Patients with cystic fibrosis who initiate ivacaftor therapy may experience significant changes in cholesterol levels, according to new study results.
In the study, published in Therapeutic Advances in Respiratory Disease, researchers analyzed serum lipid samples from 50 patients with cystic fibrosis (CF) as part of the GOAL study cohort. Using a repeated measures model, they observed a significant increase in total cholesterol (TC) levels from baseline to 3 months (+ 9.4 mg/dL, P = .004). However, by 18 months, TC levels returned closer to baseline (–3.5 mg/dL, P = 0.407). Low-density lipoprotein (LDL) and high-density lipoprotein (HDL) levels showed no statistically significant changes over time.
At baseline, participants had a mean TC level of 125.8 mg/dL (95% confidence interval [CI] = 116.7–134.9), LDL of 67.5 mg/dL (95% CI = 60.6–74.4), and HDL of 46.2 mg/dL (95% CI = 41.9–50.5). The mean body mass index (BMI) was 21.2 kg/m² (95% CI = 20.1–22.3). The cohort was 54% male, with a mean age of 25.4 years (range = 12.2–63.8). Most of the participants (88%) had severe CFTR gene mutations.
BMI was identified as a significant covariate (P = .002), with higher BMI correlating with increased TC levels. BMI also increased at each visit (P < .001), suggesting a metabolic effect of modulator therapy on lipid changes in patients with CF.
The study also surveyed 75 CF providers across the United States regarding cardiovascular risk screening. The researchers demosntrated that 67% of the providers had no standardized lipid screening policy. Among those without a policy, 69% screened patients selectively based on factors like BMI (49%), family history (46%), and age (37%).
Regarding cardiovascular risk management, 29% of adult CF providers prescribed lipid-lowering therapy over the past year, 54% initiated antihypertensive treatment, and 48% conducted ischemic cardiac evaluations. The researchers noted that many CF providers—primarily pulmonologists—are now increasingly managing cardiovascular disease risk factors in their patients.
"Lipid screening practices among CF providers were variable and providers are increasingly being confronted with managing [cardiovascular disease] risk factors. Partnering with primary care providers is likely to become increasingly important in CF care models," wrote lead study author Katherine A. Despotes, MD, and colleagues at the University of North Carolina at Chapel Hill.
The findings suggested a growing need for CF specialists to collaborate with primary care providers and cardiologists to manage cardiovascular risk.
The researchers highlighted the evolving landscape of CF care as patients live longer and encounter new health challenges. While the short-term cholesterol increases associated with ivacaftor were not sustained, continued research is needed to understand the long-term cardiovascular risks associated with highly effective modulator therapy (HEMT).
Full disclosures are detailed in the study.
