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Randomized Controlled Trial
. 2024 Mar 26;8(6):1529-1540.
doi: 10.1182/bloodadvances.2023010975.

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

Maria Gabriella Mazzucconi  1 , Francesco Rodeghiero  2 , Giuseppe Avvisati  3 , Valerio De Stefano  4 , Luigi Gugliotta  5 , Marco Ruggeri  6 , Nicola Vianelli  7 , Paola Fazi  8 , Francesca Paoloni  8 , Valeria Sargentini  8 , Erminia Baldacci  9 , Antonietta Ferretti  1   4 , Bruno Martino  10 , Iolanda Donatella Vincelli  10 , Giuseppe Carli  2 , Stefania Fortuna  2 , Mauro Di Ianni  11 , Paola Ranalli  11 , Francesca Palandri  7 , Nicola Polverelli  12 , Elisabetta Lugli  13 , Elena Rivolti  13 , Andrea Patriarca  14 , Angela Rago  15 , Mariella D'Adda  16 , Massimo Gentile  17   18 , Sergio Siragusa  19 , Silvia Sibilla  20 , Angelo Michele Carella  21 , Elena Rossi  4 , Roberta Battistini  22 , Francesco Zaja  23 , Monica Bocchia  24 , Nicola Di Renzo  25 , Pellegrino Musto  26 , Monica Crugnola  27 , Anna Chiara Giuffrida  28 , Mauro Krampera  29 , Agostino Tafuri  30 , Cristina Santoro  9
Affiliations
Randomized Controlled Trial

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

Maria Gabriella Mazzucconi et al. Blood Adv. .

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.

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Conflict of interest statement

Conflict-of-interest disclosure: C.S. participated in advisory boards for Amgen, Novartis, and Sobi, and was a member of the speakers' bureaus for Amgen, Novartis, and Sobi. E.B. participated in advisory boards for Amgen and Novartis. F.P. received honoraria from Novartis and Sobi. E. Rivolti participated in advisory boards for Novartis and Sobi, and reports presentations for Novartis and Amgen. A.P. was a consultant for Sanofi, Alexion, and Takeda; reports presentations for Sobi, Novartis, Pfizer, and Sanofi; and received congress fees from Alexion and Takeda. M.D. participated in advisory boards for Amgen and Novartis. S. Siragus collaborated with Sobi, Bayer, Amgen, Novartis, and Novo Nordisk. V.D.S. participated in advisory boards for Argenx, Grifols, Novartis, and Sobi, and was a member of the speakers' bureaus for Amgen, Grifols, and Novartis. E. Rossi participated in advisory boards for Argenx, Grifols, Novartis, Amgen, and Sobi, and was a member of the speakers' bureaus for Amgen, Novartis, and Sobi. F.Z. received personal fees from Novartis, Amgen, Roche, AbbVie, Janssen Cilag, Takeda, Grifols, Argenx, Sobi, and BeiGene; received funding for biologic studies or clinical trials from Novartis, Amgen, AbbVie, and Janssen Cilag; and received fundings for educational projects from Janssen Cilag, AbbVie, Kyowa Kirin, Takeda, BeiGene, and AstraZeneca. M.B. received conference fees from Incyte, AstraZeneca, Novartis, and Janssen. M.C. was a member of the speakers' bureaus for Novartis and Bristol Myers Squibb. A.T. received congress fees from Novartis and research funds from Janssen. N.V. participated in advisory boards and was speaker for Novartis, Amgen, Grifols, and Sobi. The remaining authors declare no competing financial interests.

Figures

None
Graphical abstract
Figure 1.
Figure 1.
Treatment scheme, therapeutic approach scheme, and steps in both study arms are reported.
Figure 2.
Figure 2.
CONSORT diagram description of patient flow in both study arms since enrollment to day 180 from the initial response. Causes for exit from the study are also reported.
See this image and copyright information in PMC

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