Immunopathology of Autoimmune Hemolytic Anemia
Autoimmune Hemolytic AnemiaAutoimmune hemolytic anemia (AIHA) is an auto-immune disease mediated by specific antibodies targeting red blood cells. Its pathogenesis is not completely understood, and the role of T cells have been rarely studied. The aim of this study is to compare the frequency of circulating T cells, T cell polarization and functions, notably regulatory T cells, during warm AIHA by comparison to healthy controls. The role of treatments, such as steroids, will also be determined in patients with warm AIHA.
Long Term Effects of Erythrocyte Lysis
Sickle Cell DiseaseHemolytic AnemiaIn this prospective observational trial, participants with chronic hemolysis will be assessed with echocardiogram for elevated tricuspid jet velocity and other evidence of pulmonary hypertension. Participants will have laboratory studies evaluating: severity of hemolysis, splenic function, inflammation, endothelial dysfunction, and hypercoagulability. There will be 3 main categories of participants enrolled in this study: (1) pediatric participants with severe sickle cell disease (SCD) (HbSS, HbS/β° thalassemia ) who are not receiving treatment (e.g., hydroxyurea or chronic transfusions); (2) pediatric participants with other forms of SCD or severe SCD (HbSS, HbS/β° thalassemia) patients being treated with hydroxyurea or chronic transfusions; and (3) pediatric and adult participants with other non-sickling hematological disorders.
Demographic, Clinical and Laboratory Characteristics of Children With Alpha Thalassemia in Northern...
Thalassemia AlphaHemolytic AnemiaThe study intends to summarize the clinical and laboratory characteristics of children with hemolytic anemia diagnosed as having alpha thalassemia mutations.
Pyruvate Kinase Deficiency Natural History Study
Pyruvate Kinase DeficiencyCongenital Non-Spherocytic Hemolytic AnemiaThe purpose of this study is to describe the range and incidence of symptoms, treatments, and complications related to pyruvate kinase deficiency (PKD). Eligible patients are those of all ages with known PKD or with a hemolytic anemia and a family member with PKD. The study will collect retrospective medical history, routine clinical care data, and quality of life measures at baseline and annually for patients with PKD.
The Role of Microparticles as a Biomarker
Atypical Hemolytic Uremic SyndromeThrombotic Thrombocytopenic Purpura2 moreThe investigators propose to characterize MPs in aHUS and TTP both at the onset and throughout treatment. The investigators believe that the number, size, and cell origin of MPs will differ between these two diseases. The hypothesis is that endothelial derived MPs will be higher in number and comprise a larger portion of the MP population in aHUS and that platelet MPs will comprise a larger number and greater proportion of MPs in TTP. The investigators believe that MP identity and number can be used to reliably differentiate between aHUS and TTP at disease onset.
Post-trial Access for Nipocalimab in Participants With Warm Autoimmune Hemolytic Anemia (wAIHA)...
Warm Autoimmune Hemolytic AnemiaThe purpose of this post-trial access (PTA) program is to provide nipocalimab for the treatment of participants with Warm Autoimmune Hemolytic Anemia (wAIHA) who are experiencing clinical benefit after completing 28-weeks open-label extension in MOM-M281-006 (NCT04119050) study.
Gall Bladder Status Among Children With Chronic Haemolytic Anemia
Gall Bladder DiseaseGall bladder status among children with chronic haemolytic anemia attending to Assuit University Children Hospital. Hemolysis is defined as the premature destruction of red blood cells (RBCs) (a shortened RBC life span). Anemia results when the rate of destruction exceeds the capacity of the marrow to produce RBCs. Normal RBC survival time is 110-120 days (half-life: 55-60 days), and thus, approximately 0.85% of the most senescent RBCs are removed and replaced each day. Patients with chronic haemolytic anemia are subjected to many complications of chronic haemolytic anemia e.g anemic heart failure, complications of blood transfusion as hepatitis and AIDS, hypersplenism, haemosiderosis ,among them there is incidence of gallbladder stone formation. This work aims to a) to determine the prevalence of gall bladder diseases among patient with chronic haemolytic anemia. b) to determine the risk factors of gall bladder diseases among patients with chronic haemolytic anemia.
Role of T Follicular Helper Cells in Autoimmune Hemolytic Anemia (TFH in AIHA)
Autoimmune Hemolytic AnemiaT follicular helper (TFH) cells represent a T cell subset dedicated to the activation of B cells. They have been involved in the pathogenesis of autoimmune diseases in humans such as lupus and Sjögren disease. We recently showed that TFH are implicated in the activation of autoreactive B lymphocytes during ITP. Autoimmune hemolytic anemia (AIHA) is an autoimmune disease due to antibodies targeting red blood cells. To date, the role of TFH in the pathogenesis of AIHA is not known. We hypothesize that AIHA is associated with an increase in the number and/or function of TFH, that could participate in the activation of autoreactive B lymphocytes.