[Guideline for diagnosis and treatment of immune thrombocytopenia].
Trombocitopenia inmune. Guía de diagnóstico y tratamiento.
idiopathic thrombocytopenic purpura
immune thrombocytopenia
pediatrics
treatment
Journal
Archivos argentinos de pediatria
ISSN: 1668-3501
Titre abrégé: Arch Argent Pediatr
Pays: Argentina
ID NLM: 0372460
Informations de publication
Date de publication:
01 12 2019
01 12 2019
Historique:
received:
20
05
2019
accepted:
05
06
2019
entrez:
24
11
2019
pubmed:
24
11
2019
medline:
28
8
2020
Statut:
ppublish
Résumé
Management, outcome, diagnosis, prognosis and treatment of immune thrombocytopenia are controversial. Several guidelines stating different experts' opinions have been published; however, no worldwide consensus regarding the management of the disease has still been reached. This guideline defines diagnostic criteria, states initial laboratory tests, establishes differential diagnosis, develops topics concerning outcome and prognosis, and enumerates available treatments for acute and chronic disease, as well as for management of life-threatening bleeding. El manejo de la trombocitopenia inmune es motivo de discusión en lo concerniente a evolución, diagnóstico, pronóstico y tratamiento. Se han publicado varias guías que expresan distintas opiniones de expertos, pero no existe aún consenso mundial sobre cuál es el manejo más adecuado de la enfermedad. Esta guía establece los criterios para definir el diagnóstico; detalla el plan de estudios de laboratorio por realizar inicialmente; plantea los distintos diagnósticos diferenciales; desarrolla aspectos relativos a evolución y pronóstico, y enumera los tratamientos disponibles para las formas agudas y las crónicas, así como para el manejo de las emergencias y en algunas situaciones especiales.
Autres résumés
Type: plain-language-summary
(spa)
El manejo de la trombocitopenia inmune es motivo de discusión en lo concerniente a evolución, diagnóstico, pronóstico y tratamiento. Se han publicado varias guías que expresan distintas opiniones de expertos, pero no existe aún consenso mundial sobre cuál es el manejo más adecuado de la enfermedad. Esta guía establece los criterios para definir el diagnóstico; detalla el plan de estudios de laboratorio por realizar inicialmente; plantea los distintos diagnósticos diferenciales; desarrolla aspectos relativos a evolución y pronóstico, y enumera los tratamientos disponibles para las formas agudas y las crónicas, así como para el manejo de las emergencias y en algunas situaciones especiales.
Identifiants
pubmed: 31758894
doi: 10.5546/aap.2019.S243
doi:
Types de publication
Journal Article
Practice Guideline
Langues
spa
Sous-ensembles de citation
IM
Pagination
S243-S254Informations de copyright
Sociedad Argentina de Pediatría.
Déclaration de conflit d'intérêts
The authors report no conflicts of interest in this work.
Références
Rodeghiero F, Stasi R, Gernsheimer T, Michel M, 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:(11)2386-93.
Cines DB, Bussel JB, Liebman HA, Luning Prak ET. The ITP syndrome: pathogenic and clinical diversity. Blood. 2009; 113(26):6511-21.
Stasi R. Immune thrombocytopenia: pathophysiologic and clinical update. Semin Thromb Hemost. 2012; 38(5):454-62.
Cines DB, Cuker A, Semple JW. Pathogenesis of immune thrombocytopenia. Presse Med. 2014; 43(4 Pt 2):e49-59.
Audia S, Mahévas M, Samson M, Godeau B, et al. Pathogenesis of immune thrombocytopenia. Autoimmun Rev. 2017; 16(6):620-32.
Emmons RV, Reid DM, Cohen RL, Meng G, et al. Human thrombopoietin levels are high when thrombocytopenia is due to megakaryocyte deficiency and low when due to increased platelet destruction. Blood. 1996; 87(10):4068-71.
Kosugi S, Kurata Y, Tomiyama Y, Tahara T, et al. Circulating thrombopoietin level in chronic immune thrombocytopenic purpura. Br J Haematol. 1996; 93(3):704-6.
Imbach P, Lazarus AH, Kühne T. Intravenous immunoglobulins induce potentially synergistic immunomodulations in autoimmune disorders. Vox Sang. 2010; 98(3 Pt 2):385-94.
