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The current literature inadequately addresses the extent to which remote monitoring should be integrated into care models for chronic respiratory diseases (CRDs). This study examined a remote monitoring program (RMP) in cystic fibrosis (CF) by exploring experiences, future perspectives, and use behavior over 3 years, with the aim of developing future directions for remote monitoring in CRDs. This was a mixed methods, multicenter, observational study in 5 Dutch CF centers following a sequential explanatory design. Self-designed questionnaires using the technology acceptance model were sent out to people with CF who had a minimum of 12 months of experience with the RMP and local health care professionals (HCPs). Questionnaire outcomes were used to inform semistructured interviews with HCPs and people with CF. Qualitative findings were reported following the COREQ (Consolidated Criteria for Reporting Qualitative Research) checklist. Anonymous data on use frequency of all people with CF were analyzed. Between the second quarter of 2020 and the end of 2022, a total of 608 people with CF were enrolled in the program, and a total of 9418 lung function tests and 2631 symptom surveys were conducted. In total, 65% (24/37) of HCPs and 89% (72/81) of people with CF responded to the questionnaire, and 7 HCPs and 12 people with CF participated in semistructured interviews. Both people with CF and HCPs were positive about remote monitoring in CF care and found the RMP a good addition to daily care (people with CF: 44/72, 61%; HCPs: 21/24, 88%). Benefits ranged from supporting individual patients to reducing health care consumption. The most valued monitoring tool was home spirometry by both people with CF (66/72, 92%) and HCPs (22/24, 92%). Downsides included the potential to lose sight of patients and negative psychosocial effects, as 17% (12/72) of people with CF experienced some form of stress due to the RMP. A large majority of people with CF (59/72, 82%) and HCPs (22/24, 92%) wanted to keep using the RMP in future, with 79% (19/24) of HCPs and 75% (54/72) of people with CF looking forward to more replacement of in-person care with digital care during periods of well-being. Future perspectives for the RMP were centered on creating hybrid care models, personalizing remote care, and balancing individual benefits with monitoring burden. Remote monitoring has considerable potential in supporting people with CF and HCPs within the CF care model. We identified 4 practice-based future directions for remote monitoring in CF and CRD care. The strategies, ranging from patient driven to prediction driven, can help clinicians, researchers, and policy makers navigate the rapidly changing digital health field, integrate remote monitoring into local care models, and align remote care with patient and clinician needs.

Portable spirometers are increasingly used to measure lung function at home, but doubts about the accuracy of these devices persist. These doubts stand in the way of the digital transition of chronic respiratory disease care, hence there is a need to address the accuracy of home spirometry in routine care across multiple settings and ages. This study aimed to assess the accuracy, reproducibility, and responsiveness to the treatment of home spirometry in long-term pediatric and adult cystic fibrosis care. This retrospective observational study was carried out in 5 Dutch cystic fibrosis centers. Home spirometry outcomes (forced expiratory volume in one second [FEV ], and forced vital capacity [FVC]) for 601 anonymized users were collected during 3 years. For 81 users, data on clinic spirometry and elexacaftor/tezacaftor/ivacaftor (ETI) use were available. Accuracy was assessed using Bland-Altman plots for paired clinic-home measurements on the same day and within 7 days of each other (nearest neighbor). Intratest reproducibility was assessed using the American Thoracic Society/European Respiratory Society repeatability criteria, the coefficient of variation, and spirometry quality grades. Responsiveness was measured by the percentage change in home spirometry outcomes after the start of ETI. Bland-Altman analysis was performed for 86 same-day clinic-home spirometry pairs and for 263 nearest neighbor clinic-home spirometry pairs (n=81). For both sets and for both FEV and FVC, no heteroscedasticity was present and hence the mean bias was expressed as an absolute value. Overall, home spirometry was significantly lower than clinic spirometry (mean ΔFEV 0.13 L, 95% CI 0.10 to 0.19; mean ΔFVC 0.20 L, 95% CI 0.14 to 0.25) and remained lower than clinic spirometry independent of age and experience. One-way ANOVA with post hoc comparisons showed significantly lower differences in clinic-home spirometry in adults than in children (Δmean 0.11, 95% CI -0.20 to -0.01) and teenagers (Δmean 0.14, 95% CI -0.25 to -0.02). For reproducibility analyses, 2669 unique measurement days of 311 individuals were included. Overall, 87.3% (2331/2669) of FEV measurements and 74.3% (1985/2669) of FVC measurements met reproducibility criteria. Kruskal-Wallis with pairwise comparison demonstrated that for both FVC and FEV , coefficient of variation was significantly lower in adults than in children and teenagers. A total of 5104 unique home measurements were graded. Grade E was given to 2435 tests as only one home measurement was performed. Of the remaining 2669 tests, 43.8% (1168/2669) and 43.6% (1163/2669) received grade A and B, respectively. The median percentage change in FEV from baseline after initiation of ETI was 19.2% after 7-14 days and remained stable thereafter (n=33). Home spirometry is feasible but not equal to clinic spirometry. Home spirometry can confirm whether lung functions remain stable, but the context of measurement and personal trends are more relevant than absolute outcomes.

