JMIR Publications

ABSTRACT

Background:

Transthyretin Amyloid Cardiomyopathy (ATTR-CM) is a systemic amyloidosis disorder with early clinical manifestations similar to other heart conditions, which complicates its diagnosis and management. The disease's insidious nature and its progression to heart failure emphasize the critical need for enhanced recognition and understanding of its clinical landscape.

Objective:

We aim to understand the natural history and current treatment patterns for managing ATTR-CM in a diverse Asian cohort from Taiwan, Hong Kong, and Malaysia.

Methods:

This study is a multicenter, non-interventional disease registry that plans to enroll patients diagnosed with ATTR-CM across approximately 17 sites in Taiwan, Hong Kong, and Malaysia. Almost 350 patients with a documented diagnosis of ATTR CM after 01 June 2019 will be enrolled in the study. Deceased patients will be enrolled without the need for consent in accordance with applicable regulations. Their data will be gathered retrospectively through a one-time review of their medical records, where permissible. Data related to clinical characteristics, treatment, and outcomes will be collected for each patient during the routine clinical practice while adhering to local standards of care. The end of data collection is planned for at least 12 months after the end of the enrollment period.

Results:

As of January 7, 2025, ethical approvals for this study have been obtained or are under review at multiple sites across Taiwan, Hong Kong, and Malaysia. In Taiwan, approvals have been secured from the National Taiwan University Hospital, Taipei Veterans General Hospital, Chang Gung Memorial Hospitals (Linkou and Kaohsiung branches), Taichung Veterans General Hospital, China Medical University & Hospital, and Taipei Mackay Memorial Hospital, with applications pending approval from Far Eastern Memorial Hospital and Chi-Mei Medical Center. In Hong Kong, the study is approved at Queen Elizabeth Hospital, Princess Margaret Hospital, and Prince of Wales Hospital while awaiting approval from Queen Mary Hospital. Additionally, an ethics application is being prepared for Tuen Mun Hospital. In Malaysia, Hospital Sultan Idris Shah in Serdang and the Sarawak Heart Centre have granted approval, while Institut Jantung Negara Sdn. Bhd. is reviewing the application. So far, twenty-seven patients have been recruited at National Taiwan University Hospital.

Conclusions:

This research will aid in understanding the demographic, clinical, and therapeutic patterns of ATTR-CM in Taiwan, Hong Kong, and Malaysia. This registry may influence advancements in early detection, diagnosis, and tailored treatment strategies, ultimately striving to enhance patient outcomes and survival rates in ATTR-CM.