Annualised bleeding rate defined as number of bleeds in total per patient per year following treatment were estimated by a Poisson model allowing for over-dispersion. The Poisson estimate is presented with a 95% confidence interval (CI). The analysis was performed for the main phase (from Visit 2 to Visit 5 [50-55 exposure day], extension phase (from Visit 6 to end of trial; exposure day 100, expected to occur between 6 and 48 months of trial participation), the combined main and extension phase (from Visit 2 to end of trial) and for the inhibitor cohort.

The number of injections of turoctocog alfa (N8) required per bleed was calculated as the number of injections of turoctocog alfa used in the time period from start of the bleed to stop of the bleed. The mean number of turoctocog alfa injections required to stop the bleed is presented. The analysis was based on the total number of bleeds. The analysis was performed for the main phase (from Visit 2 to Visit 5 [50-55 exposure day], extension phase (from Visit 6 to end of trial; exposure day 100, expected to occur between 6 and 48 months of trial participation), the combined main and extension phase (from Visit 2 to end of trial) and for the inhibitor cohort.

Mean total consumption of turoctocog alfa (N8) used for treatment (includes all injections given: prevention, treatment of bleeds and during surgery) per patient per month. The analysis was performed for the main phase (from Visit 2 to Visit 5 [50-55 exposure day], extension phase (from Visit 6 to end of trial; exposure day 100, expected to occur between 6 and 48 months of trial participation), the combined main and extension phase (from Visit 2 to end of trial) and for the inhibitor cohort.

Mean total consumption of turoctocog alfa (N8) used for treatment (includes all injections given: prevention, treatment of bleeds and during surgery) per patient per year. The analysis was performed for the main phase (from Visit 2 to Visit 5 [50-55 exposure day], extension phase (from Visit 6 to end of trial; exposure day 100, expected to occur between 6 and 48 months of trial participation), the combined main and extension phase (from Visit 2 to end of trial) and for the inhibitor cohort.

Mean consumption of turoctocog alfa (N8) used for treatment of bleed from start to stop of bleed. The analysis was based on number of bleeds. The analysis was performed for the main phase (from Visit 2 to Visit 5 [50-55 exposure day], extension phase (from Visit 6 to end of trial; exposure day 100, expected to occur between 6 and 48 months of trial participation), the combined main and extension phase (from Visit 2 to end of trial) and for the inhibitor cohort.

Mean consumption of turoctocog alfa (N8) used for preventive treatment per month per patient. The analysis was performed for the main phase (from Visit 2 to Visit 5 [50-55 exposure day], extension phase (from Visit 6 to end of trial; exposure day 100, expected to occur between 6 and 48 months of trial participation), the combined main and extension phase (from Visit 2 to end of trial) and for the inhibitor cohort.

Number of adverse events and serious adverse events per patient years of exposure. The analysis was performed for the main phase (from Visit 2 to Visit 5 [50-55 exposure day], extension phase (from Visit 6 to end of trial (exposure day 100, expected to occur between 6 and 48 months of trial participation), the combined main and extension phase (from Visit 2 to end of trial) and for the inhibitor cohort.

The incidence rates (number of patients with new inhibitor in the period/number of participants at risk, expressed in percentage) of clinically relevant inhibitors were defined as an inhibitor titre (≥ 0.6 BU) combined with a decreased recovery (<66% of expected level). Incidence rate of clinically relevant inhibitors according to the type of assay used is presented. The analysis was performed for the main phase (from Visit 2 to Visit 5 [50-55 exposure day], extension phase (from Visit 6 to end of trial; exposure day 100, expected to occur between 6 and 48 months of trial participation) and the combined main and extension phase (from Visit 2 to end of trial).

Incidence rate (number of patients with new inhibitor in the period/number of participants at risk, expressed in percentage) of high-titre inhibitors defined as inhibitor titre ≥ 5 BU (Bethesda Units)/mL). The inhibitors were evaluated for the main phase (from Visit 2 to Visit 5 [50-55 exposure day], extension phase (from Visit 6 to end of trial; exposure day 100, expected to occur between 6 and 48 months of trial participation) and the combined main and extension phase (from Visit 2 to end of trial).