Home spirometers have been widely implemented in the treatment of people with cystic fibrosis (CF). Frequent spirometry measurements at home could lead to earlier detection of exacerbations. However, previous research indicates that the long-term use of home spirometry is not well maintained by people with CF. We aimed to gain insight into the long-term uptake of home spirometry in regular multicenter CF care. Home spirometers combined with a remote monitoring platform were introduced in the treatment of people with CF in 5 Dutch CF centers starting in April 2020. Usage data from April 2020 to December 2022 were analyzed retrospectively. Survival analyses were conducted to assess use consistency over time, and t tests were used to evaluate the impact of increased pulmonary symptoms on home spirometry frequency. The effect of the initiation of a new treatment, Elexacaftor/Tezacaftor/Ivacaftor, on use frequency over time was assessed in a subgroup of participants with repeated measures ANOVA. During the observation period, a total of 604 people with CF were enrolled in the remote monitoring platform and 9930 home spirometry measurements were performed. After the initiation of home spirometry use, the number of users declined rapidly. One year after the initiation, 232 (54.2%) people with CF stopped using home spirometry. During the observation period, 67 (11.1%) users performed more than 20 measurements. Furthermore, the number of consistent home spirometry users decreased over time. After 600 days, only 1% of users had measured their lung function consistently every 31 days. Use frequency slightly increased during periods with increased pulmonary symptoms (ΔMean=0.45, t =-4,197; P<.001) and showed an initial rise followed by a decrease after starting treatment with Elexacaftor/Tezacaftor/Ivacaftor (ΔMean=0.45, t =-4,197; P<.001). Consistent uptake of home spirometry in people with CF is low but increases around periods of changing symptoms. A clear strategy for the organization of remote care seemed to improve the long-term uptake of home spirometry. Nevertheless, home spirometry and its intensity are not a goal on their own but should be used as a tool to reach individual goals within local contexts.

Background: In recent years, cystic fibrosis transmembrane regulator (CFTR) modulating therapy has made it possible to treat the underlying pathophysiological defect in children with cystic fibrosis (CF). Response to therapy varies among patients. We investigated the immune responses and exhaled breath profile changes after the initiation of CFTR modulator therapy to explore their potential as markers of therapy response. Methods: We performed a prospective, longitudinal proof-of-principle study, investigating immune responses and exhaled breath volatile organic component (VOC) profiles prior to and during the initiation of therapy with Lumacaftor/Ivacaftor in a cohort of 17 patients with CF aged 2 to 6 years old. Response to therapy was assessed based on clinical markers and the decrease in sweat chloride. Whole blood stimulation assays were performed at t = 0, 6 and 18 weeks, while VOC analysis was performed at t = 0 and 18 weeks. Results: A pattern of immune reconstitution was found in the first 4 months of therapy. The same pattern was found in responders and non-responders. Exhaled breath VOC profiles were significantly affected by therapy. A trend toward a significant difference was found between responders and non-responders. Conclusions: Pediatric CF patients show a pattern of immune reconstitution after the initiation of CFTR modulating therapy. We hypothesize that this could be explained by the need for a pro-inflammatory profile for a more effective clearance of latent airway pathogens in the initial phase. The exhaled breath profile also clearly changes after the initiation of therapy, indicating the therapy’s influence on airway inflammation and oxidative stress; thus, it might predict the response to therapy.

