Hari Parameswaran, Lin Huamao Mark, Zhu Yanyan, Berg Deborah, Richardson Paul G, Moreau Philippe
a Department of Medicine, Medical College of Wisconsin , Milwaukee , WI , USA.
b Millennium Pharmaceuticals Inc., a wholly owned subsidiary of Takeda Pharmaceutical Company Limited , Cambridge , MA , USA.
J Med Econ. 2018 Aug;21(8):793-798. doi: 10.1080/13696998.2018.1474745. Epub 2018 May 29.
The aim of this analysis was to assess healthcare resource utilization in the pivotal phase 3 TOURMALINE-MM1 study of the oral proteasome inhibitor ixazomib or placebo plus lenalidomide and dexamethasone (Rd) in relapsed and/or refractory multiple myeloma (RRMM).
In this double-blind, placebo-controlled, randomized study (NCT01564537), 722 patients with RRMM following 1-3 prior lines of therapy received Rd plus ixazomib (ixazomib-Rd; n = 360) or matching placebo (placebo-Rd; n = 362) until disease progression or unacceptable toxicity. Healthcare resource utilization data were captured on Day 1 of each 28-day cycle, every 4 weeks during follow-up for progression-free survival, and every 12 weeks during subsequent follow-up, and included medical encounters (length of stay, inpatient, outpatient, and reason) and number of missing days from work or other activities for patients and caregivers.
Exposure-adjusted rates of hospitalization were similar between the ixazomib-Rd and placebo-Rd arms, at 0.530 and 0.564 per patient year (ppy), respectively, as were outpatient visit rates (3.305 and 3.355 ppy). Mean length of hospitalization per patient was 10.0 and 10.8 days, respectively. In both arms, hospitalization and outpatient visit rates were higher in patients with two or three prior lines of treatment (ixazomib-Rd: 0.632 and 3.909 ppy; placebo-Rd: 0.774 and 3.539 ppy) compared with patients with one prior line (ixazomib-Rd: 0.460 and 2.888 ppy; placebo-Rd: 0.436 and 3.243 ppy). Patients and their caregivers who missed any work or other activity missed a median of 7 and 5 days in the ixazomib-Rd arm, respectively, vs 8 and 4 days with placebo-Rd.
The study was not powered for a statistical comparison of healthcare resource utilization between treatment arms, nor did it capture costs associated with utilization of the identified healthcare resources.
This pre-specified analysis demonstrated that the all-oral triplet regimen of ixazomib added to Rd did not increase healthcare resource utilization compared with placebo-Rd.
本分析旨在评估口服蛋白酶体抑制剂伊沙佐米或安慰剂联合来那度胺和地塞米松（Rd）用于复发和/或难治性多发性骨髓瘤（RRMM）的关键3期TOURMALINE-MM1研究中的医疗资源利用情况。
在这项双盲、安慰剂对照、随机研究（NCT01564537）中，722例接受过1 - 3线前期治疗的RRMM患者接受Rd加伊沙佐米（伊沙佐米 - Rd；n = 360）或匹配的安慰剂（安慰剂 - Rd；n = 362），直至疾病进展或出现不可接受的毒性。在每28天周期的第1天、无进展生存期随访期间每4周以及后续随访期间每12周收集医疗资源利用数据，包括医疗接触（住院时间、住院、门诊及原因）以及患者和护理人员工作或其他活动的缺勤天数。
伊沙佐米 - Rd组和安慰剂 - Rd组的暴露调整后住院率相似，分别为每人年0.530次和0.564次，门诊就诊率也相似（分别为每人年3.305次和3.355次）。每位患者的平均住院时间分别为10.0天和10.8天。在两组中，接受过两线或三线前期治疗的患者的住院率和门诊就诊率（伊沙佐米 - Rd组：每人年0.632次和3.909次；安慰剂 - Rd组：每人年0.774次和3.539次）高于接受过一线前期治疗的患者（伊沙佐米 - Rd组：每人年0.460次和2.888次；安慰剂 - Rd组：每人年0.436次和3.243次）。在伊沙佐米 - Rd组中，缺勤工作或其他活动的患者及其护理人员分别缺勤中位数7天和5天，而在安慰剂 - Rd组中分别为8天和4天。
该研究没有足够的效能对各治疗组之间的医疗资源利用进行统计学比较，也未获取与所确定的医疗资源利用相关的成本。
这项预先指定的分析表明，与安慰剂 - Rd相比，在Rd基础上加用伊沙佐米的全口服三联方案并未增加医疗资源利用。
