Value and limitations of real-world data to understand paediatric adherence to positive airway pressure therapy
Authors: Robinder G Khemani
Journal: The Lancet Digital Health (2019)

In The Lancet Digital Health, Rakesh Bhattacharjee and colleagues10Bhattacharjee R Benjafield AV Armitstead J et al.Adherence in children using positive airway pressure therapy: a big-data analysis.Lancet Digital Health. 2019; (published online Dec 23)https://doi.org/10.1016/S2589-7500(19)30214-6Summary Full Text Full Text PDF Scopus (27) Google Scholar report prevalence of adherence and risk factors for non-adherence in children who use positive airway pressure (PAP) therapy, with a focus on children with obstructive sleep apnoea. The investigators took a big-data approach, using data captured directly from medical devices by ResMed (San Diego, CA, USA). Data were uploaded into a secure cloud-based application that could be used to track patient adherence. The investigators found that 7854 (38·2%) of 20 553 analysed children had 30 consecutive days of non-usage of PAP therapy within the first 90 days of device prescription. Only 9504 (46·3%) of analysed children met the Centers for Medicare and Medicaid Services (CMS) criteria for adherence to PAP therapy, mandating at least 4 h of use per night on at least 70% of the nights during a consecutive 30-day period. This adherence prevalence is lower than that reported in adults.1Rotty MC Mallet JP Suehs CM et al.Is the 2013 American Thoracic Society CPAP-tracking system algorithm useful for managing non-adherence in long-term CPAP-treated patients?.Respir Res. 2019; 20: 209Crossref PubMed Scopus (7) Google Scholar Importantly, the investigators found that several variables were associated with adherence, including age, residual apnoea–hypopnoea index, device pressure settings, mask leak, and use of a specific patient engagement programme. Overall, there are many strengths to the study by Bhattacharjee and colleagues, including the use of real-world data from a large paediatric population of PAP users in the USA. This approach to data gathering allows clinicians to assess how these devices are used in the home by patients, without the potential biases or changes in behaviour that might be introduced when patients specifically volunteer to be in a research study.2Pepin JL Woehrle H Liu D et al.Adherence to positive airway therapy after switching from CPAP to ASV: a big data analysis.J Clin Sleep Med. 2018; 14: 57-63Crossref PubMed Scopus (44) Google Scholar Furthermore, the findings appear to have biological plausibility. Adolescents have historically had low adherence to many forms of medical therapy,3Borus JS Laffel L Adherence challenges in the management of type 1 diabetes in adolescents: prevention and intervention.Curr Opin Pediatr. 2010; 22: 405-411Crossref PubMed Scopus (221) Google Scholar a notion reinforced in this study. The youngest children in the study (aged >4 to <6 years) also had low adherence to PAP, which might be explained by developmental differences and physical interface limitations. This effect was abrogated by multivariable modelling after controlling for other factors such as mask leak, suggesting that improvement in interfaces that reduce mask leak in this young age group could be particularly helpful to improve adherence. Furthermore, increased residual apnoea–hypopnoea indices and reduced pressures were associated with nonadherence, and hence are potentially modifiable factors. Finally, a voluntary patient engagement programme that provided electronic feedback related to usage, leak, and residual apnoea–hypopnoea index was associated with increased adherence. This effect could reflect direct benefit from the programme, or a self-selection of highly motivated patients and families who volunteered for the programme. Of course, these data need to be interpreted in the context of the limitations of the study design. This was an observational study, so no direct causal inferences should be implied. This point of caution is particularly important for the patient engagement programme4Woehrle H Arzt M Graml A et al.Effect of a patient engagement tool on positive airway pressure adherence: analysis of a German healthcare provider database.Sleep Med. 2018; 41: 20-26Crossref PubMed Scopus (28) Google Scholar, 5Mendoza-Ruiz A Dylgjeri S Bour F Damagnez F Leroux K Khirani S Evaluation of the efficacy of a dedicated table to improve CPAP adherence in children: a pilot study.Sleep Med. 2019; 53: 60-64Crossref PubMed Scopus (10) Google Scholar, 6Sunwoo BY Light M Malhotra A Strategies to augment adherence in the management of sleep-disordered breathing.Respirology. 2019; (published online July 3.)DOI:10.1111/resp.13589Crossref Scopus (26) Google Scholar and PAP pressure levels, as other factors such as patient motivation or severity of disease can confound the relationships of patient engagement and PAP pressure with adherence. Additionally, the authors noted important age-related differences in adherence, but did not specifically investigate whether risk factors for adherence differ as a function of age. It is possible that, for example, the amount of leak has a differential association with adherence for a small child compared with an adolescent. Evaluating whether this difference exists would be informative to improve the design of patient-targeted interventions, which are appropriate for the child's developmental stage and age. Furthermore, age and development are not congruent for many children receiving PAP therapy, as many children have a developmental delay, which can impact adherence.7Kang EK Xanthopoulos MS Kim JY et al.Adherence to positive airway pressure for the treatment of obstructive sleep apnea in children with developmental disabilities.J Clin Sleep Med. 2019; 15: 915-921Crossref PubMed Scopus (24) Google Scholar The medical devices used in this study were from only one company, and only two device models were used. This restriction could impede the generalisability of the findings. There are many ways to classify adherence that can greatly influence the ability to compare results of different studies,5Mendoza-Ruiz A Dylgjeri S Bour F Damagnez F Leroux K Khirani S Evaluation of the efficacy of a dedicated table to improve CPAP adherence in children: a pilot study.Sleep Med. 2019; 53: 60-64Crossref PubMed Scopus (10) Google Scholar, 7Kang EK Xanthopoulos MS Kim JY et al.Adherence to positive airway pressure for the treatment of obstructive sleep apnea in children with developmental disabilities.J Clin Sleep Med. 2019; 15: 915-921Crossref PubMed Scopus (24) Google Scholar, 8Naik S Al-Halawani M Kreinin I Kryger M Centers for Medicare and Medicaid Services positive airway pressure adherence criteria may limit treatment to many medicare beneficiaries.J Clin Sleep Med. 2019; 15: 245-251Crossref PubMed Scopus (15) Google Scholar, 9Shaughnessy GF Morgenthaler TI The effect of patient-facing applications on positive airway pressure therapy adherence: a systematic review.J Clin Sleep Med. 2019; 15: 769-777Crossref PubMed Scopus (10) Google Scholar with no clear definitions specifically designed for paediatric PAP. This inconsistency is important because, as the authors note, there are age-dependent differences in the expected duration of sleep, for example. The outcomes reported attempt to investigate related but potentially distinct aspects of adherence (inadequate use over a timeframe with the CMS classification vs patient or provider instruction for termination of therapy). The risk factors leading to these outcomes might be different, although this difference was not specifically investigated in the Article. Finally, the study design precludes understanding why participants were nonadherent to therapy, leaving the possibility that the estimates of PAP nonadherence are overestimated. Nevertheless, these data provide valuable insight for researchers and practitioners in paediatric sleep medicine and provide an excellent benchmark to help to develop intervention programmes or strategies to improve PAP adherence. Furthermore, the authors have provided a method and framework that can be iteratively used to measure the effect of interventions, either through controlled trials or quality-improvement initiatives. I declare no competing interests. Adherence in children using positive airway pressure therapy: a big-data analysisTo our knowledge, our study represents the largest analysis of children using PAP therapy to date. The findings suggest that adherence to PAP therapy is lower than in previous reports from adults. However, numerous actionable factors were associated with improvements in adherence and should be used strategically in clinical decision making to improve PAP adherence in children. Full-Text PDF Open Access