Clinical and Therapeutic Impact of Molecular Markers in Myeloproliferative Disorders
- Conditions
- Myeloproliferative Neoplasms
- Registration Number
- NCT02823210
- Lead Sponsor
- Assistance Publique - Hôpitaux de Paris
- Brief Summary
Myeloproliferative neoplasms (MPN) are clonal hematopoietic disorders sharing a common natural evolution: a chronic phase, characterized by a major risk of vascular events, followed by an accelerated phase eventually leading to transformation to acute leukemia. MPN include polycythemia vera, essential thrombocythemia, primary myelofibrosis, and rarer entities. During the past years, CML became a paradigm for targeted therapy and personalized cancer medicine. For other MPNs, the discovery of the JAK2V617F mutation followed by many other mutations, opened similar perspectives. However, several questions remain to be answered in MPNs regarding the clinical implication of these major scientific discoveries: what is the clinical impact of JAK2V617F and other molecular biomarkers on the risks of complications and progression? Can these new biomarkers be used in the perspective of a personalized therapy of MPNs? his project will focus on the qualification of a series of known mutations as biomarkers in MPNs based on large multicenter cohorts of patients with well-annotated samples
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- UNKNOWN
- Sex
- All
- Target Recruitment
- 300
- Patients suffering from Myeloproliferative neoplasms (MPN) diagnosed between 2005 and 2013
- Sample DNA diagnostics available: 500 mcg
- Untreated or treated with hydroxyurea, ruxolitinib, alpha interferon,
- Patient has given his(her) own consent for the use of the sample for research on the pathology and genetic analyzes
- Refused to participate
- Patient treated with another molecule that hydroxyurea, ruxolitinib, or alpha interferon
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method cumulative incidence or progression inclusion
- Secondary Outcome Measures
Name Time Method Treatment response/resistance 3 years Disease phenotype according to WHO classification 3 years polycythemia vera, essential thrombocythemia, primary myelofibrosis, and others
Mean life-years gained 3 years The analysis will take into consideration the cost of testing, but also the costs of different treatment options with or without testing, and other disease-related costs dependent on treatment
Quality-adjusted life years gained 3 years
Trial Locations
- Locations (1)
Centre d'Investigations Cliniques
🇫🇷Paris, France