New research published in Blood Advances has shed light on the potential for early improvements in cardiac and hematologic parameters to predict improved survival outcomes for patients undergoing treatment for stage IIIb AL amyloidosis. This disease, characterized by abnormal protein buildup, typically leads to poor survival rates, with a median expectancy of 4-6 months.
Amyloidosis occurs when normal proteins misfold and create amyloid deposits in vital organs and tissues, causing organ dysfunction and ultimately leading to organ failure and death. Patients with advanced cardiac amyloidosis, which affects the heart, face a particularly grim prognosis, and treatment options have been limited by a lack of comprehensive data.
“While AL amyloidosis is a rare condition, symptoms can mimic those of many other more common diseases, like diabetes, hypertension, autoimmune disorders, and connective tissue diseases, making it difficult for clinicians to diagnose it early,” described Vaishali Sanchorawala, MD, Director of the Amyloidosis Center at Boston University Chobanian & Avedisian School of Medicine and Boston Medical Center, and senior author of this study.
“By understanding the significance of early treatment responses at one and three months after beginning treatment, we can better guide our approaches to therapy and improve patient outcomes.”
The study, conducted between 2007 and 2022, examined data from 142 patients diagnosed with stage IIIb AL amyloidosis, characterized by advanced cardiac involvement. The analysis revealed that patients had a median overall survival of nine months over a follow-up period of 60 months. Certain baseline factors were identified as predictors of poorer survival, including a longer time from symptom onset to diagnosis, higher bone marrow plasma cell counts, elevated levels of troponin I (a marker of heart damage), and limited walking capacity (less than 200 meters in six minutes).
“Stage IIIb AL amyloidosis with advanced cardiac involvement does not have to be a death sentence,” highlighted Dr Sanchorawala.
“We see patients who live four to five years after being diagnosed, and studies like this one helps us understand what factors may indicate a better prognosis. These findings help us to appropriately tailor our treatment regimens to achieve optimal outcomes.”
The study found that survival outcomes were enhanced with treatment regimens based on bortezomib and daratumumab. Importantly, patients who demonstrated early hematologic responses (within one month) and cardiac responses had significantly longer overall survival outcomes. For instance, patients achieving a very good partial hematologic response one month after treatment experienced a 47-month survival.
Dr. Sanchorawala, one of the researchers, emphasized the importance of patient participation in clinical research to advance treatment and improve overall survival for individuals grappling with this condition. The findings offer hope for better treatment strategies and improved outcomes for those affected by stage IIIb AL amyloidosis, underscoring the value of early response monitoring in guiding patient care.
“Our ability to advance science and improve patient outcomes depends on rare disease-focused clinical trials,” she stated.
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