Patel Anand Ashwin, Heng Joseph, Dworkin Emily, Monick Sarah, Derman Benjamin A, DuVall Adam S, Gurbuxani Sandeep, Kosuri Satyajit, Liu Hongtao, Thirman Michael, Godley Lucy A, Odenike Olatoyosi, Larson Richard A, Stock Wendy
Department of Medicine Section of Hematology-Oncology University of Chicago Chicago Illinois USA.
Department of Pharmacy University of Chicago Chicago Illinois USA.
EJHaem. 2021 Jun 22;2(3):413-420. doi: 10.1002/jha2.224. eCollection 2021 Aug.
Although acute lymphoblastic leukemia (ALL) is most common in pediatric and adolescent and young adult (AYA) patients, 20% of cases are diagnosed in patients ≥ 55 years old. Use of intensive pediatric regimens in AYA populations has demonstrated excellent tolerability and significant improvements in event-free survival (EFS) and overall survival (OS). The backbone of pediatric regimens includes asparaginase and corticosteroids, both of which are associated with more toxicity in older patients and those with body mass index (BMI) ≥ 30 kg/m which leads to poor tolerance of these regimens. We tested the safety and efficacy of a dose-modified The Cancer and Leukemia Group B 10403 regimen using reduced doses of pegylated (PEG)-asparaginase (ASP) and corticosteroids (RD-10403) in 30 patients with Philadelphia-chromosome negative ALL who were ≥50-year-old and younger adults with significant metabolic or hepatic co-morbidities. The complete remission rate on day 28 was 77%, 3-year EFS was 54%, and estimated 3-year OS was 55%. Grade 3+ toxicity was noted in 40% of patients during induction, and induction-related mortality was 3%. Additional prospective evaluation of RD-10403 is merited to determine efficacy and safety of this regimen and to serve as a framework for chemoimmunotherapy combination therapy.
尽管急性淋巴细胞白血病(ALL)在儿童、青少年和青年成人(AYA)患者中最为常见,但20%的病例是在年龄≥55岁的患者中确诊的。在AYA人群中使用强化儿科方案已显示出良好的耐受性,无事件生存期(EFS)和总生存期(OS)有显著改善。儿科方案的主要组成部分包括天冬酰胺酶和皮质类固醇,这两种药物在老年患者和体重指数(BMI)≥30kg/m²的患者中都与更多毒性相关,导致这些方案的耐受性较差。我们在30例年龄≥50岁且患有严重代谢或肝脏合并症的费城染色体阴性ALL的年轻成人患者中,测试了一种剂量调整的癌症与白血病B组10403方案(使用降低剂量的聚乙二醇化(PEG)-天冬酰胺酶(ASP)和皮质类固醇(RD-10403))的安全性和疗效。第28天的完全缓解率为77%,3年EFS为54%,估计3年OS为55%。40%的患者在诱导期出现3级及以上毒性,诱导相关死亡率为3%。值得对RD-10403进行进一步的前瞻性评估,以确定该方案的疗效和安全性,并作为化疗免疫联合治疗的框架。