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Since June 2021 in France, patients with haemophilia A with anti-factor VIII inhibitors and patients with severe haemophilia A without anti-factor VIII inhibitors have the choice between the community and the hospital pharmacy for dispensing emicizumab (Hemlibra®). This study aims to investigate patient-centred access to treatment by evaluating and comparing the dimensions of the Penchansky and Thomas model, between community and hospital pharmacies. The evaluation of access to treatment was based on the dimensions of the Penchansky and Thomas model: accessibility, availability, acceptability, accommodation and affordability. These were assessed using appropriate and specific indicators in the study context and calculated for patients choosing community pharmacy or hospital pharmacy for emicizumab dispensing. Geographical data collected as part of the national 'PASO DOBLE DEMI' study were used for this analysis. The findings reveal that dispensing emicizumab in community pharmacies improves accessibility by significantly reducing travel time. The availability of healthcare services is also improved due to the territorial coverage of community pharmacies. The extended opening hours and low waiting time also contribute to better access to emicizumab in community pharmacies. The dimension of acceptability must be improved, and further investigations are needed to address the affordability dimension. Several months after emicizumab became available in French community pharmacies, access to treatment has significantly improved, reducing the burden of this rare chronic disease for patients and their careers. These results suggest that this innovative model could be extended to other medicines and even other therapeutic areas. [ABSTRACT FROM AUTHOR]

Summary: Acquired haemophilia A (AHA) is a rare haemorrhagic disease characterised by new‐onset haemorrhagic symptoms associated with a dramatic decrease in factor VIII levels and an anti‐factor VIII neutralising autoantibody concentration >0.6 Bethesda units. Elderly people are often affected, whereas children are rarely affected; the paediatric incidence reported in the literature is about 0.045 case/million/year. For some time, the paediatric standard of care has been that for adults, but clinicians have often reported poor outcomes. Here, we describe the largest retrospective paediatric AHA cohort assembled to date, including eight patients diagnosed in France from 2000 to 2020. [ABSTRACT FROM AUTHOR]

Efanesoctocog alfa (ALTUVIIIOTM; [antihemophilic factor (recombinant), Fc-VWF-XTEN fusion protein-ehtl]), a von Willebrand factor (VWF) independent, recombinant DNA-derived Factor VIII (FVIII) concentrate, has been developed by Bioverativ Therapeutics, Inc (a Sanofi company) and Swedish Orphan Biovitrum AB (Sobi). Efanesoctocog alfa was approved in February 2023 in the USA for use in adults and children with hemophilia A (congenital FVIII deficiency) for: routine prophylaxis to reduce the frequency of bleeding episodes; on-demand treatment and control of bleeding episodes; perioperative management of bleeding. This article summarizes the milestones in the development of efanesoctocog alfa leading to this first approval for hemophilia A. [ABSTRACT FROM AUTHOR]

During several decades, patients with haemophilia have been treated using FVIII and FIX concentrates by haematologists working in specialized comprehensive haemophilia care centres. In the last fifteen years haemophilia treatments have been tremendously improved and novel treatment options have been developed. Gene therapy is one of these innovative approaches and will soon be available on the market. The European Association for Haemophilia and Allied Disorders and the European Haemophilia Consortium recently described a new healthcare setting for the management of patients who will receive gene therapy. This hub-and-spoke model aims to ensure the safe administration and monitoring of gene therapy by expert comprehensive haemophilia care centres, in close collaboration with the proximity haemophilia treatment centres where the patient is followed up. Any adverse events should be managed by both the expert "hubs" in collaboration with the spoke centre to provide the timeliest state-of-the-art treatment and management options to patients receiving gene therapy. All adverse events and efficacy data should be reported lifelong to a centralised registry. French reference centre for haemophilia and the patient's organization AFH worked together to adapt the European Hub and spoke model to the existing infrastructures and organization for rare bleeding disorders in France. This review describes the haemophilia patient's journey for gene therapy in France. [ABSTRACT FROM AUTHOR]

