Abstract 635P
Background
AL amyloidosis is a disorder leading to functional impairment of organs due to misfolding of light chains deposited. Few real-world studies of a large cohort of AL amyloidosis patients in Asia have been reported.
Methods
The multi-center, retrospective, observational, real-world study included patients who were pathologically diagnosed with AL amyloidosis in five centers in Sichuan Province, China between January 2011 and April 2021, with follow-up until September 2021. Demographic, clinical, treatment, and laboratory data were extracted from electronic medical records to be analyzed.
Results
348 patients were included in this study. The median age was 60 years (28-90). 204 patients (58.6%) were male. The heart (74.7%) and kidney (67.0%) were the most commonly involved organs. The majority of patients who could be analyzed were staged as Mayo 2004 Stage IIIa (42.1%) and Stage IIIb (10.0%). Due to the affordability and pessimism about the poor prognosis, only 112 patients (32.2%) had chemotherapy or ASCT records in participating centers. The median follow-up time is 16.2 months (1-100). The overall hematological response rate (RR) was 72.3% and the complete remission (CR) rate was 40.0% for all chemotherapy regimens. The bortezomib-based regimens were most commonly used in the first-line (93, 83.0%). The hematological RR, CR rate, duration of chemotherapy, and median response time for bortezomib-based regimens were 81.2%, 51.3%, 6 months (1-30) and 3 months (1-12). The daratumumab-based regimen was the most effective second-line therapy, whose RR was 100% (4) and median remission time was 1 month in this cohort. The patients who received treatment had an overall cardiac RR of 51.7% and a renal RR of 44.4%, and the median remission time for cardiac and renal involvement was 8 months (2-39) and 11 months (1-28), respectively.
Conclusions
This study reported a multi-center cohort of patients with AL amyloidosis in Asia and analyzed its demographic information and clinical outcomes. Although physicians have improved their awareness and understanding of AL amyloidosis in recent years, the prognosis is barely satisfactory due to the accessibility and affordability of medications.
Clinical trial identification
ChiCTR2100054499.
Editorial acknowledgement
Legal entity responsible for the study
The authors and West China Hospital, Sichuan University.
Funding
Janssen Pharmaceutical.
Disclosure
H. Yu: Financial Interests, Personal and Institutional, Sponsor/Funding: Janssen Pharmaceutical. C. Zhang: Financial Interests, Institutional, Sponsor/Funding: Janssen Pharmaceutical. Q. Liu: Financial Interests, Institutional, Sponsor/Funding: Janssen Pharmaceutical. Y. Wu: Financial Interests, Institutional, Sponsor/Funding: Janssen Pharmaceutical. All other authors have declared no conflicts of interest.