We hope to recruit 120 participants into the study from NHS sites across the UK.
What is involved
Participants will be asked to attend visits approximately every 3 months over a 2-year period.
For those in the standard care group:
For those randomly allocated to get standard care, participants will keep getting the best approved treatments for their sickle that they've already agreed to, like transfusions or medicines such as hydroxycarbamide and voxelotor. The medical team will talk through the options and decide together what's best for that individual. If any new treatments get approved while the study is going on, the participant can also get those. Check-ups for the study will happen at the participants’ local NHS sickle cell treatment centre.
For those in the stem cell transplant group:
For those randomly allocated to the transplant group, they'll have the transplant scheduled soon after their treatment allocation is confirmed. They will go to the hospital for the transplant procedure, following the usual UK stem cell transplant process. Before the transplant, for about 10 days, they will get chemotherapy to prepare the body for the procedure. They will also get a single dose of total body irradiation (TBI), which is part of the preparation. This treatment destroys the old bone marrow cells to make room for the new ones and lowers the immune system to reduce the risk of rejection. The treatment is given through a tube into the bloodstream to avoid lots of injections. The individual will be closely watched by the medical team to make sure they handle the treatment well.
On the transplant day, after the conditioning treatment, stem cells from a half-matched donor will be put into their bloodstream through the tube, like a blood transfusion. These cells will find their way back to the person’s bone marrow on their own, taking only a couple of hours. The transplant isn't painful, and they will be awake during it. Afterward, the individual will need to stay in the hospital for a few weeks because their immune system will be weak, putting them at risk of infection. This time is for close monitoring and recovery. They will have follow-up visits in hospital where they had the transplant to continue monitoring progress, likely weekly for the first few months after transplant and then less frequently as recovery progresses.
We will analyse the results at the end of the study and publish our findings in medical and scientific journals. We will also present results at meetings, medical conferences and share the results on this website.
Participants will be assigned to one of two groups at random (decided by chance by a computer, like at the flip of a coin). The first ‘standard of care’ group will receive the standard medical care including any currently available treatment for sickle cell. The second ‘haplo-identical stem cell transplant’ group will receive a stem cell transplant from a haplo-matched family donor. This random allocation of treatment is to make this study a fair test.
We will ask participants to donate blood and urine samples and carry out health checks to find out how participants are doing since their treatment and their last visits.