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Table 8 Change from baseline of liver and spleen volumes at Week 52

From: Neurocognitive and somatic stabilization in pediatric patients with severe Mucopolysaccharidosis Type I after 52 weeks of intravenous brain-penetrating insulin receptor antibody-iduronidase fusion protein (valanafusp alpha): an open label phase 1-2 trial

patient

liver

spleen

baseline

52 weeks

baseline

52 weeks

mL

%BW

mL

%BW

mL

%BW

mL

%BW

202

445.7

2.27

404.4

1.96

76.3

0.39

87.8

0.43

203

546.1

2.86

518.7

2.44

108.9

0.57

107.3

0.50

204

930.0

2.51

800.9

1.75

149.1

0.40

107.6

0.24

205

691.1

2.80

548.8

1.78

174.0

0.70

107.1

0.35

206

570.2

4.92

486.2

3.33

87.7

0.76

75.5

0.52

207

529.3

2.82

483.7

2.27

209.7

1.12

181.1

0.85

211

528.1

4.63

434.2a

3.37

88.0

0.77

58.1a

0.45

213

1273.5

3.31

1198.4

2.65

210.7

0.55

247.1

0.55

214

601.5

4.63

530.6

3.38

139.0

1.07

118.4

0.75

215

702.6

1.77

765.7

1.50

202.9

0.51

202.5

0.40

216

578.6

2.19

642.5

2.09

168.2

0.64

156.4

0.51

mean

672.4

3.15b

619.5

2.41b

146.8

0.68c

131.7

0.50c

SEM

71.8

0.33

69.8

0.21

15.4

0.07

17.6

0.05

  1. a52-week MRI could not be performed; difference computed from 26-week volume
  2. bLiver %BW at 52 weeks is significantly reduced compared to %BW at baseline (P<0.0005)
  3. cSpleen %BW at 52 weeks is significantly reduced compared to %BW at baseline (P<0.005)