Table 2

Rate of Medical Therapy Success Based on GR and Power Thresholds

BTT/CAP%P ≤200%%P >200%Validation%P ≤200%%P >200%
GR >1.253/4 (75.0)0/3 (0.0)GR >1.252/13 (15.4)0/7 (0.0)
GR ≤1.257/9 (77.7)0/0 (0.0)GR ≤1.2526/32 (81.3)1/1 (100.0)

Values are nsuccess/ntotal (%). For the validation group, the 81.3% success rate when below both thresholds is significantly greater than the success rate when at least 1 threshold is exceeded.

BTT = bridge to transplant; CAP = continued access protocol; GR = growth rate; %P = percent of expected power.

  • p < 0.001.