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Table 1 Cerebrospinal fluid findings following treatment initiation with nusinersen and tofersen. T1: treatment-naive; T2: month 6 of nusinersen treatment/month 2 of tofersen treatment (after completed loading phases); T3: month 54 of nusinersen treatment/month 9 of tofersen treatment

From: Macrophage inclusions in cerebrospinal fluid following treatment initiation with antisense oligonucleotide therapies in motor neuron diseases

  

Patient 1 – SMA:

Patient 2 – SOD1-ALS:

 

Reference values

T1

T2

T3

T1

T2

T3

Leucocyte count (MPt/L)

< 5

3

4

1

1

3

21

Protein (mg/L)

150–450

249

262

214

483

764

1235

Albumin (mg/L)

< 350

129

137

134

268

558

879

Albumin ratio (x10− 3)

(4 + Age/15)

3.37

3.58

3.41

5.60

12.85

18.65

IgG (mg/L)

IgG synthesis (%)

10–30

negative

14.1

0.0

12.8

0.0

12.53

0.0

44.48

1.5

69.09

0.0

115.63

0.0

IgA (mg/L)

IgA synthesis (%)

1–3

negative

1.29

0.0

1.58

0.0

1.74

0.0

5.81

0.0

10.78

0.0

17.94

0.0

IgM (mg/L)

IgM synthesis (%)

0.5–1.5

negative

< 0.5

0.0

< 0.5

0.0

< 0.12

0.0

0.33

0.0

1.82

0.0

12.26

62.6

OCBs (CSF/Serum)

negative

Type 4

Type 4

Type 1

Type 4

Type 1

Type 4

Lymphocytes (%)

50–90

93

88

97

94

85

85

Monocytes (%)

Macrophages with unspecified inclusions

10–50

none

6

9

+

1

+

3

9

+

10

+

  1. Ig: immunoglobulin; SMA: 5q-associated spinal muscular atrophy;SOD1-ALS: superoxide dismutase 1 mutation-associated amyotrophic lateral sclerosis; OCBs: oligoclonal bands; Type 1: no evidence for intrathecally produced OCBs; Type 4: equal numbers of matched OCBs in CSF and serum