Predicting response to hydroxyurea or pegylated interferon in polycythemia vera, essential thrombocythemia, and pre-myelofibrosis
Identification of Factors Associated With Treatment Response in Patients With Polycythemia Vera, Essential Thrombocythemia, and Pre-myelofibrosis.
We will test whether genetic mutations and cytokine levels at diagnosis can predict who will respond to hydroxyurea or pegylated interferon in people with polycythemia vera, essential thrombocythemia, or pre-myelofibrosis.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 120 (estimated) |
| Ages | 18 Years and up |
| Sex | All |
| Sponsor | University Hospital, Angers Government |
| Drugs / interventions | ruxolitinib |
| Locations | 6 sites (Angers and 5 other locations) |
| Trial ID | NCT05440838 on ClinicalTrials.gov |
What this trial studies
This observational, exploratory project will enroll adults with polycythemia vera, essential thrombocythemia, or pre-myelofibrosis who are starting first-line hydroxyurea or pegylated interferon. Researchers will use next-generation sequencing to characterize mutational profiles and measure cytokine levels at diagnosis, then follow hematological response at 12 months. Statistical analyses will examine associations between genetic/cytokine markers and response or loss of response. The study excludes patients with prior cytoreductive therapy, overt myelofibrosis, or other active malignancies and is conducted across several French university hospitals.
Who should consider this trial
Good fit: Adults with polycythemia vera, essential thrombocythemia, or pre-myelofibrosis who are treatment-naïve and preparing to start first-line hydroxyurea or pegylated interferon, able to consent and affiliated to social security.
Not a fit: Patients who have received prior cytoreductive treatment, have overt myelofibrosis or another active cancer, or are being treated only with phlebotomy or with drugs such as ruxolitinib, anagrelide, or pipobroman are unlikely to benefit from this protocol.
Why it matters
Potential benefit: If successful, this could identify patients unlikely to respond to hydroxyurea or pegylated interferon so alternative treatments can be considered earlier.
How similar studies have performed: Retrospective work has linked additional somatic mutations to poorer outcomes, but prospective evidence that mutational or cytokine profiles reliably predict first-line treatment response is limited, so this approach remains partly supported but exploratory.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Adults with polycythemia vera, essential thrombocythemia, or pre-myelofibrosis. * Indication for first-line treatment with hydroxyurea or pegylated interferon. * Consent to participate. * Affiliated to social security. Exclusion Criteria: * Previous treatment. * Other on-going malignancy, including overt myelofibrosis. * Other treatment such as phlebotomy solely, ruxolitinib, anagrelide, or pipobroman.
Where this trial is running
Angers and 5 other locations
- Chu Angers — Angers, France (Recruiting)
- Chu Brest — Brest, France (Recruiting)
- Chu Nantes — Nantes, France (Recruiting)
- Chu Poitiers — Poitiers, France (Recruiting)
- Chu Rennes — Rennes, France (Recruiting)
- Chu Tours — Tours, France (Recruiting)
Study contacts
- Study coordinator: Corentin Orvain, Doctor
- Email: Corentin.Orvain@chu-angers.fr
- Phone: +3302 41 35 44 75
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.