Abstract:IntroductionDirect-to-family clinical trials efficiently provide data while reducing the participation burden for children and their families. Although these trials can offer significant advantages over traditional clinical trials, the process of designing and implementing direct-to-family studies is poorly defined, especially in children with rheumatic disease. This paper provides lessons learnt from the design and implementation of a self-controlled, direct-to-family pilot trial aimed to evaluate the effects… Show more
“…None of these studies have published results to date, though one investigation has published their study protocol. 21 Figure 1.Flow of literature search according to PRISMA guidelines.…”
Section: Resultsmentioning
confidence: 99%
“…None of these studies have published results to date, though one investigation has published their study protocol. 21…”
Background Childhood-onset systemic lupus erythematosus (cSLE) is a complex multisystem autoimmune disease often associated with pain, fatigue, and mood-related disturbances. cSLE is associated with increased disease severity and higher rates of mortality as compared to adult onset SLE. Therefore, a multi-faceted approach to care, including the use of non-pharmacologic therapies, is essential to ensure optimal patient outcomes. The use of non-pharmacologic therapies as adjunctive treatments has been shown to be beneficial in adults with SLE, yet, their use and effect is less well understood in cSLE. This is the first systematic review to explore the use and quality of evidence of non-pharmacologic approaches to treat cSLE. Methods A literature review was performed using PRISMA guidelines. Studies until March 2021 with participants diagnosed with cSLE were included. The quality of the evidence was graded via OCEBM levels of evidence guidelines and bias assessed using Cochrane guidelines. Completed clinical trials (via clinicaltrials.gov ) were also searched to identify unpublished results. Results Eleven published studies consisting of 1152 patients met inclusion criteria for this review, as well as three additional studies with unpublished data on clinicaltrial.gov . Of the published trials, four studies used patient education/support, three studies used dietary supplementation, three used forms of psychotherapy (e.g., Cognitive behavioral therapy), and 1 used aerobic exercise to target the following issues: treatment adherence ( n = 3), quality of life ( n = 3), fatigue ( n = 2), pain ( n = 2), depressive symptoms ( n = 1), anxiety ( n = 1), and health-related outcomes including disease severity ( n = 3), cardiovascular disease risk (Cardiovascular disease; n = 3), and muscle function ( n = 1). Across investigations, the quality of the evidence based on study design was moderate/low. In terms of potential outcomes, dietary supplementation methods were successful in 2 of 3 studies and were associated with improvements in disease activity and fatigue. Aerobic exercise was effective in decreasing resting heart rate and increasing cardiorespiratory capacity. Patient education/support was related to significantly increased treatment adherence and decreased cardiovascular risk markers. Two of the three studies examining the impact of psychotherapy showed improvements (e.g., in treatment adherence, depression and fatigue). Conclusion This review identifies several promising non-pharmacologic therapies to use as adjunctive treatments to traditional pharmacologic regimens in health and mental health-related outcomes in patients with cSLE. Future well controlled clinical trials would be beneficial to more rigorously evaluate the effects of non-pharmacologic therapies in pediatric populations.
“…None of these studies have published results to date, though one investigation has published their study protocol. 21 Figure 1.Flow of literature search according to PRISMA guidelines.…”
Section: Resultsmentioning
confidence: 99%
“…None of these studies have published results to date, though one investigation has published their study protocol. 21…”
Background Childhood-onset systemic lupus erythematosus (cSLE) is a complex multisystem autoimmune disease often associated with pain, fatigue, and mood-related disturbances. cSLE is associated with increased disease severity and higher rates of mortality as compared to adult onset SLE. Therefore, a multi-faceted approach to care, including the use of non-pharmacologic therapies, is essential to ensure optimal patient outcomes. The use of non-pharmacologic therapies as adjunctive treatments has been shown to be beneficial in adults with SLE, yet, their use and effect is less well understood in cSLE. This is the first systematic review to explore the use and quality of evidence of non-pharmacologic approaches to treat cSLE. Methods A literature review was performed using PRISMA guidelines. Studies until March 2021 with participants diagnosed with cSLE were included. The quality of the evidence was graded via OCEBM levels of evidence guidelines and bias assessed using Cochrane guidelines. Completed clinical trials (via clinicaltrials.gov ) were also searched to identify unpublished results. Results Eleven published studies consisting of 1152 patients met inclusion criteria for this review, as well as three additional studies with unpublished data on clinicaltrial.gov . Of the published trials, four studies used patient education/support, three studies used dietary supplementation, three used forms of psychotherapy (e.g., Cognitive behavioral therapy), and 1 used aerobic exercise to target the following issues: treatment adherence ( n = 3), quality of life ( n = 3), fatigue ( n = 2), pain ( n = 2), depressive symptoms ( n = 1), anxiety ( n = 1), and health-related outcomes including disease severity ( n = 3), cardiovascular disease risk (Cardiovascular disease; n = 3), and muscle function ( n = 1). Across investigations, the quality of the evidence based on study design was moderate/low. In terms of potential outcomes, dietary supplementation methods were successful in 2 of 3 studies and were associated with improvements in disease activity and fatigue. Aerobic exercise was effective in decreasing resting heart rate and increasing cardiorespiratory capacity. Patient education/support was related to significantly increased treatment adherence and decreased cardiovascular risk markers. Two of the three studies examining the impact of psychotherapy showed improvements (e.g., in treatment adherence, depression and fatigue). Conclusion This review identifies several promising non-pharmacologic therapies to use as adjunctive treatments to traditional pharmacologic regimens in health and mental health-related outcomes in patients with cSLE. Future well controlled clinical trials would be beneficial to more rigorously evaluate the effects of non-pharmacologic therapies in pediatric populations.
