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Intrathecal cyclodextrin in the treatment of Niemann-Pick disease type C
  1. Maria Roch Santed1,
  2. Maria Josep Cabañas Poy1,
  3. Mireia Del Toro Riera2,
  4. Carme Cañete Ramírez1,
  5. Aurora Fernández Polo1,
  6. Susana Clemente Bautista1
  1. 1Pharmacy Department, Hospital Vall d'Hebron, Barcelona, Barcelona, Spain
  2. 2Pediatric Neurology Department, Hospital Vall d'Hebron, Barcelona, Barcelona, Spain
  1. Correspondence to Maria Roch Santed, Hospital Vall d'Hebron, Pharmacy department, Passeig de la Vall d'Hebron, 119-129, Barcelona, Barcelona 08035, Spain; maria24rs{at}gmail.com

Abstract

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.

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