Immune thrombocytopenia (ITP) in adults: Initial treatment and prognosis
- James N George, MD
James N George, MD
- Professor of Medicine
- University of Oklahoma Health Sciences Center
- Donald M Arnold, MD, MSc
Donald M Arnold, MD, MSc
- Associate Professor, Department of Medicine
- McMaster University
Immune thrombocytopenia (ITP) is an acquired form of thrombocytopenia due to autoantibody-mediated destruction of platelets. ITP is a diagnosis of exclusion, characterized by isolated thrombocytopenia and the lack of a clinically-apparent condition responsible for the low platelet count; there are no reliable laboratory tests to confirm the diagnosis. (See "Immune thrombocytopenia (ITP) in adults: Clinical manifestations and diagnosis".)
The major management decisions in the initial treatment of ITP are whether the patient needs therapy at all, and if so, which therapy to use. These decisions can be challenging because the goal of treatment is to prevent severe bleeding, but the risk of bleeding can be difficult to estimate for any individual patient. Severe bleeding is rare, and patients may be more likely to develop complications from the toxicities of therapy than they are to have severe bleeding.
The initial treatment and prognosis of ITP in adults is reviewed here. Second- and third-line therapies for adults who require additional treatment, and the management of ITP in children, who have a different clinical course from adults, are discussed separately. (See "Immune thrombocytopenia (ITP) in adults: Second-line and subsequent therapies" and "Immune thrombocytopenia (ITP) in children: Initial management" and "Immune thrombocytopenia (ITP) in children: Management of chronic disease".)
We use the following terms herein; this terminology is consistent with a consensus statement from an international working group on ITP published in 2009 :
●Primary ITP – Primary ITP is acquired thrombocytopenia due to autoimmune platelet destruction, not triggered by an associated condition. (See "Immune thrombocytopenia (ITP) in adults: Clinical manifestations and diagnosis".)
- Rodeghiero F, Stasi R, Gernsheimer T, et al. Standardization of terminology, definitions and outcome criteria in immune thrombocytopenic purpura of adults and children: report from an international working group. Blood 2009; 113:2386.
- Ghanima W, Godeau B, Cines DB, Bussel JB. How I treat immune thrombocytopenia: the choice between splenectomy or a medical therapy as a second-line treatment. Blood 2012; 120:960.
- George JN, Woolf SH, Raskob GE, et al. Idiopathic thrombocytopenic purpura: a practice guideline developed by explicit methods for the American Society of Hematology. Blood 1996; 88:3.
- Toltl LJ, Arnold DM. Pathophysiology and management of chronic immune thrombocytopenia: focusing on what matters. Br J Haematol 2011; 152:52.
- Lacey JV, Penner JA. Management of idiopathic thrombocytopenic purpura in the adult. Semin Thromb Hemost 1977; 3:160.
- Cortelazzo S, Finazzi G, Buelli M, et al. High risk of severe bleeding in aged patients with chronic idiopathic thrombocytopenic purpura. Blood 1991; 77:31.
- Cines DB, Bussel JB. How I treat idiopathic thrombocytopenic purpura (ITP). Blood 2005; 106:2244.
- Guthrie TH Jr, Brannan DP, Prisant LM. Idiopathic thrombocytopenic purpura in the older adult patient. Am J Med Sci 1988; 296:17.
- Stasi R, Stipa E, Masi M, et al. Long-term observation of 208 adults with chronic idiopathic thrombocytopenic purpura. Am J Med 1995; 98:436.
- Portielje JE, Westendorp RG, Kluin-Nelemans HC, Brand A. Morbidity and mortality in adults with idiopathic thrombocytopenic purpura. Blood 2001; 97:2549.
- Neylon AJ, Saunders PW, Howard MR, et al. Clinically significant newly presenting autoimmune thrombocytopenic purpura in adults: a prospective study of a population-based cohort of 245 patients. Br J Haematol 2003; 122:966.
- British Committee for Standards in Haematology General Haematology Task Force. Guidelines for the investigation and management of idiopathic thrombocytopenic purpura in adults, children and in pregnancy. Br J Haematol 2003; 120:574.
- Provan D, Stasi R, Newland AC, et al. International consensus report on the investigation and management of primary immune thrombocytopenia. Blood 2010; 115:168.
