Table 3.
Clinical efficacy assessed by investigators
| TGA (1,000 mg) n=15 |
TGB (2,000 mg) n=22 |
Total N=37 |
|
|---|---|---|---|
|
| |||
| Overall response rate to C1* | |||
|
| |||
| PR, n (%) | 3 (20) | 9 (41) | 12 (32) |
| MR, n (%) | 2 (13) | 5 (23) | 7 (19) |
| Overall response rate (MR+PR), n (%) | 5 (33) | 14 (64) | 19 (51) |
| [95% CI] | [11.8–61.6] | [40.7–82.8] | [34.4–68.1] |
|
| |||
| Overall response rate to C1 + RC* | |||
|
| |||
| PR, n (%) | 4 (27) | 11 (50) | 15 (41) |
| MR, n (%) | 3 (20) | 4 (18) | 7 (19) |
| Overall response rate (MR+PR), n (%) | 7 (47) | 15 (68) | 22 (59) |
| [95% CI] | [21.3–73.4] | [45.1–86.1] | [42.1–75.2] |
|
| |||
| Change in IgM levels | |||
|
| |||
| Median IgM change from baseline to nadir (all patients [n=37]), % | −43·6 | −39·1 | −39·8 |
| Median time to IgM nadir (all patients [n=37]), days | 190 | 228 | 218 |
| Median IgM change from baseline to nadir (responders [n=22]), % | −61·0 | −72·2 | −64·0 |
| Median time to IgM nadir (responders [n=22]), days | 351 | 289 | 298 |
|
| |||
| Improvement in haemoglobin | |||
|
| |||
| Baseline Hgb <11·0 g/dL, n | 12 | 14 | 26 |
| ≥3·0 g/dL increase in Hgb, n (%) | 6 (50) | 9 (64) | 15 (58) |
| Normalization of Hgb†, n (%) | 6 (50) | 8 (57) | 14 (54) |
| Median time to reach normal Hgb†, weeks | 25·0 | 20·9 | 20·9 |
*
There were no CRs in this study.
†
Normal Hgb for men: 12·5 g/dL; normal Hgb for women: 11·5 g/dL.
C1=Cycle 1. CI=confidence interval. CR=complete response. Hgb=haemoglobin. IgM=immunoglobin M. MR=minor response. PR=partial response. RC=redosing cycle. TGA=Treatment Group A. TGB=Treatment Group B.