Promising results: Tacrolimus plus dexamethasone in immune thrombocytopenia

The combination regimen of low-dose tacrolimus plus high-dose dexamethasone provides benefits over high-dose dexamethasone monotherapy in patients with immune thrombocytopenia (ITP), promising first-line treatment for patients with ITP.

First-line therapies with long-term effectiveness are essential

Dr Zhuo-Yu An (Peking University People’s Hospital, China) explained that approximately 30% of adult patients with ITP relapses in the first 6 months after the initiation of a first-line therapy. Therefore, first-line therapies that demonstrate long-term effectiveness are needed for patients with this condition.

The prospective, multicentre, open-label, randomised, phase 2 TARGET 020 trial (NCT04747080) compared high-dose dexamethasone (40 mg, 4 consecutive days with possible repetition after 14 days) (n=56) with high-dose dexamethasone plus low-dose tacrolimus (3–5 ng/mL, 4 consecutive days with possible repetition after 14 days) (n=48).

The primary outcome was sustained response at 6 months, defined as a platelet count ≥30x109/L (partial remission) or ≥100x109/L (complete remission), a 2-fold increase of baseline platelets, and no use of rescue medication at follow-up. The initial response rate was higher in the combination regimen arm (77%) than in the monotherapy arm (55%).

Fewer relapses, and sustained response rates

In addition, patients treated with the combination therapy showed fewer relapses (19%) than patients treated with dexamethasone monotherapy (29%). After 6 months, the sustained response rates were higher in the low-dose tacrolimus plus high-dose dexamethasone treatment group (64.6%) than in the high-dose dexamethasone monotherapy group (41.1%).

The treatment regimens were equally safe and tolerable, displaying no grade 3 or higher adverse events (AEs). The results demonstrate that low-dose tacrolimus plus high-dose dexamethasone is a promising first-line treatment regimen for patients with ITP. However, larger randomised trials are needed to validate the results of the current phase 2 trial.

An ZY, et al. Tacrolimus Plus High-Dose Dexamethasone Versus High-Dose Dexamethasone Alone As First-Line Treatment for Adult Immune Thrombocytopenia: The Phase 2, Open Label, Randomized Trial (TARGET 020). O311, ASH 2021 Scientific Sessions, 11–14 December.