MedPath

Registry of Patients With MPNs in Taiwan

Active, not recruiting
Conditions
Myeloproliferative Neoplasms
Registration Number
NCT03618485
Lead Sponsor
Chang Gung Memorial Hospital
Brief Summary

Myeloproliferative neoplasms (MPNs) are a group of clonal hematologic malignancies with great variation in reported patient life expectancy and are characterized by a relatively indolent course which can be complicated by thromboembolic events and transformation to acute myeloid leukemia (AML).

The MPNs in the 2016 World Health Organization (WHO) classification of myeloid neoplasms consist of polycythemia vera (PV), essential thrombocythemia (ET), primary myelofibrosis (PMF) including prefibrotic/early stage and over fibrotic stage, chronic myeloid leukemia, other (rare) disorders such as chronic neutrophilic leukemia and chronic eosinophilic leukemia and MPN unclassifiable (MPN-U).

The prevalence and genetic characteristics of patients with MPNs in Taiwan are still unknown. Molecular tests which are required for the diagnosis of MPNs are not available in many hospitals which hamper the accurate diagnosis and subtype classification of MPNs. Moreover, the information of current therapeutic strategy for MPNs in most medical centers in Taiwan is also not available. The purpose of this MPN registry is to collect clinical data, molecular characteristics, treatment details and response to therapy, occurrence of complications during the course, disease progression to secondary myelofibrosis from PV or ET and secondary AML (sAML) transformation as well as survival. The clinical and molecular data including the high molecular risk (HMR) genes will be examined and correlated with treatment outcomes in Taiwanese MPN patients.

The Molecular Diagnostic Laboratory at Chang Gung Memorial Hospital-Linkou is a College of American Pathologists (CAP)-accredited lab which provides high quality of molecular genetic tests for hematologic malignancies. The three driver gene mutations are the major criteria for the diagnosis of MPN, the methodologies of mutational analyses have been well set up for the clinical use in this lab. In addition, this lab is also equipped with facilities for the detection of mutated genes which were recently identified as HRM category (presence of any of ASXL1, EZH2, SRSF2, IDH1 or IDH2), and mutations of other epigenetic regulators or splicing factors.

Detailed Description

Not available

Recruitment & Eligibility

Status
ACTIVE_NOT_RECRUITING
Sex
All
Target Recruitment
500
Inclusion Criteria
  1. Male and female patients aged ≥ 20 years.
  2. Patient who had a confirmed diagnosis according to the 2016 revised WHO criteria with a Ph (-) MPN.
Exclusion Criteria
  1. Patients do not fulfill the diagnostic criteria of MPN by WHO 2016 classification.
  2. Patients unwilling to sign informed consent.

Study & Design

Study Type
OBSERVATIONAL
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
Epidemiological data of MPNs in Taiwan24 month

The prevalence of MPNs during the whole study period in Taiwan

Secondary Outcome Measures
NameTimeMethod
The frequencies of mutations of driver genes in Taiwanese patients with MPNs.24 months

The frequencies of JAK2V617F or JAK exon12, CALR and MPL in each subtype of patient's with MPN during the two years of study period.

Occurrence of vascular events36 months

Date of occurrence of thrombosis or bleeding during the study period.

Occurrence of secondary acute myeloid leukemia (sAML)36 months

Date of the development of sAML

Treatment required36 months

Date of phlebotomy and date of initiation of drug therapy

Change of symptoms from baseline using MPN-10 symptom assessment.36 months

Change of symptoms from baseline in MPN-10 at different time points (V2-5) during the study period.

Overall survival36 months

Time from diagnosis of MPN to last follow-up or date of death measured by months

Trial Locations

Locations (1)

Chang Gung Memorial Hospital-Linkou

🇨🇳

Taoyuan, State, Taiwan

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