Adding mycophenolate mofetil to a glucocorticoid for first-line treatment of immune thrombocytopenia (ITP) improved treatment response and cut the risk for refractory or relapsed disease, UK research has shown.

For the study, 120 adults with ITP were randomised to receive first-line treatment with either a glucocorticoid (standard care) or a glucocorticoid combined with mycophenolate mofetil, the latter widely being used in the UK in the second-line setting.

The results, published in the NEJM, showed that patients treated with the glucocorticoid/mycophenolate mofetil regimen experienced half the number of treatment failures (defined as a platelet count of less than 30×109 per litre and initiation of a second-line treatment) than those on standard care (22% vs 44%, respectively).

Patients in the combination group were also shown to have a greater response to treatment, with 91.5% showing platelet counts over 100×109 per litre compared to 63.9% of those receiving glucocorticoid monotherapy. 93.2% had platelet counts greater than 30×109 per litre compared with 75.4% in the glucocorticoid-only group.

Also, the researchers found that just 6.8% of patients given mycophenolate mofetil group had ITP that was refractory to treatment, versus 24.6% of patients taking only a glucocorticoid.

However, while there were no differences between the two groups with regard to bleeding, rescue treatments, or treatment side effects, the authors led by Dr Charlotte Bradbury from the Bristol Haematology and Oncology Centre in England did find that those taking the combination regimen were more likely to experience worse quality-of-life outcomes relating to physical function and fatigue than those in the glucocorticoid-only group.

In an accompanying editorial, Consultant Haematologists Dr Paula Bolton-Maggs, from Manchester University, UK and Dr James N. George, the University of Oklahoma Health Sciences Center in Oklahoma City said the study’s quality of life findings serve as “an important reminder that immune thrombocytopenia is not just about the platelet count and that randomised trials can produce unexpected findings that warrant further research”.

The paper’s authors also said further research would help clarify the role of mycophenolate mofetil in ITP treatment pathways.

“For example, mycophenolate mofetil could be used in patients for whom laboratory and clinical markers suggest that a glucocorticoid-only regimen would be expected to fail,” and “early use of mycophenolate mofetil may also be particularly valuable for patients in whom early disease control with avoidance of relapse is a priority”, they noted.

The study was funded by the UK’s National Institute for Health Research.