A Validated Composite Organ and Hematologic Response Model For Early Assessment of Treatment Outcomes In Light Chain Amyloidosis

Authors

Surbhi Sidana
Paolo Milani
Moritz Binder
Marco Basset
Nidhi Tandon
Andrea Foli
Angela Dispenzieri
Morie A Gertz
Suzanne R Hayman
Francis K Buadi
Martha Q Lacy
Prashant Kapoor
Nelson Leung
S Vincent Rajkumar
Giampaolo Merlini
Giovanni Palladini
Shaji K Kumar

Language

English

Publication Date

4-14-2020

Journal

Blood Cancer Journal

DOI

10.1038/s41408-020-0306-5

PMID

32286270

PMCID

PMC7156647

PubMedCentral® Posted Date

April 2020

PubMedCentral® Full Text Version

Post-print

Abstract

Newly diagnosed AL amyloidosis patients were evaluated to develop a model for early assessment of treatment benefit at 6 months, integrating both hematologic (HR) and organ response (OR) assessment (testing cohort, Mayo: n = 473; validation cohort, Pavia: n = 575). Multiple OR were assessed as follows: All OR (AOR): response in all organs, mixed OR (MOR): response in some organs, no OR (NOR)]. AOR rates at 6 months improved with deepening HR; complete response (CR; 38%, 35%), very good partial response (VGPR; 30%, 26%), and partial response (PR; 16%, 21%), respectively. A composite HR/OR (CHOR) model was developed using incremental scoring based on hazard ratios with scores of 0-3 for HR (0-CR, 1-VGPR, 2-PR, 3-no response) and 0-2 for OR (0-AOR, 1-MOR, 2-NOR). Patients could be divided into two distinct CHOR groups (scores 0-3 and 4-5), with median OS in group 1 and group 2: Not reached vs. 34 months, p < 0.001 [Mayo] and 87 vs. 23 months, p < 0.001 [Pavia]. In conclusion, we developed a model that can assess multiple organs concurrently, and integrate both HR and OR assessments to determine early clinical benefit with treatment, which may be used as a surrogate end-point in trials and to compare outcomes with different therapies.

Keywords

Aged, Antineoplastic Agents, Cohort Studies, Combined Modality Therapy, Female, Hematologic Tests, Hematopoietic Stem Cell Transplantation, Humans, Immunoglobulin Light-chain Amyloidosis, Male, Middle Aged, Organ Dysfunction Scores, Remission Induction, Survival Rate, Treatment Outcome

Published Open-Access

yes

Recommended Citation

Sidana, Surbhi; Milani, Paolo; Binder, Moritz; et al., "A Validated Composite Organ and Hematologic Response Model For Early Assessment of Treatment Outcomes In Light Chain Amyloidosis" (2020). Faculty, Staff and Student Publications. 1976.
https://digitalcommons.library.tmc.edu/uthmed_docs/1976