In keeping with our mission, the following has been achieved:
Dedicated Treatment and Care
- more than 1,400 children referred per year
- perform more than 30 bone marrow transplants per year, with success rates rising from 50% to 80%, and world breaking results for the most difficult mismatched transplants
- network of clinics in Edinburgh, Dublin, Manchester, Carlisle and Teesside
- improved day-by-day care for children undergoing bone marrow transplantation, including better ways of treating infection in central venous catheters, studying the use of new drugs that prevent and treat life threatening fungal (mould) infections
- part-funded the greatly improved facilities on the Unit
- provided state-of-the art equipment that enables all aspects of each child’s condition to be monitored minute by minute
- provided all the infusion pumps that are vital for administering the many difficult medications each child needs
- supported nurse education and updates, enabling staff to attend important international conferences/meetings and give presentations to benefit the Unit
provided new ‘Miltenyi’ machine, which manipulates bone marrow before the transplant in a much improved way, resulting in fewer infections and complications in the patients, as well as a more rapid recovery and earlier discharge home
- provided toys and educational equipment for each child on the Unit
Integrated Family Support
- holistic approach - close relationship with medical & nursing staff, liaison team, nursery nurses, occupational and physiotherapists, teachers and social worker
- provided for the development of the Children’s Day Unit so that more convalescent patients can go home earlier and have sometimes-complex treatments as an outpatient
- upgraded parents’ accommodation in the flats close to the hospital and on the Unit
- provided for Parents’ Support Group weekends
Focused Research
- funded research in aspects of bone marrow transplant process, as well as part-funding a consultant to further develop the Unit’s research programme
part-funded totally germ free stem cell laboratory for developing still better techniques for bone marrow and stem cell transplants, and hopefully gene
- therapy in the future
- pioneering work with placental cord blood stem cell transplantation as a cure for SCID
- research into life threatening lung inflammation after transplant – leading to new treatments and survival where previously children would have died
- successfully developed a programme to cure older children with other primary immune deficiencies other than SCID
- research into what happens to patients with Chronic Granulomatous Disease (CGD), comparing current supportive care with new curative treatments such as bone marrow transplantation
- developed UK and European programmes to cure CGD by bone marrow transplantation – successful in 10 out of 12 cases
- developed bone marrow transplant protocol to cure patients with CD40 Ligand deficiency
- research into primary immune deficiency diseases caused by defects in the mechanisms that repair DNA (the genetic blueprint), identifying new conditions
- research that has discovered the most important immune problems in Di George syndrome
- research into ensuring a fully functioning immune system develops after bone marrow transplantation
