Table 4

Refractory ITP

Definition (all should be met)

  • Failure to achieve at least R or loss of R after splenectomy*

  • Need of treatment(s) (including, but not limited to, low dose of corticosteroids) to minimize the risk of clinically significant bleeding.† Need of on-demand or adjunctive therapy alone does not qualify the patient as refractory.

  • Primary ITP confirmed by excluding other supervened causes of thrombocytopenia

 
Definition of on-demand therapy

  • Any therapy used to temporarily increase the platelet count sufficiently to safely perform invasive procedures or in case of major bleeding or trauma‡

 
Definition of adjunctive therapy

  • Any non-ITP specific therapy that may decrease bleeding (eg, antifibrinolytic agents, hormonal agents, DDAVP, recombinant factor VIIa, fibrin sealants). Platelet transfusion is also included.

 
Definition of response to therapy in refractory ITP

  • Ability to maintain a platelet count sufficient to prevent clinically significant bleeding†§

  • Ability to decrease toxic therapy (eg, corticosteroids) does not qualify for response but should be reported

 
Definition of response to on-demand therapy

  • Control of bleeding in the specific situation

  • Achievement of a platelet count sufficient to perform procedure or minimize bleeding from trauma

 
Definition (all should be met)

  • Failure to achieve at least R or loss of R after splenectomy*

  • Need of treatment(s) (including, but not limited to, low dose of corticosteroids) to minimize the risk of clinically significant bleeding.† Need of on-demand or adjunctive therapy alone does not qualify the patient as refractory.

  • Primary ITP confirmed by excluding other supervened causes of thrombocytopenia

 
Definition of on-demand therapy

  • Any therapy used to temporarily increase the platelet count sufficiently to safely perform invasive procedures or in case of major bleeding or trauma‡

 
Definition of adjunctive therapy

  • Any non-ITP specific therapy that may decrease bleeding (eg, antifibrinolytic agents, hormonal agents, DDAVP, recombinant factor VIIa, fibrin sealants). Platelet transfusion is also included.

 
Definition of response to therapy in refractory ITP

  • Ability to maintain a platelet count sufficient to prevent clinically significant bleeding†§

  • Ability to decrease toxic therapy (eg, corticosteroids) does not qualify for response but should be reported

 
Definition of response to on-demand therapy

  • Control of bleeding in the specific situation

  • Achievement of a platelet count sufficient to perform procedure or minimize bleeding from trauma

 

DDAVP indicates deamino arginine vasopressin.

*

May not be applicable in children or in patients with accessory spleen.

†

Bleeding symptoms measured by a validated scale whenever possible (requires further studies).

‡

Specific platelet thresholds cannot be provided, but in most instances, a platelet count of 50-70 × 109/L would fulfill this criterion.

§

A strict definition of response in terms of predefined platelet count cannot be given and may not be appropriate when considering the risk/benefit ratio in refractory ITP, because the trade off between efficacy of a specific treatment and its short- and long-term toxicity varies among patients.

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