Highlights from

ASH 2020

62nd Annual Meeting & Exposition of the American Society of Hematology

Virtual 5 - 8 December 2020

First randomised trial using mycophenolate to treat immune thrombocytopenia

FLIGHT is the first randomised trial using mycophenolate to treat immune thrombocytopenia (ITP), demonstrating good efficacy and tolerability, even with the inclusion of elderly patients. This treatment seemed to approximately halve the risk of refractory or relapsed ITP. Therefore, mycophenolate may be considered an effective, well tolerated first-line treatment option, alongside a short course of steroids, for some patients with ITP.

The currently recommended first-line treatment for ITP is use of high-dose corticosteroids. However, this therapy is limited by frequent side effects, heterogeneous responses, and high relapse rates. In the UK, mycophenolate is a commonly used second-line treatment for ITP. Although there are no trial data in ITP, there is retrospective evidence suggesting that mycophenolate is effective in 50-80% of patients and is well tolerated. Mycophenolate is a generic drug, which means that it is cheaper than many other second-line treatment options.

The open-label randomised controlled FLIGHT trial tested the hypothesis that mycophenolate combined with corticosteroids is a more effective first-line treatment than current standard of care, i.e. corticosteroids alone [1]. In 120 included patients, significantly fewer treatment failures occurred in patients randomised to mycophenolate (22% vs 44%, adjusted H =0.41; P=0.0064). With mycophenolate, significantly more patients responded and less were refractory. Interestingly, 2 weeks after randomisation, the responses were very similar in both treatment groups, reflecting the slower mechanism of action of mycophenolate. At the final follow-up, 56% of patients treated with corticosteroids alone had not required second-line treatment, which is higher than previous reports.

The results of FACT TH6 and ICECAP, questionnaires about bleeding and quality of life, respectively, were quite similar in both treatment groups. Self-reported quality of life was slightly worse in patients assigned to the mycophenolate group, but the reasons are unclear. This is an important reminder that disease response and patient experience may not correlate and emphasises the importance of including patient-related outcomes within clinical trials.

  1. Bradbury CA, et al. A Multicentre Randomised Trial of First Line Treatment Pathways for Newly Diagnosed Immune Thrombocytopenia: Standard Steroid Treatment Versus Combined Steroid and Mycophenolate. the Flight Trial. 62nd ASH Annual Meeting, December 5-8, 2020. Abstract LBA-2.

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