Immune thrombocytopenia (ITP) in children: Initial management
- James B Bussel, MD
James B Bussel, MD
- Professor of Pediatrics, Pediatric Hematology
- Weill Medical College of Cornell University
Immune thrombocytopenia (ITP) of childhood is characterized by isolated thrombocytopenia (platelet count <100,000/microL with normal white blood cell count and hemoglobin). The cause of ITP remains unknown in most cases, although it can be triggered by a viral infection or other immune trigger [1-3]. ITP was previously known as idiopathic thrombocytopenic purpura or immune thrombocytopenic purpura. The current term Immune ThrombocytoPenia preserves the widely-recognized acronym "ITP," and acknowledges the immune-mediated mechanism of the disorder, while allowing that patients may have little or no signs of purpura or bleeding .
The treatment and prognosis of newly diagnosed and persistent ITP in children will be reviewed here. The management of chronic ITP, and the clinical manifestations and diagnosis of ITP are discussed separately. (See "Immune thrombocytopenia (ITP) in children: Management of chronic disease" and "Immune thrombocytopenia (ITP) in children: Clinical features and diagnosis".)
The following terms are used in this topic:
●Primary ITP – ITP in the absence of other causes or disorders that may be associated with the thrombocytopenia is known as primary ITP, and is the main focus of this topic review.
●Secondary ITP – Secondary ITP refers to immune-mediated thrombocytopenia with an underlying cause, including drug-induced, or associated with systemic illness (eg, systemic lupus erythematosus, HIV). Secondary causes of immune-mediated thrombocytopenia are reviewed separately. (See "Immune thrombocytopenia (ITP) in children: Clinical features and diagnosis", section on 'Differential diagnosis' and "Causes of thrombocytopenia in children".)
- 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.
- Provan D, Stasi R, Newland AC, et al. International consensus report on the investigation and management of primary immune thrombocytopenia. Blood 2010; 115:168.
- D'Orazio JA, Neely J, Farhoudi N. ITP in children: pathophysiology and current treatment approaches. J Pediatr Hematol Oncol 2013; 35:1.
- Vesely S, Buchanan GR, Cohen A, et al. Self-reported diagnostic and management strategies in childhood idiopathic thrombocytopenic purpura: results of a survey of practicing pediatric hematology/oncology specialists. J Pediatr Hematol Oncol 2000; 22:55.
- Tarantino MD, Buchanan GR. The pros and cons of drug therapy for immune thrombocytopenic purpura in children. Hematol Oncol Clin North Am 2004; 18:1301.
- 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.
- Boruchov DM, Gururangan S, Driscoll MC, Bussel JB. Multiagent induction and maintenance therapy for patients with refractory immune thrombocytopenic purpura (ITP). Blood 2007; 110:3526.
- 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.
- Tarantino MD, Young G, Bertolone SJ, et al. Single dose of anti-D immune globulin at 75 microg/kg is as effective as intravenous immune globulin at rapidly raising the platelet count in newly diagnosed immune thrombocytopenic purpura in children. J Pediatr 2006; 148:489.
- Klaassen RJ, Mathias SD, Buchanan G, et al. Pilot study of the effect of romiplostim on child health-related quality of life (HRQoL) and parental burden in immune thrombocytopenia (ITP). Pediatr Blood Cancer 2012; 58:395.
- Kühne T, Buchanan GR, Zimmerman S, et al. A prospective comparative study of 2540 infants and children with newly diagnosed idiopathic thrombocytopenic purpura (ITP) from the Intercontinental Childhood ITP Study Group. J Pediatr 2003; 143:605.
- Rosthøj S, Hedlund-Treutiger I, Rajantie J, et al. Duration and morbidity of newly diagnosed idiopathic thrombocytopenic purpura in children: A prospective Nordic study of an unselected cohort. J Pediatr 2003; 143:302.
- Glanz J, France E, Xu S, et al. A population-based, multisite cohort study of the predictors of chronic idiopathic thrombocytopenic purpura in children. Pediatrics 2008; 121:e506.