The caregivers/parent reported treatment satisfaction is assessed by the parents using the haemophilia satisfaction questionnaire (HEMO-SAT). The questionnaire contained questions related to treatment that covered 6 domains (ease and convenience, efficacy, burden, specialist, centre and general satisfaction). Adults completing the questionnaire could achieve a score from 0 to 100, with lower scores reflecting greater treatment satisfaction. The scale range for each of the 6 domains was 0-100 with lower scores reflecting greater treatment satisfaction.

The scores of the domains at visit 3 (10th-15th ED), visit 5 (50th-55th ED) and end of trial (EoT) are presented.

Resource utilisation and caregiver burden are analysed in terms of average number of days absent from work and use of mobility aids (e.g. wheelchair or crutches) as a consequence of bleeds. The analysis was performed for the main phase (from Visit 2 to Visit 5 [50-55 exposure day], extension phase (from Visit 6 to end of trial; exposure day 100, expected to occur between 6 and 48 months of trial participation), the combined main and extension phase (from Visit 2 to end of trial) and for the inhibitor cohort.

Resource utilisation and caregiver burden are analysed in terms of average number of days of absence from work and use of mobility aids (e.g. wheelchair or crutches) as a consequence of bleeds. The analysis was performed for the main phase (from Visit 2 to Visit 5 [50-55 exposure day], extension phase (from Visit 6 to end of trial; exposure day 100, expected to occur between 6 and 48 months of trial participation), the combined main and extension phase (from Visit 2 to end of trial) and for the inhibitor cohort.

Participants received prophylactic treatment administered from completion of the main phase or completion of the inhibitor cohort until the end-of-trial visit in the extension phase. They received turoctocog alfa doses of 15-60 IU/kg body weight (BW) administered as an intravenous injection for the prevention of bleeding. The investigator decided what dose of turoctocog alfa to give based on the participant's clinical profile. After completing the main phase, participants continued in the extension phase where at least 50 participants achieved at least 100 ED, which translated into a maximum trial duration of up to 5 years (including any potential inhibitor treatment).

Participants who started preventive treatment in the main and extension phase are included in this arm. They received turoctocog alfa doses of 15-60 IU/kg body weight (BW) administered as an intravenous injection for the prevention of bleeding. The investigator decided what dose of turoctocog alfa to give based on the child's clinical profile. The trial consisted of two phases. In the main phase, an aggregated number of at least 50 participants received preventive treatment with turoctocog alfa until they reached a minimum of 50 exposure days (ED) or until they developed inhibitors. After that the participants continued in the extension phase where at least 50 participants achieved at least 100 ED, which translated into a maximum trial duration of up to 5 years (including any potential inhibitor treatment). The estimated total duration of the trial was approximately 7 years.

Participants who developed an inhibitor during the course of the trial and followed an alternative treatment regimen are included in this arm. They were offered continued treatment with turoctocog alfa for up to 24 months.

Participants received prophylactic treatment administered from completion of the main phase or completion of the inhibitor cohort until the end-of-trial visit in the extension phase. They received turoctocog alfa doses of 15-60 IU/kg body weight (BW) administered as an intravenous injection for the prevention of bleeding. The investigator decided what dose of turoctocog alfa to give based on the participant's clinical profile. After completing the main phase, participants continued in the extension phase where at least 50 participants achieved at least 100 ED, which translated into a maximum trial duration of up to 5 years (including any potential inhibitor treatment).

Participants who started preventive treatment in the main and extension phase are included in this arm. They received turoctocog alfa doses of 15-60 IU/kg body weight (BW) administered as an intravenous injection for the prevention of bleeding. The investigator decided what dose of turoctocog alfa to give based on the child's clinical profile. The trial consisted of two phases. In the main phase, an aggregated number of at least 50 participants received preventive treatment with turoctocog alfa until they reached a minimum of 50 exposure days (ED) or until they developed inhibitors. After that the participants continued in the extension phase where at least 50 participants achieved at least 100 ED, which translated into a maximum trial duration of up to 5 years (including any potential inhibitor treatment). The estimated total duration of the trial was approximately 7 years.

Participants who developed an inhibitor during the course of the trial and followed an alternative treatment regimen are included in this arm. They were offered continued treatment with turoctocog alfa for up to 24 months.

Participants received prophylactic treatment administered from completion of the main phase or completion of the inhibitor cohort until the end-of-trial visit in the extension phase. They received turoctocog alfa doses of 15-60 IU/kg body weight (BW) administered as an intravenous injection for the prevention of bleeding. The investigator decided what dose of turoctocog alfa to give based on the participant's clinical profile. After completing the main phase, participants continued in the extension phase where at least 50 participants achieved at least 100 ED, which translated into a maximum trial duration of up to 5 years (including any potential inhibitor treatment).

Participants who started preventive treatment in the main and extension phase are included in this arm. They received turoctocog alfa doses of 15-60 IU/kg body weight (BW) administered as an intravenous injection for the prevention of bleeding. The investigator decided what dose of turoctocog alfa to give based on the child's clinical profile. The trial consisted of two phases. In the main phase, an aggregated number of at least 50 participants received preventive treatment with turoctocog alfa until they reached a minimum of 50 exposure days (ED) or until they developed inhibitors. After that the participants continued in the extension phase where at least 50 participants achieved at least 100 ED, which translated into a maximum trial duration of up to 5 years (including any potential inhibitor treatment). The estimated total duration of the trial was approximately 7 years.

Participants who developed an inhibitor during the course of the trial and followed an alternative treatment regimen are included in this arm. They were offered continued treatment with turoctocog alfa for up to 24 months.

Participants received prophylactic treatment administered from completion of the main phase or completion of the inhibitor cohort until the end-of-trial visit in the extension phase. They received turoctocog alfa doses of 15-60 IU/kg body weight (BW) administered as an intravenous injection for the prevention of bleeding. The investigator decided what dose of turoctocog alfa to give based on the participant's clinical profile. After completing the main phase, participants continued in the extension phase where at least 50 participants achieved at least 100 ED, which translated into a maximum trial duration of up to 5 years (including any potential inhibitor treatment).

Participants who started preventive treatment in the main and extension phase are included in this arm. They received turoctocog alfa doses of 15-60 IU/kg body weight (BW) administered as an intravenous injection for the prevention of bleeding. The investigator decided what dose of turoctocog alfa to give based on the child's clinical profile. The trial consisted of two phases. In the main phase, an aggregated number of at least 50 participants received preventive treatment with turoctocog alfa until they reached a minimum of 50 exposure days (ED) or until they developed inhibitors. After that the participants continued in the extension phase where at least 50 participants achieved at least 100 ED, which translated into a maximum trial duration of up to 5 years (including any potential inhibitor treatment). The estimated total duration of the trial was approximately 7 years.

Participants who developed an inhibitor during the course of the trial and followed an alternative treatment regimen are included in this arm. They were offered continued treatment with turoctocog alfa for up to 24 months.

Participants received prophylactic treatment administered from completion of the main phase or completion of the inhibitor cohort until the end-of-trial visit in the extension phase. They received turoctocog alfa doses of 15-60 IU/kg body weight (BW) administered as an intravenous injection for the prevention of bleeding. The investigator decided what dose of turoctocog alfa to give based on the participant's clinical profile. After completing the main phase, participants continued in the extension phase where at least 50 participants achieved at least 100 ED, which translated into a maximum trial duration of up to 5 years (including any potential inhibitor treatment).

Participants who started preventive treatment in the main and extension phase are included in this arm. They received turoctocog alfa doses of 15-60 IU/kg body weight (BW) administered as an intravenous injection for the prevention of bleeding. The investigator decided what dose of turoctocog alfa to give based on the child's clinical profile. The trial consisted of two phases. In the main phase, an aggregated number of at least 50 participants received preventive treatment with turoctocog alfa until they reached a minimum of 50 exposure days (ED) or until they developed inhibitors. After that the participants continued in the extension phase where at least 50 participants achieved at least 100 ED, which translated into a maximum trial duration of up to 5 years (including any potential inhibitor treatment). The estimated total duration of the trial was approximately 7 years.

Participants who developed an inhibitor during the course of the trial and followed an alternative treatment regimen are included in this arm. They were offered continued treatment with turoctocog alfa for up to 24 months.

Participants received prophylactic treatment administered from completion of the main phase or completion of the inhibitor cohort until the end-of-trial visit in the extension phase. They received turoctocog alfa doses of 15-60 IU/kg body weight (BW) administered as an intravenous injection for the prevention of bleeding. The investigator decided what dose of turoctocog alfa to give based on the participant's clinical profile. After completing the main phase, participants continued in the extension phase where at least 50 participants achieved at least 100 ED, which translated into a maximum trial duration of up to 5 years (including any potential inhibitor treatment).

