Understanding PNH | More to PNH

PNH is characterized by hemolysis, thrombosis, and bone marrow failure

PNH is a rare, acquired clonal disorder of hematopoietic stem cells caused by a somatic mutation in the PIG-A gene. In PNH, red blood cells (RBCs) become deficient in the proteins CD55 and CD59, which are required to protect them from complement-mediated hemolysis.^1-3

PNH varies in presentation and is often associated with the following clinical conditions^4,5:

Hemolysis

Coombs-negative hemolytic anemia
Hemoglobinuria

Unexplained/unusual thrombosis

Venous or arterial

Bone marrow failure syndromes

Aplastic anemia
Myelodysplastic syndrome
Unexplained cytopenias

PNH by the numbers

The estimated global prevalence of PNH is ~10 to 20 per million individuals²

The estimated global incidence of PNH is ~1 to 2 per million individuals²

Median age at disease onset is 36 years⁷

PNH impacts males and females equally icon

Affects male and female patients equally⁸

Hemolysis in PNH creates a high burden of disease for patients

In an analysis of data from the International PNH Registry,* untreated patients with PNH (N=4439) had extensive histories of physician-reported symptoms and complications, including⁷:

Signs & Symptoms

Complications

Hb, hemoglobin; MOD, mechanism of disease; PIG-A, phosphatidylinositol glycan class A; PNH, paroxysmal nocturnal hemoglobinuria.

References: 1. Dingli D et al. Ann Hematol. 2022;101(2):251-263. 2. Cançado RD et al. Hematol Transfus Cell Ther. 2021;43(3):341-348. 3. Brodsky RA. Blood. 2009;113(26):6522-6527. 4. Patriquin CJ et al. Eur J Haematol. 2019;102(1):36-52. doi:101111/ejh.13176 5. Sharma VR. Clin Adv Hematol Oncol. 2013;(suppl11):13(9):2-8. 6. Sahin F et al. Am J Blood Res. 2016;6(2):19-27. 7. Schrezenmeier H et al. Ann Hematol. 2020;99(7):1505-1514. 8. Jalbert JJ et al. Blood. 2019;134(suppl1):3407. doi:10.1182/blood-2019-125867 9. Levy AR et al. Blood. 2019;134(suppl1):4803.

PNH is characterized by hemolysis, thrombosis, and bone marrow failure

PNH varies in presentation and is often associated with the following clinical conditions4,5:

PNH by the numbers

Hemolysis in PNH creates a high burden of disease for patients

There is more to PNH than you may think

Ongoing hemolysis and treatments can impact patients1,9

Explore medical expert perspectives on PNH

PNH varies in presentation and is often associated with the following clinical conditions^4,5:

Ongoing hemolysis and treatments can impact patients^1,9