“…During the pandemic, clinical trials have faced unprecedented logistical barriers including social distancing protocols, restructuring of clinical sites to accommodate inpatient surges, participants’ fear of potential exposure during study visits, reduction of in-person research staff, and policies deeming study visits non-essential, necessitating adoption of remote methods to sustain research [ 5 , 18 , 26 – 30 ]. Rather than being constrained by these limitations, researchers have capitalized on the need to transform the landscape toward a more equitable and efficient future through implementation of remote study models [ 14 , 17 , 18 , 21 , 31 , 32 ]. However, to date, there is minimal experience in defining best practices in this domain.…”
Longitudinal clinical studies traditionally require in-person study visits which are well documented to pose barriers to participation and contribute challenges to enrolling representative samples. Remote trial models may reduce barriers to research engagement, improve retention, and reach a more representative cohort. As remote trials become more common following the COVID-19 pandemic, a critical evaluation of this approach is imperative to optimize this paradigm shift in research. The TestBoston study was launched to understand prevalence and risk factors for COVID-19 infection in the greater Boston area through a fully remote home-testing model. Participants (adults, within 45 miles of Boston, MA) were recruited remotely from patient registries at Brigham and Women’s Hospital and the general public. Participants were provided with monthly and “on-demand” at-home SARS-CoV-2 RT-PCR and antibody testing using nasal swab and dried blood spot self-collection kits and electronic surveys to assess symptoms and risk factors for COVID-19 via an online dashboard. Between October 2020 and January 2021, we enrolled 10,289 participants reflective of Massachusetts census data. Mean age was 47 years (range 18–93), 5855 (56.9%) were assigned female sex at birth, 7181(69.8%) reported being White non-Hispanic, 952 (9.3%) Hispanic/Latinx, 925 (9.0%) Black, 889 (8.6%) Asian, and 342 (3.3%) other and/or more than one race. Lower initial enrollment among Black and Hispanic/Latinx individuals required an adaptive approach to recruitment, leveraging connections to the medical system, coupled with community partnerships to ensure a representative cohort. Longitudinal retention was higher among participants who were White non-Hispanic, older, working remotely, and with lower socioeconomic vulnerability. Implementation highlighted key differences in remote trial models as participants independently navigate study milestones, requiring a dedicated participant support team and robust technology platforms, to reduce barriers to enrollment, promote retention, and ensure scientific rigor and data quality. Remote clinical trial models offer tremendous potential to engage representative cohorts, scale biomedical research, and promote accessibility by reducing barriers common in traditional trial design. Barriers and burdens within remote trials may be experienced disproportionately across demographic groups. To maximize engagement and retention, researchers should prioritize intensive participant support, investment in technologic infrastructure and an adaptive approach to maximize engagement and retention.
“…Health research is especially well poised to benefit from the possibilities these technologies offer. In the last decade, there has been rapid growth in randomised controlled trials (RCTs) and clinical trials using remote and digital methodologies [1][2][3]. The COVID-19 pandemic has spotlighted these remote methods by emphasizing how useful and innovative they can be.…”
Section: Introductionmentioning
confidence: 99%
“…They often use alternative data collection locations, such as mobile or local health clinics, employ digital technologies, for example, wearable devices, or leverage online methods. Decentralised trials can potentially reach people in geographically remote areas, recruit larger and more diverse populations, improve trial participant retention, and may decrease trial costs and research waste [2,4,5].…”
Introduction: This scoping review will determine how online, randomised trials with children are conducted. The objectives of the review are: (a) to determine what methods and tools have been used to create and conduct online trials with children and (b) to identify the gaps in the knowledge in this field.
Over the last decade, randomised trials employing online methods have gained momentum. Decentralised methods lend themselves to certain types of trials and can offer advantages over traditional trial methods, potentially increasing participant reach and diversity and decreasing research waste. However, decentralised trials that have all aspects of the trial exclusively online are not yet common, and those involving children even less so. This scoping review will describe and evaluate the methods used in these trials to understand how they may be effectively employed.
Methods: Methods are informed by guidance from the Joanna Briggs Institute and the Preferred Reporting Items for Systematic Reviews and Meta-analyses extension for scoping reviews. The search strategy was developed in consultation with an information specialist for the following databases: MEDLINE, CENTRAL, CINAHL, and Embase. Grey literature searches will be completed with the consultation of experts in decentralised trials and digital health using internet searches and suitable trial registries. Once identified, included full-text studies’ references will be manually searched for any trials that may have been missed. We will include randomised and quasi-randomised trials conducted exclusively online with participants under the age of 18 published in English. We will not limit by country of conduct or date of publication. Data will be collected using a data charting tool and presented in text, graphical, and tabular formats.
Ethics and Dissemination: Ethical approval is not needed since all data sources used are publicly available. The review will be available as a preprint before publication in an open-access, peer-reviewed journal.
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