Q: Can relapsing disease be prevented and remission be induced with medical therapy in patients with chronic cITP?

The evidence is clear that, if upfront treatment is intensified in patients with newly diagnosed ITP (eg, by giving high-dose dexamethasone with rituximab), improved response rates are seen at 6 months, 12 months, and even at 24 months compared with standard first-line therapy alone. The improved response rates seen at 12 months and 24 months suggest the possibility that we could be curing patients, but I also think that it is possible that we may just be delaying relapse. Longer-term observation is needed to answer this question definitively, and the jury is still out. There are data to suggest that up to 30% of patients who are treated with a TPO-RA may be able to come off treatment with a sustained response. The question there is, are the TPO-RAs actually inducing remission in these patients or are we simply seeing the natural history of the disease? Inevitably, are there going to be some percentage of patients who will have spontaneous remissions irrespective of how you treat them? What is needed to answer these questions are randomized controlled trials, ideally of patients with newly diagnosed ITP who are treated either with or without a TPO-RA to see if upfront therapy with a TPO-RA induces long-term responses off treatment.

There are a number of patients to whom you can provide medical therapy that is nontoxic. The treatment can provide long-term maintenance of an adequate platelet count with minimal symptoms, mostly by the TPO-RAs. Also, there are patients who can develop long-term remissions with treatment. Early treatment with TPO-RAs seems to be very responsive. I think, obviously, of Dr Newland’s paper regarding the variability of the effectiveness of the existing therapies for sustained response in chronic adult ITP. We have all seen a few patients like that. The other issue is that we have other immune-modulating agents that, when combined with TPO-RAs, can induce remissions. In addition, agents that have long-term individual reports on them in terms of their use and efficacy in patients who have been refractory to the classic therapy now can be applied earlier and, particularly, in patients who are less likely to have spontaneous remissions. I think that remission can be induced, but there will be relapses. We will continue to safely tell patients that, even if they are off all medications, they are still at risk of relapse. We really do not have a strategy to identify patients who appear to have significant long-term remissions.