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. 2015 Oct;32(10):920-8.
doi: 10.1007/s12325-015-0250-0. Epub 2015 Oct 23.

Light Chain Amyloidosis: Patient Experience Survey from the Amyloidosis Research Consortium

Isabelle Lousada  1 , Raymond L Comenzo  2 , Heather Landau  3 , Spencer Guthrie  4 , Giampaolo Merlini  5
Affiliations

Light Chain Amyloidosis: Patient Experience Survey from the Amyloidosis Research Consortium

Isabelle Lousada et al. Adv Ther. 2015 Oct.

Abstract

Introduction: Information detailing the experience of patients with light chain (AL) amyloidosis is lacking. The primary aim of this study was to gather data on the patient experience to understand the challenges in diagnosis and to gain insight into barriers to accessing appropriate care.

Methods: Patients with amyloidosis, family members, and caregivers were invited to participate in an online 16-question survey (available from January 29 to February 5, 2015). Participants with AL amyloidosis were sent an eight-question follow-up survey.

Results: The initial survey was completed by 533 participants (follow-up survey completed by 201 participants). AL amyloidosis was the most common diagnosis. For 37.1% of respondents, the diagnosis of amyloidosis was not established until ≥ 1 year after the onset of initial symptoms. Diagnosis was received after visits to 1, 2, 3, 4, or ≥ 5 physicians by 7.6%, 23.5%, 20.3%, 16.8%, and 31.8% of respondents, respectively. Correct diagnosis was most often made by hematologists/oncologists (34.1%). Treatments included chemotherapy (63.1%) and stem cell transplantation (38.9%) and were difficult to tolerate for 54.1% of respondents. A significant number of respondents felt uninformed about clinical trials. Nevertheless, approximately half (46.1%) believed that enrolling in a trial would enhance their care.

Conclusions: Establishing a diagnosis of amyloidosis is difficult. Current treatments are difficult to tolerate and do not substantially improve quality of life for most patients. There is an urgent need for well-tolerated therapies with clear treatment benefit. Patient awareness of clinical trials can be improved, especially given that respondents indicated high willingness to participate.

Keywords: Disease awareness; Early diagnosis; Patient survey; Systemic amyloidosis; Treatment.

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Figures

Fig. 1
Fig. 1
Types of physicians visited before diagnosis of amyloidosis
Fig. 2
Fig. 2
Clinical trial awareness and interest
See this image and copyright information in PMC

References

    1. Merlini G, Bellotti V. Molecular mechanisms of amyloidosis. N Engl J Med. 2003;349:583–596. doi: 10.1056/NEJMra023144. - DOI - PubMed
    1. Sanchorawala V. Light-chain (AL) amyloidosis: diagnosis and treatment. Clin J Am Soc Nephrol. 2006;1:1331–1341. doi: 10.2215/CJN.02740806. - DOI - PubMed
    1. Merlini G, Comenzo RL, Seldin DC, Wechalekar A, Gertz MA. Immunoglobulin light chain amyloidosis. Exp Rev Gastroenterol Hepatol. 2014;7:143–156. - PubMed
    1. Comenzo RL, Reece D, Palladini G, Seldin D, Sanchorawala V, Landau H, et al. Consensus guidelines for the conduct and reporting of clinical trials in systemic light-chain amyloidosis. Leukemia. 2012;26:2317–2325. doi: 10.1038/leu.2012.100. - DOI - PubMed
    1. Merlini G, Wechalekar AD, Palladini G. Systemic light chain amyloidosis: an update for treating physicians. Blood. 2013;121:5124–5130. doi: 10.1182/blood-2013-01-453001. - DOI - PubMed

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