Table 3.

Mean change from baseline under therapy (last available versus baseline values)

	SMA	N	Observ. period Mean (months)	Baseline Meana	Changeb Meana	p value
CSF
WBC (/µL)	2	12	20.7	2.4	+ 0.2	0.51
	3	34	17.8	2.4	− 0.4	0.50
	Total	46	18.5	2.4	− 0.2	1.00
Glucose (mg/dL)	2	12	20.7	62.6	− 1.7	0.75
	3	34	17.6	65.7	− 2.1	0.04
	Total	46	18.4	64.9	− 2.0	0.04
Lactate (mmol/L)	2	12	20.7	1.33	+ 0.10	0.18
	3	34	17.6	1.57	+ 0.01	0.69
	Total	46	18.4	1.51	+ 0.03	0.23
Protein (mg/dL)	2	12	20.7	33.2	+ 3.8	0.75
	3	34	17.6	41.9	+ 2.6	0.01
	Total	46	18.4	39.6	+ 2.9	0.01
Blood
WBC (/nL)	2	13	17.3	7.2	+ 0.1	0.58
	3	35	17.4	7.1	− 0.2	0.74
	Total	48	17.4	7.1	− 0.1	1.00
Platelets (/nL)	2	13	17.3	315.2	− 39.0	0.27
	3	35	17.4	265.9	+ 2.7	0.86
	Total	48	17.4	279.3	− 8.6	0.77
INR	2	12	18.2	1.02	+ 0.01	0.75
	3	13	12.0	1.03	+ 0.01	1.00
	Total	25	15.0	1.02	+ 0.01	0.83
aPTT (s)	2	12	18.2	28.8	− 2.1	0.04
	3	13	12.0	28.1	− 0.7	0.09
	Total	25	15.0	28.4	− 1.4	0.004
Crea (mg/dL)	2	13	16.7	0.31	− 0.04	0.15
	3	33	17.9	0.49	− 0.11	< 0.0001
	Total	46	17.5	0.44	− 0.09	< 0.0001
Urea (mg/dL)	2	13	16.7	8.8	+ 1.5	0.27
	3	33	17.8	11.9	− 0.5	0.58
	Total	46	17.4	11.0	+ 0.0	1.00
AST (U/L)	2	13	16.3	21.0	− 1.6	0.09
	3	33	17.8	28.5	+ 1.2	1.00
	Total	46	17.4	26.4	+ 0.4	0.46
ALT (U/L)	2	13	16.3	31.5	− 4.7	1.00
	3	33	17.8	47.7	− 1.6	0.22
	Total	46	17.4	43.2	− 2.5	0.36
GGT (U/L)	2	13	16.3	36.1	+ 3.5	1.00
	3	33	17.8	43.7	− 6.2	0.49
	Total	46	17.4	41.5	− 3.5	0.46

CSF cerebrospinal fluid, SMA spinal muscular atrophy, WBC white blood cell count, INR international normalized ratio, aPTT activated partial thromboplastin time, crea creatinine, urea urea nitrogen, AST aspartate aminotransferase, ALT alanine aminotransferase, GGT gamma-glutamyltransferase, n number of patients with at least one baseline and one follow-up sample, observ. period observation period

^aUnits as specified in the first column

^bFor each patient, the last measured value was compared with the respective baseline value