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Blood, 15 February 2007, Vol. 109, No. 4, pp. 1401-1407.
Prepublished online as a Blood First Edition Paper on October 31, 2006; DOI 10.1182/blood-2005-12-015222.


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CLINICAL TRIALS AND OBSERVATIONS

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

Maria Gabriella Mazzucconi1, Paola Fazi2, Sayla Bernasconi1, Giulio De Rossi3, Giuseppe Leone4, Luigi Gugliotta5, Nicola Vianelli6, Giuseppe Avvisati7, Francesco Rodeghiero8, Angela Amendola1, Carlo Baronci3, Cecilia Carbone9, Stefano Quattrin10, Giuseppe Fioritoni11, Giulio D'Alfonso2, Franco Mandelli, for the Gruppo Italiano Malattie EMatologiche dell'Adulto (GIMEMA) Thrombocytopenia Working Party1

1 Dipartimento di Biotecnologie Cellulari ed Ematologia, Università degli Studi di Roma La Sapienza, Rome, Italy; 2 Fondazione GIMEMA, Centro Dati, Rome, Italy; 3 Divisione di Ematologia, Ospedale pediatrico Bambino Gesù, Rome, Italy; 4 Divisione di Ematologia, Università Cattolica del Sacro Cuore, Rome, Italy; 5 Servizio di Ematologia, Arciospedale Santa Maria Nuova, Reggio Emilia, Italy; 6 Istituto di Ematologia e Oncologia Medica L. e A. Seragnoli, Università di Bologna, Italy; 7 Facoltà di Medicina e Chirurgia, Libera Università Campus Bio-Medico, Rome, Italy; 8 Divisione di Ematologia, Ospedale S. Bortolo, Vicenza, Italy; 9 Sezione di Ematologia e Trapianti, Ospedali Civili, Brescia, Italy; 10 Oncoematologia, Ospedale S. Maria delle Grazie, Pozzuoli, Italy; 11 Divisione Ematologia e Trapianto, Azienda USL, Pescara, Italy

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.


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