The Immunology department at Great Ormond Street Hospital (GOSH) offers a comprehensive diagnostic and treatment service for children up to the age of 16 years with suspected or confirmed primary immunodeficiency.
Services are also provided for children with a variety of other immunological disorders (excluding allergy at present).
Primary immunodeficiency disorders
Primary immunodeficiency disorders (PIDs) form a group of rare disorders that vary widely in severity. Those at the mild end of the spectrum can predispose infants and children to frequent minor infections, while the more severe ones lead to life-threatening infections and other life-limiting complications. In some cases the deficiency resolves with increasing age, but in most cases these are lifelong disorders that require treatments to prevent infections and treat other complications and in some cases need aggressive treatment to correct the underlying problem.
The Immunology department offers diagnosis and management of all degrees of severity of PID. Most referrals are received at a tertiary level from secondary paediatric units across the UK, but direct referrals from GPs are also accepted. International referrals are also received from a variety of countries.
Services provided include inpatient, outpatient and day-case services. Infants and children who are affected by the more severe primary immunodeficiencies may require inpatient care and some require treatment by bone marrow transplantation (BMT). A small and highly selected group of children also undergo gene therapy procedures.
Day-case work includes assessments and treatments for children awaiting and being followed up after BMT, multidisciplinary assessments and treatment of children with other forms of immunodeficiency and families undergoing training for home immunoglobulin (antibody replacement) therapy.
Outpatient services are offered for assessment of children with possible immunodeficiency and follow-up of children with confirmed immunodeficiency. There are specialist clinics for certain disorders and dedicated BMT follow-up clinics. Joint clinics are held with several related sub-specialties and outreach clinics are held in a number of other centres.
Children with immunodeficiency are managed by the medical/specialist nursing team, on a shared care basis with local paediatricians, community nurses and in some cases general practitioners.
The Immunology department has well-established links with many general paediatric units throughout the UK.
When children with lifelong primary immunodeficiency reach the age of 16-18 years, appropriate preparations are made to for referral to an appropriate adult immunology centre.
Bone marrow transplantation
The more severe forms of immunodeficiency may require treatment by BMT or, in a few highly selected cases, gene therapy.
GOSH is one of the two supra-regionally funded centres in the UK for BMT for severe immunodeficiency, and is one of the largest centres worldwide. This service is provided by a close collaboration between the Immunology department and Bone Marrow Transplant Unit.
Gene therapy is performed for a small number of specific disorders, under the supervision of the gene therapy team in the recently designated Wolfson Centre for Gene Therapy.
Other primary immunodeficiency
Other forms of primary immunodeficiency require lifelong supervision and treatment to prevent infection, maintain as far as possible normal quality of life and prevent complications. In many cases this includes treatment with antibody (immunoglobulin) replacement and in others lifelong antibiotic treatment.
The majority of children requiring long-term immunoglobulin replacement have their treatment at home. There is also a specialist service for children affected by chronic granulomatous disease.
Children affected by a variety of other disorders are managed by the Immunology team, often in conjunction with other specialists.
These include the periodic fever syndromes, C1 esterase inhibitor deficiency and various inflammatory and vasculitic disorders.