A prospective randomised controlled trial of a single intravenous infusion of ferric carboxymaltose, versus single intravenous iron polymaltose or daily oral ferrous sulphate in the treatment of iron deficiency anaemia in pregnancy
Iron deficiency anaemia (IDA) is the most common nutritional deficiency affecting pregnant women worldwide. (Source: Seminars in Hematology)
Source: Seminars in Hematology - April 25, 2018 Category: Hematology Authors: Alhossain Khalafallah, Annemarie Hyppa, Anthony Chuang, Fayez Hanna, Emily Wilson, Christine Kwok, Carl Yan, Zara Gray, Ronnie Mathew, Peter Falloon, Amanda Dennis, Toly Pavlov, John Carson Allen Source Type: research
Fetal Hemoglobin Induction by Epigenetic Drugs
Fetal hemoglobin (HbF) inhibits the root cause of sickle pathophysiology, sickle hemoglobin polymerization. Individuals who naturally express high levels of HbF beyond infancy thus receive some protection from sickle complications. To mimic this natural genetic experiment using drugs, one guiding observation was that HbF is increased during recovery of bone marrow from extreme stress. This led to evaluation and approval of the cytotoxic (cell killing) drug hydroxyurea to treat sickle cell disease. (Source: Seminars in Hematology)
Source: Seminars in Hematology - April 20, 2018 Category: Hematology Authors: Donald Lavelle, James Douglas Engel, Yogen Saunthararajah Tags: Review Source Type: research
Fetal Hemoglobin Induction by Epigenetic Drugs ☆
Fetal hemoglobin (HbF) inhibits the root cause of sickle pathophysiology, sickle hemoglobin polymerization. Individuals who naturally express high levels of HbF beyond infancy thus receive some protection from sickle complications. To mimic this natural genetic experiment using drugs, one guiding observation was that HbF is increased during recovery of bone marrow from extreme stress. This led to evaluation and approval of the cytotoxic (cell killing) drug hydroxyurea to treat sickle cell disease (SCD). (Source: Seminars in Hematology)
Source: Seminars in Hematology - April 20, 2018 Category: Hematology Authors: Donald Lavelle, James Douglas Engel, Yogen Saunthararajah Source Type: research
Anticomplement Treatment in Atypical and Typical Hemolytic Uremic Syndrome
The dissection of the pathogenic mechanisms of the various forms of the hemolytic uremic syndrome (HUS) has paved the way for the design of specific efficacious treatments. Such mechanistic approach led to a revolution in the management of atypical HUS with the use of the first-in class C5 blocker, eculizumab. The availability of this anticomplement drug has also raised unsettled questions regarding the cost or burden and optimal duration of therapy and its use in secondary HUS. The efficacy of eculizumab in Shiga toxin producing Escherichia coli-associated HUS is not to date established and the results of ongoing prospect...
Source: Seminars in Hematology - April 19, 2018 Category: Hematology Authors: Fadi Fakhouri, Chantal Loirat Tags: Review Source Type: research
Novel Sickle Cell Disease Therapies: Targeting Pathways Downstream of Sickling
Sickle cell disease (SCD) is an inherited hemoglobinopathy characterized by hemolytic anemia, frequent painful episodes, poor quality of life, end organ damage and a shortened lifespan. While the seminal event is the polymerization of the abnormal hemoglobin, the downstream pathophysiology of vaso occlusion (VOC) results from heterotypic interactions between the altered, adhesive sickle cell RBC ′s, neutrophils, endothelium and platelets. Ischemia reperfusion injury, hemolysis and oxidant damage all contribute to heightened inflammation and activation of the hemostatic system. (Source: Seminars in Hematology)
Source: Seminars in Hematology - April 19, 2018 Category: Hematology Authors: Kerry Morrone, William Beau Mitchell, Deepa Manwani Source Type: research
Anti-complement treatment in atypical and typical hemolytic uremic syndrome
The dissection of the pathogenic mechanisms of the various forms of the haemolytic uremic syndrome (HUS) has paved the way for the design of specific efficacious treatments. Such mechanistic approach led to a revolution in the management of atypical HUS with the use of the first-in class C5 blocker, eculizumab. The availability of this anti-complement drug has also raised unsettled questions regarding the cost/burden and optimal duration of therapy and its use in secondary HUS. The efficacy of eculizumab in Shiga toxin producing E coli-associated-HUS is not to date established and the results of ongoing prospective studies...
