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In hematology, plasma cell dyscrasias (also termed plasma cell disorders and plasma cell proliferative diseases) are a spectrum of progressively more severe monoclonal gammopathies in which a clone or multiple clones of pre-malignant or malignant plasma cells (sometimes in association with lymphoplasmacytoid cells or B lymphocytes) over-produce and secrete into the blood stream a myeloma ...
The treatment was approved in the United Kingdom for the treatment of sickle cell disease and transfusion-dependent beta thalassemia in November 2023. [9] [10] [11] It was approved in the United States for the treatment of sickle cell disease in December 2023 and for the treatment of transfusion-dependent beta thalassemia in January 2024. [8 ...
Polycythemia is defined as serum hematocrit (Hct) or hemoglobin (HgB) exceeding normal ranges expected for age and sex, typically Hct >49% in healthy adult men and >48% in women, or HgB >16.5 g/dL in men or >16.0 g/dL in women. [8]
Diamond–Blackfan anemia (DBA) is a congenital erythroid aplasia that usually presents in infancy. [3] DBA causes low red blood cell counts (), without substantially affecting the other blood components (the platelets and the white blood cells), which are usually normal.
Hemoglobin A2 (HbA 2) is a normal variant of hemoglobin A that consists of two alpha and two delta chains (α 2 δ 2) and is found at low levels in normal human blood.Hemoglobin A2 may be increased in beta thalassemia or in people who are heterozygous for the beta thalassemia gene.
Isosthenuria may be seen in disease states as chronic kidney disease and acute kidney injury in which the kidneys lack the ability to concentrate or dilute the urine and so the initial filtrate of the blood remains unchanged despite the need to conserve or excrete water based on the body's hydration status.
Voxelotor has been shown to have disease-modifying potential by increasing hemoglobin levels and decreasing hemolysis indicators in sickle cell patients. [8] It has a safe profile in sickle cell patients and healthy volunteers, without any dose-limiting toxicity. [9] It was developed by Global Blood Therapeutics, a subsidiary of Pfizer. [10]
In diseases such as sickle cell anemia, blood is removed and replaced with donor blood. [5] In conditions such as neonatal polycythemia, a specific amount of the child’s blood is removed and replaced with normal saline, plasma (the clear liquid portion of blood), or an albumin solution.