Background: Nonacog alfa, a standard half‐life recombinant factor IX (FIX), is used as a prophylactic treatment in severe haemophilia B (SHB) patients. Its half‐life determined in clinical studies involving a limited sampling (72 h) was shown to be rather short. In our clinical practice, we suspected that its half‐life could have been underestimated. Objectives: We aimed to evaluate nonacog alfa pharmacokinetics in real world clinical practice based on FIX levels in patients receiving prophylaxis. Methods: We retrospectively collected data on patients with SHB receiving prophylaxis from eight centres across France. The terminal half‐life (THL), time to reach 5–2 IU/dl and FIX activity at 48, 72 and 96 h were derived by Bayesian estimations using NONMEM analysis. Results and conclusions: Infusion data (n = 455) were collected from 64 patients with SHB. The median THL measured in 92 pharmacokinetic (PK) studies was 43.4 h. In 26 patients ≤12 years of age, 51 PK studies showed a median time to reach 5 IU/dl of FIX of 70.5 h and a median time to reach 2 IU/dl of 121.5 h. In 38 patients 13–75 years of age, 41 PK studies showed a median time to reach 5 IU/dl of FIX of 92.0 h and a median time to reach 2 IU/dl of 167.5 h. Extending the sampling beyond 72 h makes it possible to observe a plateau, with FIX remaining between 2 and 5 IU/dl for several days and shows that the THL of nonacog alfa might be longer than previously described. Essentials: Nonacog alfa terminal half‐life (THL) in patients receiving regular prophylaxis was evaluated in clinical practice.The median THL was estimated to be 36.9 h for patients aged.8–12 years.The median THL was estimated to be 49.9 h for patients aged 13–75 years.For patients aged ≤12 and >12 years, the median times to reach 5 IU/dl were 70.5 and 92 h, respectively; to reach 3 IU/dl, 95.5 and 131.5 h, respectively; to reach 2 IU/dl, 121.5 and 167.5 h, respectively.We suggest that the half‐life of nonacog alfa might be longer than previously described in both younger and older patients. [ABSTRACT FROM AUTHOR]

What is known and objective: In older patients, multiple chronic conditions lead to the intake of multiple medications and a higher risk of adverse drug events. The exposure to inappropriate medications in older patients with bleeding disorders is poorly explored. The aim of this study was to describe the exposure to potentially inappropriate medications (PIMs) and medications with anticholinergic and sedative properties in older community‐dwelling patients with haemophilia or von Willebrand Disease (VWD). Methods: The M'HEMORRH‐AGE study (Medication in AGEd patients with HAEMORRHagic disease) is a multicentre prospective observational study. Community‐dwelling patients over 65 years with haemophilia or VWD were included in the study. PIMs were identified using the EU(7)‐PIM list, and the anticholinergic and sedative drug exposure was measured using the Drug Burden Index. Results and discussion: 142 older community‐dwelling patients with haemophilia (n = 89) or VWD (n = 53) were included (mean age: 72.8 ± 5.8 years). PIMs were used by 45.8% of older patients and were mainly represented by cardiovascular (34.9%), nervous systems (26.7%) and alimentary tract and metabolism PIMs (25.6%). Regarding anticholinergic and/or sedative medications, 37.3% of older patients were exposed mainly due to nervous system medications (68.3%), for example analgesics. What is new and conclusion: The M'HEMORRH‐AGE study showed the exposure to PIMs and anticholinergic/sedative medications was high in older community‐dwelling patients with haemophilia or VWD. Interventions focusing on deprescription of these inappropriate medications should be conducted in this specific population. [ABSTRACT FROM AUTHOR]

Acquired hemophilia (AH) is a rare, serious bleeding disorder most often associated with older age and life-threatening complications. The patient care pathway for AH is complex because of the different types of bleeding, the presence of comorbidities, and the heterogeneity of medical specialists who care for these patients. This observational study used the French national PMSI (Programme de médicalisation des systèmes d'information) database to characterize patients with AH in real-life practice and analyze their hospital pathway. In total, 180 patients with AH were identified over a 5-year study period (January 2010 to December 2014), based on three criteria: bypassing agent use, International Classification of Diseases, 10th revision code allocation, and aged over 65 years. Comparison of the incidence rate of AH versus registry data validated the PMSI as an epidemiological database. Rituximab was prescribed more often (60/180; 33.3%) than expected following guidelines and was associated in half of cases to early infections (32/60; 53.3%), surgery procedures were frequently performed during the year before AH onset (29/159; 18.2%), which may suggest a triggering effect, extended hospital stays (median: 20 days) and mortality remaining high (66/180; 36.7%) that occurred mainly during the first month after AH diagnosis. Median costs and number of injections were comparable between recombinant activated factor VII and plasma-derived activated prothrombin complex concentrate. These findings could inform future medico-economic approaches in this AH population (duration of stays, bypassing agents, rituximab use, comorbidities, hospitalizations with infections). [ABSTRACT FROM AUTHOR]

