The following is a summary of “Safety and efficacy of baricitinib in steroid-resistant or relapsed immune thrombocytopenia: An open-label pilot study,” published in the July 2024 issue of Hematology by Zhao et al.
Baricitinib, a Janus-associated kinases (JAK) inhibitor, is being explored as a treatment for steroid-resistant immune thrombocytopenia (ITP) due to its potential to improve immune function without worsening platelet levels.
Researchers conducted a retrospective study to explore the safety and efficacy of baricitinib in treating ITP.
They enrolled adults with primary ITP who had not responded to corticosteroids and one additional treatment and had platelet counts below 30 × 109/L at the start. Participants consumed approximately 4 mg of baricitinib daily for 6 months.
The results showed that at the 6-month follow-up, 20 out of 35 enrolled patients (57.1%, 95% CI 39.9 to 74.4) achieved a durable response. Initial response was seen in 23 patients (65.7%). Who responded to baricitinib, the median time to respond was 12 days (IQR 6–20), and the median peak platelet count was 94 × 109/L (IQR 72–128). Additionally, 12 out of 27 patients (44.4%) remained responsive for approximately 20 weeks after stopping baricitinib. The AEs occurred in 11 patients (31.4%), with infections in 6 patients (17.1%) during treatment. Two patients (5.7%) discontinued treatment due to AEs.
Investigators concluded that baricitinib showed promise as a potential treatment for ITP, but further studies will be needed to confirm safety, effectiveness, and optimal dosage.