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美国临床实践中三重难治性多发性骨髓瘤患者的治疗模式、生存率、生活质量及医疗资源利用情况:来自Connect MM疾病登记处的研究结果

Treatment Patterns, Survival, Quality of Life, and Healthcare Resource Use Among Patients With Triple-Class Refractory Multiple Myeloma in US Clinical Practice: Findings From the Connect MM Disease Registry.

作者信息

Lee Hans C, Ramasamy Karthik, Weisel Katja, Abonour Rafat, Hardin James W, Rifkin Robert M, Ailawadhi Sikander, Terebelo Howard R, Durie Brian G M, Tang Derek, Joshi Prashant, Liu Liang, Jou Ying-Ming, Che Min, Hernandez Gabriela, Narang Mohit, Toomey Kathleen, Gasparetto Cristina, Wagner Lynne I, Jagannath Sundar

机构信息

The University of Texas MD Anderson Cancer Center, Houston, TX.

Department of Haematology, Oxford University Hospitals NHS Trust, University of Oxford, Oxford, UK.

出版信息

Clin Lymphoma Myeloma Leuk. 2023 Feb;23(2):112-122. doi: 10.1016/j.clml.2022.11.008. Epub 2022 Nov 23.

Abstract

BACKGROUND

Adults with triple-class refractory (TCR) multiple myeloma (MM) have limited treatment options and poor prognosis, but the burden of TCR MM has not been well characterized. This study evaluated treatment patterns, overall survival (OS), health-related quality of life (HRQoL), and healthcare resource use (HCRU) among patients with TCR MM in US clinical practice.

PATIENTS AND METHODS

Patients with TCR MM in the Connect MM Registry (NCT01081028; a large, US, multicenter, prospective observational cohort study of patients with newly diagnosed MM) were included. Patient characteristics, treatment patterns, HRQoL, and HCRU were analyzed using descriptive statistics. OS was calculated using Kaplan-Meier methodology for the overall cohort and for patients with/without ≥1 post-TCR line of therapy (LOT).

RESULTS

A total of 232 patients with TCR MM were included; 155 (67%) had ≥1 post-TCR LOT (post-TCR-Treated subgroup; median 9.9 months of follow-up). Most common post-TCR treatments were carfilzomib (47%), pomalidomide (40%), and daratumumab (26%); median treatment duration was 3.3 months. Median OS was 9.9 months in the overall population, 10.8 months in post-TCR-Treated patients, and 2.6 months for those with no new post-TCR LOT. HRQoL deteriorated and pain increased over 1 year of follow-up, with clinically meaningfully changes in EQ-5D (mean, -0.06 points) and FACT-G (mean, -9.9 points). 124 (53%) patients had ≥1 all-cause hospitalization and 58 (25%) had ≥1 MM-related hospitalization; median annualized length of stay was 35.3 and 42.9 days, respectively.

CONCLUSION

The burden of TCR MM is substantial, emphasizing the need for more effective treatment options in the TCR setting.

摘要

背景

患有三重耐药(TCR)多发性骨髓瘤(MM)的成年人治疗选择有限且预后较差,但TCR-MM的疾病负担尚未得到充分描述。本研究评估了美国临床实践中TCR-MM患者的治疗模式、总生存期(OS)、健康相关生活质量(HRQoL)和医疗资源使用(HCRU)情况。

患者与方法

纳入了Connect MM注册研究(NCT01081028;一项针对新诊断MM患者的大型美国多中心前瞻性观察队列研究)中的TCR-MM患者。使用描述性统计分析患者特征、治疗模式、HRQoL和HCRU。采用Kaplan-Meier方法计算整个队列以及接受/未接受≥1线TCR后治疗方案(LOT)患者的OS。

结果

共纳入232例TCR-MM患者;155例(67%)接受了≥1线TCR后LOT(TCR后治疗亚组;中位随访时间9.9个月)。最常见的TCR后治疗药物为卡非佐米(47%)、泊马度胺(40%)和达雷妥尤单抗(26%);中位治疗持续时间为3.3个月。总体人群的中位OS为9.9个月,TCR后治疗患者为10.8个月,未接受新的TCR后LOT的患者为2.6个月。在1年的随访中,HRQoL恶化且疼痛加剧,EQ-5D(平均,-0.06分)和FACT-G(平均,-9.9分)出现具有临床意义的变化。124例(53%)患者发生≥1次全因住院,58例(25%)患者发生≥1次MM相关住院;中位年化住院天数分别为35.3天和42.9天。

结论

TCR-MM的疾病负担较重,强调在TCR背景下需要更有效的治疗选择。

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