Academic Journal
Assessing the prognostic utility of hematologic response for overall survival in patients with newly diagnosed AL amyloidosis: results of a meta-analysis
| Title: | Assessing the prognostic utility of hematologic response for overall survival in patients with newly diagnosed AL amyloidosis: results of a meta-analysis |
|---|---|
| Authors: | Efstathios Kastritis, Arpit Misra, Laura Gurskyte, Florint Kroi, Andre Verhoek, Jessica Vermeulen, Eric Ammann, Annette Lam, Sarah Cote, Ashutosh D. Wechalekar |
| Source: | Hematology, Vol 28, Iss 1 (2023) |
| Publisher Information: | Taylor & Francis Group, 2023. |
| Publication Year: | 2023 |
| Collection: | LCC:Diseases of the blood and blood-forming organs |
| Subject Terms: | AL amyloidosis, hematologic response, overall survival, meta-analysis, systematic literature review, Diseases of the blood and blood-forming organs, RC633-647.5 |
| More Details: | ABSTRACTObjectives: 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. |
| Document Type: | article |
| File Description: | electronic resource |
| Language: | English |
| ISSN: | 16078454 1607-8454 |
| Relation: | https://doaj.org/toc/1607-8454 |
| DOI: | 10.1080/16078454.2022.2157581 |
| Access URL: | https://doaj.org/article/4c7982a23cd841e7bbdd0d78214e0fd9 |
| Accession Number: | edsdoj.4c7982a23cd841e7bbdd0d78214e0fd9 |
| Database: | Directory of Open Access Journals |
| Full text is not displayed to guests. | Login for full access. |
| ISSN: | 16078454 |
|---|---|
| DOI: | 10.1080/16078454.2022.2157581 |
| Published in: | Hematology |
| Language: | English |