A multicenter randomized open-label study of rituximab plus rhTPO vs rituximab in corticosteroid-resistant or relapsed ITP

Abstract

This study aimed to compare the efficacy and safety of rituximab (RTX) plus recombinant human thrombopoietin (rhTPO) with RTX alone in patients with immune thrombocytopenia (ITP) who had failed to respond to corticosteroids or relapsed. Recruited patients were randomized at a ratio of 2:1 into 2 groups: the combination group (RTX + rhTPO, n = 77) and the monotherapy group (RTX, n = 38). Overall response was achieved in 79.2% of patients in the combination group vs 71.1% in the monotherapy group (P = .36), and the complete response (CR) rate was 45.4% in the combination group compared with 23.7% in the monotherapy group (P = .026). The combination group had significantly shorter time to response (TTR; median and range, 7 and 4-28 days) compared with the monotherapy group (28 and 4-90 days) (P < .01). There was no difference between these 2 groups in terms of the long-term response (P = .12). Our findings demonstrated that the combination of RTX and rhTPO significantly increased the CR rate and shortened TTR compared with RTX monotherapy in the treatment of corticosteroid-resistant or relapsed ITP but failed to show a beneficial effect on the long-lasting response. This study is registered at www.clinicaltrials.gov as #NCT01525836.

© 2015 by The American Society of Hematology.

Figures

Figure 1

Flowchart indicating the 2 randomized groups.

Figure 2

Box-whisker plots of platelet counts from ITP patients with initial response (including CR and R). The central horizontal line is the median; the lower and upper box limits are the first and third quartiles, respectively; and the whiskers extend to the minimal and maximal data points.

Figure 3

Kaplan-Meier analysis of patients with an initial response (including CR and R). The Kaplan-Meier curve showed time to relapse with platelet count <30 × 109/L. There was no difference between these 2 groups in terms of proportion of relapse (P = .12).