PMID- 25346067
OWN - NLM
STAT- MEDLINE
DCOM- 20160404
LR  - 20181113
IS  - 1432-1459 (Electronic)
IS  - 0340-5354 (Linking)
VI  - 262
IP  - 1
DP  - 2015 Jan
TI  - A randomized controlled pilot trial of lithium in spinocerebellar ataxia type 2.
PG  - 149-53
LID - 10.1007/s00415-014-7551-0 [doi]
AB  - Spinocerebellar ataxia type 2 (SCA2) is an autosomal dominant disorder. Lithium 
      is able to stimulate autophagy, and to reduce Ca(2+) efflux from the 
      inositol-1,4,5-triphosphate receptor. We designed a phase II, randomized, 
      placebo-controlled, double-blind, 48-week trial with lithium carbonate in 20 
      patients with SCA2. The primary objective was to determine safety and 
      tolerability of lithium. The secondary objectives were to determine disease 
      progression, quality of life, mood, and brain volume change. Sixteen patients 
      completed the trial, 8 randomized to lithium, 8 to placebo. Forty adverse events 
      (AEs) were reported during the trial, twenty-eight in the lithium and 12 in the 
      placebo group (p = 0.11). Mean AE duration was 57.4 +/- 60.8 and 77.4 +/- 68.5 days 
      (p = 0.37). Non-significant differences were observed for the SARA and for brain 
      volume change, whereas a significant reduction in the BDI-II was observed for 
      lithium group (p < 0.05). Lithium was well tolerated and reported AEs were 
      similar to those previously described for bipolar disorder patients. A correctly 
      powered phase III trial is needed to assess if lithium may slow disease 
      progression in SCA2.
FAU - Sacca, Francesco
AU  - Sacca F
AD  - Department of Neurosciences, Reproductive and Odontostomatological Sciences, 
      University Federico II, Via Pansini, 5, 80131, Napoli, NA, Italy, 
      francesco.sacca@unina.it.
FAU - Puorro, Giorgia
AU  - Puorro G
FAU - Brunetti, Arturo
AU  - Brunetti A
FAU - Capasso, Giovambattista
AU  - Capasso G
FAU - Cervo, Amedeo
AU  - Cervo A
FAU - Cocozza, Sirio
AU  - Cocozza S
FAU - de Leva, Mariafulvia
AU  - de Leva M
FAU - Marsili, Angela
AU  - Marsili A
FAU - Pane, Chiara
AU  - Pane C
FAU - Quarantelli, Mario
AU  - Quarantelli M
FAU - Russo, Cinzia Valeria
AU  - Russo CV
FAU - Trepiccione, Francesco
AU  - Trepiccione F
FAU - De Michele, Giuseppe
AU  - De Michele G
FAU - Filla, Alessandro
AU  - Filla A
FAU - Morra, Vincenzo Brescia
AU  - Morra VB
LA  - eng
PT  - Clinical Trial, Phase II
PT  - Journal Article
PT  - Randomized Controlled Trial
PT  - Research Support, Non-U.S. Gov't
DEP - 20141028
PL  - Germany
TA  - J Neurol
JT  - Journal of neurology
JID - 0423161
RN  - 0 (Enzyme Inhibitors)
RN  - 2BMD2GNA4V (Lithium Carbonate)
SB  - IM
MH  - Adult
MH  - Atrophy/pathology
MH  - Autophagy/drug effects
MH  - Brain/*pathology
MH  - Double-Blind Method
MH  - Enzyme Inhibitors/administration & dosage/adverse effects/*pharmacology
MH  - Female
MH  - Humans
MH  - Lithium Carbonate/administration & dosage/adverse effects/*pharmacology
MH  - Male
MH  - Middle Aged
MH  - Pilot Projects
MH  - Severity of Illness Index
MH  - Spinocerebellar Ataxias/*drug therapy/pathology/*physiopathology
MH  - Treatment Outcome
EDAT- 2014/10/28 06:00
MHDA- 2016/04/05 06:00
CRDT- 2014/10/28 06:00
PHST- 2014/09/22 00:00 [received]
PHST- 2014/10/18 00:00 [accepted]
PHST- 2014/10/17 00:00 [revised]
PHST- 2014/10/28 06:00 [entrez]
PHST- 2014/10/28 06:00 [pubmed]
PHST- 2016/04/05 06:00 [medline]
AID - 10.1007/s00415-014-7551-0 [doi]
PST - ppublish
SO  - J Neurol. 2015 Jan;262(1):149-53. doi: 10.1007/s00415-014-7551-0. Epub 2014 Oct 
      28.