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Monitoring and Treatment of Paroxysmal Nocturnal Hemoglobinuria in Patients with Aplastic Anemia in Asia: An Expert
Raymond Siu Ming Wong1, Jun Ho Jang2, Lily Lee Lee Wong3
1Sir Y.K. Pao Centre for Cancer & Department of Medicine and Therapeutics, Prince of Wales Hospital, The Chinese University of Hong Kong, 30-32 Ngan Shing Street, Sha Tin, Hong Kong SAR, China.
Insights
Screening for paroxysmal nocturnal hemoglobinuria (PNH) clones in aplastic anemia (AA) patients is crucial. This expert panel offers practical Asia-Pacific guidelines for PNH/AA management, focusing on monitoring and tailored treatment strategies.
Area of Science:
- Hematology
- Internal Medicine
- Clinical Practice
Background:
- Paroxysmal nocturnal hemoglobinuria (PNH) clones are frequently found in patients diagnosed with aplastic anemia (AA).
- International guidelines recommend screening for PNH clones upon AA diagnosis.
- Managing co-existing PNH and AA requires specific strategies, particularly in the Asia-Pacific region.
Purpose of the Study:
- To provide expert recommendations for managing PNH in patients with AA within the Asia-Pacific region.
- To outline best practices for monitoring PNH clones and associated hemolysis.
- To guide treatment decisions based on the predominant condition (PNH or AA).
Main Methods:
- Expert panel discussion and consensus building on current best practices.
- Review of monitoring strategies including lactate dehydrogenase (LDH) levels, reticulocyte count, and PNH clone size via flow cytometry.
- Analysis of treatment algorithms based on predominant symptoms of hemolysis or bone marrow failure.
Main Results:
- Regular monitoring of LDH, reticulocyte count, and PNH clone size is recommended.
- Flow cytometry for PNH clone monitoring is advised, with on-demand testing triggered by clinical or laboratory changes.
- Treatment decisions should prioritize PNH guidelines for significant hemolysis and AA guidelines for severe bone marrow failure.
Conclusions:
- The expert panel's recommendations offer practical approaches for PNH/AA management in the Asia-Pacific region.
- Monitoring PNH clones can inform initial AA treatment choices.
- Tailored treatment based on the dominant clinical presentation is essential for effective patient care.
Abstract:
Paroxysmal nocturnal hemoglobinuria (PNH) clones can be identified in a significant proportion of patients with aplastic anemia (AA). Screening for PNH clones at the time of an AA diagnosis is recommended by national and international guidelines. In this report, an expert panel of physicians discusses current best practices and provides recommendations for managing PNH in patients with AA in the Asia-Pacific region. Plasma/serum lactate dehydrogenase (LDH) levels and reticulocyte count should be measured with every blood test. PNH clone size should be monitored regularly by flow cytometry, with on-demand testing in the event of a rise in LDH level ± reticulocyte count or development of symptoms such as thrombosis. Monitoring for PNH clones can guide the choice of initial AA treatment, although flow cytometry has resource implications which may present a challenge in some Asia-Pacific countries. The treatment of patients with both PNH and AA depends on which condition predominates; following PNH treatment guidelines if hemolysis is the main symptom and AA treatment guidelines if bone marrow failure is severe (regardless of whether hemolysis is mild or moderate). The expert panel's recommendations on the monitoring and treatment of PNH in patients with AA are practical for healthcare systems in the Asia-Pacific region.
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