Subscribe to RSS
DOI: 10.1055/s-2004-815627
Severe Chronic Idiopathic Thrombocytopenic Purpura during Childhood: Definition, Management, and Prognosis
Publication History
Publication Date:
13 January 2004 (online)
ABSTRACT
Chronic idiopathic thrombocytopenic purpura (ITP) can be categorized as mild, moderately severe, or severe. Severe chronic ITP during childhood is a rare disorder characterized by clinically significant mucocutaneous hemorrhage, usually in the setting of marked thrombocytopenia. It can cause substantial morbidity and rarely mortality. Many patients improve with time or even fully recover, but for those whose quality of life is negatively influenced by hemorrhage or side effects of conventional therapy (corticosteroids, intravenous immunoglobulin G, or anti-D), splenectomy is recommended. If splenectomy is unsuccessful or not feasible, other drug treatments are available, but few efficacy data exist, and the toxicity and cost of these treatments can be appreciable. Their use is best avoided outside of clinical trials conducted in specialty centers or in multi-institutional networks.
KEYWORDS
ITP - thrombocytopenia - chronic - severe - splenectomy
REFERENCES
- 1 Kühne T, Imbach P, Bolton-Maggs P HB. et al . Newly diagnosed idiopathic thrombocytopenic purpura in childhood: an observational study. Lancet . 2001; 358 2122-2125
- 2 Robb L G, Tiedeman K. Idiopathic thrombocytopenic purpura: predictors of chronic disease. Arch Dis Child . 1990; 65 502-506
- 3 Lowe E J, Buchanan G R. Idiopathic thrombocytopenic purpura diagnosed during the second decade of life. J Pediatr . 2003; 141 253-258
- 4 Reid M M. Chronic idiopathic thrombocytopenic purpura: incidence, treatment, and outcome. Arch Dis Child . 1995; 72 125-128
- 5 Blanchette V S, Carcao M D. Approach to the investigation and management of immune thrombocytopenic purpura in children. Semin Hematol . 2000; 37 299-314
- 6 Cines D B, Blanchette V S. Immune thrombocytopenic purpura. N Engl J Med . 2002; 346 995-1008
- 7 British Committee for Standards in General Haematology Task Force. Guidelines for the investigation and management of idiopathic thrombocytopenic purpura in adults, children and in pregnancy. Br J Haematol . 2003; 120 574-596
- 8 George J N, Woolf S H, Raskob G E. et al . Idiopathic thrombocytopenic purpura: a practice guideline developed by explicit methods for the American Society of Hematology. Blood . 1996; 88 3-40
- 9 Kühne T, Imbach P. Chronic immune thrombocytopenic purpura in childhood. Semin Thromb Hemost . 1998; 24 549-553
- 10 Lilleyman J S. Management of childhood idiopathic thrombocytopenic purpura. Br J Haematol . 1999; 105 871-875
- 11 Walker R W, Walker W. Idiopathic thrombocytopenia, initial illness and long-term follow-up. Arch Dis Child . 1984; 59 316-322
- 12 Buchanan G R, Adix L. Outcome measures and treatment endpoints other than platelet count in childhood idiopathic thrombocytopenic purpura. Semin Thromb Hemost . 2001; 27 277-285
- 13 Lilleyman J S. Definitions and dogma in childhood idiopathic thrombocytopenic purpura. Pediatr Hematol Oncol . 1993; 10 11-14
- 14 Sutor A H, Grohmann A, Kaufmehl K, Wündisch T. Problems with platelet counting in thrombocytopenia. A rapid manual method to measure low platelet counts. Semin Thromb Hemost . 2001; 27 237-243
- 15 Wong MS-C, Chan GC-F, Ha S Y, Lau Y-L. Clinical characteristics of chronic idiopathic thrombocytopenia in Chinese children. J Pediatr Hematol Oncol . 2002; 24 648-652
- 16 Buchanan G R, Adix L. Grading of hemorrhage in children with idiopathic thrombocytopenic purpura. J Pediatr . 2002; 141 683-688
- 17 Medeiros D, Buchanan G R. Current controversies in the management of idiopathic thrombocytopenic purpura during childhood. Pediatr Clin North Am . 1996; 43 757-772
- 18 Bolton-Maggs P HB, Moon I. Assessment of UK practice for management of acute childhood idiopathic thrombocytopenic purpura against published guidelines. Lancet . 1997; 350 620-623
- 19 George J N. Initial management of immune thrombocytopenic purpura in children: is supportive counseling without therapeutic intervention sufficient?. J Pediatr . 2000; 137 598-600
- 20 Carcao M D, Blanchette V S, Stephens D. et al . Assessment of thrombocytopenic disorders using the Platelet Function Analyzer (PFA-100;rM). Br J Haematol . 2002; 117 961-964
- 21 Crosby W H. Wet purpura, dry purpura. JAMA . 1975; 232 744-745
- 22 Godeau B, Chevret S, Varet B, for the French AITP Study Group. et al . Intravenous immunoglobulin or high-dose methylprednisolone, with or without oral prednisone, for adults with untreated severe autoimmune thrombocytopenic purpura: a randomized, multicentre trial. Lancet . 2002; 359 23-29
- 23 Lacey J V, Penner J A. Management of idiopathic thrombocytopenic purpura in the adult. Semin Thromb Hemost . 1977; 3 160-174
