Table 2.

Qualifiers required for defining pediatric emergent refractory ITP and ITP that fails to respond to disease-modifying therapy

ITP qualifiers = indications + treatments + response + platelets/symptoms
Indications for treatment 
  • Emergent vs disease-modifying therapy

 
Prior trialed treatments 
  • Pharmacotherapy: administered at appropriate dosing for an adequate duration

  • Interventions (eg, splenectomy)

 
Treatment response 
  • NR: no response

  • PR

    • PR-t (transient) 

    • PR-d (durable) 

  • CR

    • CR-t (transient) 

    • CR-d (durable) 

 
Platelet response/symptom response 
  • Platelet response

  • Symptom response

    • Bleeding

    • Quality of life

    • Fatigue

 
ITP qualifiers = indications + treatments + response + platelets/symptoms
Indications for treatment 
  • Emergent vs disease-modifying therapy

 
Prior trialed treatments 
  • Pharmacotherapy: administered at appropriate dosing for an adequate duration

  • Interventions (eg, splenectomy)

 
Treatment response 
  • NR: no response

  • PR

    • PR-t (transient) 

    • PR-d (durable) 

  • CR

    • CR-t (transient) 

    • CR-d (durable) 

 
Platelet response/symptom response 
  • Platelet response

  • Symptom response

    • Bleeding

    • Quality of life

    • Fatigue

 

The duration of response to define modifiers (transient and durable) to be determined in collaboration with the IWG.

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