Hemato-Oncology Department, DMU DHI, Hôpital Saint Louis, Paris, France.
Inserm U1153 - ECSTRRA Team, Paris, France.
Leuk Lymphoma. 2022 Jul;63(7):1544-1555. doi: 10.1080/10428194.2022.2032038. Epub 2022 Feb 24.
Marginal zone lymphoma (MZL) is a heterogeneous disease and has various end-point measures. Our aim was to describe the endpoints used in trials involving patients with MZL. We searched over the last 35 years via PubMed, The Cochrane Library, clinicaltrials.govandclinicaltrialsregister.eu for published and registered clinical trials using the keyword "marginalzone lymphoma." We excluded studies focusing on pediatric populations, cutaneous MZL and on use of allogenic stem cell transplant. Endpoints were reviewed as well as their influencing factors and their definitions. Among 1192 references Q7 dentified by initial screening, 309 references were included (111 published, 198 registered), with 213 (69%) phase 2, 65 (21%) phase 1/2 and 31 (10%) phase 3 trials. The majority were open-label (n¼295, 95%) non-randomized (n¼256, 83%) trials, concerned all subtypes of MZLs at once (n¼239, 77%), and were often merged with non-MZL patients (n¼232, 75%). Among phase 1/2 and 2 trials, Overall/complete response rate (ORR/CRR) (n¼196, 70.5%) and progression-free survival (PFS,n¼28, 10.1%) were the most used primary endpoints; in phase 3 trials PFS was the most used primary endpoint (n¼18, 58.1%; ORR/CRR n¼6, 19.4%, p<0.001). Overall, the most frequent secondary endpoints were overall survival (OS, n¼153, 50%), PFS (n¼142, 46%) and ORR/CRR (n¼116, 38%). Distribution was similar when considering trials with only patients with MZL. Endpoints definitions were inconsistent across published trials (up to 9 definitions per endpoint). Trials involving patients with MZL showed marked heterogeneity both in the choice and definitions of primary and secondary endpoints, thus hampering comparability between trials.
边缘区淋巴瘤（MZL）是一种异质性疾病，有多种终点衡量指标。我们的目的是描述涉及 MZL 患者的试验中使用的终点。我们通过 PubMed、Cochrane 图书馆、clinicaltrials.gov 和 clinicaltrialsregister.eu 搜索了过去 35 年中使用关键字“marginalzone lymphoma”发表和注册的临床试验。我们排除了专注于儿科人群、皮肤 MZL 和异基因干细胞移植的研究。我们还审查了终点及其影响因素和定义。通过初步筛选确定的 1192 条参考文献 Q7，共纳入 309 条参考文献（111 条已发表，198 条已注册），其中 213 项（69%）为 2 期，65 项（21%）为 1/2 期，31 项（10%）为 3 期试验。大多数为开放标签（n¼295，95%）非随机（n¼256，83%）试验，同时涉及所有 MZL 亚型（n¼239，77%），且经常与非 MZL 患者合并（n¼232，75%）。在 1/2 期和 2 期试验中，总缓解率（ORR/CRR）（n¼196，70.5%）和无进展生存期（PFS，n¼28，10.1%）是最常用的主要终点；在 3 期试验中，PFS 是最常用的主要终点（n¼18，58.1%；ORR/CRR n¼6，19.4%，p<0.001）。总体而言，最常见的次要终点是总生存期（OS，n¼153，50%）、PFS（n¼142，46%）和 ORR/CRR（n¼116，38%）。当仅考虑 MZL 患者的试验时，分布情况相似。发表试验中终点的定义不一致（每个终点最多有 9 种定义）。涉及 MZL 患者的试验在主要和次要终点的选择和定义方面存在明显的异质性，从而阻碍了试验之间的可比性。
