Abstract
Immune thrombocytopenic purpura (ITP) is an autoimmune condition that affects nearly 1:10,000 people in the world. It is traditionally defined by a platelet count of less than 100 x 109L, but treatment typically depends on symptomology rather than on the platelet count itself. For primary idiopathic ITP, corticosteroids have been the standard first-line of treatment for symptomatic patients, with the addition of intravenous immune globulin (IVIG) or Rho(D) immune globulin (anti-RhD) for steroid-resistant cases. In cases of refractory or non-responsive ITP, second-line therapy includes splenectomy or rituximab, a monoclonal antibody against the CD20 antigen (anti-CD20). In patients who continue to have severe thrombocytopenia and symptomatic bleeding despite first- and second-line treatments, the diagnosis of “chronic refractory ITP” is appropriate, and third-line treatments are evaluated. This manuscript describes the efficacy of different treatment options for primary ITP and introduces the reader to various third-line options that are emerging as a means of treating chronic refractory ITP.
Highlights
Idiopathic thrombocytopenia or immune thrombocytopenia (ITP) is a hematological condition which is characterized by a low platelet count of less than 100 x 109L
In children, who account for half of the cases of ITP seen per year, two-thirds of cases are preceded by a febrile infectious illness [3,4]
ITP has been linked to chronic lymphocytic leukemia (CLL) (1-5% of CLL patients), as well as many other autoimmune and rheumatologic conditions [3]
Summary
Idiopathic thrombocytopenia or immune thrombocytopenia (ITP) is a hematological condition which is characterized by a low platelet count of less than 100 x 109L. A prospective study of 47 patients with chronic ITP with refractory disease after splenectomy demonstrates a 22% response rate after treatment with danazol [26]. In a long-term study of 80 patients with chronic ITP treated with romiplostim, a platelet response of greater than 50 x 109/L was seen initially in 74% of patients and was maintained two years later in 65% [47]. Ongoing studies including treatment with spleen tyrosine kinase inhibitors (Syk inhibitors) such as fostamatinib, anti-CD40 ligand, antihuman CD16 (FcγRIII) monoclonal antibody (GMA161), daclizumab, alemtuzumab, and avatrombopag are being conducted and will hopefully lead to treatments for chronic refractory ITP with a higher rate of complete and sustained remission and minimal side effects [5,44]. A summary of the ITP treatment options is shown below (Figure 1)
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