How I treat autoimmune hemolytic anemia

תאריך: 01/06/2017

The diagnosis, prognosis, and management of autoimmune hemolytic anemia (AIHA) continue to be challenging in current practice. This is related to an incomplete understanding of the pathophysiology of the disease process, complexity of initiating factors, and a lack of evidencebased standardized therapies. There is no completely validated and standard therapeutic approach to AIHA, because randomized clinical trials are difficult to implement.

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Pathophysiology, diagnosis, and treatment of paroxysmal nocturnal hemoglobinuria: a review

תאריך: 30/06/2015

Paroxysmal nocturnal hemoglobinuria (PNH) is an acquired disorder of the hematopoietic stem cell that makes blood cells more sensitive to the action of complement. Patients experience intravascular hemolysis, smooth muscle dystonia, renal failure, arterial and pulmonary hypertension, recurrent infectious diseases and an increased risk of notably dreadful thrombotic complications. The diagnosis is made by flow cytometry. Efforts have been recently performed to improve the sensitivity and the standardization of this technique. PNH is frequently associated with aplastic anemia or low-risk myelodysplasia and may be asymptomatic. Management of the classical form of PNH has been dramatically revolutionized by the development of eculizumab, which brings benefits in terms of hemolysis, quality of life, renal function, thrombotic risk, and life expectancy. Prophylaxis and treatment of arterial and venous thrombosis currently
remain a challenge in PNH.
European Journal of Haematology 95 (190–198) doi:10.1111/ejh.12543

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Paroxysmal nocturnal hemoglobinuria

תאריך: 30/10/2014

Paroxysmal nocturnal hemoglobinuria (PNH) is a rare bonemarrow failure disorder that manifests with hemolytic anemia, thrombosis, and peripheral blood cytopenias. The absence of two glycosylphosphatidylinositol
(GPI)-anchored proteins, CD55 and CD59, leads to uncontrolled complement activation that accounts for hemolysis and other PNH manifestations.
GPI anchor protein deficiency is almost always due to somatic mutations in phosphatidylinositol glycan class A (PIGA), a gene involved in the first step of GPI anchor biosynthesis; however, alternative mutations that cause PNH have recently been discovered. In addition, hypomorphic germ-line PIGA mutations that do not cause PNH have been shown to be responsible for a condition known as multiple congenital anomalies-hypotoniaseizures syndrome 2. Eculizumab, a firstin-class monoclonal antibody that inhibits terminal complement, is the treatment of choice for patients with severe manifestations of PNH. Bone marrow transplantation remains the only cure for PNH but should be reserved for patients with suboptimal response to eculizumab. (Blood. 2014;124(18):2804-2811)

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Abnormalities of the Erythrocyte Membrane

תאריך: 30/12/2013

Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, clonal, hematopoietic stem cell disorder that manifests with a hemolytic anemia from uncontrolled complement activation, bone marrow failure, and a propensity for thrombosis.
Hematol Oncol Clin North Am. 2015 June ; 29(3): 479–494. doi:10.1016/j.hoc.2015.01.005.

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New insights into childhood autoimmune hemolytic anemia: a French national observational study of 265 children

תאריך: 01/06/2011

Since 2004, a national observational study has been aiming to thoroughly describe cases and identify prognostic factors. Patients from all French hematologic pediatric units have been included if they had a hemoglobin concentration less than 11 g/dL, a positive direct antiglobulin test and hemolysis. Evans’ syndrome was defined by the association of autoimmune hemolytic anemia and immunological thrombocytopenic purpura. Data from patients' medical records were registered from birth to last follow-up. Autoimmune hemolytic anemia was classified as primary or secondary. Remission criteria, qualifying the status of anemia at last follow-up, were used with the aim of identifying a subgroup with a favorable prognosis in continuous complete remission.

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