Myeloproliferative Neoplasms (MPNs) Patient Registry

July 24, 2023 updated by: University Health Network, Toronto

Clinical and Molecular Epidemiology of Myeloproliferative Neoplasms (MPNs)

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.

Study Overview

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).

Study Type

Observational

Enrollment (Estimated)

5000

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

    • Ontario
      • Toronto, Ontario, Canada, M5G 1Z5
        • Recruiting
        • Princess Margaret Cancer Centre
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

Patients at Princess Margaret Cancer Centre with an MPN diagnosis who consent to inclusion in this registry.

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Cohort
  • Time Perspectives: Other

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
Patients with a diagnosis of MPN

Patients with a myeloproliferative neoplasm (MPN) diagnosis:

Atypical chronic myeloid leukemia (aCML), chronic eosinophilic leukemia-not otherwise specified (CEL NOS), chronic myelomonocytic leukemia (CMML), chronic neutrophilic leukemia (CNL), polycythemia vera (PV), essential thrombocythemia (ET), JMML, mastocytosis, MPN unclassifiable, myeloproliferative neoplasm/myelodysplastic syndrome unclassifiable (MPN/MDS unclassifiable), primary myelofibrosis (PMF), post-ET MF, post-PV MF, or (refractory anemia with ringed sideroblasts associated with marked thrombocytosis) RARS-T

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Survival
Time Frame: Annually or at the time of transformation of disease, up to 10 years
Survival of patients with MPN
Annually or at the time of transformation of disease, up to 10 years

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
General patient characteristics will be captured from the Hematologic Malignancy tissue bank
Time Frame: Annually or at the time of transformation of disease, up to 10 years
Type and phase of MPN, previous cancer history, age, sex
Annually or at the time of transformation of disease, up to 10 years
Disease risk score
Time Frame: Annually or at the time of transformation of disease, up to 10 years

Risk stratification (IPSS, DIPSS and DIPSS)

o Details of transformation to accelerated/phase phase disease

Annually or at the time of transformation of disease, up to 10 years
Quality of life - Neoplasm Symptom
Time Frame: Annually or at the time of transformation of disease, up to 10 years
MPN-SAF TSS questionnaire
Annually or at the time of transformation of disease, up to 10 years
Co-morbidities
Time Frame: Annually or at the time of transformation of disease, up to 10 years
HCT-CI
Annually or at the time of transformation of disease, up to 10 years
Physical symptoms of MPN
Time Frame: Annually or at the time of transformation of disease, up to 10 years
Physical examination: Splenomegaly and hepatomegaly, ascites, EMS, ECOG
Annually or at the time of transformation of disease, up to 10 years
MPN treatment type received
Time Frame: Annually or at the time of transformation of disease, up to 10 years
Medical therapies received
Annually or at the time of transformation of disease, up to 10 years
Transfusion dependence status
Time Frame: Annually or at the time of transformation of disease, up to 10 years
Transfusion status
Annually or at the time of transformation of disease, up to 10 years
Current Blood Work
Time Frame: Annually or at the time of transformation of disease, up to 10 years
CBC, INR, PT, APTT, fibrinogen, creatinine, ALP, ALT, AST, GGT, total bilirubin, LDH, urate, CRP, erythropoietin, hepatitis B and HIV
Annually or at the time of transformation of disease, up to 10 years
Identifying MPN driver mutations by using next generation sequencing.
Time Frame: Annually or at the time of transformation of disease, up to 10 years
Next generation sequencing gene panel
Annually or at the time of transformation of disease, up to 10 years
Bone marrow transplant details (if received)
Time Frame: Annually or at the time of transformation of disease, up to 10 years

Details of recipient (CMV status, ABO blood group)

  • Details of donor (gender, CMV status, ABO blood group)
  • Disease status at time of transplant (blood work disease status)
  • Transplant details (stem cell source, HLA matching, conditioning intensity & regimen, serotherapy, GVHD prophylaxis)
Annually or at the time of transformation of disease, up to 10 years
Bone marrow transplant complications (if received)
Time Frame: Annually or at the time of transformation of disease, up to 10 years
Toxicities, engraftment and chimerism, GVHD, significant infections in the first 100 days
Annually or at the time of transformation of disease, up to 10 years
Portal hypertension
Time Frame: Annually or at the time of transformation of disease, up to 10 years

Presence and details of ascites, GIT bleeding, esophageal & gastric varices, cirrhosis and portal hypertensive gastropathy

o Endoscopy results

Annually or at the time of transformation of disease, up to 10 years
Pulmonary hypertension
Time Frame: Annually or at the time of transformation of disease, up to 10 years
WHO classification, echocardiogram results, CNP, troponin, pulmonary function tests, 6 minute walk test distance, blood gas, treatment, complications
Annually or at the time of transformation of disease, up to 10 years
Thrombosis
Time Frame: Annually or at the time of transformation of disease, up to 10 years

Details of thrombosis (type, site)

o Treatment of thrombosis (type, duration)

Annually or at the time of transformation of disease, up to 10 years
Family history of MPN will be obtained from the patient record.
Time Frame: Annually or at the time of transformation of disease, up to 10 years
Relative affected (e.g. daughter, uncle, mother), details of MPN (type, phase, treatment received)
Annually or at the time of transformation of disease, up to 10 years
Disease progression
Time Frame: Annually or at the time of transformation of disease, up to 10 years
Risk stratification (IPSS, DIPSS and DIPSS)
Annually or at the time of transformation of disease, up to 10 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Investigators

  • Principal Investigator: Vikas Gupta, MD, University Health Network, Toronto

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

April 1, 2016

Primary Completion (Estimated)

October 1, 2025

Study Completion (Estimated)

October 1, 2025

Study Registration Dates

First Submitted

March 24, 2016

First Submitted That Met QC Criteria

April 28, 2016

First Posted (Estimated)

May 3, 2016

Study Record Updates

Last Update Posted (Actual)

July 25, 2023

Last Update Submitted That Met QC Criteria

July 24, 2023

Last Verified

July 1, 2023

More Information

Terms related to this study

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

De-identified participant data may be shared for REB approved research studies. Only the data necessary to achieve the study's aims will be shared and all data will be de-identified prior to sharing.

IPD Sharing Time Frame

Continuing

IPD Sharing Access Criteria

De-identified participant data may only be shared for REB approved research studies.

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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