|Paroxysmal Nocturnal Hemoglobinuria (PNH) is characterized by complement-mediated intravascular hemolysis, thrombophilia and Aplastic Anemia (AA). The treatment of PNH has been rebuilt by the anti-C5 eculizumab, which results in transfusion independence in half of the patients, while the others remain severely anemic due C3-mediated extravascular hemolysis and/or concomitant AA. The occurrence of severe AA is a possible event during the course of PNH and eventually hampers the hematological benefit from eculizumab. Treatment options include IST or Bone Marrow Transplantation.
A journal is a periodical publication intended to further progress of science, usually by reporting new research. Most journals are highly specialized, although some of the oldest journals publish articles, reviews, editorials, short communications, letters, and scientific papers across a wide range of scientific fields. Journals contain articles that peer reviewed, in an attempt to ensure that articles meet the journal's standards of quality, and scientific validity. Each such journal article becomes part of the permanent scientific record.