Efficacy and Safety of DAPSone as a Second-line Option in Adult Immune Thrombocytopenia

Summary

Due to its expected efficacy based on the retrospective data available in ITP, its relatively good safety profile and its very low cost , dapsone could be a good steroid-sparing second-line option for adults with ITP. This study is a phase III prospective multicenter randomized open trial comparing two treatment strategies: * Arm A (experimental arm): prednisone at 1 mg/kg for 3 weeks + dapsone at 100 mg per day up to week 52 if an initial response is achieved. * Arm B (control arm): prednisone alone at 1 mg/kg for 3 weeks followed by monitoring and "standard of care" The aim of the study is to demonstrate the efficacy of dapsone based on the overall response rate (including response and complete response) as a second-line treatment for adults with newly-diagnosed persistent or chronic (modified by amendment 08/11/2016) ITP not achieving a durable response with corticosteroids. The primary endpoint will be the overall response-rate (response or complete response according to standard definitions) in both arms at week 52 (1 year). The secondary endpoints are the following : * To assess the safety of dapsone over the study period and especially the incidence of cutaneous reactions. * To analyze the overall response rate (platelet count \> 30 x 109/L with at least a doubling of the pre-treatment count in the absence of any other ITP treatment) in both treatment arms at week 24. * To compare the rate of complete response and failure in both arms at 24 and 52 weeks. * To compare time to treatment failure (TTF) in both arms * To investigate the mechanisms of action of dapsone in ITP in a subgroup of patients (ancillary study)

Timeline

Start

2015-12

Primary completion

2019-05

Completion

2019-05

Publications

• Background Durand JM, Lefevre P, Hovette P, Mongin M, Soubeyrand J. Dapsone for idiopathic autoimmune thrombocytopenic purpura in elderly patients. Br J Haematol. 1991 Jul;78(3):459-60. doi: 10.1111/j.1365-2141.1991.tb04467.x. No abstract available.
• Background Godeau B, Oksenhendler E, Bierling P. Dapsone for autoimmune thrombocytopenic purpura. Am J Hematol. 1993 Sep;44(1):70-2. doi: 10.1002/ajh.2830440117.
• Background Hernandez F, Linares M, Colomina P, Pastor E, Cervero A, Perez A, Perella M. Dapsone for refractory chronic idiopathic thrombocytopenic purpura. Br J Haematol. 1995 Jun;90(2):473-5. doi: 10.1111/j.1365-2141.1995.tb05179.x.
• Background Godeau B, Durand JM, Roudot-Thoraval F, Tenneze A, Oksenhendler E, Kaplanski G, Schaeffer A, Bierling P. Dapsone for chronic autoimmune thrombocytopenic purpura: a report of 66 cases. Br J Haematol. 1997 May;97(2):336-9. doi: 10.1046/j.1365-2141.1997.412687.x.
• Background Damodar S, Viswabandya A, George B, Mathews V, Chandy M, Srivastava A. Dapsone for chronic idiopathic thrombocytopenic purpura in children and adults--a report on 90 patients. Eur J Haematol. 2005 Oct;75(4):328-31. doi: 10.1111/j.1600-0609.2005.00545.x.
• Larue M, Moulis G, Rueter M, Audia S, Comont T, Terriou L, Viallard JF, Pan-Petesch B, Royer B, Bonnotte B, Galicier L, Lambotte O, Lefrere F, Cheze S, Ebbo M, Duong TA, Boutin E, Languille L, Mahevas M, Godeau B, Canoui-Poitrine F, Michel M. Efficacy and safety of dapsone in adult primary immune thrombocytopenia. Blood Adv. 2025 Apr 22;9(8):1976-1983. doi: 10.1182/bloodadvances.2024014939.

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