Rheumatology Advance Access originally published online on October 31, 2006
Rheumatology 2006 45(12):1570-1571; doi:10.1093/rheumatology/kel319a
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Autologous haematopoeitic stem cell rescue (AHSCR) for severe rheumatic disease in children
Guidance for BSPAR members—executive summary
1Newcastle University, Newcastle-upon-Tyne, 2Yorkhill Children's Hospital NHS Trust, Glasgow, 3Royal Liverpool Children's Hospital NHS Trust, Liverpool, 4Newcastle Hospitals NHS Trust, Newcastle-upon-Tyne, and 5University College London and Great Ormond Street Hospital NHS Trust, London, UK.
Correspondence to: Dr H. Foster, School Clinical Medical Science (Rheumatology), Medical School, Framlington Place, Newcastle-upon-Tyne NE2 4HH. E-mail: h.e.foster{at}ncl.ac.uk
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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).
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1. The process of patient selection, independent assessment and referral to the transplant team
1.1 The selection of patients.
AHSCR can be considered as an option for a child or young person with any subtype of JIA, who fulfils the inclusion and exclusion criteria [7] based on:
- Severity of disease and persistent disease activity.
- Failure of immunosuppressive and anti-inflammatory therapy.
- Drug toxicity or intolerance.
In accordance with the latest Working Party guidelines (www.esid.org), proposed patients for AHSCR should meet all these criteria. Further detail for each of these criteria is given in the full guideline but the important point is to think ahead for difficult cases as the process of AHSCR takes time.
1.2 The referral for independent assessment
- Referral for AHSCR should only occur if the patient has had input from a tertiary paediatric rheumatology team (i.e. a centre with a specialist multi-disciplinary service).
- With the family's agreement, the child's case is discussed informally with the transplant centre. Once the transplant centre agrees in principle that AHSCR is to be considered, then the referral to the independent assessor is made by the referral centre, with an appropriate summary of information as suggested in the full guideline.
- The child and family are seen by the independent assessor.
- The independent assessor will compile a report to all the stakeholders, the family and primary care team.
1.3 The referral to a Transplant Centre and the AHSCR procedure (including pre- and post-AHSCR care)
- The child and the family are seen at one of the NSCAG funded transplant centres after independent assessment has taken place, given the ‘go-ahead’ for AHSCR and as soon as possible to avoid undue delay and anxiety by the family.
- A video (called ‘Josh's Story’) is available from the Newcastle transplant centre to help the family to understand the AHSCR procedure.
- The visit to the transplant centre takes place, the family will meet the transplant team staff and discussion should take place about the process and risks.
- The transplant centre makes the provisional decision to offer AHSCR, and sets a provisional date, based on the information from independent assessor, further investigations if required, and following discussion with the child and the family.
- In the ‘run-up’ to the AHSCR, the referral/tertiary centre should liaise closely with the transplant centre to ensure an agreed protocol to optimise disease control (and particularly to avoid macrophage activation syndrome (MAS) which is a potentially fatal complication of systemic onset JIA) and to support the family as necessary.
- After AHSCR, the transfer of care back to the referral centre will be done by agreement between the two centres, although follow-up by the transplant team after AHSCR will be for several years.
- In the event of a death before, during or after AHSCR, a post-mortem should be considered.
2. The roles of the ‘stakeholders’
The key is good communication between the referral team, independent assessor and the transplant team.
2.1 The roles of the referral team
- Informal discussion with the transplant centre to consider AHSCR as a treatment option.
- Discussion in broad terms with the family regarding the rationale for AHSCR, the need for independent assessment and support the family in this process.
- Production of a detailed summary for the independent assessor following the template given in the full guideline.
- Liaison with the transplant team and formal referral as soon as possible after independent assessment has been sought and at all stages of the AHSCR process.
2.2 The roles of the independent assessor.
The independent assessor will be a consultant paediatric rheumatologist, nominated by BSPAR Executive, not working in either the London or Newcastle transplant units and will ideally hold this post for several years to gain experience.
The roles of the independent assessor are to:
- Clarify that AHSCR is an appropriate option (as detailed in 1.1).
- Discuss alternative therapeutic options as appropriate.
- Make the family aware of the broad complexity of the procedure with an indication the risk involved.
- Compile and disseminate a report based on the interview, case note review and information sent from the referral centre.
- Monitor progress post-AHSCR in accordance with current recommendations.
2.3 The roles of the transplant team
- Liaise with BSPAR and EBMT regarding protocols for AHSCR and disseminate any proposed changes.
- Provide access to educational material for prospective children and families.
- Liaise with referral team and the independent assessor for all appointments.
- Share care wherever possible, based on the premise that the referral team often know the ‘child and their disease’ best, and especially to optimize disease control and minimize risk of MAS.
- Provide prompt discharge and follow-up summaries.
- Liaise with local nursing and therapy teams regarding rehabilitation.
- Liaise with the referral team to support families especially in the event of complications/death.
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Prospective children and their families will need to be assessed by an independent assessor and at least on one occasion by the transplant centre. This will incur cost to the health care organizations involved as well as time, effort and cost to the families. Such costs are currently borne by the referral centres. Once the child is selected for AHSCR, the costs are met by the Department of Health as part of the NSCAG funding to the two centres involved.
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- De Kleer IM, Brinkman DM, Ferster A, et al. (2004) Autologous stem cell transplantation for refractory juvenile idiopathic arthritis: analysis of clinical effects, mortality, and transplant related morbidity. Ann Rheum Dis 63:1318–26.
[Abstract/Free Full Text] - Barron KS, Wallace C, Woolfrey CEA, et al. (2001) Autologous stem cell transplantation for pediatric rheumatic diseases. J Rheumatol 28:2337–58.
[Abstract/Free Full Text] - Wulffraat N, van Royen A, Bierings M, Vossen J, Kuis W. (1999) Autologous haemopoietic stem-cell transplantation in four patients with refractory juvenile chronic arthritis. Lancet 353:550–3.[CrossRef][Web of Science][Medline]
- Wulffraat NM, Vastert B, Tyndall A. (2005) Treatment of refractory autoimmune diseases with autologous stem cell transplantation: focus on juvenile idiopathic arthritis. Bone Marrow Transplant 35:Suppl 1, S27–9.
- Wedderburn LR, Abinun M, Palmer P, Foster HE. (2003) Autologous haematopoietic stem cell transplantation in juvenile idiopathic arthritis. Arch Dis Child 88:201–5.
[Free Full Text] - Abinun M, Veys VP, Foster HE, Wedderburn LR. (2005) Autologous haematopoetic stem cell transplantation for refractory juvenile idiopathic arthritis in the UK: experience 2000–2005. Arch Dis Child 90:Suppl II, A52.
- Wulffraat NM, Kuis W, Petty R. (1999) Addendum: proposed guidelines for autologous stem cell transplantation in juvenile chronic arthritis. Paediatric Rheumatology Workshop. Rheumatology 38:777–8.
[Free Full Text]
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