Clinicians treating patients with immune thrombocytopenia (ITP) should be “ruthless” about reducing use of steroids, an international haematology meeting has heard.
While corticosteroids still have a role, moving to second-line therapies more quickly and taking an individualised approach with additional first-line treatments was key, Dr Charlotte Bradbury, consultant haematologist at University Hospitals Bristol, told the British Society of Haematology (BSH) 2022 conference.
She noted there are trade-offs between higher response rates seen with some additional therapies such as rituximab or mycophenolate (MMF) and the downsides of adding in more treatments.
But she also pointed out that one issue that the trials to date have not dealt with very well is taking into account patient reported outcome measures.
There are several downsides with high-dose steroids first line, including that the vast majority of patients have side effects with more than a third having to stop treatment, around 30% of patients not responding at all, and high rates of relapse among others. Only around one in five patients remains in long-term remission with this approach, Dr Bradbury said.
Recently-updated guidelines have shifted emphasis to trying to avoid prolonged high-dose steroids, Dr Bradbury said.
There is a real interest in answering whether providing more effective treatment earlier-on can produce more sustained effects in the long-term, she added.
When it comes to steroids the key message should be “less is more”, she concluded.
It means “keeping really ruthlessly to the shorter course and then choosing alternative second-line treatments rather than going for further doses of steroids if patients relapse”.
An analysis of real-world management of ITP patients in the UK during the pandemic found that the majority had a steroid but more than 40% of patients had a thrombopoietin receptor agonist alone as first-line treatment.
It was not randomised but there was an 83% response rate, she told delegates — higher than the steroid group.
The RODEX Phase 3 study of romiplostim, which will start to recruit later this year, will hopefully provide clearer answers on that approach, including on whether treatment responses are sustained, she noted.
Results from the FLIGHT trial of MMF plus steroid showed half the rate of relapse of steroids alone and a much higher response rate but patients in the MMF group had reported more fatigue which was unexpected, she said.
“We have to think about individualised therapy and again, I don’t think we should be using MMF for everybody up-front but there may be some patients who are particularly high-risk of relapse or refractoriness would be really consequential where augmenting therapy up front makes sense.”
“For example, using rituximab early if they have other autoimmune conditions.”