Identification of Factors Associated With Treatment Response in Patients With Polycythemia Vera, Essential Thrombocythemia, and Pre-myelofibrosis.
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Next-generation sequencing.
- Who it may be relevant to
- Registry conditions: Myeloproliferative Neoplasm. Basic parameters: from 18 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- France
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Overview
First-line treatment for patients with polycythemia vera, essential thrombocythemia, and pre-myelofibrosis is based on hydroxyurea or pegylated interferon. The objective of treatment is to prevent thrombotic complications and leukemic transformation. Despite overall good response rates, some patients do not respond to treatment and others lose their response over time. Both situations are associated with worse survival and there are to date no clear predictive factors for response although the existence of additional mutations seems unfavorable. In this exploratory study, we hypothesize that biological factors at diagnosis are associated with hematological response at 12 months. We will more specifically study the association between mutational profile, assessed by next-generation sequencing, and cytokine profile with hematological response. This study will help in identifying patients who will not respond to hydroxyurea or pegylated interferon and give the opportunity to try other treatments upfront, in the perspective of precision medicine. On the basic science side, this study will help in understanding the molecular and immunological factors involved in resistance to treatment.
Interventions
- Diagnostic test Next-generation sequencing
Next-generation sequencing and cytokine profile will be established in all patients before the start of treatment.
Primary outcome measures
- Complete hematological response [Time frame: 12 months]
Secondary outcome measures (2)
- Complete hematological response [Time frame: 24, 36, 48, and 60 months]
- Molecular response [Time frame: 12 and 24 months]
Eligibility 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.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Cohort
Study locations
France · 6 centers
- Chu Angers — Angers
- Chu Brest — Brest
- Chu Nantes — Nantes
- Chu Poitiers — Poitiers
- Chu Rennes — Rennes
- Chu Tours — Tours
Identifiers
NCT: NCT05440838 · 2022-A01044-39