Related Experiment Video
Updated: Jan 12, 2026
04:44
Author Spotlight: Enhancing Vascular Function and Physical Capacity in Cardiovascular Disease Through Novel Interventions and NIRS Technology
Published on: March 22, 2024
1.4K
Paroxysmal Nocturnal Hemoglobinuria
Richard C Godby1, Surbhi Shah2
1Division of Hematology, Department of Medicine, Mayo Clinic, Rochester, MN.
Mayo Clinic Proceedings
|October 31, 2025
Summary
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare blood disorder causing red blood cell destruction. Early diagnosis and new treatments improve outcomes, though stem cell transplant offers a cure.
Area of Science:
- Hematology
- Immunology
- Genetics
Background:
- Paroxysmal nocturnal hemoglobinuria (PNH) is a rare acquired clonal disorder.
- Characterized by complement-mediated intravascular hemolysis, leading to anemia, fatigue, and thrombosis.
- Historically associated with high morbidity and mortality.
Purpose of the Study:
- To provide a contemporary review of paroxysmal nocturnal hemoglobinuria (PNH).
- Focusing on pathophysiology, epidemiology, clinical manifestations, diagnosis, treatment, and outcomes.
- Emphasis on intravascular hemolysis and advancements in targeted therapies.
Main Methods:
- Review of clinically relevant articles from MEDLINE.
- Synthesis of contemporary data on PNH.
- Focus on classic PNH features and current treatment landscape.
Main Results:
- Complement inhibitors have transformed PNH treatment, improving prognosis.
- Allogeneic hematopoietic stem cell transplant remains the only curative option.
- Early diagnosis and treatment are crucial for better outcomes.
Conclusions:
- PNH management has significantly improved due to targeted therapies.
- Ongoing research and novel treatments continue to enhance patient outlook.
- A comprehensive understanding of PNH is vital for effective clinical management.
Related Concept Videos
01:27Disorders of Erythrocytes
2.0K
Disorders of erythrocytes, or red blood cells (RBCs), include a range of conditions affecting their number, shape, or function.
Erythrocyte disorders can be broadly categorized into two main types: anemic and polycythemic conditions.
A low oxygen-carrying capacity of the blood due to the loss, lower production, or destruction of erythrocytes is termed anemia. Hemorrhagic anemia, for example, occurs when bleeding from an external wound or internal ulcer reduces erythrocyte counts.
On the other...
Erythrocyte disorders can be broadly categorized into two main types: anemic and polycythemic conditions.
A low oxygen-carrying capacity of the blood due to the loss, lower production, or destruction of erythrocytes is termed anemia. Hemorrhagic anemia, for example, occurs when bleeding from an external wound or internal ulcer reduces erythrocyte counts.
On the other...
2.0K
01:19Rh Blood Group
2.7K
The Rhesus (Rh) antigen is crucial in determining blood groups and ensuring compatibility during blood transfusions.
2.7K
01:14Erythropoiesis
5.7K
Red blood cells (RBCs) transport oxygen to all body tissues. These cells survive only for 120 days and then need to be replenished. Erythropoiesis is the process of RBC production. In healthy individuals, erythropoiesis ensures all tissues are amply supplied with oxygen. In addition, blood loss due to injury leads to a drop in the physiological oxygen level that will cause erythropoiesis. Any defect in erythropoiesis leads to several physiological disorders, including thalassemia, anemia,...
5.7K
02:45Blood Transfusion and Agglutination
14.0K
Blood transfusion is a therapeutic measure to restore the blood volume after extensive blood loss due to an accident or a medical procedure. Blood transfusion involves drawing a certain amount of blood from a suitable donor and infusing it into the recipient.
History
The history of blood transfusion dates back to the 17th century, when early attempts were made in animals. In 1818 James Blundell, a British doctor, performed the first successful human blood transfusion. Later in 1900, Karl...
History
The history of blood transfusion dates back to the 17th century, when early attempts were made in animals. In 1818 James Blundell, a British doctor, performed the first successful human blood transfusion. Later in 1900, Karl...
14.0K
01:22Lifecycle of Erythrocytes
4.6K
Erythrocytes, also known as red blood cells, constantly move through blood capillaries. As a result, they damage their plasma membrane due to the continuous friction. Typically, after 100 to 120 days, erythrocytes become rigid and fragile as they wear out. As they pass through small vessels in the spleen and liver, they can get trapped and break apart into fragments.
The resident phagocytic macrophages deal with these damaged cells by engulfing them and separating their globin and heme groups....
The resident phagocytic macrophages deal with these damaged cells by engulfing them and separating their globin and heme groups....
4.6K
01:27Oxygen Transport in the Blood
5.9K
Hemoglobin (Hb) is a crucial molecule in the human body, consisting of four polypeptide chains, each bound to an iron-containing heme group. This unique structure enables hemoglobin to bind to oxygen, with each molecule capable of combining with four molecules of oxygen, leading to rapid and reversible oxygen loading. When fully loaded with oxygen, it is called oxyhemoglobin, while hemoglobin that has released oxygen is called reduced hemoglobin or deoxyhemoglobin. As hemoglobin binds oxygen,...
5.9K