Adult & Adolescent Study - Efficacy

Proven bleed protection with ALTUVIIIO¹

Low ABRs in the Prior Factor VIII Prophylaxis Group^1,2

ABR* with ALTUVIIIO prophylaxis (N=128)

* Based on treated bleeds.²
† Based on negative binomial model.²

Patients who switched to ALTUVIIIO prophylaxis experienced significant reductions in ABR²

Intrapatient ABR comparison (n=78) vs prior Factor VIII prophylaxis

* Data from prospective observational study.¹
‡ Based on treated bleeds.¹

Intrapatient ABR comparison (n=26) prior Factor VIII On-Demand Group^1,2

Mean number of bleeding episodes reported during the 12 months prior to the study was 35.7 (SD: 22.2) in the Prior Factor VIII On-Demand Group.¹

* Based on treated bleeds.¹

Proven joint bleed protection and improved joint health^1,2

* Based on treated bleeds.¹
† Based on negative binomial model.²

^‡ All patients with target joints at baseline (defined as ≥3 spontaneous bleeding episodes in a major joint which occurred in a consecutive 6-month period) achieved resolution of all target joints (45/45, 100%) with 12 months of prophylactic treatment with ALTUVIIIO (defined as ≤2 bleeding episodes in the target joint in 12 months).^1,2
§ Mean change in total score from baseline to Week 52.¹

Most patients experienced ZERO bleeds when treated with ALTUVIIIO²

Patients with zero bleeds*

Patients with zero joint bleeds*

* Based on treated bleeds.²

Effective on-demand bleed management with only 1 infusion²

Treatment of bleeding episodes in the XTEND-1 trial²

96.7% of bleeds* resolved with 1 infusion (n=350/362)

* One 50 IU/kg infusion of ALTUVIIIO (350/362 bleeds). Substantial majority of bleeding events in XTEND-1 (74%, 268/362) occurred during on-demand treatment in the Prior Factor VIII On-Demand Group.¹

† Of the 12 major surgeries, 11 surgeries required a single preoperative dose to maintain hemostasis during surgery; for 1 major surgery during routine prophylaxis, no preoperative loading dose was administered on the day of or before surgery.³

Pain and physical health scores from patient-reported outcomes

In patients on ALTUVIIIO prophylaxis who switched from prior prophylaxis in the XTEND-1 trial¹

PROMIS pain intensity*

HAEM-A-QoL Physical-Health Score in patients ≥17 years of age¹†

  • A lower score represents an overall improvement in these measures

XTEND-1 was a single-arm study, which may impact the interpretation of patient-reported outcome findings. The absence of a comparator arm may result in an under- or overestimation of treatment effect. Patient-reported nature of data may impact the reliability of findings. The PROMIS instrument has not been validated in hemophilia patients.¹

Study design: Patient-reported outcomes of pain intensity and physical functioning were evaluated in patients receiving ALTUVIIIO prophylaxis in the prior Factor VIII prophylaxis group. The PROMIS Pain Intensity 3a instrument was used to assess pain, specifically, the first question that rates the worst pain experienced during the last 7 days. Physical functioning was assessed in patients ≥17 years old using the Physical-Health Score of Haem-A-QoL, which evaluated factors such as painful swellings, presence of joint pain, pain with movement, difficulty walking far, and needing more time to get ready.¹

* Worst pain experienced during the last 7 days (1=no pain; 2=mild pain; 3=moderate pain; 4=severe pain; 5=very severe pain).¹
† LS mean based on mixed-effect model with repeated measures (MMRM) with visit as fixed effect, and baseline score as a covariate.¹

XTEND-1 treatment preference: participant-reported outcomes

Patient preference was evaluated using a 2-item questionnaire on perceived impact of treatment at the end of the study³

XTEND-1 was an open-label, single-arm study, which may impact the interpretation of patient-reported outcome findings. Patient-reported nature of data may impact the reliability of findings. The treatment preference questionnaire used is not a validated instrument.