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

Sponsor

University Hospital, Angers (Other)

Overall Status

Recruiting

CT.gov ID

NCT03869476

Collaborator

(none)

130

Enrollment

Locations

Arm

Anticipated Duration (Months)

Patients Per Site

1.4

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

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

Condition or Disease	Intervention/Treatment	Phase
Myeloproliferative Disorder Essential Thrombocythemia Primary Myelofibrosis, Prefibrotic Stage Primary Myelofibrosis, Fibrotic Stage	Diagnostic Test: Non-invasive diagnosis score	N/A

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

Study Design

Study Type:

Interventional

Anticipated Enrollment :

130 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Diagnostic

Official Title:

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

Actual Study Start Date :

Aug 30, 2019

Anticipated Primary Completion Date :

Jun 1, 2023

Anticipated Study Completion Date :

Jun 1, 2023

Arms and Interventions

Arm	Intervention/Treatment
Experimental: BioscoreSMP cohort	Diagnostic Test: Non-invasive diagnosis score spleen fibrosis by elastography plasma cytokines levels mutationnal landscape by NGS CD34 circulating cells

Arm

Intervention/Treatment

Experimental: BioscoreSMP cohort

Diagnostic Test: Non-invasive diagnosis score

spleen fibrosis by elastography plasma cytokines levels mutationnal landscape by NGS CD34 circulating cells

Outcome Measures

Primary Outcome Measures

WHO 2016 criteria for prefibrotic myelofibrosis, essential thrombocytosis and overt myelofibrosis diagnosis [At the time of diagnosis : disease classification]
Assessment of the non-invasive diagnostic score against the WHO diagnosis

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years to 99 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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;

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Chu Angers	Angers		France	49000
2	CHRU de Brest - Hôpital Morvan	Brest		France	29100

Sponsors and Collaborators

University Hospital, Angers

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

Responsible Party:

University Hospital, Angers

ClinicalTrials.gov Identifier:

NCT03869476

Other Study ID Numbers:

49RC18_0153_1

First Posted:

Mar 11, 2019

Last Update Posted:

Sep 12, 2019

Last Verified:

Sep 1, 2019

Individual Participant Data (IPD) Sharing Statement:

Yes

Plan to Share IPD:

Yes

Studies a U.S. FDA-regulated Drug Product:

Studies a U.S. FDA-regulated Device Product:

Additional relevant MeSH terms:

Primary Myelofibrosis

Thrombocytosis

Thrombocythemia, Essential

Myeloproliferative Disorders

Study Results

No Results Posted as of Sep 12, 2019