Treatment patterns and health care resource utilization among patients with relapsed/refractory systemic light chain amyloidosis

Abstract

Background: Treatment for patients with systemic light chain (AL) amyloidosis remains challenging. Our study aims to describe treatment patterns for both newly diagnosed and relapsed/refractory AL (RRAL) amyloidosis, and to assess clinical outcomes, healthcare costs, and resource utilization during the first year following a diagnosis of RRAL amyloidsis.

Methods: This was a retrospective observational study of adult patients with AL amyloidosis using the US Optum administrative claims data during 1/1/2008 to 6/30/2015. Diagnosis was based on both ICD-9 codes and treatments with a claim for AL-amyloidosis-specific anticancer systemic agents.

Results: Of 334 patients with AL amyloidosis, 43.1% were considered as RRAL amyloidosis. The majority (75%) of RRAL amyloidosis patients had organ involvement prior to the second line treatment. Proteasome-inhibitor-based regimens were most frequently used (41.0% for first-line AL, 30.6% for RRAL amyloidosis). Organ deterioration and mortality rates were 49.3% and 10.4%, respectively, during the two years following relapse. The average monthly cost was $14,369 per patient for RRAL amyloidosis including medical costs ($9441) and drug costs ($4928).

Conclusions: RRAL amyloidosis is associated with high morbidity from target organ failure and mortality, which emphasizes the need for novel medications to improve care for patients with RRAL amyloidosis.

Keywords:

• Systemic light chain amyloidosis
• relapsed
• refractory
• mortality
• clinical outcomes
• healthcare costs
• resource utilization

Disclosure statement

CVA, PH, VS, and JR are consultants for Millennium Pharmaceuticals, Inc., a wholly owned subsidiary of Takeda Pharmaceutical Company Limited, Cambridge, MA, USA. HML, CY, and DF are employed by Millennium Pharmaceuticals, Inc., a wholly owned subsidiary of Takeda Pharmaceutical Company Limited, Cambridge, MA, USA. KL was employed by Millennium Pharmaceuticals, Inc., a wholly owned subsidiary of Takeda Pharmaceutical Company Limited, Cambridge, MA, USA at the time of development of finalization of this manuscript and is currently an employee of TESARO. The roles of authors include study design (PH, HML, and CY), statistical analysis (CVA and JR), results interpretation (PH, HML, CY, KL, DF, and VS), and manuscript draft and revision (all authors).

Additional information

Funding

This study was sponsored by Millennium Pharmaceuticals, Inc., a wholly owned subsidiary of Takeda Pharmaceutical Company Limited, Cambridge, MA.