Participants who started preventive treatment in the main and extension phase are included in this arm. They received turoctocog alfa doses of 15-60 IU/kg body weight (BW) administered as an intravenous injection for the prevention of bleeding. The investigator decided what dose of turoctocog alfa to give based on the child's clinical profile. The trial consisted of two phases. In the main phase, an aggregated number of at least 50 participants received preventive treatment with turoctocog alfa until they reached a minimum of 50 exposure days (ED) or until they developed inhibitors. After that the participants continued in the extension phase where at least 50 participants achieved at least 100 ED, which translated into a maximum trial duration of up to 5 years (including any potential inhibitor treatment). The estimated total duration of the trial was approximately 7 years.

Participants who developed an inhibitor during the course of the trial and followed an alternative treatment regimen are included in this arm. They were offered continued treatment with turoctocog alfa for up to 24 months.

Participants received prophylactic treatment administered from completion of the main phase or completion of the inhibitor cohort until the end-of-trial visit in the extension phase. They received turoctocog alfa doses of 15-60 IU/kg body weight (BW) administered as an intravenous injection for the prevention of bleeding. The investigator decided what dose of turoctocog alfa to give based on the participant's clinical profile. After completing the main phase, participants continued in the extension phase where at least 50 participants achieved at least 100 ED, which translated into a maximum trial duration of up to 5 years (including any potential inhibitor treatment).

Participants who started preventive treatment in the main and extension phase are included in this arm. They received turoctocog alfa doses of 15-60 IU/kg body weight (BW) administered as an intravenous injection for the prevention of bleeding. The investigator decided what dose of turoctocog alfa to give based on the child's clinical profile. The trial consisted of two phases. In the main phase, an aggregated number of at least 50 participants received preventive treatment with turoctocog alfa until they reached a minimum of 50 exposure days (ED) or until they developed inhibitors. After that the participants continued in the extension phase where at least 50 participants achieved at least 100 ED, which translated into a maximum trial duration of up to 5 years (including any potential inhibitor treatment). The estimated total duration of the trial was approximately 7 years.

Participants who developed an inhibitor during the course of the trial and followed an alternative treatment regimen are included in this arm. They were offered continued treatment with turoctocog alfa for up to 24 months.

Participants received prophylactic treatment administered from completion of the main phase or completion of the inhibitor cohort until the end-of-trial visit in the extension phase. They received turoctocog alfa doses of 15-60 IU/kg body weight (BW) administered as an intravenous injection for the prevention of bleeding. The investigator decided what dose of turoctocog alfa to give based on the participant's clinical profile. After completing the main phase, participants continued in the extension phase where at least 50 participants achieved at least 100 ED, which translated into a maximum trial duration of up to 5 years (including any potential inhibitor treatment).

Participants who started preventive treatment in the main and extension phase are included in this arm. They received turoctocog alfa doses of 15-60 IU/kg body weight (BW) administered as an intravenous injection for the prevention of bleeding. The investigator decided what dose of turoctocog alfa to give based on the child's clinical profile. The trial consisted of two phases. In the main phase, an aggregated number of at least 50 participants received preventive treatment with turoctocog alfa until they reached a minimum of 50 exposure days (ED) or until they developed inhibitors. After that the participants continued in the extension phase where at least 50 participants achieved at least 100 ED, which translated into a maximum trial duration of up to 5 years (including any potential inhibitor treatment). The estimated total duration of the trial was approximately 7 years.

Participants who developed an inhibitor during the course of the trial and followed an alternative treatment regimen are included in this arm. They were offered continued treatment with turoctocog alfa for up to 24 months.

Participants received prophylactic treatment administered from completion of the main phase or completion of the inhibitor cohort until the end-of-trial visit in the extension phase. They received turoctocog alfa doses of 15-60 IU/kg body weight (BW) administered as an intravenous injection for the prevention of bleeding. The investigator decided what dose of turoctocog alfa to give based on the participant's clinical profile. After completing the main phase, participants continued in the extension phase where at least 50 participants achieved at least 100 ED, which translated into a maximum trial duration of up to 5 years (including any potential inhibitor treatment).

