Hungarian National Systemic Amyloidosis Registry

Systemic amyloidosis is being diagnosed with increasing frequency worldwide. Owing to recent advances in diagnostic and therapeutic modalities, the disease has received growing clinical attention in recent years. Over the past two decades, the annual number of newly diagnosed light-chain amyloidosis (AL) cases has remained stable in major international amyloidosis centers, whereas the incidence of amyloid A (AA) amyloidosis has shown a progressive decline, likely attributable to the availability of modern therapies for chronic inflammatory diseases. An increasing number of patients are being diagnosed with transthyretin amyloidosis (ATTR) globally. This trend is partly related to improvements in diagnostic techniques and their broader accessibility, as well as to heightened clinical awareness among healthcare professionals. In addition, the emergence of several disease-specific therapeutic options for ATTR amyloidosis has further contributed to increased case recognition and diagnosis. The rising prevalence of systemic amyloidosis suggests that the condition may not be as rare as previously considered, but rather historically underrecognized due to limited awareness and insufficient diagnostic attention. In response to the continuously increasing number of patients, the need has emerged for a centralized national database into which data from all centers involved in the care of patients with systemic amyloidosis across Hungary can be entered. The primary objective?primary objective The main result the study is designed to measure and report on. Other results, called secondary outcomes, are also collected. of this study is to characterize the demographic, epidemiological, and clinical data of patients with systemic amyloidosis in Hungary, as well as current national practices in diagnostic and therapeutic management and disease prognosis. Our long-term objective is to establish collaboration among healthcare centers involved in the care of patients with systemic amyloidosis in Hungary. This aim will be supported by the systemic amyloidosis registry (AMYREG). The registry will include demographic data, key imaging and laboratory parameters, as well as clinically relevant disease-specific and therapeutic data for all patients diagnosed with systemic amyloidosis in Hungary. Retrospective data collection will also be possible for patients previously diagnosed, including deceased patients. This will enable the creation of a large, unified database allowing both prospective and retrospective follow-up?follow-up The period after treatment or enrolment during which participants are observed or contacted, for example to record side effects or outcomes. of registered patients. This approach will expand knowledge regarding disease course, patient survival, prognostic factors, organ involvement, diagnostic challenges, and treatment strategies across all types of systemic amyloidosis. Furthermore, it will allow assessment of the regional distribution of diagnostic practices in Hungary and characterization of the Hungarian patient population in comparison with international data. These insights are intended to improve patient care. The study includes two patient cohorts: first, patients diagnosed with systemic amyloidosis will be prospectively enrolled from the initiation of the registry and followed longitudinally; second, patients diagnosed within the 12 months preceding study initiation may also be entered into the registry retrospectively. This is a non-interventional clinical study in which treating physicians regularly enter newly collected data from prospectively enrolled patients into the system. Data collection corresponds to secondary use of data. Patient information is provided by the treating physician. Subsequently, patients' clinical follow-up continues, and additional data may be entered retrospectively into the system. In the future, statistical analyses will be performed on the collected data, with the aim of generating scientific conclusions and publications. Data sources include electronic health records from local hospitals and healthcare providers. The study design is characterized by secondary use of data, in which relevant clinical events have already been collected as part of routine clinical practice for other purposes. The study involves secondary data utilization and does not include the collection or evaluation of adverse events?adverse events Any unwanted medical problem that happens during a study, whether or not it was caused by the treatment. A 'serious' adverse event is one that meets specific criteria, such as causing death, being life-threatening, requiring or prolonging hospital admission, causing significant disability, or another medically important outcome. as a study objective. Accordingly, no dedicated safety data collection is performed. However, if an investigator becomes aware of a spontaneous adverse event during the study period, it will be reported independently of the study through standard reporting procedures.