Utilize este identificador para referenciar este registo: http://hdl.handle.net/10451/21025
Título: Therapeutic profile of orphan medicines
Autor: Teixeira de Barros, Carla Maria
Papoila, Ana Luisa
Palavras-chave: Pharmacology & Pharmacy
Data: 2007
Citação: PHARMACOEPIDEMIOLOGY AND DRUG SAFETY. - Vol. 16, n. 4 (APR 2007), p. 435-440
Resumo: Purpose To characterise the therapeutic profile of orphan medicines. Materials and Methods A cross-sectional study was performed during 2 months in a convenience sample of seven hospital pharmacy services, in the region of Lisbon. Data were collected, from pharmaceutical service's records. Results A total of 18 orphan medicines, were dispensed to 355 patients with rare diseases. Most patients were adults (76.4%). Premature and neonates accounted with 50.0% of the paediatric patients. Differences were not found between the proportion of male and female patients across age groups (p = 0.762). Only 18.3% were inpatients. A high proportion of paediatric inpatients (58.3%) were seen in relation to adult inpatients (5.9%) (P = 0.001). In general, anti-neoplastic and immunomodulating agents for rare cancers were the most frequent dispensed medicines (51.3%). In relation to paediatrics, Caffeine Citrate for primary apnoea of premature newborns had the higher frequency distribution (57.1%). Five (71.4%) medicines dispensed for paediatrics, do not have market authorisation and the remaining (28.6%) were used off-label. For pulmonary arterial hypertension 19 of 27 patients (70.3%) were treated with Bosentan. According to evidence-based clinical practice guidelines, Bosentan has a good level of evidence and substantial benefit: grade of recommendation A. Conclusions Most orphan medicines dispensed to paediatrics and adults were not licensed. A special pharmacovigilance program and a risk management plan through the entire life cycle should be implemented, towards effectiveness and safety of orphan medicines. Copyright (c) 2006 John Wiley & Sons, Ltd.
URI: http://hdl.handle.net/10451/21025
DOI: http://dx.doi.org/10.1002/pds.1315
ISSN: 1053-8569
Aparece nas colecções:FF - Produção Científica 2000-2009

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