Background: The COVID-19 pandemic has boosted the use of forced expiratory volume in 1 second (FEV1) telemonitoring in pediatric asthma, but a consensus on its most efficient and effective implementation is still lacking. To find answers, it is important to study how such an intervention is perceived, experienced, and used by both patients and health care professionals (HCPs). Objective: The aim of this study was to provide perspectives on how FEV1 home monitoring should be used in pediatric asthma. Methods: This is a qualitative, multicenter, prospective, observational study which included patients with asthma aged 6-16 and HCPs. Primary outcomes were results of 2 surveys that were sent to all participants at study start and after 3-4 months. Secondary outcomes consisted of FEV1 device usage during 4 months after receiving the FEV1 device. Results: A total of 39 participants (26 patients and 13 HCPs) were included in this study. Survey response rates were 97% (38/39) at the start and 87% (34/39) at the end of the study. Both patients and HCPs were receptive toward online FEV1 home monitoring and found it contributive to asthma control, self-management, and disease perception. The main concerns were about reliability of the FEV1 device and validity of home-performed lung function maneuvers. FEV1 devices were used with a median frequency of 7.5 (IQR 3.3-25.5) during the 4-month study period. Conclusions: Patients and HCPs are receptive toward online FEV1 home monitoring. Frequency of measurements varied largely among individuals, yet perceived benefits remained similar. This emphasizes that online FEV1 home monitoring strategies should be used as a means to reach individual goals, rather than being a goal on their own.

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 FEV1, 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 FEV1 <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 FEV1 was 11.8/13.7% (p ≤ .001) and BMI was 0.49/1.87 kg/m2 (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. Elexacaftor/tezacaftor/ivacaftor (ETI) is effective in a real‐life setting with severe cystic fibrosis lung disease patients. The effect maintains for up to 12 months and the treatment was well tolerated and reduced the number of exacerbations. Sputum production is significantly lower after ETI initiation.

Remote care usefulness and climate change co-benefits should be addressed simultaneously to incentivize political action. To assess the changes in healthcare consumption, lung function and greenhouse gas (GHG) emissions during the COVID-19 pandemic in Dutch cystic fibrosis (CF) care. Retrospective multicentre observational study in five Dutch CF centres. Eighty-one participants were included. Healthcare consumption was described alongside the COVID-19 Stringency Index (2019-2022). Travel related GHG emissions were calculated for every clinic visit. Changes in percentage predicted Forced Expiratory Volume in one second (ppFEV1) were assessed using a paired-samples -test. Healthcare consumption patterns followed COVID-19 public health measure stringency but returned back to the \"old normal\". Emission of 5.450, 3 kg of carbon dioxide equivalents were avoided while quality of care was relatively preserved. ppFEV1 declined as expected ( Means 3.69%, 95%CI 2.11-5.28). Remote monitoring of lung function and symptoms and teleconsultations in CF can reduce GHG emissions while maintaining quality of care. As health sectors constitute a large share of national climate change footprints, digital health can partly alleviate this burden by reducing private travel.

Self-reported medical history information is included in many studies. However, data on the validity of Web-based questionnaires assessing medical history are scarce. If proven to be valid, Web-based questionnaires may provide researchers with an efficient means to collect data on this parameter in large populations. The aim of this study was to assess the validity of a Web-based questionnaire on chronic medical conditions, allergies, and blood pressure readings against obstetric records and data from general practitioners. Self-reported questionnaire data were compared with obstetric records for 519 pregnant women participating in the Dutch PRegnancy and Infant DEvelopment (PRIDE) Study from July 2011 through November 2012. These women completed Web-based questionnaires around their first prenatal care visit and in gestational weeks 17 and 34. We calculated kappa statistics (κ) and the observed proportions of positive and negative agreement between the baseline questionnaire and obstetric records for chronic conditions and allergies. In case of inconsistencies between these 2 data sources, medical records from the woman's general practitioner were consulted as the reference standard. For systolic and diastolic blood pressure, intraclass correlation coefficients (ICCs) were calculated for multiple data points. Agreement between the baseline questionnaire and the obstetric record was substantial (κ=.61) for any chronic condition and moderate for any allergy (κ=.51). For specific conditions, we found high observed proportions of negative agreement (range 0.88-1.00) and on average moderate observed proportions of positive agreement with a wide range (range 0.19-0.90). Using the reference standard, the sensitivity of the Web-based questionnaire for chronic conditions and allergies was comparable to or even better than the sensitivity of the obstetric records, in particular for migraine (0.90 vs 0.40, P=.02), asthma (0.86 vs 0.61, P=.04), inhalation allergies (0.92 vs 0.74, P=.003), hay fever (0.90 vs 0.64, P=.001), and allergies to animals (0.89 vs 0.53, P=.01). However, some overreporting of allergies was observed in the questionnaire and for some nonsomatic conditions sensitivity of both measurement instruments was low. The ICCs for blood pressure readings ranged between 0.72 and 0.92 with very small mean differences between the 2 methods of data collection. Web-based questionnaires can be used to validly collect data on many chronic disorders, allergies, and blood pressure readings among pregnant women.

