St Anna Children's Hospital and St Anna Children's Cancer Research Institute, Medical University of Vienna, Vienna, Austria.
Christian-Albrechts-University Kiel and University Medical Center Schleswig-Holstein, Kiel, Germany.
J Clin Oncol. 2023 Mar 1;41(7):1404-1422. doi: 10.1200/JCO.22.01297. Epub 2022 Oct 18.
PURPOSE: We aimed to study prognostic factors and efficacy of allogeneic hematopoietic stem-cell transplantation (allo-HSCT) in first remission of patients with noninfant childhood acute lymphoblastic leukemia (ALL) with 11q23/ rearrangements treated with chemotherapy regimens between 1995 and 2010. PATIENTS AND METHODS: Data were retrospectively retrieved from 629 patients with 11q23/-rearranged ALL from 17 members of the Ponte-di-Legno Childhood ALL Working Group. Clinical and biologic characteristics, early response assessed by minimal residual disease at the end of induction (EOI) therapy, and allo-HSCT were analyzed for their impact on outcomes. RESULTS: A specific 11q23/ translocation partner gene was identified in 84.3% of patients, with the most frequent translocations being t(4;11)(q21;q23) (n = 273; 51.5%), t(11;19)(q23;p13.3) (n = 106; 20.0%), t(9;11)(p21_22;q23) (n = 76; 14.3%), t(6;11)(q27;q23) (n = 20; 3.8%), and t(10;11)(p12;q23) (n = 14; 2.6%); 41 patients (7.7%) had less frequently identified translocation partner genes. Patient characteristics and early response varied among subgroups, indicating large biologic heterogeneity and diversity in therapy sensitivity among 11q23/-rearranged ALL. The EOI remission rate was 93.2%, and the 5-year event-free survival (EFS) for the entire cohort was 69.1% ± 1.9%, with a range from 41.7% ± 17.3% for patients with t(9;11)-positive T-ALL (n = 9) and 64.8% ± 3.0% for patients with t(4;11)-positive B-ALL (n = 266) to 91.2% ± 4.9% for patients with t(11;19)-positive T-ALL (n = 34). Low EOI minimal residual disease was associated with favorable EFS, and induction failure was particularly predictive of nonresponse to further therapy and relapse and poor EFS. In addition, EFS was not improved by allo-HSCT compared with chemotherapy only in patients with both t(4;11)-positive B-ALL (n = 64 51; = .10) and 11q23/-rearranged T-ALL (n = 16 10; = .69). CONCLUSION: Compared with historical data, prognosis of patients with noninfant 11q23/-rearranged ALL has improved, but allo-HSCT failed to affect outcome. Targeted therapies are needed to reduce relapse and treatment-related mortality rates.
目的：我们旨在研究在 1995 年至 2010 年间采用化疗方案治疗的伴有 11q23/重排的非婴儿期儿童急性淋巴细胞白血病（ALL）患者在首次缓解期接受异基因造血干细胞移植（allo-HSCT）的预后因素和疗效。
方法：我们从 Ponte-di-Legno 儿童 ALL 工作组的 17 个成员中回顾性地获取了 629 例 11q23/-重排 ALL 患者的数据。分析了临床和生物学特征、诱导结束时最小残留病（EOI）评估的早期反应以及 allo-HSCT 对结局的影响。
结果：在 84.3%的患者中鉴定出了特定的 11q23/易位伙伴基因，最常见的易位为 t(4;11)(q21;q23)（n=273；51.5%）、t(11;19)(q23;p13.3)（n=106；20.0%）、t(9;11)(p21_22;q23)（n=76；14.3%）、t(6;11)(q27;q23)（n=20；3.8%）和 t(10;11)(p12;q23)（n=14；2.6%）；41 例患者（7.7%）的易位伙伴基因较少。患者特征和早期反应在亚组之间存在差异，表明伴有 11q23/-重排的 ALL 存在较大的生物学异质性和治疗敏感性多样性。EOI 缓解率为 93.2%，整个队列的 5 年无事件生存（EFS）为 69.1%±1.9%，范围从 t(9;11)-阳性 T-ALL（n=9）患者的 41.7%±17.3%和 t(4;11)-阳性 B-ALL（n=266）患者的 64.8%±3.0%到 t(11;19)-阳性 T-ALL（n=34）患者的 91.2%±4.9%。EOI 微小残留病低与 EFS 良好相关，诱导失败尤其预示着对进一步治疗和复发的无反应和不良 EFS。此外，与单独化疗相比，allo-HSCT 并未改善伴有 t(4;11)-阳性 B-ALL（n=64 51； =.10）和 11q23/-重排 T-ALL（n=16 10； =.69）患者的 EFS。
结论：与历史数据相比，非婴儿期伴有 11q23/-重排的 ALL 患者的预后有所改善，但 allo-HSCT 并未影响结局。需要靶向治疗来降低复发和治疗相关死亡率。
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