Autologous haematopoeitic stem cell rescue (AHSCR) for severe rheumatic disease in children

Abstract

Autologous haematopoeitic stem cell rescue (AHSCR) is a treatment option for children with severe rheumatic disease and to date most experience is with juvenile idiopathic arthritis (JIA) [1-6]. Currently two transplant centres in the UK have NSCAG ( National Specialist Commissioning Advisory Group) funding to perform AHSCR for children with severe rheumatic disease; these are Newcastle (Newcastle General Hospital) and London (Great Ormond Street), both of which follow latest protocols set with the Joint European Bone Marrow Transplant (EBMT) and European Society for Immunodeficiency (ESID) Working Party Sub-Committee (www.esid.org), and submit patients to the EBMT Registry. In the absence of an evidence base to support the referral and selection process, this guideline is a consensus statement, commissioned by the BSPAR Executive, and targeted at BSPAR members. This states criteria for considering AHSCR and provides an outline of the process ( selection, independent assessment and referral to the transplant centre) and defines the roles of the stakeholders (i.e. referral team, independent assessor and transplant team). The guideline refers specifically to children with JIA, but the principles are likely to be similar for other severe rheumatic diseases of childhood. This is a short summary of the whole guideline. The full guideline is available on the journal website (see supplementary material for full guideline).