Myelofibrosis
Conditions
Keywords
Myelofibrosis, Ruxolitinib, Real world data
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.
Interventions
DRUGRuxolitinib
Observational study including patients with myelofibrosis being treated with ruxolitinib in a real world setting. Patients are treated according to current indications.
Sponsors
Margherita Maffioli
Study design
Observational model
COHORT
Time perspective
OTHER
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | 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
| Measure | Time frame | Description |
|---|---|---|
| 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) | 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 |
| 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 |
| Spleen response rate | At 3 and 6 months from ruxolitinib start | — |
| 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 |
| 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 | — |
| 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 primary secondary malignancies | Through study completion, an average of 1 year | — |
Countries
Italy
Contacts
Primary ContactMargherita Maffioli, MD
Backup ContactFrancesco Passamonti, MD
Outcome results
None listed