Reflections on Haemophilia

Experts from KHP Haematology’s hub at Waterloo share their experiences of diagnosing and treating patients from across the region, and the latest cross-partnership research into the disease.

blood testDr Pu-Lin Luo and Dr Gerry Dolan, Consultant Haematologists

It is an exciting time in world of haemophilia and the treatment landscape is ever evolving. We are proud as a Haemostasis Centre to be able to offer patients several new treatments both as standard of care and also as part of clinical trials.

Not only can are we able to offer standard and extended half-life coagulation factor replacements for patients with haemophilia, we can now provide emicizumab, a monoclonal antibody for patients with severe haemophilia A. This has significantly reduced the treatment burden for patients as they no longer need to have intravenous coagulation factor injections two-three times a week. Instead emicizumab can be delivered subcutaneously once a week or once a fortnight or even once a month.

Gene therapy has been developed which offers the potential of cure for patients with haemophilia A and B. Guy’s and St Thomas’ NHS Foundation Trust (NHS FT) has been one of the leading UK centres involved in gene therapy trials.

In addition gene therapy, patient with haemophilia A can also participate in clinical trials in other novel monoclonal antibody therapy, anti-Tissue Factor Inhibitor and anti-thrombin inhibitors.

There has been a remarkable improvement in haemophilia care over the past decade and we are privileged to be able embark on this journey with our patients.

Steve Classey, Physiotherapist

As the team physiotherapist, I have the pleasure of helping our inspiring patients to manage the acute bleeds and chronic joint changes that often occur as a result of this debilitating disease. 

Great progress has been made in the management of this disease over the last few decades and our younger patients thankfully do not suffer from the same levels of joint disease. However, it is also exciting to be involved in the research of drugs and gene therapy that will potentially eradicate pathological bleeding in patents with haemophilia within the next couple of decades.

Dr Jayanthi Alamelu, Clinical Lead for Paediatric Haemophilia

A new diagnosis of haemophilia can be a very challenging time for the family. Despite excellent treatment options now available for haemophilia, it can still be a daunting and uncertain period and parents will have lots of questions.

At Evelina London Children’s Hospital, we have a multidisciplinary team of professionals to support the family throughout the child’s journey. Along with the adult haemophilia centre next door at St Thomas’ Hospital, we provide comprehensive care to all patients with bleeding disorders.

World Federation of Haemophilia and the British Society of Haematology guidelines recommend prophylaxis (regular treatment) as the Standard of Care for those with severe and moderate haemophilia. With the commencement of early prophylaxis our patients enjoy excellent joint outcomes and improved quality of life.

As we mark World Haemophilia Day 2023, it is satisfying to acknowledge that in addition to standard factor concentrates which have been available for many decades, we are able to provide patients with newer therapeutic options like extended half-life factor concentrates and non-factor therapy prophylaxis. As the hub of the South London Paediatric Haemophilia Network, we aim to provide equitable care to all patients with haemophilia across the South London Paediatric Network so they have access to the latest therapies.

The world of haemophilia has seen an impressive shift to newer treatment options and exciting new therapies being trialled. Evelina Haemophilia Centre has been in the forefront of being able to offer our patients the option to participate in some of this latest research and early access programmes that have resulted in a landscape shift in haemophilia care and patient experience but at the same time helped answer vital research questions.

Children and families with chronic conditions build solid rapport with their paediatric teams over the years and we see this with our patients and families too. We have created a robust transition pathway to facilitate a seamless transfer of the adolescent patients to the adult haemophilia centre at St Thomas’ Hospital. Being co-located at the same site, we are unique in being able to enable a gradual transfer of care of the young person to the adult service over a couple of years and modifying this based on individual need.

Clare James, Paediatric Haemophilia Clinical Nurse Specialist

The role of a paediatric haemophilia nurse is crucial in providing comprehensive care to children and adolescents with haemophilia and other bleeding disorders. Paediatric haemophilia nurses are speciality trained to support and manage the unique needs of children and adolescents with a bleeding disorder. This includes:

  • Assessment and Education: The role includes conducting thorough assessments of children with haemophilia, including their physical, emotional, and psychosocial well-being. From diagnosis through to transition to adult care, we provide education and counselling to patients and their families on the condition, its management, and the importance of adhering to treatment plans.

  • Treatment Management: We play a vital role in coordinating and managing the treatment plans for children with haemophilia. The haemophilia nurse organises training of parents and children to administer their medication either via portacath, directly into their veins or to give sub cutaneous injections. This training is given both in the hospital and home setting to facilitate parents and children to manage independently at home. 

  • Support and Counselling: The impact of a diagnosis of haemophilia requires emotional and psychological support in addition to medical management . The haemophilia CNS helps patients and families cope with the challenges of living with a chronic bleeding disorder by quickly addressing concerns, providing coping strategies, and connecting families with appropriate resources for support.

  • Research and Education: Haemophilia nurses at Evelina London Children’s Hospital have direct involvement in the clinical trials and quality improvement projects with an aim to improve patient outcomes.

Trupti Bhandari, Paediatric Haemophilia Physiotherapist

The role of the physiotherapist within the care of a child/person with haemophilia is vital. At the Evelina, we provide a service from diagnosis to 18 years of age where we see the children for acute joint bleeds and review assessments. Bleeds can affect the cartilage within the joints and over time this leads to arthropathy and can prevent people with haemophilia staying active into adulthood.

It is important during an acute joint bleed to provide support, advice, guidance and exercises to help them to return to normal function as quickly as possible. This requires daily adjustment of exercises to ensure that full range of movement and muscle strength returns to allow the affected joint to be protected from further bleeds.

In review clinics we monitor growth, development and conduct joint assessments using the haemophilia joint health score (HJHS) and point of care ultrasound (HEAD-US). These are a vital part of haemophilia care, as the child grows and develops it is important for the joints and muscles to be optimal for all the activities they want to do. I call it an ‘MOT’ for their body, to make sure they can continue to do all the activities they love without harm.

Education around how to recognise joint bleeds and to seek out assistance from their haemophilia centre is crucial to ensure that bleeds are dealt with in a timely manner to minimise any damage caused to the joint and the surrounding muscles.

King’s Health Partners Haematology brings together our strengths in clinical service, research, and education to deliver exceptional outcomes for haematology patients