Pilot Study for the Development of a Diagnostic Score to Differentiate Myeloproliferative Neoplasms.

Essential Thrombocythemia Clinical Trial

— BioScoreSMP  

Official title:

Pilot Study for the Development of a Non-invasive Diagnostic Score to Differentiate Between Essential Thrombocythemia, Premyelofibrosis and Myelofibrosis

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT03869476
• Other study ID #: 49RC18_0153_1
• Status: Recruiting
• Phase: N/A
• Start date: July 30, 2019
• Est. completion date: January 30, 2024

Study information

• Verified date: September 2023
• Source: University Hospital, Angers
• Contact: Damien Luque Paz, Pharm.D.
• Phone: 0241355590
• Email: damien.luquepaz[@]chu-angers.fr
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

Prospective study for the development of a non-invasive score for differentiating prefibrotic myelofibrosis from essential thrombocytosis and overt myelofibrosis.

Description:

The diagnostic criteria for myeloproliferative neoplasia have recently been revised in the WHO 2016 classification, which now recognizes a new entity between essential thrombocythemia (TE) and overt myelofibrosis (or primary myelofibrosis): prefibrotic myelofibrosis. Prefibrotic myelofibrosis patients seem to have a poorer prognosis compared to essential thrombocythemia in term of overall survival and myelofibrotic evolution. The dichotomy between these diseases is based on the bone marrow biopsy evaluation and is challenging with a lack of reproducibility showed in the literature.

This study aims to develop a diagnostic non-invasive score for differentiating prefibrotic myelofibrosis from essential thrombocytosis and overt myelofibrosis.

The parameters studied are :

• spleen fibrosis by elastography

• plasma cytokines levels

• mutationnal landscape by NGS

• CD34 circulating cells

Bone marrow biopsy were reviewed by 2 anatomopathologists. Number of patients to be enrolled : 130

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 130
• Est. completion date: January 30, 2024
• Est. primary completion date: January 30, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 99 Years

Eligibility

Inclusion Criteria:

• Adult (18 years of age or older) with the diagnosis of essential thrombocytosis, prefibrotic myelofibrosis or overt myelofibrosis according to WHO 2016 criteria;

• Patient who has not received specific treatment for neoplasms;

• Patient who has signed the consent to participate in the study;

• Patient who has consented to be included in the "Malignant Haemopathies" collection at the University Hospital of Angers or Brest and for whom the samples necessary for the study are available in the biocollection.

Exclusion Criteria:

• Patient with another hematologic neoplasm or progressive cancer at the time of diagnosis;

• Patient with a diagnosis of secondary myelofibrosis;

• Pregnant or breastfeeding woman;

• Person prived of liberty by judicial or administrative decision;

• Person subject to a legal protection measure;

Related Conditions & MeSH terms

• Essential Thrombocythemia
• Myeloproliferative Disorder
• Myeloproliferative Disorders
• Primary Myelofibrosis
• Primary Myelofibrosis, Fibrotic Stage
• Primary Myelofibrosis, Prefibrotic Stage
• Thrombocythemia, Essential
• Thrombocytosis

Intervention

Diagnostic Test:
• Non-invasive diagnosis score
spleen fibrosis by elastography plasma cytokines levels mutationnal landscape by NGS CD34 circulating cells

Locations

Country	Name	City	State
France	Chu Angers	Angers
France	CHRU de Brest - Hôpital Morvan	Brest

Sponsors (1)

Lead Sponsor	Collaborator
University Hospital, Angers

Country where clinical trial is conducted

France, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	WHO 2016 criteria for prefibrotic myelofibrosis, essential thrombocytosis and overt myelofibrosis diagnosis	Assessment of the non-invasive diagnostic score against the WHO diagnosis	At the time of diagnosis : disease classification