Symptoms and General Pathology

All Conditions

Blood and Lymph Conditions

Heart and Blood Diseases

Rare Diseases

Syndrome

Myeloproliferative Disorders

Primary Myelofibrosis

Thrombocytosis

Thrombocythemia, Essential

Bone Marrow Diseases

Hematologic Diseases

Blood Platelet Disorders

Hemostatic Disorders

Blood Coagulation Disorders

Hemorrhagic Disorders

Chronic Myeloproliferative Disorders

Essential Thrombocythemia

* DNA extraction from blood sample for CALR mutation quantification (fragment analysis)
* at diagnosis and follow-up (inclusion period: 3 years)
* max 1 sample/year
* secondary outcome: mutational landscape by Next Generation Sequencing (NGS) analysis at diagnosis

CALR allele burden quantification

Laurane COTTIN, Doctor

Emma BLANCHET

Laurane COTTIN, Dr

Prospective study to evaluate the relevance of CALR allele burden monitoring as a molecular marker of disease progression.

A first local study on 45 patients showed the prognostic impact of CALR mutation quantification in follow-up, independently of the European LeukemiaNet (ELN) prognostic score validated in this group of patients.

This study aims to evaluate a multicenter cohort of 260 patients, including all types of CALR-mutated MPNs and several follow-up samples, to model the temporal evolution of CALR allele burden.

Blood of MPN patients will be collected, at the time of diagnosis and for 3 years (max 1 sample/year), for the quantification of the CALR allele burden. During follow-up, a clinicobiological score to define the progression or not of the disease for each patient will be evaluated in Essential Thrombocythemia (ET) and MyeloFibrosis (MF).

Interest of CALR Allele Burden in Diagnosis and Follow-up of Patients With CALR Mutated Myeloproliferative Syndromes (CALRSUIVI)

Clinicobiological score :

For ET, disease progression if ≥ 1 of:

* leukocytosis \> 12 G/L or myelemia \> 10% or erythroblasts \> 1%,
* anemia or thrombocytopenia not related to drug toxicity,
* development or worsening of splenomegaly,
* development of thrombocytosis (platelets \> 450 G/L) on cytoreductive therapy,
* poor disease control (at least one change in therapy for reasons other than adverse events),
* hematologic transformation or death related to hematologic pathology.

For MF, disease progression if ≥ 1 of:

* anemia \< 100 g/L, neutropenia \< 1 G/L, thrombocytopenia \< 100 G/L and/or development of general signs not previously present or recurring after improvement,
* increase in leukocytosis \> 25 G/L not previously present,
* appearance or aggravation of transfusion dependence,
* appearance (\> 5 cm) or aggravation (\> 50%) of splenomegaly,
* leukemic transformation or death related to the hematologic pathology.

Ligue contre le cancer, France

University Hospital, Angers

Neoplasms

Polycythemia Vera

Polycythemia

Bone Marrow Neoplasms

Hematologic Neoplasms

Harinder Gill, MD

Garret Leung, MD

Yok-lam Kwong, MD

This is a multinational, multicenter, prospective and retrospective, observational, cohort study of patients with myeloproliferative neoplasm.

This is a multinational, multicenter, prospective and retrospective, observational, cohort registry of patients with MPN. The registry mainly involves study centres in Asia including but not limited to Hong Kong, Singapore, Malaysia, Taiwan, Korea and Thailand. Clinicopathologic characteristics, cytogenetic characteristics, mutational characteristics, treatment characteristics, outcomes and survivals of Asian patients MPN diagnosed between 2010-2025 will be collected to establish clinical registry.

Data on the clinicopathologic characteristics, cytogenetics, mutational profile, prognostic scoring treatment characteristics, responses to treatment, outcome and survivals will be collected through routine clinic visits and/or reviewing medical records. Data will be collected at diagnosis, and approximately every 6 months thereafter (for prospective data) and at progression until death/study termination.

Peripheral blood and bone marrow aspirate

Asian Myeloproliferative Neoplasm (MPN) Registry

Asian Myeloproliferative Neoplasm (MPN) Registry - An Asian Myeloid Working Group (AMWG) Project.

Measured in months, from date of diagnosis of MPN to date of documentation of secondary myelofibrosis (event), death (event) or latest follow-up (censor). Patient without an event will be censored at date last known to be progression-free

Measured in months, from date of diagnosis of MPN to the date of the first MDS or AML or death due to any cause. Patient without an event will be censored at date last known to be progression-free and alive.

Measured in months from the date of diagnosis of MDS until the date of death due to any cause. Patient who is alive will be censored at the date last known alive

Novartis

The University of Hong Kong

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

Non-invasive diagnosis score

Damien Luque Paz, Pharm.D.

Corentin Orvain, M.D.

Damien Luque Paz

Corentin Orvain

Jean-Christophe Ianotto

Eric Lippert

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

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

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

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

Data of study protocol, Statiscal analysis plan and the infromed consent form will be made available after the study completion

Assessment of the non-invasive diagnostic score against the WHO diagnosis

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
Interest of CALR Allele Burden in Diagnosis and Follow-up of Patients With CALR Mutated Myeloproliferative Syndromes (CALRSUIVI)	NCT04942080	Myeloproliferat... Essential Throm... Primary Myelofi... Primary Myelofi...	CALR allele bur...	18 Years -	University Hospital, Angers
Asian Myeloproliferative Neoplasm (MPN) Registry	NCT05882773	Myeloproliferat... Polycythemia Ve... Essential Throm... Post-polycythem... Post-essential ... Primary Myelofi... Primary Myelofi... Primary Myelofi...		18 Years -	The University of Hong Kong
Interest of CALR Allele Burden in Diagnosis and Follow-up of Patients With CALR Mutated Myeloproliferative Syndromes (CALRSUIVI)	NCT04942080	Myeloproliferat... Essential Throm... Primary Myelofi... Primary Myelofi...	CALR allele bur...	18 Years -	University Hospital, Angers
Interest of CALR Allele Burden in Diagnosis and Follow-up of Patients With CALR Mutated Myeloproliferative Syndromes (CALRSUIVI)	NCT04942080	Myeloproliferat... Essential Throm... Primary Myelofi... Primary Myelofi...	CALR allele bur...	18 Years -	University Hospital, Angers
Pilot Study for the Development of a Diagnostic Score to Differentiate Myeloproliferative Neoplasms.	NCT03869476	Myeloproliferat... Essential Throm... Primary Myelofi... Primary Myelofi...	Non-invasive di...	18 Years - 99 Years	University Hospital, Angers

Spots Global Cancer Trial Database for primary myelofibrosis, fibrotic stage