Clinical trial NCT03869476: Pilot Study for the Development of a Diagnostic Score to Differentiate Myeloproliferative Neoplasms.
Official title: Pilot Study for the Development of a Non-invasive Diagnostic Score to Differentiate Between Essential Thrombocythemia, Premyelofibrosis and Myelofibrosis
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: NA
Conditions: Myeloproliferative Disorder
Essential Thrombocythemia
Primary Myelofibrosis, Prefibrotic Stage
Primary Myelofibrosis, Fibrotic Stage
Lead sponsor: University Hospital, Angers
Sponsor class: OTHER_GOV
First posted: 2019-03-11
Enrollment: 133
Sex: ALL
Minimum age (years): 18
Maximum age (years): 99
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: DIAGNOSTIC

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

Detailed 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

Eligibility criteria: 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;

Primary outcomes:
measure: WHO 2016 criteria for prefibrotic myelofibrosis, essential thrombocytosis and overt myelofibrosis diagnosis
timeFrame: At the time of diagnosis : disease classification
Secondary outcomes:
Other outcomes:
Locations:
city: Angers
country: France
facility: Chu Angers
city: Brest
country: France
facility: CHRU de Brest - Hôpital Morvan
Study officials:
References: