Abstract Number: PB0599
Meeting: ISTH 2021 Congress
Theme: Hemophilia and Rare Bleeding Disorders » Hemophilia - Clinical
Background: Haemophilia A (HA) is a disorder characterized by deficiency in clotting FVIII that affects daily lives of patients and caregivers. Emicizumab is a humanized, bispecific, monoclonal antibody, which substitutes missing activated FVIII.
Aims: Effectiveness and safety on emicizumab in clinical practice.
Methods: Hemolife study initiated in March 2020, describes the socio-occupational impact of HA without inhibitors in patients and caregivers, in terms of quality of life, activity, and productivity/absenteeism, among others. At that time, both COVID-19 pandemic and emicizumab approval for PwHA without inhibitors happened.
A multidisciplinary Scientific Committee of experts in HA, rethought Hemolife study to include a cohort of patients treated with emicizumab (Hemolution), resulting in a more complete and stronger RWE study, and avoiding logistical duplications that a separate study would mean to centers and patients.
2020 has been a year for adaptation, efficiency and re-design; also, in RWE context.
100 PwHA will be followed for 12 months within Hemolife. At any time, a patient prescribed with emicizumab may be enrolled in Hemolution cohort, followed for at least 8 months after initiation of emicizumab to collect efficiency and safety data (Figure 1). 
Figure 1
Finally, Hemolife involves telemedicine; in a pandemic setting, Hemolution could also benefit from this live data collection and may shape a new and more dynamic form of care in HA that avoids being in the hospital.
Results: Ongoing study. Protocol was submitted and approved by authorities/ethics committee’s (Table 1).
| Region | Hospital |
| Andalucía | Hospital Universitario Virgen del Rocío |
| Hospital Regional Universitario de Málaga | |
| Hospital Puerta del Mar de Cádiz | |
| Hospital Universitario Reina Sofía | |
| Hospital del SAS Jerez de la Frontera Hospital Virgen de las Nieves |
|
| Aragón | Hospital Universitario Miguel Servet |
| Asturias | Hospital Universitario Central de Asturias |
| Castilla La Mancha & Castilla León | Hospital Universitario General de Toledo |
| Hospital Universitario de Guadalajara | |
| Hospital del Río Hortega | |
| Hospital Universitario de Salamanca | |
| Cataluña | Hospital de la Santa Creu i Sant Pau |
| Hospital Sant Joan de Déu Barcelona Hospital Universitario Vall d’Hebron |
|
| Galicia | CHUAC – Complexo Hospitalario Universitario A Coruña |
| Complejo Hospitalario Universitario de Vigo | |
| Islas Baleares & Islas Canarias | Hospital Universitari Son Espases Hospital Universitario de Gran Canaria Doctor Negrín |
| Madrid | Hospital Universitario La Paz |
| Navarra | Complejo Hospitalario de Navarra |
Table 1
Conclusions: By this novel approach we will gather information on emicizumab in clinical practice and provide relevant information to the Haemophilia community, contributing to improve patient care in HA.
To cite this abstract in AMA style:
Álvarez Román MT, Soto I, de la Corte H, Bosh P, Lavandeira Á, Alvarez C, Guerra I. Prospective, Non-interventional Study with a Cohort to Assess Real-world Effectiveness (RWE) and Safety of Emicizumab in Persons with Hemophilia A (PwHA) without Inhibitors in Spain, Hemolution Cohort [abstract]. Res Pract Thromb Haemost. 2021; 5 (Suppl 2). https://abstracts.isth.org/abstract/prospective-non-interventional-study-with-a-cohort-to-assess-real-world-effectiveness-rwe-and-safety-of-emicizumab-in-persons-with-hemophilia-a-pwha-without-inhibitors-in-spain-hemolution-cohort/. Accessed October 1, 2023.« Back to ISTH 2021 Congress
ISTH Congress Abstracts - https://abstracts.isth.org/abstract/prospective-non-interventional-study-with-a-cohort-to-assess-real-world-effectiveness-rwe-and-safety-of-emicizumab-in-persons-with-hemophilia-a-pwha-without-inhibitors-in-spain-hemolution-cohort/