Management of patients with severe haemophilia a without inhibitors on prophylaxis with emicizumab: AICE recommendations with focus on emergency in collaboration with SIBioC, SIMEU, SIMEUP, SIPMeL and SISET. Issue 6 (23rd October 2020)

• Record Type: Journal Article
• Authors: Coppola, Antonio; Castaman, Giancarlo; Santoro, Rita C.; Mancuso, Maria Elisa; Franchini, Massimo; Marino, Renato; Rivolta, Gianna Franca; Santoro, Cristina; Zanon, Ezio; Sciacovelli, Laura; Manca, Salvatore; Lubrano, Riccardo; Golato, Maria; Tripodi, Armando; Rocino, Angiola
• Other Names: Bernardini Sergio investigator.; Biasoli Chiara investigator.; Borchiellini Alessandra investigator.; Cultrera Dorina investigator.; De Cristofaro Raimondo investigator.; Di Minno Giovanni investigator.; Gamba Gabriella investigator.; Giampaolo Adele investigator.; Giuffrida Anna C. investigator.; Giuffrida Clemente investigator.; Gresele Paolo investigator.; Mazzucconi Maria G. investigator.; Morfini Massimo investigator.; Hassan Hamisa J. investigator.; Molinari Angelo C. investigator.; Pollio Berardino investigator.; Sargentini Vittorio investigator.; Tagliaferri Annarita investigator.; Tosetto Alberto investigator.; Zampogna Stefania investigator.
• Is Part Of: Haemophilia. Volume 26:Issue 6(2020)
• Page Start: 937
• Page End: 945
• Publication Date: 2020-10-23
• Subjects: bleeding -- emergency -- emicizumab -- FVIII -- haemophilia A -- surgery; Hemophilia -- Periodicals; 616.1572005
• DOI: 10.1111/hae.14172
• Languages: English
• ISSNs: 1351-8216

Abstract

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.