AB0937 IDENTIFYING THE PRIMARY OUTCOME MEASURE AND PROTOCOL COMPONENTS FOR A PROSPECTIVE FEASIBILITY STUDY OF CORTICOSTEROID REGIMENS FOR CHILDREN AND YOUNG PEOPLE WITH JUVENILE IDIOPATHIC ARTHRITIS USING CONSENSUS METHODS WITH YOUNG PEOPLE, FAMILIES AND PROFESSIONALS

Abstract Background Juvenile idiopathic arthritis (JIA) is an umbrella term for seven relapsing-remitting inflammatory conditions in children and young people (CYP). Early, intensive treatment can prevent long-term damage; however, established drugs exhibit a delayed response, prompting the need for rapid-onset treatment in the form of corticosteroids. Given a lack of consensus as to which corticosteroid induction regimen should be used for CYP with JIA, a feasibility trial of different regimens is needed. The aim was to achieve consensus among CYP, families, and healthcare professionals (HCPs) about the primary outcome measures and protocol components to include in a prospective feasibility study. Methods A modified Nominal Group Technique was used to achieve consensus on the most appropriate primary outcome measure to be included in a prospective feasibility study, in addition to other protocol components such as inclusion/exclusion criteria. Fifteen participants participated in the process, including a combination of CYP with JIA, families (n = 9) and HCPs (n = 6). Results In the first vote, participants agreed that Juvenile Arthritis Disease Activity Score (JADAS) and Physician Global Assessment Score were most meaningful. During sub-group discussions, the need for a composite score which captured the voice of CYP and families was emphasised. In the second vote, JADAS and the JIA Core Set were identified as the most important. Further discussions led to the results of the third vote, agreeing JADAS as the primary outcome measure of choice being measured at 6 weeks after commencement of treatment. The majority of HCPs, CYP and families voted for all JIA sub-types to be included in a prospective feasibility study, with some queries about the inclusion of systemic JIA given its unique presentation. Participants also identified the need for more frequent data collection time points to capture the rapid onset of corticosteroid action, while CYP and families opted for accessible mechanisms for participation, such as digital follow-up strategies. Conclusion It is feasible to include CYP, families and HCPs in synthesising complex concepts to agree by consensus the design components of clinical research. The primary outcome measure for inclusion in a prospective feasibility study of corticosteroid regimens in CYP with JIA was co-prioritised, with CYP and families taking a leading role in the ultimate selection of an appropriate outcome measure and other study protocol components. Using consensus methods with CYP, families and HCPs is a systematic and rigorous way in which to select outcome measures that are both meaningful and relevant to everyone involved in the care and treatment of CYP with JIA. Conflicts of Interest The authors declare no conflicts of interest.

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Telephone-guided self-help for mental health difficulties in neurological conditions: a randomised pilot trial

Archives of Disease in Childhood ◽

10.1136/archdischild-2019-318577 ◽

2021 ◽

pp. archdischild-2019-318577

Author(s):

Sophie D Bennett ◽

Isobel Heyman ◽

Anna E Coughtrey ◽

Sophia Varadkar ◽

Terence Stephenson ◽

...

Keyword(s):

