Paroxysmal Nocturnal Hemoglobinuria: From Bench to Bed.
Paroxysmal nocturnal hemoglobinuria (PNH) is an acquired hemolytic anemia with highly variable clinical symptoms making the diagnosis and prediction of its outcome difficult. It is caused by the expansion of a hematopoietic progenitor cell that has acquired a mutation in the X-linked phosphatidylinositol glycan class A (PIGA) gene that results in deficiency of the glycosylphosphatidylinositol anchor structure responsible for fixing a wide spectrum of proteins particularly CD55 and CD59. The clinical features of this disease arise as a result of complement-mediated hemolysis in unprotected red cells, leukocytes, and platelets as well as the release of free hemoglobin. Patients may present with a variety of clinical manifestations, such as anemia, thrombosis, kidney disease, smooth muscle dystonias, abdominal pain, dyspnea, and extreme fatigue. PNH is an outstanding example of how an increased understanding of pathophysiology may directly improve clinical symptoms and treat disease-associated complications when we inhibit the terminal complement cascade. This topic will discuss PNH overview to assist specialists looking after PNH patients.
Medical Oncology Department, Faculty of Medicine, Zagazig University, Zagazig, Egypt ; Oncology Center, King Abdullah Medical City-Holy Capital, Makkah, Saudi Arabia ; Muzdallifa Street, P.O. Box 57657, Makkah, 21995 Saudi Arabia.
Amrallah A
AA
Mohammed
Oncology Center, King Abdullah Medical City-Holy Capital, Makkah, Saudi Arabia.
Hani
H
El-Tanni
Faculty of Medicine, Umm Al-Qura University, Makkah, Saudi Arabia.
Tariq Al-Malki
TA
Atiah
Faculty of Medicine, Umm Al-Qura University, Makkah, Saudi Arabia.
Arwa Al-Malki
AA
Atiah
Faculty of Health Administration, Umm Al-Qura University, Makkah, Saudi Arabia.
Marwan Al-Malki
MA
Atiah
Medical Oncology Department, Faculty of Medicine, Zagazig University, Zagazig, Egypt.
Ayman A
AA
Rasmy
Complement; Paroxysmal; Phosphatidylinositol