Prednisone vs high-dose dexamethasone in newly diagnosed adult primary Immune Thrombocytopenia: a randomised trial.

A debate is still ongoing regarding which type of corticosteroids [standard dose prednisone (PDN) or high-dose dexamethasone (HD-DXM)] is the best first-line treatment for newly diagnosed untreated primary immune thrombocytopenia (pITP) adult patients. An ad hoc study compared PDN to HD-DXM in newly diagnosed untreated adult pITP patients (>18<80 years, platelet count <20x109/L or >20<50x109/L and bleeding score >8). Patients were randomised to receive PDN 1 mg/kg/day from day 0 to 28 (ARM A) or HD-DXM 40 mg/day for 4 days, every 14 days, for 3 consecutive courses (ARM B). Fifty-nine/113 patients (52.2%) were randomised to ARM A, 54/113 (47.8%) to ARM B. In evaluable patients total initial responses [Complete Response (CR), Partial Response (PR), Minimal Response (MR)] were 44/56 (78.57%) in ARM A, and 46/49 (93.88%) in ARM B at days 42 and 46, respectively (P=0.0284). Total final responses (CR+PR+MR at day 180 from initial response) were 26/43 (60.47%) in ARM A and 23/39 (58.97%) in ARM B (P=0.8907). Persistent responses (CR+PR+MR, at 12 months from initial response), were 25/31 (80.65%) in ARM A and 20/36 (55.56%) in ARM B (P=0.0292). Seven relapses occurred: 2 in ARM A, 5 in ARM B. Median follow-up was 44.4 months. Overall survival was 100% at 48 months, overall disease-free survival was 81.11% at 48 months from day 180. Prednisone and pulsed HD-DXM were well tolerated; HD-DXM allows effective initial responses, but less long-lasting than PDN. GIMEMA Protocol ITP 0207-EudraCT number 2008-000417-30, was registered at ClinicalTrials.gov #NCT00657410.