Survey on knowledge and perception of paroxysmal nocturnal hemoglobinuria among Colombian physicians: A cross-sectional study

Abstract

Introduction

Paroxysmal nocturnal hemoglobinuria (PNH) is a rare disease with severe clinical manifestations, for which diagnosis and treatment may be limited by medical knowledge and barriers to specialized care access.

Objective

To assess the perception and level of knowledge about PNH among physicians from different specialties in a national context.

Methods

A cross-sectional study was conducted using a structured survey targeting general practitioners, residents, and specialists. The questionnaire assessed six dimensions: signs and symptoms, pathophysiology, diagnosis, treatment, prognosis, and access to diagnosis and treatment. Knowledge perception was evaluated, and objective questions were included to measure the level of knowledge in each dimension. The internal consistency of the instrument was high, with an adequate Cronbach's alpha.

Results

Two hundred eighteen responses were received from a calculated sample of 355. Most surveyed physicians perceived their knowledge of PNH as low. However, objective knowledge assessment suggests a gap between general practitioners and specialists, with an overall knowledge level considered moderate. Hematologists demonstrated a high level of knowledge, but access barriers to specialized care may contribute to delayed diagnoses.

Conclusion

Using a standardized instrument, it is demonstrated that knowledge gaps regarding PNH across different levels of medical training could affect the early recognition of the disease. Therefore, it is recommended to strengthen medical education on PNH and improve strategies for access to specialized care to optimize disease management.