- Revel-Vilk S, Yacobovich J, Frank S, et al. Age and duration of bleeding symptoms at diagnosis best predict resolution of childhood immune thrombocytopenia at 3, 6, and 12 months. J Pediatr 2013; 163:1335.
- Sadowitz D, Souid AK, Terndrup TE. Idiopathic thrombocytopenic purpura in children: recognition and management. Pediatr Emerg Care 1996; 12:222.
- Beck CE, Nathan PC, Parkin PC, et al. Corticosteroids versus intravenous immune globulin for the treatment of acute immune thrombocytopenic purpura in children: a systematic review and meta-analysis of randomized controlled trials. J Pediatr 2005; 147:521.
- Kühne T, Imbach P, Bolton-Maggs PH, et al. Newly diagnosed idiopathic thrombocytopenic purpura in childhood: an observational study. Lancet 2001; 358:2122.
- Blanchette VS, Luke B, Andrew M, et al. A prospective, randomized trial of high-dose intravenous immune globulin G therapy, oral prednisone therapy, and no therapy in childhood acute immune thrombocytopenic purpura. J Pediatr 1993; 123:989.
- Lilleyman JS. Management of childhood idiopathic thrombocytopenic purpura. Br J Haematol 1999; 105:871.
- McWilliams NB, Maurer HM. Acute idiopathic thrombocytopenic purpura in children. Am J Hematol 1979; 7:87.
- Sartorius JA. Steroid treatment of idiopathic thrombocytopenic purpura in children. Preliminary results of a randomized cooperative study. Am J Pediatr Hematol Oncol 1984; 6:165.
- Buchanan GR, Holtkamp CA. Prednisone therapy for children with newly diagnosed idiopathic thrombocytopenic purpura. A randomized clinical trial. Am J Pediatr Hematol Oncol 1984; 6:355.
- Imbach P, Wagner HP, Berchtold W, et al. Intravenous immunoglobulin versus oral corticosteroids in acute immune thrombocytopenic purpura in childhood. Lancet 1985; 2:464.
- Mazzucconi MG, Francesconi M, Fidani P, et al. Treatment of idiopathic thrombocytopenic purpura (ITP): results of a multicentric protocol. Haematologica 1985; 70:329.
- Bellucci S, Charpak Y, Chastang C, Tobelem G. Low doses v conventional doses of corticoids in immune thrombocytopenic purpura (ITP): results of a randomized clinical trial in 160 children, 223 adults. Blood 1988; 71:1165.
- Khalifa AS, Tolba KA, el-Alfy MS, et al. Idiopathic thrombocytopenic purpura in Egyptian children. Acta Haematol 1993; 90:125.
- Ozsoylu S, Sayli TR, Oztürk G. Oral megadose methylprednisolone versus intravenous immunoglobulin for acute childhood idiopathic thrombocytopenic purpura. Pediatr Hematol Oncol 1993; 10:317.
- 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.
- Albayrak D, Işlek I, Kalaycí AG, Gürses N. Acute immune thrombocytopenic purpura: a comparative study of very high oral doses of methylprednisolone and intravenously administered immune globulin. J Pediatr 1994; 125:1004.
- Kumar M, Vik TA, Johnson CS, et al. Treatment, outcome, and cost of care in children with idiopathic thrombocytopenic purpura. Am J Hematol 2005; 78:181.
- Imbach P, Barandun S, d'Apuzzo V, et al. High-dose intravenous gammaglobulin for idiopathic thrombocytopenic purpura in childhood. Lancet 1981; 1:1228.
- Tarantino MD, Madden RM, Fennewald DL, et al. Treatment of childhood acute immune thrombocytopenic purpura with anti-D immune globulin or pooled immune globulin. J Pediatr 1999; 134:21.
- Bussel JB, Goldman A, Imbach P, et al. Treatment of acute idiopathic thrombocytopenia of childhood with intravenous infusions of gammaglobulin. J Pediatr 1985; 106:886.
