Real world data of a German Parkinson’s disease population: effectiveness and safety of safinamide in routine clinical practice

Figures & data

Table 1. Demographic and clinical characteristics of patients in the total cohort and in subgroups of interest.

		FAS (N = 181)	Patients aged > 75 years (N = 52)	Patients with relevant comorbidities (N = 153)	Patients with psychiatric conditions (N = 69)
Sex (N, %)	Male	128 (70.7%)	37 (71.2%)	108 (70.6%)	42 (60.9%)
	Female	53 (29.3%)	15 (28.8%)	45 (29.4%)	27 (39.1%)
Age at enrolment (years), mean (SD)		69.1 (8.7)	79.7 (3.1)	69.5 (8.8%)	67.2 (9.5)
Race (N, %)	Caucasian	181 (100.0%)	52 (100.0%)	153 (100.0%)	69 (100.0%)
Diagnosis (N, %)	Idiopathic PD	181 (100.0%)	52 (100.0%)	153 (100.0%)	69 (100.0%)
	Atypical Parkinsonism	0 (0.0%)	0 (0.0%)	0 (0.0%)	0 (0.0%)
Time from PD diagnosis (y): mean (SD)		7.2 (5.3)	8.1 (6.0)	7.1 (5.3)	6.8 (5.1)
Time from PD onset of symptoms (years):		8.8 (6.6)	10.2 (7.5)	8.8 (6.8)	7.9 (5.3)
mean (SD)
Age at onset of symptoms (years): mean (SD)		59.8 (10.5)	68.8 (8.4)	60.2 (10.8)	58.8 (10.6)
Hoehn & Yahr stage: mean (SD)		2.5 (0.8)	2.9 (0.9)	2.6 (0.8)	2.7 (0.9)
N(%)	1	5 (3.4%)	1 (2.6%)	4 (3.1%)	2 (3.4%)
	2	80 (54.1%)	12 (30.8%)	67 (52.3%)	28 (48.3%)
	3	46 (31.1%)	16 (41.0%)	41 (32.0%)	18 (31.0%)
	4	15 (10.1%)	8 (20.5%)	14 (10.9%)	8 (13.8%)
	5	2 (1.4%)	2 (5.1%)	2 (1.6%)	2 (3.4%)

Abbreviations. FAS, Full Analysis Set; N, number of patients; SD, standard deviation; PD, Parkinson’s disease.

Percentages (%) were computed by column.

Figure 1. Parkinson’s disease symptoms at baseline.

Table 2. Concomitant PD treatments at the start of safinamide.

Categories	Total number of evaluable patients (FAS) (N = 181)
Levodopa	181 (100.0%)
Dopamine agonists	106 (58.6%)
COMT inhibitors	76 (42.0%)
Amantadine	40 (22.1%)
Anticholinergics	3 (1.7%)
MAO-B inhibitors	0 (0.0%)
Deep brain stimulation	10 (5.5%)

Abbreviations. PD, Parkinson’s disease; FAS, Full Analysis Set; N, number of patients; COMT, catechol-o-methyltransferase; MAO-B, monoamine-oxidase B.

Percentages are computed out of the total number of patients evaluable for FAS. A patient could have more than one PD treatment.

Table 3. AEs and SAEs overview in the FAS and relevant subgroups.

	Total number of evaluable patients (FAS)	Aged 75+ years	Relevant comorbidities	Psychiatric conditions
	(n = 181)	(n = 52)	(n = 153)	(n = 69)
By patients	N (%)	N (%)	N (%)	N (%)
Patients with at least one adverse event (AE) (N, %)	113 (62.4%)	34 (65.4%)	99 (64.7%)	42 (60.9%)
Patients with at least one serious adverse event (SAE) (N, %)	29 (16.0%)	13 (25.0%)	26 (17.0%)	15 (21.7%)
Patients with at least one adverse drug reaction (ADR) (N, %)	46 (25.4%)	17 (32.7%)	40 (26.1%)	16 (23.2%)
Patients with at least one serious adverse drug reaction (SADR) (N, %)	5 (2.8%)	3 (5.8%)	5 (3.3%)	4 (5.8%)
By events
Total number of adverse events (N)	330	106	306	127
AE with definite  relationship to safinamide (N, %)	5 (1.5%)	1 (0.9%)	5 (1.6%)	1 (0.8%)
Total number of serious adverse events (N, %)	44 (13.3%)	24 (22.6%)	41 (13.4%)	27 (21.3%)
Total number of adverse drug reactions (N)	76	26	69	30
Total number of serious adverse drug reaction (N)	7	4	7	6

Table 4. Fluctuations at the start of treatment with safinamide and during the follow-up.

