Recognizing the Burden of Paroxysmal Nocturnal Hemoglobinuria

Announcer:
You’re listening to Project Oncology on ReachMD, and this episode is sponsored by Novartis. Here’s your host, Dr. Brian McDonough.

Dr. McDonough:
Welcome to Project Oncology on ReachMD. I’m Dr. Brian McDonough and today, we’ll be taking a closer look at the disease burden of paroxysmal nocturnal hemoglobinuria, also known as PNH.

PNH is a rare, acquired, chronic disorder of hematopoietic stem cells that can result in a variety of complications.¹ Some of the most common ones include fatigue, which can be disabling, smooth muscle dystonia, which is often seen in the form of abdominal pain, hemoglobinuria, and chronic kidney disease.^1,2 Life-threatening complications may include thrombosis and bone marrow failure, with thrombotic events being the leading cause of mortality.¹ If left untreated, patients with PNH have a ten-year mortality rate of 29 percent.¹

For patients, these complications can impact quality of life and employment, and result in hospitalizations, as well as the need for transfusion therapy.³

Lastly, delayed diagnosis remains a significant challenge for patients with PNH. In fact, 79 percent of patients see more than one physician to obtain a diagnosis.⁴ And overall, almost one-quarter of PNH cases take at least five years to diagnose.4 That’s why the importance of an early and accurate diagnosis cannot be understated as it can help us alleviate the burden of PNH on our patients. And for ReachMD, I’m Dr. Brian McDonough.

Announcer:
This episode of Project Oncology was sponsored by Novartis. To access this and other episodes in this series, visit ReachMD.com/Project Oncology, where you can Be Part of the Knowledge. Thanks for listening!

References:

1. Bektas M et al. J Manag Care Spec Pharm. 2020;26(12-b Suppl):S3-S8. doi:10.18553/jmcp.2020.26.12-b.s3
2. Schrezenmeier H et al. Ann Hematol. 2020;99(7):1505-1514. doi:10.1007/s00277-020-04052-z
3. Schrezenmeier H et al. Haematologica. 2014;99(5):922-929. doi:10.3324/haematol.2013.093161
4. Mitchell R et al. SM Clin Med Oncol. 2017;1(1):1001. Accessed July 19, 2023. https://www.researchgate.net/profile/Ellen-Salkeld/publication/331412219_Path_to_Diagnosis_of_Paroxysmal_Nocturnal_Hemoglobinuria_The_Results_of_an_Exploratory_Study_Conducted_by_the_Aplastic_Anemia_and_MDS_International_Foundation_and_the_National_Organization_for_Rare_Di/links/5c781f66458515831f780d3f/Path-to-Diagnosis-of-Paroxysmal-Nocturnal-Hemoglobinuria-The-Results-of-an-Exploratory-Study-Conducted-by-the-Aplastic-Anemia-and-MDS-International-Foundation-and-the-National-Organization-for-Rare-Di.pdf