Kappers-Klunne MC, De Haan M, Struijk PC, Van Vliet HH. Serum thrombopoietin levels in relation to disease status in patients with immune thrombocytopenic purpura. Br J Haematol. 2001; 115(4):1004-6.
Hoemberg M, Stahl D, Schlenke P, Sibrowski W, et al. The isotype of autoantibodies influences the phagocytosis of antibody-coated platelets in autoimmune thrombocytopenic purpura. Scand J Immunol. 2011; 74(5):489-95.
George JN, Woolf SH, Raskob GE, Wasser JS, et al. Idiopathic thrombocytopenic purpura: a practice guideline developed by explicit methods for the American Society of Hematology. Blood. 1996; 88(1):3-40.
De Mattia D, Del Principe D, Del Vecchio GC, Jankovic M, et al. Acute childhood idiopathic thrombocytopenic purpura: AIEOP consensus guidelines for diagnosis and treatment. Haematologica. 2000; 85(4):420-4.
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(4):574-96.
Shirahata A, Ishii E, Eguchi H, Okawa H, et al. Consensus guideline for diagnosis and treatment of childhood idiopathic thrombocytopenic purpura. Int J Hematol. 2006; 83(1):29-38.
Tarantino MD, Bolton-Maggs PH. Update on the management of immune thrombocytopenic purpura in children. Curr Opin Hematol. 2007; 14(5):526-34.
Comité Nacional de Hematología. Púrpura trombocitopénica inmunitaria. Guía de diagnóstico y tratamiento. Arch Argent Pediatr. 2010; 108(2):173-8.
Rapetti MC, Donato H, Fassi D, Fondevila C, et al. Trombocitopenia inmune. En: Sociedad Argentina de Hematología. Guías de Diagnóstico y Tratamiento. Buenos Aires: SAH; 2017.Págs.213-28.
Kühne T, Berchtold W, Tran VB, Tran VB, et al. Ethnicity and environment may affect the phenotype of immune thrombocytopenic purpura in children. Pediatr Res. 2000; 48(3):374-9.
Kühne T, Imbach P, Bolton-Maggs PHB, Berchtold W, et al. Newly diagnosed idiopathic thrombocytopenic purpura in childhood: an observational study. Lancet. 2001; 358(9299):2122-5.
Imbach P, Zimmerman S. Local and cultural aspects of childhood idiopathic thrombocytopenic purpura: a summary of statements from the 12 countries worldwide. J Pediatr Hematol Oncol. 2003; 25(Suppl 1):S68-73.
Kühne T, Buchanan GR, Zimmerman S, Michaels LA, 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(5):605-8.
Donato H, Picón A, Rapetti MC, Rosso A, et al. Demographic data, outcome and prognosis of children with Idiopathic Thrombocytopenic Purpura (ITP) in Argentina. Results from a multicentered study. Hematol J. 2004; 5(Suppl 3):a482.
Watts RG. Idiopathic thrombocytopenic purpura: A 10-year natural history study at the Childrens Hospital of Alabama. Clin Pediatr (Phila). 2004; 43(8):691-702.
Imbach P, Kühne T, Muller D, Berchtold W, et al. Childhood ITP: 12 months follow-up data from the prospective Registry I of the Intercontinental Childhood ITP Study Group. Pediatr Blood Cancer. 2006; 46(3):351-6.
Donato H, Picón A, Martinez M, Rapetti MC, 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(4):491-6.
Lowe EJ, Buchanan GR. Idiopathic thrombocytopenic purpura diagnosed during the second decade of life. J Pediatr. 2002; 141(2):253-8.
Sandoval C, Visintainer P, Ozkaynak MF, Tugal O, et al. Clinical features and treatment outcomes of 79 infants with immune thrombocytopenic purpura. Pediatr Blood Cancer. 2004; 42(1):109-12.
Donato H, Picón A, Rapetti MC, Rosso A, et al. Splenectomy and spontaneous remission in children with chronic idiopathic thrombocytopenic purpura. Pediatr Blood Cancer. 2006;47(Suppl 5):S737-9.
Glanz J, France E, Xu S, Hayes T, et al. A population-based, multisite cohort study of the predictors of chronic idiopathic thrombocytopenic purpura in children. Pediatrics. 2008; 121(3):e506-12.
Yacobovich J, Revel-Vilk S, Tamary H. Childhood immune thrombocytopenia – Who will spontaneously recover? Semin Hematol. 2013; 50(Suppl 1):S71-4.
