PB1126 - Safety and efficacy of simoctocog alfa in patients with haemophilia A in routine clinical practice (GENA-99)

Tuesday, July 12, 2022

6:30 PM – 7:30 PM

Location: Exhibition

Presentation Format: In-Person

Presenting Author(s)

Claire Berger, MD PhD

Hemophilia CRC Hospital, INSERM, U 1059, Jean Monnet University,
Saint-Etienne, Rhone-Alpes, France

Co-Author(s)

MS

Maria Sol Cruz

Director of CEDINOR
Centro Diabetologico del Norte (CEDINOR)
Salta, Salta, Argentina
SC

Silvana Carolina Oliva

Attending physician
PEDIAS INC
Guatemala City, Alta Verapaz, Guatemala
AM

Alicia Molina Pantoja

Haematologist
Teodoro Maldonado Carbo Specialty Hospital
Guayaquil, Guayas, Ecuador
MM

Mary Mathias

Haemophilia Centre Director
Great Ormond Street Hospital for Children, NHS Foundation Trust
London, England, United Kingdom
EZ

Ezio Zanon

Dirigente medico di I Livello
Medicina Generale, Azienda Ospedaliera di Padova
Padova, Veneto, Italy
SS

Sergio Siragusa

Full Professor
AOUP Policlinico Giaccone
Palermo, Sicilia, Italy
BP

Berardino Pollio

SSD Medicina Trasfusionale, Dirigente Medico Ematologo
Regina Margherita Children Hospital Azienda Ospedaliero-Universitaria Città della Salute e della Scienza
Torino, Piemonte, Italy
HG

Heidi Glosli

Medical Doctor
Division of Paediatric and Adolescent Medicine Centre for Rare Disorders Oslo University Hospital Oslo Norway.
Oslo, Oslo, Norway
AB

Angelika Batorova

Head of the Department of Hematology and Transfusion Medicine and National Hemophilia Centre
National Haemophilia Center
Bratislava, Bratislava, Slovakia
Evgeny Dmitriev

Practising Haematologist
Belarussian Research Centre for Paediatric Oncology, Haematology and Immunology
Minsk, Minskaya Voblasts', Belarus
MJ

Maissaa Janbain

Associate Director
Louisiana Center for Bleeding and Clotting Disorders, Tulane University Medical Center, LA, USA
New Orleans, Louisiana, United States
JM

Jorge Martinez

Head of Department of Immunohemotherapy of Center Hospital
University of Cova da Beira, Centro Hospitalar Universitário Cova da Beira, EPE
Colviha, Castelo Branco, Portugal
SS

Sonata Saulyte Trakymiene

Pediatric hematologist/oncologist and Lecturer
Clinic of Children’s diseases, Faculty of Medicine, Vilnius University, Vilnius University Hospital Santaros Klinikos
Vilnius, Vilniaus Apskritis, Lithuania
MP

Miroslav Penka

Head of the Department of Clinical Hematology
Teaching Hospital of Faculty of Medicine of Masaryk University
Brno, Jihomoravsky kraj, Czech Republic
PR

Paolo Radossi

Medical Doctor
Istituto Oncologico Veneto, IRCCS
Castelfranco Veneto, Veneto, Italy
SW

Sylvia Werner

Senior Director Clinical R&D
Octapharma USA
Paramus, New Jersey, United States
Gita Pezeshki, PhD

Associate Director, Clinical Operations
Octapharma USA
Paramus, New Jersey, United States
GD

Giovanni Di Minno

Direttore, Centro di Coordinamento Regionale per le Emocoagulopatie
Azienda Ospedaliera Universitaria "Federico II"
Napoli, Campania, Italy

Background: Clinical studies have demonstrated the efficacy and safety of simoctocog alfa in patients with severe haemophilia A. GENA-99 (NCT02962765) was a prospective, multinational, non-interventional study that assessed the immunogenicity, safety and effectiveness of simoctocog alfa in routine clinical practice.

Aims: To generate real-world data on the use of simoctocog alfa for the routine care of previously treated patients (PTPs) with haemophilia A.

Methods: Male PTPs (≥150 exposure days [EDs] to FVIII) with haemophilia A (FVIII:C ≤2%) of any age/ethnicity were enrolled in 13 countries and followed for 100 EDs. The study was approved by independent ethics committees and institutional review boards, with written informed consent obtained before any study-related activities. Adverse drug reactions (ADRs), including hypersensitivity reactions, FVIII inhibitor development and thromboembolic events, were monitored throughout the study. The effectiveness of simoctocog alfa in prevention of bleeds, and treatment of bleeding episodes (BEs) and surgery was assessed.

Results: Seventy-eight PTPs (Table 1) were enrolled and treated with a total of 14,628,000 IU of simoctocog alfa. No patient experienced an ADR, and no inhibitors were reported. Of the 77 patients who received prophylaxis, 32 (41.6%) patients received a 3x/week regimen, 18 (23.4%) every-other-day, and 17 (22.1%) 2x/week. In 74 patients who received ≥3 months of prophylaxis, mean duration was 10.2 months (maximum 21.2 months). Median ABR was comparable to those in the clinical registration studies GENA-08 and GENA-03 (Table 2). In the 77 patients who received prophylaxis, 246 BEs occurred and were treated with simoctocog alfa. BEs were rated with excellent (67.9%) or good (20.3%) treatment efficacy, for a success rate of 88.2%. Efficacy of all 5 rated surgeries was rated excellent.

Conclusion(s): These data indicate that the long-term safety and efficacy of simoctocog alfa in the real-life clinical setting are comparable to those in clinical registration studies.