Prednisone vs high-dose dexamethasone in newly diagnosed adult primary immune thrombocytopenia: a randomized trial.

Valeria Sargentini, Marco Ruggeri, Monica Bocchia, Sergio Siragusa, Giuseppe Avvisati, Agostino Tafuri, Iolanda Donatella Vincelli, Elena Rivolti, Maria Gabriella Mazzucconi, Luigi Gugliotta, Cristina Santoro, Erminia Baldacci, Antonietta Ferretti, Giuseppe Carli, Stefania Fortuna, Paola Ranalli, Elisabetta Lugli, Silvia Sibilla, Roberta Battistini, Anna Chiara Giuffrida, Monica Crugnola, Francesca Palandri, Mauro Krampera, Nicola Vianelli, Francesco Rodeghiero, Andrea Patriarca, Mauro Di Ianni, Angela Rago, Valerio De Stefano, Angelo Michele Carella, Elena Rossi, Francesca Paoloni, Nicola Di Renzo, Pellegrino Musto, Paola Fazi, Nicola Polverelli, Mariella D'Adda, Francesco Zaja, Bruno Martino, Massimo Gentile

Journal: Blood advances 2024;8(6):1529-1540

PMID: 38231017

Free Full Text on Elsevier Science View on PubMed

Abstract

A debate exists regarding which type of corticosteroids (standard-dose prednisone [PDN] or high-dose dexamethasone [HD-DXM]) is the best first-line treatment for adult patients with newly diagnosed untreated primary immune thrombocytopenia (pITP). An ad hoc study compared PDN with HD-DXM in newly diagnosed untreated patients with pITP (aged ≥18 but ≤80 years, platelet count of ≤20 or >20 but <50 × 109/L, and bleeding score of ≥8). Patients were randomised to receive PDN 1 mg/kg per day from days 0 to 28 (Arm A) or HD-DXM 40 mg per day for 4 days, every 14 days, for 3 consecutive courses (Arm B). Fifty-nine of 113 patients (52.2%) were randomized to Arm A and 54 of 113 (47.8%) to Arm B. In evaluable patients, total initial responses (complete response [CR], partial response [PR], minimal response [MR]) were 44 of 56 (78.57%) in Arm A and 46 of 49 (93.88%) in Arm B at days 42 and 46, respectively (P = 0.0284). Total final responses (at day 180 from initial response) were 26 of 43 (60.47%) in Arm A and 23 of 39 (58.97%) in Arm B (P = 0.8907). Total persistent responses (at 12 months from initial response) were 25 of 31 (80.65%) in Arm A and 20 of 36 (55.56%) in Arm B (P = 0.0292). Seven relapses occurred. Median follow-up was 44.4 months. Overall survival was 100% at 48 months, overall disease-free survival was 81.11% at 48 months from day 180. PDN and pulsed HD-DXM were well tolerated; HD-DXM allows effective initial responses but less long lasting than PDN. This trial was registered at www.clinicaltrials.gov as #NCT00657410.

© 2024 by The American Society of Hematology. Licensed under Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 International (CC BY-NC-ND 4.0), permitting only noncommercial, nonderivative use with attribution. All other rights reserved.