Participants who started preventive treatment in the main and extension phase are included in this arm. They received turoctocog alfa doses of 15-60 IU/kg body weight (BW) administered as an intravenous injection for the prevention of bleeding. The investigator decided what dose of turoctocog alfa to give based on the child's clinical profile. The trial consisted of two phases. In the main phase, an aggregated number of at least 50 participants received preventive treatment with turoctocog alfa until they reached a minimum of 50 exposure days (ED) or until they developed inhibitors. After that the participants continued in the extension phase where at least 50 participants achieved at least 100 ED, which translated into a maximum trial duration of up to 5 years (including any potential inhibitor treatment). The estimated total duration of the trial was approximately 7 years.

Participants received prophylactic treatment administered from completion of the main phase or completion of the inhibitor cohort until the end-of-trial visit in the extension phase. They received turoctocog alfa doses of 15-60 IU/kg body weight (BW) administered as an intravenous injection for the prevention of bleeding. The investigator decided what dose of turoctocog alfa to give based on the participant's clinical profile. After completing the main phase, participants continued in the extension phase where at least 50 participants achieved at least 100 ED, which translated into a maximum trial duration of up to 5 years (including any potential inhibitor treatment).

Participants who started preventive treatment in the main and extension phase are included in this arm. They received turoctocog alfa doses of 15-60 IU/kg body weight (BW) administered as an intravenous injection for the prevention of bleeding. The investigator decided what dose of turoctocog alfa to give based on the child's clinical profile. The trial consisted of two phases. In the main phase, an aggregated number of at least 50 participants received preventive treatment with turoctocog alfa until they reached a minimum of 50 exposure days (ED) or until they developed inhibitors. After that the participants continued in the extension phase where at least 50 participants achieved at least 100 ED, which translated into a maximum trial duration of up to 5 years (including any potential inhibitor treatment). The estimated total duration of the trial was approximately 7 years.

Participants received prophylactic treatment administered from completion of the main phase or completion of the inhibitor cohort until the end-of-trial visit in the extension phase. They received turoctocog alfa doses of 15-60 IU/kg body weight (BW) administered as an intravenous injection for the prevention of bleeding. The investigator decided what dose of turoctocog alfa to give based on the participant's clinical profile. After completing the main phase, participants continued in the extension phase where at least 50 participants achieved at least 100 ED, which translated into a maximum trial duration of up to 5 years (including any potential inhibitor treatment).

Participants who started preventive treatment in the main and extension phase are included in this arm. They received turoctocog alfa doses of 15-60 IU/kg body weight (BW) administered as an intravenous injection for the prevention of bleeding. The investigator decided what dose of turoctocog alfa to give based on the child's clinical profile. The trial consisted of two phases. In the main phase, an aggregated number of at least 50 participants received preventive treatment with turoctocog alfa until they reached a minimum of 50 exposure days (ED) or until they developed inhibitors. After that the participants continued in the extension phase where at least 50 participants achieved at least 100 ED, which translated into a maximum trial duration of up to 5 years (including any potential inhibitor treatment). The estimated total duration of the trial was approximately 7 years.

Participants who developed an inhibitor during the course of the trial and followed an alternative treatment regimen are included in this arm. They were offered continued treatment with turoctocog alfa for up to 24 months.

Participants received prophylactic treatment administered from completion of the main phase or completion of the inhibitor cohort until the end-of-trial visit in the extension phase. They received turoctocog alfa doses of 15-60 IU/kg body weight (BW) administered as an intravenous injection for the prevention of bleeding. The investigator decided what dose of turoctocog alfa to give based on the participant's clinical profile. After completing the main phase, participants continued in the extension phase where at least 50 participants achieved at least 100 ED, which translated into a maximum trial duration of up to 5 years (including any potential inhibitor treatment).

Participants who started preventive treatment in the main and extension phase are included in this arm. They received turoctocog alfa doses of 15-60 IU/kg body weight (BW) administered as an intravenous injection for the prevention of bleeding. The investigator decided what dose of turoctocog alfa to give based on the child's clinical profile. The trial consisted of two phases. In the main phase, an aggregated number of at least 50 participants received preventive treatment with turoctocog alfa until they reached a minimum of 50 exposure days (ED) or until they developed inhibitors. After that the participants continued in the extension phase where at least 50 participants achieved at least 100 ED, which translated into a maximum trial duration of up to 5 years (including any potential inhibitor treatment). The estimated total duration of the trial was approximately 7 years.

Participants who developed an inhibitor during the course of the trial and followed an alternative treatment regimen are included in this arm. They were offered continued treatment with turoctocog alfa for up to 24 months.