Rare disease education
PNH beyond haematology
Paroxsymal nocturnal haemoglobinuria
A PODCAST SERIES
Paroxysmal nocturnal haemoglobinuria (PNH) is an acquired, rare, chronic, potentially life-threatening haematologic disease characterised by haemolysis, bone marrow failure and thrombosis.1-2 PNH is associated with patient burden and multiple clinical manifestations, including persistently low haemoglobin, anaemia, fatigue, and thrombosis, all of which impact morbidity, mortality and quality of life for patients.2-5 Beyond haematological consequences, haemolysis and anaemia may lead to pathophysiological changes resulting in multisystem complications.6-8
Podcast Episodes
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Episode 1
Uncovering the common multi-system consequences of a rare disease – chronic kidney disease
A podcast with Prof Elena Solomou, Haematologist and PNH expert and Prof Alberto Ortiz, Nephrologist provides an overview of PNH as a complex rare disease, explores the need for better understanding of extra-haematological complications, focusing on the impact on kidney disease and addresses ways to support cross-specialty care between haematologists and nephrologists.
Preparation:
May 2025
NP-41349 -
Episode 2
Uncovering the common multi-system consequences of a rare disease – cardiorespiratory disease
A podcast with Dr Alex Pike, Haematologist and PNH expert and Dr Jivendra Gosai, Cardiologist, to be released in July 2025
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Episode 3
Uncovering the common multi-system consequences of a rare disease – the multidisciplinary team
A podcast with Dr Danny Hsu, Haematologist and PNH expert and Surabhi Chaturvedi, Senior Haematology Psychotherapist to be released by the end of 2025
References
1. Hill A, Kelly RJ, Hillmen P. Thrombosis in paroxysmal nocturnal hemoglobinuria. Blood 2013;121:4985–4996 2. Hill A, DeZern AE, Kinoshita T, et al. Paroxysmal nocturnal haemoglobinuria. Nat Rev Dis Primers 2017 3. Kelly RJ, Holt M, Vidler J, et al. Treatment outcomes of complement protein C5 inhibition in 509 UK patients with paroxysmal nocturnal hemoglobinuria. Blood 2024;143:1157–1166 4. Meyers G, Weitz I, Lamy T, et al. Disease-related symptoms reported across a broad population of patients with paroxysmal nocturnal hemoglobinuria. Blood 2007;110:3683 5. Schrezenmeier H, et al. Baseline clinical characteristics and disease burden in patients with paroxysmal nocturnal hemoglobinuria (PNH): updated analysis from the International PNH Registry. Ann Hematol 2020;99:1514; 6. Hill A, Rother RP, Wang X, et al. Effect of eculizumab on haemolysis-associated nitric oxide depletion, dyspnoea, and measures of pulmonary hypertension in patients with paroxysmal nocturnal haemoglobinuria. Br J Haematol 2010;149:414–425 7. Socié G, Schrezenmeier H, Muus P, et al. Changing prognosis in paroxysmal nocturnal haemoglobinuria disease subcategories: An analysis of the International PNH Registry. Intern Med J 2016;46:1044–1053. 8. Hillmen P, Elebute M, Kelly R, et al. Long-term effect of the complement inhibitor eculizumab on kidney function in patients with paroxysmal nocturnal hemoglobinuria. Am J Hematol 2010;85:553–559