Longitudinal Health Status and Quality of Life in Congenital Diaphragmatic Hernia

OBJECTIVES: To longitudinally evaluate self-reported health status (HS) and quality of life (QoL) a in 8- and 12-year-old survivors of congenital diaphragmatic hernia (CDH). We hypothesized that HS would improve with age—as associated health problems tend to decline—whereas QoL would decrease, as the children start to compare themselves with peers. METHODS: Self-reported HS and QoL of 133 children born between 1999 and 2013 who had joined our standardized follow-up program were routinely assessed at the ages of 8 and 12 with generic, internationally validated, standardized instruments. Longitudinal evaluation of total and subscale scores was performed using general linear model analyses. In addition, we compared these scores to sex- and age-specific normative data. RESULTS: Between ages 8 and 12, boys born with CDH perceived a decline in HS (mean difference 7.15, P &lt; .001). Self-reported QoL did not change over time in both boys and girls. At both ages, HS was significantly lower than that of healthy peers (effect size 5 0.71, P 5 .001 for boys, and effect size 5 0.69, P 5 .003 for girls), whereas differences in QoL were small. CONCLUSIONS: Children born with CDH are at risk for declining HS between 8 and 12 years, but not QoL, compared with healthy peers. Given that children born with CDH tend to grow into deficits, our findings highlight the need for continued somatic and psychological assessments in adolescent and adult CDH survivors.</p

Development of a core outcome set for congenital pulmonary airway malformations: study protocol of an international Delphi survey

Introduction A worldwide lack of consensus exists on the optimal management of asymptomatic congenital pulmonary airway malformation (CPAM) even though the incidence is increasing. Either a surgical resection is performed or a wait-and-see policy is employed, depending on the treating physician. Management is largely based on expert opinion and scientific evidence is scarce. Wide variations in outcome measures are seen between studies making comparison difficult thus highlighting the lack of universal consensus in outcome measures as well. We aim to define a core outcome set which will include the most important core outcome parameters for paediatric patients with an asymptomatic CPAM.Methods and analysis This study will include a critical appraisal of the current literature followed by a three-stage Delphi process with two stakeholder groups. One surgical group including paediatric as well as thoracic surgeons, and a non-surgeon group including paediatric pulmonologists, intensive care and neonatal specialists. All participants will score outcome parameters according to their level of importance and the most important parameters will be determined by consensus.Ethics and dissemination Electronic informed consent will be obtained from all participants. Ethical approval is not required. After the core outcome set has been defined, we intend to design an international randomised controlled trial: the COllaborative Neonatal NEtwork for the first CPAM Trial, which will be aimed at determining the optimal management of patients with asymptomatic CPAM

Long-term effectiveness of dopamine agonists and monoamine oxidase B inhibitors compared with levodopa as initial treatment for Parkinson\u27s disease (PD MED): A large, open-label, pragmatic randomised trial

\ua9 2014 Elsevier Ltd. Background Whether initial treatment for Parkinson\u27s disease should consist of levodopa, dopamine agonists, or monoamine oxidase type B inhibitors (MAOBI) is uncertain. We aimed to establish which of these three classes of drug, as initial treatment, provides the most effective long-term control of symptoms and best quality of life for people with early Parkinson\u27s disease. Methods In this pragmatic, open-label randomised trial, patients newly diagnosed with Parkinson\u27s disease were randomly assigned (by telephone call to a central office; 1:1:1) between levodopa-sparing therapy (dopamine agonists or MAOBI) and levodopa alone. Patients and investigators were not masked to group assignment. Primary outcomes were the mobility dimension on the 39-item patient-rated Parkinson\u27s disease questionnaire (PDQ-39) quality-of-life scale (range 0-100 with six points defined as the minimally important difference) and cost-effectiveness. Analysis was intention to treat. This trial is registered, number ISRCTN69812316. Findings Between Nov 9, 2000, and Dec 22, 2009, 1620 patients were assigned to study groups (528 to levodopa, 632 to dopamine agonist, 460 to MAOBI). With 3-year median follow-up, PDQ-39 mobility scores averaged 1\ub78 points (95% CI 0\ub75-3\ub70, p=0\ub7005) better in patients randomly assigned to levodopa than those assigned to levodopa-sparing therapy, with no increase or attrition of benefit during 7 years\u27 observation. PDQ-39 mobility scores were 1\ub74 points (95% CI 0\ub70-2\ub79, p=0\ub705) better in patients allocated MAOBI than in those allocated dopamine agonists. EQ-5D utility scores averaged 0\ub703 (95% CI 0\ub701-0\ub705; p=0\ub70002) better with levodopa than with levodopa-sparing therapy; rates of dementia (hazard ratio [HR] 0\ub781, 95% CI 0\ub761-1\ub708, p=0\ub714), admissions to institutions (0\ub786, 0\ub763-1\ub718; p=0\ub74), and death (0\ub785, 0\ub769-1\ub706, p=0\ub717) were not significantly different, but the upper CIs precluded any substantial increase with levodopa compared with levodopa-sparing therapy. 179 (28%) of 632 patients allocated dopamine agonists and 104 (23%) of 460 patients allocated MAOBI discontinued allocated treatment because of side-effects compared with 11 (2%) of 528 patients allocated levodopa (p&lt;0\ub70001). Interpretation Very small but persistent benefits are shown for patient-rated mobility scores when treatment is initiated with levodopa compared with levodopa-sparing therapy. MAOBI as initial levodopa-sparing therapy was at least as effective as dopamine agonists. Funding UK National Institute for Health Research Health Technology Assessment Programme and UK Department of Health