Juvenile chronic arthritis (JCA), the most frequent transplanted children with JCA is in line with this ‘theoretical concept’ [7]. autoimmune disease of childhood, is not a benign disease. Functional disability occurs in 20–30% of patients Of course, many questions have to be dealt with. Is ASCT the treatment of choice? After ASCT, the with JCA and 5–10% develop a serious handicap [1]. Children with a polyarticular and systemic-onset form immunogenetic genotype does not change. However, the alternative, allogeneic stem cell transplantation, is not are especially at risk. To improve the outcome of these patients is a challenge for paediatric rheumatologists. acceptable because of the higher mortality risk in a nonlethal disease. What is the best conditioning regimen? A To improve disease outcome, immunosuppressive drugs like methotrexate [2], cyclosporin [3] and azathiohot topic is the use of total body irradiation (TBI). In experimental animal models, TBI is necessary to sustain prine [4] have been introduced in the treatment of JCA. Although these drugs have been proven to be effective remission [8]. On the other hand, TBI is rather toxic with a risk for infertility, growth retardation and cancer in a substantial number of the JCA patients, the disease lingers on and results in more or less serious functional [9]. Another issue is the handling of the graft. Should we deplete for T cells and which method do we use for handicaps. Treatment strategies resembling those used as an depletion [10]? Last, but not least, is the question whether we should use bone marrow as a source of stem induction regimen for haematological malignancies are now being introduced. An example is the treatment cells or pure blood. The latter method certainly has advantages. Collection is much easier and in general regimen built up from methothrexate, pulses of methylprednisolone and cyclophosphamide [5]. The toxicity of more stem cells can be harvested. However, the patient should be pre-treated with G-CSF, which in theory this treatment regimen is high and effectiveness has not been proven. Case reports of adult patients with autocould worsen the disease and, especially in the systemiconset form, the risk of inducing macrophage-activating immune diseases treated with autologous or allogeneic stem cell transplantation for a secondary haematological syndrome. In this issue of Rheumatology, many experts are malignancy or bone marrow aplasia showed that patients may be cured of their original ‘autoimmune’ invited to give their opinion on the above-mentioned questions. Of course, we cannot solve all the answers at disease [6 ]. This success motivated rheumatologists and haematologists to use autologous stem cell transplantathis stage, but the experience in experimental models and of the use of bone marrow (stem cell ) transplanttion (ASCT ) for the treatment of autoimmune diseases and there is a rapid expanding experience with ASCT ation in haematological malignancies can direct our way. In this issue, the first results are presented after ASCT [6 ]. The advantage of ASCT is that the conditioning treatment leads to radical eradication of mature T cells, of four children with JCA [7], one child with systemic scleroderma [11] and patients with systemic lupus erythwhile the stem cells are rescued by collecting them in advance. Theoretically, another advantage is that naive ematosus [12]. Although the follow-up period is rather short, the first results are promising. Of course, longT cells should be re-educated after ASCT, resulting in peripheral tolerance. In this way, ASCT should not only term follow-up is necessary to determine the real place of ASCT in the treatment of these autoimmune diseases. be regarded as a heavy immunosuppressive (myeloablative) treatment, but should also induce protective In May, during the ‘First workshop on the use of autologous stem cell transplantation in rheumatic dis‘anti-inflammatory’ responses. The experience in the first eases of childhood’, we reached consensus about inclusion criteria for JCA, the conditioning regimen and graft Accepted 15 March 1999. handling, which is published in this issue [13]. It is Correspondence to: N. M. Wulffraat, Department of Paediatric important that we restrict ourselves to a very limited Immunology, University Hospital for Children, ‘HET Wilhelmina Kinderziekenhuis’, Utrecht, The Netherlands. number of protocols, so we can really compare and
Read full abstract