What Is Most Suitable for Children With Cystic Fibrosis—The Relationship Between Spirometry, Oscillometry, and Multiple Breath Nitrogen Washout

IntroductionIn cystic fibrosis (CF), pathological lung changes begin early in life. The technological progress currently gives many diagnostic possibilities. However, pulmonary function testing in children remains problematic.ObjectivesOur study aimed to correlate the results of impulse oscillometry (IOS) with those of multiple breath nitrogen washout (MBNW) in our pediatric CF population. We also compared those parameters between the groups with and without spirometric features of obturation.MethodsWe collected 150 pulmonary function test sets, including spirometry, IOS, and MBNW in patients with CF aged 12.08 ± 3.85 years [6–18]. The study group was divided into two subgroups: IA (without obturation) and IB (with obturation). We also compared Sacin, Scond, and oscillometry parameters of 20 patients aged 14–18 years who reached the appropriate tidal volume (VT) during MBNW.ResultsStatistical analysis showed a negative correlation between lung clearance index (LCI) and spimoetric parameters. Comparison of subgroups IA (n = 102) and IB (n = 48) indicated a statistically significant difference in LCI (p < 0.001) and FEV1z-score (p < 0.001), FEV1% pred (p < 0.001), MEF25z-score (p < 0.001), MEF50 z-score (p < 0.001), MEF75 z-score (p < 0.001), R5% pred (p < 0.05), and R20% pred (p < 0.01). LCI higher than 7.91 was found in 75.33% of the study group, in subgroup IB—91.67%, and IA−67.6%.ConclusionsLCI derived from MBNW may be a better tool than IOS for assessing pulmonary function in patients with CF, particularly those who cannot perform spirometry.

Stride Velocity 95th Centile: Insights into Gaining Regulatory Qualification of the First Wearable-Derived Digital Endpoint for use in Duchenne Muscular Dystrophy Trials

In 2019, stride velocity 95th centile (SV95C) became the first wearable-derived digital clinical outcome assessment (COA) qualified by the European Medicines Agency (EMA) for use as a secondary endpoint in trials for Duchenne muscular dystrophy. SV95C was approved via the EMA’s qualification pathway for novel methodologies for medicine development, which is a voluntary procedure for assessing the regulatory acceptability of innovative methods used in pharmaceutical research and development. SV95C is an objective, real-world digital ambulation measure of peak performance, representing the speed of the fastest strides taken by the wearer over a recording period of 180 hours. SV95C is correlated with traditional clinic-based assessments of motor function and has greater sensitivity to clinical change over 6 months than other wearable-derived stride variables, for example, median stride length or velocity. SV95C overcomes many limitations of episodic, clinic-based motor function testing, allowing the assessment of ambulation ability between clinic visits and under free-living conditions. Here we highlight considerations and challenges in developing SV95C using evidence generated by a high-performance wearable sensor. We also provide a commentary of the device’s technical capabilities, which were a determining factor in the regulatory approval of SV95C. This article aims to provide insights into the methods employed, and the challenges faced, during the regulatory approval process for researchers developing new digital tools for patients with diseases that affect motor function.

Annual Baseline King-Devick Oculomotor Function Testing Is Needed Due to Scores Varying by Age

Objective: To document baseline King-Devick (K-D) oculomotor function scores for male and female participants aged between 4 and 20 years old. Methods: Utilising a cross section of schools, rugby clubs and gymnastic clubs, 1936 participants (1300 male, 636 female) completed the spiral-bound K-D test for the identification of disturbed oculomotor function. Results: This study identified that overall, the baseline scores of the K-D test became faster by 1.4 (0.3 to 4.5) s per year, when compared with the previous age group in the same number of reading card groups. When comparing normative values of the original K-D validation study with the same age groups of the current cohort, participants aged 6 to 11 years recorded a faster baseline time (range 3.5 to 8.6 s), while those in the 12 to 14 years. age group recorded slower baseline times (range −3.9 to −7.9 s). Discussion: In general, there were age group differences, but not sex differences, for K-D test times in the current cohort. Analysis of single card times, across all age groups, showed changes likely due to improved reading time. Conclusion: The results support the need for individualised annual pre-injury baseline testing of the K-D test.

Olfactory function testing before and after anesthesia

This study aimed to determine whether anesthesia would affect olfactory function. Patients who were admitted for surgical intervention that did not include the nasal cavity and paranasal sinuses were included in this prospective cohort study. Structured medical history was taken from the patients, including the following: age, sex, smoking history, alcohol intake, current medications, and sleep deficits prior to surgery. Before surgery, patients were asked for a self-rating of their olfactory function. Olfactory function was also measured using Sniffin’ Sticks comprising measures of odor threshold, discrimination, and identification. The mean interval between olfactory tests was 6 days (range 3–12 days). Seventy-three patients were included in the study, 34 men and 39 women. Olfactory scores were consistent before and after surgery as indicated by correlative analyses (p < 0.05). Odor thresholds, discrimination, identification, and composite TDI scores did not change significantly, whereas odor identification scores increased (p = 0.011) after surgery. In conclusion, post-operative olfactory scores remained stable. However, identification scores exhibited a slight increase which can be attributed to a retest effect. Overall, the present results indicate that surgery outside of the nasal and paranasal sinus region performed in general anesthesia has no major effect on the sense of smell.