Mental Health ◽

Young People ◽

Primary Outcome ◽

Outcome Measure ◽

Mental Health Problems ◽

Pilot Trial ◽

Primary Outcome Measure ◽

Self Help ◽

Mental Health Difficulties ◽

Neurological Conditions

ObjectiveThis study aimed to conduct a randomised pilot trial to assess the feasibility of a randomised controlled trial (RCT) to investigate the effect of telephone-guided self-help for the treatment of mental health difficulties in children with neurological conditions.DesignPreliminary RCT. The primary outcome measure was the Strengths and Difficulties Questionnaire.SettingNeurology clinics in a national tertiary paediatric hospital.PatientsYoung people attending neurology clinics who met criteria for mental health difficulties according to the Development and Wellbeing Assessment.Interventions12 weeks of telephone-guided self-help based on a modular approach to psychological therapy for children delivered to children and/or their parents (n=17; eight males; mean age 12.04 years, SD=3.34) or a waiting list for telephone-guided self-help with no additional intervention over 12 weeks (n=17; nine males; mean age 10.53 years, SD=3.14).Results124 participants completed the DAWBA, and 34 children and young people were entered into the trial. 65% of those randomised to the intervention arm completed the full intervention, and the intervention was acceptable to those completing it. However, there were significant problems related to lack of data completion (38% data loss for primary outcome measure), choice of control comparator and outcome measures. Due to significant loss of data at follow-up, the effect size findings are considered unreliable.ConclusionsFurther feasibility work should be conducted to improve data completeness before progression to a definitive trial of guided self-help for mental health problems in children with neurological conditions can be recommended.Trial registration numberISRCTN21184717.

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Therapist- and parent-guided Internet-delivered behaviour therapy for paediatric Tourette’s Disorder: a pilot randomised controlled trial with long-term follow-up

10.31234/osf.io/dp3qz ◽

2018 ◽

Cited By ~ 1

Author(s):

Per Andrén ◽

Kristina Aspvall ◽

Lorena Fernández de la Cruz ◽

Paulina Wiktor ◽

Sofia Romano ◽

...

Keyword(s):

Young People ◽

Primary Outcome ◽

Outcome Measure ◽

Primary Outcome Measure ◽

Behaviour Therapy ◽

Tourette’S Disorder ◽

Tourette's Disorder ◽

Tic Severity ◽

Randomised Controlled

Objective: Behaviour therapy (BT) for Tourette’s Disorder (TD) and Persistent Motor or Vocal Tic Disorder (PTD) is rarely available. We evaluated the feasibility of adapting two existing BT protocols for TD/PTD (habit reversal training [HRT] and exposure and response prevention [ERP]) into a therapist- and parent-guided online self-help format.Design: A pilot, single-blind, parallel group randomised controlled trial.Setting: A university hospital in Sweden.Participants: Twenty-three young people with TD/PTD, aged 8-16.Interventions: Two 10-week therapist- and parent-guided Internet-delivered programmes (called BIP TIC HRT and BIP TIC ERP).Outcome: The primary outcome measure was the Yale Global Tic Severity Scale (YGTSS). Blinded evaluators rated symptoms at baseline, post-treatment, and 3-month follow-up (primary endpoint). All participants were naturalistically followed-up to 12 months after treatment.Results: Patients and parents rated the interventions as highly acceptable, credible, and satisfactory. While both interventions resulted in reduced tic-related impairment, parent-rated tic severity, and improved quality of life, only BIP TIC ERP resulted in a significant improvement on the primary outcome measure. Within-group effect sizes and responder rates were, respectively: d=1.12 and 75% for BIP TIC ERP, and d=0.50 and 55% for BIP TIC HRT. The therapeutic gains were maintained up to 12 months after the end of the treatment. Adverse events were rare in both groups. The average therapist support time was around 25 minutes per participant per week.Conclusions: Internet-delivered BT has the potential to greatly increase access to evidence-based treatment for young people with TD/PTD. Further evaluation of the efficacy and cost-effectiveness of this treatment modality is warranted.Trial registration number: NCT02864589 (ClinicalTrials.gov).Funding: The trial was supported by the Stockholm County Council (PPG project 2016-0108).

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Therapist-guided and parent-guided internet-delivered behaviour therapy for paediatric Tourette’s disorder: a pilot randomised controlled trial with long-term follow-up

BMJ Open ◽

10.1136/bmjopen-2018-024685 ◽

2019 ◽

Vol 9 (2) ◽

pp. e024685 ◽

Cited By ~ 17

Author(s):

Per Andrén ◽

Kristina Aspvall ◽

Lorena Fernández de la Cruz ◽

Paulina Wiktor ◽

Sofia Romano ◽

...

