Dexamethasone effective as initial therapy for immune thrombocytopenic purpura [Classics Series]

1. Of 125 patients with newly diagnosed immune thrombocytopenic purpura, 106 (85%) had an initial response to high-dose dexamethasone therapy

2. Half of the 106 patients with an initial response to high-dose dexamethasone had a sustained response after six months of follow-up

Original Date of Publication: August 28, 2003

Study Rundown: This study assessed the effectiveness of high-dose dexamethasone as an initial therapy for newly diagnosed immune thrombocytopenic purpura (ITP). The results were promising as 85% of patients showed an initial response to therapy and half of these patients had a sustained response after six months of follow-up. This demonstrated that a short course (four days) of high dose glucocorticoid could be effective as an initial treatment for ITP, avoiding the numerous and potentially severe complications associated with longer courses of prednisone, which was the standard therapy. The high-dose dexamethasone was well-tolerated. No patients discontinued treatment due to side effects in this study. This prospective case series involved a relatively large sample of patients; however, the study lacked a control group to compare its performance to a placebo or treatment with prednisone.

Click to read the study in NEJM

Click to read an accompanying editorial in NEJM

In-Depth [case series study]: This study recruited consecutive adult patients who presented with a new diagnosis of ITP. Of 157 consecutive patients, 125 met eligibility criteria – a platelet count of less than 20,000 per mm³, or a platelet count of less than 50,000 per mm³  and clinically significant bleeding. The exclusion criteria were relapsed ITP, treatment with corticosteroids in the previous 6 months, a history of clinically significant adverse effects from previous corticosteroid treatment (e.g., psychosis, avascular necrosis), uncontrolled hypertension or diabetes mellitus, and pregnancy. Patients were treated with 40 mg daily of oral dexamethasone for four days. Initial treatment response was defined as an increase in platelet count of at least 30,000 per mm³, a platelet count greater than 50,000 mm³ by day 10 after treatment was started, and bleeding cessation. A sustained response was defined as a platelet count above 50,000 per mm³ after six months of follow-up.

Of the 125 patients included, 106 (85%) had an initial response to high-dose dexamethasone. Of the 19 patients who did not have a treatment response, 14 responded to either intravenous immune globulin or anti-D immune globulin. The remaining patients underwent splenectomy or received cytotoxic therapy. The median follow-up period was 30.5 months. Of the 106 patients with an initial response to dexamethasone therapy, 53 (50%) had a sustained response and required no further treatment. The remaining 53 patients had a relapse but responded to a second course of high-dose dexamethasone. No patient discontinued treatment due to adverse effects.

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