Patients suffering from paroxysmal nocturnal hemoglobinuria ( PNH ) appear to be especially at risk for Budd Chiari syndrome, more than other forms of thrombophilia : up to 39 % develop venous thromboses and 12 % may acquire Budd-Chiari.
32.
A urine test strip showing positive for blood can also indicate hemoglobinuria, which is not detectable using a microscope due to the lysis of red blood cells in the urinary tract ( particularly in alkaline or dilute urine ), or intravascular hemolysis.
33.
"' Alexion Pharmaceuticals Inc . "'is an American pharmaceutical company best known for its development of Soliris, a drug used to treat the rare disorders atypical hemolytic uremic syndrome ( aHUS ) and paroxysmal nocturnal hemoglobinuria ( PNH ).
34.
Many patients with aplastic anemia also have clones of cells characteristic of the rare disease paroxysmal nocturnal hemoglobinuria ( PNH, anemia with thrombopenia and / or thrombosis ), sometimes referred to as AA / PNH . Occasionally PNH dominates over time, with the major manifestation intravascular hemolysis.
35.
They are usually associated with multiple sclerosis or pertussis, but they may also be observed in other trauma, stroke, asthma, trigeminal neuralgia, breath-holding spells, epilepsy, malaria, tabes dorsalis, and Beh�et's disease, paroxysmal nocturnal hemoglobinuria ( PNH ).
36.
"' David L . Hallal "'is the former President and Chief Executive Officer ( CEO ) of Alexion Pharmaceuticals, the American pharmaceutical company best known for its development of Soliris, a drug used to treat the rare disorders atypical hemolytic uremic syndrome ( aHUS ) and paroxysmal nocturnal hemoglobinuria ( PNH ).
37.
Decreased platelet numbers may be due to various causes, including insufficient production ( e . g ., in myelodysplastic syndrome or other bone marrow disorders ), destruction by the immune system ( immune thrombocytopenic purpura / ITP ), and consumption due to various causes ( thrombotic thrombocytopenic purpura / TTP, hemolytic-uremic syndrome / HUS, paroxysmal nocturnal hemoglobinuria / PNH, disseminated intravascular coagulation / DIC, heparin-induced thrombocytopenia / HIT ).
38.
In December 2014 the provincial government of Ontario, Canada was in ongoing price negotiations with the Soliris manufacturer, the only drug approved by Health Canada to treat aHUS . Patients who require the apply for it on " compassionate grounds " " on a case-by-case basis for individuals who have been urgently hospitalized due to an immediate life-, limb-, or organ-threatening complication . " Soliris is already " funded by the Ontario government for the treatment of another rare illness, paroxysmal nocturnal hemoglobinuria ( PNH ), through a bulk-buy deal reached by the provincial premiers in 2011 ."
39.
Eculizumab was approved by the U . S . Food and Drug Administration ( FDA ) on March 13, 2007 for the treatment of paroxysmal nocturnal hemoglobinuria ( PNH ), a rare, progressive, and sometimes life-threatening disease characterized by excessive hemolysis; and on September 23, 2011 for the treatment of atypical hemolytic uremic syndrome ( aHUS ) It was approved by the European Medicines Agency for the treatment of PNH on June 20, 2007, and on November 29, 2011 for the treatment of aHUS . However, of note is the exceedingly high cost of treatment, with one year of the drug costing over $ 500, 000.
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