The following article features coverage from the American Society of Hematology 2020 meeting. Click here to read more of Hematology Advisor‘s conference coverage.

Mycophenolate in combination with corticosteroids may be efficacious and tolerable as first-line treatment for patients with immune thrombocytopenia (ITP), according to study results presented at the virtual 62nd American Society of Hematology (ASH) Annual Meeting and Exposition.

Although high-dose corticosteroids are currently used as first-line treatment for ITP, the therapy is associated with high rates of side effects, heterogeneous responses, and relapses.  In addition, long-term remission has been reported in only 20% of this patient population. The use of mycophenolate as second-line treatment has been reported to be effective in 50% to 80% of patients with good, but delayed, tolerability.


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A team of UK-based investigators conducted a multicenter, open-label, randomized controlled trial (FLIGHT; ClinicalTrials.gov Identifier: NCT03156452) to determine whether mycophenolate would be more effective as first-line treatment for ITP compared with the current standard of care.

A total of 120 patients (median age, 54 years; 54.2% women) with ITP were randomly assigned (1:1) to receive either combined mycophenolate with corticosteroid (n=59) or corticosteroid treatment alone (n=61). The primary efficacy outcome was time to treatment failure from randomization (defined as platelets <30 x109/L and a clinical need for second-line treatment). Secondary outcomes included side effects, bleeding events, and patient-reported outcomes.

Significantly fewer treatment failures were reported among patients in the mycophenolate cohort compared with patients who only received standard of care (22% vs 44%; adjusted hazard ratio [aHR], 0.41; P =.0064). After excluding patients with secondary ITP (n=13), results were even more favorable for mycophenolate (aHR, 0.37; P =.0029).

Rates of significant adverse events, bleeding events, rescue treatments, hospital admissions, and side effects were similar between the 2 treatment groups; however, quality-of-life factors such as physical role, physical function, and fatigue were worse among patients treated with mycophenolate.

Among patients in the standard-of-care cohort, 56% did not require second-line treatment at the final follow-up visit.

“It is unclear why some aspects of [quality of life] were worse in the [mycophenolate] group,” the authors noted. “This is an important reminder that disease response and patient experience may not correlate and [emphasizes] the importance of including [patient reported] outcomes within trials.”

Read more of Hematology Advisor’s coverage of the ASH 2020 meeting by visiting the conference page.

Reference

Bradbury CA, Greenwood R, Pell J, 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. Presented at: American Society of Hematology (ASH) 62nd Annual Meeting and Exposition; December 5-8, 2020. LBA-2.