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The only form of haemophilia treatment that is able to prevent arthropathy and other consequences of bleeding symptoms in patients with severe haemophilia is prophylaxis started at an early age (primary prophylaxis). It is also highly beneficial for the psychological and social wellbeing of patients and their families. Scientific institutions and international organizations such as WHO, the World Federation of Hemophilia (WFH) and the National Haemophilia Foundation (NHF) have recommended that prophylaxis be considered optimum therapy. This paper discusses the barriers to prophylaxis, such as the perceived need, costs and availability, and difficulty of venous access, and describes the authors' experiences with the therapy. [ABSTRACT FROM AUTHOR]

Further studies are needed to complete biological assessments of all patients, and a prospective study, particularly on patient evolution with prophylactic treatment, could be of interest. All the patients in our study presented their first joint bleed during childhood, even though spontaneous joint bleeds are unusual in mHB patients.2 In addition, some significant BE may have been underestimated. Four patients had arthrodesis/plasty, from 1 to 4 joint surgeries/patient, and the median age at first arthrodesis/plasty (total joint replacement, hip, knee, elbow) was 37 years (range 25-52 years). This is a retrospective cohort with patients from different French centre with clinical and biological data relying on patient treatment diaries and medical records. [Extracted from the article]

Introduction: The development of an anti‐FVIII inhibitor is the most serious complication of haemophilia A occurring in up to 30% of severe haemophilic patients. The current management of haemophilia A with inhibitor uses bypassing agents (BPA) and represents a significant therapeutic burden together with a limited adherence to prophylactic treatment. Emicizumab is the first monoclonal antibody developed in haemophilia A approved for the prevention of bleeding episodes in patients with anti‐FVIII inhibitor. Aim: The purpose of this study is to evaluate the incremental cost‐effectiveness ratio (ICER) of emicizumab versus BPAs. Methods: A Markov model was developed over a five‐year time horizon to estimate the comparative costs and benefits of the different therapeutic approaches in this rare disease. Model inputs were clinical, including annual bleeding rate and quality of life, and economical including mainly costs of prophylaxis, bleeds and adverse events. Results: Emicizumab treatment is dominant, ie lest costly and more effective, in the base‐case analysis saving 234 191 € for a gain of 0.88 QALY. This is confirmed by both the deterministic and probabilistic sensitivity analyses. The main limit of the study remains the absence of long‐term clinical data allowing to relate treatment consumption to clinical benefit, especially in the progression of haemophilic arthropathy. Conclusion: Our results show that emicizumab is a cost‐effective treatment allowing to consider an easy to implement prophylactic treatment for haemophilia A patients with anti‐FVIII inhibitors. [ABSTRACT FROM AUTHOR]

Abstract Objective This article looks at how users and doctors in France have rethought the question of shared decision-making in the clinical field of haemophilia following a major crisis – that of the infected blood affair. Design We did a qualitative survey based on semi-structured interviews in three regions of France. Setting and participants The interviews covered 31 clinical doctors of haemophilia and 31 users: 21 adult males with severe haemophilia (21/31), infected (14/21) or not (7/21) with HIV, the infected wife of one of the latter (1/31) and nine parents of young patients with severe haemophilia (9/31), either HIV positive (6/9) or negative (3/9). Results and conclusions The results show the infected blood affair to be a major individual and collective ordeal. It has caused users and doctors to rethink their roles within clinical relationships and to develop new ways of sharing medical decision-making. Prior to the crisis, the dominant model was based upon a distinction between the medical aspect, governed by the doctors, and the psychosocial aspect, which involved the patients and their families. Since the crisis, medicoscientific knowledge has been shared between users and doctors. This general trend nevertheless permits the existence of different patient, family and doctor profiles which in turn correspond to different notions of what a clinical decision should be. Some users remain attached to the idea of complementarity between doctors and patients (new partnership model), whilst others put doctors and patients on an equal footing (negotiation model). On the doctors’ side, whilst some still prefer the initial model for therapeutic decision-making, the majority have reassessed their perceptions and viewpoints. A certain number believe that decisions should be made by both doctor and patient in accordance with scientific procedures (decision-making controlled by scientific standards) or regulatory procedures (decision-making controlled by legal standards). Yet others feel that multiple points of view are acceptable within the decision-making process (decision-making model as interactivity). [ABSTRACT FROM AUTHOR]

Summary. Because peculiar profiles for some genetic haematological diseases have been described among Basques, we aimed to investigate the distribution of haemophilia among this specific population. Hence, we retrospectively assessed all the cases of factor (F) VIII and FIX deficiencies seen in the French Basque country during a 16-year period. Data on 41 patients with haemophilia (FVIII or FIX = 25%) were compiled. Incidence and prevalence for the whole population ranged within the classical limits (but with an unusually high A : B ratio) and tended to be slightly lower in autochthonous Basques (P = n.s.). Our data did not support significant differences in the distribution of this disease among French Basques. [ABSTRACT FROM AUTHOR]