Providing multimedia information to children and young people increases recruitment to trials: pre-planned meta-analysis of SWATs.

BACKGROUND: Randomised controlled trials are often beset by problems with poor recruitment and retention. Information to support decisions on trial participation is usually provided as printed participant information sheets (PIS), which are often long, technical, and unappealing. Multimedia information (MMI), including animations and videos, may be a valuable alternative or complement to a PIS. The Trials Engagement in Children and Adolescents (TRECA) study compared MMI to PIS to investigate the effects on participant recruitment, retention, and quality of decision-making. METHODS: We undertook six SWATs (Study Within A Trial) within a series of host trials recruiting children and young people. Potential participants in the host trials were randomly allocated to receive MMI-only, PIS-only, or combined MMI + PIS. We recorded the rates of recruitment and retention (varying between 6 and 26 weeks post-randomisation) in each host trial. Potential participants approached about each host trial were asked to complete a nine-item Decision-Making Questionnaire (DMQ) to indicate their evaluation of the information and their reasons for participation/non-participation. Odds ratios were calculated and combined in a meta-analysis. RESULTS: Data from 3/6 SWATs for which it was possible were combined in a meta-analysis (n = 1758). Potential participants allocated to MMI-only were more likely to be recruited to the host trial than those allocated to PIS-only (OR 1.54; 95% CI 1.05, 2.28; p = 0.03). Those allocated to combined MMI + PIS compared to PIS-only were no more likely to be recruited to the host trial (OR = 0.89; 95% CI 0.53, 1.50; p = 0.67). Providing MMI rather than PIS did not impact on DMQ scores. Once children and young people had been recruited to host trials, their trial retention rates did not differ according to intervention allocation. CONCLUSIONS: Providing MMI-only increased the trial recruitment rate compared to PIS-only but did not affect DMQ scores. Combined MMI + PIS instead of PIS had no effect on recruitment or retention. MMIs are a useful tool for trial recruitment in children and young people, and they could reduce trial recruitment periods.

Towards development of treat to target (T2T) in childhood-onset systemic lupus erythematosus: PReS-endorsed overarching principles and points-to-consider from an international task force.

OBJECTIVES: Application of 'treat-to-target' (T2T) in childhood-onset systemic lupus erythematosus (cSLE) may improve care and health outcomes. This initiative aimed to harmonise existing evidence and expert opinion regarding T2T for cSLE. METHODS: An international T2T Task Force was formed of specialists in paediatric rheumatology, paediatric nephrology, adult rheumatology, patient and parent representatives. A steering committee formulated a set of draft overarching principles and points-to-consider, based on evidence from systematic literature review. Two on-line preconsensus meeting Delphi surveys explored healthcare professionals' views on these provisional overarching principles and points-to-consider. A virtual consensus meeting employed a modified nominal group technique to discuss, modify and vote on each overarching principle/point-to-consider. Agreement of >80% of Task Force members was considered consensus. RESULTS: The Task Force agreed on four overarching principles and fourteen points-to-consider. It was agreed that both treatment targets and therapeutic strategies should be subject to shared decision making with the patient/caregivers, with full remission the preferred target, and low disease activity acceptable where remission cannot be achieved. Important elements of the points-to-consider included: aiming for prevention of flare and organ damage; glucocorticoid sparing; proactively addressing factors that impact health-related quality of life (fatigue, pain, mental health, educational challenges, medication side effects); and aiming for maintenance of the target over the long-term. An extensive research agenda was also formulated. CONCLUSIONS: These international, consensus agreed overarching principles and points-to-consider for T2T in cSLE lay the foundation for future T2T approaches in cSLE, endorsed by the Paediatric Rheumatology European Society.

Pharmacovigilance Reports Received from Children and Young People, and Development of Information to Aid Future Reporting from this Age Group.

OBJECTIVES: This study aimed to determine the contribution of children and young people (CYP) to the Medicines and Healthcare Products Regulatory Agency (MHRA) Yellow Card Scheme (YCS), and to develop age appropriate information with CYP through focus groups to help aid future reporting by this cohort. METHODS: Reports for suspected adverse drug reactions (ADRs) in patients < 19 years of age received by the MHRA YCS from 1 January 2008 to 29 November 2018 were collated and analysed. Consultation activities with CYP from regional, national and international groups were undertaken from November 2017 to July 2018 to develop CYP-appropriate information from current available literature about reporting ADRs to the YCS. RESULTS: CYP contributed 2.3% of YCS reports for patients < 19 years (948 self-reports from a total of 41,630 YCS reports). Patients from 10 years of age contributed YCS reports, and the number of CYP reports represented in the total YCS reports rose by 3% in October 2018 compared with the previous year. Self-reported YCS from the CYP contain different suspected medications and reactions compared with YCS reported on behalf of patients aged < 19 years. The most reported medicines by self-reported YCS from CYP were adolescent vaccinations (such as the human papilloma virus [HPV] vaccine, n = 69), oral contraceptives, acne medication, anti-infectives, and antidepressants. The most commonly reported suspected ADRs submitted by CYP to the YCS were headache (n = 107), nausea and fatigue. CYP-generated reports included alternative suspected ADRs compared with adult reports about ADRs in CYP; these included depression, anxiety and suicidal ideation. The second part of the study used focus groups involving CYP from various backgrounds to develop two information leaflets regarding reporting suspected ADRs in the YCS; this was highlighted in phase I by CYP who identified divergent information needs dependent on age. Phase II-VI updated and improved the relevant information required for both age groups in a succinct and satisfactory manner. Overall, more than 300 CYP contributed to the development of the information. CONCLUSIONS: CYP's contribution to the YCS is limited, but increasing, and demonstrates distinct patterns of suspected medications and reactions. Age-appropriate information for CYP to aid reporting of suspected ADRs has been developed.