A special bond
The Royal Marsden has been pioneering stem cell transplants in the treatment of blood cancers for more than 45 years, and the specialist team at our Oak Centre for Children and Young People supports more than 20 patients and their families through the intense process each year.
Fifteen year old sisters Kareece and Kameeka Stone-Small have a special connection that surpasses even that of being twins. Four years ago, Kareece was diagnosed with acute lymphoblastic leukaemia (ALL). But after two years of treatment at The Royal Marsden, and just as the girls were about to start secondary school, their mum Lorette was given the devastating news that Kareece had relapsed and would need a stem cell transplant (SCT).
Fortunately, the family had been tested when Kareece was diagnosed, and Kameeka was already known to be a sibling match. Lorette says: “The team had already tested all of the family just in case, so we could move quite quickly. In a way, we were the lucky ones as I knew Kameeka was a good match. Not everyone has that, which must be awful. I am forever grateful to her because without her, we would not have Kareece.”
Their story is just one example of The Royal Marsden’s world-leading work in stem cell transplantation in adult and paediatric patients, and highlights how identifying a well-matched donor increases the chances of a positive outcome. In 1973, we became one of the first cancer centres in Europe to carry out an SCT (also known as a bone marrow transplant). Today, the Trust is one of the largest SCT centres in Europe, carrying out more than 200 transplants every year. Despite tremendous advances in chemotherapy drugs in the intervening years, the SCT remains one of the most effective strategies to successfully treat some types of high-risk blood cancer.
The specialist clinical team in our Oak Centre for Children and Young People (OCCYP) comprises three paediatric transplant consultants, a clinical nurse specialist and a clinical fellow, supported by a multidisciplinary team of allied health professionals. The OCCYP is one of 12 specialist paediatric transplant centres in the UK, and our consultants work closely with other national experts to help decide the best course of treatment for patients across the country.
Dr Caroline Furness, Consultant Paediatric Haematologist and Lead for Paediatric SCT, explains that SCTs – which replace damaged stem cells with healthy ones – are most commonly undertaken to treat diseases such as ALL and acute myeloid leukaemia (AML).
“The process is very complex,” she says. “As soon as a patient is identified as needing a transplant, we send off blood tests to establish their tissue type and, in parallel, test family members to see if a siblingmatch will be identified. A sibling match is usually the preferred donor, but if this is not available, we’re able to search international registries to identify a matched unrelated donor as an alternative.
“In the event that there is no good unrelated donor, an alternative donor source will be used – usually an umbilical cord donation, or occasionally a parent, known as a haploidentical transplant. “We also need to achieve the ideal disease status to get the patient into transplant. The optimal state in acute leukaemia is MRD [minimal residual disease] negative, which means there is no detectable disease after treatment. Once we reach that status, we get the patient into transplant as soon as possible.”
A sibling match is usually the preferred donor, but if this is not available, we’re able to search international registries to identify a matched unrelated donor as an alternative
Making a match
The decision to undertake an SCT is never taken lightly. It is usually only performed in patients with high-risk disease where there is a significant chance of relapse, or when a patient relapses despite standard chemotherapy treatments. Careful assessment of the need for an SCT is essential due to the potential for side effects. Dr Furness says: “Although a transplant is potentially curative, it can also cause complications such as unusual or severe infections, or graft versus host disease [GvHD].”
GvHD occurs when the incoming donor immune system recognises the host (patient) as ‘foreign’. It can be an acute problem during the initial transplant admission, leading to skin rash, diarrhoea and liver dysfunction; or can become a more chronic issue, with long-term effects on quality of life. “The severity of GvHD can be related to the degree of donor match, which is why we focus on getting the best possible donor for our patients via high-resolution donor typing,” says Dr Furness.
“Over the past 20 years, through better matching and supportive care, we have improved our patients’ overall outcomes. We now need to work on improving the treatment of transplant-related complications and GvHD. “We have been expanding our team so we can run more research into these side effects. We opened three trials in the past year and hope to expand on that this year. We also recently recruited a research nurse, so we have the team in place to support more patients on clinical trials.”
Patients undergoing an SCT can often be in hospital for anything from a few weeks to a few months. Even when they are discharged, the side effects can trigger numerous overnight stays at our shared care hospitals or, in some cases, back at the OCCYP. Patients come in for weekly check-ups for up to six months following a successful SCT, then with decreasing frequency for five years. They also attend our long-term follow-up clinic for monitoring of growth, puberty and heart, lung and kidney function. Innie Johnson, Clinical Nurse Specialist, says: “I look after the families through what is likely to be one of the most difficult times of their lives. The parents are juggling jobs, caring for other family members and, of course, looking after their unwell child.
“The OCCYP is a safe and welcoming environment where we offer support for children and their families from play specialists, psychologists and teachers. Most of their care can be provided within the OCCYP and in collaboration with their local hospitals.” It is this level of care, combined with The Royal Marsden’s SCT expertise, that has led to Kareece’s recovery. Lorette says: “She is doing brilliantly now. She is possibly underdeveloped for her age, but she is here and alive. The team at The Royal Marsden were, and continue to be, amazing.”
As for Kareece, she says she is grateful to her sister Kameeka, but received more than just her twin’s stem cells from the transplant. “Since the SCT, I can no longer eat peanuts, celery or raw carrots – I managed to get Kameeka’s food intolerances, which is weird!”