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Table 3 Effect summary for targeted therapy

From: Efficacy of tocilizumab therapy in a patient with severe pancytopenia associated with a STAT3 gain-of-function mutation

Cause of targeted therapy

Case number

Remission number

No response

Proportion of remission

enteropathy

5

3

2

60%

ILD

5

4

1

80%

arthritis

3

3

0

100%

cytopenia

3

3

0

100%

hepatosplenomegaly

2

2

0

100%

dermopathy

2

1

1

50%

hepatitis/liver failure

1

0

1

0%