Table 1.
Adjudicator evaluation of GTR rFVIIa efficacy data
| Adjudicator evaluation of efficacy – bleeding episodes16
| ||||||
|---|---|---|---|---|---|---|
| Treatment group | Number of patientsa | Number of episodes | Success n (%) | Failure n (%) | Insufficient data n (%) | No consensus n (%) |
| All rFVIIab | 92 | 266 | 251 (94.4) | 4 (1.5) | 6 (2.3) | 5 (1.9) |
| By treatment regimen | ||||||
| rFVIIa only | 44 | 109 | 101 (92.7) | 2 (1.8) | 4 (3.7) | 2 (1.8) |
| rFVIIa ± platelets ± other hemostatic agents | 69 | 157 | 150 (95.5) | 2 (1.3) | 2 (1.3) | 3 (1.9) |
| By antibody/refractory group | ||||||
| Refractoriness ± platelet-specific antibodiesc,d | 31 | 79 | 75 (94.9) | 2 (2.5) | 2 (2.5) | 0 (0.0) |
| Platelet-specific antibodiesc,d | 8 | 10 | 10 (100.0) | 0 (0.0) | 0 (0.0) | NA |
| Neither or unknownd,e | 57 | 177 | 166 (93.8) | 2 (1.1) | 4 (2.3) | 5 (2.8) |
| Adjudicator evaluation of efficacy – surgical procedures16
| ||||
|---|---|---|---|---|
| Treatment group | Number of patientsa | Number of procedures | Success n (%) | Insufficient dataf n (%) |
| All rFVIIab | 77 | 160 | 159 (99.4) | 1 (0.6) |
| By treatment regimen | ||||
| rFVIIa only | 35 | 66 | 65 (98.5) | 1 (1.5) |
| rFVIIa ± platelets ± other hemostatic agents | 57 | 94 | 94 (100.0) | 0 (0.0) |
| By antibody/refractory group | ||||
| Refractoriness ± platelet-specific antibodiesc,g | 33 | 70 | 69 (98.6) | 1 (1.4) |
| Platelet-specific antibodiesc,g | 11 | 24 | 24 (100.0) | 0 (0.0) |
| Neither or unknowne,g | 36 | 66 | 66 (100.0) | 0 (0.0) |
Notes:
Patient numbers are not additive. Patients may have episodes with different treatment regimens and have more than one antibody/refractory status.
All treatment regimens that included treatment with rFVIIa.
Includes GPIIb/IIIa, HLA, and unspecified platelet-specific antibodies.
Treatment was rFVIIa only for 26/79 episodes with refractoriness with or without antibodies, 2/10 episodes with platelet-specific antibodies only, and 81/177 episodes with neither or unknown. The remainder received rFVIIa with platelets and/or antifibrinolytic agents.
Assumes no platelet-specific antibodies or refractoriness reported or antibody and refractory status unknown.
No reports of failure or lack of consensus were reported.
Treatment was rFVIIa only for 22/70 episodes with refractoriness with or without antibodies, 13/24 episodes with platelet-specific antibodies only, and 31/66 episodes with neither or unknown. The remainder received rFVIIa with platelets and/or antifibrinolytic agents.
Abbreviations: GP, glycoprotein; GTR, Glanzmann’s Thrombasthenia Registry; HLA, human leukocyte antigen; NA, not available; rFVIIa, recombinant activated coagulation factor VII.