Pediatric Nephrology

, Volume 24, Issue 9, pp 1749–1752 | Cite as

Rituximab therapy in two children with autoimmune thrombotic thrombocytopenic purpura

  • Jumana H. Albaramki
  • Juliana Teo
  • Stephen I. Alexander
Brief Report


Thrombotic thrombocytopenic purpura (TTP) is a rare disease among pediatric patients, in whom it may be mistaken for hemolytic uremic syndrome (HUS) and idiopathic thrombocytopenic purpura (ITP). Familial forms are caused by mutations in the ADAMTS13 gene, whereas acquired forms may result from an inhibitory antibody directed against ADAMTS13, a metalloprotease that cleaves very large multimers of Von Willebrand factor (VWF), thereby preventing platelet aggregation in blood vessels. We report two cases of TTP. The first was a 15-year-old girl with her first episode of TTP that failed to respond after 10 days of plasmapheresis and was treated with rituximab; she has remained in remission at 12 months of follow-up. The second was a 6-year-old boy with acquired relapsing TTP previously managed with plasmapheresis and prednisolone, who presented with a third relapse that was treated with plasmapheresis and rituximab; he remains in remission 17 months after treatment. Rituximab has been used by pediatricians for treating B cell malignancy, autoimmune diseases and antibody-mediated diseases, such as the Factor VIII inhibitors in hemophilia A, and may also have a promising role in children with acute refractory or chronic relapsing TTP.


B lymphocyte Plasmapheresis Rituximab Thrombotic thrombocytopenic purpura 


  1. 1.
    George JN (2006) Clinical practice. Thrombotic thrombocytopenic purpura. N Engl J Med 354:1927–1935PubMedCrossRefGoogle Scholar
  2. 2.
    Sadler JE (2008) Von Willebrand factor, ADAMTS13, and thrombotic thrombocytopenic purpura. Blood 112:11–18PubMedCrossRefGoogle Scholar
  3. 3.
    Licht C, Stapenhorst L, Simon T, Budde U, Schneppenheim R, Hoppe B (2004) Two novel ADAMTS13 gene mutations in thrombotic thrombocytopenic purpura/hemolytic-uremic syndrome (TTP/HUS). Kidney Int 66:955–958PubMedCrossRefGoogle Scholar
  4. 4.
    Rufer A, Brodmann D, Gregor M, Kremer Hovinga JA, Lammle B, Wuillemin WA (2007) Rituximab for acute plasma-refractory thrombotic thrombocytopenic purpura. A case report and concise review of the literature. Swiss Med Wkly 137:518–524PubMedGoogle Scholar
  5. 5.
    Yomtovian R, Niklinski W, Silver B, Sarode R, Tsai HM (2004) Rituximab for chronic recurring thrombotic thrombocytopenic purpura: a case report and review of the literature. Br J Haematol 124:787–795PubMedCrossRefGoogle Scholar
  6. 6.
    George JN, Woodson RD, Kiss JE, Kojouri K, Vesely SK (2006) Rituximab therapy for thrombotic thrombocytopenic purpura: a proposed study of the Transfusion Medicine/Hemostasis Clinical Trials Network with a systematic review of rituximab therapy for immune-mediated disorders. J Clin Apher 21:49–56PubMedCrossRefGoogle Scholar
  7. 7.
    Garvey B (2008) Rituximab in the treatment of autoimmune haematological disorders. Br J Haematol 141:149–169PubMedCrossRefGoogle Scholar
  8. 8.
    Nwobi O, Abitbol CL, Chandar J, Seeherunvong W, Zilleruelo G (2008) Rituximab therapy for juvenile-onset systemic lupus erythematosus. Pediatr Nephrol 23:413–419PubMedCrossRefGoogle Scholar
  9. 9.
    Dotsch J, Muller-Wiefel DE, Kemper MJ (2008) Rituximab: is replacement of cyclophosphamide and calcineurin inhibitors in steroid-dependent nephrotic syndrome possible? Pediatr Nephrol 23:3–7PubMedCrossRefGoogle Scholar
  10. 10.
    Curtillet C, Poullin P, Dore E, Fossat C, Lefevre P, Michel G (2006) Paediatric case report of an acquired autoimmune thrombotic thrombocytopenic purpura. Arch Pediatr 13:1521–1524PubMedCrossRefGoogle Scholar
  11. 11.
    Kosugi S, Matsumoto M, Ohtani Y, Take H, Ishizashi H, Fujimura Y, Kuyama J (2005) Rituximab provided long-term remission in a patient with refractory relapsing thrombotic thrombocytopenic purpura. Int J Hematol 81:433–436PubMedCrossRefGoogle Scholar
  12. 12.
    Jasti S, Coyle T, Gentile T, Rosales L, Poiesz B (2008) Rituximab as an adjunct to plasma exchange in TTP: a report of 12 cases and review of literature. J Clin Apher 23:151–156PubMedCrossRefGoogle Scholar
  13. 13.
    Leandro MJ, Cambridge G, Edwards JC, Ehrenstein MR, Isenberg DA (2005) B-cell depletion in the treatment of patients with systemic lupus erythematosus: a longitudinal analysis of 24 patients. Rheumatology (Oxford) 44:1542–1545CrossRefGoogle Scholar
  14. 14.
    Fakhouri F, Vernant JP, Veyradier A, Wolf M, Kaplanski G, Binaut R, Rieger M, Scheiflinger F, Poullin P, Deroure B, Delarue R, Lesavre P, Vanhille P, Hermine O, Remuzzi G, Grunfeld JP (2005) Efficiency of curative and prophylactic treatment with rituximab in ADAMTS13-deficient thrombotic thrombocytopenic purpura: a study of 11 cases. Blood 106:1932–1937PubMedCrossRefGoogle Scholar
  15. 15.
    Suzan F, Ammor M, Ribrag V (2001) Fatal reactivation of cytomegalovirus infection after use of rituximab for a post-transplantation lymphoproliferative disorder. N Engl J Med 345:1000PubMedCrossRefGoogle Scholar

Copyright information

© IPNA 2009

Authors and Affiliations

  • Jumana H. Albaramki
    • 1
  • Juliana Teo
    • 1
  • Stephen I. Alexander
    • 1
  1. 1.Centre for Kidney Research, Childrens’ Hospital at WestmeadWestmeadAustralia

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