Edslev PW, Rosthøj S, Treutiger I, Rajantie J, et al. A clinical score predicting a brief and uneventful course of newly diagnosed idiopathic thrombocytopenic purpura in children. Br J Haematol. 2007; 138(4):513-6.
Schifferli A, Holbro A, Chitlur M, Coslovsky M, et al. A comparative prospective observational study of children and adults with immune thrombocytopenia: 2-year followup. Am J Hematol. 2018; 93(6):751-9.
Bennett CM, Neunert C, Grace RF, Buchanan G, et al. Predictors of remission in children with newly diagnosed immune thrombocytopenia: Data from the Intercontinental Cooperative ITP Study Group Registry II participants. Pediatr Blood Cancer. 2018; 65(1):e26736.
Butros LJ, Bussel JB. Intracranial hemorrhage in immune thrombocytopenic purpura: a retrospective analysis. J Pediatr Hematol Oncol. 2003; 25(8):660-4.
Psaila B, Petrovic A, Page LK, Menell J, et al. Intracranial hemorrhage (ICH) in children with immune thrombocytopenia (ITP): study of 40 cases. Blood. 2009; 114(23):4777-83.
Beardsley D, Nathan DG. Platelet abnormalities in infancy and childhood. En: Nathan DG, Orkin SH, Oski F (eds.). Nathan and Oski’s Hematology of Infancy and Childhood. 5th ed. Philadelphia: WB Saunders; 1998.Págs.1585-1630.
Cines DB, Blanchette VS. Immune thrombocytopenic purpura. N Engl J Med. 2002; 346(13):995-1008.
Imbach P, Barandun S, d’Apuzzo V, Baumgartner C, et al. High-dose intravenous gammaglobulin for idiopathic thrombocytopenic purpura in childhood. Lancet.1981; 1(8232):1228-31.
Salama A, Müeller-Eckhardt C, Kiefel V. Effect of intravenous immunoglobulin in immune thrombocytopenia. Lancet. 1983; 2(8343):193-5.
Scaradavou A, Woo B, Woloski BMR, Cunningham-Rundles S, et al. Intravenous anti-D treatment of immune thrombocytopenic purpura: experience in 272 patients. Blood. 1997; 89(8):2689-700.
Semple JW, Allen D, Rutherford M, Woloski M, et al. Anti-D (WinRho SD) treatment of children with chronic autoimmune thrombocytopenic purpura stimulates transient cytokine/chemokine production. Am J Hematol. 2002; 69(3):225-7.
Benesch M, Kerbl R, Lackner H, Berqhold A, et al. Low-dose versus high-dose immunoglobulin for primary treatment of acute immune thrombocytopenic purpura in children: results of a prospective, randomized single-center trial. J Pediatr Hematol Oncol. 2003; 25(10):797-800.
Beck CE, Nathan PC, Parkin PC, Blanchette VS, 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(4):521-7.
Lioger B, Maillot F, Ternant D, Passot C, et al. Efficacy and safety of anti-D immunoglobilins versus intravenous immunoglobulins for immune thrombocytopenia in children: Systematic review and meta-analysis of randomized controlled trials. J Pediatr. 2019; 204:225-33.e8.
Donato H. PTI. “Segun pasan los años”. Conferencia Nacional Dr. Gregorio Bomchil. Hematología. 2017; 21(Extraordinario): 137-51.
Flores A, Klaassen RJ, Buchanan G, Neunert CE. Patterns and influences in health-related quality of life in children with immune thrombocytopenia: A study from the Dallas ITP Cohort. Pediatr Blood Cancer. 2017; 64(8):e26405.
McMillan R, Bussel JB, George JN, Lalla D, et al. Self-reported health-related quality of life in adults with chronic immune thrombocytopenic purpura. Am J Hematol. 2008; 83(2):150-4.
Aronis S, Platokouki H, Mitsika A, Haidas S, et al. Seventeen years of experience with chronic idiopathic thrombocytopenic purpura in childhood. Is therapy always better? Pediatr Hematol Oncol. 1994; 11(5):487-98.
Choudhry VP, Kashyap R, Ahlawat S, Pati HP. Vinblastine and Danazol therapy in steroid resistant childhood chronic idiopathic thrombocytopenic purpura. Int J Hematol. 1995; 61(3):157-62.
