Rituximab as first-line treatment for acquired thrombotic thrombocytopenic purpura

Abstract

ObjectiveTo investigate the efficacy and safety of rituximab (RTX) as first-line treatment of acquired thrombotic thrombocytopenic purpura (aTTP).MethodsTwenty-five patients with acute aTTP and/or severe a disintegrin and metalloproteinase with a thrombospondin type 1 motif, member 13 (ADAMTS13) deficiency were admitted to our centre from April 2009 to March 2015. Fourteen patients received RTX plus standard therapy (plasma exchange and corticosteroids) at acute episodes. Haemoglobin, platelet count, schistocytes, lactate dehydrogenase levels, ADAMTS13 activity and its inhibitors, and the ratio of B lymphocytes in the peripheral blood, were monitored. The number of plasma exchange (PEXs), total plasma volume, remission time, relapse ratio, and adverse effects were recorded.ResultsThe median number of PEXs was 5 (2–17) sessions and median total plasma volume was 168.43 ml/kg (62.86–469.52 ml/kg). Patients achieved haematological remission at a median of 15 days (5–22 days), and the median time of immunological remission was 2 weeks (2–8 weeks) with a median follow-up of 13 months (3–61 months). ADAMTS13 activity significantly increased after 2 weeks. The B lymphocyte percentage in peripheral blood was reduced 1 week after the first dose of RTX infusion compared with before treatment (2.21% ± 5.23% vs 18.47% ± 7.34%, P = 0.000 [the result of statistical software]), and began to gradually increase 9 months later. Severe adverse effects and relapsing TTP were not observed during therapy and follow-up. However, one patient who had sustained immunological remission died of severe pneumonia 7 months later.ConclusionAlthough our study was limited by its small sample number and it was a non-controlled, clinical trial, it showed potential benefits of RTX therapy for acute aTTP. RTX may be administered as a first-line therapy for lowering patients’ relapse rate in the long term. Randomized, controlled trials of RTX for aTTP are required.

Preventing infectious complications when treating non‐malignant immune‐mediated hematologic disorders

“…Rituximab is a chimeric monoclonal antibody targeting the B‐cell antigen CD20, approved for various malignant hematologic and rheumatologic disorders. It has shown efficacy in autoimmune hemolytic anemia, acquired coagulation factor deficiencies, thrombotic thrombocytopenic purpura, immune thrombocytopenic purpura (ITP), and has been used in other disorders such as antiphospholipid syndrome, and aplastic anemia, but its use in these non‐malignant immune‐mediated hematologic disorders is off‐label.…”

Efficacy and safety of rituximab in autoimmune and microangiopathic hemolytic anemia: a systematic review and meta-analysis

“…Indeed, there was no records for this in the present meta-analysis. In the epidemiological studies, the average global prevalence of HBV infection is 4.8% whereas the figure is as high as 23.7% in Taiwan, which is among the highest in the Asia-Pacific region [55]. The absence of HBV related AE in the present meta-analysis is conceivable because the included studies were all carried out in Western countries.…”

“…Besides, there were 3 mortalities (0.3%), including one early death from uncontrolled hemolysis [38]. The 2nd fatal patient died because of delayed treatment [55]. The 3rd fatal patient died shortly after RTX therapy, which was also claimed to be partially contributing to the death [42].…”

Positive Response and Increase in ADAMTS13 with Scheduled Rituximab in a Patient with Relapsing Thrombotic Thrombocytopenic Purpura

“…The present case suggests a regimen with good efficacy and minimal side effects that may serve as a template for such a guideline. Continued research will be needed to establish a guideline for treatment [5]. The efficacy of this treatment regimen does offer hope for patients who suffer from TTP and the distress of recurrence.…”