Abstract | BACKGROUND: RESULTS: Here we present safety, tolerability and efficacy data from 12 patients treated intravenously (IV) for over 7 years with HPβCD in the US and Brazil. Some patients subsequently received intrathecal (IT) treatment with HPβCD following on average 13 months of IV HPβCD. Several patients transitioned to an alternate HPβCD. Moderately affected NPC patients treated with HPβCD showed slowing of disease progression. Severely affected patients demonstrated periods of stability but eventually showed progression of disease. Neurologic and neurocognitive benefits were seen in most patients with IV alone, independent of the addition of IT administration. Physicians and caregivers reported improvements in quality of life for the patients on IV therapy. There were no safety issues, and the drug was well tolerated and easy to administer. CONCLUSIONS: These expanded access data support the safety and potential benefit of systemic IV administration of HPβCD and provide a platform for two clinical trials to study the effect of intravenous administration of HPβCD in NPC patients.
|
Authors | Caroline Hastings, Camilo Vieira, Benny Liu, Cyrus Bascon, Claire Gao, Raymond Y Wang, Alicia Casey, Sharon Hrynkow |
Journal | Orphanet journal of rare diseases
(Orphanet J Rare Dis)
Vol. 14
Issue 1
Pg. 228
(10 21 2019)
ISSN: 1750-1172 [Electronic] England |
PMID | 31639011
(Publication Type: Journal Article, Research Support, Non-U.S. Gov't)
|
Chemical References |
- 2-Hydroxypropyl-beta-cyclodextrin
|
Topics |
- 2-Hydroxypropyl-beta-cyclodextrin
(administration & dosage, therapeutic use)
- Adolescent
- Adult
- Child
- Child, Preschool
- Female
- Humans
- Infant
- Male
- Niemann-Pick Disease, Type C
(drug therapy)
- Retrospective Studies
- Young Adult
|