More data on the clinical outcomes of emicizumab and other treatment options would guide the appropriate positioning of these agents among haemophilia management approaches. The purpose of this study was to review the clinical outcomes of emicizumab prophylaxis and other treatment options in children and adults with severe haemophilia A (HA) at Sheikh Khalifa Medical City (SKMC) and Tawam Hospital in the United Arab Emirates (UAE). This retrospective study consisted of charts review of all individuals with HA diagnosed and treated with emicizumab and other treatment options at both centres between January 2019 and December 2023. The primary endpoint was annualised bleeding rate (ABR) from baseline. Secondary endpoints included the presence of FVIII inhibitors, joint bleed episodes, health-related quality of life (HRQoL), need for rescue medication, and Emergency Department visits at Month 12 after switching to emicizumab. All persons were males; mean age was 16.8±11.4 years. Mean duration of emicizumab therapy was 11±2.3 months. The ABR decreased significantly after therapy initiation (2.93±2.48 versus 0.0±0.0; p<0.001). None of the persons required rescue medication during emicizumab therapy (p<0.001) and FVIII inhibitors significantly decreased (p<0.001). After emicizumab therapy, number of Emergency Department visits decreased significantly (3.3±2.8 versus 0.1±0.3; p <0.001), no joint bleed episodes had been reported (p<0.001) and HRQoL index scores were significantly improved (p<0.001). Emicizumab was associated with improved clinical outcomes in terms of ABR, joint bleeding episodes, use of rescue medications, and FVIII inhibitors level. Emicizumab prophylaxis also improved HRQoL.
Concizumab's potential – A subcutaneous TFPI inhibitor for haemophilia, offering an alternative to intravenous therapySafety profile – Meta-analysis of RCTs shows no significant increase in adverse events compared to standard treatmentsSerious adverse events – Lower, though not statistically significant, risk in the concizumab groupBleeding reduction – Potential to reduce joint bleeding episodes, enhancing haemophilia managementFuture research – Larger, long-term studies are needed to confirm safety and efficacy
Persons with haemophilia (PwH) have historically been advised to avoid physical activity (PA) to limit bleeding risk, leading many to adopt sedentary lifestyles. The paradigm shift in treatment for PwH toward prophylaxis with extended half-life factor concentrates and non-factor replacement therapy has substantially reduced joint bleeding and deterioration, allowing PwH to lead more active lives with improved protection against bleeds. This has led to increased recognition of the importance of regular PA for musculoskeletal strength, joint and cardiovascular health, quality of life, and social/psychological well-being among PwH. Nonetheless, hesitation regarding risks of PA for PwH persists, and key clinical questions including haemostatic protection for safe participation in PA remain largely unanswered. This narrative review summarises the considerations for PA in PwH and its role in haemophilia management with associated recommendations from key organisations, focusing on shared decision-making and individualised treatment planning. Key strategies to increase PA among PwH include promoting sustained behavioural change and potential use of wearable activity trackers. Higher haemostatic potential is consistently associated with protection against PA-related bleeds. An increasing evidence base indicates that both factor and non-factor therapies can safely enhance physical function and support greater PA, although additional evidence is needed for novel and emerging treatments. Further research is needed to help clinicians individualise prophylaxis regimens in a holistic manner that includes consideration of PA. Recommendations for PA in PwH must continue to progress alongside the evolving therapeutic landscape.
With correct diagnosis and treatment, women with bleeding disorders can live their lives without the pressure of having heavy menstrual bleeding (HMB) and the concern of postpartum bleeding, thus improving quality of life (QoL). As health care professionals (HCPs), we have the opportunity to advise, support and educate our patients, guiding them to optimal treatment and care.
Greater treatment choice and the associated flexibility in regimens has necessitated new conversations with people with haemophilia about their personal goals. Innovations in haemophilia treatment drive the need for innovation in care service delivery. Supported by Sobi™, CATCH (Collaborate & Address Treatment Challenges in Haemophilia) is an annual, non-promotional, medical education meeting that brings together members of the haemophilia multidisciplinary team from across the UK and Ireland to discuss all aspects of haemophilia care and management. This report summarises the key issues that were explored and discussed during the CATCH 2024 meeting, including understanding the personal motivations of people with haemophilia and how these influence treatment choices and support; facilitating behaviour change to improve outcomes; reviewing the practicalities and possibilities for eliminating bleeds and new arthropathy in people with haemophilia; and developing new models of care to elevate haemophilia services.
Acquired haemophilia A (AHA) is a rare autoimmune bleeding disorder characterized by the formation of inhibitory antibodies against endogenous Factor VIII (FVIII), interfering with its function. Though a rare disorder, with around 1.5 cases per million, it is more prevalent among the elderly. There is a wide phenotype of bleed presentation, and nearly half of patients will have an underlying malignancy or autoimmune disorder. Mortality is estimated to be more than 20% in patients aged over 65 years.
