Therapy with high-dose dexamethasone (HD-DXM) in previously untreated patients affected by idiopathic thrombocytopenic purpura: a GIMEMA experience

• Published in: Blood Vol. 109; no. 4; pp. 1401 - 1407
• Main Authors: Mazzucconi, Maria Gabriella, Fazi, Paola, Bernasconi, Sayla, De Rossi, Giulio, Leone, Giuseppe, Gugliotta, Luigi, Vianelli, Nicola, Avvisati, Giuseppe, Rodeghiero, Francesco, Amendola, Angela, Baronci, Carlo, Carbone, Cecilia, Quattrin, Stefano, Fioritoni, Giuseppe, D'Alfonso, Giulio, Mandelli, Franco
• Format: Journal Article
• Language: English
• Published: Washington, DC Elsevier Inc 15.02.2007; The Americain Society of Hematology
• Subjects: Adolescent; Adult; Age Factors; Aged; Biological and medical sciences; Child; Child, Preschool; Dexamethasone - administration & dosage; Disease-Free Survival; Drug Administration Schedule; Female; Glucocorticoids - administration & dosage; Hematologic and hematopoietic diseases; Humans; Infant; Male; Medical sciences; Middle Aged; Pilot Projects; Platelet diseases and coagulopathies; Prospective Studies; Purpura, Thrombocytopenic, Idiopathic - drug therapy; Remission Induction; Human; Corticosteroid; Platelet; Dexamethasone; Treatment; Hematology; Steroid hormone; Hemopathy; Immune thrombocytopenic purpura; High dose
• ISSN: 0006-4971; 1528-0020
• DOI: 10.1182/blood-2005-12-015222

In idiopathic thrombocytopenic purpura (ITP), corticosteroids have been widely recognized as the most appropriate first-line treatment, even if the best therapeutic approach is still a matter of debate. Recently, a single high-dose dexamethasone (HD-DXM) course was administered as first-line therapy in adult patients with ITP. In this paper we show the results of 2 prospective pilot studies (monocentric and multicentric, respectively) concerning the use of repeated pulses of HD-DXM in untreated ITP patients. In the monocenter study, 37 patients with severe ITP, age at least 20 years and no more than 65 years, were enrolled. HD-DXM was given in 4-day pulses every 28 days, for 6 cycles. Response rate was 89.2%; relapse-free survival (RFS) was 90% at 15 months; long-term responses, lasting for a median time of 26 months (range 6-77 months) were 25 of 37 (67.6%). In the multicenter study, 95 patients with severe ITP, age at least 2 years and no more than 70 years, were enrolled. HD-DXM was given in 4-day pulses every 14 days, for 4 cycles; 90 patients completed 4 cycles. Response rate (85.6%) was similar in patients classified by age (< 18 years, 36 of 42 = 85.7%; ≥ 18 years, 41 of 48 = 85.4%, P = not significant), with a statistically significant difference between the second and third cycle (75.8% vs 89%, P = .018). RFS at 15 months 81%; long-term responses, lasting for a median time of 8 months (range 4-24 months) were 67 of 90 (74.4%). In both studies, therapy was well tolerated. A schedule of 3 cycles of HD-DXM pulses will be compared with standard prednisone therapy (eg, 1 mg/kg per day) in the next randomized Gruppo Italiano Malattie EMatologiche dell'Adulto (GIMEMA) trial.