Case report: Transfusion independence and abolition of extravascular hemolysis in a PNH patient treated with pegcetacoplan

Letter Hematology

Experience of compassionate-use pegcetacoplan for paroxysmal nocturnal hemoglobinuria

Morag Griffin et al.

BLOOD (2023)

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Letter Hematology

Pegcetacoplan for refractory paroxysmal nocturnal haemoglobinuria associated with the C5 genetic variant

Shinobu Tamura et al.

BRITISH JOURNAL OF HAEMATOLOGY (2022)

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Article Hematology

Inhibition of C3 with pegcetacoplan results in normalization of hemolysis markers in paroxysmal nocturnal hemoglobinuria

Raymond S. M. Wong et al.

Summary: The study evaluated the safety and efficacy of the newly approved complement inhibitor, pegcetacoplan, in patients with PNH and found that it improved clinical indicators by controlling hemolysis.

ANNALS OF HEMATOLOGY (2022)

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Article Hematology

Somatic mutations and clonal expansions in paroxysmal nocturnal hemoglobinuria

Kohei Hosokawa et al.

Summary: Paroxysmal nocturnal hemoglobinuria (PNH) is an acquired hematopoietic stem cell disorder caused by a PIGA gene mutation. Clonal expansion of GPI(-) cells is common in PNH, but tiny GPI(-) cell populations can also be found in healthy individuals. In patients with acquired aplastic anemia, the expansion of PNH clones is related to immune attack on hematopoietic stem cells. However, the mechanisms underlying the selection and expansion of GPI(-) cells remain unclear.

SEMINARS IN HEMATOLOGY (2022)

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Review Hematology

Safety and efficacy of pegcetacoplan in paroxysmal nocturnal hemoglobinuria

Raymond S. M. Wong

Summary: This article reviews multiple clinical studies on the efficacy and safety of pegcetacoplan treatment in adults with PNH. The studies found that pegcetacoplan showed superior improvements in hemoglobin levels and other hematologic parameters, and effectively managed anemia and anemia-related complications.

THERAPEUTIC ADVANCES IN HEMATOLOGY (2022)

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Article Medicine, General & Internal

Pegcetacoplan versus Eculizumab in Paroxysmal Nocturnal Hemoglobinuria

Peter Hillmen et al.

Summary: The study demonstrated that Pegcetacoplan was superior to eculizumab in improving hemoglobin and clinical and hematologic outcomes in patients with PNH by providing broad hemolysis control, including control of intravascular and extravascular hemolysis.

NEW ENGLAND JOURNAL OF MEDICINE (2021)

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Article Medicine, General & Internal

Comparative effectiveness of pegcetacoplan versus ravulizumab in patients with paroxysmal nocturnal hemoglobinuria previously treated with eculizumab: a matching-adjusted indirect comparison

Rachel H. Bhak et al.

Summary: This study compared the effectiveness of C3 complement inhibitor pegcetacoplan and C5 complement inhibitor ravulizumab among PNH patients previously treated with eculizumab. Results showed that patients treated with pegcetacoplan had better outcomes in terms of transfusion avoidance, hemoglobin stabilization, LDH normalization, and blood transfusion reduction compared to patients receiving ravulizumab. Additionally, pegcetacoplan led to improvements in fatigue, global health status, physical functioning, and fatigue symptoms in comparison to ravulizumab.

CURRENT MEDICAL RESEARCH AND OPINION (2021)

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Article Hematology