates encouraging. Blinatumomab is a valuable bridging salvage therapy for relapsed or refractory patients to a second or even third HSCT.
患有复发或难治性急性淋巴细胞白血病的儿科患者预后较差。我们在此评估接受blinatumomab 治疗的复发/难治性急性淋巴细胞白血病儿科患者的缓解率、不良事件和长期随访结果。
对 10 年间单中心使用blinatumomab 的 38 例患者进行回顾性分析。
治疗时的中位年龄为 10 岁（1-21 岁）。71%的患者在接受blinatumomab 治疗前至少接受过一次造血干细胞移植（HSCT）。我们观察到 38 例患者中有 13 例（34%）对blinatumomab 有反应。主要的不良反应是发热反应，近一半的患者发生细胞因子释放综合征。在 78 个周期中记录到 8 例神经毒性事件（15%）。迄今为止，9 名患者（24%）存活且处于完全分子缓解状态。所有幸存者在接受blinatumomab 治疗后均进行了单倍体 HSCT。
尽管大多数患者均有大量预处理，但严重不良事件罕见，缓解率令人鼓舞。blinatumomab 是复发或难治性患者进行第二次甚至第三次 HSCT 的有价值的桥接挽救性治疗。