Stakeholder-led organisations play a critical role in bridging gaps in care and advocacy. Understanding the lived experiences and contributions of core facilitators within these communities is essential to strengthen their impact.
The Contaminated Blood Scandal (CBS) infected many people with haemophilia (PwH) with blood-borne diseases. While studies have focused on those directly affected, little is known about the intergenerational impact on their children and siblings. Many adult children were subject to multiple adverse childhood experiences (ACEs) as relatives became unwell or died, experiencing isolation, stigma, grief and loss, from both society and healthcare professionals (HCPs). These experiences occurred within broader ecological systems that compounded their impact across individual, family, community, and societal levels.
Emicizumab is a humanised, bispecific monoclonal antibody that bridges activated factor (F) IX and FX; it is approved for treatment of people with haemophilia A (PwHA) of all ages, with or without FVIII inhibitors. Qualitative experiences of PwHA switching to non-factor treatments, such as emicizumab, are needed to facilitate shared decision-making (SDM).
For people with haemophilia, surgical procedures, whether haemophilia-related or for other conditions, are associated with a risk of bleeding. Factor activity levels must be maintained for any invasive intervention. Clinical studies have shown that extended half-life (EHL) recombinant factor products are beneficial in this context. Clinical nurse specialists working in comprehensive care centres play a central role in preparing patients for surgery and in coordinating the surgical process. This case series reports on the nurse experience of using EHL-FIX products in surgery in a small UK cohort of people with haemophilia B (PwHB).
Emicizumab has recently been investigated for prophylactic use in patients with acquired haemophilia A. While it shows promising results in effectively reducing bleeding episodes, there remains a very delicate question concerning the risk of thromboembolic events. Recently published prospective studies reported thrombotic events in 4% to 8% of cases. However, these studies excluded patients with an increased thrombotic risk or those undergoing treatment for a thromboembolic disease, situations frequently occurring in this elderly patient group. Therefore, we urge clinicians to critically assess which patients would benefit from emicizumab treatment and which would not.
Haemophilia is an inherited bleeding disorder requiring regular factor replacement therapy, often necessitating frequent hospital visits. Home therapy (HT) has emerged as a viable alternative, improving patient independence and adherence. This study evaluates the impact of a structured home therapy intervention at Assam Medical College and Hospital on treatment adherence, self-infusion rates, and quality of life among haemophilia patients.
Occult hepatitis B virus (HBV) infection (OBI) is defined as the presence of HBV DNA in the absence of HBV surface antigen (HBsAg) serological markers. Despite the high prevalence of HBV infection in many communities, OBI prevalence among people with haemophilia (PWH) remains poorly characterised in South Africa.
Haemophilia is an inherited bleeding disorder characterised by a deficiency in coagulation factors, leading to an increased risk of bleeding, including after procedures such as immunisation. While immunisation is crucial for preventing infectious diseases, it poses a bleeding risk in people with haemophilia, particularly when administered intramuscularly.
Haemophilia A and B (HA/HB) are congenital, X-linked recessive bleeding disorders caused by deficiency of clotting factor VIII (FVIII) or IX (FIX), respectively. People with haemophilia (PwH) have increased risk of spontaneous or traumatic bleeding in joints, muscles, or soft tissues, which can be severe in people with HA/HB with inhibitors (HAwI/HBwI). Despite advances in haemophilia treatment, there are remaining and emerging unmet needs in PwH.
Haemophilia carriers (HCs) face psychosocial challenges, including emotional distress, health concerns, and pregnancy-related issues. Understanding their needs is crucial for optimal healthcare support. This study examined the emotional impact on haemophilia carriers (HC).
Historically people with haemophilia B (PwHB) have experienced adverse clinical outcomes including joint bleeds, pain and arthropathy. The current management of severe and moderate haemophilia B in the UK is largely through the use of factor replacement therapy often given as prophylaxis. Despite this, research reveals reduced quality of life (QoL) and psychological burden in PwHB of all severities, highlighting unmet need. Contemporary therapeutic developments including extended half-life factor IX products, novel non-factor agents, and gene therapy offer opportunities to address treatment-related issues and improve QoL. We investigated what PwHB identify as meaningful when considering their personal goals and the potential for gene therapy.
Data on clinical characteristics and bleeding outcome of Omani children with haemophilia A (HA) is limited. This study aims to describe the clinical profile and outcome of Omani children with HA at the Royal Hospital from 2006 to 2019.
Severe haemophilia A is associated with serious spontaneous bleeding in muscles, soft tissues, and joints. Haemarthrosis, bleeding into joints, is a serious complication of haemophilia. Emicizumab is a novel non-factor replacement agent for preventing or reducing the frequency of bleeding episodes in adults and children with haemophilia A with or without inhibitors.
Prophylaxis is the global standard of care for haemophilia A (HA), and its adoption has been accelerated by wide use of emicizumab prophylaxis globally. Reports on the prophylaxis in people with haemophilia living in Africa are limited.
