Clinical Trial: Myeloproliferative Neoplasms (MPNs) Patient Registry

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Observational [Patient Registry]

Official Title: Clinical and Molecular Epidemiology of Myeloproliferative Neoplasms (MPNs)

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 Summary:

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).


Sponsor: University Health Network, Toronto

Current Primary Outcome: Survival [ Time Frame: Bi-annually or at the time of transformation of disease, up to 10 years ]

Original Primary Outcome: Same as current

Current Secondary Outcome:

  • General patient characteristics will be captured from the Hematologic Malignacy tissue bank [ Time Frame: Bi-annually or at the time of transformation of disease, up to 10 years ]
    Type and phase of MPN, previous cancer history, age, sex
  • Disease risk score [ Time Frame: Bi-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

  • Quality of life [ Time Frame: Bi-annually or at the time of transformation of disease, up to 10 years ]
    MPN-SAF TSS questionnaire
  • Co-morbidities [ Time Frame: Bi-annually or at the time of transformation of disease, up to 10 years ]
    HCT-CI
  • Physical symptoms of MPN [ Time Frame: Bi-annually or at the time of transformation of disease, up to 10 years ]
    Physical examination: Splenomegaly and hepatomegaly, ascites, EMS, ECOG
  • MPN treatment type [ Time Frame: Bi-annually or at the time of transformation of disease, up to 10 years ]
  • Transfusion dependence [ Time Frame: Bi-annually or at the time of transformation of disease, up to 10 years ]
  • Current Blood Work [ Time Frame: Bi-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
  • Identifying MPN driver mutations by using next generation sequencing. [ Time Frame: Bi-annually or at the time of transformation of disease, up to 10 years ]
  • Bone marrow transplant details (if received) [ Time Frame: Bi-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)
  • Bone marrow transplant complications (if received) [ Time Frame: Bi-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
  • Portal hypertension [ Time Frame: Bi-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
    • Endoscopy results
  • Pulmonary hypertension [ Time Frame: Bi-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
  • Thrombosis [ Time Frame: Bi-annually or at the time of transformation of disease, up to 10 years ]
    • Details of thrombosis (type, site)
    • Treatment of thrombosis (type, duration)
  • Family history of MPN will be obtained from the patient record. [ Time Frame: Bi-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)
  • Disease progression [ Time Frame: Bi-annually or at the time of transformation of disease, up to 10 years ]
    Risk stratification (IPSS, DIPSS and DIPSS)


Original Secondary Outcome: Same as current

Information By: University Health Network, Toronto

Dates:
Date Received: March 24, 2016
Date Started: April 2016
Date Completion: October 2025
Last Updated: April 28, 2016
Last Verified: April 2016