Keyword(s):

Randomised Controlled Trial ◽

Young People ◽

Primary Outcome ◽

Outcome Measure ◽

Controlled Trial ◽

Primary Outcome Measure ◽

Behaviour Therapy ◽

Tic Severity ◽

Randomised Controlled

ObjectiveBehaviour therapy (BT) for Tourette’s disorder (TD) and persistent (chronic) motor or vocal tic disorder (PTD) is rarely available. We evaluated the feasibility of adapting two existing BT protocols for TD/PTD (habit reversal training (HRT) and exposure and response prevention (ERP)) into a therapist-guided and parent-guided online self-help format.DesignA pilot, single-blind, parallel group randomised controlled trial.SettingA specialist outpatient clinic in Sweden.ParticipantsTwenty-three young people with TD/PTD, aged 8–16.InterventionsTwo 10-week therapist-guided and parent-guided internet-delivered programmes (called BIP TIC HRT and BIP TIC ERP).OutcomeThe primary outcome measure was the Yale Global Tic Severity Scale. Blinded evaluators rated symptoms at baseline, post-treatment and 3-month follow-up (primary endpoint). All participants were naturalistically followed up to 12 months after treatment.ResultsPatients and parents rated the interventions as highly acceptable, credible and satisfactory. While both interventions resulted in reduced tic-related impairment, parent-rated tic severity and improved quality of life, only BIP TIC ERP resulted in a significant improvement on the primary outcome measure. Within-group effect sizes and responder rates were, respectively: d=1.12 and 75% for BIP TIC ERP, and d=0.50 and 55% for BIP TIC HRT. The therapeutic gains were maintained up to 12 months after the end of the treatment. Adverse events were rare in both groups. The average therapist support time was around 25 min per participant per week.ConclusionsInternet-delivered BT has the potential to greatly increase access to evidence-based treatment for young people with TD/PTD. Further evaluation of the efficacy and cost-effectiveness of this treatment modality is warranted.Trial registration numberNCT02864589; Pre-results.

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Supporting self-management amongst young people with acne vulgaris: feasibility randomised trial of a web-based behavioural intervention (Preprint)

10.2196/preprints.25918 ◽

2020 ◽

Author(s):

Athena Ip ◽

Ingrid Muller ◽

Adam Geraghty ◽

Kate Rumsby ◽

Beth Stuart ◽

...

Keyword(s):

Young People ◽

Usual Care ◽

Primary Outcome ◽

Outcome Measure ◽

Randomised Trial ◽

Intervention Group ◽

Primary Outcome Measure ◽

Self Management ◽

Web Based ◽

Topical Treatments

BACKGROUND Acne is a common skin condition that is most prevalent in young people. It can have substantial impact on quality of life, which can be minimised with appropriate use of topical treatments. Nonadherence to topical treatments for acne is common often leading to treatment failure. OBJECTIVE The objective of this study was to develop a web-based behavioural intervention to support self-management of acne and to assess the feasibility of recruitment, retention and engagement in a parallel unblinded randomised trial of the intervention compared with usual care alone. METHODS The intervention was developed iteratively using LifeGuide software and following the Person-Based Approach (PBA) to intervention development. The target behaviour was ‘Appropriate use of topical treatments’ and barriers and facilitators identified from the qualitative research and evidence from the wider literature were used to identify techniques to improve and promote their use. Young people with acne aged 14-25 years who had received treatment for acne within the last 6 months were invited to participate through mail-out from primary care practices in the South of England. Participants were randomised to: (1) usual care, or (2) usual care plus access to web-based intervention. Usage data was collected and a series of questionnaires including the primary outcome measure for skin specific quality of life (Skindex-16) were collected at baseline, 4-weeks and 6-weeks follow-up. RESULTS A total of 1193 participants were invited and 53 young people with acne were randomised to usual care (n=27) or usual care plus intervention (n=26). The response rate for the primary outcome measure (Skindex-16) was 87% at 4-weeks, 6-weeks or both time points. The estimate of mean scores between groups (with 95% confidence) using linear regression showed a trend in the direction of benefit for the web-based intervention group in the primary outcome measure (Skindex-16), and secondary measures (Patient Health Questionnaire 4 and the Problematic Experiences of Therapy Scale). Intervention usage data showed a high uptake of the core module in the usual care plus web-based intervention group with 23 of 26 (88%) completing the module. Uptake of the optional modules were low with less than half visiting each (Myth-busting quiz: 27%; Living with spots or acne: 42%; Oral antibiotics: 19%; What are spots or acne: 27%; Other treatments: 27%; Talking to your GP: 12%). CONCLUSIONS This study demonstrated the feasibility of delivering a trial of a web-based intervention to support self-management for young people with acne. Additional work is needed prior to a full definitive trial including enhancing engagement with the intervention, recruitment and follow-up rates. CLINICALTRIAL International Standard Randomized Controlled Trial Number (ISRCTN): 78626638; https://doi.org/10.1186/ISRCTN78626638.

