Safety and efficacy of rituximab in adult immune thrombocytopenia: results from a prospective registry including 248 patients

Abstract

Key Points• Tolerance of rituximab is acceptable in ITP, and with its benefit/risk ratio may remain a valid option for treating ITP in adults.We conducted a prospective multicenter registry of 248 adult patients with immune thrombocytopenia (ITP) treated with rituximab to assess safety. We also assessed response and predictive factors of sustained response. In total, 173 patients received 4 infusions of 375 mg/m 2 and 72 received 2 fixed 1-g infusions 2 weeks apart. The choice of the rituximab regimen was based on the physician's preference and not patient characteristics. Overall, 38 patients showed minor intolerance to rituximab infusions; infusions had to be stopped for only 3 patients. Seven showed infection (n 5 11 cases), with an incidence of 2.3 infections/100 patient-years. Three patients died of infection 12 to 14 months after rituximab infusions, but the role of rituximab was questionable. In total, 152 patients (61%) showed an overall initial response (platelet count ‡30 3 10 9 /L and ‡2 baseline value). At a median follow-up of 24 months, 96 patients (39%) showed a lasting response. On multivariate analysis, the probability of sustained response at 1 year was significantly associated with ITP duration <1 year (P 5 .02) and previous transient complete response to corticosteroids (P 5 .05). The pattern of response was similar with the 2 rituximab regimens. With its benefit/risk ratio, rituximab used off-label may remain a valid option for treating persistent or chronic ITP in adults. This trial was registered at www.clinicaltrials.gov as #NC1101295. (Blood. 2014;124(22):3228-3236)

How we manage immune thrombocytopenia in the elderly

“…It has been reported that younger age was associated with better response (Khellaf et al, 2014). A meta-analysis of studies of non-splenectomized patients receiving rituximab confirmed that younger age was the most relevant predictive factor of response in multivariate analysis (Auger et al, 2012).…”

“…A meta-analysis of studies of non-splenectomized patients receiving rituximab confirmed that younger age was the most relevant predictive factor of response in multivariate analysis (Auger et al, 2012). Rituximab may be associated with better response in the early stages of the disease (Khellaf et al, 2014). Some studies reported that the response could be better in females (Godeau et al, 2008;, but conflicting results have been reported (Auger et al, 2012;Patel et al, 2012;Khellaf et al, 2014).…”

“…Rituximab in ITP leads to an overall initial response rate of 60%, and 30-40% after 2 years of follow-up (Arnold et al, 2007;Godeau et al, 2008;Auger et al, 2012;Khellaf et al, 2014). It has been reported that younger age was associated with better response (Khellaf et al, 2014).…”

The European Hematology Association Roadmap for European Hematology Research: a consensus document

“…221 New innovative therapies have been successfully introduced and have questioned current therapeutic strategies and stimulated expert discussions. 222,223 Despite the relative rarity of FNAIT, several important advances have been made in the past ten years. Firstly, new diagnostic methods, including fetal genotyping on maternal blood samples and antibody detection using aptamers or recombinant peptides, have been developed and are gradually being introduced into clinical practice.…”

A Case of Pregnancy With Chronic Itp Managed With Ivig: A Report

“…Of the available therapies, rituximab was not used as it is slow to act and also, there is a high risk of infections 7 . Of the two available pulse therapies (methylprednisolone and IVIg), IVIg was preferred as it has shown superior efficacy to raise platelet counts in the short term and, it has a better residual effect to maintain the platelet levels 8 .…”