Pb2135: treatment patterns in patients with newly diagnosed al amyloidosis

Topic: 14. Myeloma and other monoclonal gammopathies - Clinical Background: Treatment options for AL amyloidosis remain limited. Aims: To describe current patterns of medication use in patients with newly diagnosed AL amyloidosis. Methods: This retrospective analysis used 2017-2020 data from the Merative® MarketScan® Commercial and Medicare Supplemental and the IQVIA Pharmetrics Plus® databases to identify adult patients (newly diagnosed) with ≥1 inpatient or ≥2 outpatient claims for AL amyloidosis (ICD-10-CM code E85.81) in any diagnosis field during the identification period (1/1/2018-12/31/2020). The first observed AL amyloidosis diagnosis date was the index date. Continuous enrollment in a health plan during the 1 year before (baseline) and ≥3 months after (follow-up) index was required. Patients with an AL amyloidosis diagnosis during the baseline period or who received no treatment were excluded. Study outcomes included demographic/clinical characteristics and treatment patterns. We reported on lines of treatment, duration (defined as continuous use from first line to end of days’ supple without a gap of ≥120 days), and adherence (proportion of days covered, PDC, ≥80% while persistent) to first-line therapy. Results: The final sample consisted of 338 patients with newly diagnosed AL amyloidosis. Mean (SD) age at diagnosis was 61.2 (9.1) years and 42.3% of patients were female. As the first-line therapy, 72.5% patients received bortezomib-containing regimens, whereas only 13.9% received daratumumab-containing regimens. The most common first-line treatment was bortezomib-cyclophosphamide-dexamethasone (34.0%), with mean (SD) duration of 138.7 (100.9) days and mean (SD) PDC of 98.4% (5.8%). Overall, for patients with first-line treatment (except for hematopoietic stem cell transplant [HSCT]), the mean (SD) duration was 143.2 (136.7) days, mean (SD) PDC was 0.978 (0.064), and 97.2% were adherent (PDC ≥80%). For patients with second-line treatment, the most common was HSCT (23.4%), followed by bortezomib-daratumumab-dexamethasone and daratumumab alone (both 11.1%). Summary/Conclusion: Bortezomib-containing regimens were the most common first-line therapy and HSCT was the most common second-line therapy. Even though treatment availability is limited for AL amyloidosis, newly diagnosed patients in this study were highly adherent to their first-line therapy. Additional therapies for AL amyloidosis are currently under development. Keywords: Treatment, Bortezomib, AL amyloidosis, Real world data