Background

Long-term changes in medication dispensings post cystic fibrosis transmembrane conductance regulator (CFTR) modulator initiation have not been described. Our study aimed to investigate changes in medication use following the initiation of modulator therapy in people with cystic fibrosis (PwCF) in Australia.

Methods

Using a 10% sample of the Australian Pharmaceutical Benefits Scheme (PBS) data between 2013 and 2022, linear regression was used to analyse dispensings in PwCF who initiated any modulator (cases) and matched PwCF controls not dispensed a modulator. The difference in mean number of total monthly dispensings pre- and post-modulator initiation was analysed, with separate analyses by medication class.

Results

A total of 247 cases were matched 1:1 to controls (case and control median age 21 years (IQR: 13–32), 55.1% male). Immediately after modulator initiation, the mean number of dispensings was 0.9 higher in the modulator group, but then decreased to the level of controls after approximately 5 years. After 7.5 years, cases had decreased opioids compared to the pre-modulator period (β-coefficient: −0.00131, 95% CI: −0.00164, −0.00097) whilst controls did not (β: −0.00014, 95% CI: −0.00042, 0.00014). Over the same time period controls had an increase in psychotropics (β: 0.00389, 95% CI:0.00295, 0.00484) whilst cases remained stable (β: −0.00014, 95% CI: −0.0006, 0.00031). Women's health medications increased in cases (β:0.00026, 95% CI:0.0001, 0.00042) but decreased in controls (β: 0.00044, 95% CI: 0.00063, −0.00025).

Conclusions

Modulator initiation in PwCF was associated with decreased dispensings of opioids and psychotropics, and increased dispensings of women's health medications, suggesting improved patient outcomes across multiple clinical domains.

中文翻译：

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囊性纤维化患者在调节剂治疗前后的药物使用情况

背景

尚未描述囊性纤维化跨膜电导调节剂（CFTR）调节剂启动后药物分配的长期变化。我们的研究旨在调查澳大利亚囊性纤维化 (PwCF) 患者开始调节剂治疗后药物使用的变化。

方法

使用 2013 年至 2022 年间澳大利亚药品福利计划 (PBS) 数据的 10% 样本，使用线性回归来分析 PwCF 中启动任何调节剂（病例）的配药情况以及未配发调节剂的匹配 PwCF 对照。分析了调节剂启动前和启动后每月总配药平均次数的差异，并按药物类别进行单独分析。

结果

总共 247 例病例与对照进行 1:1 匹配（病例和对照中位年龄 21 岁（IQR：13-32），55.1% 为男性）。调节剂启动后，调节剂组的平均配药次数增加了 0.9 次，但大约 5 年后下降到对照组的水平。7.5年后，与调制前相比，病例阿片类药物减少（β系数：-0.00131，95% CI：-0.00164，-0.00097），而对照组则没有（β：-0.00014，95% CI：-0.00042， 0.00014）。同一时期内，对照精神药物的使用有所增加（β：0.00389，95％CI：0.00295，0.00484），而病例保持稳定（β：-0.00014，95％CI：-0.0006，0.00031）。病例中妇女的保健药物增加（β：0.00026，95％CI：0.0001，0.00042），但对照组减少（β：0.00044，95％CI：0.00063，-0.00025）。

结论

PwCF 中调节剂的启动与阿片类药物和精神药物的配药减少以及女性健康药物的配药增加相关，这表明多个临床领域的患者治疗结果得到改善。