Presenting features, treatment patterns and predictors of outcomes in AL amyloidosis at a tertirary care cancer centre

Abstract

Abstract Introduction Light Chain (AL) amyloidosis is the most common form of systemic amyloidosis, characterized by the deposition of misfolded monoclonal light chains or their fragments in tissues, leading to progressive organ dysfunction. The treatment of AL Amyloidosis has been particularly challenging for hematologists, with therapy needing to be carefully tailored based on the severity of organ involvement and clonal characteristics, along with the need for constant monitoring. Data in developing countries remains scarce due to the heterogeneous nature of the disease, particularly in settings with limited access to novel agents and autologous stem cell transplantation (ASCT). Methods We conducted a retrospective observational study of patients diagnosed with AL amyloidosis at our institution between January 2013 and April 2025. Data was obtained from the Electronic Medical Records. Demographic details, clinical features, treatment regimens, and outcomes were analysed from the recorded data using SPSS 31.0. Results For analysis, a total of 93 patients were included. The median age at diagnosis was 60 years (range: 29–85 years), with 64 (68.8%) patients being male. The median duration of symptoms before diagnosis was 6 months (range: 1–24 months). The most common organ involvement was the kidney in 66 (71%) of patients, followed by the heart in 59(63.4%) patients, with 53 (63.4%) patients having two or more organs involved at presentation. Revised Mayo Staging was evaluated for 61 patients, with 19 (30.6%) patients presenting at stage III or higher. The most common induction regimen used was VCD in 50 (53.8%) followed by VRD in 6 (6.5%) patients, while a Daratumumab based induction regimen (Dara-VCD) was started in only 2 (2.2%) patients. ASCT was performed in 6 (6.4%) of patients. Post-induction haematological responses were observed in 89.6%, with complete response (CR) in 24.1%, very good partial response (VGPR) in 41.4%, and partial response (PR) in 24.1%. The median follow-up duration was 18 months (range: 4–145 months), with a 24-month progression-free survival (PFS) of 58% (95% CI: 45%–71%). The 24-month and 60-month overall survival (OS) rates were 72% (95% CI: 62%–82%) and 59% (95% CI: 48%–70%), respectively. Early mortality (< 6 months) occurred in 10 (41.6%) of the total deaths. On univariate and multivariate analyses, renal involvement (HR: 3.85; 95% CI: 1.14–12.93; P = 0.029) and lack of post-induction haematological response (HR: 4.2; 95% CI: 1.2–14.92; P = 0.022) were significantly associated with poor outcomes. Conclusions This study provides key insights into the presentation and outcomes of AL amyloidosis in an Indian cohort, highlighting delayed diagnosis, frequent multiorgan involvement, and limited access to advanced therapies such as Daratumumab and ASCT. High early mortality rates underscore the urgent need for timely diagnosis, aggressive supportive care, and access to novel agents. Renal involvement and failure to achieve a post-induction hematologic response emerged as significant predictors of poor prognosis.