Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
. 2019 Dec;187(5):588-594.
doi: 10.1111/bjh.16096. Epub 2019 Jul 12.

Ten-year survivors in AL amyloidosis: characteristics and treatment pattern

Eli Muchtar  1 , Morie A Gertz  1 , Martha Q Lacy  1 , Ronald S Go  1 , Francis K Buadi  1 , David Dingli  1 , Martha Grogan  2 , Omar F AbouEzzeddine  2 , Suzanne R Hayman  1 , Prashant Kapoor  1 , Nelson Leung  1   3 , Amie Fonder  1 , Miriam Hobbs  1 , Yi Lisa Hwa  1 , Wilson Gonsalves  1 , Rahma Warsame  1 , Taxiarchis V Kourelis  1 , Stephen Russell  1 , John A Lust  1 , Yi Lin  1 , Steven Zeldenrust  1 , Robert A Kyle  1 , S Vincent Rajkumar  1 , Shaji K Kumar  1 , Angela Dispenzieri  1
Affiliations

Ten-year survivors in AL amyloidosis: characteristics and treatment pattern

Eli Muchtar et al. Br J Haematol. 2019 Dec.

Abstract

Improvement in survival in Light chain (AL) amyloidosis has been seen over recent decades, enabling more patients to achieve long-term survival. Patients with AL amyloidosis who survived ≥10 years from time of diagnosis (n = 186) were the subject of this study. Ten-year survivors represented 22% of the total population. These patients were characterized by favourable patient, organ and plasma cell features. Of note, trisomies were less common among 10-year survivors compared to those who did not survive to 10 years. All-time best haematological response was complete response in 67%, very good partial response in 30%, partial response in 2% and no response in 1%, with 11% having received a consolidative strategy for inadequate response to first line therapy. The overall organ response rate to first-line therapy was 76%, which increased to 86% when considering subsequent line(s) of therapy. Forty-seven percent of the 10-year survivors did not require a second-line therapy. The median treatment-free survival (TFS) among the 10-year survivors was 10·5 years (interquartile range 7·4-12·2). On multivariate analysis independent predictors for TFS were the achievement of complete haematological response and lack of cardiac involvement. Long-term survivors are increasingly seen in AL amyloidosis and present distinct patient, organ and clonal disease features.

Keywords: mortality; outcome; prognosis; response; treatment; trend.

PubMed Disclaimer

Conflict of interest statement

Conflict-of-interest disclosure:

Eli Muchtar: None; Morie A. Gertz: Consultancy (Milleniu) and honoraria (Celgene, Millenium, Onyx, Novartis, Smith Kline, Prothena, Ionis). Martha Q. Lacy: Research funding (Celgene); Ronald S. Go: None; Francis Buadi: None; David Dingli: Research funding (Karyopharm Therapeutics, Amgen, Millenium Pharmaceuticals); Martha Grogan: None; Omar F. Abou Ezzeddine: None; Suzanne R. Hayman: None; Prashant Kapoor: Research funding (Takeda, Celgene, Amgen); Nelson Leung: None; Amie Fonder: None; Miriam Hobbs: None; Yi Lisa Hwa: None; Wilson Gonsalves: none; Rahma Warsame: None; Taxiarchis v. Kourelis: None; Stephen Russel: None; John A. Lust: None; Yi Lin: None; Steven Zeldenrust: None; Robert A. Kyle: None; Vincent Rajkumar: None; Shaji Kumar: Consultancy (Celgene, Millennium, Onyx, Janssen, BMS); and research funding (Celgene, Millennium, Novartis, Onyx AbbVie, Janssen, BMS). Angela Dispenzieri: Research funding (Celgene, Millennium, Pfizer, Janssen), Travel grant (Pfizer)

Figures

Figure 1:
Figure 1:
Time to next therapy
Figure 2:
Figure 2:
Treatment-free survival: A. Entire Cohort. B. By autologous stem cell transplantation (ASCT) and standard therapy sub-categorization
See this image and copyright information in PMC

Comment in

References

    1. Bochtler T, Hegenbart U, Kunz C, Granzow M, Benner A, Seckinger A, Kimmich C, Goldschmidt H, Ho AD, Hose D, Jauch A & Schonland SO (2015) Translocation t(11;14) is associated with adverse outcome in patients with newly diagnosed AL amyloidosis when treated with bortezomib-based regimens. Journal of clinical oncology : official journal of the American Society of Clinical Oncology, 33, 1371–1378. - PubMed
    1. Gertz MA, Comenzo R, Falk RH, Fermand JP, Hazenberg BP, Hawkins PN, Merlini G, Moreau P, Ronco P, Sanchorawala V, Sezer O, Solomon A & Grateau G (2005) Definition of organ involvement and treatment response in immunoglobulin light chain amyloidosis (AL): a consensus opinion from the 10th International Symposium on Amyloid and Amyloidosis, Tours, France, 18–22 April 2004. American journal of hematology, 79, 319–328. - PubMed
    1. Kyle RA & Gertz MA (1995) Primary systemic amyloidosis: clinical and laboratory features in 474 cases. Semin Hematol, 32, 45–59. - PubMed
    1. Kyle RA, Gertz MA, Greipp PR, Witzig TE, Lust JA, Lacy MQ & Therneau TM (1999) Long-term survival (10 years or more) in 30 patients with primary amyloidosis. Blood, 93, 1062–1066. - PubMed
    1. Lakshman A, Paul S, Rajkumar SV, Ketterling RP, Greipp PT, Dispenzieri A, Gertz MA, Buadi FK, Lacy MQ, Dingli D, Fonder AL, Hayman SR, Hobbs MA, Gonsalves WI, Hwa YL, Kapoor P, Leung N, Go RS, Lin Y, Kourelis TV, Warsame R, Lust JA, Russell SJ, Zeldenrust SR, Kyle RA & Kumar SK (2018) Prognostic significance of interphase FISH in monoclonal gammopathy of undetermined significance. Leukemia, 32, 1811–1815. - PubMed

Publication types

MeSH terms