Table 3.
Safety outcomes
|
Number of patients (N = 37) a |
Number of AEs (E/R) |
|
|---|---|---|
| Total years in trial b | 65.6 | |
| Exposure | ||
| Total number of exposure days per patient, median (range) | 59.0 (1‐197) | … |
| Number of prophylaxis doses per patient, median (range) | 78.5 (1‐196) | … |
| AEs, n (%) | 36 (97.3) | 380 (5.79) |
| AEs according to severity | ||
| Mild | 35 (94.6) | 331 (5.04) |
| Moderate | 19 (51.4) | 38 (0.58) |
| Severe | 9 (24.3) | 11 (0.17) |
| Most common AEs, n (%) | ||
| Pyrexia | 19 (51.4) | 51 (0.78) |
| Upper respiratory tract infection | 17 (45.9) | 24 (0.37) |
| Nasopharyngitis | 15 (40.5) | 31 (0.47) |
| Cough | 10 (27.0) | 15 (0.23) |
| Rhinorrhea | 8 (21.6) | 14 (0.21) |
| Diarrhea | 7 (18.9) | 8 (0.12) |
| SAEs, c n (%) | 14 (37.8) | 24 (0.37) |
| AEs judged by the investigator to be related to treatment, d n (%) | 7 (18.9) | 13 (0.20) |
| MESIs, n (%) | 9 (24.3) | 14 (0.21) |
| Drug hypersensitivity | 2 (5.4) | 3 (0.05) |
| Anaphylactic reaction e | 1 (2.7) | 1 (0.02) |
| Hypersensitivity | 1 (2.7) | 1 (0.02) |
| Rash | 1 (2.7) | 4 (0.06) |
| Papular rash | 1 (2.7) | 1 (0.02) |
| FIX inhibition e | 2 (5.4) | 2 (0.03) |
| Accidental underdose | 1 (2.7) | 1 (0.02) |
| ASD d | 1 (2.7) | 1 (0.02) |
| AEs leading to withdrawal, n (%) | 3 (8.1) | 5 (0.08) |
Abbreviations: AE, adverse event; ASD, autism spectrum disorder; ED, exposure day; E/R, number of AEs/patient‐years of exposure; FIX, coagulation factor IX; MESI, medical event of special interest; N9‐GP, nonacog beta pegol; SAE, serious adverse event; SOC, system organ class.
Includes all treated patients (preprophylaxis and prophylaxis).
Includes follow‐up period for patients with inhibitors who were off treatment and discontinued patients (from time of discontinuation until end of trial).
SAE SOCs included infections and infestations; gastrointestinal disorders; blood and lymphatic system disorders; investigations; immune system disorders; injury, poisoning, and procedural complications; skin and subcutaneous tissue disorders; and vascular disorders.
The patient with ASD was among the 17 who underwent a neurological exam. Communication with the patient’s family was complicated, as they communicated via a translator. This patient, aged 4 years, had abnormal findings in the neurological exam that were described by the investigator as possibly related to N9‐GP. He was enrolled in October 2015 at 15 months old with no abnormalities reported, and the first N9‐GP dose was administered 9 months later. The patient showed increasingly aberrant behavior over time and did not react to verbal or visual cues appropriately or exhibit normal social interaction. In January 2018, after ~ 27 months on the trial (101 EDs), the patient was diagnosed with hypoacusis (drainage of the ear was performed in May 2019). Nine months later, in October 2018, after > 130 EDs, the investigator reported that the patient (now aged ~ 4 years) was suspected to have ASD with the start dated to October 2017 (87 EDs). The patient received N9‐GP until June 2019 and remains in the trial for follow‐up. He has been referred to relevant medical specialists for further assessment and support.
SAE that was also considered a MESI.