Table 2.
Moxetumomab pasudotox was associated with a high response rate and MRD negativity among complete responders
| Parameter | Value (N = 80), ITT population | |
|---|---|---|
| Blinded independent central review | Investigator assessment | |
| Durable CR [primary endpoint], n (%) | 29 (36) | 39 (49) |
| 95% CIa | 26, 48 | 37, 60 |
| CR with HR ≥ 360 days, n (%) | 26 (33) | 36 (45) |
| 95% CIa | 22, 44 | 34, 57 |
| Best overall response | ||
| CR, n (%) | 33 (41) | 42 (53) |
| 95% CIa | 30, 53 | 41, 64 |
| CR and MRD–, n (%)b | 27 (34) | 26 (33) |
| 95% CIa | 24, 45 | 22, 44 |
| CR and MRD + , n (%)b | 6 (8) | 6 (8) |
| 95% CIa | 3, 16 | 3, 16 |
| PR, n (%) | 27 (34) | 21 (26) |
| Objective response rate [CR or PR], n (%) | 60 (75) | 63 (79) |
| 95% CIa | 64, 84 | 68, 87 |
| Median duration of response, months (range) | 66.7 (0+ to 66.7) | 42.1 (0.0 + to 69.0) |
| Median duration of CR, months (range) | 62.8 (0.1+ to 62.8) | 56.6 (0.0 + to 69.0) |
CR was defined as clearing of the bone marrow of hairy cells (determined by routine hematoxylin and eosin stain), radiologic resolution of pre-existing lymphadenopathy and/or organomegaly, and HR. PR was defined as ≥ 50% decrease or normalization (< 500/mm3) in peripheral blood lymphocyte count, reduction of pre-existing lymphadenopathy and/or organomegaly, and HR or 50% improvement in neutrophils, platelets, and hemoglobin over baseline (without transfusions or growth factors for at least 4 weeks)
CI confidence interval, CR complete response, HR hematologic remission, IHC immunohistochemistry, ITT intent-to-treat, MRD minimal residual disease, PR partial response
aTwo-sided CI was calculated using the exact probability method based on the binomial distribution. bDetermined by IHC