Kastritis, Efstathios;
Misra, Arpit;
Gurskyte, Laura;
Kroi, Florint;
Verhoek, Andre;
Vermeulen, Jessica;
Ammann, Eric;
... Wechalekar, Ashutosh D; + view all
(2023)
Assessing the prognostic utility of hematologic response for overall survival in patients with newly diagnosed AL amyloidosis: results of a meta-analysis.
Hematology
, 28
(1)
, Article 2157581. 10.1080/16078454.2022.2157581.
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Abstract
Objectives: 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. Methods: Studies were identified via systematic literature review. Pooled effect estimates were generated by a random-effects model. Results: 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. Conclusions: 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.
| Type: | Article |
|---|---|
| Title: | Assessing the prognostic utility of hematologic response for overall survival in patients with newly diagnosed AL amyloidosis: results of a meta-analysis |
| Location: | England |
| Open access status: | An open access version is available from UCL Discovery |
| DOI: | 10.1080/16078454.2022.2157581 |
| Publisher version: | https://doi.org/10.1080/16078454.2022.2157581 |
| Language: | English |
| Additional information: | © 2023 The Author(s). Published by Informa UK Limited, trading as Taylor & Francis Group. This is an Open Access article distributed under the terms of the Creative Commons Attribution License (http://creativecommons.org/licenses/by/4.0/), which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited. |
| Keywords: | Science & Technology, Life Sciences & Biomedicine, Hematology, AL amyloidosis, hematologic response, overall survival, meta-analysis, systematic literature review, HIGH-DOSE MELPHALAN, PRIMARY SYSTEMIC AMYLOIDOSIS, DEXAMETHASONE, CYCLOPHOSPHAMIDE, BORTEZOMIB, OUTCOMES, IMPACT, TRANSPLANTATION, LENALIDOMIDE, CRITERIA |
| UCL classification: | UCL UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences > Div of Medicine UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences > Div of Medicine > Inflammation |
| URI: | https://discovery.ucl.ac.uk/id/eprint/10166160 |
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