Use of 2 hydroxypropyl-beta-cyclodextrin therapy in two adult Niemann Pick Type C patients

Journal of the Neurological Sciences

Volumn 366, Issue , 2016, Pages 65-67

  García Robles, Ana Alejandra ^a   Company Albir, María José ^a   Megías Vericat, Juan Eduardo ^a   Fernández Megía, María José ^a   Pérez Miralles, Francisco Carlos ^b   López Briz, Eduardo ^a   Alcalá Vicente, Carmen ^b   Galeano, Inmaculada ^a   Casanova, Bonaventura ^b   Poveda, José Luis ^a  

^a HOSPITAL UNIVERSITARIO LA FE   (Spain)

^b UNIVERSITY OF VALENCIA   (Spain)

Author keywords

2 Hydroxypropyl cyclodextrin; Intrathecal; Metabolic disease; Niemann Pick; Toxicity

Indexed keywords

2 HYDROXYPROPYL BETA CYCLODEXTRIN; MIGLUSTAT; 2-HYDROXYPROPYL-BETA-CYCLODEXTRIN; BETA CYCLODEXTRIN DERIVATIVE;

ADULT; ARTICLE; ASPIRATION PNEUMONIA; BRAIN RADIOGRAPHY; CANDIDIASIS; CASE REPORT; CLINICAL EFFECTIVENESS; COMPASSIONATE USE; COMPUTER ASSISTED TOMOGRAPHY; DIARRHEA; DISEASE COURSE; DRUG SAFETY; DRUG WITHDRAWAL; ECHOGRAPHY; FEMALE; FEVER; GASTROINTESTINAL DISEASE; GENE MUTATION; HEPATOSPLENOMEGALY; HISTIOCYTOSIS; HUMAN; LUMBAR PUNCTURE; MENINGITIS; MENTAL DETERIORATION; NIEMANN PICK DISEASE; NUCLEAR MAGNETIC RESONANCE IMAGING; OMMAYA RESERVOIR; POSITRON EMISSION TOMOGRAPHY; PRIORITY JOURNAL; SPLENOMEGALY; VISUAL EVOKED POTENTIAL; FATALITY; GENETICS; INTRASPINAL DRUG ADMINISTRATION; MIDDLE AGED; NIEMANN-PICK DISEASE, TYPE C;

ADULT; BETA-CYCLODEXTRINS; COMPASSIONATE USE TRIALS; FATAL OUTCOME; FEMALE; HUMANS; INJECTIONS, SPINAL; MIDDLE AGED; NIEMANN-PICK DISEASE, TYPE C;

EID: 84966320579     PISSN: 0022510X     EISSN: 18785883     Source Type: Journal    
DOI: 10.1016/j.jns.2016.04.048     Document Type: Letter

References (15)

1. M.T. Vanier Niemann-Pick disease type C Orphanet J. Rare Dis. 5 2010 16 10.1186/1750-1172-5-16
2. M.C. Patterson, C.J. Hendriksz, M. Walterfang, and et al. Recommendations for the diagnosis and management of Niemann-Pick disease type C: an update Mol. Genet. Metab. 106 2012 330 344 10.1016/j.ymgme.2012.03.012
3. E.A. Ottinger, M.L. Kao, N. Carrillo-Carrasco, and et al. Collaborative development of 2-hydroxypropyl-β-cyclodextrin for the treatment of Niemann-Pick type C1 disease Curr. Top. Med. Chem. 14 2014 330 339 10.2174/1568026613666131127160118
4. C. Hastings Request for intrathecal delivery of HPBCD for Niemann Pick Type C patients, Caroline Hastings, M.D. Principal Investigator Department of Pediatric Hematology Oncology Children's Hospital & Research Center Oakland Submission Date to FDA: August 13 http://addiandcassi.com/wordpress/wp-content/uploads/Hempel-Cyclodextrin-Intrathecal-FDA-Filing-2010-Aug.pdf 2010 (accessed 15.02.2016)
5. M. Matsuo, M. Togawa, K. Hirabaru, and et al. Effects of cyclodextrin in two patients with Niemann-Pick Type C disease Mol. Genet. Metab. 108 2013 76 81 10.1016/j.ymgme.2012.11.005
6. M. Matsuo, K. Shraishi, K. Wada, and et al. Effects of intracerebroventricular administration of 2-hydroxypropyl-β-cyclodextrin in a patient with Niemann-Pick Type C disease Mol. Genet. Metab. Rep. 1 2014 391 400 10.1016/j.ymgmr.2014.08.004
7. T.J. Maarup, A.H. Chen, F.D. Porter, and et al. Intrathecal 2-hydroxypropyl-beta-cyclodextrin in a single patient with Niemann-Pick C1 Mol. Genet. Metab. 116 2015 75 79 10.1016/j.ymgme.2015.07.001
8. N.M. Yanjanin, J.I. Velez, A. Gropman, and et al. Linear clinical progression, independent of age of onset, in Niemann-Pick disease, type C Am. J. Med. Genet. B Neuropsychiatr. Genet. 153B 2010 132 140 10.1002/ajmg.b.30969
9. N. Morikawa, T. Mori, H. Kawashima, M. Takeyama, and S. Hori Pharmacokinetics of anticancer drugs in cerebrospinal fluid Ann. Pharmacother. 32 1998 1008 1012 10.1345/aph.17362
10. F.D. Porter, N. Yanjanin, A. Liu, and et al. Therapeutic trials for Niemann-Pick disease, Type C1: 2-hydroxypropyl-β-cyclodextrin Michael, Marcia, and Christa Parseghian Scientific Conference for Niemann-Pick Type C Research June 12-14, 2014. Abstract 7:18 2014
11. C.A. Naranjo, U. Busto, E.M. Sellers, and et al. A method for estimating the probability of adverse drug reactions Clin. Pharmacol. Ther. 30 1981 239 245 10.1038/clpt.1981.154
12. B. Liu, H. Li, J.J. Repa, S.D. Turley, and J.M. Dietschy Genetic variations and treatments that affect the lifespan of the NPC1 mouse J. Lipid Res. 49 2008 663 669 10.1194/jlr.M700525-JLR200
13. B. Liu, S.D. Turley, D.K. Burns, A.M. Miller, J.J. Repa, and J.M. Dietschy Reversal of defective lysosomal transport in NPC disease ameliorates liver dysfunction and neurodegeneration in the npc1 -/- mouse Proc. Natl. Acad. Sci. U. S. A. 106 2009 2377 2382 10.1073/pnas.0810895106
14. C.H. Vite, J.H. Bagel, G.P. Swain, M. Prociuk, T.U. Sikora, V.M. Stein, P. O'Donnell, T. Ruane, S. Ward, A. Crooks, S. Li, E. Mauldin, S. Stellar, M. De Meulder, M.L. Kao, D.S. Ory, C. Davidson, M.T. Vanier, and S.U. Walkley Intracisternal cyclodextrin prevents cerebellar dysfunction and Purkinje cell death in feline Niemann-Pick type C1 disease Sci. Transl. Med. 7 276 2015 276ra26 10.1126/scitranslmed.3010101
15. C. Vite, E. Mauldin, S. Ward, V. Stein, M. Prociuk, M.E. Haskins, and et al. Intrathecal cyclodextrin therapy of feline Niemann-Pick Type C disease Mol. Genet. Metab. 102 2011 S44 10.1016/j.ymgme.2010.11.148