Abstract

Multiple myeloma (MM) is an incurable hematological cancer requiring multiple lines of anti-myeloma regimens to promote disease remission and increase patient survival. The study assessed the incidence and reasons for discontinuation of first-line therapy in outpatients who started MM therapy in Belo Horizonte, Brazil from 2009 to 2020. A historical cohort study in which patients were followed from treatment initiation until discontinuation of first-line therapy. Discontinuation of first-line therapy was characterized as (i) discontinuation followed by a second-line therapy, and (ii) discontinuation that prevented patients from receiving a subsequent line of treatment. Non-parametric competing risk analysis with a 95% confidence interval estimated the cumulative incidences of discontinuation followed by a second-line therapy. The probability of discontinuation was compared according to selected variables using the Gray’s test at a significance level of 5%. Approximately half of the participants (n = 260) were female and younger than 65 years. Discontinuation of first-line therapy followed by a second-line therapy accounted for 50.4% of the patients and occurred up to 30th month. The main reason for discontinuation not qualifying patients for receiving second-line therapy was to achieve a response to treatment. The maximum times for discontinuation not followed by a second-line therapy ranged from 12 to 20 months due to deaths or response to treatment. The probability of receiving second-line therapy was higher among patients initiating therapy in 2009–2014 and those not undergoing transplantation. In conclusion, discontinuation of first-line therapy followed by second-line treatment occurred as likely as the discontinuation not followed by a subsequent line.

中文翻译：

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多发性骨髓瘤患者的发病率和停止一线治疗的原因：2009-2020 年巴西历史队列的结果

摘要

多发性骨髓瘤 (MM) 是一种无法治愈的血液癌症，需要多种抗骨髓瘤治疗方案来促进疾病缓解并提高患者生存率。该研究评估了 2009 年至 2020 年在巴西贝洛奥里藏特开始接受 MM 治疗的门诊患者中止一线治疗的发生率和原因。这是一项历史队列研究，对患者从治疗开始到中止一线治疗进行随访。一线治疗中止的特征是（i）中止后进行二线治疗，以及（ii）中止导致患者无法接受后续治疗。具有 95% 置信区间的非参数竞争风险分析估计了二线治疗后停药的累积发生率。根据选定的变量，使用格雷检验以 5% 的显着性水平比较停药的概率。大约一半的参与者 ( n  = 260) 是女性，年龄小于 65 岁。50.4% 的患者在停止一线治疗后又接受二线治疗，且持续时间长达 30 个月。不符合接受二线治疗资格的患者停止治疗的主要原因是为了获得治疗反应。由于死亡或治疗反应而停药且未接受二线治疗的最长停药时间为 12 至 20 个月。2009-2014 年开始治疗的患者和未接受移植的患者接受二线治疗的可能性较高。总之，中止一线治疗后进行二线治疗的可能性与中止后未进行后续治疗的可能性一样。