Poster Session Abstracts: Abstracts 216–281

• Publisher: John Wiley and Sons
• Year: 1998
• Tongue: English
• Weight: 324 KB
• Volume: 26
• Category: Article
• ISSN: 8755-6863
• DOI: 10.1002/(sici)1099-0496(199810)26:17+<260::aid-ppul11>3.0.co;2-v

✦ Synopsis

tgAAVCF (AAV-CFTR) was used in a phase II, double-blind, randomized, placebo-controlled clinical trial to transfer CFTR cDNA into respiratory epithelial cells in the maxillary sinus of 23 CF patients with maxillary antrostomies. The maxillary sinuses serve as a surrogate model of CF lung disease because of similar clinical manifestations as well as having the same ion transport systems and microbiology. A dose of 100,000 replication units tgAAVCF was used to treat one sinus in all patients. The contralateral sinus was treated with placebo in a randomized, blinded fashion. Clinically-deÞned and endoscopically conÞrmed recurrent sinusitis is the primary outcome measure. Secondary outcomes include maxillary sinus transepithelial potential differences, semi-quantitative DNA PCR, and semi-quantitative RT-PCR. Although patient recruitment is complete, the study is still in progress and is not yet unblinded. To date, the rate of recurrent sinusitis is 76% during three months of follow-up visits. One case of acute peripheral vestibulopathy was temporally associated with tgAAVCF treatment. Vector shedding was also seen in this patient at day 7, but not on day 1 or day 14; no vector shedding was observed in any other patient. Symptoms of vestibulopathy in this patient completely resolved. Seven subacute pulmonary deteriorations requiring hospitalization but not related to tgAAVCF treatment occurred in 17 patients who have Þnished follow-up visits.