TY - JOUR
T1 - Comprehensive systematic review summary
T2 - Treatment of cerebellar motor dysfunction and ataxia
AU - Zesiewicz, Theresa A.
AU - Wilmot, George
AU - Han Kuo, Sheng
AU - Perlman, Susan
AU - Greenstein, Patricia E.
AU - Sarah, Ying
AU - Tetsuo, Ashizawa
AU - Subramony, S. H.
AU - Schmahmann, Jeremy D.
AU - Figueroa, K. P.
AU - Mizusawa, Hidehiro
AU - Schöls, Ludger
AU - Shaw, Jessica D.
AU - Dubinsky, Richard M.
AU - Armstrong, Melissa J.
AU - Gronseth, Gary S.
AU - Sullivan, Kelly L.
N1 - Funding Information:
T. Zesiewicz has served as a clinical advisor for Steminent Biotherapeutics; has received travel reimbursement from the Department of Neurology at University of Southern Florida; has received travel reimbursement for a Biohaven Pharmaceuticals meeting; has served on the editorial board for Neurodegenerative Disease Management and Tremor and other Hyperkinetic Movements; has a patent for Methods of Treating Disease-Induced Ataxia and Non-Ataxic Imbalance (US Patent No. 9463190 B2); and has received research support for her division for approximately 20 clinical trials for Parkinson disease Friedreich ataxia, and spinocerebellar ataxias (SCAs). G. Wilmot has served on scientific advisory panels for Bio-haven Pharmaceuticals and Santhera Pharmaceuticals, and has received financial or material research support or compensation from Friedreich’s Ataxia Research Alliance, Reata Pharmaceuticals, and Shire. S.-H. Kuo and S. Perlman report no disclosures relevant to the manuscript. P. Greenstein has received an R21 grant award from the NIH to study the effect of transcranial magnetic stimulation on SCA (grant awarded in August of 2013; the study began in January 2014; no preliminary data yet available). S. Ying received a salary from Shire; received a salary during her employment with Pfizer Inc. and Takeda Pharmaceuticals Inc.; and received grant funding from the NIH. T. Ashizawa has nonfinancial competing interests with the Marigold Foundation, Myotonic Dystrophy Foundation, and the Muscular Dystrophy Association (MDA); receives honoraria from the NIH National Institute of Neurological Disorders and Stroke (NINDS) Neurological Sciences and Disorders B Study Section; received travel reimbursement from the MDA Medical Advisory Committee and the National Ataxia Foundation for the Ataxia Investigator Meeting; serves as an editor for PLoS ONE; has a patent (US Patent No. 6855497) on a DNA test for SCA type 10 (SCA10); receives funding for an NINDS research grant award R01NSNS083564; participates in a clinical trial of BHV-4157 (NCT02960893); and has received royalty payments from Baylor College of Medicine for a DNA test for SCA10 (US Patent No. 6855497). S. Subramony has received compensation for a lecture from Athena Diagnostics in October 2013; and has received travel reimbursement or honoraria from Reata Pharmaceuticals, ISIS Pharmaceuticals (now Ionis Pharmaceuticals), the NIH, the National Ataxia Foundation, and the Friedreich’s Ataxia Research Alliance. J. Schmahmann serves as a consultant to Ataxion, Biogen, Biohaven, Pfizer, and Takeda, and receives grant support from the National Ataxia Foundation and the A-T Children’s Project. K. Figueroa, H. Mizusawa, L. Schöls, and J. Shaw report no disclosures relevant to the manuscript. R. Dubinsky serves on the scientific advisory board for Allergan Pharmaceuticals; has received travel funding from the American Academy of Neurology (AAN), Allergan Pharmaceuticals, and the Huntington Study Group; serves as Level of Evidence associate editor for the AAN; receives honoraria from Allergan Pharmaceuticals; serves on the speakers bureau for Allergan Pharmaceuticals; and is involved with the commercial entity Allergan Pharmaceuticals and the government entities the NIH and the Agency for Healthcare Research and Quality. His spouse holds stock in Abbott Laboratories. M. Armstrong serves on the Level of Evidence editorial board for Neurology (not compensated financially) and is an AAN evidence-based methodologist. G. Gronseth serves as an associate editor (level of evidence review) for Neurology, serves on the editorial advisory board for Neurology Now, and is compensated by the AAN for methodologic activities. K. Sullivan has received research support from the Georgia Governor’s Office of Highway Safety and has a patent for Methods of Treating Disease-Induced Ataxia and Non-Ataxic Imbalance (US Patent No. 9463190 B2). Go to Neurology. org/N for full disclosures.
