Effect of enzyme therapy and prognostic factors in 69 adults with Pompe disease: An open-label single-center study

Abstract. Background: Enzyme replacement therapy (ERT) in adults with Pompe disease, a progressive neuromuscular disorder, is of promising but variable efficacy. We investigated whether it alters the course of disease, and also identified potential prognostic factors. Methods. Patients in this open-label single-center study were treated biweekly with 20 mg/kg alglucosidase alfa. Muscle strength, muscle function, and pulmonary function were assessed every 3-6 months and analyzed using repeated-measures ANOVA. Results: Sixty-nine patients (median age 52.1 years) were followed for a median of 23 months. Muscle strength increased after start of ERT (manual muscle testing 1.4 percentage points per year (pp/y); hand-held dynamometry 4.0 pp/y; both p < 0.001). Forced vital capacity (FVC) remained stable when measured in upright, but declined in supine position (-1.1 pp/y; p = 0.03). Muscle function did not improve in all patients (quick motor function test 0.7 pp/y; p = 0.14), but increased significantly in wheelchair-independent patients and those with mild and moderate muscle weakness.Relative to the pre-treatment period (49 patients with 14 months pre-ERT and 22 months ERT median follow-up), ERT affected muscle strength positively (man

Katalytische oxidatie van SO2 in rookgas

Document(en) uit de collectie Chemische Procestechnologie.DelftChemTechApplied Science

Een aanvullend acheologisch onderzoek op het Oldehoofsterkerkhof te Leeuwarden

Lit.opg

Effectiveness and toxicity of hypofractionated high-dose intensity-modulated radiotherapy versus 2- and 3-dimensional radiotherapy in incurable head and neck cancer

BACKGROUND: This retrospective study evaluates efficacy and tolerability of high-dose hypofractionated radiotherapy (RT) in patients with head and neck cancer. METHODS: All patients with head and neck cancer treated between September 2003 and September 2013 with 12 x 4 Gy RT were included. Two and 3D-RT or intensity-modulated radiotherapy (IMRT) were used. Overall survival (OS), tumor response, and palliative effect were evaluated. RESULTS: Palliative effect occurred in 63% of 81 included patients, lasted a median of 4.6 months, and was correlated with tumor response (p = .006). Median OS was 7.2 months. Confluent mucositis occurred more often in patients treated with 2D/3D-RT than IMRT (26% vs 44%; p = .04) and lasted for a median of 2 weeks. CONCLUSION: High-dose hypofractionated RT resulted in meaningful palliation in 63%, lasting for almost 5 months. IMRT should be the technique of choice, as it results in less high-grade toxicity. The 12 x 4 schedule should be opted for patients with reasonable functional capacities and a life expectancy of >6 months. (c) 2015 Wiley Periodicals, Inc. Head Neck 38: E1264-E1270, 2016

Development of a rating scale for determining competence in basketball referees : implications for sport psychology

Construction et validation d'une échelle d'évaluation de la compétence chez les arbitres de basket-ball, l'échelle BARS (Behaviorally Anchored Rating Scale)