- Heitink-Polle KM, Uiterwaal CS, Pocelijn L, et al. Treatment with Intravenous Immunoglobulin Does Not Prevent Chronic Immune Thrombocytopenia in Children: Results of a Randomized Controlled Trial. Blood 2016; :866.
- Niebanck AE, Kwiatkowski JL, Raffini LJ. Neutropenia following IVIG therapy in pediatric patients with immune-mediated thrombocytopenia. J Pediatr Hematol Oncol 2005; 27:145.
- Heitink-Pollé KM, Nijsten J, Boonacker CW, et al. Clinical and laboratory predictors of chronic immune thrombocytopenia in children: a systematic review and meta-analysis. Blood 2014; 124:3295.
- Scaradavou A, Woo B, Woloski BM, et al. Intravenous anti-D treatment of immune thrombocytopenic purpura: experience in 272 patients. Blood 1997; 89:2689.
- Despotovic JM, Lambert MP, Herman JH, et al. RhIG for the treatment of immune thrombocytopenia: consensus and controversy (CME). Transfusion 2012; 52:1126.
- Package label for WinRho, Rh(D) immune globulin intravenous. Available at: http://dailymed.nlm.nih.gov/dailymed/lookup.cfm?setid=a9f7654f-cf56-4aec-9e08-7c4b0dc54ff1 (Accessed on April 17, 2013).
- Handin RI, Stossel TP. Effect of corticosteroid therapy on the phagocytosis of antibody-coated platelets by human leukocytes. Blood 1978; 51:771.
- Handin RI, Stossel TP. Phagocytosis of antibody-coated platelets by human granulocytes. N Engl J Med 1974; 290:989.
- Kitchens CS, Pendergast JF. Human thrombocytopenia is associated with structural abnormalities of the endothelium that are ameliorated by glucocorticosteroid administration. Blood 1986; 67:203.
- Donato H, Picón A, Martinez M, et al. Demographic data, natural history, and prognostic factors of idiopathic thrombocytopenic purpura in children: a multicentered study from Argentina. Pediatr Blood Cancer 2009; 52:491.
- Imbach P, Kühne T, Müller D, et al. Childhood ITP: 12 months follow-up data from the prospective registry I of the Intercontinental Childhood ITP Study Group (ICIS). Pediatr Blood Cancer 2006; 46:351.
- Zeller B, Rajantie J, Hedlund-Treutiger I, et al. Childhood idiopathic thrombocytopenic purpura in the Nordic countries: epidemiology and predictors of chronic disease. Acta Paediatr 2005; 94:178.
- Bruin M, Bierings M, Uiterwaal C, et al. Platelet count, previous infection and FCGR2B genotype predict development of chronic disease in newly diagnosed idiopathic thrombocytopenia in childhood: results of a prospective study. Br J Haematol 2004; 127:561.
- Ahmed S, Siddiqui AK, Shahid RK, et al. Prognostic variables in newly diagnosed childhood immune thrombocytopenia. Am J Hematol 2004; 77:358.
- Tamminga R, Berchtold W, Bruin M, et al. Possible lower rate of chronic ITP after IVIG for acute childhood ITP an analysis from registry I of the Intercontinental Cooperative ITP Study Group (ICIS). Br J Haematol 2009; 146:180.
- Treutiger I, Rajantie J, Zeller B, et al. Does treatment of newly diagnosed idiopathic thrombocytopenic purpura reduce morbidity? Arch Dis Child 2007; 92:704.
- General measures
- Treatment approach
- - Overview
- - Life-threatening bleeding
- - Severe non-life-threatening bleeding
- - High bleeding risk
- - Moderate bleeding risk
- - Low bleeding risk
- - Surgery/invasive procedures
- Target platelet count
- Response to treatment
- - Expected response
- - Poor response to initial therapy
- - Relapses
- FIRST-LINE THERAPIES
- Anti-D immune globulin
- SECOND-LINE THERAPIES
- Disease course
- Bleeding complications
- INFORMATION FOR PATIENTS
- SUMMARY AND RECOMMENDATIONS