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Presentation Details
| Patient- and caregiver-reported outcomes with subcutaneous Mim8 prophylaxis in pediatric patients with hemophilia A with or without factor VIII inhibitors: phase 3 FRONTIER3 study Gili Kenet1, Matteo Luciani2, Paula Kjöllerström3, Rubén Berrueco4, Amalie Rhode Høgh Nielsen5, Jay Jay Thaung Zaw5, Emily Waters6, Karin Fijn van Draat7, Ryan Rodriguez6. 1National Hemophilia Center and Institute of Thrombosis & Hemostasis, Sheba Medical Center, Tel Aviv, Israel.2Onco-Hematology, Cell and Gene Therapy and Bone Marrow Transplant Clinic Area, Bambino Gesù Children’s Hospital, Rome, Italy.3Pediatric Hematology Unit, Hospital Dona Estefânia, ULS São José, Lisbon, Portugal.4Pediatric Hematology Department, Hospital Sant Joan de Déu, Universitat de Barcelona, Barcelona, Spain.5Novo Nordisk A/S, Søborg, Denmark.6Novo Nordisk Inc., Plainsboro, NJ, USA.7Department of Paediatric Haematology, Emma Children’s Hospital, Amsterdam UMC, University of Amsterdam, Amsterdam, Netherlands |
Abstract
Background: The burden of hemophilia A (HA) and its treatment can impair the quality of life (QoL) of pediatric patients and their caregivers. Mim8 (denecimig) is an activated factor VIII mimetic bispecific antibody under investigation in clinical trials for subcutaneous prophylaxis (PPX) in HA. Objectives: This analysis from the phase 3, open-label FRONTIER3 study (NCT05306418) assessed the impact of Mim8 once-every-week (QW) PPX on treatment preference, treatment burden, and QoL in pediatric patients. Methods: Pediatric patients aged 1–11 years, with HA with or without inhibitors, of any severity, who received Mim8 QW PPX for 26 weeks and responded to questionnaires at baseline and Week 26, were included in this analysis. This study assessed treatment preference of caregivers using the Caregiver Hemophilia Patient Preference Questionnaire (Caregiver H-PPQ), caregiver-reported treatment burden of all patients using the Child Hemophilia Treatment Experience Measure (Hemo-TEM) v1.0 (lower score indicates less treatment burden), and change in health-related QoL using the Pediatric QoL Inventory (PedsQL) v4.0 for patients aged 5–11 years (higher score indicates better health-related QoL). Results: Seventy patients (36 aged 1–5 years; 34 aged 6–11 years) completed 26-week treatment with Mim8 QW; 60 (86%) had severe HA. At baseline, 41 patients (59%) had previously received PPX with factor VIII or bypassing agent, 25 (36%) were treated on-demand, 2 (3%) were untreated, and 2 (3%) had immune tolerance induction. At Week 26, 60/70 caregivers had responded; most (n=59/60, 98%) preferred Mim8 to prior treatment, a subgroup of 44/60 (73%) very strongly preferred Mim8. With Mim8 QW PPX, treatment burden mean total score (Child Hemo-TEM) was 32.0 at baseline and 15.8 at Week 26; mean (standard deviation [SD]) change: –16.2 (24.0). Mean physical functioning score (PedsQL) was 77.8 at baseline and 89.0 at Week 26; mean (SD) change: 11.1 (19.1). QoL mean total score (PedsQL) and scores for emotional, school and social functioning domains also increased from baseline to Week 26. Conclusions: Changes in patient- and caregiver-reported outcomes suggest Mim8 has the potential to reduce treatment burden and improve physical functioning and QoL of children with HA. Previously presented at the 18th Annual European Association for Haemophilia and Allied Disorders (EAHAD) Congress, Milan, Italy, 4–7 February 2025.
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