Table 2

Proposed criteria for assessing response to ITP treatments

Quality of response*†

  • CR: platelet count ≥ 100 × 109/L and absence of bleeding

  • R: platelet count ≥ 30 × 109/L and at least 2-fold increase the baseline count and absence of bleeding

  • Time to response: time from starting treatment to time of achievement of CR or R‡

  • NR: platelet count < 30 × 109/L or less than 2-fold increase of baseline platelet count or bleeding

  • Loss of CR or R: platelet count below 100 × 109/L or bleeding (from CR) or below 30 × 109/L or less than 2-fold increase of baseline platelet count or bleeding (from R)

 
Timing of assessment of response to ITP treatments

  • Variable, depends on the type of treatment (see Table 3)

 
Duration of response§

  • Measured from the achievement of CR or R to loss of CR or R

  • Measured as the proportion of the cumulative time spent in CR or R during the period under examination as well as the total time observed from which the proportion is derived

 
Corticosteroid-dependence

  • The need for ongoing or repeated doses administration of corticosteroids for at least 2 months to maintain a platelet count at or above 30 × 109/L and/or to avoid bleeding (patients with corticosteroid dependence are considered nonresponders)

 
Supplemental outcomes (whenever possible)

  • Bleeding symptoms measured by a validated scale (requires additional studies)

  • Health-related quality of life assessment measured by a validated instrument (requires additional studies)

 
Quality of response*†

  • CR: platelet count ≥ 100 × 109/L and absence of bleeding

  • R: platelet count ≥ 30 × 109/L and at least 2-fold increase the baseline count and absence of bleeding

  • Time to response: time from starting treatment to time of achievement of CR or R‡

  • NR: platelet count < 30 × 109/L or less than 2-fold increase of baseline platelet count or bleeding

  • Loss of CR or R: platelet count below 100 × 109/L or bleeding (from CR) or below 30 × 109/L or less than 2-fold increase of baseline platelet count or bleeding (from R)

 
Timing of assessment of response to ITP treatments

  • Variable, depends on the type of treatment (see Table 3)

 
Duration of response§

  • Measured from the achievement of CR or R to loss of CR or R

  • Measured as the proportion of the cumulative time spent in CR or R during the period under examination as well as the total time observed from which the proportion is derived

 
Corticosteroid-dependence

  • The need for ongoing or repeated doses administration of corticosteroids for at least 2 months to maintain a platelet count at or above 30 × 109/L and/or to avoid bleeding (patients with corticosteroid dependence are considered nonresponders)

 
Supplemental outcomes (whenever possible)

  • Bleeding symptoms measured by a validated scale (requires additional studies)

  • Health-related quality of life assessment measured by a validated instrument (requires additional studies)

 

For response criteria in refractory ITP, see Table 4.

HRQoL indicates health-related quality-of-life assessment.

*

Platelet counts should be confirmed on at least 2 separate occasions (at least 7 days apart when used to define CR, R) or 1 day apart when used to define NR or loss of response.

†

Baseline platelet count refers to platelet count at the time of starting of the investigated treatment; for postsplenectomy response evaluation, basal platelet count refers to the platelet count before patient was first treated (initial treatment).

‡

Late responses not attributable to the investigated treatment should not be defined as CR or R (see Table 3).

§

The 2 definitions are not mutually exclusive: the first definition, collectively represented using Kaplan-Meyer analysis, is more suitable for short-course treatments aimed at inducing prolonged remission of the disease, whereas the second one is more suitable to evaluate the overall benefit of continuous or intermittent repeated administration of agents requiring dose adjustments with anticipated temporary losses of CR or R.

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