The combination of the corrector lumacaftor with the potentiator ivacaftor has been approved for treatment of cystic fibrosis (CF) patients homozygous for the Phe508del CFTR mutation. There are no reports detailing the effect of lumacaftor-ivacaftor on physical activity (PA) and exercise tolerance. We performed incremental cardiopulmonary exercise testing (CPET) and we assessed PA pre-and post 2 years initiation of lumacaftor-ivacaftor in three CF adults. PA of mild intensity improved by +13% in patient 1, + 84% in patients 2 and + 89% in patient 3. Oxygen uptake increased both at anaerobic threshold and at peak exercise (patient 1 + 33%, patient 2 + 42% and patient 3 + 20%). Daily physical activities and exercise tolerance improved after two years of lumacaftorivacaftor therapy.

Background Regular physical activity (PA) is a valued part of cystic fibrosis (CF) care. Although the accelerometer, SenseWear Armband (SWA), accurately measures habitual PA in CF, it is mostly used for research purposes. For the first time, we analyzed different methods of measuring PA in daily life by the use of smartphones and other electronic devices such as smartwatch and Fitbit. Methods Twenty-four stable adults with CF (mean age 37.5 ± 11.5SD yrs.; FEV1 58 ± 19% predicted, BMI 22.9 ± 3.2) were studied. Daily PA was monitored for seven consecutive days. All patients wore the accelerometer SWA and at the same time they monitored PA with the electronic device they used routinely. They were allocated into one of four arms according to their device: Smartwatch, Fitbit, Android smartphones and iOS smartphones. PA related measurements included: duration of PA, energy expenditure, number of steps. Results There was a good agreement between SWA and Fitbit for number of steps (p = 0.605) and energy expenditure (p = 0.143). iOS smartphones were similar to SWA in monitoring the number of steps (p = 0.911). Significant differences were found between SWA and both Smartwatch and Android smartphones. Conclusions Fitbit and iOS smartphones seem to be a valuable approach to monitor daily PA. They provide a good performance to measure step number compared to SWA.

Introduction: Systemic sclerosis (SSc) is an autoimmune disease characterized by the overproduction of collagen leading to fibrosis of the skin and internal organs. Interstitial lung disease (ILD) is one of the major causes of death in patients with SSc. Exercise tolerance can be investigated by cardio-pulmonary exercise testing (CPET). First-line therapies in patients with SSc associated with ILD (SSc-ILD) include cyclophosphamide and mycophenolate mofetil (MMF). The aim of this study was to evaluate the response of patients with SSc-ILD to MMF by means of CPET. Methods: Ten consecutive SSc patients were enrolled in this study. All SSc patients underwent clinical evaluation, echocardiography, pulmonary function tests, high-resolution computed tomography (HRCT) and CPET at baseline and after 2 years of therapy with MMF. Results: After 24 months of treatment with MMF (target dose 1500 mg twice daily), forced vitality capacity, diffusing capacity of the lungs for carbon monoxide and systolic pulmonary arterial pressure had not improved significantly and there were no significant differences in HRCT findigns. In addition, peak oxygen uptake (V 0 O 2 peak) and ventilatory equivalents for carbon dioxide production (V 0 E/V 0 CO 2 slope) had not improved significantly. In contrast, there was a significant improvement from baseline to 24 months of treatment in the respiratory exchange ratio [median (interquartile range): 1.07 (0.92-1.22) vs. 1.26 (1.22-1.28), respectively; p \ 0.01] and in the Borg scale for leg discomfort [median (interquartile range): 5 (5-7) vs. 4 (3-4), respectively; p \ 0.01]. Conclusion: These data from our pilot study on a small cohort of SSc patients are the first to demonstrate that treatment with MMF can improves exercise tolerance and leg discomfort in patients with SSc-ILD. These preliminary results need to be confirmed in large randomized studies.

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