Attainment of personal goals in the first year of intrathecal baclofen treatment in dyskinetic cerebral palsy: a prospective cohort study

Figures & data

Table 1. Patient characteristics at baseline (n = 34).

Sex
Male	23 (67.6%)
Female	11 (32.4%)
Age at pump implantation (years)	14.2 ± 4.2 (7.7–22.7)
Heights (cm)	147.3 ± 20.3 (110.0–181.0)
Weight (kg)	33.8 ± 12.5 (17.0–75.0)
BMI (kg/m^2)	15.2 ± 2.8 (11.2–22.9)
GMFCS level
IV	13 (38.2%)
V	21 (61.7%)
MACS level
III	3 (8.8%)
IV	9 (26.4%)
V	22 (64.7%)
C-BiLLT^a	60 ± 25 (0–86)^b
MRI pattern^c
Basal ganglia/thalamic lesions	22 (64.7%)
Kernicterus/globus pallidus	4 (11.8%)
Multicystic encephalomalacia	1 (2.9%)
Periventricular leukomalacia	7 (20.6%)
MRI grading^c
White matter total score
Mild (7–11)	22 (68.8%)
Moderate (12–16)	5 (15.6%))
Severe (17–21)	5 (15.6%)
Grey matter total score
Mild (6–9)	12 (37.5%)
Moderate (10–14)	20 (62.5%)
Severe (15–18)	0 (0%)

%: percentage; SD: standard deviation; GMFCS: Gross Motor Functioning Classification System, MACS: Manual Ability Classification System; C-BiLLT: Computer-Based instrument for Low Motor Language Testing; BMI: body mass index.

Data are presented as number (%) or mean ± SD (range).

^a Data available for 29 of 34 individuals.

^b Age equivalent of younger than 18 months (pretest not possible = 0) to maximal score of C-BiLLT = 86 (i.e., comprehension of spoken language of older than 8 years).

^c Data available for 32 of 34 individuals, some MRIs show multiple patterns (mostly basal ganglia in combination with periventricular leukomalacia).

Table 2. Treatment characteristics.

Characteristic	Number (%) or mean ± SD (range)
Catheter tip position
Th3 or higher	27 (80%)
Th4–Th9	5 (14.3%)
Th10 or lower	2 (5.7%)
(Serious) Adverse events
Individuals with SAE
Yes	11 (32.3%)
No	23 (65.7%)
Number AE	32
Number SAE	14
Type AE/SAE
CSF leakage AE/SAE	11/2
Pump infection AE/SAE	0/2
Catheter related AE/SAE	0/3
Other AE/SAE^a	21/7
Sleeping-related breathing disorder
No change	23 (63.9%)
Decreased (less risk)	4 (11.1%)
Increase (higher risk)	7 (19.4%)
Dosage ITB at 12 months
Total dosage (µg/day)	258.9 ± 197.0 (72–837)
Use of one or more bolus over the day
Yes	27 (79.4%)
No	7 (20.6%)
Oral anti-spastic or dyskinetic medication change since baseline
Yes
Decreased	15 (44.1%)
Increased	1 (2.9%)
No	18 (44.1%)

%: percentage; SD: standard deviation; CSF: cerebrospinal fluid; ITB: intrathecal baclofen; Th: thoracic level; SAE: serious adverse events; AE: adverse events.

^a Other SAE were infections (pneumonia, gastrointestinal infection), obstipation, apnea, and seizure.

Table 3. Outcomes at baseline and 12 month follow-up.

