Evaluating the Integration of a New PNH Treatment in the SUS: Public Input Welcome

Friday, December 29, 2023

AstraZeneca underscores the critical nature of Paroxysmal Nocturnal Hemoglobinuria (PNH), emphasizing its position among the approximately 7,000 rare diseases globally, each presenting unique challenges. PNH, characterized by life-threatening severity, stems from acquired somatic mutations in the PIG-A gene within stem cells. This mutation increases the vulnerability of red blood cells to destruction, leading to complications such as thrombosis, anemia, and damage to vital organs, significantly impacting patients' quality of life.

Typically affecting individuals aged 30 to 50, PNH exhibits no gender bias. The incidence is estimated to be between 1 and 10 cases per 1 million individuals over their lifetime. Current treatment strategies aim to mitigate red blood cell destruction, alleviate symptoms, and prevent or address thromboembolic episodes and associated complications. Administration of necessary medications often involves frequent hospital or clinic visits and extended procedural durations, particularly when delivered via infusion. Notably, allogeneic bone marrow transplantation remains the only curative option, though it is reserved for specific cases.

In response to these challenges, a recently ANVISA-approved treatment option for PNH emerges as a promising alternative. This new therapy, endorsed by AstraZeneca, not only promises enhanced quality of life for patients by reducing fatigue and thrombosis risk but also necessitates fewer infusions compared to the existing medication available in the SUS. This advancement allows for an extended infusion interval of every 8 weeks, providing a tangible improvement over the current standard of every 2 weeks.





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