Chronic Eosinophilic Leukemia-not Otherwise Specified, Essential Thrombocythemia, Leukemia, Myeloid, Chronic, Atypical, BCR-ABL Negative, Leukemia, Myelomonocytic, Chronic, Leukemia, Myelomonocytic, Juvenile, Mastocytosis, Myelodysplastic-Myeloproliferative Diseases, Neoplasms, Polycythemia Vera, Primary Myelofibrosis
Conditions
Keywords
myeloproliferative neoplasm, myelodysplastic syndrome, registry, leukemia, Bone Marrow Diseases, Hematologic Diseases, Neoplasms
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).
Interventions
OTHERObservational
Sponsors
University Health Network, Toronto
Study design
Observational model
COHORT
Time perspective
OTHER
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Survival | Annually or at the time of transformation of disease, up to 10 years | Survival of patients with MPN |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| 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 |
| 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 |
| 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) |
| 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 |
Countries
Canada
Contacts
Primary ContactVikas Gupta, MD
Backup ContactJaime O. Claudio, PhD
Outcome results
None listed