The DOI matches its Crossref record; 42 of the 42 references it cites were found in a registry or an index.
Clinical Effectiveness of Elexacaftor/Tezacaftor/Ivacaftor in People with Cystic Fibrosis: A Clinical Trial
Open accessAbstract
Rationale: The cystic fibrosis (CF) modulator drug, elexacaftor/tezacaftor/ivacaftor (ETI), proved highly effective in controlled clinical trials for individuals with at least one F508del allele, which occurs in at least 85% of people with CF. Objectives: PROMISE is a postapproval study to understand the broad effects of ETI through 30 months’ clinical use in a more diverse U.S. patient population with planned analyses after 6 months. Methods: Prospective, observational study in 487 people with CF age 12 years or older with at least one F508del allele starting ETI for the first time. Assessments occurred before and 1, 3, and 6 months into ETI therapy. Outcomes included change in percent predicted FEV1 (ppFEV1), sweat chloride concentration, body mass index (BMI), and self-reported respiratory symptoms. Measurements and Main Results: Average age was 25.1 years, and 44.1% entered the study using tezacaftor/ivacaftor or lumacaftor/ivacaftor, whereas 6.7% were using ivacaftor, consistent with F508del homozygosity and G551D allele, respectively. At 6 months into ETI therapy, ppFEV1 improved 9.76 percentage points (95% confidence interval [CI], 8.76 to 10.76) from baseline, cystic fibrosis questionnaire–revised respiratory domain score improved 20.4 points (95% CI, 18.3 to 22.5), and sweat chloride decreased −41.7 mmol/L (95% CI, −43.8 to −39.6). BMI also significantly increased. Changes were larger in those naive to modulators but substantial in all groups, including those treated with ivacaftor at baseline. Conclusions: ETI by clinical prescription provided large improvements in lung function, respiratory symptoms, and BMI in a diverse population naive to modulator drug therapy, using existing two-drug combinations, or using ivacaftor alone. Each group also experienced significant reductions in sweat chloride concentration, which correlated with improved ppFEV1 in the overall study population. Clinical trial registered with www.clinicaltrials.gov (NCT NCT04038047).
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Self-citationElexacaftor–Tezacaftor–Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele
Middleton, P. G., Mall, M. A., Dřevínek, P., Lands, L. C., McKone, E. F., Polineni, D., Ramsey, B. W., Taylor-Cousar, J. L., Tullis, E., Vermeulen, F., et al. (2019) · New England Journal of Medicine · doi:10.1056/nejmoa1908639 · Cited by 2,198
Self-citationA CFTR Potentiator in Patients with Cystic Fibrosis and the G551D Mutation
Ramsey, B. W., Davies, J., McElvaney, N. G., Tullis, E., Bell, S. C., Dřevínek, P., Griese, M., McKone, E. F., Wainwright, C. E., Konstan, M. W., et al. (2011) · New England Journal of Medicine · doi:10.1056/nejmoa1105185 · Cited by 2,195
Self-citationHeijerman, H. G. M., McKone, E. F., Downey, D. G., Van Braeckel, E., Rowe, S. M., Tullis, E., Mall, M. A., Welter, J. J., Ramsey, B. W., McKee, C. M., et al. (2019) · The Lancet · doi:10.1016/s0140-6736(19)32597-8 · Cited by 1,414
Self-citationThe future of cystic fibrosis care: a global perspective
Bell, S. C., Mall, M. A., Gutierrez, H., Macek, M., Madge, S., Davies, J. C., Burgel, P.-R., Tullis, E., Castaños, C., Castellani, C., et al. (2020) · The Lancet Respiratory Medicine · doi:10.1016/s2213-2600(19)30337-6 · Cited by 974
Self-citationDiagnosis of Cystic Fibrosis: Consensus Guidelines from the Cystic Fibrosis Foundation
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Self-citationStallings, V. A., Stark, L. J., Robinson, K. A., Feranchak, A. P., & Quinton, H. (2008) · Journal of the American Dietetic Association · doi:10.1016/j.jada.2008.02.020 · Cited by 732
Tezacaftor–Ivacaftor in Residual-Function Heterozygotes with Cystic Fibrosis
Rowe, S. M., Daines, C., Ringshausen, F. C., Kerem, E., Wilson, J., Tullis, E., Nair, N., Simard, C., Han, L., Ingenito, E. P., et al. (2017) · New England Journal of Medicine · doi:10.1056/nejmoa1709847 · Cited by 522
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CFTR modulator therapy: Transforming the landscape of clinical care in cystic fibrosis
Taylor-Cousar, J. L., Robinson, P. D., Shteinberg, M., & Downey, D. G. (2023) · The Lancet · doi:10.1016/s0140-6736(23)01609-4 · Cited by 173
Putman, M. S., Norris, A. W., Hull, R. L., Rickels, M. R., Sussel, L., Blackman, S. M., Chan, C. L., Ode, K. L., Daley, T., Stecenko, A. A., et al. (2023) · Diabetes Care · doi:10.2337/dc23-0380 · Cited by 35
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Metadata
- Authors
- Nichols, D. P., Paynter, A. C., Heltshe, S. L., Donaldson, S. H., Frederick, C. A., Freedman, S. D., Gelfond, D., Hoffman, L. R., Kelly, A., Narkewicz, M. R., Pittman, J. E., Ratjen, F., Rosenfeld, M., Sagel, S. D., Schwarzenberg, S. J., Singh, P. K., Solomon, G. M., Stalvey, M. S., Clancy, J. P., Kirby, S., Van Dalfsen, J. M., Kloster, M. H., & Rowe, S. M.
- Venue
- American Journal of Respiratory and Critical Care Medicine
- Publisher
- Oxford University Press (OUP)
- Published
- 2022-03-01
- Volume
- 205
- Issue
- 5
- Pages
- 529-539
- ISSN
- 1073-449X (ISSN-L)
- 1535-4970
- OpenAlex
- W3214022819
- Cited by
- 398
- References
- 42
- FWCI
- 32.62
- Type
- journal article
- Language
- en
- Open access
- hybrid
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- Nichols, D. P., Paynter, A. C., Heltshe, S. L., Donaldson, S. H., Frederick, C. A., Freedman, S. D., Gelfond, D., Hoffman, L. R., Kelly, A., Narkewicz, M. R., Pittman, J. E., Ratjen, F., Rosenfeld, M., Sagel, S. D., Schwarzenberg, S. J., Singh, P. K., Solomon, G. M., Stalvey, M. S., Clancy, J. P., . . . Rowe, S. M. (2022). Clinical Effectiveness of Elexacaftor/Tezacaftor/Ivacaftor in People with Cystic Fibrosis: A Clinical Trial. American Journal of Respiratory and Critical Care Medicine. https://doi.org/10.1164/rccm.202108-1986oc
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Bibliographic data: Semantic Scholar, OpenAlex, Crossref.
Contains information from the Semantic Scholar Academic Graph Datasets, made available under the ODC Attribution License (ODC-BY 1.0).
Semantic Scholar’s data is described in: Kinney, R., et al. (2023). The Semantic Scholar Open Data Platform. arXiv:2301.10140.