Adams DM, Kinney TR, O’Branski-Rupp E, Ware RE. High-dose oral dexamethasone therapy for chronic childhood idiopathic thrombocytopenic purpura. J Pediatr. 1996; 128(2):281-3.
Chen JS, Wu JM, Chen YJ, Yeh TF. Pulsed high-dose dexamethasone therapy in children with chronic idiopathic thrombocytopenic purpura. J Pediatr Hematol Oncol. 1997; 19(6):526-9.
Chandra J, Dua T, Narayan S, Jain V, et al. Dexamethasone therapy in chronic ITP. Indian Pediatr. 2000; 37(6):647-50.
Hemmila R, Foley DS, Castle VP, Hirschl RB. The response to splenectomy in pediatric patients with idiopathic thrombocytopenic purpura who fail high-dose intravenous immune globulin. J Pediatr Surg. 2000; 35(6):967-71.
Mantadakis E, Buchanan GR. Elective splenectomy in children with idiopathic thrombocytopenic purpura. J Pediatr Hematol Oncol. 2000; 22(2):148-53.
Tarantino MD. Treatment options for chronic immune (idiopathic) thrombocytopenia purpura in children. Semin Hematol. 2000; 3(1 Suppl 1):S35-41.
Gesundheit B, Cividalli G, Freeman A, Yatziv S, et al. Cyclosporin A in the treatment of refractory immune thrombocytopenia purpura in children. Eur J Haematol. 2001; 66(5):347-51.
El-Alfy MS, El-Tawil MM, Shahein N. 5- to 16- year followup following splenectomy in chronic immune thrombocytopenic purpura in children. Acta Haematol. 2003; 110(1):20-4.
Wang J, Wiley JM, Luddy R, Greenberg J, et al. Chronic immune thrombocytopenic purpura in children: assessment of rituximab treatment. J Pediatr. 2005; 146(2):217-21.
Parodi E, Nobili B, Perrotta S, Rosaria Matarese SM, et al. Rituximab (Anti-CD20 monoclonal antibody) in children with chronic refractory symptomatic immune thrombocytopenic purpura: efficacy and safety of treatment. Int J Haematol. 2006; 84(1):48-53.
Peñalver FJ, Jimenez-Yuste V, Almagro M, Alvarez-Larrán A, et al. Rituximab in the management of chronic immune thrombocytopenic purpura: an effective and safe therapeutic alternative in refractory patients. Ann Hematol. 2006; 85(6):400-6.
Tamminga RY, Bruin MC. Rituximab treatment for symptomatic chronic ITP. Pediatr Blood Cancer. 2006; 47(Suppl 5):S714-6.
Franchini M, Zaffanello M, Veneri D, Lippi G. Rituximab for the treatment of childhood chronic idiopathic thrombocytopenic purpura and hemophilia with inhibitors. Pediatr Blood Cancer. 2007; 49(1):6-10.
Kühne T, Blanchette V, Buchanan GR, Ramenghi U, et al. Splenectomy in children with idiopathic thrombocytopenic purpura: A prospective study of 134 children from the Intercontinental Childhood ITP Study Group. Pediatr Blood Cancer. 2007; 49(6):829-34.
Bennett CM, Rogers ZR, Kinnamon DD, Bussel JB, et al. Prospective phase 1/2 study of rituximab in childhood and adolescent chronic immune thrombocytopenic purpura. Blood. 2006; 107(7):2639-42.
Chaturvedi S, Arnold DM, McCrae KR. Splenectomy for immune thrombocytopenia: down but not out. Blood. 2018; 131(11):1172-82.
Grainger JD, Locatelli F, Chotsampancharoen T, Donyush E, et al. Eltrombopag for children with chronic immune thrombocytopenia (PETIT2): a randomized, multicentre, placebo-controlled trial. Lancet. 2015; 386(10004):1649-58.
Elalfy MS, Abdelmaksoud AA, Eltonbary KY. Romiplostim in children with chronic refractory ITP: randomized placebo controlled study. Ann Hematol. 2011; 90(11):1341-4.
Bussel JB, Buchanan GR, Nugent DJ, Gnarra DJ, et al. A randomized, double-blind study of romiplostin to determine its safety and efficacy in children with immune thrombocytopenia. Blood. 2011; 118(1):28-36.
