hematology and oncology
Immune Thrombocytopenia (ITP)
Treatment of ITP
Oral corticosteroids IV immune globulin (IVIG) IV anti-D immune globulin Sometimes, splenectomy Thrombopoietin receptor agonists (TPO-RA) Rituximab Fostamatinib Other immunosuppressants For severe bleeding, IVIG, IV anti-D immune globulin, IV corticosteroids, and/or platelet transfusions 2019 guidelines are now available Treatment references . Asymptomatic patients with a platelet count > 30,000/mcL (> 30 × 10 Adults with newly diagnosed ITP with bleeding and a platelet count < 30,000/mcL (< 30 × 109/L) are usually given an oral corticosteroid (eg,prednisone1 mg/kg orally once a day) initially. An alternative, probably equally effective, corticosteroid regimen isdexamethasone40 mg orally once a day for 4 days. In most patients, the platelet count increases within 2 to 5 days. However, in some patients, a response may take 2 to 4 weeks. When the corticosteroid is tapered after response, most adult patients relapse. Repeated corticosteroid treatments may be effective but increase the risk of adverse effects. Corticosteroids should not usually be continued beyond the first several months; other drugs may be tried in an attempt to avoid splenectomy. If medical therapy is effective, most guidelines recommend continuing it for at least one year before considering splenectomy. ) can also be used before invasive procedures but should not be used for childbirth. IVIG and IV anti-D immune globulin are also useful for Life-threatening bleeding in ITP (ITP) but are rarely used for chronic treatment because their response may last for only a few days to weeks. /L]) in whom bleeding risk cannot be controlled with medical therapy or those whose disease persists after 12 months. If thrombocytopenia can be controlled with second-line medical therapies, splenectomy is often not necessary ( Treatment references ). Splenectomy results in an increased risk of thrombosis and infection (particularly with encapsulated bacteria such as pneumococcus); patients require vaccination against Second-line medical therapiesare available for patients with immune thrombocytopenia Who are seeking to defer splenectomy in hope of a spontaneous remission Who are not candidates for or refuse splenectomy In whom splenectomy has not been effective Such patients usually have platelet counts<10,000 to 20,000/mcL (< 10 to 20 × 109/L), and thus are at risk for bleeding. Second-line medical therapies include thrombopoietin receptor agonists (TPO-RAs),rituximab,fostamatinib, or other immunosuppressive drugs. Thrombopoietin receptor agonists, such asromiplostim1 to 10 mcg/kg subcutaneously once a week,eltrombopag25 to 75 mg orally once a day, andavatrombopag20 mg orally once a dayy have response rates > 85%. TPO-RAs often need to be administered continuously to maintain the platelet count>50,000/mcL (> 50 × 109/L), but data suggest that one third of adults will undergo a treatment-free remission after 1 year and > 50% after 2 years. IV once a week for 4 weeks) has a response rate of 57%, but only 21% of adult patients remain in remission after 5 years ( Treatment references ). Alternate dosing schemes are also effective (eg, doses of 1000 mg IV given 2 weeks apart). /L— Treatment references ). More intensive immunosuppression may be required with drugs such ascyclophosphamide,cyclosporine,mycophenolate, andazathioprinein patients unresponsive to other drugs who have severe, symptomatic thrombocytopenia. In children or adults with immune thrombocytopenia and life-threatening bleeding, rapid phagocytic blockade is attempted by giving IVIG 1 g/kg once a day for 1 to 2 days or, in Rh-positive patients, a single dose of IV anti-D immune globulin 75 mcg/kg. IV anti-D immunoglobulin is only effective in patients who have not had a splenectomy and may be associated with severe complications such as severe hemolysis and Disseminated Intravascular Coagulation (DIC) . This treatment usually causes the platelet count to rise within 2 to 4 days, but the count remains high for only 2 to 4 weeks. High-dosemethylprednisolone(1 g IV once a day for 3 days) is easier to administer than IVIG or IV anti-D immune globulin but may not be as effective. Patients with ITP and life-threatening bleeding are also given platelet transfusions. Platelet transfusions are not used prophylactically. Vincristine(1.4 mg/m2; maximum dose of 2 mg) has also been used in emergency situations but may produce neuropathy with repeated administration. Early use of TPO-RA may also be effective in combination with the therapies above ( Treatment references ). Treatment of children with immune thrombocytopenia is usually supportive because most children spontaneously recover. Even after months or years of thrombocytopenia, most children have spontaneous remissions. If mucosal bleeding occurs, corticosteroids or IVIG may be given. Corticosteroid and IVIG use is controversial because the increased platelet count may not improve clinical outcome. Splenectomy is rarely done in children. However, if thrombocytopenia is severe and symptomatic for>6 months, then thrombopoietin receptor agonists (eg,romiplostim,eltrombopag) should be considered. 1.Neunert C, Terrell DR, Arnold DM, et al: American Society of Hematology 2019 guidelines for immune thrombocytopenia.Blood Adv3(23):3829–3866, 2019. doi: 10.1182/bloodadvances.2019000966 2.Provan D, Arnold DM, Bussel JB, et al: Updated international consensus report on the investigation and management of primary immune thrombocytopenia.Blood Adv3(22):3780–3817, 2019. doi: 10.1182/bloodadvances.2019000812 3.Patel VL, Mahevas M, Lee SY, et al: Outcomes 5 years after response torituximabtherapy in children and adults with immune thrombocytopenia.Blood119:5989–5995, 2012. doi: 10.1182/blood-2011-11-393975 4.Bussel J, Arnold DM, Grossbard E, et al:Fostamatinibfor the treatment of adult persistent and chronic immune thrombocytopenia: Results of two phase 3, randomized, placebo‐controlled trials.Am J Hematol93: 921–930, 2018. doi: 10.1002/ajh.25125 5.Kuter DJ, Tarantino MD, Lawrence T: Clinical overview and practical considerations for optimizingromiplostimtherapy in patients with immune thrombocytopenia.Blood Rev49:100811, 2021. doi: 10.1016/j.blre.2021.100811 6.Lozano ML, Godeau B, Grainger J, et al:Romiplostimin adults with newly diagnosed or persistent immune thrombocytopenia.Expert Rev Hematol13(12):1319–1332, 2020. doi: 10.1080/17474086.2020.1850253 