Registry of Patients With MPNs in Taiwan

Registry of Patients With Myeloproliferative Neoplasms (MPNs) in Taiwan -Taiwan MPN Working Group

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.

Study Overview

• Status: Active, not recruiting
• Conditions: Myeloproliferative Neoplasms

• Study Type: Observational
• Enrollment (Actual): 500

Contacts and Locations

Study Locations

• Taiwan
  • State
    • Taoyuan, State, Taiwan, 33305
      • Chang Gung Memorial Hospital-Linkou

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 20 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: N/A

• Sampling Method: Non-Probability Sample

Study Population

Adult patients with MPNs newly diagnosed and followed in Chang Gung Memorial Hospital and cooperative hospitals.Registry will be limited to patients newly diagnosed between 2016 and 2019. Newly diagnosed means having the first bone marrow biopsy within 12 months from diagnosis.

Description

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 Plan

How is the study designed?

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Epidemiological data of MPNs in Taiwan	The prevalence of MPNs during the whole study period in Taiwan	24 month

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
The frequencies of mutations of driver genes in Taiwanese patients with MPNs.	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.	24 months
Treatment required	Date of phlebotomy and date of initiation of drug therapy	36 months
Change of symptoms from baseline using MPN-10 symptom assessment.	Change of symptoms from baseline in MPN-10 at different time points (V2-5) during the study period.	36 months
Occurrence of vascular events	Date of occurrence of thrombosis or bleeding during the study period.	36 months
Occurrence of secondary acute myeloid leukemia (sAML)	Date of the development of sAML	36 months
Overall survival	Time from diagnosis of MPN to last follow-up or date of death measured by months	36 months

Collaborators and Investigators

• Sponsor: Chang Gung Memorial Hospital

Study record dates

Study Major Dates

• Study Start (Actual): April 1, 2017
• Primary Completion (Actual): July 31, 2023
• Study Completion (Estimated): December 31, 2025

Study Registration Dates

• First Submitted: July 16, 2018
• First Submitted That Met QC Criteria: August 5, 2018
• First Posted (Actual): August 7, 2018

Study Record Updates

• Last Update Posted (Actual): March 25, 2025
• Last Update Submitted That Met QC Criteria: February 13, 2025
• Last Verified: February 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Hematologic Diseases; Bone Marrow Diseases; Neoplasms; Myeloproliferative Disorders
• Other Study ID Numbers: 201700356B0

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No