All-Trans Retinoic Acid With High-Dose Dexamethasone in Immune Thrombocytopenia

In an article published in The Lancet Haematology, investigators showed that “the combination of all-trans retinoic acid and high-dose dexamethasone was safe and active in newly diagnosed patients with primary immune thrombocytopenia, providing a sustained response,” supporting it as a potentially more effectiveness first-line treatment than dexamethasone alone for patients with immune thrombocytopenia.

The randomized clinical trial, led by Qiu-Sha Huang, included 129 patients who received either all-trans retinoic acid (10 mg orally twice a day for 12 weeks) plus high-dose dexamethasone (40 mg intravenously on four consecutive days), or high-dose dexamethasone alone. The primary endpoint was six-month sustained response, with response defined as a sustained platelet count of ≥30 × 10⁹ platelets/L that was at least twice the patient’s baseline, without bleeding events or rescue medication.

According to the authors, a significantly higher proportion of patients in the all-trans retinoic acid plus high-dose dexamethasone group achieved a sustained response at month six compared to in the dexamethasone alone group (68% vs. 41%; odds ratio [OR] = 3.095; 95% confidence interval [CI], 1.516–6.318; p = 0.0017). Regarding adverse events, dry skin (48%), headaches (19%), and insomnia (19%) were the most common adverse events in the combined regimen group, while insomnia (15%) and anxiety or mood disorders (12%) were the most common in the monotherapy group. Overall, the authors stated that both treatments were well-tolerated, with no grade IV or higher adverse events or treatment-related deaths.

Ultimately, the authors advanced that “this regimen represents a potential first-line treatment in this setting,” though they did acknowledge that “further studies are needed to validate its efficacy and safety.”