Table 3.

Clinical features of patients who were responding to BNZ-1

PatientDose (mg/kg)STAT3 mutationIndicationPrior treatmentBaseline ANCBaseline HgB (g/dL)ANC best responseHgB best responseOverall
response
LTE (Y/N)
L-OSU-004 STAT3; p.Y640F, c.1919 A>T Neutropenia, Infection None (for LGL); abatacept for RA 490 11.5 2230 (D28) 12.1 PR 
L-OSU-006 WT Transfusion-dependent anemia, neutropenia MTX 580 9.2 3850 (D28) 8.4 (stable) PR 
L-UVA-004 WT Transfusion-dependent anemia MTX, Cytoxan+pred, CsA, alemtuzumab 430 7.7 880 (LTE WK 22) 10.5 (LTE WK 9) PR 
L-UVA-007 D661Y
 
Transfusion-dependent anemia MTX 1140 10.4 2810 (WK 10) 10.9 (WK 8) PR 
PatientDose (mg/kg)STAT3 mutationIndicationPrior treatmentBaseline ANCBaseline HgB (g/dL)ANC best responseHgB best responseOverall
response
LTE (Y/N)
L-OSU-004 STAT3; p.Y640F, c.1919 A>T Neutropenia, Infection None (for LGL); abatacept for RA 490 11.5 2230 (D28) 12.1 PR 
L-OSU-006 WT Transfusion-dependent anemia, neutropenia MTX 580 9.2 3850 (D28) 8.4 (stable) PR 
L-UVA-004 WT Transfusion-dependent anemia MTX, Cytoxan+pred, CsA, alemtuzumab 430 7.7 880 (LTE WK 22) 10.5 (LTE WK 9) PR 
L-UVA-007 D661Y
 
Transfusion-dependent anemia MTX 1140 10.4 2810 (WK 10) 10.9 (WK 8) PR 

CsA, cyclosporine; EO; end of treatment; HgB, hemoglobin; MTX, methotrexate; pred, prednisone; RA, rheumatoid arthritis; WT, wild-type.

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