Scholarlink

<p>Paroxysmal nocturnal hemoglobinuria (PNH) is a rare hematological disorder. Despite the improvements in clinical outcomes with C5 complement inhibitors, one of the drawbacks is the development of clinically significant extravascular hemolysis in a substantial number of patients. Pegcetacoplan, a proximal C3 complement inhibitor (C3i), was able to overcome this issue, which led to the approval of the drug based on data from PEGASUS trial. Most clinical trials exclude PNH patients presenting with acute thrombosis. We present a case of a 33-year-old male C5i-naive patient presenting with thrombosis. The clinical picture comprising of severe compensated intravascular hemolysis and atypical site thrombosis in the form of Budd-Chiari syndrome as well as flow cytometry analysis confirmed the diagnosis of classical PNH. Pegcetacoplan was given as initial therapy. The patient’s clinical outcomes improved after administering pegcetacoplan without any further progression of thrombosis. This report suggests that C3i in the form of pegcetacoplan can be used as initial therapy to treat PNH patients presenting with acute thrombosis.</p>

错误：搜索内容不能为空，请输入英文关键词
错误：关键词超出字数限制，请精简
高级检索

Improving Outcome of Paroxysmal Nocturnal Hemoglobinuria with Active Thrombosis by Pegcetacoplan: A Case Report

  • 期刊论文

  • 发表日期：

    2025年11月8日

查看全文
  • Hazzaa Alzahrani,
  • Yazeed S. Bujuaifer,
  • Ahmad Alzahrani,
  • Aisha Khan,
  • Raaina Mahevish

摘要

Paroxysmal nocturnal hemoglobinuria (PNH) is a rare hematological disorder. Despite the improvements in clinical outcomes with C5 complement inhibitors, one of the drawbacks is the development of clinically significant extravascular hemolysis in a substantial number of patients. Pegcetacoplan, a proximal C3 complement inhibitor (C3i), was able to overcome this issue, which led to the approval of the drug based on data from PEGASUS trial. Most clinical trials exclude PNH patients presenting with acute thrombosis. We present a case of a 33-year-old male C5i-naive patient presenting with thrombosis. The clinical picture comprising of severe compensated intravascular hemolysis and atypical site thrombosis in the form of Budd-Chiari syndrome as well as flow cytometry analysis confirmed the diagnosis of classical PNH. Pegcetacoplan was given as initial therapy. The patient’s clinical outcomes improved after administering pegcetacoplan without any further progression of thrombosis. This report suggests that C3i in the form of pegcetacoplan can be used as initial therapy to treat PNH patients presenting with acute thrombosis.