Immune Thrombocytopenia

Abstract

Immune thrombocytopenia (ITP) is a common hematologic disorder characterized by isolated thrombocytopenia. ITP presents as a primary form characterized by isolated thrombocytopenia (platelet count < 100 × 109/L) in the absence of other causes or disorders that may be associated with thrombocytopenia, or a secondary form in which immune thrombocytopenia develops in association with another disorder that is usually immune or infectious. ITP may affect individuals of all ages, with peaks during childhood and in the elderly, in whom the age specific incidence of ITP is greatest. Bleeding is the most common clinical manifestation of ITP, with the risk of bleeding and related morbidity increased in elderly patients. The pathogenesis of ITP is complex, involving alterations in humoral and cellular immunity. Thrombocytopenia is caused by antibodies that react with glycoproteins expressed on platelets and megakaryocytes (glycoprotein IIb/IIIa, Ib/IX and others), causing shortened survival of circulating platelets and impairing platelet production. Diminished numbers and function of regulatory T cells, as well as the effects of cytotoxic T cells also contribute to the pathogenesis of ITP. Corticosteroids remain the most common first line therapy for ITP, occasionally in conjunction with intravenous immunoglobulin (IVIg) and anti-Rh(D). However, these agents do not lead to durable remissions in the majority of adults with ITP, and considerable heterogeneity exists in the use of second line approaches, which may include splenectomy, Rituximab, or thrombopoietin receptor agonists (TRAs). This review summarizes the classification and diagnosis of primary and secondary ITP, as well as the pathogenesis and options for treatment. Remarkable advances in the understanding and management of ITP have been achieved over the last decade, though many questions remain.

Fostamatinib for Persistent/Chronic Adult Immune Thrombocytopenia

“…is an orally bioavailable prodrug that is metabolized into a biologically active form, R406 (N4-2,2-dimethyl-3-oxo-4H-pyrid [1,4]oxazin-6-yl)-5-fluoro-N2-(3,4,5-trimethoxyphenyl)-2,4-pyrimidinediamine), a selective small molecule inhibitor of Syk (Figure 5) [31,46]. R406 was initially identified as a relatively specific inhibitor of Fc R-dependent mast cell activation in a cell-based screen for structure activity relationships with primary human mast cells [37].…”

“…In adults, the prevalence of ITP increases with age [6]. Some reports have suggested that incidence is slightly higher in women of reproductive age and the elderly [4,5].…”

“…The combined effects of platelet destruction and impaired platelet production often leads to severe thrombocytopenia with skin and mucosal bleeding and risk of serious intracranial and gastrointestinal bleeding complications [2][3][4]. Prevalence of ITP is estimated at 9.5 per 100,000 adults, and incidence rates have been reported at 3.3 adults per 100,000 years [5].…”

“…Adult ITP is a heterogeneous disease that can persist for years, even with best available care, and treatments are infrequently curative [4]. Despite availability of several treatments with differing mechanisms of action, chronicity of disease continues to be a problem.…”

Use of intrauterine balloon tamponade in successful conservative management of life threatening menorrhagia in a patient with idiopathic thrombocytopenic purpura and severe anemia

“…Corticosteroids and IVIg are the first line therapy for ITP. 19 In our case, platelet transfusion was given in view of active bleeding. Also the ITP responded well to steroids and IV Immunoglobulins.…”