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
. 2021 Mar;106(3):354-361.
doi: 10.1111/ejh.13554. Epub 2020 Dec 4.

Characterization and prognostic implication of delayed complete response in AL amyloidosis

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

Characterization and prognostic implication of delayed complete response in AL amyloidosis

Eli Muchtar et al. Eur J Haematol. 2021 Mar.

Abstract

Introduction: Little is known on continued response following completion of therapy in light chain (AL) amyloidosis.

Methods: We studied 373 AL amyloidosis patients who achieved complete response (CR) or very good partial response (VGPR) to first-line therapy.

Results: By end of therapy (EOT), 46% of patients achieved a CR and 54% a VGPR. With no further therapy, 17.5% of patients were upstaged from VGPR to CR (delayed CR), with a median of 9 months. Compared with CR and VGPR at EOT, patients with a delayed CR were characterized by higher proportion of t(11;14) and lower rate of trisomies. Autologous stem cell transplant was more frequent in the delayed CR group. Patients with a delayed CR were characterized by minimal residual disease negativity and organ response rates similar to patients with CR at EOT and higher than patients achieving VGPR at EOT. Patients with a delayed CR had a longer PFS/OS compared to patients with CR or VGPR by EOT (median PFS 149 vs 92 vs 52 months, P < .001; 10-year OS 87% vs 71% vs 56%, P < .001).

Conclusions: This study characterizes delayed CR in AL amyloidosis, highlights its prognostic impact which is at least similar to those who achieved CR at EOT, and underlines another aspect of response monitoring.

Keywords: autologous stem cell transplant; immunoparesis; off-therapy; survival.

PubMed Disclaimer

Conflict of interest statement

CONFLICT OF INTEREST

Morie A. Gertz received consultancy from Millennium and honoraria from Celgene, Millennium, Onyx, Novartis, Smith Kline, Prothena, and Ionis. Shaji Kumar received consultancy from Celgene, Millennium, Onyx, Janssen, and BMS; and research funding from Celgene, Millennium, Novartis, Onyx AbbVie, Janssen, and BMS. Martha Q. Lacy received research funding from Celgene; David Dingli received research funding from Karyopharm Therapeutics, Amgen, and Millennium Pharmaceuticals; Prashant Kapoor received research funding from Takeda, Celgene, and Amgen; Angela Dispenzieri received research funding from Celgene, Millennium, Pfizer, and Alnylam, travel grant from Pfizer, and advisory board from Janssen. Eli Muchtar, Nelson Leung, Francis Buadi, Suzanne R. Hayman, Ronald S. Go, Wilson Gonsalves, Taxiarchis v. Kourelis, Rahma Warsame, Yi Lisa Hwa, Amie Fonder, Miriam Hobbs, Stephen Russel, John A. Lust, Mustaqueem Siddiqui, Vincent Rajkumar, and Robert A. Kyle have no conflict of interest.

Figures

FIGURE 1
FIGURE 1
Overall organ response stratified by the hematological response groups and assessed organs
FIGURE 2
FIGURE 2
Graded organ response stratified by the hematological response groups. A, Cardiac response. B, Renal response. C, Hepatic response
FIGURE 3
FIGURE 3
Kaplan-Meier curves of survival based on level of response. A, Progression-free survival (PFS) in the whole cohort. B, PFS in the ASCT sub-group. C, Overall survival (OS) in the whole cohort. D, OS in the ASCT sub-group
See this image and copyright information in PMC

Similar articles

See all similar articles

References

    1. Palladini G, Dispenzieri A, Gertz MA, et al. New criteria for response to treatment in immunoglobulin light chain amyloidosis based on free light chain measurement and cardiac biomarkers: impact on survival outcomes. J Clin Oncol. 2012;30(36):4541–4549. - PubMed
    1. Merlini G, Dispenzieri A, Sanchorawala V, et al. Systemic immunoglobulin light chain amyloidosis. Nat Rev Dis Primers. 2018;4(1):38. - PubMed
    1. Muchtar E, Dispenzieri A, Kumar SK, et al. Immunoparesis in newly diagnosed AL amyloidosis is a marker for response and survival. Leukemia. 2017;31(1):92–99. - PubMed
    1. Palladini G, Hegenbart U, Milani P, et al. A staging system for renal outcome and early markers of renal response to chemotherapy in AL amyloidosis. Blood. 2014;124(15):2325–2332. - PubMed
    1. Gertz MA, Comenzo R, Falk RH, et al. 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. Am J Hematol. 2005;79(4):319–328. - PubMed

MeSH terms