Table 5

Trial-adapted criteria for eligibility and outcome assessments in ITP

Eligibility (all should be met)

  • Previously treated or untreated patients fitting within one of the different phases of the disease (in Table 1). Refractory patients defined as in Table 4.

  • Entry platelet count: at least < 30 × 109/L. At least < 50 × 109/L in specific clinical settings or patients on steroids, or in the presence of bleeding symptoms

  • Patients should be on a stable treatment or off any treatment for a time sufficient to exclude a late effect (see Table 3)

 
Supplemental specifications

  • Pediatric and adult patients analyzed separately

  • Response to previous treatment(s), if any, should be reported

 
End points

  • Primary end points:

    • CR or R based on platelet count as in Table 2 *

  • Secondary end points:

    • Adverse events (safety), need for rescue interventions, corticosteroids/concomitant treatment reduction, rate of splenectomies.

    • Could become primary end points according to the design of the clinical trial or patient characteristics.

    • Bleeding scale, HRQoL assessment and, whenever possible, pharmacoeconomic analysis should be included

 
Timing of assessment of primary end points and duration of response

  • Depends on the type of treatment

  • Patients enrolled while on a stable treatment with one or more agents must be no longer receiving these treatments for a time sufficient to exclude any protracted effect†

  • Duration could be calculated as follows depending on study design:

    • The time from CR or R as defined in Table 2 to loss of response‡

    • The cumulative time spent in CR or R or cumulative time spent without meeting a predefined end point(s)

 
Adverse events

  • Bleeding episodes, rescue interventions, frequency of splenectomy, and treatment-related side effects occurring during or after the time of exposure to the experimental agent always reported. Duration of side effects monitoring time after the end of experimental treatment should be provided.

  • For assessment of rebound thrombocytopenia or bleeding, the immediate period after the suspension of the agent up to the attainment of a stable platelet count§ or institution of a new treatment should be considered. This treatment should be recorded.

  • A predefined exceedingly high platelet count induced by treatment could be considered an adverse event, depending on the agent under investigation

 
Eligibility (all should be met)

  • Previously treated or untreated patients fitting within one of the different phases of the disease (in Table 1). Refractory patients defined as in Table 4.

  • Entry platelet count: at least < 30 × 109/L. At least < 50 × 109/L in specific clinical settings or patients on steroids, or in the presence of bleeding symptoms

  • Patients should be on a stable treatment or off any treatment for a time sufficient to exclude a late effect (see Table 3)

 
Supplemental specifications

  • Pediatric and adult patients analyzed separately

  • Response to previous treatment(s), if any, should be reported

 
End points

  • Primary end points:

    • CR or R based on platelet count as in Table 2 *

  • Secondary end points:

    • Adverse events (safety), need for rescue interventions, corticosteroids/concomitant treatment reduction, rate of splenectomies.

    • Could become primary end points according to the design of the clinical trial or patient characteristics.

    • Bleeding scale, HRQoL assessment and, whenever possible, pharmacoeconomic analysis should be included

 
Timing of assessment of primary end points and duration of response

  • Depends on the type of treatment

  • Patients enrolled while on a stable treatment with one or more agents must be no longer receiving these treatments for a time sufficient to exclude any protracted effect†

  • Duration could be calculated as follows depending on study design:

    • The time from CR or R as defined in Table 2 to loss of response‡

    • The cumulative time spent in CR or R or cumulative time spent without meeting a predefined end point(s)

 
Adverse events

  • Bleeding episodes, rescue interventions, frequency of splenectomy, and treatment-related side effects occurring during or after the time of exposure to the experimental agent always reported. Duration of side effects monitoring time after the end of experimental treatment should be provided.

  • For assessment of rebound thrombocytopenia or bleeding, the immediate period after the suspension of the agent up to the attainment of a stable platelet count§ or institution of a new treatment should be considered. This treatment should be recorded.

  • A predefined exceedingly high platelet count induced by treatment could be considered an adverse event, depending on the agent under investigation

 

HRQoL indicates health-related quality of life.

*

At variance with Table 4, these definitions should be also adopted for refractory cases, considering the experimental nature of clinical trials requiring objective measurements.

†

Specify the duration that a subject should be off other treatments and/or the time elapsed after any rescue medication at the time of response evaluation, see also Table 3. For patients enrolled while on a stable concomitant treatment, still requiring it at the time of response evaluation, only secondary end points can be assessed

‡

For some agents requiring continuous treatment like TPO agonists an upper limit of acceptable platelet count should be predefined and thus cumulative time spent within a therapeutic window is most suitable.

§

Defined as a platelet count not requiring treatment or dosage modification for at least 15 days.

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