- 24 Barnard D, Woloski M, Feeny D, for the Canadian Children's Platelet Study Group. et al . Development of disease-specific health-related quality-of-life instruments for children with immune thrombocytopenic purpura and their parents. J Pediatr Hematol Oncol . 2003; 25 56-62
- 25 Bolton-Maggs P HB, Dickerhoff R, Vora A J. The nontreatment of childhood ITP (or “the art of medicine consists of amusing the patient until nature cures the disease”). Semin Thromb Hemost . 2001; 27 269-275
- 26 Dickerhoff R, von Ruecker A. The clinical course of immune thrombocytopenic purpura in children who did not receive intravenous immunoglobulins or sustained prednisone treatment. J Pediatr . 2000; 137 629-632
- 27 Buchanan G R. ITP: how much treatment is enough?. Contemp Pediatr . 2000; 17 112-121
- 28 Lilleyman J S. Intracranial hemorrhage in idiopathic thrombocytopenic purpura. Arch Dis Child . 1994; 71 251-253
- 29 Miller E, Waight P, Farrington P. et al . Idiopathic thrombocytopenic purpura and MMR vaccine. Arch Dis Child . 2001; 84 227-229
- 30 Vlacha V, Forman E N, Miron D, Peter G. Recurrent thrombocytopenic purpura after repeated measles-mumps-rubella vaccination. Pediatrics . 1996; 97 738-739
- 31 Tarantino M D, Madden R M, Fennewald D L, Patel C C, Bertolone S J. Treatment of childhood acute immune thrombocytopenic purpura with anti-D immune globulin or pooled immune globulin. J Pediatr . 1999; 134 21-26
- 32 Newman G C, Novoa M V, Fodero E M. et al . A dose of 75 μg/kg/d of iv anti-D increases the platelet count more rapidly and for a longer period of time than 50 μg/kg/d in adults with immune thrombocytopenic purpura. Br J Haematol . 2001; 112 1076-1078
- 33 Andrew M, Blanchette V S, Adams M. et al . A multicenter study of the treatment of childhood chronic idiopathic thrombocytopenic purpura with anti-D. J Pediatr . 1992; 120 522-527
- 34 Bussel J B. Splenectomy-sparing strategies for the treatment and long-term maintenance of chronic idiopathic (immune) thrombocytopenic purpura. Semin Hematol . 2000; 37 1-4
- 35 Andersen J C. Response of resistant idiopathic thrombocytopenic purpura to pulsed high-dose dexamethasone therapy. N Engl J Med . 1994; 330 1560-1564
- 36 Kühne T, Freedman J, Semple J W. et al . Platelet and immune responses to oral cyclic dexamethasone therapy in childhood chronic immune thrombocytopenic purpura. J Pediatr . 1997; 130 17-24
- 37 Chen J-S, Wu J-M, Chen Y-J, Yeh T-F. Pulsed high-dose dexamethasone therapy in children with chronic idiopathic thrombocytopenic purpura. J Pediatr Hematol Oncol . 1997; 19 526-529
- 38 Mantadakis E, Buchanan G R. Elective splenectomy in children with idiopathic thrombocytopenic purpura. J Pediatr Hematol Oncol . 2000; 22 148-153
- 39 Szold A, Schwartz J, Abu-Abeid S, Bulvik S, Eldor A. Laparoscopic splenectomies for idiopathic thrombocytopenic purpura: experience of sixty cases. Am J Hematol . 2000; 63 7-10
- 40 Farah R A, Rogers Z R, Thompson W R. et al . Comparison of laparoscopic and open splenectomy in children with hematologic disorders. J Pediatr . 1997; 131 41-46
- 41 Holt D, Brown J, Terrill K. et al . Response to intravenous immunoglobulin predicts splenectomy response in children with immune thrombocytopenic purpura. Pediatrics . 2003; 111 87-90
- 42 Law C, Marcaccio M, Tam P, Heddle N, Kelton J G. High-dose intravenous immune globulin and the response to splenectomy in patients with idiopathic thrombocytopenic purpura. N Engl J Med . 1997; 336 1494-1498
- 43 Najean Y, Rain J-D, Billotey C. The site of destruction of autologous 111In-labelled platelets and the efficiency of splenectomy in children and adults with idiopathic thrombocytopenic purpura: a study of 578 patients with 268 splenectomies. Br J Haematol . 1997; 97 547-550
- 44 Hemmila M R, Foley D S, Castle V P, Hirschl R B. The response to splenectomy in pediatric patients with idiopathic thrombocytopenic purpura who fail high-dose intravenous immune globulin. J Pediatr Surg . 2000; 35 967-972
- 45 Williams J A, Boxer L A. Combination therapy for refractory idiopathic thrombocytopenic purpura in adolescents. J Pediatr Hematol Oncol . 2003; 25 232-235
- 46 Quiquandon I, Fenaux P, Caulier M T. et al . Re-evaluation of the role of azathioprine in the treatment of adult chronic idiopathic thrombocytopenic purpura: a report on 53 cases. Br J Haematol . 1990; 74 223-228
- 47 Donato H, Kohan R, Picón A. et al . α-Interferon therapy induces improvement of platelet counts in children with chronic idiopathic thrombocytopenic purpura. J Pediatr Hematol Oncol . 2001; 233 598-603
- 48 Hrstková H, Bajer M, Michálek, J. Recombinant human interferon α-2a therapy in children with chronic immune thrombocytopenic purpura. J Pediatr Hematol Oncol . 2002; 24 299-303
- 49 Stasi R, Pagano A, Stipa E L, Amadori S. Rituximab chimeric anti-CD20 monoclonal antibody treatment for adults with chronic idiopathic thrombocytopenic purpura. Blood . 2001; 98 952-957
- 50 Zecca M, Nobili B, Ramenghi U. et al . Rituximab for the treatment of refractory autoimmune hemolytic anemia in children. Blood . 2003; 101 3857-3861