BackgroundThe use of patient‐reported outcomes measures (PROMs), such as quality of life or symptoms like pain or fatigue, is increasingly embraced within patient‐centred care and shared decision making.ObjectivesTo investigate: (a) how patients and health professionals think about using PROMs during routine medical consultations; (b) for which purpose(s), patients and health professionals want to use PROMs during those consultations; and (c) how patients interpret PROMs information presented in various formats. People with Parkinson's disease and their health professionals served as case example.MethodsWe performed semi‐structured interviews with patients (N = 13) and professionals (N = 7 neurologists; N = 7 physiotherapists). We also used a survey in which patients (N = 115) were shown six figures displaying different information types. Presentation formats of this information varied (line/bar graphs). Interpretation by patients, perceived usefulness of information, attitude towards using information during routine medical consultations and (hypothetical) decisions were assessed.FindingsPatients and professionals were generally positive about using PROMs during medical consultations. Professionals stressed the opportunity to monitor changes in individual PROMs over time. Patients were primarily positive about aggregated PROMs to make treatment decisions. This information was also most often interpreted correctly, especially when presented through a line graph (90.1% correct). Professionals thought patients should take the initiative in discussing PROMs, whereas patients thought professionals should do so.Conclusion/DiscussionWhen used in routine medical consultations, PROMs seem to have potential to support shared decision making and facilitate patient‐professional communication. However, training seems needed for both patients and professionals to facilitate actual discussion and proper interpretation.

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Background: The novel cystic fibrosis transmembrane conductance regulator (CFTR) modulators, ivacaftor, lumacaftor, and tezacaftor, are the first drugs directly targeting the underlying pathophysiological mechanism in cystic fibrosis (CF); however, independent studies describing their pharmacokinetics are lacking. The aim of this study was to develop a quantification method for ivacaftor and its 2 main metabolites, lumacaftor and tezacaftor, in plasma and sputum using liquid chromatography with tandem mass spectrometry. Methods: The developed method used a small sample volume (20 µL) and simple pretreatment method; protein precipitation solution and internal standard were added in one step to each sample. Liquid chromatography with tandem mass spectrometry was performed for a total run time of 6 minutes. The method was validated by assessing selectivity, carryover, linearity, accuracy and precision, dilution, matrix effects, and stability. Results: The selectivity was good as no interference from matrices was observed. In the concentration range from 0.01 to 10.0 mg/L, calibration curves were linear with a correlation coefficient >0.9997 for all compounds. The within-run and between-run accuracy were between 99.7% and 116% at the lower limit of quantitation (LLOQ) and between 95.8% and 112.9% for all concentrations above LLOQ for all analytes in plasma and sputum. Within-run and between-run precisions were <12.7% for LLOQ and <6.7% for the higher limit of quantitation. Samples were stable, with no significant degradation at examined temperatures and time points. Clinical applicability was revealed by analyzing samples from 2 patients with CF. Conclusions: The presented method enables simultaneous quantification of ivacaftor, lumacaftor, and tezacaftor in plasma and sputum and is an improvement over previous methods because it uses smaller sample volumes, a simple pretreatment protocol, and includes tezacaftor. In future studies, it can be applied for examining pharmacokinetics characteristics of new CF transmembrane conductance regulator modulators.

Elexacaftor/tezacaftor/ivacaftor (ETI) is a cystic fibrosis (CF) transmembrane conductance regulator modulator, which has shown efficacy in CF patients (≥6 years) with ≥1 Phe508del mutation and a minimal function mutation. In October 2019, ETI became available on compassionate use basis for Dutch CF patients with severe lung disease. Our objective was to investigate safety and efficacy of ETI in this patient group in a real‐life setting. A multicenter longitudinal observational study was conducted to examine changes in FEV 1 , BMI, and adverse events at initiation and 1, 3, 6, and 12 months after starting ETI. The number of exacerbations was recorded in the 12 months before and the 12 months after ETI treatment. Patients eligible for compassionate use had a FEV 1 <40% predicted. Wilcoxon signed‐rank test analyzed changes over time. Twenty subjects were included and followed up for up to 12 months after starting ETI. Treatment was well tolerated with mild side effects reported, namely, rash (15%) and stomach ache (20%) with 80% resolving within 1 month. Mean absolute increase of FEV 1 was 11.8/13.7% ( p ≤ .001) and BMI was 0.49/1.87 kg/m 2 ( p < .001–0.02) after 1/12 months, respectively. In comparison to the number of exacerbations pretrial, there was a marked reduction in exacerbations after initiation. Our findings show long‐term effects of treatment with ETI in patients with severe CF lung disease in a real‐life setting. Treatment with ETI is associated with increased lung function and BMI, less exacerbations, and only mild side effects.