Controversies in the management of children with idiopathic thrombocytopenic purpura (ITP)

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Controversies in the management of children with
idiopathic thrombocytopenic purpura (ITP)
Thomas Kühne, MDUniversity Childrens Hospital
BaselSwitzerland
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Idiopathic Thrombocytopenic Purpura (ITP)

• Incidence approx. 4100,000 children/year(
  acute leukemia)
• acute ITP approx. 80
• chronic ITP approx. 20

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Idiopathic Thrombocytopenic Purpura (ITP)

• Controversies
• Dogmas
• Expert opinion
• Lack of- data from large clinical trials-
  collaborative research activities-
  centralization of care

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Idiopathic Thrombocytopenic Purpura (ITP)
Statements childhood ITP
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Idiopathic Thrombocytopenic Purpura

• Adequate parental education about the condition
  from an experienced specialist is essential,
  together with open access for children should
  they develop any problems.
• Bolton-Maggs PHB, Dickerhoff R, Vora AJ, Semin
  Thromb Hemost 2001

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Idiopathic Thrombocytopenic Purpura

• DiscrepancyLow platelet count relatively mild
  bleeding symptoms
• ? Large platelets (gt30fl)
• ? Platelet microparticles (lt2fl)
• Enumeration of platelets in the low range
• ? Conventional automated hematology analyzer
• ? Flow cytometry
• ? New generations of hematology analyzers

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Idiopathic Thrombocytopenic Purpura

• Platelet number does not reflect platelet
  function
• ? S hemostatic function
• ? S platelet function
• ? Reality Platelet count
• ? Bleeding symptoms

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ITP Guidelines, Surveys

• Eden OB, Lilleyman JS. Arch Dis Child
  19926710561058
• George JN, et al. Blood 1996883-40
• Zeller B, et al. Pediatr Hematol Oncol
  200017551-558
• Sutor AH, et al. Semin Thromb Hemost
  200127253-267
• Brit Soc Haematol, Br J Haematol 2003120574-596
• Bolton-Maggs PHB, Moon I. Lancet 1997350620-623
• Bolton-Maggs PHB, Moon I. Blood 20019858b
  (abstract)
• Vesely S, et al. J Pediatr Hematol Oncol
  20002255-61
• Vesely S, et al. J Pediatr Hematol Oncol
  200325130-133
• Kühne T, et al. Lancet 20013582122-2125

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Published Experience in Childhood ITP
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Clinical Course of ITP in Children
George et al., 1996
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Randomized trials of children with previously
untreated ITP
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Idiopathic Thrombocytopenic Purpura
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Treated versus untreated children with ITP
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The Intercontinental ChildhoodITP Study
Groupwww.unibas.ch/itpbaselFax 41-61-685-6566
Registry I
Registry II
Splenectomy Registry
PARC ITP
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Idiopathic Thrombocytopenic Purpura (ITP)

• Conclusions
• ? Evidence-based medicine ? dogmas
• Untreated ITP with minor hemorrhage
• Long-term follow-up studies
• Individual bleeding risk at initial diagnosis
• Refractory ITP with long-lasting severe
  thrombocytopenia
• Alternative outcome measures other than
  platelet count. Platelet count increments do not
  reflect clinical requirements
• Evaluation of different drugs and comparison
  with untreated patients (Treatment versus
  Prophylaxis)
• Financial aspects of the management of ITP

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