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Clinical change 2 years from start of elexacaftor-tezacaftor-ivacaftor in severe cystic fibrosis

McCoy, Karen S; Blind, Jill; Johnson, Terri; Olson, Patti; Raterman, Laura; Bai, Shasha; Eisner, Mariah; Sheikh, Shahid I; Druhan, Stephan; Young, Cody; Pasley, Kimberly
RATIONALE/BACKGROUND:Limited published research is available on the impact of elexacaftor/tezacaftor/ivacaftor (ETI) beyond the initial few months postdrug initiation, especially for those who initiated therapy via individual investigational new drug application. The experiences of patients with cystic fibrosis (CF) experiencing severe lung disease were reviewed for significant improvements in clinical symptoms and quality of life. OBJECTIVES/OBJECTIVE:To examine clinical outcomes 2 years post-ETI in patients with CF and advanced lung disease. METHODS:This single center institutional review board-approved, retrospective chart review assessed clinical markers (percent predicted forced expiratory volume in 1 s, weight, sweat chloride), quality of life and computed tomography scans in patients with advanced lung disease who met criteria for compassionate use/expanded access program due to high risk of death or transplant need within 2 years. RESULTS:Eighteen identified patients (ages 15-49 years) initiated drug between July and September 2019. Clinical markers indicated that therapy was well tolerated, not discontinued by any participant, and lab values did not indicate medical concern or discontinuation. Monitoring results indicated the safety of modulator therapy as there were no adverse clinical occurrences and all patients presented universal stabilization. There were no deaths and no transplants by the end of the study. CONCLUSIONS:, subjective reports of clinical status, level of activity, and a reduction in burden of treatment.
PMID: 36650567
ISSN: 1099-0496
CID: 5426362

Successful sonographically guided drainage of epidural abscess [Case Report]

Druhan, Stephen M; Shiels, William E; Kang, D Richard; Elton, Scott W; Koranyi, Katalin
PMID: 17056883
ISSN: 1546-3141
CID: 4996562