Myeloproliferative Neoplasms (MPNs) Patient Registry
Study Details
Study Description
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.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
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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 Design
Arms and Interventions
| Arm | 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 |
Other: Observational
|
Outcome Measures
Primary Outcome Measures
- Survival [Annually or at the time of transformation of disease, up to 10 years]
Survival of patients with MPN
Secondary Outcome Measures
- General patient characteristics will be captured from the Hematologic Malignancy tissue bank [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 [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 - Neoplasm Symptom [Annually or at the time of transformation of disease, up to 10 years]
MPN-SAF TSS questionnaire
- Co-morbidities [Annually or at the time of transformation of disease, up to 10 years]
HCT-CI
- Physical symptoms of MPN [Annually or at the time of transformation of disease, up to 10 years]
Physical examination: Splenomegaly and hepatomegaly, ascites, EMS, ECOG
- MPN treatment type received [Annually or at the time of transformation of disease, up to 10 years]
Medical therapies received
- Transfusion dependence status [Annually or at the time of transformation of disease, up to 10 years]
Transfusion status
- Current Blood Work [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. [Annually or at the time of transformation of disease, up to 10 years]
Next generation sequencing gene panel
- Bone marrow transplant details (if received) [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) [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 [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
- Pulmonary hypertension [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 [Annually or at the time of transformation of disease, up to 10 years]
Details of thrombosis (type, site) o Treatment of thrombosis (type, duration)
- Family history of MPN will be obtained from the patient record. [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 [Annually or at the time of transformation of disease, up to 10 years]
Risk stratification (IPSS, DIPSS and DIPSS)
Eligibility Criteria
Criteria
Inclusion Criteria:
Diagnosis of one of the following myeloproliferative neoplasms (MPNs):
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Atypical CML (aCML)
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Chronic eosinophilic leukemia-not otherwise specified (CEL, NOS),
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Chronic myelomonocytic leukemia (CMML)
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Chronic neutrophilic leukemia (CNL),
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Essential thrombocythemia (ET),
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Juvenile myelomonocytic leukemia (JMML),
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Mastocytosis, MPN unclassifiable
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MPN/MDS unclassifiable,
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Primary myelofibrosis (PMF),
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Post-essential thrombocythemia myelofibrosis (post-ET MF),
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Post-polycythemia vera MF (post-PV MF)
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Refractory anemia with ringed sideroblasts associated with marked thrombocytosis (RARS-T)
Exclusion Criteria:
- None
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Princess Margaret Cancer Centre | Toronto | Ontario | Canada | M5G 1Z5 |
Sponsors and Collaborators
- University Health Network, Toronto
Investigators
- Principal Investigator: Vikas Gupta, MD, University Health Network, Toronto
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- UHN REB 15-9814 CE