Matches in SemOpenAlex for { <https://semopenalex.org/work/W2806334396> ?p ?o ?g. }
Showing items 1 to 71 of
71
with 100 items per page.
- W2806334396 abstract "Background The term ultra-rare diseases has been coined to describe very infrequent diseases. The European Union defines them as those whose prevalence is less than 1 per 50 000 inhabitants. Purpose Describe ultra-rare diseases treated in our hospital and analyse the budgetary impact. Material and methods Descriptive analysis of a cohort of seven patients suffering from an ultra-rare disease in a third-level hospital during 2016. Results The hospital serves a population of 5 00 000 inhabitants. It currently treats seven patients suffering from an ultra-rare disease: one patient with Matoteaux Lamy disease (mucopolysaccharidosis type IV), two patients with Gaucher syndrome, two patients with paroxysmal nocturnal haemoglobinuria (PNH), one patient with atypical haemolytic uremic syndrome (SHUa) and one patient with Hunter’s disease. The drugs with high budgetary impact used to treat these diseases are: gasulfase 5 mg/ml for Matoteaux Lamy disease, imiglucerase 400 U/vial for Gaucher syndrome, eculizumab 300 mg/30 ml for PNH and for SHUa, and idursulfase for Hunter’s disease. The following table shows the average monthly consumption of these drugs and their cost: In total, the monthly cost is €199,029.94 (€2,388,359.24 per year). This represents approximately 4% of the hospital’s annual drug budget. Conclusion Complexity and high cost of treatment of ultra-rare diseases makes their management a challenge, both clinically and logistically. The pharmacy service is in charge of ensuring the availability of drugs necessary for the treatment of these patients, and must also perform a correct pharmacotherapeutic follow-up. Reference and/or Acknowledgements 1. Conference of orphan drug and rare diseases, Hospital la Paz, Madrid2017. No conflict of interest" @default.
- W2806334396 created "2018-06-13" @default.
- W2806334396 creator A5012465405 @default.
- W2806334396 creator A5029135402 @default.
- W2806334396 creator A5030552012 @default.
- W2806334396 creator A5033055030 @default.
- W2806334396 creator A5061329864 @default.
- W2806334396 creator A5062012854 @default.
- W2806334396 creator A5089696416 @default.
- W2806334396 date "2018-03-01" @default.
- W2806334396 modified "2023-10-17" @default.
- W2806334396 title "2SPD-012 Budgetary impact of ultra-rare diseases in a third-level hospital" @default.
- W2806334396 doi "https://doi.org/10.1136/ejhpharm-2018-eahpconf.33" @default.
- W2806334396 hasPublicationYear "2018" @default.
- W2806334396 type Work @default.
- W2806334396 sameAs 2806334396 @default.
- W2806334396 citedByCount "1" @default.
- W2806334396 countsByYear W28063343962021 @default.
- W2806334396 crossrefType "proceedings-article" @default.
- W2806334396 hasAuthorship W2806334396A5012465405 @default.
- W2806334396 hasAuthorship W2806334396A5029135402 @default.
- W2806334396 hasAuthorship W2806334396A5030552012 @default.
- W2806334396 hasAuthorship W2806334396A5033055030 @default.
- W2806334396 hasAuthorship W2806334396A5061329864 @default.
- W2806334396 hasAuthorship W2806334396A5062012854 @default.
- W2806334396 hasAuthorship W2806334396A5089696416 @default.
- W2806334396 hasBestOaLocation W28063343962 @default.
- W2806334396 hasConcept C104863432 @default.
- W2806334396 hasConcept C126322002 @default.
- W2806334396 hasConcept C177713679 @default.
- W2806334396 hasConcept C187212893 @default.
- W2806334396 hasConcept C194828623 @default.
- W2806334396 hasConcept C199360897 @default.
- W2806334396 hasConcept C2779134260 @default.
- W2806334396 hasConcept C2779256446 @default.
- W2806334396 hasConcept C2779701055 @default.
- W2806334396 hasConcept C2908647359 @default.
- W2806334396 hasConcept C41008148 @default.
- W2806334396 hasConcept C512399662 @default.
- W2806334396 hasConcept C60644358 @default.
- W2806334396 hasConcept C71924100 @default.
- W2806334396 hasConcept C72563966 @default.
- W2806334396 hasConcept C75480439 @default.
- W2806334396 hasConcept C86803240 @default.
- W2806334396 hasConcept C99454951 @default.
- W2806334396 hasConceptScore W2806334396C104863432 @default.
- W2806334396 hasConceptScore W2806334396C126322002 @default.
- W2806334396 hasConceptScore W2806334396C177713679 @default.
- W2806334396 hasConceptScore W2806334396C187212893 @default.
- W2806334396 hasConceptScore W2806334396C194828623 @default.
- W2806334396 hasConceptScore W2806334396C199360897 @default.
- W2806334396 hasConceptScore W2806334396C2779134260 @default.
- W2806334396 hasConceptScore W2806334396C2779256446 @default.
- W2806334396 hasConceptScore W2806334396C2779701055 @default.
- W2806334396 hasConceptScore W2806334396C2908647359 @default.
- W2806334396 hasConceptScore W2806334396C41008148 @default.
- W2806334396 hasConceptScore W2806334396C512399662 @default.
- W2806334396 hasConceptScore W2806334396C60644358 @default.
- W2806334396 hasConceptScore W2806334396C71924100 @default.
- W2806334396 hasConceptScore W2806334396C72563966 @default.
- W2806334396 hasConceptScore W2806334396C75480439 @default.
- W2806334396 hasConceptScore W2806334396C86803240 @default.
- W2806334396 hasConceptScore W2806334396C99454951 @default.
- W2806334396 hasLocation W28063343961 @default.
- W2806334396 hasLocation W28063343962 @default.
- W2806334396 hasOpenAccess W2806334396 @default.
- W2806334396 hasPrimaryLocation W28063343961 @default.
- W2806334396 isParatext "false" @default.
- W2806334396 isRetracted "false" @default.
- W2806334396 magId "2806334396" @default.
- W2806334396 workType "article" @default.