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复发/难治性多发性骨髓瘤三联疗法的医疗成本和治疗模式:来自意大利的真实世界证据

Healthcare Costs and Treatment Patterns of Triplet Therapies in Relapsed/Refractory Multiple Myeloma: Real World Evidence from Italy.

作者信息

Perrone Valentina, Leogrande Melania, Giacomini Elisa, Cappuccilli Maria, Degli Esposti Luca

机构信息

CliCon S.R.L. Società Benefit, Health Economics and Outcomes Research, Bologna, Italy.

出版信息

Clinicoecon Outcomes Res. 2025 Jul 19;17:495-505. doi: 10.2147/CEOR.S529788. eCollection 2025.

Abstract

PURPOSE

This analysis sought to characterize patients with relapsed or refractory multiple myeloma (RRMM) on triplet therapy with immunomodulatory agents/proteasome inhibitors/monoclonal antibodies combined with dexamethasone, describing their demographic and clinical features, therapeutic pathways and the related healthcare costs for the Italian National Health Service (NHS).

PATIENTS AND METHODS

A retrospective observational analysis was conducted on administrative databases of Italian healthcare entities, covering about 3.6 million health-assisted residents. From 2017 to 2020, patients receiving at least one triplet combination reimbursed by the Italian NHS for the treatment of RRMM were included. RRMM treatment pathways were evaluated in terms of duration of therapy and treatment lines. Healthcare costs per patient were assessed on monthly basis during the therapy period by computing expenditures for drugs, hospitalizations and outpatient specialist services.

RESULTS

A total of 209 RRMM patients on triplet combinations were identified, with a mean age of 67.4 years, 56% males and mild-to-moderate comorbidity profile, with heart disease and renal disease as the most common coexisting conditions (respectively, 13.4% and 7.7% of patients). KRd (carfilzomib/lenalidomide/dexamethasone) was the triplet administrated to the largest proportion of patients (44%), then DaraRd (daratumumab/lenalidomide/dexamethasone) triplet (24%). Treatment duration was on average 7 months for overall patients, 7.7 months for 118 patients with triplet as second line at inclusion, and 6 months for 91 patients with triplet as third or further line at inclusion. The monthly overall costs were € 9,517, with drug expenses accounting for 93% of total expenditures. Progressing to later treatment lines, cost analysis showed comparable trends, being drugs as the most impactive item.

CONCLUSION

This analysis on RRMM patients under triplet medication in real-life Italian clinical practice confirmed the complex multifaceted profile of this frail population, highlighting a challenging clinical management for the oncologists and a major economic burden for the NHS.

摘要

目的

本分析旨在描述接受免疫调节剂/蛋白酶体抑制剂/单克隆抗体联合地塞米松的三联疗法治疗的复发或难治性多发性骨髓瘤(RRMM)患者的特征,阐述其人口统计学和临床特征、治疗途径以及意大利国家医疗服务体系(NHS)的相关医疗费用。

患者与方法

对意大利医疗保健机构的行政数据库进行回顾性观察分析,涵盖约360万接受医疗救助的居民。纳入2017年至2020年期间接受意大利NHS报销的至少一种三联组合疗法治疗RRMM的患者。从治疗持续时间和治疗线数方面评估RRMM的治疗途径。通过计算药物、住院和门诊专科服务的费用,在治疗期间每月评估每位患者的医疗费用。

结果

共确定了209例接受三联组合疗法的RRMM患者,平均年龄67.4岁,男性占56%,合并症为轻度至中度,最常见的并存疾病是心脏病和肾病(分别占患者的13.4%和7.7%)。KRd(卡非佐米/来那度胺/地塞米松)是应用于最大比例患者(44%)的三联疗法,其次是DaraRd(达雷妥尤单抗/来那度胺/地塞米松)三联疗法(24%)。总体患者的治疗持续时间平均为7个月,纳入时将三联疗法作为二线治疗的118例患者为7.7个月,纳入时将三联疗法作为三线或更后线治疗的91例患者为6个月。每月总费用为9517欧元,药物费用占总支出的93%。随着治疗线数的增加,成本分析显示出类似趋势,药物是影响最大的项目。

结论

对意大利现实临床实践中接受三联药物治疗的RRMM患者的本分析证实了这一脆弱人群复杂多面的特征,凸显了肿瘤学家面临的具有挑战性的临床管理以及NHS的重大经济负担。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/fcec/12285882/40e6fb8ace67/CEOR-17-495-g0001.jpg

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