Ruxolitinib in Myelofibrosis Patients in Lombardy, Italy

Study Overview
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Brief Summary
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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.

Official Title
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Observational, Retrospective and Prospective Study on the Use of Ruxolitinib in Myelofibrosis Patients in Lombardy, Italy

Conditions
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Myelofibrosis

Intervention / Treatment
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* Drug: Ruxolitinib


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Intervention/Treatment |
| --- |
|Drug: Ruxolitinib|Observational study including patients with myelofibrosis being treated with ruxolitinib in a real world setting. Patients are treated according to current indications.|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Rate of infectious events after ruxolitinib exposure in myelofibrosis patients |  | Through study completion, an average of 1 year | 
| Rate of vascular events after ruxolitinib exposure in myelofibrosis patients |  | Through study completion, an average of 1 year | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Spleen response rate |  | At 3 and 6 months from ruxolitinib start | 
| Rate of primary secondary malignancies |  | Through study completion, an average of 1 year | 
| Acute myeloid leukemia transformation rate |  | Through study completion, an average of 1 year | 
| Rate of infectious events according to driver mutational status (i.e., mutations of JAK2, CALR, or MPL) | Association of rate of infectious events with driver mutational status | Through study completion, an average of 1 year | 
| Rate of vascular events according to driver mutational status (i.e., mutations of JAK2, CALR, or MPL) | Association of rate of vascular events with driver mutational status | Through study completion, an average of 1 year | 
| Spleen response rate according to driver mutational status (i.e., mutations of JAK2, CALR, or MPL) | Association of spleen response rate with driver mutational status | 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) | Association of rate of primary secondary malignancies with driver mutational status | Through study completion, an average of 1 year | 
| Acute myeloid leukemia transformation rate according to driver mutational status (i.e., mutations of JAK2, CALR, or MPL) | Association of acute myeloid leukemia transformation rate with driver mutational status | Through study completion, an average of 1 year | 
| Rate of infectious events according to the presence of additional mutations | Association of rate of infectious events with the presence of additional mutations | Through study completion, an average of 1 year | 
| Rate of vascular events according to the presence of additional mutations | Association of rate of vascular events with the presence of additional mutations | Through study completion, an average of 1 year | 
| Spleen response rate according to the presence of additional mutations | Association of spleen response rate with the presence of additional mutations | Through study completion, an average of 1 year | 
| Rate of primary secondary malignancies according to the presence of additional mutations | Association of rate of primary secondary malignancies with the presence of additional mutations | Through study completion, an average of 1 year | 
| Acute myeloid leukemia transformation rate according to the presence of additional mutations | Association of acute myeloid leukemia transformation rate with the presence of additional mutations | Through study completion, an average of 1 year | 
| Evaluation of overall survival after ruxolitinib start and, if applicable, discontinuation |  | Through study completion, an average of 1 year | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Myelofibrosis, Ruxolitinib, Real world data