Clinical trial NCT03959371: Ruxolitinib in Myelofibrosis Patients in Lombardy, Italy
Official title: Observational, Retrospective and Prospective Study on the Use of Ruxolitinib in Myelofibrosis Patients in Lombardy, Italy
Study type: OBSERVATIONAL
Overall status: UNKNOWN
Conditions: Myelofibrosis
Keywords: Myelofibrosis
Ruxolitinib
Real world data
Lead sponsor: Ospedale di Circolo - Fondazione Macchi
Sponsor class: OTHER
First posted: 2019-05-22
Enrollment: 620
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: OTHER
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients with primary myelofibrosis diagnosis according to the WHO (World Health Organization) 2016 classification or post-essential thrombocythemia/post-polycythemia vera myelofibrosis diagnosis according to the IWG-MRT (International Working Group for Myelofibrosis Research and Treatment) 2008 classification, with an intermediate-1, intermediate-2, or high risk score according to the IPSS (International Prognostic Scoring System), treated with ruxolitinib in accordance with current indications in Italy and pertaining to centers of the regional Hematology Network of Lombardy (Rete Ematologica Lombarda), Italy.

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.


Eligibility criteria: Inclusion Criteria:

* 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

Exclusion Criteria:

* 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

Primary outcomes:
measure: Rate of infectious events after ruxolitinib exposure in myelofibrosis patients
timeFrame: Through study completion, an average of 1 year
measure: Rate of vascular events after ruxolitinib exposure in myelofibrosis patients
timeFrame: Through study completion, an average of 1 year
Secondary outcomes:
measure: Spleen response rate
timeFrame: At 3 and 6 months from ruxolitinib start
measure: Rate of primary secondary malignancies
timeFrame: Through study completion, an average of 1 year
measure: Acute myeloid leukemia transformation rate
timeFrame: Through study completion, an average of 1 year
measure: Rate of infectious events according to driver mutational status (i.e., mutations of JAK2, CALR, or MPL)
timeFrame: Through study completion, an average of 1 year
measure: Rate of vascular events according to driver mutational status (i.e., mutations of JAK2, CALR, or MPL)
timeFrame: Through study completion, an average of 1 year
measure: Spleen response rate according to driver mutational status (i.e., mutations of JAK2, CALR, or MPL)
timeFrame: Through study completion, an average of 1 year
measure: Rate of primary secondary malignancies according to driver mutational status (i.e., mutations of JAK2, CALR, or MPL)
timeFrame: Through study completion, an average of 1 year
measure: Acute myeloid leukemia transformation rate according to driver mutational status (i.e., mutations of JAK2, CALR, or MPL)
timeFrame: Through study completion, an average of 1 year
measure: Rate of infectious events according to the presence of additional mutations
timeFrame: Through study completion, an average of 1 year
measure: Rate of vascular events according to the presence of additional mutations
timeFrame: Through study completion, an average of 1 year
measure: Spleen response rate according to the presence of additional mutations
timeFrame: Through study completion, an average of 1 year
measure: Rate of primary secondary malignancies according to the presence of additional mutations
timeFrame: Through study completion, an average of 1 year
measure: Acute myeloid leukemia transformation rate according to the presence of additional mutations
timeFrame: Through study completion, an average of 1 year
measure: Evaluation of overall survival after ruxolitinib start and, if applicable, discontinuation
timeFrame: Through study completion, an average of 1 year
Other outcomes:
Locations:
city: Bergamo
country: Italy
facility: ASST Papa Giovanni XXIII
city: Brescia
country: Italy
facility: U.O. Ematologia, ASST Spedali Civili
city: Brescia
country: Italy
facility: U.S.D. Trapianti di Midollo Osseo, ASST Spedali Civili
city: Lecco
country: Italy
facility: U.O.C. di Ematologia Clinica, ASST Lecco
city: Milan
country: Italy
facility: U.O. Ematologia, ASST Fatebenefratelli-Sacco
city: Milan
country: Italy
facility: U.O. Ematologia, Fondazione IRCCS Istituto Nazionale Tumori
city: Milan
country: Italy
facility: U.O. Ematologia, Grande Ospedale Metropolitano Niguarda
city: Milan
country: Italy
facility: U.O. Ematologia, Humanicas Cancer Center
city: Milan
country: Italy
facility: U.O. Ematologia, Ospedale San Raffaele
city: Milan
country: Italy
facility: U.O. Oncoematologia, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
city: Monza
country: Italy
facility: Clinica Ematologica, Ospedale San Gerardo
city: Varese
country: Italy
facility: Ospedale di Circolo, ASST Sette Laghi
Study officials:
affiliation: Ospedale di Circolo ASST Sette Laghi, Università dell'Insubria, Varese, Italy
name: Francesco Passamonti, MD
role: PRINCIPAL_INVESTIGATOR
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