Assessing the prognostic utility of hematologic response for overall survival in patients with newly diagnosed AL amyloidosis: results of a meta-analysis

Efstathios Kastritis; Arpit Misra; Laura Gurskyte; Florint Kroi; Andre Verhoek; Jessica Vermeulen; Eric Ammann; Annette Lam; Sarah Cote; Ashutosh D. Wechalekar

doi:10.1080/16078454.2022.2157581

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Assessing the prognostic utility of hematologic response for overall survival in patients with newly diagnosed AL amyloidosis: results of a meta-analysis

Journal article

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Assessing the prognostic utility of hematologic response for overall survival in patients with newly diagnosed AL amyloidosis: results of a meta-analysis

Efstathios Kastritis, Arpit Misra, Laura Gurskyte, Florint Kroi, Andre Verhoek, Jessica Vermeulen, Eric Ammann, Annette Lam, Sarah Cote and Ashutosh D. Wechalekar

Hematology (Luxembourg), Vol.28(1), pp.2157581-2157581

12/31/2023

DOI: 10.1080/16078454.2022.2157581

PMID: 36607151

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Abstract

Amyloid light-chain (AL) amyloidosis is a rare disease characterized by amyloid fibril deposits made up of toxic light chains causing progressive organ dysfunction and death. Recent studies suggest that hematologic response may be an important prognostic indicator of overall survival (OS) in AL amyloidosis. The aim of this study was to evaluate the trial-level association between hematologic complete response (CR) or very good partial response or better (≥ VGPR) and OS in newly diagnosed patients. Studies were identified via systematic literature review. Pooled effect estimates were generated by a random-effects model. Nine observational studies reporting hematologic CR or ≥VGPR and OS hazard ratios (HRs) were included in the meta-analysis. Achieving hematologic CR was associated with improved OS (HR, 0.21; 95% confidence interval [CI] 0.13-0.34). Achieving ≥ VGPR was also associated with improved OS (HR 0.21; 95% CI 0.17-0.26). Results of a sensitivity analysis excluding one outlier study revealed no heterogeneity and a better overall HR estimate. Potential limitations of this meta-analysis include the small number of eligible studies (consistent with the rarity of the disease) and inconsistencies in reporting of results. Overall, our findings support the use of deep hematologic response (CR or ≥VGPR) as a clinical trial endpoint in newly diagnosed AL amyloidosis. This study provides evidence that early hematologic response is a strong patient-level surrogate for long-term OS in patients with AL amyloidosis receiving frontline therapy. Structured data collection of depth of response in future trials will further strengthen these observations.

AL amyloidosis

hematologic response

meta-analysis

overall survival

systematic literature review

Details

Title: Subtitle: Assessing the prognostic utility of hematologic response for overall survival in patients with newly diagnosed AL amyloidosis: results of a meta-analysis
Creators: Efstathios Kastritis - National and Kapodistrian University of Athens
Arpit Misra
Laura Gurskyte
Florint Kroi
Andre Verhoek
Jessica Vermeulen
Eric Ammann
Annette Lam
Sarah Cote
Ashutosh D. Wechalekar - University College London
Resource Type: Journal article
Publication Details: Hematology (Luxembourg), Vol.28(1), pp.2157581-2157581
DOI: 10.1080/16078454.2022.2157581
PMID: 36607151
NLM abbreviation: Hematology
ISSN: 1024-5332
eISSN: 1607-8454
Publisher: Taylor & Francis
Grant note: Janssen Global Services (10.13039/100010412)
Language: English
Date published: 12/31/2023
Academic Unit: Epidemiology
Record Identifier: 9984363620202771

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