Spectrum of Clinical Research in Juvenile Idiopathic Arthritis: A Cross-Sectional Analysis of Registered Studies in Clinicaltrials.gov and Clinicaltrialsregister.eu

The management of juvenile idiopathic arthritis (JIA) has improved tremendously in recent years due to the introduction of new drug therapies but remains complex in terms of non-pharmaceutical issues. In order to determine the direction of scientific progress by characterizing the current spectrum of ongoing clinical research in JIA, we analyzed all ongoing studies in the field of JIA—registered in clinicaltrials.gov and clinicaltrialsregister.eu—concerning sponsoring, enrollment, duration, localization, and particularly objectives. The close of the database was 7 January 2021. After identifying double-registered studies, n = 72 went into further analysis. Of these, 61.1% were academia-sponsored and 37.5% were sponsored by the pharma industry. The majority of the studies was of the interventional type (77.8%), while others (22.2%) were observational. The median planned enrollments were 100 participants (interventional studies) and 175 participants (observational studies), respectively. The duration differed remarkably from one month to more than 15 years, with a median of 42.5 months. A total of 61.1% of studies were located in a single country, and 38.9% were in several. Europe and North America clearly dominated the study localizations. The study objectives were DMARDs (56.9%), followed by diagnostics and disease activity measurement (18.1%), and medication other than DMARD (12.5%), besides others. Studies on DMARDs were mainly sponsored by industry, predominantly interventional studies on established and novel biologics, with several on specific issues such as systemic JIA and others. The spectrum of registered studies is currently centered on drug therapy and diagnostics, while other issues in JIA play a subordinated role in current research. Drug development was transferred from adult rheumatology into the JIA population with little innovation for children. Future research should take specific pediatric needs better into account.

Spectrum of Clinical Research in Juvenile Idiopathic Arthritis: a Cross-Sectional Analysis of Registered Studies in Clinicaltrials.gov and Clinicaltrialsregister.eu

Management of Juvenile idiopathic arthritis (JIA) has improved tremendously in recent years due to the introduction of new drug therapies but remains complex also in terms of non-pharmaceutical issues. In order to determine the direction of scientific progress by characterizing the current spectrum of ongoing clinical research in JIA, we analyzed all ongoing studies in the field of JIA registered in clinicaltrials.gov and clinicaltrialsregister.eu concerning sponsoring, enrollment, duration, localization, and particularly objectives. Close of database was 7 January 2021. After identifying doubled-registered studies, N=72 went into further analysis. Of these, 61.1% were academia-sponsored and 37.5% by pharma industry. The majority of studies was of interventional type (77.8%), while others (22.2%) were observational. Median planned enrollments were 100 participants (interventional studies) and 175 participants (observational studies), respectively. Duration differed remarkably from one month to more than 15 years with a median of 42.5 months. 61.1% of studies were located in a single country, 38.9% were in several. Europe and North America clearly dominated study localizations. Study objectives were DMARDs (56.9%), followed by diagnostics and disease activity measurement (18.1%), and medication other than DMARD (12.5%), besides others. Studies on DMARDs were mainly sponsored by industry, predominantly interventional studies on established and novel biologics, with several on specific issues like systemic JIA and others. The spectrum of registered studies is currently centered on drug therapy and diagnostics, while other issues in JIA play a subordinated role.

Patient insights on living with idiopathic inflammatory myopathy and the limitations of disease activity measurement methods – a qualitative study

