title: Therapy with high-dose dexamethasone (HD-DXM) in previously untreated patients affected by idiopathic thrombocytopenic purpura: a GIMEMA experience.
abstract: 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%; >or=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.
authors.affiliation: Dipartimento di Biotecnologia Cellulari ed Ematologia, Università degli Studi di Roma La Sapienza, and Divisione di Ematologia, Ospedale pediatrico Bambino Gesù, Rome, Italy. mazzucconi@bce.uniroma1.it
authors.forename: Maria Gabriella
authors.initials: MG
authors.lastname: Mazzucconi
authors.forename: Paola
authors.initials: P
authors.lastname: Fazi
authors.forename: Sayla
authors.initials: S
authors.lastname: Bernasconi
authors.forename: Giulio
authors.initials: G
authors.lastname: De Rossi
authors.forename: Giuseppe
authors.initials: G
authors.lastname: Leone
authors.forename: Luigi
authors.initials: L
authors.lastname: Gugliotta
authors.forename: Nicola
authors.initials: N
authors.lastname: Vianelli
authors.forename: Giuseppe
authors.initials: G
authors.lastname: Avvisati
authors.forename: Francesco
authors.initials: F
authors.lastname: Rodeghiero
authors.forename: Angela
authors.initials: A
authors.lastname: Amendola
authors.forename: Carlo
authors.initials: C
authors.lastname: Baronci
authors.forename: Cecilia
authors.initials: C
authors.lastname: Carbone
authors.forename: Stefano
authors.initials: S
authors.lastname: Quattrin
authors.forename: Giuseppe
authors.initials: G
authors.lastname: Fioritoni
authors.forename: Giulio
authors.initials: G
authors.lastname: D'Alfonso
authors.forename: Franco
authors.initials: F
authors.lastname: Mandelli
mesh_terms: D000293:Adolescent; D000328:Adult; D000367:Age Factors; D000368:Aged; D002648:Child; D002675:Child, Preschool; D003907:Dexamethasone / Q000008:administration & dosage*; D018572:Disease-Free Survival; D004334:Drug Administration Schedule; D005260:Female; D005938:Glucocorticoids / Q000008:administration & dosage*; D006801:Humans; D007223:Infant; D008297:Male; D008875:Middle Aged; D010865:Pilot Projects; D011446:Prospective Studies; D016553:Purpura, Thrombocytopenic, Idiopathic / Q000188:drug therapy*; D012074:Remission Induction
chemical_list: D005938:Glucocorticoids; D003907:Dexamethasone