a Policy Analysis Inc. (PAI) , Brookline , MA , USA.
b Amgen Inc , South San Francisco , CA , USA.
J Med Econ. 2019 Aug;22(8):818-829. doi: 10.1080/13696998.2019.1614932. Epub 2019 May 17.
To compare monthly healthcare resource utilization (HRU) and costs among adult patients with multiple myeloma (MM) receiving second or subsequent line of treatment (LOT) with carfilzomib or pomalidomide as monotherapy or in combination with dexamethasone. Adult MM patients who received carfilzomib or pomalidomide as second/subsequent LOT between 2006 and 2014 were selected from the MarketScan databases. LOT was determined using Medical/pharmacy claims using a published algorithm. For each patient, first LOT with carfilzomib or pomalidomide was defined as index LOT. Patients with first LOT as index LOT, who received other chemotherapy in combination with carfilzomib or pomalidomide, or who underwent stem cell transplant (STC) during index LOT were excluded. Monthly HRU and costs during index LOT were compared using inverse probability of treatment weights (IPTW) based on propensity scores for receipt of carfilzomib estimated by logistic regression with LOT, patient demographics, Charlson index, comorbidities, pre-index healthcare cost, and receipt of prior SCT as covariates. After weighting, baseline characteristics were well balanced among 114 carfilzomib and 144 pomalidomide patients. Mean (95% CI) numbers of outpatient visits per month were 7.1 (5.2-8.0) with carfilzomib and 4.7 (3.9-6.1) with pomalidomide ( = 0.006). Otherwise, there were no statistically significant differences between the groups in mean monthly HRU and costs or median time to therapy discontinuation. Mean (95% CI) monthly total healthcare costs were $19,776 (15,322-27,748) with pomalidomide and $17,321 (12,412-21,874) with carfilzomib ( = 0.522). Comparison of carfilzomib vs pomalidomide may be biased if there are unobserved factors not balanced by IPTW. The relatively small sample size limits the power of analyses to detect potential differences between treatment groups. Monthly HRU and costs are similar among patients with relapse or refractory MM patients receiving carfilzomib or pomalidomide as monotherapy or in combination with dexamethasone.
比较接受卡非佐米或泊马度胺二线或后续线治疗（LOT）的多发性骨髓瘤（MM）成年患者的每月医疗资源利用（HRU）和成本，这些患者接受卡非佐米或泊马度胺单药或联合地塞米松治疗。 从 MarketScan 数据库中选择 2006 年至 2014 年间接受卡非佐米或泊马度胺二线/后续 LOT 的成年 MM 患者。LOT 是使用已发表的算法通过医疗/药房索赔确定的。对于每位患者，卡非佐米或泊马度胺的首次 LOT 被定义为索引 LOT。索引 LOT 中接受其他化疗联合卡非佐米或泊马度胺或接受干细胞移植（STC）的患者，或在索引 LOT 期间接受 STC 的患者，排除在外。通过基于逻辑回归估计的 LOT、患者人口统计学、Charlson 指数、合并症、指数前医疗保健费用和接受先前 SCT 的协变量对卡非佐米的接受概率的逆概率治疗权重（IPTW）比较索引 LOT 期间的每月 HRU 和成本。加权后，114 名卡非佐米和 144 名泊马度胺患者的基线特征在治疗组之间得到很好的平衡。每月门诊就诊次数的平均值（95%CI）分别为卡非佐米组 7.1（5.2-8.0）和泊马度胺组 4.7（3.9-6.1）（ = 0.006）。否则，两组在每月 HRU 和成本或治疗中断的中位时间方面没有统计学上的显著差异。每月总医疗保健费用的平均值（95%CI）分别为泊马度胺组 19776 美元（15322-27748 美元）和卡非佐米组 17321 美元（12412-21874 美元）（ = 0.522）。如果存在未观察到的、不受 IPTW 平衡的因素，那么卡非佐米与泊马度胺的比较可能存在偏倚。较小的样本量限制了检测治疗组之间潜在差异的分析能力。接受卡非佐米或泊马度胺单药或联合地塞米松治疗的复发性或难治性 MM 患者的每月 HRU 和成本相似。