Objective: The Hemraude study was conducted to describe the profile of patients with HA, disease management, and economic burden in a collective perspective. Methods: This retrospective study was conducted using the French administrative healthcare claims database SNIIRAM/SNDS. Male patients treated for hemophilia A with a long-term illness (ALD) status or invalidity were included in the study between January 1, 2016 and December 31, 2017. Patients were classified in six treatment groups: no treatment, on-demand FVIII, prophylactic FVIII, FVIII in immune tolerance induction (ITI) protocol, on-demand bypassing agents, and prophylactic bypassing agents. Patients treated with FVIII in ITI protocol and those treated with bypassing agents are deemed to have developed inhibitors. HA patients were compared to a control population without coagulation disorder and matched (ratio 1:3) on age and sex. Results: A total of 4172 patients were included in the analysis, aged on average 35.2 years, 5.3% had HIV infection, and 8.8% had hepatitis B or C. In 2017, half of the patients received no treatment for HA, 46.7% were treated with FVIII (25% on demand, 20.6% with prophylaxis, and 1.1% ITI), 1.5% with bypassing agents. Patients treated with prophylactic treatments, either inhibitor or non-inhibitor, were less likely to be hospitalized for severe bleeding compared to patients receiving on-demand treatments. The average annual costs for HA management per patient were 72,209.60 €. The highest costs were observed in patients treated with FVIII in ITI protocol and those receiving prophylactic bypassing agents. Conclusion: Direct costs of HA treatments for HA may be very high especially in the small percentage of patients developing inhibitors or treated with ITI protocol. [ABSTRACT FROM AUTHOR]

Background: New therapies provide a favorable evolution in the care management of persons with hemophilia. However, the impact of these new therapies on patient care organization remains to be determined. A qualitative study will be implemented to analyze patients' perception regarding the impact of innovation on the organization of their care management. Secondary objectives will include refining specific factors related to persons with hemophilia (barriers or facilitators, especially the place of treatment) to consider within an organizational impact analysis. Patients and Methods: Semi-structured individual interviews will be conducted via videoconferencing or by phone by two researchers using an interview guide. Participants will be recruited from the Rhône-Alpes region, in France. Physicians from two hemophilia treatment centers will identify eligible patients. Moreover, a call for volunteers will be launched by the Rhône-Alpes committee of the French hemophilia association. Interviews will be conducted with adult patients, adolescent patients or parents of a minor with hemophilia regularly treated prophylactically or on demand. Data analysis will be performed with NVivo® software. Each interview will be analyzed by two researchers using an inductive content analytic method. Discussion: The INNOVHEMO study is an original study analyzing the way patients perceive the impact of an innovation on their care management organization. The resulting patient-specific factors, identified as barriers or facilitators, will need to be integrated into a more comprehensive analysis of the impact of innovation on care management organization. [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: Lightening the injection burden is commonly believed to improve prophylaxis adherence. Efmoroctocog alfa (rFVIIIFc) is the first recombinant FVIII‐Fc fusion protein available in France. This clotting factor with an extended half‐life could thus improve medication adherence. Aim: The study primarily aimed to assess the real‐life impact on prophylaxis adherence of haemophilia A patients, when switching from a standard to an extended half‐life FVIII. Methods: This study was an observational, monocentre, non‐interventional study aiming at assessing haemophilia A patients' real‐life adherence during the first‐year post‐rFVIIIFc prophylaxis initiation. Medication adherence was assessed using two methods: the medication possession ratio (MPR), which is based on the hospital pharmacy dispensing data, and self‐reported VERITAS‐Pro® questionnaire. Patients on rFVIIIFc prophylaxis for at least 12 months, following a 12‐month standard FVIII prophylaxis, were eligible for inclusion. Results: In 2019, 47 male patients were undergoing rFVIIIFc prophylaxis in our Hemophilia Center, among which 36 meeting the inclusion criteria. Switching from standard to extended half‐life FVIII prophylaxis resulted in increased mean dosing, while the mean number of weekly prophylactic injections (2.6 ± 0.5 vs 1.8 ± 0.3) decreased. Following rFVIIIFc initiation, a non‐significant increase in median MPR occurred and the self‐reported VERITAS‐Pro® questionnaire demonstrated improved adherence to rFVIIIFc prophylaxis. Comparing adherent and non‐adherent patients revealed age as the only factor likely to impact adherence (p =.07). Conclusion: Our patient cohort exhibited high adherence levels before and after FVIII switching, based on MPR and VERITAS‐Pro® questionnaire. The latter is likely a useful tool to quantity prophylaxis adherence from a patient's perspective in daily use. [ABSTRACT FROM AUTHOR]

