Mycophenolate, Corticosteroids as First-Line Treatment for Immune Thrombocytopenia

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.

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.