The Combination of ATRA and High-dose Dexamethasone as First-line Treatment in Adult Immune Thrombocytopenia

Phase 2 Completed 132 enrolled Peking University People's Hospital Beijing Aerospace General Hospital · collab Beijing Hospital · collab Beijing Tongren Hospital · collab Navy General Hospital, Beijing · collab Qilu Hospital of Shandong University · collab

RandomizedParallel-groupOpen-labelTreatment

Summary

Randomized, open-label, multicenter study to compare the efficacy and safety of ATRA plus high-dose dexamethasone compared to high-dose dexamethasone monotherapy for the first-line treatment of adults with primary immune thrombocytopenia (ITP).

Timeline

Start: 2020-01-01
Primary completion: 2020-06-30
Completion: 2020-06-30

Outcome

Met primary endpoint

paper 45 [68%] of 66) than in the high-dose dexamethasone monotherapy group (27 [41%] of 66) had a sustained response (OR 3·095, 95% CI 1·516-6·318; p=0·0017). PMID 34560012 ↗

Publications

Huang QS, Liu Y, Wang JB, Peng J, Hou M, Liu H, Feng R, Wang JW, Xu LP, Wang Y, Huang XJ, Zhang XH. All-trans retinoic acid plus high-dose dexamethasone as first-line treatment for patients with newly diagnosed immune thrombocytopenia: a multicentre, open-label, randomised, controlled, phase 2 trial. Lancet Haematol. 2021 Oct;8(10):e688-e699. doi: 10.1016/S2352-3026(21)00240-4.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Dexamethasone	Small molecule	40 mg	Intravenous
Subject	Tretinoin	Unknown	10 mg	Oral