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Timed walk as primary outcome measure of treatment response in clinical trials for HTLV-1-associated myelopathy: a feasibility study

Pilot and Feasibility Studies ◽

10.1186/s40814-015-0031-1 ◽

2015 ◽

Vol 1 (1) ◽

Cited By ~ 2

Author(s):

Fabiola Martin ◽

Eisuke Inoue ◽

Irene C. M. Cortese ◽

Ramon de Almeida Kruschewsky ◽

Adine Adonis ◽

...

Keyword(s):

Clinical Trials ◽

Treatment Response ◽

Feasibility Study ◽

Primary Outcome ◽

Outcome Measure ◽

Primary Outcome Measure

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P121 The efficacy of treatment interventions on fatigue in adolescents with juvenile idiopathic arthritis: a literature review

Rheumatology ◽

10.1093/rheumatology/keab247.117 ◽

2021 ◽

Vol 60 (Supplement_1) ◽

Author(s):

Sebastian Moshtael ◽

Sonia Khanom ◽

Janet E McDonagh

Keyword(s):

Quality Of Life ◽

Juvenile Idiopathic Arthritis ◽

Young People ◽

Rating Scale ◽

Outcome Measure ◽

Therapeutic Interventions ◽

Current Evidence ◽

Original Research ◽

Qualitative Synthesis

Abstract Background/Aims Fatigue has been reported by young people and professionals alike as a major challenge for people living with juvenile idiopathic arthritis (JIA) . It remains unclear as to how therapeutic interventions impacts on this major symptom. The aim of this review was to determine the current evidence for the effectiveness of therapeutic interventions, non- pharmacological and pharmacological, on improving fatigue in adolescents with JIA. Methods Three electronic databases (MEDLINE, EMBASE, PsycINFO) were searched from 2000 to Feb 2020; in addition to manual searches. Articles were eligible for inclusion if they (i) were original research papers, (ii) had fatigue as a primary outcome measure (iii) included adolescents (10-25 years) and (iv) were available in the English language. Results Of the 3,142 records identified, 31 underwent full text assessment and 4 studies were included in the qualitative synthesis including 2 from the Netherlands, 1 Germany, and 1 from Canada. The total number of participants across the 4 studies was 824. Three were randomised controlled trials of nonpharmacological interventions and one was a cohort study from a national drug registry including older adolescents JIA (mean: 19-years). Measurement tools included the Paediatric Quality of life multidimensional fatigue scale, (2 studies), the Checklist for Individual Strength CIS-20 (1 study), a numeric rating scale (1 study). In one study a visual analogue scale for energy level was also utilised. In 2 studies baseline prevalence of fatigue was reported as 60% and 76% respectively. Significant reduction in fatigue was observed in all three nonpharmacological studies. In the registry study, fatigue was noted to be prevalent in spite of the disease being in inactive or minimally active on biologic therapy. In 3 studies the relationship between health-related quality of life and fatigue was highlighted and showed a decreased quality of life in fatigued young people. Conclusion Fatigue is a significant problem in JIA during adolescence and influences quality of life. Improvements in fatigue with non-pharmacological interventions have been reported. However, at present, data is insufficient to conclusively decide which treatment intervention is most efficacious in treating fatigue in young people with JIA. Fatigue should be considered as an important outcome measure for the management of JIA in future evaluations of interventions. Disclosure S. Moshtael: None. S. Khanom: None. J.E. McDonagh: None.

