Haema 2010; 1(2): 172-182
by Maria Papaioannou, Dimitrios Pilalas
Department of Haematology, First Department of Internal Medicine, AHEPA University Hospital Aristotle University of Thessaloniki School of Medicine
Abstract
Despite the introduction of new classes of therapeutic agents and a greater understanding of the disease pathophysiology, treatment of adult patients with primary immune thrombocytopenia (ITP) remains a real challenge. The goal of treatment is to raise the platelet count to high enough levels to prevent bleeding using the least toxic therapy, recognizing the generally benign nature of the illness. Corticosteroids, intravenous immune globulin or anti-D globulin remain mainstays of first line treatment. However, only 10-30% of the patients will have a sustained response to the initial management. Second line treatment is required for the majority of the patients. Splenectomy is an effective option for many patients; however, it is frequently avoided by physicians due to concerns about safety risks and is often declined by patients reluctant to undergo surgery. Immune suppressant medications and cytotoxic drugs continue to be used in patients with severe and chronic refractory ITP with some success; however, estimates of the effect of these agents are limited by the lack of randomized trials using clinical end points. Rituximab, an anti-CD20 antibody, although effective in many patients, randomized controlled trials of rituximab for ITP are still needed. Recently two novel agonists of TPO receptors have been made available: romiplostim and eltrombopag. In several phase III studies, both agents demonstrated increase of platelet counts in about 80% of chronic ITP patients within 2 to 3 weeks. These agents substantially broaden the therapeutic options for patients with chronic ITP, although long-term results and safety are still pending. In the era of new treatment options for ITP an International Consensus Report was published very recently with the purpose to comment upon new data and provide recommendations relating to diagnosis and treatment of ITP. The take home message of the report is: Final judgment regarding care of individual patients should lie with the responsible healthcare professional and be based on careful investigation of individual circumstances.deacetylase inhibitors are currently under investigation.