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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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Submitted December 30, 2005
Accepted August 22, 2006

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

Maria Gabriella Mazzucconi*, Paola Fazi, Sayla Bernasconi, Giulio De Rossi, Giuseppe Leone, Luigi Gugliotta, Nicola Vianelli, Giuseppe Avvisati, Francesco Rodeghiero, Angela Amendola, Carlo Baronci, Cecilia Carbone, Stefano Quattrin, Giuseppe Fioritoni, Giulio D'Alfonso, and Franco Mandelli

Universita degli Studi di Roma "La Sapienza", Italy
Fondazione GIMEMA, Italy
Ospedale pediatrico Bambino Gesu, Italy
Universita Cattolica del Sacro Cuore, Italy
Arciospedale "Santa Maria Nuova", Italy
Universita di Bologna, Italy
Libera Universita "Campus Bio-Medico", Italy
Ospedale "S. Bortolo", Italy
Ospedali Civili, Italy
Ospedale "S. Maria delle Grazie", Italy
Azienda USL, Italy

* Corresponding author; email: mazzucconi{at}bce.uniroma1.it.

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 HD-DXM course was administered as first-line therapy in adult ITP patients. In this paper we show the results of two prospective pilot studies (monocentric and a multicentric, respectively) concerning the use of repeated pulses of HD-DXM in untreated ITP patients. Monocentre study: 37 severe ITP patients, age ≥ 20 and ≤ 65, were enrolled. HD-DXM, in 4 day pulses, every 28 days, for 6 cycles was given. Response rate was 89.2%; relapse free survival (RFS) 90% at 15 months; long-term responses, lasting for a median time of 26 months (6-77), were 25/37 (67.6%). Multicentre study: 95 severe ITP patients, age ≥ 2 and ≤ 70, were enrolled HD-DXM in 4 days pulses, every 14 days, for 4 cycles was given, 90 completed 4 cycles. Response rate (85.6%) was similar in patients classified by age (<18 years, 36/42= 85.7%; ≥ 18 years, 41/48= 85.4%, p=n.s.), with a statistically significant difference between the second and third cycle (75.8% vs 89%, p=0.018). RFS at 15 months 81%; long-term responses, lasting for a median time of 8 months (4-24), were 67/90 (74.4%). In both studies, therapy was well tolerated. A schedule of three cycles of HD-DXM pulses will be compared with standard prednisone therapy (e.g.1mg/Kg/day) in a next randomized GIMEMA trial.


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