Source: Seminars in Hematology - April 19, 2018 Category: Hematology Authors: Fadi Fakhouri, Chantal Loirat Source Type: research
Novel Sickle Cell Disease Therapies: Targeting Pathways Downstream of Sickling
Sickle cell disease (SCD) is an inherited hemoglobinopathy characterized by hemolytic anemia, frequent painful episodes, poor quality of life, end organ damage and a shortened lifespan. While the seminal event is the polymerization of the abnormal hemoglobin, the downstream pathophysiology of vaso occlusion (VOC) results from heterotypic interactions between the altered, adhesive sickle cell RBC ′s, neutrophils, endothelium and platelets. Ischemia reperfusion injury, hemolysis and oxidant damage all contribute to heightened inflammation and activation of the hemostatic system. (Source: Seminars in Hematology)
Source: Seminars in Hematology - April 19, 2018 Category: Hematology Authors: Kerry Morrone, William Beau Mitchell, Deepa Manwani Source Type: research
Anti-complement treatment in atypical and typical hemolytic uremic syndrome
The dissection of the pathogenic mechanisms of the various forms of the haemolytic uremic syndrome (HUS) has paved the way for the design of specific efficacious treatments. Such mechanistic approach led to a revolution in the management of atypical HUS with the use of the first-in class C5 blocker, eculizumab. The availability of this anti-complement drug has also raised unsettled questions regarding the cost/burden and optimal duration of therapy and its use in secondary HUS. The efficacy of eculizumab in Shiga toxin producing E coli-associated-HUS is not to date established and the results of ongoing prospective studies...
Source: Seminars in Hematology - April 19, 2018 Category: Hematology Authors: Fadi Fakhouri, Chantal Loirat Source Type: research
Somatic mutations in Philadelphia chromosome-negative myeloproliferative neoplasms
Myeloproliferative neoplasms (MPN) include polycythemia vera (PV), essential thrombocythemia (ET), and primary myelofibrosis (PMF). MPN are characterized by clonal proliferation of myeloid progenitors leading to erythrocytosis, thrombocytosis and/or leukocytosis, and risk of hemorrhagic and thrombotic events, as well as myelofibrosis and blast transformation.The discovery of somatic mutations in MPN, namely JAK2 V617F, JAK2 exon 12, MPL, and CALR mutations, has permitted a more specific approach to diagnosis and treatment. (Source: Seminars in Hematology)
Source: Seminars in Hematology - April 17, 2018 Category: Hematology Authors: S érgio Ferreira Cristina, Blanca Polo, João F. Lacerda Source Type: research
Congenital defects in the expression of the glycosylphosphatidylinositol-anchored complement regulatory proteins CD59 and decay-accelerating factor
CD59 and decay-accelerating factor (DAF) are glycosylphosphatidylinositol (GPI)-anchored complement regulatory proteins critical for regulating complement activation on the host cell surface. Defective expressions of CD59 and/or DAF caused by mutations in the genes coding for these proteins or genes involved in the biosynthesis of GPI, such as PIGA, PIT and PIGM, are associated with various clinical symptoms that are mediated by dysregulated activation of complement, especially the C5 component. (Source: Seminars in Hematology)
Source: Seminars in Hematology - April 16, 2018 Category: Hematology Authors: Taroh Kinoshita Source Type: research
Complement in Pathophysiology and Treatment of Transplant-Associated Thrombotic Microangiopathies
Transplant-associated thrombotic microangiopathy (TA-TMA) is a form of microangiopathy specifically occurring in the context of hematopoietic stem cell transplantation. Similarly, to other microangiopathies, TA-TMA is characterized by hemolytic anemia, thrombocytopenia, and organ failure due to endothelial injury. Although its clinical association with medications (eg, calcineurin inhibitors), immune reactions (eg, graft vs host disease) or infectious complications is well established, the pathophysiology remains largely unknown. (Source: Seminars in Hematology)
Source: Seminars in Hematology - April 11, 2018 Category: Hematology Authors: Sonata Jodele Tags: Review Article Source Type: research
Complement in pathophysiology and treatment of transplant-associated thrombotic microangiopathies (TA-TMA)
Transplant-associated thrombotic microangiopathy (TA-TMA) is a form of microangiopathy specifically occurring in the context of hematopoietic stem cell transplantation (HSCT). Similarly, to other microangiopathies, TA-TMA is characterized by hemolytic anemia, thrombocytopenia, and organ failure due to endothelial injury. While its clinical association with medications (e.g., calcineurin inhibitors), immune reactions (e.g., graft versus host disease) or infectious complications is well established, the pathophysiology remains largely unknown. (Source: Seminars in Hematology)
Source: Seminars in Hematology - April 11, 2018 Category: Hematology Authors: Sonata Jodele Source Type: research
