Feng F-E et al. -In the current study, the efficacy and safety of all-trans retinoic acid (ATRA) plus danazol versus danazol in non-splenectomised patients with corticosteroid-resistant or relapsed primary immune thrombocytopenia (PIT) was determined. It was shown that patients with PIT given ATRA plus danazol had a rapid and sustained response compared with danazol monotherapy.

Methods

• A multicentre, randomised, open-label, phase 2 study of adult patients (greater than or equal to 18 years of age) with PIT from 5 different tertiary medical centres in China was conducted.
• The participants were non-splenectomised, resistant to corticosteroid treatment or relapsed, and had a platelet count less than 30 × 10[9] per L.
• Patients received oral ATRA (10 mg twice daily) plus oral danazol (200 mg twice daily) or oral danazol monotherapy (200 mg twice daily) for 16 weeks.
• All patients were assessed every week during the first 8 weeks of treatment, and at 2-week intervals thereafter.
• The primary endpoint was 12-month sustained response (a platelet count of 30 × 10[9] per L or more and at least a doubling of baseline platelet count [partial response] or a platelet count of 100 × 10[9] per L or more [complete response] and the absence of bleeding without rescue medication at the 12-month follow-up.

Results

• 93 patients were included in the modified intention-to-treat analysis (45 in the ATRA plus danazol group and 48 in the danazol group).
• A sustained response was more frequent in patients receiving ATRA plus danazol than in patients receiving danazol monotherapy (28 of 45 vs. 12 of 48; OR = 4.94).