Background: Hemophilic arthropathy is a major complication in patients with severe hemophilia. A plastic knee model has been developed for the therapeutic education of patients to promote improved care management and self-treatment skills. The objective of this study was to evaluate the impact of this hemarthrosis-simulating artificial knee (HSAK) on patients' knowledge of their disease and its treatment. Methods: In this observational study, the impact of HSAK was assessed during individualized education in patients with severe/moderately severe hemophilia A or B at seven hemophilia treatment centers in France. Participants provided written informed consent and completed questionnaires to assess knowledge of their disease (score range: 0– 7) and knowledge of their treatment (score range: 0– 4). Questionnaires were completed before, immediately after and 6 months after HSAK use. The scores obtained before and after the use of the HSAK were compared. Results: The participants comprised 32 children, 29 teenagers, and 31 adults. The mean (SD) disease knowledge score increased significantly in all age groups of patients from 4.5 (2.0) to 5.9 (1.5; p< 0.001) immediately after the training and remained unchanged at 6 months. Mean (SD) treatment knowledge scores were unchanged, but Wilcoxon signed rank testing showed a significant increase after the training course that was maintained at 6 months in children and teenagers. Conclusion: These findings suggest that an individualized training course can enhance the understanding of hemophilia in patients of all ages, especially in children and teenagers, and that the HSAK may assist in improving patients' management of their disease. [ABSTRACT FROM AUTHOR]

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]

Objectives: In France, only hospital pharmacies can dispense clotting factor concentrates to persons with hemophilia, which limits the access to care for the treatment and the prevention of bleeding episodes. Moreover, the cost of clotting factor concentrates may restrain the maintenance of sufficient stocks in hospital pharmacies. The aim of this study was to investigate the accessibility of clotting factor concentrates to persons with hemophilia in the context of long-term prophylaxis and emergency treatment in the Rhone-Alpes region of France.Methods: A geographic information system was used for evaluating accessibility of clotting factor concentrates. Persons with hemophilia and hospital pharmacies were geolocalized with the use of postal data, and the evaluation of accessibility was based on the road network.Results: Approximately 72% of the study area was accessible in less than 30 minutes to a hospital pharmacy. Eighty-five percent of persons with hemophilia had access to clotting factor concentrates for prophylactic treatment in less than 20 minutes. Most of them were patients with severe or moderate hemophilia. Regarding emergency doses, factor VIII was accessible in less than 30 minutes in 45.6% of the study area, and factor IX in 30.5%.Conclusion: This study highlights that spatial access to clotting factor concentrates by persons with hemophilia in the Rhône-Alpes region is good for prophylactic treatment but is more uneven for emergency doses. [ABSTRACT FROM AUTHOR]

Background: Replacement therapy in haemophilia plays an important role in the effective management of this rare bleeding disorder and requires a high level of knowledge and practical skills.Objective: To evaluate and to compare the knowledge and skills about the medicines and their management among people with haemophilia treated with clotting factor concentrates, and their informal caregivers.Setting: Eight Hospital Pharmacies working closely with Haemophilia Care Centres in France. Method In this cross-sectional and multi-centre study, 26-item questionnaire was specifically developed to assess the knowledge and skills. Face-to-face interviews using a questionnaire were conducted with patients and caregivers.Main Outcome Measure: Level of knowledge and skills about disease, treatment and medication management. Results This study included 80 patients and 55 caregivers. Although most interviewees had basic knowledge of the treatment, only 43.7 % knew the effect of clotting factor concentrates on the haemostasis process. Similarly, only 12.7 % of the patients and 30.9 % of the caregivers referred to inhibitors as adverse effects. Despite intensive training for home treatment, 55.7 % reported difficulties with reconstitution or injection. The knowledge required for the responsible management of their medications had not been totally acquired: only 17 participants were indeed familiar with the medication storage conditions; 29 patients and 9 caregivers had already experienced an emergency which they had to treat with no medicine available at home; and 47.4 % of participants had already thrown away an unused drug vial.Conclusion: This study shows the need of a regular update and to renew awareness of haemophilia treatment specificities among these populations. The identified needs suggest that we should more invest in educational techniques or therapeutic education programs more focused on medication management. [ABSTRACT FROM AUTHOR]

