Intrathecal cyclodextrin in the treatment of Niemann-Pick disease type C
| Autor: | Maria Josep Cabañas Poy, Maria Roch Santed, Aurora Fernández Polo, Carme Cañete Ramírez, Susana Clemente Bautista, Mireia Del Toro Riera |
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| Rok vydání: | 2016 |
| Předmět: |
0301 basic medicine
Drug Pediatrics medicine.medical_specialty media_common.quotation_subject Initial dose Case Report Disease Intrathecal 03 medical and health sciences Hydroxypropyl-beta-cyclodextrin 0302 clinical medicine Miglustat polycyclic compounds medicine General Pharmacology Toxicology and Pharmaceutics Adverse effect media_common Niemann–Pick disease type C business.industry technology industry and agriculture medicine.disease carbohydrates (lipids) 030104 developmental biology Anesthesia business 030217 neurology & neurosurgery medicine.drug |
| Zdroj: | European Journal of Hospital Pharmacy. 24:185-188 |
| ISSN: | 2047-9964 2047-9956 |
| DOI: | 10.1136/ejhpharm-2016-001067 |
| Popis: | Case A child with Niemann-Pick disease type C was started on miglustat therapy at the age of 2 years. Intrathecal administration of hydroxypropyl-β-cyclodextrin was added 5 months later. The initial dose of 175 mg was gradually increased over the first 6 months to reach 325 mg. The drug was administered every 15 days, and the patient received 43 doses. A slight delay in progression of the disease was seen during the first year of intrathecal hydroxypropyl-β-cyclodextrin. However, additional symptoms have emerged since that time, suggesting a lack of effectiveness of the drug. Our patient has shown no drug-related adverse events. Conclusions Intrathecal hydroxypropyl-β-cyclodextrin therapy is safe, but its efficacy seems questionable in a patient with the severe infantile form of Niemann-Pick disease type C. |
| Databáze: | OpenAIRE |
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