CAR-T(CAR-T)细胞疗法在非肿瘤性疾病中的应用
Chimeric antigen receptor T (CAR-T) cell therapy in non-oncological diseases.
CAR-T(CAR-T)细胞在血液系统恶性肿瘤中的应用推动了这种免疫治疗形式的显著进展。
CELL INTELLIGENCE · 肿瘤细胞治疗研究
肿瘤细胞治疗研究
英文原题:Burden of illness among patients with relapsed or refractory multiple myeloma, and eligible for B-cell maturation antigen-targeted therapies.
Burden of illness among patients with relapsed or refractory multiple myeloma, and eligible for B-cell maturation antigen-targeted therapies.
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重度经治的 RRMM 患者承受着较高的疾病负担,可能从新型疗法中获益。
靶向B细胞成熟抗原(BCMA)的疗法已获批用于接受过4线治疗、且既往暴露于三类药物的复发/难治性多发性骨髓瘤(RRMM)患者。
本项基于理赔数据的回顾性分析,考察了2016年1月1日至2022年9月30日期间符合BCMA靶向疗法条件并开始新治疗的美国患者,其临床和经济负担。
符合条件的228例患者(中位年龄64岁,男性52.6%,白人68.7%)常见骨髓瘤相关疾病和并发症,治疗失败率较高(6个月内为44.7%)。住院率从48.3%升至71.1%;每名患者每月医疗费用从12个月基线期的35,964美元升至随访期的41,358美元(中位随访11.8个月)。
既往接受多线治疗的RRMM患者疾病负担沉重,有望从新型疗法中获益。本文摘要介绍什么?尽管治疗选择已有显著进展,多发性骨髓瘤(MM)仍无法治愈。接受多线治疗的患者最终会产生耐药并复发或转为难治。需要新治疗的患者选择有限。本研究考察接受过4线治疗且可能符合新获批BCMA靶向CAR-T 细胞或双特异性疗法条件的复发/难治性MM(RRMM)患者,其临床和经济负担。结果如何?研究发现,美国患者中很大比例存在MM相关疾病和合并症,如贫血、高血压和肾功能障碍。开始新一线治疗后,每名患者每月医疗费用由基线期的35,964美元升至随访期的41,358美元,原因是随访期间住院、治疗及门诊费用增加。6个月内治疗失败较为常见(44.7%)。这些结果意味着什么?既往接受多线RRMM治疗的患者承受显著临床和经济负担。获得新型疗法(如BCMA靶向CAR-T 或双特异性抗体治疗)可能减轻RRMM患者的疾病负担。
B-cell maturation antigen (BCMA)-targeted therapies were approved for triple-class exposed relapsed/refractory multiple myeloma (RRMM) after 4 lines of therapy.
This claims-based retrospective analysis examined clinical and economic burden among United States patients who met eligibility for BCMA-targeted therapies and started a new therapy between 1 January 2016 and 30 September 2022.
Of 228 eligible patients (median age: 64, 52.6% male, 68.7% White), clinical burden was characterized by prevalent MM-related conditions and complications, and high treatment failure rate (44.7% in 6 months). Hospitalization rate increased from 48.3% to 71.1%, while healthcare costs per-member-per-month increased from $35,964 during 12-month baseline to $41,358 during follow-up (median: 11.8 months).
Patients with heavily pre-treated RRMM endure high disease burden and could benefit from novel therapies. What is this summary about? Multiple myeloma (MM) remains incurable despite considerable advances in treatment options. Patients treated with multiple lines of therapy eventually become resistant and develop relapse or refractory disease. Patients who require new treatments have limited options. This study examined the clinical and economic burden of patients with relapsed or refractory MM (RRMM) who had been treated with 4 lines of therapy and could be eligible for newly approved B-cell maturation antigen (BCMA)-targeted chimeric antigen receptor T-cell (CAR-T) or bispecific therapies. What were the results? The study found that a high proportion of United States patients suffer from MM-related conditions and comorbidities such as anemia, hypertension, and renal dysfunction. After initiating a new line of therapy, the healthcare costs per-member-per-month increased from $35,964 in the baseline to $41,358 during follow-up. The increase was due to higher inpatient hospitalization, treatment costs, and outpatient visits during follow-up. Treatment failure was common (44.7%) within 6 months. What do the results mean? The results highlight the significant clinical and economic burdens among patients with extensive previous RRMM treatment. Access to novel therapies (e.g. BCMA-targeted, CAR-T, or bispecific antibody therapies) may reduce the clinical burden of disease among patients with RRMM.
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