Trab Trine, Chanchiri Iman, Al-Mashhadi Ahmed Ludvigsen, Dall Emma Berggren, Rasch Stine, Johansen Mette, Husby Simon, Simonsen Mikkel Runason, Clausen Michael Roost, Larsen Thomas Stauffer, Christensen Jacob Haaber, Pedersen Robert Schou, Frederiksen Mikael, Jerkeman Mats, Poulsen Christian Bjørn, Haunstrup Laura Mors, Brown Peter, El-Galaly Tarec Christoffer, Grønbæk Kirsten
Department of Hematology, Rigshospitalet, Copenhagen, Denmark.
Biotech Research and Innovation Center, University of Copenhagen, Copenhagen, Denmark.
Blood Neoplasia. 2025 Jun 9;2(3):100128. doi: 10.1016/j.bneo.2025.100128. eCollection 2025 Aug.
Ibrutinib was approved for relapsed/refractory (R/R) mantle cell lymphoma (MCL) based on high response rates in clinical trials, but it is unclear how effective ibrutinib is in the real-world setting. This study provides population-based response rates and survival estimates and characterization of prognostic indicators and adverse events (AEs) to ibrutinib for patients with R/R MCL. All patients diagnosed with MCL in Denmark from 2010 to 2022 were identified in the Danish Lymphoma Registry and screened for eligibility. Data were collected from health records. Patients receiving ibrutinib in second or later lines were included and followed from ibrutinib start until death or last follow-up. End points were overall response rate (ORR), progression-free survival (PFS), overall survival (OS), frequency of AEs, and AE-related discontinuation and dose reductions. In total, 146 patients were included (median age, 73 years); 90 (62%) received ibrutinib in second line. ORR was 56%, median PFS 5.8 months, and median OS 12.0 months. In Cox regressions, factors associated with inferior PFS were Ki67 of ≥50% (hazard ratio [HR], 2.34; 95% confidence interval [CI], 1.47-3.71), blastoid or pleomorphic subtype (HR, 3.00; 95% CI, 2.04-4.41), early relapses (HR, 1.65; 95% CI, 1.15-2.36), and refractory disease (HR, 1.57; 95% CI, 1.07-2.30). Three-year cumulative incidences of discontinuation and dose reductions owing to AEs were 19% and 22%, respectively. Median OS after ibrutinib discontinuation was 1.9 months. In conclusion, real-world outcomes after initiation of ibrutinib for R/R MCL were poorer than observed in clinical trials, and dose-limiting toxicities were common, emphasizing the need for more effective treatments and dose-optimization studies.
依鲁替尼基于临床试验中的高缓解率被批准用于复发/难治性(R/R)套细胞淋巴瘤(MCL),但尚不清楚依鲁替尼在现实环境中的有效性如何。本研究提供了基于人群的缓解率、生存估计以及R/R MCL患者使用依鲁替尼的预后指标和不良事件(AE)特征。在丹麦淋巴瘤登记处识别出2010年至2022年期间在丹麦诊断为MCL的所有患者,并筛选其是否符合条件。数据从健康记录中收集。纳入接受二线或更后线依鲁替尼治疗的患者,并从依鲁替尼开始使用直至死亡或末次随访进行随访。终点指标为总缓解率(ORR)、无进展生存期(PFS)、总生存期(OS)、AE的发生频率以及与AE相关的停药和剂量减少情况。总共纳入了146例患者(中位年龄73岁);90例(62%)接受二线依鲁替尼治疗。ORR为56%,中位PFS为5.8个月,中位OS为12.0个月。在Cox回归分析中,与较差PFS相关的因素包括Ki67≥50%(风险比[HR],2.34;95%置信区间[CI],1.47 - 3.71)、母细胞样或多形性亚型(HR,3.00;95%CI,2.04 - 4.41)、早期复发(HR,1.65;95%CI,1.15 - 2.36)以及难治性疾病(HR,1.57;95%CI,1.07 - 2.30)。因AE导致停药和剂量减少的3年累积发生率分别为19%和22%。依鲁替尼停药后的中位OS为1.9个月。总之,R/R MCL患者开始使用依鲁替尼后的实际疗效比临床试验中观察到的要差,且剂量限制性毒性常见,这凸显了对更有效治疗方法和剂量优化研究的需求。