Identification of Factors Associated With Treatment Response in Patients With Polycythemia Vera, Essential Thrombocythemia, and Pre-myelofibrosis.

NCT05440838 | Recruiting

• 1 other identifier: 2022-A01044-39
• Study Type: observational
• Target: 120
• Locations: 1 country
• Sites: 6

Brief Summary

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.

Conditions

Myeloproliferative Neoplasm

Outcome Measures

Primary Outcomes (1)

• Complete hematological response

  ELN-2013 criteria by meeting all of the following: * Durable resolution of disease-related signs including palpable hepatosplenomegaly, large symptoms improvement, AND * Durable peripheral blood count remission, defined as: platelet count ≤400 ×109/L, WBC count \<10 × 109/L, Ht lower than 45% without phlebotomies (for PV patients), absence of leukoerythroblastosis, AND * Without signs of progressive disease, and absence of any hemorrhagic or thrombotic events.

  12 months

Secondary Outcomes (2)

• Complete hematological response

  24, 36, 48, and 60 months

• Molecular response

  12 and 24 months

Interventions

Next-generation sequencing DIAGNOSTIC_TEST

Next-generation sequencing and cytokine profile will be established in all patients before the start of treatment.

Also known as: Cytokines

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Adults with polycythemia vera, essential thrombocythemia, or pre-myelofibrosis requiring first-line treatment with hydroxyurea or pegylated interferon.

You may qualify if:

• 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.

You may not qualify if:

• Previous treatment.
• Other on-going malignancy, including overt myelofibrosis.
• Other treatment such as phlebotomy solely, ruxolitinib, anagrelide, or pipobroman.

Sponsors & Collaborators

• University Hospital, Brest: collaborator
• Nantes University Hospital: collaborator
• Poitiers University Hospital: collaborator
• Rennes University Hospital: collaborator
• University Hospital, Tours: collaborator
• University Hospital, Angers: lead

Study Sites (6)

Chu Angers

Angers, France

RECRUITING

Chu Brest

Brest, 29606, France

RECRUITING

Chu Nantes

Nantes, 44093, France

RECRUITING

Chu Poitiers

Poitiers, 86021, France

RECRUITING

Chu Rennes

Rennes, 35033, France

RECRUITING

Chu Tours

Tours, 37044, France

RECRUITING

MeSH Terms

Conditions

Myeloproliferative Disorders

Condition Hierarchy (Ancestors)

Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases

Central Study Contacts

CORENTIN ORVAIN, DOCTOR

CONTACT

+3302 41 35 44 75; Corentin.Orvain@chu-angers.fr

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER GOV
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 10, 2022
• First Posted: July 1, 2022
• Study Start: May 17, 2023
• Primary Completion (Estimated): February 17, 2027
• Study Completion (Estimated): February 17, 2032
• Last Updated: March 6, 2026

Record last verified: 2026-03

Locations

FR (6)