Division of Gastroenterology, Hepatology, and Nutrition, the Ohio State University Wexner Medical Center, Columbus, OH, USA.
Center for Biostatistics, Department of Biomedical Informatics, the Ohio State University College of Medicine, Columbus, OH, USA.
Am J Gastroenterol. 2021 Dec 1;116(12):2446-2454. doi: 10.14309/ajg.0000000000001527.
Acute pancreatitis (AP) occurs among patients with pancreas-sufficient cystic fibrosis (PS-CF) but is reportedly less common among patients with pancreas-insufficient cystic fibrosis (PI-CF). The incidence of AP may be influenced by cystic fibrosis transmembrane conductance regulator (CFTR) modulator use. We hypothesized that CFTR modulators would reduce AP hospitalizations, with the greatest benefit in PS-CF.
MarketScan (2012-2018) was queried for AP hospitalizations and CFTR modulator use among patients with CF. Multivariable Poisson models that enabled crossover between CFTR modulator treatment groups were used to analyze the rate of AP hospitalizations on and off therapy. Pancreas insufficiency was defined by the use of pancreas enzyme replacement therapy.
A total of 10,417 patients with CF were identified, including 1,795 who received a CFTR modulator. AP was more common in PS-CF than PI-CF (2.9% vs 0.9%, P = 0.007). Overall, the observed rate ratio of AP during CFTR modulator use was 0.33 (95% confidence interval [CI] 0.10, 1.11, P = 0.07) for PS-CF and 0.38 (95% CI 0.16, 0.89, P = 0.03) for PI-CF, indicating a 67% and 62% relative reduction in AP hospitalizations, respectively. In a subset analysis of 1,795 patients who all had some CFTR modulator use, the rate ratio of AP during CFTR modulator use was 0.36 (95% CI 0.13, 1.01, P = 0.05) for PS-CF and 0.53 (95% CI 0.18, 1.58, P = 0.26) for PI-CF.
CFTR modulator use is associated with a reduction in AP hospitalizations among patients with CF. These observational data support the prospective study of CFTR modulators to reduce AP hospitalizations among patients with CF.
急性胰腺炎（AP）发生于胰腺功能正常的囊性纤维化（PS-CF）患者中，但在胰腺功能不全的囊性纤维化（PI-CF）患者中较少见。CFTR 调节剂的使用可能会影响 AP 的住院率，PS-CF 患者的获益最大。
通过 MarketScan（2012-2018 年）查询 CF 患者的 AP 住院率和 CFTR 调节剂的使用情况。采用允许 CFTR 调节剂治疗组交叉的多变量泊松模型分析治疗和停药期间 AP 住院率。胰腺功能不全定义为使用胰腺酶替代疗法。
共纳入 10417 例 CF 患者，其中 1795 例接受 CFTR 调节剂治疗。PS-CF 患者的 AP 发生率高于 PI-CF（2.9%比 0.9%，P = 0.007）。总体而言，CFTR 调节剂治疗期间 AP 的观察到的发生率比值比（RR）在 PS-CF 为 0.33（95%置信区间 [CI] 0.10，1.11，P = 0.07），在 PI-CF 为 0.38（95% CI 0.16，0.89，P = 0.03），表明 AP 住院率分别降低了 67%和 62%。在 1795 例均有 CFTR 调节剂使用的患者的亚组分析中，CFTR 调节剂治疗期间 AP 的 RR 在 PS-CF 为 0.36（95% CI 0.13，1.01，P = 0.05），在 PI-CF 为 0.53（95% CI 0.18，1.58，P = 0.26）。
CFTR 调节剂的使用与 CF 患者 AP 住院率的降低相关。这些观察性数据支持前瞻性研究 CFTR 调节剂以降低 CF 患者的 AP 住院率。
