**Author details**

20 Immunodeficiency

**11. Future prospects** 

vaccine antigens can be assessed once the primary vaccination schedule is complete. Vaccination with live vaccines such as MMR should only be considered once normal T cell proliferation has been demonstrated and an antibody response to the primary vaccination schedule has been confirmed. Prophylaxis against polysaccharide coated organisms should be continued for at least 2 years post transplant[66]. Patients should however have received the conjugated pneumococcal and meningococcal vaccines. Once the response to polysaccharide organisms has been demonstrated in those with normal splenic function, antibiotic prophylaxis can be discontinued. Patients should be monitored for evidence of endocrine dysfunction - particularly thyroid dysfunction[67]. Thyroid dysfunction occurs in up to 10% of post transplant patients. Usually this is hypothyroidism but more rarely hyperthyroidism has been described and each should be managed appropriately. In the long term there may be issues with fertility due to the conditioning regimens and patients should be counselled accordingly – referral to fertility clinics or endocrine specialists may be necessary. Growth is usually normal, and growth retardation due to the underlying illness may be reversed post transplant. Ongoing care of previous end organ damage such as bronchiectasis may require specialist input with regular monitoring of lung function and radiological changes. The quality of life post transplant has not been extensively assessed in primary immune deficiency patients. One study looking at the outcome of patients transplanted for severe primary immune deficiency demonstrated an increased risk of long term cognitive difficulties with associated emotional and behavioural difficulties. Specific genetic diagnosis and a severe clinical course were specifically associated with poor outcome[68]. Conversely a recent study looking at patients with CGD found significantly better quality of life skills in those who had undergone transplantation compared to those who were not transplanted, with the post transplant patients score similar to normal controls. As more patients survive the transplant procedure and a longer follow up is

achieved further work will be needed in this area to determine quality of life.

Transplantation for primary immunodeficiency is a successful treatment leading to cure of disease and normal life quality for the majority of patients. Future work will need to address optimal timing of transplantation, which may be gauged as future registry data becomes available. Less damaging conditioning regimens, particularly for newborns identified with the newborn screening programmes will become important. Treosulfan, fludarabine containing regimens are less toxic than busulphan containing regimens but targeting conditioning using radiotherapy or monoclonal antibodies may play a great role in the future. Accelerating thymopoiesis and immuno-reconstitution will be important particularly when pre-existing viral infection is present. Agents including exogenous interleukin 7 or keratinous growth factor may have a role to play. Graft manipulation may improve outcomes for some patients - better use of T cell depleted donors when no matched donors are available and particularly improving immune reconstitution through TCR alpha beta depletion may be appropriate. Expansion of stem cells in cord blood transplantation, the use Mary A. Slatter\* and Andrew R. Gennery *The Institute for Cellular Medicine, Newcastle upon Tyne University, Newcastle upon Tyne, UK* 
