Real-world treatment patterns, costs, and outcomes in patients with AL amyloidosis: analysis of the Optum EHR and commercial claims databases

Abstract

Background

This study characterised real-world treatment patterns, clinical outcomes, and cost-of-illness in patients with light-chain (AL) amyloidosis.

Methods

Data were extracted from the US-based Optum® EHR and Clinformatics® Data Mart (claims) databases (2008–2019) for patients newly diagnosed with AL amyloidosis and who initiated anti-plasma cell therapies. Healthcare resource utilisation (HCRU) and related costs were compared across lines of therapy (LOT). Incidences of cardiac and renal failure were evaluated using the Kaplan-Meier method.

Results

About 1347 patients (EHR, n = 776; claims, n = 571) were included. Median age was 68 years; 56.8% were male. At initial diagnosis, 33.1% and 15.1% of patients had cardiac and renal failure, respectively. Most patients received bortezomib-containing treatment in LOT1 (69%); bortezomib-cyclophosphamide-dexamethasone was most common (26%). HCRU was similar across LOTs. Mean per-patient-per-month and per-patient-per-LOT costs were $19,343 and $105,944 for LOT1, $19,183 and $95,793 for LOT2, and $16,611 and $128,446 for LOT3, respectively. Costs were primarily driven by anti-plasma cell therapies, outpatient visits, and hospitalisations. The 5-year cardiac and renal failure rates following initial diagnosis were 64.5% and 39.0%, respectively.

Conclusion

AL amyloidosis is associated with substantial costs and suboptimal outcomes, highlighting the need for new therapeutic approaches to prevent organ deterioration, and reduce disease burden.

Keywords:

• Real-world treatment patterns
• optum claims
• optum EHR
• AL amyloidosis
• burden of illness

Acknowledgements

Medical writing and editorial support were provided by Justine Lempart, PhD, and Noopur Mandrekar, PhD of Eloquent Scientific Solutions and were funded by Janssen Global Services, LLC.

Disclosure statement

AD: Janssen, Celgene, Takeda, Pfizer, Alnylam, Intellia (research funding); JZ: BMS, Celgene, Takeda, Amgen, Alnylam (advisory committee, research funding), Intellia, Regeneron, Caelum, Oncopeptides, Janssen (consultancy); JH: Loxo (equity holder), Celgene (honoraria and speakers bureau); SWW: Janssen, Fortis, Genentech, Bristol Myers Squibb, GSK (research funding), Amgen (consultancy), Sanofi (advisory committee); ML: Adaptive, Caelum, Celgene, GSK, Janssen, Jazz Pharmaceuticals (advisory committee), Pfizer (honoraria); RA: BMS, Takeda, Janssen, Celgene (consultancy, research funding, honoraria); ADS: Janssen (consultancy), TeneoBio, Sanofi, Takeda (research funding), Imbrium, Akcea, Pfizer (honoraria); CL: Janssen (current employment); SC: Janssen (current employment); RP: SmartAnalyst (current employment); EA: Janssen (current employment, equity holder); NPT: Janssen (current employment, equity holder); AL: Janssen (current employment); SN: Janssen (current employment).

Data availability statement

The source data used for the present study were licenced from Optum, and hence are not publicly available. While the study investigators are not permitted to share the underlying patient-level data, other researchers may licence the databases used for the present study from Optum.

Additional information

Funding

This work was supported by Janssen Global Services, LLC.