Clinical trial NCT02760238: Myeloproliferative Neoplasms (MPNs) Patient Registry
Official title: Clinical and Molecular Epidemiology of Myeloproliferative Neoplasms (MPNs)
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Primary Myelofibrosis
Polycythemia Vera
Essential Thrombocythemia
Mastocytosis
Leukemia, Myeloid, Chronic, Atypical, BCR-ABL Negative
Leukemia, Myelomonocytic, Juvenile
Chronic Eosinophilic Leukemia-not Otherwise Specified
Myelodysplastic-Myeloproliferative Diseases
Neoplasms
Leukemia, Myelomonocytic, Chronic
Keywords: myeloproliferative neoplasm
myelodysplastic syndrome
registry
leukemia
Bone Marrow Diseases
Hematologic Diseases
Neoplasms
Lead sponsor: University Health Network, Toronto
Sponsor class: OTHER
First posted: 2016-05-03
Enrollment: 5000
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: OTHER
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients at Princess Margaret Cancer Centre with an MPN diagnosis who consent to inclusion in this registry.

Brief summary: The mandate of this MPN registry is to collect clinical information, including molecular results, from consenting patients with a variety of MPNs at different time points during the course of their disease.

Detailed description: The myeloproliferative neoplasms (MPNs) are a group of rare hematological malignancies in which the bone marrow cells that produce the body's blood cells develop and function abnormally.

Despite the gains that have already been made in understanding and treatment of MPNs there is much that can still be learned. This registry will establish a clinical annotation database would help to better understand this group of diseases and to more effectively assign individual patients to the optimal therapy and so, improve their outcomes. This project will provide new insights on the molecular profiling of patients with MPN. It will be used as future resource for observational studies related to MPN.

The registry involves the collection of clinical information from patients with diagnosis of MPN at different time points during the course of their disease. The clinical data is collected following written informed consent from the Hematologic Malignancy tissue bank (UHN REB 01-0573C).

Data collected includes: a range of clinical measures, disease-associated factors, details of treatment and its results, complications during treatment, molecular and cytogenetic data, symptom assessment and survival outcome (up to 10 years).

Data will be collected prospectively and retrospectively, in both cases after obtaining written informed consent as per the study standard operating procedure (SOP).

Eligibility criteria: Inclusion Criteria:

Diagnosis of one of the following myeloproliferative neoplasms (MPNs):

* Atypical CML (aCML)
* Chronic eosinophilic leukemia-not otherwise specified (CEL, NOS),
* Chronic myelomonocytic leukemia (CMML)
* Chronic neutrophilic leukemia (CNL),
* Essential thrombocythemia (ET),
* Juvenile myelomonocytic leukemia (JMML),
* Mastocytosis, MPN unclassifiable
* MPN/MDS unclassifiable,
* Primary myelofibrosis (PMF),
* Post-essential thrombocythemia myelofibrosis (post-ET MF),
* Post-polycythemia vera MF (post-PV MF)
* Refractory anemia with ringed sideroblasts associated with marked thrombocytosis (RARS-T)

Exclusion Criteria:

* None

Primary outcomes:
measure: Survival
timeFrame: Annually or at the time of transformation of disease, up to 10 years
Secondary outcomes:
measure: General patient characteristics will be captured from the Hematologic Malignancy tissue bank
timeFrame: Annually or at the time of transformation of disease, up to 10 years
measure: Disease risk score
timeFrame: Annually or at the time of transformation of disease, up to 10 years
measure: Quality of life - Neoplasm Symptom
timeFrame: Annually or at the time of transformation of disease, up to 10 years
measure: Co-morbidities
timeFrame: Annually or at the time of transformation of disease, up to 10 years
measure: Physical symptoms of MPN
timeFrame: Annually or at the time of transformation of disease, up to 10 years
measure: MPN treatment type received
timeFrame: Annually or at the time of transformation of disease, up to 10 years
measure: Transfusion dependence status
timeFrame: Annually or at the time of transformation of disease, up to 10 years
measure: Current Blood Work
timeFrame: Annually or at the time of transformation of disease, up to 10 years
measure: Identifying MPN driver mutations by using next generation sequencing.
timeFrame: Annually or at the time of transformation of disease, up to 10 years
measure: Bone marrow transplant details (if received)
timeFrame: Annually or at the time of transformation of disease, up to 10 years
measure: Bone marrow transplant complications (if received)
timeFrame: Annually or at the time of transformation of disease, up to 10 years
measure: Portal hypertension
timeFrame: Annually or at the time of transformation of disease, up to 10 years
measure: Pulmonary hypertension
timeFrame: Annually or at the time of transformation of disease, up to 10 years
measure: Thrombosis
timeFrame: Annually or at the time of transformation of disease, up to 10 years
measure: Family history of MPN will be obtained from the patient record.
timeFrame: Annually or at the time of transformation of disease, up to 10 years
measure: Disease progression
timeFrame: Annually or at the time of transformation of disease, up to 10 years
Other outcomes:
Locations:
city: Toronto
country: Canada
facility: Princess Margaret Cancer Centre
state: Ontario
Study officials:
affiliation: University Health Network, Toronto
name: Vikas Gupta, MD
role: PRINCIPAL_INVESTIGATOR
References: