Kate Khair

Haemnet · Research

Background: Qualitative studies have explored why people with haemophilia (PwH) might consider or forgo gene therapy, the impact it has had on those who have received it and what support might be needed throughout the process. No studies have yet examined what withdrawal prior to transfection might mean for PwH and their families. Aims: To underst...

Introduction: The "problem joint" (PJ) concept was developed to address patient-centric needs for a more holistic assessment of joint morbidity for people with haemophilia (PwH). Aim: To quantify the humanistic burden of PJs in PwH to further support validation of the PJ outcome measure. Methods: Multivariable regression models evaluated the r...

Introduction: The use of virtual interventions is of interest to patients with chronic disease and healthcare professionals. This study aimed to determine the effect of virtual child disease management programme on burden and social adjustment of caregivers of children with coagulation factor deficiencies. Moreover, the effect of this intervention...

Introduction: Gene therapy has the potential to change the life experience of people with haemophilia and their families. A growing number of studies have examined the experience for those who have had gene therapy. A few studies have examined the process with other gene therapy among a wider cross-section of the haemophilia community. Exigency is...

Background Gene therapy has the potential to change the life experience of people with haemophilia and family members. Few studies have sought to explore the impact of gene therapy on both individuals and families. The aim of this study was to capture real-life experiences of gene therapy in People with haemophilia and their families. Results Sixt...

Background: Research studies have described the morbidity associated with inherited bleeding disorders such as hemophilia and von Willebrand disease in women, but their effect on daily living has long been underrecognized. This systematic review sought to document the lived experience of women with a bleeding disorder by assessing research finding...

Introduction: Despite therapeutic advances in bleeding disorder treatment, over the past 20 years women with bleeding disorders have reported delayed diagnosis, impaired quality of life, dismissive attitudes from health professionals and inappropriate care. Aim: To explore the lived experience of women with a diagnosed bleeding disorder and to i...

Purpose To explore the experiences, views and beliefs of people with severe haemophilia and healthcare professionals (HCPs) on approaches for pain management, as well as their views on exercise being used as an aspect of management. Methods Taking a qualitative inquiry approach using focus groups and semi-structured interviews, participants includ...

Purpose To explore the life experiences of pain in people with severe haemophilia and understand how such experiences influence beliefs and sensation of pain in adulthood. Methods A qualitative inquiry approach using focus groups and semi-structured individual interviews was used. Participants included people with severe haemophilia living with ch...

Background: People with haemophilia A and inhibitors (PwHi) suffer more orthopaedic complications, bleeding and pain than those without inhibitors. The advent of emicizumab as a prophylactic treatment has led to a reduction in bleed frequency and a significant in overall quality of life. No research to date has examined the nature of this improvem...

Introduction Many young men with hemophilia engage in physical activity and sport but face challenges to participation because of their hemophilia. Project GYM aimed to investigate the feasibility of a hemophilia-specific fitness program led by a personal trainer (PT) and its impact on gym activity, motivation, and adherence to exercise. Methods T...

Introduction Contemporary hemophilia care supports physical activity, its benefits being well recognized. Despite recognition of the psychological challenges encountered by people with hemophilia, little is known about the psychological impact of physical fitness in this population. Aim To identify changes in psychological well-being in young men...

Women who have the gene variant for haemophilia are labelled solely as ‘carriers’ unless they have a factor VIII activity of ≤40%. This term, which describes an individual who can pass on a disorder but are themselves unaffected, reflects a legacy that extends from the 18th century to the present day. There is strong evidence that women labelled as...

Introduction: Neutralizing antibodies to coagulation factor VIII (FVIII) remain a major complication associated with FVIII replacement therapy. Aim: To assess safety and efficacy of immune tolerance induction (ITI) therapy with ADVATE® (antihemophilic factor [recombinant] [rAHF]) in patients who participated in the Prospective ADVATE Immune Tole...

Hemophilia A and von Willebrand disease (VWD) are inherited rare bleeding disorders affecting normal hemostasis. Patients with VWD, especially those with severe disease types, share some similarities to patients with hemophilia A in their burden of disease: they suffer from an increased risk of potentially severe and life-threatening bleeds and ass...

Introduction Despite increasing awareness of issues faced by women and girls with inherited BDs (WGBD), standards of care are lacking, with disparities in diagnosis and treatment for WGBD across Europe. We aimed to develop practical principles of care (PoC) to promote standardization of care for WGBD within European Haemophilia Treatment and Compre...

Gene therapy for haemophilia has the potential to change the life experience of individuals and their families. Few studies at this time have sought to explore the impact of gene therapy on individuals and their families. The aim of the Exigency study is to capture the real-life experience of gene therapy in people with haemophilia and their famili...

Compared with people with haemophilia A, people with haemophilia A and inhibitors (PwHi) have been disproportionately affected by their condition, suffering more orthopaedic complications, bleeding and pain. The advent of emicizumab as a prophylactic treatment has led to a reduction in bleed frequency and a significant improvement in overall qualit...

Introduction For many people with haemophilia (PwH) gene therapy offers a potential functional cure. However, some have stated that they do not wish to have gene therapy either now or in the future. Aim This sub-study, part of the larger Exigency programme, assesses the attitudes, views and understanding of those who do not wish to undergo gene th...

Background The efficacy and safety of wilate (human von Willebrand factor/coagulation factor VIII) in patients with von Willebrand disease (VWD) has been demonstrated in clinical trials. Here, we present real-world data on the use of wilate for the routine care of patients with VWD. Objectives The objectives of this observational, prospective, phas...

Aim To share our experience of implementing a programme of interventions aimed at building research capacity and capability of nurses and allied health professionals in a specialist children's hospital. Background Clinicians at the forefront of care are well positioned to lead on research to improve outcomes and experiences of patients but some pr...

Background and Aims Despite advances in haemophilia care, inhibitor development remains a significant complication. Although viable treatment options exist, there is some divergence of opinion in the appropriate standard approach to care and goals of treatment. The aim of this study was to assess consensus on United Kingdom (UK) standard of care fo...

Introduction The paediatric Haemophilia Activities List (pedHAL) was developed to measure activities and participation in children and youth with haemophilia. Results from international studies provide an opportunity to determine which items are universally important. Aim The aim of this study was to determine which items of the pedHAL are redunda...

Introduction Pain is recognised as a subjective phenomenon, often defined as ‘whatever the experiencing person says it is, existing whenever the experiencing person says it does’. Pain is a critical aspect of life for many people with haemophilia (PWH) but is under-recognised and inconsistently managed by clinicians. As haemophilia management moves...

Introduction People with haemophilia (PwH) experience recurrent bleeds in weight-bearing joints. Optimal management for people with severe haemophilia involves prophylaxis with factor VIII or IX, which helps to reduce the risk of bleeds and joint damage. However, this is expensive and frequently not an option in economically developing countries, w...

Abstract Introduction Gene therapy is used in life‐limiting conditions of childhood. While not a current therapeutic option for children with haemophilia, it may be considered in the future especially for those where access to treatment is limited. Aim To assess the attitudes and opinions of parents of children with haemophilia about gene therapy...

Introduction: Long-term effectiveness and safety data in patients treated in routine clinical practice settings can be captured from real-world studies. The international (INT) and German (GER) Antihemophilic factor (recombinant; rAHF) Hemophilia A (HA) outcome Database (AHEAD) studies assess long-term effectiveness and safety outcomes in patients...

Introduction: Hemophilia A (HA) is a congenital bleeding disorder, caused by a deficiency in clotting factor VIII (FVIII) and characterized by uncontrolled bleeding and progressive joint damage. This analysis assesses the impact of disease burden on the daily life of children with hemophilia A (CwHA) and their caregivers, addressing a deficit of cu...

Introduction Severe hemophilia (FVIII/FIX level <1%) is characterized by spontaneous hemarthrosis leading to progressive joint deterioration and chronic pain in the affected individual. Unless these recurrent hemarthroses can be prevented, e.g. with the use of prophylactic factor replacement therapy, these patients will develop chronic synovitis, p...

Introduction: Hemophilia A (HA) is a congenital bleeding disorder caused by a deficiency in clotting factor VIII (FVIII). There are currently limited data on the impact of HA on daily life. Here we examine the impact of HA on the daily life of adult persons with HA (PwHA) without current FVIII inhibitors according to disease severity. Methods: The...

Background Some studies suggest that people with haemophilia (PwH) who use prophylaxis value low frequency of clotting factor administration more than a lower risk of bleeding. However, more frequent infusions offer the potential of reducing joint disease and pain, which in turn may improve functioning and quality of life. Aims To explore the impa...

Introduction Hemophilia A is a bleeding disorder traditionally managed with standard half-life (SHL) factor (F) VIII concentrates. Extended half-life (EHL) FVIII products and emicizumab-kywh, a nonfactor therapy, are newer treatment options. Additional nonfactor agents and gene therapy are expected to reach the market in the near future. Areas cov...

Haemophilia nurses in the UK are instrumental in supporting people with haemophilia in self-management, including managing treatment options, recording treatment use and understanding the budgetary impact of prescribing practice. The widespread use of prophylaxis identified haemophilia as a high cost disorder to treat, resulting in a financially su...

Management of haemophilia A requires administration of factor VIII therapy which, for those with severe haemophilia A in the UK, is predominantly self-administered at home in a prophylactic regimen to prevent or minimise bleeding. The UK undertakes a national tendering process every three years to ensure access to current and new therapies at a cos...

Purpose Approximately 35%‐50% of people with haemophilia (PWH) report living with chronic musculoskeletal pain. Although exercise based rehabilitation is effective for pain in other arthritises, there are no published guidelines for management of chronic pain in PWH. This review aims to evaluate and appraise the current evidence of effectiveness of...

Introduction Nurses play a central co‐ordinating role in delivering comprehensive care for people with haemophilia and allied bleeding disorders, for which they need a broad range of competencies. The UK Haemophilia Nurses Association (HNA) published a role description in 1994 which was developed into a competency framework in 2014. This has now be...

Young adults with long-term health conditions (LTHCs) are increasing due to improvements in care, therapy regimens and medical technology. In the UK, transition to adult services occurs at a stage when they are also learning self-management skills. For some, transfer from paediatric to adult care providers may result in a loss of psychosocial suppo...

Aims and objectives: To describe the perceived burden on parents of children with severe or moderate haemophilia and the impact of sociodemographic aspects and the child's medical condition on this. Background: Parents of children with haemophilia face a multitude of demands. The child needs frequent intravenous injections, hospital visits, extr...

Introduction: Inherited bleeding disorders are rare, for some such as haemophilia, there is a growing body of evidence about caregiver burden and associated support needs. Whilst for other disorders, which may be present with less clinically significant bleeding, there is a paucity of data about parents support needs. Aims: To evaluate the suppo...

Introduction Extended half‐life (EHL) clotting factors have been shown to offer people with haemophilia (PwH) protection from bleeding with fewer infusions, which might reduce treatment burden. Aim The HOw Patients view Extended half‐life products (HOPE) study aimed to explore, understand and describe patient expectations around the prophylactic u...

Spirituality plays an important role in coping with chronic diseases. However, the meaning of spirituality is not known in hemophilia, as a chronic disease. This study aimed to explore the essence of spirituality in hemophilia patients. This qualitative study with a hermeneutic phenomenological approach was conducted on twelve Muslim adult hemophil...

Haemophilia is an inherited bleeding disorder associated with a reduction or absence of coagulation factor VIII or IX. In severe haemophilia, recurrent, spontaneous bleeding occurs into joints, without treatment this leads to crippling joint deformity. Haemophilia is an X-linked disorder yet there is no family history in approximately one third of...

