Management of Huntington’s disease: role of tetrabenazine

Figures & data

Table 1 Main data across the selected studies about TBZ in HD patients

Study	Year	Study design	No. of HD patients under TBZ	No. of HD patients on placebo	No. of follow-up	Maximum time of observation	Mean TBZ dosage	Short-term change of TMC	Long-term change of TMC	Clinical global impression of change	Adverse effects	Serious adverse accidents or causes of withdrawal
Paleacu et al^Citation18	2004	Open-label long-term follow-up	28 (HD plus other types of chorea) among a total of 150 patients with different movement disorders			88 weeks	Up to 150 mg/die (76.2 ± 38.4 mg, range 25–175)			32.14% marked improvement (among the total chorea patients)	Not defined in HD patients	Worsening (3), lack of benefit (7 patients with chorea including HD)
Huntington Study Group^Citation15	2006	Multicenter, prospective, double-blind, placebo-controlled, dose-finding	54	30	1	12 weeks	Up to 100 mg/die (range 25–100)	–5 ± 0.5 (M ± SE) (P = 0.0001 vs placebo)		40% much improved (P = 0.0007 vs placebo)	Sedation (13), akatisia (4), parkinsonism, (2), depression (5)	Suicide (1), intracerebral hemorrhage consequent to a fall (1), breast cancer (1)
Fasano et al^Citation17	2008	Long-term retrospective	68	0	2	137.6 + 100 weeks	57.5 ± 14.7 mg (range 6.25–150)	–21% (0–20) (P = 0.00001 vs baseline)	–9% (1–22) (n.s. vs baseline)		Drowsiness (17), depression (11), disturbances of gut function (8), worsening of parkinsonism (4), xerostomia (2), hypotension (1)	Worsening of psychiatric disturbance (1), disabling asthenia (1)
Frank^Citation16	2009	Open-label extension study	45	0	3	80 weeks	Up to 200 mg/die (range 12.5–175 mg)	–5.8 ± 5 (M + SD) (P < 0.05 vs baseline)	–4.6 ± 5.5 (M + SD) (P < 0.05 vs baseline)		Sedation (18), depressed mood (17), anxiety (13), insomnia (10), fatigue (7), agitation (5), fall (4), dysphagia (3), dystonia (3)	Vocal tics, depression, delusions (3); methastatic breast cancer (1); elevated bilirubine level, akatisia (1); elevated liver enzyme (1)

Abbreviations: HD, Huntington’s disease; TBZ, tetrabenazine; TMC, Total Maximal Chorea score.

Table 2 Mean values and standard deviations of the principal clinical features in our HD patients

	Tetrabenazine N = 28			Clotiapine N = 10
	Basal	1 year	2 years	Basal	1 year	2 years
TFC	5.9 ± 3.9	5.8 ± 2.3	5.5 ± 3.9	7.13 ± 5.4	6.9 ± 4.5	5.7 ± 4.3
UHDRSM	43.77 ± 22	44.5 ± 20	50.21 ± 27	40.26 ± 28	44.3 ± 19.8	55.88 ± 22.3
Chorea	10 ± 5.96	9.6 ± 4.66	10.7 ± 4.29	9.8 ± 5.4	9.9 ± 6.3	12.12 ± 4.2

Abbreviations: HD, Huntingdon’s disease; TFC, Total Functional Capacity Scale; UHDRSM, Motor Section of the Unified Huntington’s Disease Rating Scale.

Figure 1 Mean values and standard deviations of A) the global motor score of the Unified Huntington’s Disease Rating Scale (UHDRSM), B) the chorea score, and C) the Total Functional Capacity (TFC) Scale in a cohort of Huntington’s disease patients before and after 2 years of treatment. The results of two-way analysis of variance (ANOVA) are reported.

PaleacuDGiladiNMooreOTetrabenazine treatment in movement disordersClin Neuropharmacol20042723023315602104

 PubMed Web of Science ®Google Scholar

Huntington Study GroupTetrabenazine as antichorea therapy in Huntington disease: a randomized controlled trialNeurology200666336637216476934

 PubMed Web of Science ®Google Scholar

FasanoACadedduFGuidubaldiAThe long-term effect of tetrabenazine in the management of Huntington diseaseClin Neuropharmacol200831631331819050408

 PubMed Web of Science ®Google Scholar

FrankSTetrabenazine as anti-chorea therapy in Huntington disease: an open-label continuation study. Huntington Study Group/TETRA-HD InvestigatorsBMC Neurol200996220021666

 PubMed Web of Science ®Google Scholar