Martin J Scott1,2,3, Hua Xiang1, Daniel P Hart4, Benedict Palmer1, Peter W Collins5, David Stephensen4,6, Camelia S Sima7, Charles R M Hay1,2,8. 1. UK National Haemophilia Database, Manchester, UK. 2. University Department of Clinical Haematology, Manchester Royal Infirmary, Manchester, UK. 3. Institute of Cancer Sciences, Faculty of Biology, Medicine and Health, The University of Manchester, Manchester, UK. 4. The Royal London Hospital Haemophilia Centre, Barts and The London School of Medicine and Dentistry, Queen Mary University London, London, UK. 5. School of Medicine, Cardiff University, University Hospital of Wales, Cardiff, UK. 6. Kent Haemophilia & Thrombosis Centre, East Kent Hospitals University NHS Trust, Canterbury, UK. 7. Roche/Genentech, South San Francisco, California. 8. School of Vascular Medicine, The University of Manchester, Manchester, UK.
Abstract
INTRODUCTION: The THUNDER study provides an analysis of treatment patterns and outcomes in UK patients with severe or moderate haemophilia A (SHA/MHA) in 2015. METHODS: Patients with SHA or MHA registered with the UK National Haemophilia Database (NHD) were segregated by severity, inhibitor status and age. Haemophilia joint health score (HJHS) was derived from NHD records and treatment regimen and annualized bleed/joint-bleed rate (ABR/AJBR) from Haemtrack (HT) in HT-compliant patients. RESULTS: We report 1810 patients with SHA and 864 with MHA. Prophylaxis was used in 94.9% (n = 130/137) of HT-compliant children <12 years with SHA, falling to 74.1% (n = 123/166) aged ≥40 years. Median ABR increased with age (1.0, IQR 0.0-5.0, <12 years; 3.0 IQR, 1.0-8.0, ≥40 years). Inhibitors were present in 159 (8.8%) SHA and 34 (3.9%) MHA. Median ABR increased from 2.0 (<12 years) to 21.0 (≥40 years) in SHA inhibitor patients using prophylaxis. Prophylaxis was used by 68.8% of HT-compliant MHA patients (n = 106) (median FVIII baseline 0.01 IU/mL) associated with a median (IQR) ABR of 3.0 (1.0-7.0). Median HJHS (n = 453) increased with age in SHA and MHA. Median (IQR) HJHS was higher in SHA inhibitor (17.0, 0.0-64.5) than non- or past inhibitor patients (7.0, 0.0-23.0). CONCLUSIONS: Increasing ABR with age persists despite current prophylaxis regimens. SHA and MHA had similar ABR/AJBR and HJHS, leading to a suspicion that a subgroup of MHA may be relatively undertreated. More intensive prophylaxis may improve outcomes, but this requires further study.
INTRODUCTION: The THUNDER study provides an analysis of treatment patterns and outcomes in UK patients with severe or moderate haemophilia A (SHA/MHA) in 2015. METHODS:Patients with SHA or MHA registered with the UK National Haemophilia Database (NHD) were segregated by severity, inhibitor status and age. Haemophilia joint health score (HJHS) was derived from NHD records and treatment regimen and annualized bleed/joint-bleed rate (ABR/AJBR) from Haemtrack (HT) in HT-compliant patients. RESULTS: We report 1810 patients with SHA and 864 with MHA. Prophylaxis was used in 94.9% (n = 130/137) of HT-compliant children <12 years with SHA, falling to 74.1% (n = 123/166) aged ≥40 years. Median ABR increased with age (1.0, IQR 0.0-5.0, <12 years; 3.0 IQR, 1.0-8.0, ≥40 years). Inhibitors were present in 159 (8.8%) SHA and 34 (3.9%) MHA. Median ABR increased from 2.0 (<12 years) to 21.0 (≥40 years) in SHA inhibitor patients using prophylaxis. Prophylaxis was used by 68.8% of HT-compliant MHApatients (n = 106) (median FVIII baseline 0.01 IU/mL) associated with a median (IQR) ABR of 3.0 (1.0-7.0). Median HJHS (n = 453) increased with age in SHA and MHA. Median (IQR) HJHS was higher in SHA inhibitor (17.0, 0.0-64.5) than non- or past inhibitor patients (7.0, 0.0-23.0). CONCLUSIONS: Increasing ABR with age persists despite current prophylaxis regimens. SHA and MHA had similar ABR/AJBR and HJHS, leading to a suspicion that a subgroup of MHA may be relatively undertreated. More intensive prophylaxis may improve outcomes, but this requires further study.
Authors: Erik Berntorp; Kathelijn Fischer; Daniel P Hart; Maria Elisa Mancuso; David Stephensen; Amy D Shapiro; Victor Blanchette Journal: Nat Rev Dis Primers Date: 2021-06-24 Impact factor: 52.329
Authors: Richard A Wilkins; David Stephensen; Heidi Siddle; Martin J Scott; Hua Xiang; Elizabeth Horn; Ben Palmer; Graham J Chapman; Michael Richards; Rebecca Walwyn; Anthony Redmond Journal: BMJ Open Date: 2022-01-12 Impact factor: 2.692
Authors: Erik Berntorp; Petra LeBeau; Margaret V Ragni; Munira Borhany; Yasmina L Abajas; Michael D Tarantino; Katharina Holstein; Stacy E Croteau; Raina Liesner; Cristina Tarango; Manuela Carvalho; Catherine McGuinn; Eva Funding; Christine L Kempton; Christoph Bidlingmaier; Alice Cohen; Johannes Oldenburg; Susan Kearney; Christine Knoll; Philip Kuriakose; Suchitra Acharya; Ulrike M Reiss; Roshni Kulkarni; Michelle Witkop; Stefan Lethagen; Rebecca Krouse; Amy D Shapiro; Jan Astermark Journal: Haemophilia Date: 2022-03-08 Impact factor: 4.263
Authors: Richard A Wilkins; Lara S Chapman; Jenny C Emmel; Thuvia Flannery; Graham J Chapman; Rebecca E A Walwyn; Anthony C Redmond; Heidi J Siddle Journal: Haemophilia Date: 2022-03-04 Impact factor: 4.263