Myeloproliferative Neoplasms (MPNs) Patient Registry

Study Overview
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Brief Summary
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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
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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).

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

Conditions
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Leukemia, Myeloid, Chronic, Atypical, BCR-ABL Negative, Primary Myelofibrosis, Polycythemia Vera, Essential Thrombocythemia, Mastocytosis, Leukemia, Myelomonocytic, Juvenile, Chronic Eosinophilic Leukemia-not Otherwise Specified, Myelodysplastic-Myeloproliferative Diseases, Neoplasms, Leukemia, Myelomonocytic, Chronic

Intervention / Treatment
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* Other: Observational


Participation Criteria
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Eligibility Criteria
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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
Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Patients with a diagnosis of MPN<br>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<br> <br> |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Survival | 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 | 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 | 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 | MPN-SAF TSS questionnaire | Annually or at the time of transformation of disease, up to 10 years | 
| Co-morbidities | HCT-CI | Annually or at the time of transformation of disease, up to 10 years | 
| Physical symptoms of MPN | 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 | Medical therapies received | Annually or at the time of transformation of disease, up to 10 years | 
| Transfusion dependence status | Transfusion status | Annually or at the time of transformation of disease, up to 10 years | 
| Current Blood Work | 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. | Next generation sequencing gene panel | Annually or at the time of transformation of disease, up to 10 years | 
| Bone marrow transplant details (if received) | 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) | 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 | 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 | 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 | 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. | 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 | Risk stratification (IPSS, DIPSS and DIPSS) | Annually or at the time of transformation of disease, up to 10 years | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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myeloproliferative neoplasm, myelodysplastic syndrome, registry, leukemia, Bone Marrow Diseases, Hematologic Diseases, Neoplasms