• Layla Diab-Cáceres, Rosa María Girón-Moreno, María Teresa Pastor-Sanz, Esther Quintana-Gallego, Isabel Delgado-Pecellín, Marina Blanco-Aparicio, Luis Maiz, Marta María García-Clemente, Carmen Luna-Paredes, Pedro Mondéjar-López, Marta Ruiz-de-Valbuena, Ofelia Fernández, Maribel Barrio, Maribel González, Alejandro López-Neyra, María Cols-I-Roig, Alexandre Palou-Rotger, and Francisco Javier Gómez-de-Terreros-Caro.
• Hospital de La Princesa, Respirology, Madrid, Spain. Electronic address: layladch@gmail.com.
• Arch. Bronconeumol. 2018 Dec 1; 54 (12): 614-618.

BackgroundThe most common cystic fibrosis (CF)-causing mutation is deltaF508 (F508del), which is present in 28% of CF Spanish patients. While the literature based on real-life studies on CF patients homozygous F508del treated with lumacaftor/ivacaftor is limited, it demonstrates the need for better strategies to prevent related adverse events (AEs) as well as the development of newer drugs.MethodsWe conducted a multicenter, retrospective, observational study to describe the effects of lumacaftor/ivacaftor treatment in real-life in Spain. 20 CF patients were included, all aged 6 and upwards and presented with ppFEV1<40%, chosen from CF units country-wide. For the purposes of the study, they were treated with lumacaftor/ivacaftor 200/125mg two tablets twice a day on a compassionate use programme throughout 2016. The primary endpoint was measured in all of the sample patients. Data were analysed from ppFEV1 at baseline and was measured every 6 months.ResultsThe mean age was 26.65 (range of 10-45), while the mean ppFEV1 before the treatment was 32.4% and mean BMI was 19.9kg/m2. We analysed the changes in ppFEV1 and BMI from baseline during the treatment with lumacaftor/ivacaftor, but no differences were found. However, a moderate association between days of intravenous antibiotic needed and the use of lumacaftor/ivacaftor (p=0.001) was established. Indeed, under the lumacaftor/ivacaftor, patients required 5.8 days of intravenous antibiotic treatment compared to 14.9 days prior to study. Also, severe pulmonary exacerbations requiring hospitalisation were statistically fewer under lumacaftor/ivacaftor treatment (p=0.003). Finally, 75% of the sample presented with AEs, which led 35% of the subjects to discontinue the treatment.ConclusionsWhile treatment with lumacaftor/ivacaftor resulted in an improvement in the number of pulmonary severe exacerbations, no improvement in ppFEV1 or BMI was found.Copyright © 2018 SEPAR. Publicado por Elsevier España, S.L.U. All rights reserved.

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