January 05, 2016
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High-dose dexamethasone confers favorable outcomes in adult primary ITP

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High-dose dexamethasone may be a preferred corticosteroid strategy compared with conventional prednisone for first-line treatment of adult primary immune thrombocytopenia, according to the results of a randomized prospective study.

“Immune thrombocytopenia (ITP) is an autoimmune thrombocytopenic syndrome characterized by decreased platelet count and an increased risk for bleeding,” Ming Hou, MD, a hematologist at Qilu Hospital at Shandong University in Jinan, China, and colleagues wrote. “Corticosteroids are recommended as the first-line therapeutic strategy in practical guidelines.”

Prednisone currently serves as the standard initial treatment for adult patients with primary ITP, according to study background. However, only a small number of patients treated with prednisone achieve long-lasting remission.

Thus, Hou and colleagues sought to compare the safety and efficacy of high-dose dexamethasone and conventional prednisone in a randomized cohort of newly diagnosed patients with primary ITP.

The study included data from 195 patients. The researchers randomly assigned patients to 4 days of dexamethasone (n = 95; 40 mg per day) or 4 weeks of prednisone (n = 97; 1 mg/kg per day).

Patients assigned dexamethasone who did not respond had the option to receive an additional 4-day treatment course.

Initial response and sustained response served as the primary endpoints.

The researchers observed that dexamethasone was associated with higher rates of overall initial response (82.1% vs. 67.4%; P = .044) and complete response (50.5% vs. 26.8%; P = .001).

Further, patients assigned dexamethasone experienced a shorter time to response (median, 3 days vs. 6 days; P < .001).

In both arms, a baseline bleeding score of 8 or higher appeared associated with a decreased likelihood of initial response (dexamethasone: OR = 0.25; 95% CI, 0.06-0.92; prednisone: OR = 0.21; 95% CI, 0.06-0.7).

A similar proportion of patients in both arms achieved sustained response (dexamethasone, 40%; prednisone, 41.2%).

Initial complete response served as a positive predictor for sustained response. Initial complete responders were less likely to lose their response when treated with dexamethasone (OR = 0.17; 95% CI, 0.06-0.51) or prednisone (OR = 0.25; 95% CI, 0.07-0.88). However, the presence of antiplatelet autoantibodies served as a negative predictor of sustained response in both arms (dexamethasone, P = .021; prednisone, P = .024).

No deaths occurred in either arm. Both treatment regimens appeared well tolerated, with mostly mild adverse events observed. Two patients assigned prednisone discontinued treatment due to adverse events compared with no patients assigned dexamethasone.

“One or two courses of high-dose dexamethasone provides a more effective and more rapid response as initial treatment of ITP, with at least comparable long-term prognosis and better tolerance when compared with conventional prednisone,” Hou and colleagues wrote. “Therefore, high-dose dexamethasone could become a preferred corticosteroid approach for first-line management of adult primary ITP. Furthermore, as it has been shown that repeated courses of medication may yield better long-term outcomes, future randomized controlled trials should be designed to compare the effect of repeated courses vs. a limited number of high-dose dexamethasone.” – by Cameron Kelsall

Disclosure: The researchers report no relevant financial disclosures.