2 Clinical need and practice

2.1 Kidney transplant is used to treat established kidney failure, which is severe and irreversible impairment of kidney function. After a kidney transplant, immunosuppressive therapy is used to reduce the risk of rejection of the transplanted kidney (or 'graft') and prolong its survival. Between April 2016 and March 2017, 127 kidney transplants were done in the UK for children and young people under 18 years; 116 of these were in England.

2.2 Kidney transplant in children and young people can differ from adults in several important aspects including the cause of kidney failure, the pharmacokinetic properties of immunosuppressive therapies and how they are metabolised, the immune response after transplant, the measures of success of the transplant procedure, the susceptibility to post-transplant complications, and the degree of adherence to treatment.

2.3 Immunosuppressive therapy aims to prevent acute rejection and optimise the function of the transplanted kidney, while minimising the adverse effects of immunosuppression (such as increased risk of infection, cancer, diabetes and cardiovascular disease). Immunosuppressive therapy can be categorised as induction therapy or maintenance therapy. Induction therapy is an intensive immunosuppression regimen that is used for up to 2 weeks around the time of transplant and may include polyclonal or monoclonal antibodies. Maintenance therapy starts immediately after transplant and continues for life.

2.4 NICE's technology appraisal guidance on immunosuppressive therapy for kidney transplantation in children and adolescents was published in 2006. It recommended basiliximab, daclizumab, tacrolimus, mycophenolate mofetil and sirolimus, in certain circumstances, as options for immunosuppressive therapy for kidney transplant in children and young people. Since that appraisal, the marketing authorisation for daclizumab has been withdrawn, new technologies (rabbit anti-human thymocyte immunoglobulin, mycophenolate sodium, belatacept, a prolonged-release formulation of tacrolimus, and everolimus) have received marketing authorisations, but some of the marketing authorisations exclude children and young people. In addition, some of the technologies are available as generics.

  • National Institute for Health and Care Excellence (NICE)