A decade of biologic treatment observation in juvenile idiopathic arthritis: Lessons learned from the Dutch ABC Register

Since 1999, the treatment of juvenile idiopathic arthritis (JIA) has been extended with a new category of drugs: biologic agents (also known as biologicals or biologic disease modifying anti-rheumatic drugs). Biologic agents consist of natural proteins, like antibodies and cytokines, and have been developed to target one or more steps of the immune response. Elucidation of many of the cellular and molecular mechanisms of the immune system involved in inflammatory arthritis has resulted in an increasing development of different biologic agents and, in the future, this number will expand even further. Tumour necrosis factor (TNF) is a proinflammatory cytokine that plays a central role in the pathogenesis of juvenile idiopathic arthritis. In systemic disease, interleukin (IL)-1 (a proinflammatory cytokine synthesised by fibroblasts in the synovium and macrophages) and IL-6 (concentrations of which correlate with fever, disease activity, and platelet counts) are also thought to be important. If inhibition of these cytokines is not sufficient, other drugs that aim at T cell blockade and B cell depletion are available. Only etanercept (TNF-alpha receptor antagonist), adalimumab (anti-TNF-alpha antibody), abatacept (selective T-cell co-stimulation modulator) and tocilizumab (IL-6 receptor antibody) have been approved by the European Medicines Agency and the Food and Drug Administration for the treatment of JIA. Until now, also infliximab (anti-TNF-alpha antibody), anakinra (IL-1 receptor antagonist), canakinumab (anti-IL-1 antibody) and rilonacept (IL-1 receptor antagonist), though not approved, are available options or under investigation for the treatment of JIA. All of the above mentioned agents have been studied in randomized controlled clinical trials with inclusion of JIA patients.But after approval and sometimes off-label use in daily clinical practice, prospective observational studies are crucial to evaluate the longterm effectiveness and safety in a non-selected patient group. Furthermore, additional information with regard to the optimal use and the use in specific subgroups of patients is required. These studies are known as Phase IV post-marketing surveillance studies. Because of the long-term aspect and the requirements of studying a large population, such studies have for practical and financial reasons an observational character, as for example the Dutch national Arthritis and Biologics in Children (ABC) Register

Online focus groups as a tool to collect data in hard-to-include populations: examples from paediatric oncology

Contains fulltext : 81501.pdf (publisher's version ) (Open Access)BACKGROUND: The purpose of this article is to describe and evaluate the methodology of online focus group discussions within the setting of paediatric oncology. METHODS: Qualitative study consisting of separate moderated asynchronous online discussion groups with 7 paediatric cancer patients (aged 8-17), 11 parents, and 18 survivors of childhood cancer (aged 8-17 at diagnosis). RESULTS: All three participant groups could be actively engaged over a one-week period. Respondents highly valued the flexibility and convenience of logging in at their own time and place to join the discussion. Adolescent patients and survivors emphasized that the anonymity experienced made them feel comfortable to express their views in detail. The findings indicate a strong preference for online group discussions across all participant groups. CONCLUSION: The findings show that online focus group methodology is a feasible tool for collecting qualitative data within the setting of paediatric oncology, and may offer new opportunities to collect data in other hard-to-include populations. The evaluations seem to indicate that the online group discussions have given participants an opportunity to articulate their experiences and views in a way they might not have done in a traditional group discussion.9 p

Efficacy of smoking prevention program 'Smoke-free Kids': study protocol of a randomized controlled trial

Contains fulltext : 77005.pdf (publisher's version ) (Open Access)Background - A strong increase in smoking is noted especially among adolescents. In the Netherlands, about 5% of all 10-year olds, 25% of all 13-year olds and 62% of all 17-year olds report ever smoking. In the U.S., an intervention program called 'Smoke-free Kids' was developed to prevent children from smoking. The present study aims to assess the effects of this home-based smoking prevention program in the Netherlands. Methods - A randomized controlled trial is conducted among 9 to 11-year old children of primary schools. Participants are randomly assigned to the intervention and control conditions. The intervention program consists of five printed activity modules designed to improve parenting skills specific to smoking prevention and parent-child communication regarding smoking. These modules will include additional sheets with communication tips. The modules for the control condition will include solely information on smoking and tobacco use. Initiation of cigarette smoking (first instance of puffing on a lighted cigarette), susceptibility to cigarette smoking, smoking-related cognitions, and anti-smoking socialization will be the outcome measures. To collect the data, telephone interviews with mothers as well as with their child will be conducted at baseline. Only the children will be examined at post-intervention follow-ups (6, 12, 24, and 36 months after the baseline). Discussion - This study protocol describes the design of a randomized controlled trial that will evaluate the effectiveness of a home-based smoking prevention program. We expect that a significantly lower number of children will start smoking in the intervention condition compared to control condition as a direct result of this intervention. If the program is effective, it is applicable in daily live, which will facilitate implementation of the prevention protocol. Trial registration: Netherlands Trial Register NTR146510 p

Smoking onset and the time-varying effects of self-efficacy, environmental smoking, and smoking-specific parenting by using discrete-time survival analysis

This study examined the timing of smoking onset during mid- or late adolescence and the time-varying effects of refusal self-efficacy, parental and sibling smoking behavior, smoking behavior of friends and best friend, and parental smoking-specific communication. We used data from five annual waves of the ‘Family and Health’ project. In total, 428 adolescents and their parents participated at baseline. Only never smokers were included at baseline (n = 272). A life table and Kaplan–Meier survival curve showed that 51% of all adolescents who did not smoke at baseline did not start smoking within 4 years. The risk for smoking onset during mid- or late adolescence is rather stable (hazard ratio between 16 and 19). Discrete-time survival analyses revealed that low refusal self-efficacy, high frequency of communication, and sibling smoking were associated with smoking onset one year later. No interaction effects were found. Conclusively, the findings revealed that refusal self-efficacy is an important predictor of smoking onset during mid- or late adolescence and is independent of smoking-specific communication and smoking behavior of parents, siblings, and (best) friend(s). Findings emphasize the importance of family prevention programs focusing on self-efficacy skills