Although extremely rare, acquired haemophilia A (AHA) can cause severe bleeding, which may be fatal. The underlying causes of autoantibody development are not fully understood. Treatment goals are bleeding control and autoantibody eradication. At the time of our study, there was no consensus on a standard treatment strategy for AHA. Previous data were mainly retrospective or from single-centre cohorts. We conducted a prospective, controlled, registry-based study of patients with AHA in France. The prospective French registry (Surveillance des Auto antiCorps au cours de l'Hémophilie Acquise [SACHA]) collected data on prevalence, clinical course, disease associations and outcomes for haemostatic treatment and autoantibody eradication in 82 patients with a 1-year follow-up. Similar to earlier studies, the prevalence of AHA was higher in the elderly, with two thirds of patients aged >70 years. Around half of AHA cases were associated with underlying disease, most commonly autoimmune disease and cancer in younger and older patients respectively. Haemostatic treatment was initially administered to 46% of patients. Complete resolution or improvement of initial bleeding occurred in 22/27 (81%) rFVIIa-treated patients and in all six cases receiving pd- aPCC. The majority of patients (94%) received immunosuppressive therapy, with complete remission at 3 months in 61% (36/59) and in 98% (50/51) at 1 year. Overall mortality was 33%: secondary to bleeding in only three patients but to sepsis in 10. Bypassing agents were effective at controlling bleeding in patients with AHA. Immunosuppressive therapy should be used early but with caution, particularly in elderly patients. [ABSTRACT FROM AUTHOR]

. Deaths occurring in the context of acquired haemophilia (AH) may be related to inter-connected causes and mechanisms including bleeding, specific or older patient co-morbidities or iatrogenic complications. However, their magnitude remains unknown. This study aimed to determine the respective weight and frequency of the various causes of death in AH. Multiple-cause analysis based on death certificates data is used in this purpose. Over a 10-year period (2000-2009), 121 deaths with AH as a cause were registered in France. All the deaths were of adults (extremes: 47 and 99 years; mean age: 80.7 years). The average number of causes per death certificate was 4.7. AH was the underlying cause of death (UCD) in 69.4% of the cases, and was more frequent in the older subjects. In contrast, before age of 75 years, AH was more often a contributing cause of death. No postpartum or obvious thromboembolism-related deaths were registered. Haemorrhagic shock was the most frequent direct cause of death (DCD), followed by infectious events, cardiac dysfunction, metabolic and nutritional disorders with muscle wasting and decubitus complications, and cancers (52.9%, 26.4%, 7.5%, 5.8% and 4.1%, respectively). However, when AH was not reported as an UCD, infections become the first DCD (32.4%) followed by bleeding events (16.2%). Best prophylactic and curative strategies for infections are particularly required to improve the prognosis in AH. Moreover, as several of its DCD correspond also to steroids side effects, best tolerated immunosuppressant regimen with steroid-sparing agents adjoining are particularly awaited in AH population. [ABSTRACT FROM AUTHOR]

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Intracranial haemorrhage (ICH) is known to be a severe although uncommon complication of haemophilia. A national survey has been conducted in France in order to collect information about ICHs which occurred in haemophiliacs between 1991 and 2001 and to propose recommendations for the diagnostic and treatment of ICH. Within this period, 123 episodes of ICH were recorded from 106 patients. Two-thirds of ICH concerned patients with severe haemophilia. Half of the cases occurred in patients under 15 years of age, 67.2% of which were post-traumatic. Ten cases occurred in neonates with three fatal outcomes. Overall mortality was high (21.9%) suggesting that availability of clotting factor concentrates has not improved the prognosis of this event. Morbidity was also high with 60% of long-term sequelae. The following parameters have been identified as prognostic factors for death: thrombocytopenia, HCV infection, intraventricular or intraparenchymatous haemorrhage. A delay in diagnosis was mentioned in 43.3% of cases, often related to the lack of recognition of the initial symptoms, which may be very common (apathy, tearfulness in young children and headache in elder patients). Delayed replacement therapy was recorded in 37.2% of cases. Emergency units initially dealt with half of these patients. Information concerning recognition and management of these episodes, not only in severe haemophilia, but also in moderate and mild forms, should be regularly supplied to paediatricians in maternity and physicians from emergency units, as well as to patients and their relatives. [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]

