Treating for stability: an ethnographic study of aspirations and limitations in haemophilia treatment in Europe

Abstract Background Recent improvements in approaches to treatment have opened a window of opportunity to redefine and expand the goals of treatment in haemophilia This article explores treatment culture in light of these improvements and its potential impact on the range of possibilitis in the lived experience of haemophilia. Aims The aim of this article is to further investigate findings related to how health care professionals (HCPs) approach haemophilia treatment and care, one of the main themes identified in an ethnographic study of the everyday life of people with haemophilia (PwH). This large-scale study investigated PwH's beliefs and experiences related to their condition, their treatment, and their personal ways of managing the condition. Methods The study used ethnographic research methods. Five haemophilia experts helped frame the research design by providing historical and disease area context prior to the initation of field research. In the field, study researchers collected data through 8–12 hours of participant observation, semi-structured interviews, written exercises, facilitated group dialogues, and on-site observations of the interactions of PwH with friends, family, and HCPs. Study researchers also conducted on-site observation at haemophilia treatment centres (HTCs) and interviewed HCPs. The study employed a multi-tiered grounded theory approach and combined data were analysed using techniques such as inductive and deductive analysis, cross-case analysis, challenge mapping, and clustering exercises. This article explores findings related specifically to how HCPs approach haemophilia treatment and care, and is thus focused on a subset of the data from the study. Results Fifty-one PwH in Italy, Germany, Spain, UK, and Ireland were interviewed and followed in their daily lives. Eighteen HCPs from seven HTCs were interviewed, and on-site observation was undertaken at six of the HTCs. Most haematologists in the study ‘treated for stability’, rather than to guide PwH to overcome limitations. ‘Treating for stability’ here refers to an approach to haemophilia care that focuses on measuring success in terms of annual bleed rate, instilling a focus on mitigating risk, rather than an approach that allows PwH to overcome the limitations they face due to their condition. However, some haematologists had moved beyond treating for stability to instead treat for possibilities, enabling a better quality of life for PwH. Conclusions These results suggest that a culture of ‘treating for stability’ could be limiting progress in expanding the goals of treatment in haemophilia. Expanded metrics of success, more flexible approaches to treatment, and higher ambitions on behalf of PwH may be needed in treatment and care, in order for PwH to fully benefit from treatment advances and to increase their quality of life.