Observational Study of IncobotulinumtoxinA for Cervical Dystonia or Blepharospasm (XCiDaBLE): Interim Results for the First 170 Subjects with Blepharospasm

Hubert H. Fernandez; Joseph Jankovic; John B. Holds; Daniel Lin; John Burns; Amit Verma; Kapil Sethi; Eric J. Pappert; on behalf of the XCiDaBLE Study Group

doi:10.5334/tohm.181

Figures & Tables

Table 1

Subject Demographics and Disease Characteristics

Characteristic	Blepharospasm N = 170
Female gender, n (%)	131 (77.1)
Race,¹ n (%)
Asian	5 (2.9)
Black	7 (4.1)
White	156 (91.8)
Other	2 (1.2)
Age at enrollment (years), mean (SD)	63.7 (11.4)
Age at onset (years), mean (SD)	n = 169 53.9 (12.6)
Estimated duration of disease (years), mean (SD)	n = 169 10.9 (8.3)
Age at first botulinum toxin treatment prior to enrollment on this study (years), mean (SD)	n = 157 58.7 (11.1)
Time since most recent botulinum toxin injection (months), mean (SD)	n = 163 4.3 (9.7)
Subjects with previous botulinum toxin therapy, n (%)	164 (96.5)
Previous botulinum toxin treatments²
AbobotulinumtoxinA, n (%)	4 (2.4)
Mean number of treatments (SD)	n = 4 2.3 (1.3)
IncobotulinumtoxinA, n (%)	43 (25.3)
Mean number of treatments (SD)	n = 43 1.6 (1.1)
OnabotulinumtoxinA, n (%)	162 (95.3)
Mean number of treatments (SD)	n = 152 21.8 (19.1)
RimabotulinumtoxinB, n (%)	6 (3.5)
Mean number of treatments (SD)	n = 6 10.7 (20.8)
Effect of previous botulinum toxin treatment
None	1 (0.6)
Partial	35 (20.6)
Near complete/full	127 (74.7)
Unknown	7 (4.1)
Previous botulinum toxin duration (days), mean (SD)	n = 157 79.0 (31.6)
Baseline employment
Employed at time of onset	N = 169
Yes	100 (59.2)
If employed at onset, was employment status affected
Different job with less responsibility	4 (4.0)
Loss of employment	11 (11.0)
No	75 (75.0)
Same job, less pay	1 (1.0)
Unknown	9 (9.0)
Receiving or seeking disability benefits?	n = 161
Yes	19 (11.8)

{ label (or @symbol) needed for fn[@id='TFN01t01'] } Abbreviation: SD, Standard Deviation.

1 A subject may select multiple races.

2 Subjects may have received more than one serotype and thus the total number of subjects per treatment sums to more than the total number of subjects.

Percentages are based on non-missing values.

Table 2

Summary of IncobotulinumtoxinA Dosing

	Blepharospasm N = 170
Dose at first injection visit, mean units (SD)	71.5 (50.6)
Most frequent volume (mL) of saline/100 U incobotulinumtoxinA at 1st injection visit [range]	2 (1–10)
Dosing by muscle for both eyes: total at first injection visit,¹ mean units (SD)
Orbicularis oculi, n = 168	26.2 (14.7)
Procerus, n = 77	7.1 (5.7)
Corrugator supercilii, n = 118	8.5 (7.1)
Summary of muscle identification at first injection visit, n (%)
Anatomical location	168 (98.8)
Electromyography	2 (1.2)
Electrical stimulation	—
Sonography	—

Abbreviation: SD, Standard Deviation.

1 More than one muscle could have been treated.

Table 3

Global Impressions (Investigator and Patient)

Blepharospasm, n (%)
Severity				Improvement
Category	Baseline	Baseline	Category	4 Weeks
	(1st Injection Visit)	(1st Injection Visit)		Post 1st Injection
	Investigator Severity	Patient Severity		Patient Improvement
	N = 169	N = 165		N = 159
Not assessed	0	2 (1.2)	Not assessed	5 (3.1)
Normal (1)	12 (7.1)	34 (20.6)	Very much improved (1)	24 (15.1)
Borderline (2)	10 (5.9)	18 (10.9)	Much improved (2)	57 (35.9)
Mildly (3)	32 (18.9)	33 (20.0)	Minimally improved (3)	43 (27.0)
Moderately (4)	71 (42.0)	40 (24.2)	No change (4)	17 (10.7)
Markedly (5)	29 (17.2)	23 (13.9)	Minimally worse (5)	5 (3.1)
Severely (6)	15 (8.9)	12 (7.3)	Much worse (6)	7 (4.4)
Extremely (7)	0	3 (1.8)	Very much worse (7)	1 (0.6)

Table 4

Summary of Work Productivity and Activity Impact Questionnaire

	All Subjects		Employed Subjects
	Currently employed (yes), n (%)	How much did health affect non-work during the previous week? (range 0–10), mean (SD)	Hours worked in the previous week, mean (SD)	Hours missed from work due to ill health during previous week, mean (SD)	Hours missed from work due to other reasons during previous week, mean (SD)	How much did health affect productivity during previous week? (range 0–10), mean (SD)
Blepharospasm
Baseline (N = 167)	53 (31.7)	n = 165 3.1 (2.8)	n = 52 34.2 (17.0)	n = 52 1.2 (5.7)	n = 52 5.2 (11.0)	n = 52 1.9 (2.3)
Week 1 (N = 158)	47 (29.7)	n = 150 2.9 (2.9)	n = 47 33.2 (19.5)	n = 47 1.4 (6.3)	n = 47 5.3 (14.2)	n = 43 1.6 (1.7)
Week 2 (N = 154)	47 (30.5)	n = 146 3.1 (3.0)	n = 46 26.6 (17.1)	n = 46 5.2 (19.4)	n = 46 4.3 (10.8)	n = 38 1.5 (2.1)
Week 3 (N = 154)	46 (29.9)	n = 148 2.6 (2.7)	n = 46 30.3 (19.6)	n = 46 1.9 (7.8)	n = 46 4.6 (10.1)	n = 40 1.5 (2.1)
Week 4 (N = 170)	49 (28.8)	n = 159 2.8 (2.8)	n = 49 28.7 (19.2)	n = 49 1.1 (5.9)	n = 49 3.5 (8.9)	n = 41 1.6 (2.0)

[i] Abbreviation: SD, Standard Deviation.

[ii] The productivity and daily activities questions are based on a 10-point scale where 0 = health problems had no effect on daily activity and 10 = health problems completely prevented productivity/activities. Percentages are based on non-missing values.