Rodríguez-Arbolí Eduardo, Rodríguez-Veiga Rebeca, Soria-Saldise Elena, Bergua Juan M, Caballero-Velázquez Teresa, Arnán Montserrat, Vives Susana, Serrano Josefina, Bernal Teresa, Martínez-Sánchez Pilar, Tormo Mar, Rodríguez-Medina Carlos, Herrera-Puente Pilar, Lavilla-Rubira Esperanza, Boluda Blanca, Acuña-Cruz Evelyn, Cano Isabel, Cáceres Sara, Ballesteros Juan, Falantes José, Martínez-Cuadrón David, Pérez-Simón José A, Montesinos Pau
Department of Hematology, Hospital Universitario Virgen del Rocío, Instituto de Biomedicina de Sevilla (IBiS/CSIC), University of Seville, Seville, Spain.
Department of Hematology, Hospital Universitari i Politècnic La Fe, Valencia, Spain.
Cancer. 2025 Jan 1;131(1):e35618. doi: 10.1002/cncr.35618. Epub 2024 Oct 30.
LAMVYX was a multicenter, single-arm, phase 2 trial designed to validate the safety and efficacy of CPX-351 in patients aged 60-75 years with newly diagnosed, secondary acute myeloid leukemia and to generate evidence on key issues not addressed in the preceding regulatory pivotal trial.
The primary end point of the study was the complete remission (CR)/CR with incomplete hematologic recovery (CRi) rate after induction. Eligible patients were recommended to undergo allogeneic hematopoietic stem cell transplantation after the first consolidation cycle. Alternatively, patients could undergo up to six maintenance cycles with CPX-351.
Twenty-nine patients (49%; 95% exact confidence interval [CI], 37%-62%) patients achieved a CR/CRi after one or two cycles of induction, with a measurable residual disease negativity rate of 67% as assessed by centralized, multiparameter flow cytometry. Among patients who had serial next-generation sequencing analyses available, clearance of somatic mutations that were present at diagnosis was achieved in 7 (35%). The median follow-up among survivors was 16.8 months (range, 8.7-24.3 months). The median event-free survival was 3.0 months (95% CI, 1.4-7.3 months), and the median overall survival was 7.4 months (95% CI, 3.7-12.7 months). In landmark analyses at day +100 from diagnosis, the 1-year overall and event-free survival rate among patients who underwent allogeneic hematopoietic stem cell transplantation was 70% (95% CI, 47%-100%) and 70% (95% CI, 47%-100%), respectively. The corresponding values were 89% (95% CI, 71%-100%) and 44% (95% CI, 21%-92%), respectively, for patients who entered the maintenance phase. No significant longitudinal changes were observed in severity index or quality-of-life visual analog scale scores.
The current data provide novel insights that might inform the clinical positioning and optimal use of CPX-351, complementing previous results (ClinicalTrials.gov identifier NCT04230239).
LAMVYX是一项多中心、单臂2期试验，旨在验证CPX-351对年龄在60 - 75岁、新诊断的继发性急性髓系白血病患者的安全性和有效性，并为之前监管关键试验中未涉及的关键问题提供证据。
该研究的主要终点是诱导后完全缓解（CR）/伴有血细胞计数未完全恢复的CR（CRi）率。符合条件的患者在第一个巩固周期后建议接受异基因造血干细胞移植。或者，患者可以接受多达六个周期的CPX-351维持治疗。
29例患者（49%；95%确切置信区间[CI]，37% - 62%）在1个或2个周期诱导后达到CR/CRi，通过集中式多参数流式细胞术评估，可测量残留病阴性率为67%。在可进行系列下一代测序分析的患者中，7例（35%）实现了诊断时存在的体细胞突变清除。幸存者的中位随访时间为16.8个月（范围8.7 - 24.3个月）。中位无事件生存期为3.0个月（95% CI，1.4 - 7.3个月），中位总生存期为7.4个月（95% CI，3.7 - 12.7个月）。在诊断后第100天的标志性分析中，接受异基因造血干细胞移植患者的1年总生存率和无事件生存率分别为70%（95% CI，47% - 100%）和70%（95% CI，47% - 100%）。进入维持期患者的相应值分别为89%（95% CI，71% - 100%）和44%（95% CI，21% - 92%）。严重程度指数或生活质量视觉模拟量表评分未观察到显著的纵向变化。
当前数据提供了新的见解，可能为CPX-351的临床定位和最佳使用提供参考，补充了先前的结果（ClinicalTrials.gov标识符NCT04230239）。