- Neunert C, Lim W, Crowther M, et al. The American Society of Hematology 2011 evidence-based practice guideline for immune thrombocytopenia. Blood 2011; 117:4190.
- Kojouri K, Perdue JJ, Medina PJ, George JN. Occult quinine-induced thrombocytopenia. J Okla State Med Assoc 2000; 93:519.
- George JN. Management of patients with refractory immune thrombocytopenic purpura. J Thromb Haemost 2006; 4:1664.
- Neunert CE, Buchanan GR, Imbach P, et al. Severe hemorrhage in children with newly diagnosed immune thrombocytopenic purpura. Blood 2008; 112:4003.
- Psaila B, Petrovic A, Page LK, et al. Intracranial hemorrhage (ICH) in children with immune thrombocytopenia (ITP): study of 40 cases. Blood 2009; 114:4777.
- Goel R, Ness PM, Takemoto CM, et al. Platelet transfusions in platelet consumptive disorders are associated with arterial thrombosis and in-hospital mortality. Blood 2015; 125:1470.
- Neunert C, Noroozi N, Norman G, et al. Severe bleeding events in adults and children with primary immune thrombocytopenia: a systematic review. J Thromb Haemost 2015; 13:457.
- Gardner FH, Helmer RE 3rd. Aminocaproic acid. Use in control of hemorrhage in patients with amegakaryocytic thrombocytopenia. JAMA 1980; 243:35.
- Bartholomew JR, Salgia R, Bell WR. Control of bleeding in patients with immune and nonimmune thrombocytopenia with aminocaproic acid. Arch Intern Med 1989; 149:1959.
- Kalmadi S, Tiu R, Lowe C, et al. Epsilon aminocaproic acid reduces transfusion requirements in patients with thrombocytopenic hemorrhage. Cancer 2006; 107:136.
- Godeau B, Chevret S, Varet B, et al. Intravenous immunoglobulin or high-dose methylprednisolone, with or without oral prednisone, for adults with untreated severe autoimmune thrombocytopenic purpura: a randomised, multicentre trial. Lancet 2002; 359:23.
- Cooper N, Woloski BM, Fodero EM, et al. Does treatment with intermittent infusions of intravenous anti-D allow a proportion of adults with recently diagnosed immune thrombocytopenic purpura to avoid splenectomy? Blood 2002; 99:1922.
- Mizutani H, Furubayashi T, Imai Y, et al. Mechanisms of corticosteroid action in immune thrombocytopenic purpura (ITP): experimental studies using ITP-prone mice, (NZW x BXSB) F1. Blood 1992; 79:942.
- Wei Y, Ji XB, Wang YW, et al. High-dose dexamethasone vs prednisone for treatment of adult immune thrombocytopenia: a prospective multicenter randomized trial. Blood 2016; 127:296.
- Mazzucconi MG, Fazi P, Bernasconi S, et al. Therapy with high-dose dexamethasone (HD-DXM) in previously untreated patients affected by idiopathic thrombocytopenic purpura: a GIMEMA experience. Blood 2007; 109:1401.
- Cheng Y, Wong RS, Soo YO, et al. Initial treatment of immune thrombocytopenic purpura with high-dose dexamethasone. N Engl J Med 2003; 349:831.
- Godeau B, Caulier MT, Decuypere L, et al. Intravenous immunoglobulin for adults with autoimmune thrombocytopenic purpura: results of a randomized trial comparing 0.5 and 1 g/kg b.w. Br J Haematol 1999; 107:716.
- Ware RE, Zimmerman SA. Anti-D: mechanisms of action. Semin Hematol 1998; 35:14.
- Bussel JB, Graziano JN, Kimberly RP, et al. Intravenous anti-D treatment of immune thrombocytopenic purpura: analysis of efficacy, toxicity, and mechanism of effect. Blood 1991; 77:1884.
- Newman GC, Novoa MV, Fodero EM, et al. A dose of 75 microg/kg/d of i.v. anti-D increases the platelet count more rapidly and for a longer period of time than 50 microg/kg/d in adults with immune thrombocytopenic purpura. Br J Haematol 2001; 112:1076.
- George JN, Raskob GE, Vesely SK, et al. Initial management of immune thrombocytopenic purpura in adults: a randomized controlled trial comparing intermittent anti-D with routine care. Am J Hematol 2003; 74:161.
