TY - JOUR T1 - Intrathecal cyclodextrin in the treatment of Niemann-Pick disease type C JF - European Journal of Hospital Pharmacy JO - Eur J Hosp Pharm SP - 185 LP - 188 DO - 10.1136/ejhpharm-2016-001067 VL - 24 IS - 3 AU - Maria Roch Santed AU - Maria Josep Cabañas Poy AU - Mireia Del Toro Riera AU - Carme Cañete Ramírez AU - Aurora Fernández Polo AU - Susana Clemente Bautista Y1 - 2017/05/01 UR - http://ejhp.bmj.com/content/24/3/185.abstract N2 - 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. ER -