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Identifying and Treating Refractory ITP: Difficulty in Diagnosis and Role of Combination Treatment.

07:00 EST 22nd November 2019 | BioPortfolio

Summary of "Identifying and Treating Refractory ITP: Difficulty in Diagnosis and Role of Combination Treatment."

Immune Thrombocytopenia (ITP) is the commonest acquired thrombocytopenia after chemotherapy-induced thrombocytopenia. Existing guidelines describe the management and treatment of most patients who overall do well, even if they present with chronic disease, and are usually not at high risk of bleeding. However, a small percentage of patients is refractory and difficult to manage. Patients classified as refractory either have a diagnosis that is not really ITP or have disease that is difficult to manage. ITP is a diagnosis of exclusion; no specific tests exist to confirm the diagnosis. Response to treatment is the only affirmative confirmation of diagnosis. However, refractory patients do not respond to front-line or other treatments, and thus, no confirmation of diagnosis exists. This review carefully evaluates the diagnostic considerations in patients with refractory ITP. The second section describes combination treatment for refractory cases of ITP. The reported combinations are divided into the era before thrombopoietin(TPO) and rituximab and the current era. Current therapy appears to have increased effectiveness. However, the definition of refractory, if it includes insufficient response to TPO agents, describes a group with more severe and difficult to treat disease. The biology of refractory ITP is largely unexplored and includes oligoclonality, lymphocyte pumps, and other possibilities. Newer treatments, especially rapamycin, fostamatinib, FcRn and BTK inhibitors, may be useful components of future therapy given their mechanisms of action but TPO agents, notwithstanding failure as monotherapy, appear to be critical components. In summary, refractory ITP is a complicated entity in which precise, specific diagnosis is as important as development of effective combination treatments.

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Name: Blood
ISSN: 1528-0020
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