Prednisone or Dexamethasone in Newly Diagnosed, Previously Untreated Primary Immune Thrombocytopenic Purpura
- Conditions
- Nonneoplastic Condition
- Interventions
- Registration Number
- NCT00657410
- Brief Summary
RATIONALE: Drugs, such as prednisone and dexamethasone, may change the immune system and be an effective treatment for primary immune thrombocytopenic purpura. It is not yet known which drug is more effective in treating primary immune thrombocytopenic purpura.
PURPOSE: This randomized phase III trial is studying high-dose dexamethasone to see how well it works compared to standard-dose prednisone in treating patients with newly diagnosed, previously untreated primary immune thrombocytopenic purpura.
- Detailed Description
OBJECTIVES:
Primary
* To evaluate the role of therapy intensification in adult patients with newly diagnosed, previously untreated primary immune thrombocytopenic purpura with high-dose dexamethasone (HD-DXM), in terms of improvement of response at 6 months after initial response, in comparison with standard-doses of prednisone.
Secondary
* Compare rate of initial response.
* Compare quality of response.
* Compare rate of final responses and rate of persistent response.
* Compare rate of bleeding events.
* Determine rate of resumed response with HD-DXM in non-responder patients or patients who have lost response (arm I only).
* Compare time to platelet number increase until a hemostatically effective level is reached and/or disappearance of bleeding symptoms.
* Compare rate of rescue interventions.
* Compare rate of eligible patients for splenectomy.
* Compare rate of patients who underwent splenectomy.
* Compare rate of patients who develop connective tissue diseases or underlying hematological diseases (myelodysplastic syndromes, chronic lymphoproliferative diseases, others).
* Compare patient's self reported quality of life.
OUTLINE: This is a multicenter study. Patients are stratified by treating center. Patients are randomized to 1 of 2 treatment arms.
* Arm I (Standard-dose prednisone): Patients receive oral prednisone at a standard dose (1 mg/Kg) once daily on days 1-28 followed by a 14-day taper.
Patients considered non-responders at day 42 or who have lost response before evaluation of final response (day 180) are crossed to arm II.
* Arm II (High-dose dexamethasone): Patients receive oral dexamethasone at a high dose (40 mg/day) once daily on days 1-4. Treatment repeats every 14 days for 3 courses.
Quality of life is assessed at baseline, on day 42 (arm I) or 46 (arm II) (initial response evaluation day), 180 days after initial response evaluation, and at 3, 9, 12 months after randomization.
After completion of study treatment, patients are followed monthly until 1 year after randomization, every 2 months for 1 year, and then every 3 months for 1 year.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 150
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description ARM B - DXM dexamethasone DXM is administered orally at single fixed daily doses of 40 mg for 4 consecutive days, every 14 days, for 3 consecutive courses. If platelet count is £ 20x109/L or bleeding symptoms related to thrombocytopenia are present, lowdose DXM (0.035 mg/Kg/day) between courses is given. The patients (either from ARM A+B or from ARM B) considered NOT RESPONDER at day 46 or who HAVE LOST THE RESPONSE within the evaluation of final response (see paragraph 8.1), will be considered OFF TREATMENT. For these patients a second line therapy will be considered, according to the medical practice of the Centre (splenectomy or other). ARM B - DXM quality-of-life assessment DXM is administered orally at single fixed daily doses of 40 mg for 4 consecutive days, every 14 days, for 3 consecutive courses. If platelet count is £ 20x109/L or bleeding symptoms related to thrombocytopenia are present, lowdose DXM (0.035 mg/Kg/day) between courses is given. The patients (either from ARM A+B or from ARM B) considered NOT RESPONDER at day 46 or who HAVE LOST THE RESPONSE within the evaluation of final response (see paragraph 8.1), will be considered OFF TREATMENT. For these patients a second line therapy will be considered, according to the medical practice of the Centre (splenectomy or other). ARM A - PDN quality-of-life assessment PDN is administered orally at the daily dose of 1 mg/Kg for 4 consecutive weeks (from day 0 to day 28), then, therapy is tapered within 14 days. The patients considered NOT RESPONDER at day 42 or WHO HAVE LOST THE RESPONSE within the evaluation of final response (see paragraph 8.1), will be crossed to ARM B. ARM A - PDN prednisone PDN is administered orally at the daily dose of 1 mg/Kg for 4 consecutive weeks (from day 0 to day 28), then, therapy is tapered within 14 days. The patients considered NOT RESPONDER at day 42 or WHO HAVE LOST THE RESPONSE within the evaluation of final response (see paragraph 8.1), will be crossed to ARM B.
