The addition of mycophenolate (MMF) to corticosteroid yields improved outcomes over corticosteroid alone as a first-line therapy for immune thrombocytopenia (ITP), according to results of the UK-based FLIGHT trial presented at the ASH 2020 Meeting.

“Mycophenolate seems to approximately halve the risk of refractory or relapsed ITP,” said Dr Charlotte Bradbury of the University of Bristol, who presented the results.

“Therefore, mycophenolate may be considered first-line alongside a short course of corticosteroids for some patients with ITP.”

High-dose corticosteroids are currently recommended as first-line treatment, but most patients suffer from toxicities with this approach and about 30% fail to respond at all.

Dr Bradbury said MMF was commonly used as a second-line treatment for ITP in the UK. Prior to the FLIGHT trial there were no randomised trial data on MMF, though retrospective evidence suggested efficacy between 50% and 80%.

In the 120-patient cohort, significantly fewer treatment failures occurred in patients randomised to receive MMF along with a corticosteroid. The treatment failure rate was 22% with the combination regimen, compared with 44% with corticosteroid alone, for an adjusted hazard ratio of 0.41 (P = .0064).

Only 6.8% of those in the MMF group were refractory, meaning they achieved neither a partial or complete response, compared with 24.6% of those in the corticosteroid monotherapy group.

Dr Bradbury noted that after two weeks of treatment the responses were very similar between the groups before eventually favouring MMF, which reflected the drug’s slower mechanism of action.

Side effects were similar between the groups and were generally related to the corticosteroid. The most common side effects included weight gain, difficulty sleeping and mood change. There was no difference with regard to infection or GI side effects, and no patients in the MMF group developed neutropenia. Bleeding rates were also no different between groups, with bleeding episodes reported in 22.0% of MMF patients and in 24.5% of corticosteroid alone patients (P = 0.83).

Patients in the MMF group fared somewhat worse on certain patient-reported outcomes. This included the physical health summary based on the SF36 questionnaire, but Dr Bradbury noted that none of these differences reached statistical significance.

“This is the first randomised controlled trial using MMF to treat ITP, and it shows, we think, very good efficacy and surprising tolerability considering the inclusion of so many elderly patients,” she said. A total of 27.5% of the cohort were over the age of 70, and 15.8% were over 75.

“It’s unclear why some aspects of quality of life seemed a bit worse in the MMF group –  and whether that reflects the slightly older age range recruited, we just don’t know,” she added.

The researchers are now examining the trial data to search for potential biomarkers of response, including the IL-10 to IL-17 ratio. “I don’t believe that this is a first-line treatment for all patients, but I’d like to see in the future … a more individualised approach,” Dr Bradbury said.