Myeloproliferative Neoplasms (MPNs) Patient Registry

Recruiting

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

• Registration Number: NCT02760238
• Lead Sponsor: University Health Network, Toronto

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).

Study Timeline

• Study Start: 2015-04
• Study First Submitted: 2016-03-24
• Study First Submitted QC: 2016-04-28
• Study First Posted: 2016-05-03
• Status Verified: 2024-08
• Last Update Submitted: 2024-08-26
• Last Update Posted: 2024-08-27
• Primary Completion: 2027-10-31
• Study Completion: 2027-10-31

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 5000

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 & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Patients with a diagnosis of MPN	Observational	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

Primary Outcome Measures

Name	Time	Method
Survival	Annually or at the time of transformation of disease, up to 10 years	Survival of patients with MPN

Secondary Outcome Measures

Name	Time	Method
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
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
Co-morbidities	Annually or at the time of transformation of disease, up to 10 years	HCT-CI
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
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
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)
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
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
Quality of life - Neoplasm Symptom	Annually or at the time of transformation of disease, up to 10 years	MPN-SAF TSS questionnaire
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
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)
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)
Disease progression	Annually or at the time of transformation of disease, up to 10 years	Risk stratification (IPSS, DIPSS and DIPSS)

Trial Locations

Locations (1)

Princess Margaret Cancer Centre; 🇨🇦 Toronto, Ontario, Canada