Address: Hematology, Dipartimento Medicina Traslazionale e di Precisione, Università degli Studi Sapienza, Rome, Italy.; Hematology Project Foundation, Vicenza Affiliated to the Department of Hematology, San Bortolo Hospital, Vicenza, Italy.; Fondazione Policlinico Universitario Campus Bio-Medico, Rome, Italy.; Sezione di Ematologia, Dipartimento di Scienze Radiologiche ed Ematologiche, Università Cattolica, Fondazione Policlinico Universitario Agostino Gemelli, Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS), Rome, Italy.; Unità Operativa Complessa di Ematologia, Arcispedale Santa Maria Nuova, Reggio Emilia, Italy.; Unità Operativa Complessa (UOC) Ematologia, Azienda Unità Locale Socio Sanitaria 8 Berica-Ospedale di Vicenza, Vicenza, Italy.; Istituto di Ematologia Seràgnoli, IRCCS Azienda Ospedaliero, Universitaria di Bologna, Bologna, Italy.; Gruppo Italiano Malattie EMatologiche Adulto Foundation, Franco Mandelli Onlus, Rome, Italy.; UOC Ematologia, Azienda Ospedaliero Universitaria Policlinico Umberto I, Rome, Italy.; Hematology, Dipartimento Medicina Traslazionale e di Precisione, Università degli Studi Sapienza, Rome, Italy.; Sezione di Ematologia, Dipartimento di Scienze Radiologiche ed Ematologiche, Università Cattolica, Fondazione Policlinico Universitario Agostino Gemelli, Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS), Rome, Italy.; Dipartimento Emato-Oncologia, Azienda Ospedaliera Bianchi-Melacrino-Morelli, Reggio Calabria, Italy.; UOC Ematologia Clinica, Azienda Sanitaria Locale (ASL) Pescara PO Santo Spirito, Pescara, Italy.; Struttura Semplice a Valenza Dipartimentale Centro Trapianti Midollo per Adulti, Azienda Socio Sanitaria Territoriale (ASST) degli Spedali civili di Brescia, Brescia, Italy.; Ematology, Azienda Unità Sanitaria Locale IRCCS, Reggio Emilia, Italy.; Struttura Complessa a Direzione Universitaria Ematologia, Azienda Ospedlaiero Universitaria Maggiore della Carità di Novara, Novara, Italy.; UOC Ematologia, ASL Latina-Ospedale S.M. Goretti, Latina, Italy.; Unità Operativa (UO) Ematologia, ASST degli Spedali Civili di Brescia, Brescia, Italy.; Hematology Unit, Azienda Ospedaliera of Cosenza, Cosenza, Italy.; Department of Pharmacy, Health and Nutritional Sciences, University of Calabria, Rende, Italy.; UOC di Ematologia, Azienda Ospedaliera Universitaria Policlinico di Palermo, Palermo, Italy.; UO Ematologia, Ente Ecclesiastico Cardinale G. Panico, Tricase, Italy.; UOC Ematologia e Centro Trapianti Cellule Staminali Emopoietiche, Fondazione IRCCS Casa Sollievo della Sofferenza, San Giovanni Rotondo, Italy.; UOC Ematologia e Centro Trapianti CSE, Azienda Ospedaliera San Camillo Forlanini, Rome, Italy.; Dipartimento Scienze Mediche, Università degli Studi di Trieste Unità Complessa Operativa Ematologia, Azienda Sanitaria Universitaria Giuliano Isontina, Trieste, Italy.; UOC Ematologia e Trapianti, Azienda Ospedlaiero Universitaria Senese Policlinico Le Scotte, Siena, Italy.; UOC di Ematologia e Trapianto di Cellule Staminali, Ospedale Vito Fazzi ASL Lecce, Lecce, Italy.; Department of Precision and Regenerative Medicine and Ionian Area, Aldo Moro University School of Medicine and Unit of Hematology and Stem Cell Transplantation, Azienda Ospedaliero Universitaria Consorziale Policlinico, Bari, Italy.; Hematology Unit and Bone Marrow Transplantation Center, Azienda Ospedaliera Universitaria, Parma, Italy.; UOC Medicina Trasfusionale, Azienda Ospedaliera Universitaria Integrata, Verona, Italy.; UOC di Ematologia e Centro Trapianto di Midollo Osseo, Azienda Ospedaliera Universitaria Integrata, Verona, Italy.; Hematology, University Hospital Sant'Andrea, Department of Clinical and Molecular Medicine, Sapienza University of Rome, Rome, Italy.

DOI: 10.1182/bloodadvances.2023010975

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MeSH Terms: Adult, Humans, Prednisone, Purpura, Thrombocytopenic, Idiopathic, Dexamethasone, Platelet Count, Disease-Free Survival