Summary. Replacement therapy in haemophiliacs has a major economic impact on health establishments. We assessed in this prospective study the cost of clotting factor concentrate therapy for haemophilia A or B patients. We compared the overall costs of treated patients with or without inhibitors. In six French haemophilia centres, 278 consecutive hospitalizations were collected and analysed between June 97 and June 99. Haemophilia must be considered as the main cost factor during hospitalization. The severity of bleeds and surgical procedures increase the total cost. Furthermore, the daily and total costs are closely linked to the presence or the absence of inhibitors. This study should enable the hospital administration to evaluate the necessary resources to the clotting factor therapy in haemophiliacs with or without inhibitors during hospitalization. [ABSTRACT FROM AUTHOR]

In patients with severe haemophilia receiving clotting factor concentrates, the risk of immunisation against their usual treatment is still patent and feared. New haemophilia drug treatments with an extended half-life have become available over the past few years. The risk of inhibitor development to these new treatments is unclear. We report the case of a 51-year-old man with severe haemophilia A, who was previously treated with no history of inhibitor development. Soon after a switch in his treatment to efmoroctocog alfa he developed an inhibitor against this recombinant Fc fusion extended half-life FVIII (rFc-FVIII) product. The patient was on an on-demand treatment regimen and was treated for mucosal bleeding. The inhibitor was characterised as type I, with classical epitope mapping. The spontaneous evolution of this inhibitor was favourable, but an anamnestic response led to a switch in his treatment to emicizumab. [ABSTRACT FROM AUTHOR]

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]

The development of an anti-FVIII inhibitor is the most serious complication of haemophilia A. Since February 7 SP th sp 2019, emicizumab is reimbursed in France for the prevention of bleeding episodes in patients with haemophilia A with FVIII inhibitor. Over 5 years, the mean total cost per patient was 2,3 M€ in patients treated with emicizumab, versus 2,6 M€ with BPA, associated to 3,3 and 2,4 QALYs respectively. [Extracted from the article]

Objectives: Hemophilia is a rare genetic disease, characterized by uncontrolled bleeding. Injections of clotting factor are the principal are the principal treatment. This drug is very expensive. The objectives of this study are to determine the cost of clotting factor for in patients and the factor impacting this cost., Methods: A retrospective study was carried on hemophiliac in patients between 1 January 2014 and 31 December 2015 in Cochin hospital and having received at least an injection of clotting factor during their hospitalization. A collection of clinical data and treatments received during the hospitalization was realized for every patient., Results: Fifty-one patients were included in the study with a total of 68 hospitalizations. The median cost of clotting factors by hospitalization was 16,908€. The median part of clotting factors on the total cost of the hospitalization was 68.2%. The cost of factors by stay was higher for the severe haemophiliacs (P=0.015) and for the major surgeries (P<0.0001). The daily median cost of clotting factors was 3124€. This cost was higher at the haemophiliacs B (P=0.0112), the severe haemophiliacs (P<0.0001) and the haemophiliacs with inhibitor (P=0.0053)., Conclusions: Clotting factors represent the most part of the cost of hospitalization of a haemophiliac. Their cost in hospitalization varies according to many factors. It may evolve with the arrival of long-acting clotting factors., (Copyright © 2018 Académie Nationale de Pharmacie. Published by Elsevier Masson SAS. All rights reserved.)

Until 1990, congenital hemorrhagic disorders were treated by plasma-derived concentrates. The first recombinant drug, recombinant factor VIII was available after this date and few years later recombinant factor IX could also be proposed to patients. The evolution of market share in France was different between these two drugs: while recombinant factor VIII took a large place in hemophilia A treatment (85%), plasma-derived factor IX represent 50% of the French market. In the next years, the arrival of long-acting antihemophilic factors may lead to the dramatically reduce the amount of plasma-derived antihemophilic factors used to treat hemophilia. For rare bleeding coagulation disorders, plasma-derived concentrates are still widely used, while they are the only concentrates available in most diseases. This situation is unlikely to evolve significantly in the next years., (Copyright © 2015 Elsevier Masson SAS. All rights reserved.)