K Khair1, M G Mazzucconi2, R Parra3, E Santagostino4, D A Tsakiris5, C Hermans6, J Oldenburg7, G Spotts8, K Steinitz-Trost9, A Gringeri9. 1. Haemophilia Comprehensive Care Centre, Great Ormond Street Hospital for Children, NHS Foundation Trust, London, UK. 2. Universita' Roma La Sapienza, Policlinico Umberto I, Rome, Italy. 3. Unidad de Hemofilia-Banc de Sang i Teixits, Hospital Universitari Vall d'Hebron, Barcelona, Spain. 4. Angelo Bianchi Bonomi Haemophilia and Thrombosis Centre, IRCCS Fondazione Ca' Granda, Ospedale Maggiore Policlinico, Milan, Italy. 5. Diagnostic Hematology, University Hospital, Basel, Switzerland. 6. Haemophilia Clinic, Division of Haematology, St-Luc University Hospital, Brussels, Belgium. 7. Institute for Experimental Hematology and Transfusion Medicine, Bonn University Clinic, Bonn, Germany. 8. Shire, Chicago, IL, USA. 9. Shire, Vienna, Austria.
Abstract
INTRODUCTION: Outcome data on treatment of patients with haemophilia A spanning several years of real-world evidence collection are currently very limited. AIM AND METHODS: The global prospective long-term Advate® Haemophilia A Outcome Database (AHEAD) cohort study collects real-world data from patients with severe and moderate haemophilia. We report an interim data read-out after three years of observation. RESULTS: A total of 522 patients were enrolled from 21 countries: 334 completed year 1 follow-up, 238 completed year 2 and 136 completed year 3, with an overall follow-up of 811 patient-years. Median annual bleeding rates (ABR) were 1.7 in the prophylaxis group and 8.9 in the on-demand group at year 1 visit, 1.6 and 13.0, respectively, at year 2 visit and 2.2 and 10.3, respectively, at year 3 visit. Moreover, about 42% of patients on prophylaxis vs 12% of patients on on-demand had zero annual joint bleeding rates (AJBR). Effectiveness of prophylaxis and on-demand treatment was deemed excellent/good in the majority of cases. Octocog alfa (Advate® ) was well tolerated. The inhibitors that developed in nine patients all disappeared spontaneously. Three patients had been previously exposed to FVIII for ≤50 exposure days (EDs), 3 for >50 EDs and 3 showed a borderline positive inhibitory activity (≤0.6 BU/mL). CONCLUSIONS: These data confirm that the goal of zero bleeds is achievable, although not yet achieved in all patients. Understanding reasons behind the lower response to standard prophylaxis regimens in some patients and personalizing prophylactic treatment may further improve outcome in patients with haemophilia A.
INTRODUCTION: Outcome data on treatment of patients with haemophilia A spanning several years of real-world evidence collection are currently very limited. AIM AND METHODS: The global prospective long-term Advate® Haemophilia A Outcome Database (AHEAD) cohort study collects real-world data from patients with severe and moderate haemophilia. We report an interim data read-out after three years of observation. RESULTS: A total of 522 patients were enrolled from 21 countries: 334 completed year 1 follow-up, 238 completed year 2 and 136 completed year 3, with an overall follow-up of 811 patient-years. Median annual bleeding rates (ABR) were 1.7 in the prophylaxis group and 8.9 in the on-demand group at year 1 visit, 1.6 and 13.0, respectively, at year 2 visit and 2.2 and 10.3, respectively, at year 3 visit. Moreover, about 42% of patients on prophylaxis vs 12% of patients on on-demand had zero annual joint bleeding rates (AJBR). Effectiveness of prophylaxis and on-demand treatment was deemed excellent/good in the majority of cases. Octocog alfa (Advate® ) was well tolerated. The inhibitors that developed in nine patients all disappeared spontaneously. Three patients had been previously exposed to FVIII for ≤50 exposure days (EDs), 3 for >50 EDs and 3 showed a borderline positive inhibitory activity (≤0.6 BU/mL). CONCLUSIONS: These data confirm that the goal of zero bleeds is achievable, although not yet achieved in all patients. Understanding reasons behind the lower response to standard prophylaxis regimens in some patients and personalizing prophylactic treatment may further improve outcome in patients with haemophilia A.
Authors: Barbara A Konkle; Doris V Quon; Leslie Raffini; Michael Recht; Vlad C Radulescu; Shannon L Carpenter; Amy L Dunn; Mei Lu; Maureen Watt Journal: J Blood Med Date: 2021-10-14