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December 8, 2025BloodOpen Access

A phase 1/2a trial of amsulostat, a novel pan-lysyl oxidase inhibitor, in patients with advanced myelofibrosis as an add-on to ruxolitinib treatment for up to 52 weeks

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Authors

RBRoss BakerSCStanley CheungHHHui-Hua Hsiao

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Overview

Phase 1/2a trial shows improved tolerability and safety for amsulostat in myelofibrosis, suggesting enhanced treatment options.

Key Points

  • Improvements in symptom scores and tolerability observed with amsulostat combined with ruxolitinib in myelofibrosis.
  • At 38 weeks, 75% of patients achieved significant reduction in total symptom score (TSS50).
  • Assessment through a multi-center trial analyzed safety and tolerability of amsulostat as an add-on treatment.
  • Supports progression to larger confirmatory trials based on safety and additional efficacy outcomes.

Cite This Study

Baker et al. (2025) studied this question.

synapsesocial.com/papers/69362f4b4fa91c937236d7c7https://doi.org/10.1182/blood-2025-2027
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Also Consider

Synapse has enriched 5 closely related papers on similar clinical questions. Consider them for comparative context:

  1. 1Phase 3 randomized double-blind study evaluating selinexor, an XPO1 inhibitor, plus ruxolitinib in jaki-naïve myelofibrosis: Study rationale and preliminary patient characteristics2025
  2. 2A phase I study of the combination of ruxolitinib and the ERK1/2 inhibitor ulixertinib in previously treated myelofibrosis patients.2025
  3. 3Durable efficacy and long-term safety with pelabresib plus ruxolitinib in JAK Inhibitor–Naive myelofibrosis: 96-week Results from the Phase III MANIFEST-2 study2025 · 6 citations
  4. 4Preliminary analysis of a phase 2b, randomized, active-control study of flonoltinib maleate (FM) vs. ruxolitinib in intermediate-2 or high-risk myelofibrosis patients2025 · 1 citations
  5. 5Real-world treatment duration of ruxolitinib and use of transfusion among 2268 patients with myelofibrosis: An analysis of the Medicare fee-for-service claims database2025