Cystic fibrosis triple therapy outcomes improve regardless of prior modulators
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Key takeaways:
- Triple therapy improved lung function, BMI and other factors.
- Patients naïve to cystic fibrosis transmembrane conductance regulator modulators had comparable improvements to patients with prior exposure.
BOSTON — Having vs. not having past cystic fibrosis transmembrane conductance regulator modulator exposure did not change positive outcomes seen with elexacaftor/tezacaftor/ivacaftor, according to data presented at the CHEST Annual Meeting.
“Our results suggest that in real life, elexacaftor/tezacaftor/ivacaftor (ETI) is equally effective among those who were either previously exposed or naïve to previous cystic fibrosis transmembrane conductance regulator (CFTR) modulators,” Shahid I. Sheikh, MD, pediatric pulmonologist at Nationwide Children’s Hospital, told Healio.
“[This was] not only in clinical outcomes but there are no significant differences in improvement in their chronic rhinosinusitis as shown by sinus CT scans with 1 year therapy of ETI,” Sheikh continued.
In a prospective, longitudinal, observational study, Sheikh and colleagues assessed 115 patients (median age, 23 years; 46% male) with cystic fibrosis (CF) to see if 1-year ETI (Trikafta, Vertex Pharmaceuticals) performs differently in 49 patients (median age, 20 years; 42.9% male) naïve to CFTR modulators vs. 66 patients (median age, 26 years; 48.5% male) with prior CFTR modulator exposure before receiving the triple therapy.
From baseline (start of ETI) to the 1-year mark, researchers reported significant (P < .05) improvements in several factors, all of which were similar when comparing the naïve group with the past CFTR therapy group:
- Percent-predicted FEV1 (67% to 79%);
- BMI (22.1 kg/m2 to 23.8 kg/m2);
- Pseudomonas aeruginosa positivity (61 patients to 30 patients); and
- methicillin-resistant Staphylococcus aureus positivity (32 patients to 17 patients).
Another significant improvement — taking place between the year before starting ETI and 1 year after starting ETI — that occurred independently of prior exposure to CFTR modulators was found in CF exacerbations, according to the abstract. The same was true for the observed reduction in use of Pulmozyme (dornase alfa, Genentech) from 99 patients at baseline to 44 patients and HyperSal (sodium chloride, Pari Innovative Manufacturers Inc.) from 74 patients to 37 patients.
“Study results were expected as we found that in real life, there were no significant differences in ETI efficacy based on previous exposure to CFTR modulators,” Sheikh told Healio.
Further, the total cohort included 21 patients with baseline and 1-year sinus CT scans, and two scoring systems signaled that CF-associated chronic rhinosinusitis significantly improved between the two time periods (median total score, Lund-Mackay system, 5.5 to 2.8; Sheikh-Lund system, 3.8 to 2.2; both P < .001).
Researchers again found that patients naïve to CFTR modulators and patients with prior CFTR modulator exposure had comparable changes in these scores.
“This study suggests that in real-life previous exposure to other CFTR modulators does not limit benefits from new CFTR modulator (ETI) therapy,” Sheikh told Healio.
“CFTR modulator therapies have revolutionized CF care,” Sheikh added. “With time, newer therapies will emerge, and hopefully their efficacy may also not be impacted by previous exposures to other similar therapies.”
For more information:
Shahid I. Sheikh, MD, can be reached at shahid.sheikh@nationwidechildrens.org.