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Charlotte Bradbury: Adding Mycophenolate to Corticosteroid for ITP

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So the flight clinical trial was a randomized open label um multi-center trial comparing uh standard of care which is corticosteroid alone versus corticosteroid plus mycophenolate for first-line treatment of itp and the reason why we wanted to test this approach is that the standard of care of corticosteroid alone has several downsides so not only do most

Patients have side effects from steroids but also there’s a heterogeneity of responses with some patients about 30 percent not responding at all and the other patients most likely to relapse at some point in the future which means that only about 20 percent stay well long-term uh with this approach in the uk we use mycophenolate quite a lot as a second line

Treatment and there’s retrospective data that suggests it has good efficacy and tolerability so the hypothesis was if we treat patients with microphenylate at the beginning um then potentially we can prevent that almost inevitable treatment failure of corticosteroid alone so the primary outcome was time to treatment failure which we defined as a platelet

Count of less than 30 and a clinical need for second line treatment and inc included in the treatment failures were patients refractory to treatment who didn’t respond at all and also patients uh who initially responded but then subsequently relapsed for the combination there were only 6.8 percent who were refractory and overall 22 percent experience of

Treatment failure for um corticosteroids alone it was 44 to experience the treatment failure and there was a significant difference between the two groups so you’re roughly two times as likely to have experienced a treatment failure if you were randomized to corticosteroid alone well interestingly when we looked at side effects uh they were actually really

Similar between the two groups and predominantly were related to corticosteroid treatments so um that would be weight gain um psychological disturbance um and things like that in actual fact the the rates of infections were really similar between the groups as well as the gi side effects so it didn’t seem like mycophenolate was adding uh much in the way of

Side effects which was really interesting particularly as it was a very elderly cohort that were included in the trial i would say that’s a really difficult question and i think it’s very difficult to say that this should now be a routine first line treatment option for itp and that’s in part because over half of the patients treated with corticosteroid

Alone and remained in first remission at follow-up um so potentially had they received microphonolate over half of those may have been over treated and then the other thing is that some aspects of the quality of life appear to be a little bit worse with mycophenolate um and we don’t really understand why and whether that’s because of the elderly uh population

That were included and we just don’t know um so i think the key would be to try and um better stratify patients at diagnosis and try and target mycophenolate potentially for those who would be expected to do poorly on corticosteroid alone

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Charlotte Bradbury: Adding Mycophenolate to Corticosteroid for ITP By American Society of Hematology