Background The idiopathic inflammatory myopathies (IIMs) are chronic autoimmune conditions, typically resulting in proximal muscle weakness and impacting upon quality of life. Accurate measurement of IIM disease activity is imperative for appropriate medical management and carrying out valid clinical trials. The International Myositis Assessment and Clinical Studies Group (IMACS) “Disease Activity Core Set Measures” are the current gold-standard of IIM disease activity assessment. Anecdotally, patients with an IIM report that the IMACS Core Set Measures and other available methods do not necessarily capture their perceived disease activity. Investigating the patient experiences of living with an IIM and their views on the accuracy of the IMACS Core Set Measures will provide valuable insights for both clinical and research purposes. Methods Eighteen interviews with patients with an IIM were carried out and analysed thematically, using a grounded theory approach. Experiences on living with an IIM and perceptions on the accuracy of disease activity measurement methods were explored. Results Interview analysis revealed four themes: 1) fatigue, 2) pain, 3) day-to-day symptom variation, 4) limitations of creatine kinase levels and manual muscle testing. Conclusions This study has provided valuable insights into patient experiences of living with an IIM. Aspects of IIM disease activity perceived not to be wholly measured by the IMACS Core Set Measures have also been identified. These findings have implications for future IIM clinical care and research, in particular providing justification for research into pain, fatigue and symptom variation.

Evaluation of Mobile Apps Targeted at Patients With Spondyloarthritis for Disease Monitoring: Systematic App Search

Background There are many apps developed for patients with spondyloarthritis in the market, but their purpose and quality are not objectively evaluated. Objective The objective of this study was to identify and evaluate existing publicly available, high-quality apps that use validated measurement instruments for monitoring spondyloarthritis disease activity. Methods We conducted a review of apps available on the Apple App Store and the Google Play Store based on a combination of keywords and inclusion and exclusion criteria. Validated disease activity measurement instruments were identified. Data regarding app characteristics, including the presence of validated disease activity measurement, were extracted. The Mobile App Rating Scale (MARS) was used to review the apps for user experience. Results A total of 1253 apps were identified in the app stores, and 5 apps met the criteria and were further analyzed. Moreover, 2 apps (MySpA and Group for Research and Assessment of Psoriasis and Psoriatic Arthritis App) contained some of the validated disease activity monitoring instruments for specific spondyloarthritis subtypes. These 2 apps were also rated good on the MARS (with total mean scores ≥4 out of 5), whereas the other apps scored poorly in comparison. Conclusions There are 2 high-quality spondyloarthritis disease activity monitoring apps publicly available, but they only target 2 spondyloarthritis subtypes—ankylosing spondylitis and psoriatic arthritis. There is a lack of high-quality apps that can measure disease activity for other spondyloarthritis subtypes, and no app that consolidates all validated disease activity instruments across subtypes was available.

Evaluation of Mobile Apps Targeted at Patients With Spondyloarthritis for Disease Monitoring: Systematic App Search (Preprint)

BACKGROUND There are many apps developed for patients with spondyloarthritis in the market, but their purpose and quality are not objectively evaluated. OBJECTIVE The objective of this study was to identify and evaluate existing publicly available, high-quality apps that use validated measurement instruments for monitoring spondyloarthritis disease activity. METHODS We conducted a review of apps available on the Apple App Store and the Google Play Store based on a combination of keywords and inclusion and exclusion criteria. Validated disease activity measurement instruments were identified. Data regarding app characteristics, including the presence of validated disease activity measurement, were extracted. The Mobile App Rating Scale (MARS) was used to review the apps for user experience. RESULTS A total of 1253 apps were identified in the app stores, and 5 apps met the criteria and were further analyzed. Moreover, 2 apps (MySpA and Group for Research and Assessment of Psoriasis and Psoriatic Arthritis App) contained some of the validated disease activity monitoring instruments for specific spondyloarthritis subtypes. These 2 apps were also rated good on the MARS (with total mean scores ≥4 out of 5), whereas the other apps scored poorly in comparison. CONCLUSIONS There are 2 high-quality spondyloarthritis disease activity monitoring apps publicly available, but they only target 2 spondyloarthritis subtypes—ankylosing spondylitis and psoriatic arthritis. There is a lack of high-quality apps that can measure disease activity for other spondyloarthritis subtypes, and no app that consolidates all validated disease activity instruments across subtypes was available.