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December 8, 2025BloodOpen Access

An open-label multicenter Phase 1/2 trial of venetoclax-dexamethasone in Relapsed/Refractory t(11;14)-positive systemic AL amyloidosis

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Authors

ADAnita D’SouzaSBSusan BalOAOthman Salim Akhtar

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Overview

Phase 1/2 trial demonstrated significant hematologic overall response in relapsed AL amyloidosis patients, suggesting potential for frontline treatment options.

Key Points

  • Hematologic overall response rate reached 91.7% among participants, indicating strong efficacy of treatment.
  • Patients experienced favorable outcomes with no dose-limiting toxicities reported during this phase.
  • The maximum tolerated dose identified was 400 mg of venetoclax combined with 20 mg of dexamethasone weekly.
  • Further investigation is warranted for venetoclax-based combinations in newly diagnosed systemic AL amyloidosis.

Cite This Study

D’Souza et al. (2025) studied this question.

synapsesocial.com/papers/69362f6e4fa91c937236e097https://doi.org/10.1182/blood-2025-694
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