盐酸米托蒽醌脂质体注射液治疗外周T细胞淋巴瘤有效性和安全性的多中心、非干预性、双向性队列真实世界研究(MOMENT)

Efficacy and safety of mitoxantrone hydrochloride liposome injection in treatment of peripheral T-cell lymphomas:a multicenter,non-interventional,ambispective cohort,real-world study(MOMENT)

目的评价真实世界中盐酸米托蒽醌脂质体注射液治疗外周T细胞淋巴瘤(PTCL)的有效性和安全性。方法本研究为双向性队列的真实世界研究(MOMENT研究)(中国临床试验注册号:ChiCTR2200062067)。收集2022年1月至2023年1月我国37家医院接受盐酸米托蒽醌脂质体注射液单药或联合治疗的198例患者临床资料,其中回顾性队列166例,前瞻性队列32例;初治10例,复发难治188例。总结患者临床特征、疗效及不良事件发生情况,分析总生存(OS)和无进展生存(PFS)情况。结果198例患者接受盐酸米托蒽醌脂质体注射液治疗的中位周期数为3个(范围1~7个);盐酸米托蒽醌脂质体注射液单药治疗28例,联合方案治疗170例。188例复发难治患者中,完全缓解(CR)45例(23.9%),部分缓解(PR)82例(43.6%),疾病稳定(SD)28例(14.9%),疾病进展(PD)33例(17.6%),客观缓解率(ORR)为67.6%(127/188);10例初治患者中,CR 4例(40.0%),PR 5例(50.0%),PD 1例(10.0%),ORR为90.0%(9/10)。中位随访时间为2.9个月(95%CI 2.4~3.7个月),复发难治组及初治组患者的中位PFS和OS时间均未达到。复发难治患者中,盐酸米托蒽醌脂质体注射液不同治疗线数患者的ORR[二线、三线、≥四线分别为74.4%(67/90)、73.9%(34/46)、50.0%(26/52)]比较,差异有统计学意义(P=0.008)。198例PTCL患者中,182例(91.9%)发生了至少1次治疗相关的不良事件,≥3级不良事件发生率为66.7%(132/198),主要以血液学不良事件为主。≥3级血液学不良事件主要包括淋巴细胞计数降低、中性粒细胞计数降低、白细胞数降低及贫血;非血液学不良事件多为1~2级,主要包括色素沉着障碍和上呼吸道感染。结论采用含盐酸米托蒽醌脂质体注射液方案治疗PTCL具有确切的疗效,且耐受性较好,是PTCL患者新的治疗选择。

Objective To evaluate the efficacy and safety of mitoxantrone hydrochloride liposome injection in the treatment of peripheral T-cell lymphoma(PTCL)in a real-world setting.Methods This was a real-world ambispective cohort study(MOMENT study)(Chinese clinical trial registry number:ChiCTR2200062067).Clinical data were collected from 198 patients who received mitoxantrone hydrochloride liposome injection as monotherapy or combination therapy at 37 hospitals from January 2022 to January 2023,including 166 patients in the retrospective cohort and 32 patients in the prospective cohort;10 patients in the treatment-naïve group and 188 patients in the relapsed/refractory group.Clinical characteristics,efficacy and adverse events were summarized,and the overall survival(OS)and progression-free survival(PFS)were analyzed.Results All 198 patients were treated with mitoxantrone hydrochloride liposome injection for a median of 3 cycles(range 1-7 cycles);28 cases were treated with mitoxantrone hydrochloride liposome injection as monotherapy,and 170 cases were treated with the combination regimen.Among 188 relapsed/refractory patients,45 cases(23.9%)were in complete remission(CR),82 cases(43.6%)were in partial remission(PR),and 28 cases(14.9%)were in disease stabilization(SD),and 33 cases(17.6%)were in disease progression(PD),with an objective remission rate(ORR)of 67.6%(127/188).Among 10 treatment-naïve patients,4 cases(40.0%)were in CR,5 cases(50.0%)were in PR,and 1 case(10.0%)was in PD,with an ORR of 90.0%(9/10).The median follow-up time was 2.9 months(95%CI 2.4-3.7 months),and the median PFS and OS of patients in relapsed/refractory and treatment-naïve groups were not reached.In relapsed/refractory patients,the difference in ORR between patients with different number of treatment lines of mitoxantrone hydrochloride liposome injection[ORR of the second-line,the third-line and≥the forth-line treatment was 74.4%(67/90),73.9%(34/46)and 50.0%(26/52)]was statistically significant(P=0.008).Of the 198 PTCL patients,182 cases(91.9%)experienced at least 1 time of treatment-related adverse events,and the incidence rate of≥grade 3 adverse events was 66.7%(132/198),which was mainly characterized by hematologic adverse events.The≥grade 3 hematologic adverse events mainly included decreased lymphocyte count,decreased neutrophil count,decreased white blood cell count,and anemia;non-hematologic adverse events were mostly grade 1-2,mainly including pigmentation disorders and upper respiratory tract infection.Conclusions The use of mitoxantrone hydrochloride liposome injection-containing regimen in the treatment of PTCL has definite efficacy and is well tolerated,and it is a new therapeutic option for PTCL patients.