ICEECE2012 Symposia Transition from paediatric to adult care - do we have progress? (3 abstracts)
GH therapy in children with PWS was approved in the US in 2000, based on short term growth data, and in Europe in 2006, based on growth and body composition data. GH therapy in PWS has been used by the medical community and advocated by parental support groups since this time. The advent of GH therapy for individuals with PWS represents a unique therapeutic challenge with its intersection of GH treatment for individuals with cognitive disability, varied therapeutic goals that are not focused only on increased height, and potential association with life-threatening adverse events. Given the availability of new results regarding efficacy and safety in infants, children, and adults with PWS from randomised controlled trials as well as from a recent Cochrane review, a CPG Workshop sponsored by the GRS systematically reviewed the literature, graded the available evidence and provided concise recommendations for the use of GH in this context. Forty-three experts (pediatric and adult endocrinologists, clinical and basic geneticists, epidemiologists, a nutrition specialist, an orthopaedic surgeon, a psychiatrist, health technology assessment specialists, a bioethicist, health economist, and a patient advocate) participated. The 4-day workshop followed the CPG development recommendations outlined by the AGREE Collaboration (www.agreetrust.org/). Strength of evidence was graded using the EVIDEM Quality Assessment instrument, using questions organized into 8 domains including study population, intervention and comparators, outcomes measures, design, adverse events ascertainment, time horizon, types of analyses and results validity and relevance (www.evidem.org/praderwilli). For clinical evidence in the pediatric population, randomized controlled trials (RCTs), comparative observational studies and long term studies (>3.5 years) were included. Adult studies included RCTs of GH treatment for ≥6 months and uncontrolled trials, since data was more limited. Safety data was obtained from pharmaceutical registries (Phase 4) and clinical trials (Phase 3). Data on disease, therapeutic context, and economic, ethical and societal aspects were assessed to reflect the international context. This presentation will review the data discussed in the Workshop and present recommendations for GH treatment of individuals with PWS. We will also address a process for obtaining informed consent/assent of patients with PWS and/or their families.