Introduction: Caring for a child with haemophilia is burdensome and impacting on caregivers' emotional and financial status. This paper assesses the impact of psychosocial determinants on caregivers' burden across European countries. Methods: This non-interventional study enrolled caregiver/child dyads at haemophilia treatment centres (HTCs) usi...

Introduction Treatment burden for the people with haemophilia has been documented, as has the burden of caring for a child with a common chronic disease such as asthma or diabetes. However, there remains a paucity of data about caregiver burden in haemophilia. Aims The aim of this study was to evaluate the impact of bleeding on caregivers of child...

Haemophilia impacts on the person who has it as well as his close family and friends. The majority of healthcare provider focus is with the person with haemophilia and his carers during childhood, and then on the person himself as he becomes able to self-manage. There is a belief that the family and healthcare team support the patient equally. In t...

Transition is the term used to describe the process of approaching and crossing the chronological boundary between paediatric and adult care services. Transfer of care describes the administrative arrangements associated with moving from a paediatric to an adult service across this boundary. Transforming Transition was a nurse-led initiative design...

Introduction and Objective: Inhibitors to coagulation factor VIII (FVIII) are the most serious complication of haemophilia A treatment. Previously untreated patients (PUPs) are at the greatest risk of inhibitors, which generally occur within the first 20 exposure days (ED) to FVIII. Immune tolerance induction (ITI) is the only clinically proven str...

Background The need for a more integrated, multidisciplinary approach to care for individuals with bleeding or clotting disorders has been highlighted in recent years. Evidence‐based education adapted to nurses’ needs is essential for a successful evolution. However, limited data currently exist on the clinical challenges nurses face in this specia...

Background Pain management can prevent long-term burdens in haemophilia patients and improve their quality of life. The present study aimed to describe and interpret pain experiences in haemophilia patients, focusing on pain self-management in their lives. Methods This was a qualitative study undertaken using a hermeneutic phenomenological approac...

Overweight and obesity may carry a significant disease burden for patients with haemophilia (PWH), who experience reduced mobility due to joint inflammation, muscle dysfunction and haemophilic arthropathy. This review aimed to define the prevalence and clinical impact of overweight/obesity in the global population of PWH. A detailed literature sear...

Introduction: Contemporary haemophilia management recommends sport and physical activity in children with haemophilia. Assessment of subjective physical functioning requires standardized and validated instruments. Aims: To adapt and psychometrically test the adult Haemophilia & Exercise Project-Test-Questionnaire (HEP-Test-Q) for children (aged...

The development of high‐titre inhibitory antibodies (inhibitors) against factor VIII (FVIII) remains a challenge in the management of patients with haemophilia A (HA). Patients with high‐titre inhibitors are more likely to experience uncontrolled bleeding, physical disability from chronic arthropathy and premature death compared with those without...

Management of haemophilia involves on-demand or prophylactic intravenous administration of recombinant or plasma-derived replacement clotting factors or bypassing agents. These products are provided as lyophilised powder and diluent, which need to be mixed to produce a solution for infusion. While this process has previously involved multiple time-...

Long-standing inhibitors present many day-today difficulties for the affected individual; the unpredictability of bleeds, bleed management, pain and treatment efficacy all affect quality of life. This study explored these issues through focus groups of affected individuals aged 16-25 in the UK. The data from the focus groups was analysed for recurr...

Hemophilia causes bleeding that may affect patients’ psychological aspects and quality of life. This study aims to evaluate the impact of psychological aspects and pain on health-related quality of life (HRQoL) in adult patients with hemophilia. This cross-sectional study was conducted on 103 patients with hemophilia. Data were collected using hemo...

Introduction: Outcome data on treatment of patients with haemophilia A spanning several years of real-world evidence collection are currently very limited. Aim and methods: The global prospective long-term Advate(®) Haemophilia A Outcome Database (AHEAD) cohort study collects real-world data from patients with severe and moderate haemophilia. We...

Introduction: Contemporary haemophilia care demands Patient-Reported Outcomes. SO-FIT is a UK multi-centre study, assessing self-reported function, health-related quality of life (HRQoL) and joint health in boys with severe haemophilia. Methods: Subjective physical function (PedHAL, HEP-Test-Q) and HRQoL (Haemo-QoL Short Form [SF]) were assessed...

