First Line Treatment For Newly Diagnosed Immune Thrombocytopenia: Standard Steroid Treatment Versus Combined Steroid And Mycophenolate. The Flight Trial

In the late breaking abstract session at the 2020 American Society of hematology meeting, investigators reported on the multicenter randomized trial of newly diagnosed ITP with combination mycophenolate and steroids.  The FLIGHT trial, was a multicenter, United Kingdom based, open label, randomized control trial where 120 patients with ITP who required first-line treatment were randomized to corticosteroid therapy alone versus combined corticosteroid and mycophenolate.  

Patients were randomized in a one-to-one fashion.  Patients had to be at least 16 years old.  Baseline platelet counts had to be less than 30,000.  Patients with underlying HIV, CVID, pregnancy, breast-feeding or unwillingness to follow contraception advice if assigned to MMF were excluded.  Dosing of corticosteroids following international consensus guidance, either dexamethasone pulses or prednisolone initially dosed at 1 mg/kg then tapered off, and dosing of mycophenolate used a strategy to taper and stop half a year after starting treatment.  The primary efficacy outcome from the time of randomization to treatment failure was defined as a platelet count less than 30,000 as well as a clinical need for second line treatment.  Secondary outcomes included adverse events, patient reported outcomes, bleeding events which were measured at baseline as well as 2, 4, 6 and 12 months through a variety questionnaires.  

Over a 120 patients with a mean baseline platelet count of 7,000 and the mean follow-up of 18 months were enrolled.  Significantly fewer treatment failures occurred in patients randomized to mycophenolate, 22%, 13 of 59 patients versus 44%, 27-61 patients, with a hazard ratio of 0.21.  The P value was 0.0064.  When secondary ITP patients were excluded, hazard ratio of 0.37, with a P value of 0.0029 was confirmed. The adverse events profile was similar between the 2 groups.  Infection was seen in 14 patients in each group, with neutropenia seen in the corticosteroid group while GI side effects were also well documented.

Patients in the mycophenolate arm had a worsening of quality of life.

The authors concluded that mycophenolate may be considered an effective, well-tolerated first-line treatment option, alongside a short course of steroids for some patients with ITP, approximately reducing the risk of refractory or relapsed ITP by 50%.  They also reported that at final follow-up, 56% of patients treated with corticosteroid monotherapy, had not required second line treatment.