Meier-Gorlin syndrome (MGS) is a rare autosomal recessive primordial dwarfism disorder, characterized by microtia, patellar applasia/hypoplasia, and a proportionate short stature. Associated clinical features encompass feeding problems, congenital pulmonary emphysema, mammary hypoplasia in females and urogenital anomalies, such as cryptorchidism and hypoplastic labia minora and majora. Typical facial characteristics during childhood comprise a small mouth with full lips and micro-retrognathia. During ageing, a narrow, convex nose becomes more prominent. The diagnosis MGS should be considered in patients with at least two of the three features of the clinical triad of microtia, patellar anomalies, and pre- and postnatal growth retardation. In patients with short stature and/or microtia, the patellae should be assessed with care by ultrasonography before age 6 or radiography thereafter. Mutations in one of five genes ( ORC1 , ORC4 , ORC6 , CDT1 , and CDC6 ) of the pre-replication complex, involved in DNA-replication, are detected in approximately 67-78 % of patients with MGS. Patients with ORC1 and ORC4 mutations appear to have the most severe short stature and microcephaly. Management should be directed towards in-depth investigation, treatment and prevention of associated problems, such as growth retardation, feeding problems, hearing loss, luxating patellae, knee pain, gonarthrosis, and possible pulmonary complications due to congenital pulmonary emphysema with or without broncho- or laryngomalacia. Growth hormone treatment is ineffective in most patients with MGS, but may be effective in patients in whom growth continues to decrease after the first year of life (usually growth velocity normalizes after the first year) and with low levels of IGF1. At present, few data is available about reproduction of females with MGS, but the risk of premature labor might be increased. Here, we propose experience-based guidelines for the regular care and treatment of MGS patients.

Objective To validate use of the Manchester triage system in paediatric emergency care.Design Prospective observational study.Setting Emergency departments of a university hospital and a teaching hospital in the Netherlands, 2006-7.Participants 17 600 children (aged <16) visiting an emergency department over 13 months (university hospital) and seven months (teaching hospital).Intervention Nurses triaged 16 735/17 600 patients (95%) using a computerised Manchester triage system, which calculated urgency levels from the selection of discriminators embedded in flowcharts for presenting problems. Nurses over-ruled the urgency level in 1714 (10%) children, who were excluded from analysis. Complete data for the reference standard were unavailable in 1467 (9%) children leaving 13 554 patients for analysis.Main outcome measures Urgency according to the Manchester triage system compared with a predefined and independently assessed reference standard for five urgency levels. This reference standard was based on a combination of vital signs at presentation, potentially life threatening conditions, diagnostic resources, therapeutic interventions, and follow-up. Sensitivity, specificity, and likelihood ratios for high urgency (immediate and very urgent) and 95% confidence intervals for subgroups based on age, use of flowcharts, and discriminators.Results The Manchester urgency level agreed with the reference standard in 4582 of 13 554 (34%) children; 7311 (54%) were over-triaged and 1661 (12%) under-triaged. The likelihood ratio was 3.0 (95% confidence interval 2.8 to 3.2) for high urgency and 0.5 (0.4 to 0.5) for low urgency; though the likelihood ratios were lower for those presenting with a medical problem (2.3 (2.2 to 2.5) v 12.0 (7.8 to 18.0) for trauma) and in younger children (2.4 (1.9 to 2.9) at 0-3 months v 5.4 (4.5 to 6.5) at 8-16 years).Conclusions The Manchester triage system has moderate validity in paediatric emergency care. It errs on the safe side, with much more over-triage than under-triage compared with an independent reference standard for urgency. Triage of patients with a medical problem or in younger children is particularly difficult.