Introduction: Central venous access devices facilitate home treatment in boys with haemophilia. These are usually fully implanted lines, referred to as ports. Caregivers are taught to manage the port using sterile techniques and maintaining patency by flushing with saline or heparin solution. National and international guidelines for the home care...

Haemophilia caregivers face limitations in their life leading to perceived burden. This single-centre study investigates the impact of burden on caregivers’ health-related quality of life (HRQoL). Methods: Questionnaires for caregivers comprised demographic data, HRQoL (EQ-5D, SF-36) and caregiver burden (IOF: Impact on Family Scale). Children were...

The terms ‘nurse-led service’ and ‘nurse-led care’ are often used, but are frequently not well defined. As health care delivery evolves across the world and struggles to cope with changes in medical care and with the rising number of people living longer with long term conditions, it is frequently suggested that more care should be delivered and co...

Prophylaxis for hemophilia A with conventional factor VIII (FVIII) products requires frequent intravenous dosing, which may reduce adherence. Recombinant factor VIII Fc fusion protein (rFVIIIFc) has a prolonged half-life compared with conventional rFVIII, and has demonstrated safety and efficacy for the prevention and treatment of bleeding episodes...

Introduction and Objective Prospective surveillance studies are a useful tool to monitor drug safety and efficacy in the post-approval period and provide treaters and caregivers with real-life information about von Willebrand disease (VWD) treatment. This ongoing study aims to demonstrate consistency of safety and efficacy data of a VWF/FVIII conce...

Introduction: Long-term, real-world data on natural history of hemophilia A patients, safety and treatment outcome are still insufficient, particularly as far as the impact of bleeding on patient lives is concerned, as most of clinical trials and PASS studies are limited in study population size and length of follow-up, often no longer than 12 mont...

Background: Pain, as a crucial subsequence of joint hemorrhages in hemophilia patients, is chronic, debilitating, and distracting. This study aimed to describe and interpret pain experiences of hemophilia patients in their lives. Methods: This qualitative study with hermeneutic phenomenological approach was conducted on fourteen hemophilia patient...

Haemophilia nursing roles continue to develop alongside nursing as a profession. There are now nurses who practice autonomously, much like a medical practitioner, and many who have extended their roles to deliver direct patient care, education and research. There has been little, if any, comparison with haemophilia nurse roles internationally, nor...

Wider access to modern treatment of haemophilia has led to a growing interest in the family’s role in management. An increasing amount of research has sought to understand the psychosocial impact of living with a child with haemophilia. Understanding how such demands affect parents and families who live with the daily threat of bleeding can help he...

Introduction: Intracranial haemorrhage (ICH) is the most serious bleeding event for patients with inherited bleeding disorders (IBD). The risks and long-term consequences remain unknown. Aim: This single-centre service evaluation aimed to identify the incidence, risks and long-term outcomes following ICH in patients with IBD. Methods: The IBD...

Background: There is increasing recognition that sport is important for individuals with haemophilia; however, there remains a paucity of data of the importance of this in adults, many of whom already have joint pathology related to childhood bleeds and treatment access. This multicentre, cross-sectional study presents the impact of sport on healt...

Pain is a major problem in haemophilia patients’ lives. The perspective of pain in such patients is unique and may be different from other chronic illnesses. This qualitative hermeneutic phenomenological study aims to describe and interpret pain experience of haemophilia patients. Participants were selected from a haemophilia clinic affiliated with...

Prophylactic coagulation factor replacement is increasingly the treatment modality of choice for people with haemophilia (PWH). Currently available recombinant factor products require reconstitution from a lyophilised powder and diluent, and a range of infusion systems is available to assist in this process. This study aimed to understand the prope...

Pain is a major clinical problem in patients with bleeding disorders. This study aimed to determine the level of pain and how it is managed in children and adolescents with bleeding disorders. This cross-s