Physical fitness and habitual physical activity in children with cystic fibrosis - do they improve with elexacaftor/tezacaftor/ivacaftor therapy?

Objectives: The aim of this ongoing longitudinal study is to examine the effects of elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) on aspects of physical fitness (PF, strength endurance, explosive power, coordination under time pressure, coordination for the precision aspect, trunk flexibility, peak workload, habitual physical activity (HPA, steps/day, intensity), and zscores of FEV1 and BMI in children with cystic fibrosis (CF)). All children have been receiving ELX/TEZ/IVA for 19.2 ± 10.4 (11–33) months. Methods: Six children with CF agreed to participate (3f/3 m) mean age 13 ± 4yrs (9–17 yrs), FEV1 z-score − 1.017 ± 1.817 (-3.2–0.8). PFwas assessed using 5 test items at baseline and 4.51 ± 0.98 years later;strength endurance (PU push-ups, SU sit-ups), explosive power (SLJ standing long jump), coordination under time pressure (JSS jumping side to side), coordination for the precision aspect (BB balancing backward on beams), and trunk flexibility (FB, forward bend). Cycle-ergometry (Godfrey-protocol) was used to determine Wpeak. Lung function was measured by spirometry. Results: Significant improvements were seen in test items representing strength endurance (PU, SU) and coordination under time pressure (JSS) (p < .05). Although HPA expressed as steps/day remained the same, thetime of moderate to vigorous intensity of HPA decreases significantly (p < .05). A slight, not significant, improvement of BMI Z-score was observed (p > .05). No or small changes could be observed for the parameter FEV1 z-score, Wpeak, PF, SLJ, BB and FB (p > 0.05). Conclusion: ELX/TEZ/IVA therapy seems to be a facilitator to increase some aspects of PF. The decrease in intensity of HPA is possibly an effect of the COVID-19 Pandemic that has already been described in healthy and asthmatic children. In this ongoing study, we assume that clearer effects will be shown with a greater number of children included. However, ELX/ TEZ/IVA enhanced muscle strength endurance in children with CF.

ECMO as a bridging to recovery in a 19-year-old patient with e-cigarette / vaping-product associated lung injury (EVALI)

Introduction: E-cigarettes are becoming increasingly popular among the younger generation. An increasing number of e-cigarette or vaping-product associated lung injuries (EVALI) has been published with some severe and even lethal outcomes. We report an unusually severe case with veno-venous ECMO being used as a bridging to recovery. Case Report: A previously healthy 19- year-old male was admitted to a hospital with shortness of breath. He had been suffering from progressive dyspnoea for about four weeks. The patient had been a frequent user of e-cigarettes and hookahs. Treatment was begun with noninvasive ventilation and continued with endotracheal intubation after rapid deterioration. Since hypercarbic pulmonary failure persisted the patient was transferred to an academic referral hospital. After a futile conservative approach with prone positioning, veno-venous ECMO was initiated. Extensive tests for infectious diseases (including SARS-CoV-2) or autoimmune conditions were nega- tive and the diagnosis of EVALI was made after transbronchial biopsy. Steroids and azithromycin were administered, resulting in a slow improvement after three weeks. The patient was eventually weaned from ECMO and ventilation after tracheostomy. He was discharged to a rehab facility and is now almost asymptomatic. However, pulmonary function testing does still demonstrate a marked obstruction predominantly affecting the small airways. Conclusion: After exclusion of other causes, and given the appropriate case history, EVALI must be considered as a rare diagnosis for pulmonary failure. Since the time to recovery can be substantial, veno-venous ECMO might be a therapeutic option bridging the patient until pulmonary function improves. Early transfer to a specialised facility appears appropriate.