The relationship between cost and the recommendation, refusal, and discontinuation of treatment for chronic myeloid leukemia and multiple myeloma in Japan: a cross-sectional exploratory survey

This study aimed to ascertain the number of patients with chronic myelogenous leukemia (CML) and transplant-ineligible patients with multiple myeloma (MM) not recommended by their physicians for optimal drug treatment or who refuse, discontinue, reduce, or skip treatment owing to cost in Japan. A cross-sectional survey was conducted among hematologists, hematologic oncologists, and oncologists in Japan treating ≥1 patient with CML or ≥5 transplant-ineligible patients with MM per year. A total of 212 physicians participated: 105 treating patients with CML and 107 treating transplant-ineligible patients with MM. While treatment cost did not lead to non-optimal treatment most patients, physicians reported that they recommended non-optimal treatment to 6.53% of their patients with CML and 1.41% of their transplant-ineligible patients with MM, that 1.51 and 0.35% of their patients, respectively, refused treatment and that 1.97 and 0.71% discontinued treatment owing to treatment cost. However, no significant differences in the effect of treatment cost on recommendation, discontinuation, refusal, or reduction of treatment were observed. Non-recommendation of optimal treatment owing to treatment cost was most common for third-line CML and fourth-line transplant-ineligible MM treatment. Discontinuation due to treatment cost was most common in third-line treatment for both. Our results show that non-optimal treatment due to treatment cost occurs among some physicians in Japan for patients with CML and transplant-ineligible patients with MM, but it may be limited to a small percentage of patients. Further research is needed to identify the drivers of treatment decisions for physicians and patients, including those involving treatment cost.

本研究旨在明确日本境内，因治疗费用问题未获医师推荐最优药物治疗，或拒绝、中断、减量、漏用治疗方案的慢性髓性白血病（chronic myelogenous leukemia, CML）患者，以及经医师评估不适合移植的多发性骨髓瘤（multiple myeloma, MM）患者的人数规模。
本研究针对日本血液科医师、血液肿瘤医师及肿瘤医师开展横断面调查，纳入对象为年接诊≥1例CML患者，或年接诊≥5例不适合移植的MM患者的医师。最终共有212名医师参与本次研究，其中105名接诊CML患者，107名接诊不适合移植的MM患者。
尽管整体而言，治疗费用并非多数患者接受非最优治疗的主要诱因，但受访医师报告称，其分别向6.53%的CML患者、1.41%的不适合移植MM患者推荐了非最优治疗方案；两类患者中分别有1.51%、0.35%拒绝接受治疗，另有1.97%、0.71%因治疗费用中断治疗。不过，未观察到治疗费用对治疗方案推荐、中断、拒绝或减量的影响存在显著统计学差异。
因治疗费用未推荐最优治疗的情况，在CML三线治疗、不适合移植MM四线治疗中最为常见；而因治疗费用中断治疗的情况，则在两类患者的三线治疗中占比最高。
本研究结果显示，日本部分医师接诊的CML患者与不适合移植的MM患者中，存在因治疗费用导致非最优治疗的情况，但此类患者占比整体偏低。未来仍需开展进一步研究，以明确医师与患者治疗决策的各类驱动因素，其中包括治疗成本相关的影响环节。