• Parameswaran Hari, Huamao Mark Lin, Carl V Asche, Jinma Ren, Candice Yong, Katarina Luptakova, Douglas V Faller, and Vaishali Sanchorawala.
• a Department of Medicine , Medical College of Wisconsin , Milwaukee , WI , USA.
• Amyloid. 2018 Mar 1; 25 (1): 1-7.

BackgroundTreatment 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.MethodsThis 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.ResultsOf 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).ConclusionsRRAL 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.

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