Haematological response and overall survival in two consecutive Dutch patient cohorts with AL amyloidosis diagnosed between 2008 and 2016

Abstract

Background: Although survival has improved in recent decades, the short-term prognosis of patients with immunoglobulin light chain (AL) amyloidosis remains grim. We aimed to assess overall survival (OS) of AL amyloidosis patients by comparing cohorts in two consecutive time periods. Methods: Data were collected and compared on 126 patients from two tertiary referral centres in The Netherlands during the time periods 2008-2012 and 2013-2016. Results: There was a non-significant trend to improved 6-month OS in the last cohort (78% vs. 67%, p ¼ .216, crude odds ratio 1.66, 95%CI 0.74-3.70, adjusted odds ratio 2.22, 95%CI 0.88-5.56). Patients in this cohort had higher Mayo risk scores (stage III 40% vs. 24%, p < .001 and revised stage IV 14% vs. 11%, p < .001), higher use of bortezomib (50% vs. 30%), and better haematological response (complete response/very good partial response in 39% vs. 27%, p < .001). Diagnostic delay was similar in both time periods. Conclusions: In the 2013-2016 cohort there was a trend toward improved 6-month OS, and an improved haematological response. Patients in this cohort had more advanced cardiac disease and received bortezomib more frequently, but diagnostic delay was similar to the 2008-2012 cohort. For further prognostic improvement, practitioners should be more alert, especially for cardiac amyloidosis.

Restrictive Atrial Dysfunction in Cardiac Amyloidosis: Differences between Immunoglobulin Light Chain and Transthyretin Cardiac Amyloidosis Patients

“…This stage can be avoided by initiating early treatment. The hematologic treatment of AL development has shown much progression in the last decade [ 4 ]. In addition, therapeutic agents have become available for ATTR cardiac amyloidosis.…”

“…In the past years, several novel treatments have become available including transthyretin stabilizers and transthyretin gene silencing approaches for ATTR cardiac amyloidosis [ 1 ]. Moreover, the treatment for AL cardiac amyloidosis has improved significantly over the past decade [ 4 ]. Nevertheless, the treatment for both types of cardiac amyloidosis is most beneficial in patients presenting in an early stage of the disease.…”

Cardiac amyloidosis: the need for early diagnosis

“…Treatment-related mortality is high as the treatment by itself can be cardiotoxic leading to worsening heart failure. Although chemotherapy followed by autologous stem cell transplantation (SCT) is the preferred therapy to obtain complete remission, only a minority of patients are eligible after patient selection [7, 8, 41]. Severe cardiac involvement is an important contraindication (serum NTproBNP level is >5000 pg/ml, serum troponin T level is >0.06 ng/ml, LVEF <45%, New York Heart Association [NYHA] class ≥ III) next to severe kidney dysfunction, hypotension, age >70 years and impaired performance status [34, 35].…”

Differences and Similarities in Treatment Paradigms and Goals between AL Amyloidosis and Multiple Myeloma

“…More similar are the male-to-female ratio and the median age at diagnosis (Table 1). In both diseases, there is a delay in diagnosis, but some reports suggest that this delay is much more prominent in AL amyloidosis, ranging from 180 to 441 days [11,12]. In addition, many patients diagnosed with AL amyloidosis need to consult numerous different physicians before receiving a correct diagnosis [13].…”