Autoimmune Cytopenias

At the 63rd Annual Meeting of the American Society of Hematology (ASH), researchers from Peking University People's Hospital, Beijing, China, presented results (abstract 13) from TARGET 020, a multicenter, open-label, randomized, phase 2 trial (ClinicalTrials.gov Identifier: NCT04747080) evaluating the impact of adding tacrolimus to high-dose dexamethasone (HD-DXM) in first-line treatment for adult immune thrombocytopenia (ITP). A total of 140 patients (48.6% males) with newly diagnosed ITP were assigned to 1 of 2 groups: HD-DXM monotherapy (n=68) or HD-TXM plus tacrolimus (n=72). At 6-month follow-up, the proportion of patients exhibiting a sustained response (SR, defined as platelet count maintained higher than 50×109/L without any additional ITP-modifying therapy) was 65.3% in HD-TXM plus tacrolimus group vs 42.6% with HD-DXM monotherapy (P=.007). The 14-day early remission rate was higher with combination therapy vs monotherapy (76.4% vs 55.9%; P=.001) and the rate of treatment failure was twice as high in the monotherapy group (19.4% vs 38.2%, respectively; P=.0014). During follow-up, median time to relapse was 36 days with HD-DXM monotherapy vs 77 days with HD-DXM plus tacrolimus. Patients randomized to combination therapy also had lower bleeding scores, and a smaller proportion experienced bleeding events. The incidence of serious AEs, rescue therapy, and treatment side effects was similar, without any statistically significant differences in AEs between the 2 groups. No grade 4 AEs or treatment-related deaths were reported. Owing to the sustained prolonged response and safety seen with HD-DXM plus tacrolimus, the investigators concluded that this combination "may be a new treatment option for adult patients with ITP."