	Baseline	12-month follow-up	Difference baseline-12 months follow-up^a
Variable	Mean (SD)	Mean (SD)	Mean (95% CI)	p Value	ES
GAS T-scores	23.08 (0.87)	42.58 (14.90)	19.41 (14.33; 24.48)	<0.001*	1.39
DIS DYS rest	0.58 (0.18)	0.49 (0.19)	–0.09 (–0.15; −0.03)	0.003	–0.47
DIS DYS action	0.72 (0.13)	0.67 (0.14)	–0.06 (–0.10; −0.02)	0.007	–0.36
DIS DYS total	0.66 (0.14)	0.59 (0.16)	–0.07 (–0.11; −0.03)	<0.001*	–0.47
DIS CA rest	0.16 (0.17)	0.11 (0.14)	–0.04 (–0.08; −0.01)	0.017	–0.27
DIS CA action	0.21 (0.18)	0.12 (0.16)	–0.07 (–0.12; −0.03)	0.006	–0.39
DIS CA total	0.17 (0.16)	0.12 (0.15)	–0.06 (–0.09; −0.02)	0.002*	–0.38
DIS total	0.42 (0.11)	0.35 (0.11)	–0.07 (–0.10; −0.04)	<0.001*	–0.64
BADS	20.22 (6.51)	16.94 (6.34)	–3.44 (–5.57; −1.30)	0.002*	–0.56
H/M ratio	0.41 (0.28)	0.20 (0.16)	–0.16 (–0.24; −0.08)	<0.001*	–0.70
SPAT score	0.48 (0.37)	0.47 (0.32)	0.00 (–0.09; 0.09)	0.975	0.00
Pain score	4.28 (2.54)	3.18 (2.42)	–1.04 (–2.07; 0.01)	0.049	–0.41
Comfort score (caregiver)	5.97 (2.72)	6.27 (2.11)	0.23 (–0.85; 1.34)	0.650	0.1
Comfort score (child)	4.42 (1.39)	5.06 (0.98)	0.59 (0.12; 1.05)	0.083	0.49
PEDI mobility	10.83 (9.32)	11.53 (9.61)	0.50 (–0.07; 1.06)	0.028	0.05
PEDI self-care	11.81 (10.80)	12.26 (10.47)	0.87 (0.27; 1.47)	0.005	0.08
PEDI social functioning	36.58 (18.21)	37.65 (17.80)	1.32 (0.67; 1.96)	<0.001*	0.07

ES: effect size (Cohen's d); SD: standard deviation; GAS: Goal Attainment Scaling; DIS: Dyskinesia Impairment Scale; DYS: dystonia; CA: choreo-athetosis; BADS: Barry-Albright Dystonia Scale; H/M ratio: ratio of the soleus Hoffmann reflex (H reflex) to M wave (H/M ratio); SPAT: spasticity test; PEDI: Pediatric Evaluation of Disability Inventory.

Outcomes are derived from model estimates with correction for possible covariates.

* Significant difference with the Holm correction of p value accounting for multiple comparison.

^a Mixed linear model analysis.

Figure 1. Patient outcomes on Goal Attainment Scaling. For the outcome on the Goal Attainment Scale (GAS), improvement is defined as goal being partially achieved, achieved, more than achieved or much more than achieved. Other possible GAS outcomes are goal being unchanged or deterioration.

Figure showing outcomes for Goal Attainment Scaling. Most patients (70.59%) show improvement on two or more goals, 11.76% show improvement on two goals and deterioration on the 3rd goal, and 2.94% show deterioration on one or two goals without any improvement on the other goals. The other 14.7% accounts for improvement on one goal and either unchanged remaining goals (2.94%) or deterioration on one or two remaining goals (both 5.88%).

Table 4. Goal attainment at 12 month follow-up.

ICF-component	Subcategory	Number of goals
		Deterioration	Baseline score, no change	Partially achieved	Goal achieved	More than goal achieved	Much more than goal achieved	Total (%)
Body functions and structures	Comfort/pain/fatigue	0	0	3	3	1	1	8 (8.3%)
	Sleeping	1	2	2	0	0	0	5 (5.2%)
	Weight gain	0	0	1	0	0	0	1 (1.0%)
	Total	1	2	6	3	1	1	14 (14.6%)
Activity and participation
Mobility	Changing and maintaining body position^a	2	0	4	1	2	1	10 (10.4%)
	Moving around using equipment^b	3	1	2	2	0	3	11 (11.5%)
	Hand and arm use	0	3	3	0	5	0	11 (11.5%)
	Total	5	4	9	3	7	4	32 (33.3%)
Communication	Communication^c	3	0	1	2	0	0
	Total	3	0	1	2	0	0	6 (6.3%)
Environmental factors	Dressing	2	3	8	6	5	2	26 (27.1%)
	Diaper change	0	1	4	5	2	0	12 (12.5%)
	Washing/bathing	1	0	1	1	0	1	4 (4.2%)
	Caregiving general	0	0	0	0	2	0	2 (2.1%)
	Total	3	4	13	12	9	3	44 (45.8%)
Total number of goals (%)		12 (12.5%)	10 (10.4%)	29 (30.2%)	20 (20.8%)	17 (17.7%)	8 (8.3%)	96 (100%)
Number of subjects		9 (26.5%)	10 (29.4%)	23 (67.6%)	14 (41.2%)	13 (38.2%)	7 (20.6%)	33 (100%)

^a For example, standing, sitting, transfers.

^b For example, walking, operating electric wheelchair.

^c For example, use of communication device.