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December 8, 2025Blood

Disease monitoring and management among pediatric patients with pyruvate kinase deficiency: Real-world practices from pyruvate kinase deficiency registries prior to 2024 international expert guidelines

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Authors

AGAndreas GlenthoejUniversity of Copenhagen BGBertil GladerStanford Medicine YYYan YanFirst Hospital of China Medical University

Discussion

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Implication

Retrospective analysis reveals gaps in disease monitoring and management among pediatric patients with pyruvate kinase deficiency, indicating need for improved care practices.

Key Points

Disease monitoring and management practices vary significantly among pediatric patients with pyruvate kinase deficiency.
Iron overload screening was properly conducted for all regularly transfused patients, with 90.6% having a history of IO.
Observational analysis from two registries highlights gaps in vitamin D monitoring and endocrine function checks for NRT patients.
Consistent care and monitoring improvements are essential for managing pediatric patients with PK deficiency effectively.

Cite This Study

Glenthoej et al. (2025) studied this question.

synapsesocial.com/papers/69362f714fa91c937236e215 https://doi.org/10.1182/blood-2025-4454

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Implication

Retrospective analysis reveals gaps in disease monitoring and management among pediatric patients with pyruvate kinase deficiency, indicating need for improved care practices.

Disease monitoring and management among pediatric patients with pyruvate kinase deficiency: Real-world practices from pyruvate kinase deficiency registries prior to 2024 international expert guidelines

Discussion

Member takes

Implication

Key Points

Cite This Study

Disease monitoring and management among pediatric patients with pyruvate kinase deficiency: Real-world practices from pyruvate kinase deficiency registries prior to 2024 international expert guidelines

Discussion

Member takes

Implication

Key Points

Cite This Study

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