The Hong Kong CML Registry

A Multi-centre Study of Epidemiology, Characteristics, Treatment and Outcome of Chronic Myeloid Leukaemia in Adults in Hong Kong

Chronic myeloid leukaemia (CML) is a malignant disorder of the haematopoietic system. It is characterized by the chromosomal translocation between chromosomes 9 and 22, resulting in the formation of the Philadelphia chromosome which contains the BCRABL1 fusion gene. The projected prevalence of CML is rising steadily, due to the significantly improved survival of CML patients and that the incidence rate increases with age. The efficacious yet costly tyrosine kinase inhibitors pose a significant financial burden to both patients and the health care system, while they carry their own side effects and long-term risks. This study aims to set up a local disease registry of CML to improve the knowledge concerning this disease, including epidemiology,characteristics and treatment outcome of CML in Hong Kong,as well as long-term safety and toxicities of therapeutic agents.

Study Overview

• Status: Recruiting
• Conditions: Chronic Myeloid Leukemia

Detailed Description

Chronic myeloid leukaemia (CML) is a malignant disorder of the haematopoietic system characterized by the presence of the Philadelphia chromosome arising from t(9;22)(q34;q 11).The annual incidence of CML is reported to be around 1-2 cases per 100,000 population.In Hong Kong, the age-standardised incidence rate of CML has remained static in recent years.

However, as the incidence of CML increases with age and Hong Kong has an ageing population, the crude incidence rate is expected to follow a rising trend. On the other hand, the prognosis of CML patients has improved significantly since the advent of tyrosine kinase inhibitors (TKis) more than two decades ago. With the efficacious TKis, CML has changed from a deadly cancer to a treatable chronic condition. It has been shown that life expectancy of CML patients is approaching that of the general population now.The prevalence of CML is expected to rise considerably in view of the increasing incidence in an ageing population and improved prognosis. The Hong Kong Cancer Registry only provides data on the incidence rates of cancers but it has yet to provide cancer prevalence data. A clinical registry is "an organized system that uses observational study methods to collect uniform data to evaluate specific outcomes". A disease registry specifically designed for CML would provide more accurate and comprehensive data and allow an in-depth study of the epidemiology and characteristics of CML in Hong Kong.

At present,there are three TKis approved for front-line treatment of newly diagnosed chronic-phase CML,viz.,imatinib, nilotinib and dasatinib.lmatinib is a first-generation TKI while nilotinib and dasatinib are second-generation TKis (2G-TKI) . Nilotinib and dasatinib demonstrate better in vitro efficacy and achieve molecular responses faster and deeper than imatinib,but these drugs not been proven superior to imatinib in conventional clinical endpoints including overall survival and progression-free survival.Besides, nilotinib and dasatinib are not without side effects and their long-term safety profiles are unclear. For example, nilotinib is associated with increased cardiovascular risk while dasatinib often leads to pleural effusion and less frequently,pulmonary hypertension.The safety of imatinib has been well established over the past 15 years and its side effects are generally considered tolerable. However, the generic formulation of imatinib has been recently introduced to public hospitals and substituted for branded imatinib in CML patients. There have been reports that patient switching to generic imatinib develop adverse drug reactions not experienced with branded imatinib. Other less commonly used therapeutic agents include bosutinib and ponatinib, which still play an important role in the management of CML. Therefore, it is important to determine the "real-world" clinical effectiveness of various drugs, and monitor their side effects and long-term toxicities in a population-based approach.

As physicians in Hong Kong are facing a growing patient population on long-term TKI,the demand on health care services and financial burden of drug costs will escalate accordingly. lt is pivotal for the investigators to understand the disease and patients in the locality better by systematic data collection, in order to improve quality of care, allow service planning and facilitate medical research and advances .

• Study Type: Observational
• Enrollment (Anticipated): 200

Contacts and Locations

• Study Contact: Name: Dr Carol Cheung, MBBS; Phone Number: 852-22553456; Email: drcarolcheung@gmail.com

Study Locations

• Hong Kong
  • Hong Kong, Hong Kong, 0000
    • Recruiting
    • The University of Hong Kong
    • Contact: Dr Carol Cheung, MBBS; Phone Number: 852-22553456; Email: drcarolcheung@gmail.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Probability Sample

Study Population

Descriptive statistics will be used to analyse the characteristics of the CML cohort. Categorical data will be presented with frequency, percentage and 95% confidence interval. Quantitative variables will be presented with median and range, mean and standard deviation when relevant. Survival data will be analysed by the Kaplan-Meier method and competing risks methods. A P value of less than 0.05 will be considered statistically significant. All analyses will be performed using SPSS version 24 and Rsoftware version3.3.2.

Description

Inclusion Criteria:

1. Patients aged 18 years or above
2. Diagnosed with Philadelphia chromosome positive and/or BCR-ABLl positive chronic myeloid leukaemia in all phases
3. Managed in any of the participating centres in the period of 2007 to 2017

Exclusion Criteria:

1) Patients less than 18years of age

Study Plan

How is the study designed?

Design Details

• Observational Models: Case-Only
• Time Perspectives: Cross-Sectional

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort
chronic myeloid leukaemia; chronic myeloid leukaemia diagnosed from 2007 - 2017

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Demographics	percentage of ethnic distribution	January 2007 to December 2017
Age	percentage of age distribution	January 2007 to December 2017
Etiology	percentage of etiology	January 2007 to December 2017
Clinical features	percentage of clinical features	January 2007 to December 2017
Sex	ratio of male to female patient	January 2007 to December 2017

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
number of CML patients in Hong Kong	Estimated prevalence of CML in Hong Kong	up to 10 years
number of newly diagnosis CML patient each year	Estimated incidence of CML in Hong Kong	up to 10 years
Current treatment pattern of CML	Current treatment pattern of CML	up to 10 years
record AE of each treatment	Safety data ofTKI	up to 10 years
disease free and survival free	Treatment outcome of CML patients	up to 10 years
disease free and survival free data in advanced phases (accelerated and blast phases)	Outcome of patients in advanced phases (accelerated and blast phases)	up to 10 years

Collaborators and Investigators

• Sponsor: The University of Hong Kong
• Investigators: Principal Investigator: Dr Carol Cheung, MBBS, Queen Mary Hospital, Hong Kong

Study record dates

Study Major Dates

• Study Start (Actual): April 1, 2018
• Primary Completion (Anticipated): March 31, 2023
• Study Completion (Anticipated): September 30, 2023

Study Registration Dates

• First Submitted: July 25, 2018
• First Submitted That Met QC Criteria: August 9, 2018
• First Posted (Actual): August 10, 2018

Study Record Updates

• Last Update Posted (Actual): August 10, 2018
• Last Update Submitted That Met QC Criteria: August 9, 2018
• Last Verified: August 1, 2018

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Neoplasms by Histologic Type; Neoplasms; Bone Marrow Diseases; Hematologic Diseases; Myeloproliferative Disorders; Leukemia; Leukemia, Myeloid; Leukemia, Myelogenous, Chronic, BCR-ABL Positive
• Other Study ID Numbers: QMH-CML-003

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: Undecided

Drug and device information, study documents

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