- Blanchette V, Imbach P, Andrew M, et al. Randomised trial of intravenous immunoglobulin G, intravenous anti-D, and oral prednisone in childhood acute immune thrombocytopenic purpura. Lancet 1994; 344:703.
- Barbolla L, Nieto S, Llamas P, et al. Severe immune haemolytic anaemia caused by intravenous immunoglobulin anti-D in the treatment of autoimmune thrombocytopenia. Vox Sang 1993; 64:184.
- Hong F, Ruiz R, Price H, et al. Safety profile of WinRho anti-D. Semin Hematol 1998; 35:9.
- Gaines AR. Acute onset hemoglobinemia and/or hemoglobinuria and sequelae following Rh(o)(D) immune globulin intravenous administration in immune thrombocytopenic purpura patients. Blood 2000; 95:2523.
- Tarantino MD, Bussel JB, Cines DB, et al. A closer look at intravascular hemolysis (IVH) following intravenous anti-D for immune thrombocytopenic purpura (ITP). Blood 2007; 109:5527; author reply 5528.
- Gaines AR. Response: Further examination of intravascular hemolysis (IVH) following intravenous anti-D for immune thrombocytopenic purpura (ITP). Blood. 2007; 109:5528.
- Garratty G. What is the mechanism for acute hemolysis occurring in some patients after intravenous anti-D therapy for immune thrombocytopenic purpura? Transfusion 2009; 49:1026.
- http://www.fda.gov/Safety/MedWatch/SafetyInformation/SafetyAlertsforHumanMedicalProducts/ucm203739.htm (Accessed on March 10, 2010).
- Robak T, Windyga J, Trelinski J, et al. Rozrolimupab, a mixture of 25 recombinant human monoclonal RhD antibodies, in the treatment of primary immune thrombocytopenia. Blood 2012; 120:3670.
- Tarantino MD, Fogarty PF, Shah P, Brainsky A. Dental procedures in 24 patients with chronic immune thrombocytopenia in prospective clinical studies of eltrombopag. Platelets 2015; 26:93.
- http://clinicaltrials.gov/show/NCT01621204 (Accessed on May 05, 2014).
- Stasi R, Sarpatwari A, Segal JB, et al. Effects of eradication of Helicobacter pylori infection in patients with immune thrombocytopenic purpura: a systematic review. Blood 2009; 113:1231.
- Arnold DM, Stasi R. Does Helicobacter pylori eradication therapy result in a platelet count improvement in adults with immune thrombocytopenic purpura regardless of H pylori infection? ASH evidence-based review 2008. Hematology Am Soc Hematol Educ Program 2008; :31.
- Rostami N, Keshtkar-Jahromi M, Rahnavardi M, et al. Effect of eradication of Helicobacter pylori on platelet recovery in patients with chronic idiopathic thrombocytopenic purpura: a controlled trial. Am J Hematol 2008; 83:376.
- Suzuki T, Matsushima M, Masui A, et al. Effect of Helicobacter pylori eradication in patients with chronic idiopathic thrombocytopenic purpura-a randomized controlled trial. Am J Gastroenterol 2005; 100:1265.
- McMillan R, Durette C. Long-term outcomes in adults with chronic ITP after splenectomy failure. Blood 2004; 104:956.
- Bourgeois E, Caulier MT, Delarozee C, et al. Long-term follow-up of chronic autoimmune thrombocytopenic purpura refractory to splenectomy: a prospective analysis. Br J Haematol 2003; 120:1079.
- Vianelli N, Valdrè L, Fiacchini M, et al. Long-term follow-up of idiopathic thrombocytopenic purpura in 310 patients. Haematologica 2001; 86:504.
- OVERVIEW OF OUR APPROACH
- Indications for treatment
- Other therapeutic considerations
- Hematologist referral
- TREATMENT OF BLEEDING
- FIRST-LINE THERAPIES
- Choice of initial first-line therapy
- SECOND- AND THIRD-LINE THERAPIES
- SPECIAL SCENARIOS
- Surgery/invasive procedures
- Myelosuppressive chemotherapy
- ITP during pregnancy
- Secondary ITP
- Helicobacter pylori infection
- High risk physical activities
- Disease course
- INFORMATION FOR PATIENTS
- SUMMARY AND RECOMMENDATIONS