Thorat Teja, McGarry Lisa J, Jariwala-Parikh Krutika, Limone Brendan, Bonafede Machaon, Chandarana Keval, Konstan Michael W
Vertex Pharmaceuticals Incorporated, 50 Northern Ave, Boston, MA, 02210, USA.
IBM Watson Health, Boston, MA, USA.
Pulm Ther. 2021 Jun;7(1):281-293. doi: 10.1007/s41030-021-00154-9. Epub 2021 Apr 28.
Ivacaftor was first approved in 2012 for the treatment of a select population of individuals with cystic fibrosis (CF), a rare, life-shortening genetic disease. Reductions in healthcare resource utilization (HCRU) associated with ivacaftor have been observed during limited follow-up and for selected outcomes in real-world studies. This study aimed to further describe the long-term impact of ivacaftor treatment on multiple measures of HCRU among people with CF (pwCF).
This retrospective study used US commercial and Medicaid claims data from 2011-2018. We included pwCF ≥ 6 years of age with ≥ 1 claim for ivacaftor and 12 months of continuous health plan enrollment before ivacaftor initiation ("pre-ivacaftor" period) who also had 36 months of continuous enrollment and persistent ivacaftor use (i.e., no gap ≥ 90 days between refills) following initiation ("post-ivacaftor" period). We compared comorbidities occurring pre-ivacaftor versus the last 12 months post-ivacaftor. HCRU outcomes included medication use, inpatient admissions, and outpatient office visits. We compared medication use pre-ivacaftor versus the last 12 months post-ivacaftor and inpatient admissions and outpatient office visits pre-ivacaftor versus the post-ivacaftor period annualized across 36 months.
Seventy-nine pwCF met all criteria, including persistent ivacaftor use during the post-ivacaftor period. Ivacaftor treatment was associated with a significant reduction in pneumonia prevalence (10.1% vs. 26.6%; p < 0.001) and significantly fewer mean [SD] antibiotics claims (8.0 [7.3] vs. 12.3 [11.1]; p < 0.001) in the last 12 months post-ivacaftor versus pre-ivacaftor. In comparing the 36-month post-ivacaftor period to the pre-ivacaftor period, we also observed fewer mean [SD] annual inpatient admissions (0.2 [0.4] vs. 0.4 [0.7]), CF-related inpatient admissions (0.1 [0.2] vs. 0.2 [0.5]), and outpatient office visits (8.8 [4.9] vs. 9.9 [5.4]) (all, p < 0.05).
Long-term ivacaftor treatment reduced HCRU, consistent with trends observed in prior real-world studies. Our results support the sustained, long-term value of ivacaftor treatment in reducing CF burden.
依伐卡托于2012年首次获批用于治疗特定人群的囊性纤维化（CF），这是一种罕见的、缩短寿命的遗传性疾病。在有限的随访期间以及真实世界研究的特定结局中，已观察到与依伐卡托相关的医疗资源利用（HCRU）减少。本研究旨在进一步描述依伐卡托治疗对CF患者（pwCF）多种HCRU指标的长期影响。
这项回顾性研究使用了2011 - 2018年美国商业保险和医疗补助索赔数据。我们纳入了年龄≥6岁、有≥1次依伐卡托索赔记录且在开始使用依伐卡托前连续参加健康计划12个月（“依伐卡托前”时期），并且在开始使用依伐卡托后（“依伐卡托后”时期）连续参保36个月且持续使用依伐卡托（即两次取药间隔无≥90天的间断）的pwCF。我们比较了依伐卡托前与依伐卡托后最后12个月出现的合并症。HCRU结局包括药物使用、住院入院和门诊就诊。我们比较了依伐卡托前与依伐卡托后最后12个月的药物使用情况，以及依伐卡托前与依伐卡托后时期年化后的36个月内的住院入院和门诊就诊情况。
79例pwCF符合所有标准，包括在依伐卡托后时期持续使用依伐卡托。与依伐卡托前相比，依伐卡托治疗与依伐卡托后最后12个月肺炎患病率显著降低（10.1%对26.6%；p < 0.001）以及平均[标准差]抗生素索赔显著减少（8.0 [7.3]对12.3 [11.1]；p < 0.001）相关。在比较依伐卡托后36个月时期与依伐卡托前时期时，我们还观察到平均[标准差]年度住院入院次数减少（0.2 [0.4]对0.4 [0.7]）、CF相关住院入院次数减少（0.1 [0.2]对0.2 [0.5]）以及门诊就诊次数减少（8.8 [4.9]对9.9 [5.4]）（所有p < 0.05）。
依伐卡托长期治疗可降低HCRU，与先前真实世界研究中观察到的趋势一致。我们的结果支持依伐卡托治疗在减轻CF负担方面具有持续的长期价值。
