High-dose dexamethasone vs prednisone for treatment of adult immune thrombocytopenia: a prospective multicenter randomized trial

Abstract

Key Points• HD-DXM is a preferred strategy to conventional prednisone as first-line management of newly diagnosed adult primary ITP.This study compared the efficacy and safety of high-dose dexamethasone (HD-DXM) and conventional prednisone (PDN) on the largest cohort to date as first-line strategies for newly diagnosed adult primary immune thrombocytopenia (ITP). Patients enrolled were randomized to receive DXM 40 mg/d for 4 days (n 5 95, nonresponders received an additional 4-day course of DXM) or prednisone 1.0 mg/kg daily for 4 weeks and then tapered (n 5 97). One or 2 courses of HD-DXM resulted in a higher incidence of overall initial response (82.1% vs 67.4%, P 5 .044) and complete response (50.5% vs 26.8%, P 5 .001) compared with prednisone. Time to response was shorter in the HD-DXM arm (P < .001), and a baseline bleeding score ‡8 was associated with a decreased likelihood of initial response. Sustained response was achieved by 40.0% of patients in the HD-DXM arm and 41.2% in the PDN arm (P 5 .884). Initial complete response was a positive indicator of sustained response, whereas presence of antiplatelet autoantibodies was a negative indicator. HD-DXM was generally tolerated better. We concluded that HD-DXM could be a preferred corticosteroid strategy for first-line management of adult primary ITP. This study is registered at www.clinicaltrials.gov as #NCT01356511. (Blood. 2016;127(3):296-302)

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“…1a). [6][7][8][9][10]13,14 This tendency was the same if analyzed with the random-effects model (RR, 1.23; 95% CI, 0.84-1.79; p ¼ 0.28).…”

“…If the random-effects model was used, RR was 1.78 (95% CI, 1.37-2.32; p < 0.01). On the other hand, three studies using Dex monotherapy (only one course of Dex; additional courses of Dex were administered only to nonresponders) [8][9][10] showed inferior SR incidence compared with that of PSL with borderline significance (N ¼ 437; RR, 0.80; 95% CI, 0.62-1.03; p ¼ 0.08; ►Fig. 1b).…”

“…The primary search yielded 157 citations, of which 7 RCTs met the inclusion criteria. [6][7][8]10,[12][13][14] A search of conference proceedings revealed one additional relevant study (abstract only). 9 In total, we included eight RCTs totaling 704 patients with acute primary ITP (κ ¼ 0.83).…”

“…The intervention groups (Dex) consisted of high-dose Dex (40 mg/day for 4 days in most studies; 0.6 mg/kg/day for 4 days in one study 14 ). In three studies, patients were administered Dex monotherapy (one course of Dex; additional courses were added only in nonresponders) without any consolidation or tapering of the corticosteroid administration, [8][9][10] while in five studies, patients were administered Dex with posttherapy (two to six courses of Dex for all patients 6,13,14 or low-dose Dex 13 or PSL 7,12 accompanying the preceding courses of Dex, irrespective of the treatment effects in the first course of Dex). Total dosage of corticosteroids in the interventional arm were calculated (estimating the body weight was 40-80 kg), and converted to the equivalent amounts of PSL (►Table 1 and ►Table S5 [supplementary table available in the online version only]).…”

“…Cui et al 6 and Mashhadi et al 7 showed the superiority of Dex in terms of long-term sustained response (SR), while Li et al 8 and Bae et al 9 showed inferior SR in the Dex arm. Moreover, the newest and the largest study from Wei et al 10 indicated that Dex and PSL were equivalent in terms of SR.…”