PMID- 28155026
OWN - NLM
STAT- MEDLINE
DCOM- 20180309
LR  - 20220318
IS  - 1590-3478 (Electronic)
IS  - 1590-1874 (Linking)
VI  - 38
IP  - 5
DP  - 2017 May
TI  - Early experience with compassionate use of 2 hydroxypropyl-beta-cyclodextrin for 
      Niemann-Pick type C disease: review of initial published cases.
PG  - 727-743
LID - 10.1007/s10072-017-2833-9 [doi]
AB  - Niemann-Pick type C (NP-C) is a rare neurodegenerative disorder. Management is 
      mainly supportive and symptomatic. The investigational use of 
      2-hydroxypropyl-beta-cyclodextrin (HP-beta-CD) showed a promising role in treating 
      NP-C, although efficacy and safety have not been established. We conducted 
      searches of MEDLINE, Cochrane, EMBASE, and other databases of reported cases of 
      HP-beta-CD compassionate use in NP-C disease. Sixteen reported cases were eligible, 
      including evaluable information of 17 patients. The median onset age of HP-beta-CD 
      was 14 years (range 2-49 years). Intrathecal route was employed in 16 patients, 
      in 3 patients simultaneously to IV infusions. Intracerebroventricular route was 
      used in two patients. An objective improvement of clinical outcomes was measured 
      in 14 patients, mainly by the NIH NP-C Clinical Severity Score and brainstem 
      auditory evoked potential. Besides, an increase in metabolism and activities of 
      the brain were observed in image tests and cholesterol biomarkers. Most patients 
      showed some clinical benefit or a stabilization of NP-C progression. There were 
      17 adverse events (AEs) reported in 11 patients, 11 of them related to the drug 
      and 6 to the route of administration. Loss of hearing was reported in four 
      patients. The most severe AE were fever and chemical meningitis. Results suggest 
      that efficacy may be partial and dependent on the early administration of the 
      drug, the severity of the disease, and interpersonal variability. HP-beta-CD could 
      help stabilize NP-C with low toxicity potential, although some AEs have been 
      reported. Moreover, controlled clinical trials would be necessary to evaluate the 
      role of HP-beta-CD in NP-C.
FAU - Megias-Vericat, Juan Eduardo
AU  - Megias-Vericat JE
AD  - Servicio de Farmacia, Area del Medicamento, Hospital Universitari i Politecnic La 
      Fe, Avda. Fernando Abril Martorell, 106, 46026, Valencia, Spain.
FAU - Garcia-Robles, Ana
AU  - Garcia-Robles A
AD  - Servicio de Farmacia, Area del Medicamento, Hospital Universitari i Politecnic La 
      Fe, Avda. Fernando Abril Martorell, 106, 46026, Valencia, Spain.
FAU - Company-Albir, Maria Jose
AU  - Company-Albir MJ
AD  - Servicio de Farmacia, Area del Medicamento, Hospital Universitari i Politecnic La 
      Fe, Avda. Fernando Abril Martorell, 106, 46026, Valencia, Spain. 
      company_mjoalb@gva.es.
FAU - Fernandez-Megia, Maria Jose
AU  - Fernandez-Megia MJ
AD  - Servicio de Farmacia, Area del Medicamento, Hospital Universitari i Politecnic La 
      Fe, Avda. Fernando Abril Martorell, 106, 46026, Valencia, Spain.
FAU - Perez-Miralles, Francisco Carlos
AU  - Perez-Miralles FC
AD  - Unidad Mixta de Esclerosis Multiple y Neurorregeneracion, IIS Hospital La Fe, 
      Universitat de Valencia, Hospital Universitari i Politecnic La Fe, Avda. Fernando 
      Abril Martorell, 106, 46026, Valencia, Spain.
FAU - Lopez-Briz, Eduardo
AU  - Lopez-Briz E
AD  - Servicio de Farmacia, Area del Medicamento, Hospital Universitari i Politecnic La 
      Fe, Avda. Fernando Abril Martorell, 106, 46026, Valencia, Spain.
FAU - Casanova, Bonaventura
AU  - Casanova B
AD  - Unidad Mixta de Esclerosis Multiple y Neurorregeneracion, IIS Hospital La Fe, 
      Universitat de Valencia, Hospital Universitari i Politecnic La Fe, Avda. Fernando 
      Abril Martorell, 106, 46026, Valencia, Spain.
FAU - Poveda, Jose Luis
AU  - Poveda JL
AD  - Servicio de Farmacia, Area del Medicamento, Hospital Universitari i Politecnic La 
      Fe, Avda. Fernando Abril Martorell, 106, 46026, Valencia, Spain.
LA  - eng
PT  - Journal Article
PT  - Review
DEP - 20170202
PL  - Italy
TA  - Neurol Sci
JT  - Neurological sciences : official journal of the Italian Neurological Society and 
      of the Italian Society of Clinical Neurophysiology
JID - 100959175
RN  - 0 (Excipients)
RN  - 1I96OHX6EK (2-Hydroxypropyl-beta-cyclodextrin)
SB  - IM
MH  - 2-Hydroxypropyl-beta-cyclodextrin/*therapeutic use
MH  - Databases, Bibliographic/statistics & numerical data
MH  - Excipients/*therapeutic use
MH  - Humans
MH  - Niemann-Pick Disease, Type C/*drug therapy
OTO - NOTNLM
OT  - 2-Hydroxypropyl-beta-cyclodextrin
OT  - Effectiveness
OT  - Niemann-Pick
OT  - Toxicity
EDAT- 2017/02/06 06:00
MHDA- 2018/03/10 06:00
CRDT- 2017/02/04 06:00
PHST- 2016/10/13 00:00 [received]
PHST- 2017/01/25 00:00 [accepted]
PHST- 2017/02/06 06:00 [pubmed]
PHST- 2018/03/10 06:00 [medline]
PHST- 2017/02/04 06:00 [entrez]
AID - 10.1007/s10072-017-2833-9 [pii]
AID - 10.1007/s10072-017-2833-9 [doi]
PST - ppublish
SO  - Neurol Sci. 2017 May;38(5):727-743. doi: 10.1007/s10072-017-2833-9. Epub 2017 Feb 
      2.