Real-World Treatment Patterns Following Update to National Comprehensive Cancer Network Guidelines for Light-Chain Amyloidosis: Results from a Us Administrative Claims Database

Background: Light-chain amyloidosis (AL) is a rare, multi-organ disease that can cause serious organ damage. In January 2021, a new therapy, daratumumab, was FDA-approved for the treatment of AL in combination with current standard-of-care (SoC) of bortezomib/cyclophosphamide/dexamethasone (CyBorD) and was classified as SoC according to the National Comprehensive Cancer Network (NCCN) guidelines. The objective of this study is to describe current real-world treatment patterns amongst patients with AL who newly initiated treatment January/2021 through June/2022.

Methods: This study included patients aged ≥18 identified in the IQVIA PharMetrics database (>72 million lives 2021-2022 total) who were newly initiating pharmacological treatment with bendamustine, bortezomib, carfilzomib, cyclophosphamide, daratumumab, ixazomib, lenalidomide, melphalan, pomalidomide, or venetoclax 01Jan2021-30Jun2022 (date of first therapy classified as “index date”). Patients were required to have an ICD-10 diagnosis of AL (E85.81) identified during full study period (01Jan2020-30Sep2022) as well as continuous medical and prescription insurance enrollment from 1 year pre-treatment-index through 90-days post-treatment-index. Patients were excluded if they had any of the following: a claim indicating treatment with stem cell transplant; a diagnosis of familial mediterranean fever; a diagnosis of secondary, transthyretin, or hereditary amyloidosis; a diagnosis of a non-myeloma/non-lymphoma malignancy. Treatment patterns assessed included index treatment regimen and daratumumab adoption/use patterns at index, 30-days post-index, and 90-days post-index.

Results: A total of 194 patients met all eligibility criteria, out of which the majority were male (N=119; 61%), with the largest insurance coverage being self-insured (N=80; 41%), and a mean age of 62 years (SD=9.8). The most common index treatment regimen was CyBorD+daratumumab (N=53; 27%) followed by dexamethasone+daratumumab (N=24;12%) and daratumumab monotherapy (N=16; 8%) ( Table). Across all treatment regimens at index date, daratumumab was found to be utilized by 62% (N=121) of patients. Furthermore, 76% (N=148) and 80% (N=155) of patients were found to have utilized daratumumab within 30-days or 90-days of treatment initiation, respectively.

Discussion: AL is life threatening disease that impacts organ function and quality of life. Until 2021, there were no FDA approved therapies for AL. Daratumumab use was rapidly adopted to treat patients with AL following approval, in accordance with national guidelines, primarily in combination with other treatments such as CyBorD. This has changed the treatment paradigm providing hope to patients and clinicians to treat this devasting disease and should be informative for current and future clinical trial considerations in development of medications to treat AL.