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December 8, 2025Blood

Investigating belumosudil in children with refractory moderate-to-severe chronic graft-versus-host disease: Design of a phase 1/2 study

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Authors

ABAdriana Balduzzi

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Overview

Trial evaluates safety and efficacy of belumosudil in children with refractory chronic graft-versus-host disease, suggesting alternate treatment options.

Key Points

  • Overall response rate assessed by Week 24 in participants aged 1 to <18 years with moderate-to-severe cGvHD.
  • Multicenter open-label study investigating belumosudil administering a daily oral formulation for systemic therapy.
  • Key inclusion requires prior hematopoietic stem cell transplantation and moderate-to-severe cGvHD as defined by NIH criteria.
  • Study aims for safety evaluation and long-term follow-up to improve treatment outcomes for pediatric patients.

Cite This Study

Adriana Balduzzi (2025) studied this question.

synapsesocial.com/papers/69362f3a4fa91c937236d404https://doi.org/10.1182/blood-2025-7753
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