Hematology

Paroxysmal Nocturnal Hemoblobinuria(PNH) and Myelodysplastic syndromes: Treatment Approach – pro

It is rare to have MDS and PNH coexist as two full blown disorders but PNH clones can be present in MDS and PNH can resemble some features of MDS. Among acquired stem cell disorders, pathological links between myelodysplastic syndromes (MDS) and aplastic anaemia (AA), and paroxysmal nocturnal haemoglobinuria (PNH) and AA, have been often described, whereas the relationship between MDS and PNH is not entirely clear. Many reports identified small PNH

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Exjade for transfusion iron overload and thalassemia – pro

Iron overload is a cumulative, potentially life-threatening, consequence of frequent blood transfusions or conditions such as thalassemia. Iron starts to build up in the body after as few as 10 transfusions because the body cannot remove it on its own. Iron chelation is the only effective drug treatment for transfusion-related iron overload. In thalassemia, ineffective erytrhopoieisis causes iron overload as well. In iron chelation, an agent binds

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ALSCT for MF – pro

Idiopathic myelofibrosis (IMF; also referred to as agnogenic myeloid metaplasia or myelosclerosis with myeloid metaplasia) is a chronic myeloproliferative disorder characterized by bone marrow fibrosis secondary to cytokine release from a clonal proliferation of hematopoietic stem cells and resulting in extramedullary hemopoiesis. Myeloablative allogeneic stem cell transplantation (allo-SCT) is the only curative treatment for myelofibrosis, although

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Arixtra and Leiden – pro

Factor V Leiden, or factor V G1691A, is a single-point mutation in the gene that codes for coagulation factor V.2 It involves a G (guanine)-to-A (adenine) substitution at nucleotide 1691 (G1691A) in exon 10, which predicts the replacement of arginine at amino acid residue 506 by glutamine (Arg506Gln). The mutation, transmitted through autosomal dominant inheritance, renders factor V resistant to inactivation by APC (a natural anticoagulant protein).

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Novoseven off-label – pro

Recombinant activated factor seven (VIIa) is a vitamin K-dependent glycoprotein that is structurally similar to human plasma-derived factor VIIa. It has received approval from the Food and Drug Administration (FDA) for the treatment of bleeding episodes in patients with hemophilia A or B who have developed inhibitors to factor VIII or factor IX, respectively. It has recently been approved in the EU for Glantzman's thromabasthenia. However, because

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IgG for CVI – pro

Common variable immune deficiency (CVID) is characterized by humoral immune deficiency with onset after 24 months of age and usually in young adulthood, resulting in increased susceptibility to infections and diminished responses to protein and polysaccharide vaccines. The most common infections are sinopulmonary and include Streptococcus pneumonia, Hemophilis influenza, Klebsiella pneumonia, and sometimes mycoplasma infections. Individuals may experience

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Soliris for PNH – pro

Soliris is the first therapy approved for paroxysmal nocturnal hemoglobinuria (PNH), a rare, disabling and life-threatening blood disorder defined by chronic red blood cell destruction, or hemolysis. Soliris is FDA indicated for the treatment of patients with PNH to reduce hemolysis and it was approved on March 16th of 2007. Soliris has proven to be a safe and effective therapy for PNH in three multi-national clinical studies: TRIUMPH, a placebo-controlled

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Eythropietics for anemia of chronic disease – pro

Anemia of chronic disease, the anemia that is the second most prevalent after anemia caused by iron deficiency, occurs in patients with acute or chronic immune activation. The condition has thus been termed "anemia of inflammation" but in many cases no obvious inflammatory casue is apparent. It is imperative to rule out iron deficincy coexistisng with chronic disease but in the presence of chronic disease, iron studies are unreliable and a bone marrow

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FUO – pro

Fever of unknown origin (FUO) refers to the presence of a documented fever for a specified time, for which a cause has not been found after a basic medical evaluation. The classic criteria developed in 1961 included: temperature greater than 101°F (38.3°C), for at least three weeks, and inability to find a cause after one week of study. Within the past decade, a revision has been proposed that categorizes FUO into classic, hospital acquired FUO,

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Soliris – pro

On Friday, March 16, 2007, the U.S. Food and Drug Administration (FDA) approved Soliris (eculizumab) for the treatment of paroxysmal nocturnal hemoglobinuria (PNH), a disorder in which abnormal breakdown of red blood cells occurs. Made by Alexion Pharmaceuticals, Soliris does not cure the disease, but treats the breakdown of the blood cells. Soliris has proven to be a safe and effective therapy for PNH in three multi-national clinical studies.  

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