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Clinical impact of left and right axis deviations with narrow QRS complex on 3-year outcomes in a hospital-based population in Japan

Scientific Reports ◽

10.1038/s41598-021-88259-8 ◽

2021 ◽

Vol 11 (1) ◽

Author(s):

Yuta Seko ◽

Takao Kato ◽

Yuhei Yamaji ◽

Yoshisumi Haruna ◽

Eisaku Nakane ◽

...

Keyword(s):

Cardiovascular Events ◽

Primary Outcome ◽

Outcome Measure ◽

Conduction Block ◽

Primary Outcome Measure ◽

Excess Risk ◽

Major Adverse Cardiovascular Events ◽

Prognostic Impact ◽

Axis Deviation ◽

The Right

AbstractWhile the prognostic impact of QRS axis deviation has been assessed, it has never been investigated in patients without conduction block. Thus, we evaluated the prognostic impact of QRS-axis deviation in patients without conduction block. We retrospectively analyzed 3353 patients who had undergone both scheduled transthoracic echocardiography and electrocardiography in 2013 in a hospital-based population, after excluding patients with a QRS duration of ≥ 110 ms, pacemaker placement, and an QRS-axis − 90° to − 180° (northwest axis). The study population was categorized into three groups depending on the mean frontal plane QRS axis as follows: patients with left axis deviation (N = 171), those with right axis deviation (N = 94), and those with normal axis (N = 3088). The primary outcome was a composite of all-cause death and major adverse cardiovascular events. The cumulative 3-year incidence of the primary outcome measure was significantly higher in the left axis deviation group (26.4% in the left axis deviation, 22.7% in the right axis deviation, and 18.4% in the normal axis groups, log-rank P = 0.004). After adjusting for confounders, the excess risk of primary outcome measure remained significant in the left axis deviation group (hazard ratio [HR] 1.44; 95% confidence interval [CI] 1.07–1.95; P = 0.02), while the excess risk of primary outcome measure was not significant in the right axis deviation group (HR 1.22; 95% CI 0.76–1.96; P = 0.41). Left axis deviation was associated with a higher risk of a composite of all-cause death and major adverse cardiovascular events in hospital-based patients without conduction block in Japan.

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Computerised cognitive–behavioural therapy for adults with intellectual disability: randomised controlled trial

The British Journal of Psychiatry ◽

10.1192/bjp.bp.117.198630 ◽

2017 ◽

Vol 211 (2) ◽

pp. 95-102 ◽

Cited By ~ 13

Author(s):

Patricia Cooney ◽

Catherine Jackman ◽

David Coyle ◽

Gary O'Reilly

Keyword(s):

Intellectual Disability ◽

Randomised Controlled Trial ◽

Primary Outcome ◽

Outcome Measure ◽

Controlled Trial ◽

Behavioural Therapy ◽

Primary Outcome Measure ◽

Cognitive Behavioural ◽

Randomised Controlled

BackgroundDespite the evidence base for computer-assisted cognitive–behavioural therapy (CBT) in the general population, it has not yet been adapted for use with adults who have an intellectual disability.AimsTo evaluate the utility of a CBT computer game for adults who have an intellectual disability.MethodA 2 × 3 (group × time) randomised controlled trial design was used. Fifty-two adults with mild to moderate intellectual disability and anxiety or depression were randomly allocated to two groups: computerised CBT (cCBT) or psychiatric treatment as usual (TAU), and assessed at pre-treatment, post-treatment and 3-month follow-up. Forty-nine participants were included in the final analysis.ResultsA significant group x time interaction was observed on the primary outcome measure of anxiety (Glasgow Anxiety Scale for people with an Intellectual Disability), favouring cCBT over TAU, but not on the primary outcome measure of depression (Glasgow Depression Scale for people with a Learning Disability). A medium effect size for anxiety symptoms was observed at post-treatment and a large effect size was observed after follow-up. Reliability of Change Indices indicated that the intervention produced clinically significant change in the cCBT group in comparison with TAU.ConclusionsAs the first application of cCBT for adults with intellectual disability, this intervention appears to be a useful treatment option to reduce anxiety symptoms in this population.

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