Immunosuppressant-Glucocorticoid Combo Increases Platelets in Immune Thrombocytopenia

(Reuters Health) – In patients with immune thrombocytopenia, the immunosuppressant mycophenolate mofetil, when added to a glucocorticoid, can increase platelet counts and reduce the number of treatment failures compared with glucocorticoid therapy, a new study of 120 volunteers in the United Kingdom has concluded.

But the double-therapy did not appear to affect the risk of bleeding, infection, the need for rescue treatments or the odds of mental health problems.

And quality of life metrics that measured physical function and fatigue showed poorer outcomes among mycophenolate mofetil recipients.

The results of the multicenter open-label FLIGHT trial appear in The New England Journal of Medicine.

Immune thrombocytopenia usually shows up as bruising and bleeding due to a low platelet count. It can produce severe fatigue. Because the immune system is turning on the body, production of platelets is throttled down and consumption of platelets is increased.

Standard treatment with high-dose glucocorticoids carries its own troubling side effects such as mood swings, weight gain, diabetes, insomnia, and high blood pressure.

Only about 20% have a long-term remission with glucocorticoids and up to 30% of patients have no response. But doctors don’t have a lot of other options.

Mycophenolate mofetil is a second-line therapy. The FLIGHT researchers wanted to see if the combination would offer better results.

All the volunteers had a platelet count of less than 30 x 10 to the 9th platelets per liter. Women who might become pregnant were required to use some type of contraception because of mycophenolate mofetil’s associations with miscarriage and birth defects. Prednisolone was the most common glucocorticoid used.

After a minimum of 12 months, the primary outcome — a lower platelet count and initiation of a second-line treatment — was reached in 22% of the mycophenolate mofetil group compared with 44% with a glucocorticoid alone (P=0.008).

Adding mycophenolate mofetil did not seem to add to the side effect profile expected with glucocorticoid therapy but it did produce greater fatigue, poorer physical health and less satisfaction among patients.

“The reasons for patient-reported aspects of quality of life appearing less favorable among patients receiving mycophenolate mofetil are unclear,” said the research team, led by Charlotte Bradbury, University of Bristol. “The quality-of-life differences do not seem to be explained by specific side effects of mycophenolate mofetil, such as infection or diarrhea. Possible reasons include the length of treatment with mycophenolate mofetil, with a potential psychological effect related to treatment duration. The open-label design of the FLIGHT trial is another potential limitation.”

Dr. Bradbury did not respond to questions about the study.

SOURCE: The New England Journal of Medicine, online September 1, 2021.

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