Clinical trial NCT03618485: Registry of Patients With MPNs in Taiwan
Official title: Registry of Patients With Myeloproliferative Neoplasms (MPNs) in Taiwan -Taiwan MPN Working Group
Study type: OBSERVATIONAL
Overall status: ACTIVE_NOT_RECRUITING
Conditions: Myeloproliferative Neoplasms
Lead sponsor: Chang Gung Memorial Hospital
Sponsor class: OTHER
First posted: 2018-08-07
Enrollment: 500
Sex: ALL
Minimum age (years): 20
Standard ages: ADULT
OLDER_ADULT
Observational model: CASE_ONLY
Time perspective: PROSPECTIVE
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.

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.


Eligibility criteria: 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.

Primary outcomes:
measure: Epidemiological data of MPNs in Taiwan
timeFrame: 24 month
Secondary outcomes:
measure: The frequencies of mutations of driver genes in Taiwanese patients with MPNs.
timeFrame: 24 months
measure: Treatment required
timeFrame: 36 months
measure: Change of symptoms from baseline using MPN-10 symptom assessment.
timeFrame: 36 months
measure: Occurrence of vascular events
timeFrame: 36 months
measure: Occurrence of secondary acute myeloid leukemia (sAML)
timeFrame: 36 months
measure: Overall survival
timeFrame: 36 months
Other outcomes:
Locations:
city: Taoyuan
country: Taiwan
facility: Chang Gung Memorial Hospital-Linkou
state: State
Study officials:
References: