Severe Aplastic Anemia (SAA)

Also known as: Severe Aplastic Anemia

DrugDrug NameDrug Description
DB09312Antilymphocyte immunoglobulin (horse)Equine anti-thymocyte globulin is composed of purified gamma globulin containing primarily IgG against human thymus lymphocytes. It is formed by inoculating a horse with an antigen (human thymoyctes) which then induces the horse immune system's B-lymphocytes to produce IgG immunoglobulins specific for that antigen. The result is polyclonal IgG that is then purified from the horse's serum to produce a usable drug product that can be used for immunosuppression. Although the exact mechanism of action is unknown, equine anti-thymocyte globulin targets a variety of immune system proteins including lymphocyte surface proteins, granulocytes, platelets, bone marrow cells, and other cell types. Equine ATG is currently indicated for the suppression of the immune system to prevent renal transplant rejection and in the treatment of aplastic anemia. Induction of T cell apoptosis and resulting T-cell lymphopenia found in vivo is credited for its therapeutic effect in these conditions. There are currently various ATG products available, which differ in the source of inoculated animal (rabbit, horse, or pig) and in the type of antigen product used to produce immunoglobulin (thymocytes, peripheral T cells, etc.).
DB06626AxitinibAxitinib is a second generation tyrosine kinase inhibitor that works by selectively inhibiting vascular endothelial growth factor receptors (VEGFR-1, VEGFR-2, VEGFR-3).[L6676] Through this mechanism of action, axitinib blocks angiogenesis, tumour growth and metastases. It is reported to exhibit potency that is 50-450 times higher than that of the first generation VEGFR inhibitors.[L6676] Axitinib is an indazole derivative.[A179398] It is most commonly marketed under the name Inlyta® and is available in oral formulations.
DB06210EltrombopagEltrombopag is used to treat low blood platelet counts in adults with chronic immune (idiopathic) thrombocytopenia (ITP), when certain other medicines, or surgery to remove the spleen, have not worked well enough. ITP is a condition that may cause unusual bruising or bleeding due to an abnormally low number of platelets in the blood. Eltrombopag has also been recently approved (late 2012) for the treatment of thrombocytopenia (low blood platelet counts) in patients with chronic hepatitis C to allow them to initiate and maintain interferon-based therapy.
DB06273TocilizumabTocilizumab is a recombinant, humanized, anti-human interleukin 6 (IL-6) receptor monoclonal antibody that achieves a significant therapeutic response rate. The light chain is made up of 214 amino acids. The heavy chain is made up of 448 amino acids. The four polypeptide chains are linked intra- and inter-molecularly by disulfide bonds. FDA approved on January 8, 2010. Tocilizumab (injection) was further approved by the FDA for the treatment of adults with giant cell arteritis, an inflammation of the blood vessels (vasculitis) in May, 2017. In a double-blind, placebo-controlled study, the patients achieved sustained remission from Week 12 through Week 52, which was associated with significant improvements in symptoms of giant cell arteritis, normalization of inflammatory laboratory tests and tapering the use of corticosteroids [L943].
DrugDrug NamePhaseStatusCount
DB00087Alemtuzumab1 / 2Terminated1
DB09312Antilymphocyte immunoglobulin (horse)1 / 2Recruiting1
DB00531Cyclophosphamide1 / 2Completed2
DB00091Cyclosporine1 / 2Completed1
DB00091Cyclosporine1 / 2Recruiting1
DB06210Eltrombopag1 / 2Recruiting1
DB01073Fludarabine1 / 2Completed1
DB01073Fludarabine1 / 2Terminated1
DB00087Alemtuzumab2Active Not Recruiting1
DB00098Antithymocyte immunoglobulin (rabbit)2Recruiting4
DB09110Coenzyme M2Recruiting1
DB00531Cyclophosphamide2Active Not Recruiting1
DB00091Cyclosporine2Active Not Recruiting1
DB06210Eltrombopag2Active Not Recruiting1
DB01073Fludarabine2Unknown Status1
DB00688Mycophenolate mofetil2Recruiting4
DB01024Mycophenolic acid2Recruiting1
DB09312Antilymphocyte immunoglobulin (horse)3Active Not Recruiting1
DB06210Eltrombopag3Active Not Recruiting1
DB00098Antithymocyte immunoglobulin (rabbit)4Recruiting1
DB00092AlefaceptNot AvailableTerminated1
DB09312Antilymphocyte immunoglobulin (horse)Not AvailableRecruiting1
DB00098Antithymocyte immunoglobulin (rabbit)Not AvailableRecruiting1
DB00098Antithymocyte immunoglobulin (rabbit)Not AvailableWithdrawn1
DB00531CyclophosphamideNot AvailableRecruiting1
DB00531CyclophosphamideNot AvailableWithdrawn1
DB00091CyclosporineNot AvailableRecruiting1
DB01073FludarabineNot AvailableRecruiting1
DB01073FludarabineNot AvailableWithdrawn1
DB00563MethotrexateNot AvailableRecruiting1