Bussel JB, Hsieh L, Buchanan GR, Stine K, et al. Long-term use of the thrombopoietin-mimetic romiplostim in children with severe chronic immune thrombocytopenia (ITP). Pediatr Blood Cancer. 2015; 62(2):208-13.
Cines DB, Wasser J, Rodeghiero F, Chong BH, et al. Safety and efficacy of romiplostim in splenectomized and nonsplenectomized patients with primary immune thrombocytopenia. Haematologica. 2017; 102(8):1342-51.
Khellaf M, Viallard JF, Hamidou M, Cheze S, et al. A retrospective pilot evaluation of switching thrombopoietic receptor-agonists in immune thrombocytopenia. Haematologica. 2013; 98(6):881-7.
Grace RF, Shimano KA, Bhat R, Neunert C, et al. Secondline treatments in children with immune thrombocytopenia: Effect on platelet count and patient-centered outcomes. Am J Hematol. 2019; 94(7):741-50.
Tumaini Massaro J, Chen Y, Ke Z. Efficacy and safety of thrombopoietin receptor agonists in children with chronic immune thrombocytopenia: meta-analysis. Platelets. 2019; Feb 27: 1-8. [Epub ahead of print].
Zhang J, Liang Y, Ai Y, Xie J, et al. Thrombopoietin-receptor agonists for children with immune thrombocytopenia: A systematic review. Expert Opin Pharmacother. 2017; 18(15):1543-51.
Grainger JD, Thind S. A practical guide to the use of eltrombopag in children with chronic immune thrombocytopenia. Pediatr Hematol Oncol. 2017; 34(2):73-89.
Grainger JD, Bolton-Maggs PHB, Godeau B, Bussel J, et al. Diagnosis and management of chronic ITP: comments from an ICIS expert group. Ann Hematol. 2010; 89(Suppl 1):S11-7.
Danielson PD, Shaul DB, Phillipos JD, Stein JE, et al. Technical advances in pediatric laparoscopy have had a beneficial impact on splenectomy. J Pediatr Surg. 2000; 35(11):1578-81.
Esposito C, Schaarschmidt K, Settimi A, Montupet P. Experience with laparoscopic splenectomy. J Pediatr Surg. 2001; 36(2):309-11.
Rescorla FJ, Engum SA, West KW, Tres Scherer LR 3rd, et al. Laparoscopic splenectomy has become the gold standard in children. Am Surg. 2002; 68(3):297-302.
Rosen M, Brody F, Walsh RM, Tarnoff M, et al. Outcome of laparoscopic splenectomy based on hematologic indication. Surg Endosc. 2002; 16(2):272-9.
Taube T, Schmid H, Reinhard H, Von Stackelberg A, et al. Effect of a simgle dose of rituximab in chronic immune thrombocytopenic purpura in childhood. Haematologica. 2005; 90(2):281-3.
Shenoy S, Kelly M, Grossman V, Lee S, et al. Rituximab therapy in children with chronic refractory immune cytopenia: Long-term efficacy and immune function analysis. Blood. 2003; 102:a1020.
Parodi E, Rivetti E, Amendola G, Bisogno G, et al. Longterm follow-up analysis after rituximab therapy in children with refractory symptomatic ITP: identification of factors predictive of a sustained response. Br J Haematol. 2008; 144(4):552-8.
Mueller BU, Bennett CM, Feldman HA, Bussel JB, et al. One year follow-up of children and adolescents with chronic immune thrombocytopenic purpura (ITP) treated with rituximab. Pediatr Blood Cancer. 2009; 52(2):259-62.
Moskowitz IPG, Gaynon PS, Shahidi NT, Cripe TP. Lowdose Cyclosporin A therapy in children with refractory immune thrombocytopenic purpura. J Pediatr Hematol Oncol. 1999; 21(1):77-9.
Donato H. Trastornos de las plaquetas. En: Donato H, Rapetti MC (eds.). Hematología Neonatal. Buenos Aires: FUNDASAP; 2007.Págs.227-54.
Valat AS, Caulier MT, Devos P, Rugeri L, et al. Relationships between severe neonatal thrombocytopenia and maternal characteristics in pregnancies associated with autoimmune thrombocytopenia. Br J Haematol. 1998; 103(2):397-401.
Kim BJ, Kim HS, Jang HJ, Kim JH. Helicobacter pylori eradication in idiopathic thrombocytopenic purpura: A metaanalysis of randomized trials. Gastroenterol Res Pract. 2018; 2018:6090878.