Prof. Zhang Xiaohui’s Team Publishes New Research Results in Blood
On January 20th, 2022, Prof. Zhang Xiaohui’s team from the Department of Hematology at Peking University People’s Hospital and Peking University Institute of Hematology published an article entitled “All-trans retinoic acid plus low-dose rituximab vs low-dose rituximab in corticosteroid-resistant or relapsed ITP” in the top international journal Blood, which was designated as a continuing medical education (CME) article for the journal. In the same issue, Blood published a commentary stating that the findings “further the possibilities of ATRA as a novel treatment of ITP and advance its utilization especially in combination perhaps with dexamethasone as recently reported6 as well as with rituximab". On January 27th, 2022, the NEJM Journal Watch published a review commenting “the authors suggest a novel future combination of ATRA with thrombopoietin agonists, given their potentially synergistic mechanisms of action”.
The study aimed to compare the efficacy and safety of all-trans retinoic acid (ATRA) plus low-dose rituximab (LD-RTX) with LD-RTX monotherapy in corticosteroid-resistant or relapsed immune thrombocytopenia (ITP) patients. Recruited patients were randomized at a ratio of 2:1 into 2 groups: 112 patients received LD-RTX plus ATRA, and 56 patients received LD-RTX monotherapy. Overall response (OR), defined as achieving a platelet count of ≥30 ×109/L confirmed on ≥2 separate occasions (≥7 days apart), at least a doubling of the baseline platelet count without any other ITP-specific treatment, and the absence of bleeding within 1 year after enrollment, was observed in more patients in the LD-RTX plus ATRA group (80%) than in the LD-RTX monotherapy group (59%) (between-group difference, 0.22; 95% CI, 0.07-0.36). Sustained response (SR), defined as maintenance of a platelet count >30 × 109/L, an absence of bleeding, and no requirement for any other ITP-specific treatment for 6 consecutive months after achievement of OR during 1 year following enrollment, was achieved by 68 (61%) patients in the combination group and 23 (41%) patients in the monotherapy group (between-group difference, 0.20; 95% CI, 0.04-0.35). The two most common adverse events (AEs) for the combination group were dry skin and headache or dizziness. The research findings demonstrated that ATRA plus LD-RTX significantly increased the overall and sustained response, indicating a promising treatment option for corticosteroid-resistant or relapsed adult ITP.
MD student Wu Yejun is the first author of the paper. Prof. Zhang Xiaohui and Prof. Huang Xiaojun are the corresponding authors.