Ruxolitinib in Myelofibrosis Patients in Lombardy, Italy
- Conditions
- Myelofibrosis
- Registration Number
- NCT03959371
- Lead Sponsor
- Margherita Maffioli
- Brief Summary
The RUXOREL-MF observational study includes patients with primary and post-essential thrombocythemia/post-polycythemia vera myelofibrosis (MF) being treated with the oral JAK1-/JAK2-inhibitor ruxolitinib in a "real world" setting. Patients are treated according to current indications in Italy (i.e., primary and secondary MF patients with intermediate-1, intermediate-2, and high risk IPSS (International Prognostic Scoring System) scores and symptomatic splenomegaly and/or systemic symptoms). Patients are treated at facilities pertaining to the regional Hematology Network of Lombardy (Rete Ematologica Lombarda) in Italy. Efficacy data, data related to infectious and vascular events, data related to second primary malignancies, data regarding disease progression/transformation, and molecular information in relationship to ruxolitinib treatment will be collected and analyzed.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- UNKNOWN
- Sex
- All
- Target Recruitment
- 620
- Age >= 18 years
- Diagnosis of primary myelofibrosis diagnosis according to the WHO 2016 classification or post-essential thrombocythemia/post-polycythemia vera myelofibrosis according to the IWG-MRT 2008 classification
- Patients with an intermediate-1, intermediate-2, or high risk score according to the IPSS (International Prognostic Scoring System)
- Patients treated with ruxolitinib in accordance with current indications in Italy
- Patients eligible or ineligible to hematopoietic stem cell transplant or who have already undergone a hematopoietic stem cell transplant
- Diagnoses other than primary myelofibrosis or post-essential thrombocythemia/post-polycythemia vera myelofibrosis
- Patients treated with ruxolitinib having a platelet count at treatment initiation <50 x10^9/L
- Patients treated with ruxolitinib for conditions other than primary myelofibrosis or post-essential thrombocythemia/post-polycythemia vera myelofibrosis
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Rate of vascular events after ruxolitinib exposure in myelofibrosis patients Through study completion, an average of 1 year Rate of infectious events after ruxolitinib exposure in myelofibrosis patients Through study completion, an average of 1 year
- Secondary Outcome Measures
Name Time Method Rate of primary secondary malignancies Through study completion, an average of 1 year Spleen response rate At 3 and 6 months from ruxolitinib start Acute myeloid leukemia transformation rate Through study completion, an average of 1 year Rate of primary secondary malignancies according to driver mutational status (i.e., mutations of JAK2, CALR, or MPL) Through study completion, an average of 1 year Association of rate of primary secondary malignancies with driver mutational status
Acute myeloid leukemia transformation rate according to driver mutational status (i.e., mutations of JAK2, CALR, or MPL) Through study completion, an average of 1 year Association of acute myeloid leukemia transformation rate with driver mutational status
Rate of infectious events according to the presence of additional mutations Through study completion, an average of 1 year Association of rate of infectious events with the presence of additional mutations
Rate of vascular events according to the presence of additional mutations Through study completion, an average of 1 year Association of rate of vascular events with the presence of additional mutations
Rate of infectious events according to driver mutational status (i.e., mutations of JAK2, CALR, or MPL) Through study completion, an average of 1 year Association of rate of infectious events with driver mutational status
Rate of vascular events according to driver mutational status (i.e., mutations of JAK2, CALR, or MPL) Through study completion, an average of 1 year Association of rate of vascular events with driver mutational status
Spleen response rate according to driver mutational status (i.e., mutations of JAK2, CALR, or MPL) Through study completion, an average of 1 year Association of spleen response rate with driver mutational status
Spleen response rate according to the presence of additional mutations Through study completion, an average of 1 year Association of spleen response rate with the presence of additional mutations
Rate of primary secondary malignancies according to the presence of additional mutations Through study completion, an average of 1 year Association of rate of primary secondary malignancies with the presence of additional mutations
Acute myeloid leukemia transformation rate according to the presence of additional mutations Through study completion, an average of 1 year Association of acute myeloid leukemia transformation rate with the presence of additional mutations
Evaluation of overall survival after ruxolitinib start and, if applicable, discontinuation Through study completion, an average of 1 year
Trial Locations
- Locations (12)
U.O. Ematologia, ASST Spedali Civili
🇮🇹Brescia, Italy
Clinica Ematologica, Ospedale San Gerardo
🇮🇹Monza, Italy
U.O.C. di Ematologia Clinica, ASST Lecco
🇮🇹Lecco, Italy
U.O. Ematologia, ASST Fatebenefratelli-Sacco
🇮🇹Milan, Italy
U.O. Ematologia, Grande Ospedale Metropolitano Niguarda
🇮🇹Milan, Italy
U.O. Oncoematologia, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
🇮🇹Milan, Italy
U.S.D. Trapianti di Midollo Osseo, ASST Spedali Civili
🇮🇹Brescia, Italy
ASST Papa Giovanni XXIII
🇮🇹Bergamo, Italy
U.O. Ematologia, Fondazione IRCCS Istituto Nazionale Tumori
🇮🇹Milan, Italy
U.O. Ematologia, Humanicas Cancer Center
🇮🇹Milan, Italy
U.O. Ematologia, Ospedale San Raffaele
🇮🇹Milan, Italy
Ospedale di Circolo, ASST Sette Laghi
🇮🇹Varese, Italy