Promising Phase III Trial for Immune Thrombocytopenia Treatment

Immune thrombocytopenia (ITP) is an autoimmune disease where the immune system attacks platelets in the blood, resulting in a low platelet count. Because platelets normally help the blood clot, low platelet counts can lead to significant bruising or bleeding.

Patients with ITP can be treated with steroids, but those that don’t respond to this require a different type of medication. Medications, such as eltrombopag, can help increase platelet counts but often require daily or weekly dosing. 

Novartis recently published the results of a Phase III clinical trial showing that treatment with the monoclonal antibody ianalumab alongside eltrombopag can help extend the “time to treatment failure” compared to eltrombopag alone. The “time to treatment failure” data point can indicate how long a treatment lasts, with longer times indicating that a therapy holds up better. Those treated with both drugs had a time to treatment failure of over one year while those on eltrombopaq had a time to treatment failure of 4.7 months. If approved, this drug can give patients the possibility of reducing the need for chronic treatment.

Treatment with ianalumab decreases the autoreactive B cells that cause ITP and this drug has also been explored to treat other autoimmune diseases.

Citation

Cuker, A., Stauch, T., Cooper, N., Al-Samkari, H., Michel, M., Ghanima, W., Urban, P., Fronczek, J., Foster, M., Weill, M., Zhang, L., Hou, M., Zander, T., Sharif, A., Sun, J., Nath, U. K., Schutgens, R., Rossi, E., Deleu, L., Červinek, L., … VAYHIT2 Investigators (2025). Ianalumab plus Eltrombopag in Immune Thrombocytopenia. The New England Journal of Medicine, 10.1056/NEJMoa2515168. Advance online publication. https://doi.org/10.1056/NEJMoa2515168