PMID- 30518496 OWN - NLM STAT- MEDLINE DCOM- 20191024 LR - 20210518 IS - 2173-5751 (Electronic) IS - 2173-5751 (Linking) VI - 54 IP - 12 DP - 2018 Dec TI - Compassionate Use of Lumacaftor/Ivacaftor in Cystic Fibrosis: Spanish Experience. PG - 614-618 LID - S0300-2896(18)30193-5 [pii] LID - 10.1016/j.arbres.2018.05.004 [doi] AB - BACKGROUND: The 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. METHODS: We 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. RESULTS: The 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/m(2). 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. CONCLUSIONS: While treatment with lumacaftor/ivacaftor resulted in an improvement in the number of pulmonary severe exacerbations, no improvement in ppFEV1 or BMI was found. CI - Copyright (c) 2018 SEPAR. Publicado por Elsevier Espana, S.L.U. All rights reserved. FAU - Diab-Caceres, Layla AU - Diab-Caceres L AD - Hospital de La Princesa, Respirology, Madrid, Spain. Electronic address: layladch@gmail.com. FAU - Giron-Moreno, Rosa Maria AU - Giron-Moreno RM AD - Hospital de La Princesa, Respirology, Madrid, Spain. FAU - Pastor-Sanz, Maria Teresa AU - Pastor-Sanz MT AD - Hospital de La Princesa, Respirology, Madrid, Spain. FAU - Quintana-Gallego, Esther AU - Quintana-Gallego E AD - Hospital Universitario Virgen del Rocio, Sevilla, Spain. Centro de Investigacion Biomedica en Red de Enfermedades Respiratorias (CIBERES), Instituto de Salud Carlos III, Madrid, Spain. FAU - Delgado-Pecellin, Isabel AU - Delgado-Pecellin I AD - Hospital Universitario Virgen del Rocio, Sevilla, Spain. Centro de Investigacion Biomedica en Red de Enfermedades Respiratorias (CIBERES), Instituto de Salud Carlos III, Madrid, Spain. FAU - Blanco-Aparicio, Marina AU - Blanco-Aparicio M AD - Complejo Hospitalario La Coruna, Respirology, La Coruna, Spain. FAU - Maiz, Luis AU - Maiz L AD - Hospital Ramon y Cajal, Respirology, Madrid, Spain. FAU - Garcia-Clemente, Marta Maria AU - Garcia-Clemente MM AD - Hospital Central de Asturias, Respirology, Oviedo, Spain. FAU - Luna-Paredes, Carmen AU - Luna-Paredes C AD - Hospital 12 de Octubre, Madrid, Spain. FAU - Mondejar-Lopez, Pedro AU - Mondejar-Lopez P AD - Hospital Arrixaca, Murcia, Spain. FAU - Ruiz-de-Valbuena, Marta AU - Ruiz-de-Valbuena M AD - Hospital La Paz, Madrid, Spain. FAU - Fernandez, Ofelia AU - Fernandez O AD - Hospital San Pedro de Alcantara, Caceres, Spain. FAU - Barrio, Maribel AU - Barrio M AD - Hospital San Pedro de Alcantara, Caceres, Spain. FAU - Gonzalez, Maribel AU - Gonzalez M AD - Hospital Infantil Nino Jesus, Madrid, Spain. FAU - Lopez-Neyra, Alejandro AU - Lopez-Neyra A AD - Hospital Ramon y Cajal, Paediatric, Madrid, Spain. FAU - Cols-I-Roig, Maria AU - Cols-I-Roig M AD - Hospital San Joan de Deu, Barcelona, Spain. FAU - Palou-Rotger, Alexandre AU - Palou-Rotger A AD - Hospital Son Espases, Palma de Mallorca, Spain. FAU - Gomez-de-Terreros-Caro, Francisco Javier AU - Gomez-de-Terreros-Caro FJ AD - Hospital San Pedro de Alcantara, Caceres, Spain. LA - eng LA - spa PT - Journal Article PT - Multicenter Study PT - Observational Study DEP - 20180707 PL - Spain TA - Arch Bronconeumol (Engl Ed) JT - Archivos de bronconeumologia JID - 101777538 RN - 0 (Aminophenols) RN - 0 (Aminopyridines) RN - 0 (Benzodioxoles) RN - 0 (Chloride Channel Agonists) RN - 0 (Drug Combinations) RN - 0 (Quinolones) RN - 1Y740ILL1Z (ivacaftor) RN - EGP8L81APK (lumacaftor) SB - IM MH - Adolescent MH - Adult MH - Aminophenols/*administration & dosage MH - Aminopyridines/*administration & dosage MH - Benzodioxoles/*administration & dosage MH - Child MH - Chloride Channel Agonists/*administration & dosage MH - *Compassionate Use Trials MH - Cystic Fibrosis/*drug therapy MH - Drug Combinations MH - Female MH - Humans MH - Male MH - Middle Aged MH - Quinolones/*administration & dosage MH - Retrospective Studies MH - Spain MH - Young Adult OTO - NOTNLM OT - Compassionate use OT - Cystic fibrosis OT - Exacerbacion pulmonar OT - Fibrosis quistica OT - Lumacaftor/ivacaftor OT - Pulmonary exacerbation OT - Uso compasivo OT - ppFEV1 EDAT- 2018/12/07 06:00 MHDA- 2019/10/28 06:00 CRDT- 2018/12/07 06:00 PHST- 2017/10/27 00:00 [received] PHST- 2018/04/09 00:00 [revised] PHST- 2018/05/01 00:00 [accepted] PHST- 2018/12/07 06:00 [entrez] PHST- 2018/12/07 06:00 [pubmed] PHST- 2019/10/28 06:00 [medline] AID - S0300-2896(18)30193-5 [pii] AID - 10.1016/j.arbres.2018.05.004 [doi] PST - ppublish SO - Arch Bronconeumol (Engl Ed). 2018 Dec;54(12):614-618. doi: 10.1016/j.arbres.2018.05.004. Epub 2018 Jul 7.