- Primary Outcome Measures
Name Time Method Final response (complete, partial, and minimal response) rate from evaluation of initial response At day +180 from evaluation of initial response
- Secondary Outcome Measures
Name Time Method Initial response rate At day 42 (arm I), at day 46 (arm II) Quality of response per arm At initial evaluation and at final evaluation Final response rate At day 180 from the statement of initial response Rate of rescue interventions After day 180 from evaluation of initial response Rate of splenectomy eligible patients At 12 months from enrollment Rate of bleeding events At 3 years from study entry Time to platelet number increase until a hemostatically effective level is reached and/or disappearance of bleeding symptoms At 3 years from study entry Rate of persistent response At 12 months from the statement of initial response Association of type of initial response with final and persistent response (in patients with final and persistent response) At 3 years from study entry Rate of patients who have undergone splenectomy during follow-up At 3 years from study entry Resumed response rate in non-responder patients (at day 42) or patients who have lost response before day 180 from the first evaluation (arm I only) At day 42 or before day 180 from the first evaluation Rate of patients who develop connective tissue diseases or underlying hematological diseases (myelodysplastic syndromes, chronic lymphoproliferative diseases, others) during follow-up At 3 years from study entry
Trial Locations
- Locations (42)
S.O.C. di Ematologia - Azienda Ospedaliera - SS. Antonio e Biagio e Cesare Arrigo
🇮🇹Alessandria, Italy
USL 8 - Ospedale S.Donato
🇮🇹Arezzo, Italy
UO Ematologia con trapianto- AOU Policlinico Consorziale di Bari
🇮🇹Bari, Italy
University of Bologna Medical School
🇮🇹Bologna, Italy
Ospedale Maggiore - Div.Medicina Crema
🇮🇹Crema, Italy
Struttura Complessa di Ematologia Ospedali Riuniti Foggia - Azienda Ospedaliero-Universitaria
🇮🇹Foggia, Italy
Ospedale Santa Maria Goretti
🇮🇹Latina, Italy
Istituto Scientifico Romagnoli per lo Studio e la Cura dei Tumori- IRST
🇮🇹Meldola, Italy
Azienda Ospedaliera Universitaria - Policlinico G. Martino Dipartimento di Medicina Interna - U.O. Messina
🇮🇹Messina, Italy
ASL Le1 P.O. Vito Fazzi - U.O. di Ematologia
🇮🇹Lecce, Italy
Azienda Ospedaliera San Paolo - Unità di Ematologia e Trombosi
🇮🇹Milano, Italy
S.C.D.U. Ematologia - DIMECS e Dipartimento Oncologico - Università del Piemonte Orientale Amedeo Avogadro
🇮🇹Novara, Italy
Divisione di Ematologia con trapianto di midollo - A.U. Policlinico "Paolo Giaccone"
🇮🇹Palermo, Italy
Unità Operativa Ematologia e Centro Trapianti - Dipartimento di Oncologia ed Ematologia - AUSL Ospedale di Piacenza
🇮🇹Piacenza, Italy
Dipartimento Oncologico - Ospedale S.Maria delle Croci
🇮🇹Ravenna, Italy
Dipartimento Emato-Oncologia A.O."Bianchi-Melacrino-Morelli"
🇮🇹Reggio Calabria, Italy
Az. Ospedaliera "Sant' Andrea"-Università la Sapienza Seconda Facoltà di Medicina e Chirurgia
🇮🇹Roma, Italy
Divisione di Ematologia - Ospedale S. Camillo
🇮🇹Roma, Italy
Divisione Ematologia - Università Campus Bio-Medico
🇮🇹Roma, Italy
Ospedale "Infermi"
🇮🇹Rimini, Italy
UOC Pronto Soccorso e Accettazione Ematologica - Dipartimento Biotecnologie Cellulari ed Ematologia - Università degli Studi di Roma "Sapienza"
🇮🇹Rome, Italy
Policlinico A. Gemelli - Universita Cattolica del Sacro Cuore
🇮🇹Rome, Italy
Istituto di Ematologia - IRCCS Ospedale Casa Sollievo della Sofferenza
🇮🇹San Giovanni Rotondo, Italy
U.O.C. Ematologia e Trapianti - A.O. Senese - Policlinico " Le Scotte"
🇮🇹Siena, Italy
Divisione di Ematologia dell' Università degli Studi di Torino - "Città della Salute e della Scienza di Torino"
🇮🇹Torino, Italy
Struttura Complessa II Medicina - Ematologia - Centro di Riferimento Ematologico - Ospedale Maggiore
🇮🇹Trieste, Italy
Clinica Ematologica - Policlinico Universitario
🇮🇹Udine, Italy
Ospedale San Bortolo
🇮🇹Vicenza, Italy
U.O. di Ematologia - Azienda Ospedaliera - Pia Fondazione di Culto e di Religione Card. G.Panico
🇮🇹Tricase, (le), Italy
Ospedali Riuniti di Bergamo
🇮🇹Bergamo, Italy
Sezione di Ematologia e Trapianti Spedali Civili
🇮🇹Brescia, Italy
Struttura Complessa di Oncologia Medica - Azienda Ospedaliera - Ospedale di Circolo di Busto Arsizio
🇮🇹Busto Arsizio, Italy
Med. Int. ed Oncologia Medica IRCCS Policlinico S. Matteo
🇮🇹Pavia, Italy
Unità Operativa Complessa di Ematologia - Arcispedale S. Maria Nuova
🇮🇹Reggio Emilia, Italy
Marche U.O. di Medicina Interna - ASUR Marche 8 - Ospedale Civile
🇮🇹Civitanova - Marche, Italy
U.O. Ematologia - P.O. Annunziata - A.O. di Cosenza
🇮🇹Cosenza, Italy
Azienda ospedaliera Nuovo Ospedale "Torrette"
🇮🇹Ancona, Italy
Cattedra di Ematologia CTMO Università degli Studi di Parma
🇮🇹Parma, Italy
U.O. Ematologia Clinica - Azienda USL di Pescara
🇮🇹Pescara, Italy
Policlinico G. B. Rossi - Borgo Roma
🇮🇹Verona, Italy
Dipartimento Area Medica - Presidio Ospedaliero "C. e G.Mazzoni"
🇮🇹Ascoli, Italy
Pordenone Unità operativa Medicina II Az. Osp. S. M. degli Angeli
🇮🇹Pordenone, Italy