Publisher Copyright:
© 2018 American Academy of Neurology.
PY - 2018
Y1 - 2018
N2 - Objective To systematically review evidence regarding ataxia treatment. Methods A comprehensive systematic review was performed according to American Academy of Neurology methodology. Conclusions For patients with episodic ataxia type 2, 4-Aminopyridine 15 mg/d probably reduces ataxia attack frequency over 3 months (1 Class I study). For patients with ataxia of mixed etiology, riluzole probably improves ataxia signs at 8 weeks (1 Class I study). For patients with Friedreich ataxia or spinocerebellar ataxia (SCA), riluzole probably improves ataxia signs at 12 months (1 Class I study). For patients with SCA type 3, valproic acid 1,200 mg/d possibly improves ataxia at 12 weeks. For patients with spinocerebellar degeneration, thyrotropin-releasing hormone possibly improves some ataxia signs over 10 to 14 days (1 Class II study). For patients with SCA type 3 who are ambulatory, lithium probably does not improve signs of ataxia over 48 weeks (1 Class I study). For patients with Friedreich ataxia, deferiprone possibly worsens ataxia signs over 6 months (1 Class II study). Data are insufficient to support or refute the use of numerous agents. For nonpharmacologic options, in patients with degenerative ataxias, 4-week inpatient rehabilitation probably improves ataxia and function (1 Class I study); transcranial magnetic stimulation possibly improves cerebellar motor signs at 21 days (1 Class II study). For patients with multiple sclerosis-associated ataxia, the addition of pressure splints possibly has no additional benefit compared with neuromuscular rehabilitation alone (1 Class II study). Data are insufficient to support or refute use of stochastic whole-body vibration therapy (1 Class III study).
AB - Objective To systematically review evidence regarding ataxia treatment. Methods A comprehensive systematic review was performed according to American Academy of Neurology methodology. Conclusions For patients with episodic ataxia type 2, 4-Aminopyridine 15 mg/d probably reduces ataxia attack frequency over 3 months (1 Class I study). For patients with ataxia of mixed etiology, riluzole probably improves ataxia signs at 8 weeks (1 Class I study). For patients with Friedreich ataxia or spinocerebellar ataxia (SCA), riluzole probably improves ataxia signs at 12 months (1 Class I study). For patients with SCA type 3, valproic acid 1,200 mg/d possibly improves ataxia at 12 weeks. For patients with spinocerebellar degeneration, thyrotropin-releasing hormone possibly improves some ataxia signs over 10 to 14 days (1 Class II study). For patients with SCA type 3 who are ambulatory, lithium probably does not improve signs of ataxia over 48 weeks (1 Class I study). For patients with Friedreich ataxia, deferiprone possibly worsens ataxia signs over 6 months (1 Class II study). Data are insufficient to support or refute the use of numerous agents. For nonpharmacologic options, in patients with degenerative ataxias, 4-week inpatient rehabilitation probably improves ataxia and function (1 Class I study); transcranial magnetic stimulation possibly improves cerebellar motor signs at 21 days (1 Class II study). For patients with multiple sclerosis-associated ataxia, the addition of pressure splints possibly has no additional benefit compared with neuromuscular rehabilitation alone (1 Class II study). Data are insufficient to support or refute use of stochastic whole-body vibration therapy (1 Class III study).
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U2 - 10.1212/WNL.0000000000005055
DO - 10.1212/WNL.0000000000005055
M3 - Article
C2 - 29440566
AN - SCOPUS:85047192462
SN - 0028-3878
VL - 90
SP - 464
EP - 471
JO - Neurology
JF - Neurology
IS - 10
ER -