Antonio Coppola1, Giancarlo Castaman2, Rita C Santoro3, Maria Elisa Mancuso4, Massimo Franchini5, Renato Marino6, Gianna Franca Rivolta1, Cristina Santoro7, Ezio Zanon8, Laura Sciacovelli9, Salvatore Manca10, Riccardo Lubrano11, Maria Golato12, Armando Tripodi13, Angiola Rocino14. 1. Regional Reference Centre for Inherited Bleeding Disorders, University Hospital of Parma, Parma, Italy. 2. Centre for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy. 3. Centre for Haemorrhagic and Thrombotic Disorders, 'Pugliese Ciaccio' Hospital, Catanzaro, Italy. 4. Centre for Thrombotic and Haemorrhagic Diseases, Humanitas Clinical and Research Center - IRCCS, Rozzano, Italy. 5. Department of Haematology and Transfusion Medicine, Carlo Poma Hospital, Mantova, Italy. 6. Haemophilia and Thrombosis Centre, University Hospital of Bari, Bari, Italy. 7. Haematology, Umberto I University Hospital, Rome, Italy. 8. Haemophilia Centre, University Hospital of Padua, Padua, Italy. 9. President of SIBioC; Department of Laboratory Medicine, University Hospital of Padua, Padua, Italy. 10. President of SIMEU; Department of Emergency Medicine and Intensive Care, S. Martino Hospital, Oristano, Italy. 11. President of SIMEUP; Unit of Paediatric Nephrology, Department of Paediatrics, Sapienza University of Rome, Rome, Italy. 12. President of SIPMeL; Unit of Clinical Pathology, 'SS Annunziata' University Hospital, ASL Lanciano-Vasto-Chieti, Chieti, Italy. 13. President of SISET; IRCCS Cà Granda, Ospedale Maggiore Policlinico Foundation, Angelo Bianchi Bonomi Haemophilia and Thrombosis Centre and 'Luigi Villa' Foundation, Milan, Italy. 14. President of AICE; Haemophilia and Thrombosis Centre, Haematology, Ospedale del Mare, ASL Napoli 1 Centro, Naples, Italy.
Abstract
INTRODUCTION: The factor VIII (FVIII)-mimetic bispecific monoclonal antibody, emicizumab, previously approved for prophylaxis in haemophilia A with inhibitors, has been recently licensed in several countries also in patients with severe haemophilia A (PWSHA) without inhibitors. The introduction of this innovative agent requires the development of specific pathways at Haemophilia Treatment Centres (HTC), particularly regarding laboratory testing and treatment of breakthrough bleeds and invasive procedures/surgeries, even more critical when patients are managed by non-specialist professionals. Limited literature data and clinical experience in PWSHA without inhibitors on emicizumab are currently available. AIM: To promote awareness and overcome these challenges, the Italian Association of Haemophilia Centres (AICE) issued a guidance on the management of PWSHA without inhibitors on emicizumab prophylaxis, focused on emergency and shared with other National Scientific Societies in the field. METHODS: The document, drafted by an AICE expert panel and approved through online consultation, was further revised by a multidisciplinary working group, including members of 5 haemostasis, laboratory and emergency scientific societies. The final version was approved by the Council of each society. RESULTS: General recommendations about use of FVIII concentrates for the treatment of bleeding or haemostatic coverage of invasive procedures/surgeries and laboratory monitoring in PWSHA without inhibitors on emicizumab are provided. Specific issues of the management in the emergency room are focused, highlighting the need for direct involvement or formalized supervision by specialist HTC physicians. CONCLUSIONS: This guidance provides a reference pathway to be implemented in the different healthcare organizations, especially for the challenging emergency management in this setting.
INTRODUCTION: The factor VIII (FVIII)-mimetic bispecific monoclonal antibody, emicizumab, previously approved for prophylaxis in haemophilia A with inhibitors, has been recently licensed in several countries also in patients with severe haemophilia A (PWSHA) without inhibitors. The introduction of this innovative agent requires the development of specific pathways at Haemophilia Treatment Centres (HTC), particularly regarding laboratory testing and treatment of breakthrough bleeds and invasive procedures/surgeries, even more critical when patients are managed by non-specialist professionals. Limited literature data and clinical experience in PWSHA without inhibitors on emicizumab are currently available. AIM: To promote awareness and overcome these challenges, the Italian Association of Haemophilia Centres (AICE) issued a guidance on the management of PWSHA without inhibitors on emicizumab prophylaxis, focused on emergency and shared with other National Scientific Societies in the field. METHODS: The document, drafted by an AICE expert panel and approved through online consultation, was further revised by a multidisciplinary working group, including members of 5 haemostasis, laboratory and emergency scientific societies. The final version was approved by the Council of each society. RESULTS: General recommendations about use of FVIII concentrates for the treatment of bleeding or haemostatic coverage of invasive procedures/surgeries and laboratory monitoring in PWSHA without inhibitors on emicizumab are provided. Specific issues of the management in the emergency room are focused, highlighting the need for direct involvement or formalized supervision by specialist HTC physicians. CONCLUSIONS: This guidance provides a reference pathway to be implemented in the different healthcare organizations, especially for the challenging emergency management in this setting.
Authors: Anouk A M T Donners; László Gerencsér; Kim C M van der Elst; Toine C G Egberts; Moniek P M de Maat; Albert Huisman; Rolf T Urbanus; Mohsin El Amrani Journal: Res Pract Thromb Haemost Date: 2022-06-08
Authors: Anouk A M T Donners; Carin M A Rademaker; Lisanne A H Bevers; Alwin D R Huitema; Roger E G Schutgens; Toine C G Egberts; Kathelijn Fischer Journal: Clin Pharmacokinet Date: 2021-08-13 Impact factor: 5.577