	Total number of evaluable patients (FAS) (N = 181)	Total evaluable patients at 4 months (N = 160)	Total evaluable patients at 8 months (N = 147)	Total evaluable patients at 12 months (N = 149)	Improvement^a
Motor complications	N (%)	N (%)	N (%)	N (%)	%
Any	181 (100.0%)	128 (80.0%)	115 (78.2%)	106 (71.1%)	41.4
Wearing-off	142 (78.5%)	94 (58.8%)	86 (58.5%)	82 (55.0%)	42.3
Unpredictable fluctuations	17 (9.4%)	9 (5.6%)	5 (3.4%)	10 (6.7%)	41.2
Early morning fluctuations	60 (33.1%)	40 (25.5%)	40 (27.2%)	36 (24.2%)	40.0
Delayed on fluctuation	9 (5.0%)	3 (1.9%)	3 (2.0%)	4 (2.7%)	55.6
Dyskinesia	50 (27.6%)	34 (21.3%)	25 (17.4%)	22 (14.8%)	56.0
Other	8 (4.4%)	3 (1.9%)	6 (4.1%)	3 (2.0%)	62.5

Abbreviations. FAS, Full analysis Set; N, number of patients.

Motor evaluation was done by UPDRS III (UPDRS scores were summarized at each time point). Dyskinesia was part of that assessment.

Percentages were computed out of the number evaluable for the FAS and for each follow-up visits.

^aPercentage difference in rate of patients with motor complications between baseline and end of study.

Table 5. Distribution of adverse events (AE) severity by system organ class.

System organ class	Any AE (N = 330)	Mild AE N = 177 (53.6%)	Moderate AE N = 116 (35.2%)	Severe AE N = 37 (11.2%)
Nervous system disorders	80 (24.2)	49 (14.8)	29 (8.8)	2 (0.6)
Psychiatric disorders	51 (15.5)	27 (8.2)	20 (6.1)	4 (1.2)
Gastrointestinal disorders	19 (5.8)	11 (3.3)	7 (2.1)	1 (0.3)
Injury, poisoning and procedural complications	29 (8.8)	9 (2.7)	11 (3.3)	9 (2.7)
Musculoskeletal and connective tissue disorders	28 (8.5)	16 (4.8)	11 (3.3)	1 (0.3)
General disorders	28 (8.5)	16 (4.8)	10 (3.0)	2 (0.6)
Infections and infestations	14 (4.2)	5 (1.5)	5 (1.5)	4 (1.2)
Eye disorders	8 (2.4)	7 (2.1)	1 (0.3)	0 (0.0)
Vascular disorders	11 (3.3)	5 (1.5)	5 (1.5)	1 (0.3)
Respiratory, thoracic and mediastinal disorders	14 (4.2)	8 (2.4)	3 (0.9)	3 (0.9)
Skin and subcutaneous tissue disorders	12 (3.6)	9 (2.7)	2 (0.6)	1 (0.3)
Renal and urinary disorders	4 (1.2)	1 (0.3)	2 (0.6)	1 (0.3)
Cardiac disorders	5 (1.5)	1 (0.3)	1 (0.6)	2 (0.6)
Metabolism and nutrition disorders	6 (1.8)	4 (1.2)	2 (0.6)	0 (0.0)
Investigations	5 (1.5)	4 (1.2)	1 (0.3)	0 (0.0)
Neoplasms benign, malignant and unspecified	6 (1.8)	1 (0.3)	1 (0.3)	4 (1.2)
Surgical and medical procedures	2 (0.6)	0 (0.0)	2 (0.6)	0 (0.0)
Ear and labyrinth disorders	3 (0.9)	3 (0.9)	0 (0.0)	0 (0.0)
Blood and lymphatic system disorders	1 (0.3)	0 (0.0)	0 (0.0)	1 (0.3)
Reproductive system and breast disorders	2 (0.6)	0 (0.0)	1 (0.3)	1 (0.3)
Endocrine disorders	0 (0.0)	0 (0.0)	0 (0.0)	0 (0.0)

Values are N (%).

Figure 2. Summary for AE and SAE.

Table 6. UPDRS scores.

	UPDRS scores (SD)				N (%) patients with clinically relevant improvement
	Baseline		12 months
	N	Mean (SD)	N	Mean (SD)
UPDRS part III	104	24.5 (12.8)	82	20.3 (10.9)	36 (45.0%)
UPDRS total score	104	37.8 (18.1)	82	33.4 (17.0)	40 (50.0%)

Abbreviations. SD, standard deviation; UPDRS, Unified Parkinson’s Disease Rating Scale.

Clinically relevant improvement is defined as difference > 4.3 points for the UPDRS Total Score (subscales I, II, III) and > 2.5 points for the UPDRS Part III (Motor Examination Score) according to the criteria developed by Shulman et al.²⁵

Shulman LM, Gruber-Baldini AL, Anderson KE, et al. The clinically important difference on the unified Parkinson’s disease rating scale. Arch Neurol. 2010;67(1):64–70. doi: 10.1001/archneurol.2009.295.

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Data availability statement

The raw data supporting the conclusions of this article will be made available by the authors, without undue reservation upon request.