# Expert consensus on clonal screening and monitoring of complement inhibitor therapy in paroxysmal nocturnal hemoglobinuria (2024)

## TL;DR

This paper presents expert consensus on screening and monitoring for PNH, a rare blood disorder, during complement inhibitor therapy to improve patient outcomes.

## Contribution

The paper provides a consensus on best practices for PNH screening and monitoring during complement inhibitor treatment.

## Key findings

- Clonal screening and rapid diagnosis are critical for identifying PNH patients needing complement inhibitor therapy.
- Monitoring for breakthrough hemolysis and extravascular hemolysis during treatment is essential for improving survival and quality of life.
- The consensus integrates recent research and expert experience to guide clinical practice in PNH management.

## Abstract

阵发性睡眠性血红蛋白尿症（paroxysmal nocturnal hemoglobinuria，PNH）是一种罕见的造血干细胞异常克隆性疾病，以血管内溶血性贫血、血栓形成和外周血细胞减少为主要表现，呈慢性进展性病程，严重者可危及生命。补体抑制剂是治疗PNH溶血相关症状的一线推荐药物。随着补体抑制剂领域的快速发展，加强对PNH的筛查、快速诊断，判断需要用补体抑制剂治疗的患者，在补体抑制剂治疗过程中监测突破性溶血、血管外溶血等，对患者的生存、生活质量改善有着重要意义。为促进PNH临床诊疗的规范，本共识参考国内外最新指南和文献，荟萃国内外最新研究成果，并结合专家团队经验，聚焦 PNH筛查原则、PNH克隆检测意义、末端C5补体抑制剂治疗后监测等问题，旨在为PNH的筛查、诊断和补体抑制剂时代的治疗监测提供参考意见。