Ragoonanan Dristhi, Bhar Saleh, Mohan Gopi, Beltramo Fernando, Khazal Sajad J, Hurley Caitlin, Andersen Clark, Margossian Steven, Neelapu Sattva S, Shpall Elizabeth, Gutierrez Cristina, Tewari Priti, Shoberu Basirat, Talleur Aimee, McCall David, Nunez Cesar, Cuglievan Branko, Tambaro Francesco Paolo, Petropoulos Demetrios, Abdel-Azim Hisham, Mahadeo Kris M
Department of Pediatrics, Stem Cell Transplantation and Cellular Therapy, University of Texas MD Anderson Cancer Center, Houston, TX, United States.
Texas Children's Hospital, Baylor College of Medicine, Houston, TX, United States.
Front Oncol. 2022 Oct 24;12:1022901. doi: 10.3389/fonc.2022.1022901. eCollection 2022.
Tisagenlecleucel is associated with remarkable outcomes in treating patients up to the age of 25 years with refractory B-cell acute lymphoblastic leukemia (ALL). Yet, due to unique and potentially life-threatening complications, access remains limited to higher-resource and certified centers. Reports of inequity and related disparities in care are emerging. In this multicenter study of ALL patients admitted for anti-leukemia therapy, who required pediatric intensive care (ICU) support (n = 205), patients receiving tisagenlecleucel (n = 39) were compared to those receiving conventional chemotherapy (n = 166). The median time to ICU transfer was 6 (0-43) versus 1 (0-116) days, respectively (p < 0.0001). There was no difference in the use of vasopressor, ionotropic, sedating, and/or paralytic agents between groups, but use of dexamethasone was higher among tisagenlecleucel patients. Patients receiving tisagenlecleucel were more likely to have cardiorespiratory toxicity (p = 0.0002), but there were no differences in diagnostic interventions between both groups and/or differences in ICU length of stay and/or overall hospital survival. Toxicities associated with tisagenlecleucel are generally reversible, and our findings suggest that resource utilization once admitted to the ICU may be similar among patients with ALL receiving tisagenlecleucel versus conventional chemotherapy. As centers consider improved access to care and the feasibility of tisagenlecleucel certification, our study may inform strategic planning.
替沙格赛定在治疗25岁及以下难治性B细胞急性淋巴细胞白血病（ALL）患者方面具有显著疗效。然而，由于存在独特且可能危及生命的并发症，其应用仍局限于资源更丰富且具备认证资质的中心。关于医疗不公平及相关差异的报道不断涌现。在这项针对因抗白血病治疗入院且需要儿科重症监护（ICU）支持的ALL患者的多中心研究中（n = 205），将接受替沙格赛定治疗的患者（n = 39）与接受传统化疗的患者（n = 166）进行了比较。转至ICU的中位时间分别为6（0 - 43）天和1（0 - 116）天（p < 0.0001）。两组之间在使用血管加压药、离子otropic、镇静和/或麻痹剂方面无差异，但替沙格赛定治疗的患者中地塞米松的使用更高。接受替沙格赛定治疗的患者更易出现心肺毒性（p = 0.0002），但两组在诊断干预、ICU住院时长和/或总体医院生存率方面无差异。与替沙格赛定相关的毒性通常是可逆的，我们的研究结果表明，ALL患者接受替沙格赛定治疗与接受传统化疗相比，入住ICU后的资源利用情况可能相似。随着各中心考虑改善医疗可及性以及替沙格赛定认证的可行性，我们的研究可能为战略规划提供参考。
