Consensus guidelines for the conduct and reporting of clinical trials in systemic light-chain amyloidosis

Melphalan and dexamethasone with or without bortezomib in newly diagnosed AL amyloidosis: a matched case–control study on 174 patients

“…32,33 In 2011, a randomized clinical trial comparing MDex with BMDex was initiated (NCT01277016), which is still ongoing in Europe and Australia. However, the results of this trial will not be available for at least 2 years, although the increasing use of bortezomib combinations in routine clinical practice urgently requires some indications on the most appropriate setting to employ this drug in AL amyloidosis.…”

Epidemiology of AL amyloidosis: a real-world study using US claims data

“…Therefore, the literature suggests an estimated incidence between 3 and 12 cases per million persons per year, and an estimated prevalence of 30 000 to 45 000 AL amyloidosis patients in the United States and the European Union. 1,7,[11][12][13][14] Additionally, studies have shown that men have a slightly increased rate of AL amyloidosis compared with women, and the majority of patients are over the age of 65 years. 1,7,15 Given the lack of contemporary data on AL amyloidosis in the United States, this study sought to examine the prevalence (proportion of the population having the condition at a given time) and incidence (annual rate of newly identified cases) of AL amyloidosis over a 9-year period (2007-2015) using health insurance claims data.…”

Organ response in patients with AL amyloidosis treated with NEOD001, an amyloid‐directed monoclonal antibody

“…B: Patient 2 (46‐year‐old man) had elevated NT‐proBNP levels (>3,000 pg/mL) both after he achieved CR from previous chemotherapy (combined cyclophosphamide, bortezomib, and dexamethasone) and at screening 9.6 months later. After infusion 8 (4 mg/kg NEOD001), his NT‐proBNP level dropped to 2196 pg/mL (−33.7% of baseline) and met consensus criteria for cardiac response 5. At infusion 14, his dose was escalated to the MTD (orange arrow), and his best response to date was recorded after 31 infusions (−72% of baseline; 929 pg/mL NT‐proBNP).…”

“…He was treated with PCD therapy and experienced complete HR. However, his N‐terminal fragment of probrain natriuretic peptide (NT‐proBNP) level remained elevated and unimproved (3,914 pg/mL; >332 pg/mL predicts poor prognosis 5), indicating persistent cardiac dysfunction.…”

“…His cardiac troponin level was <0.06 ng/mL, and his NYHA classification was II. After eight infusions, he achieved a cardiac response by consensus criteria 5 (NT‐proBNP level was 2,196 pg/mL; 34% reduction from baseline) and normal cardiac troponin (<0.01 ng/mL). The patient was escalated to 24 mg/kg at his 14‐month infusion, and his NT‐proBNP continued to decrease (Fig.…”