Objective. To assess the safety and efficacy of intensive immunosuppression followed by T celldepleted autologous hematopoietic stem cell transplantation (ASCT) for induction of disease remission in children with refractory progressive juvenile idiopathic arthritis (JIA).Methods. Twenty-two patients with progressive refractory JIA were followed up over a median period of 80 months after pretreatment with intensive immunosuppression followed by ASCT in a multicenter, prospective, phase II clinical trial. Hematopoietic stem cells were harvested from the patients' bone marrow, depleted of T cells, and kept frozen until used for ASCT. Pretreatment of patients consisted of a combination of antithymocyte globulin, cyclophosphamide, and lowdose total body irradiation. Patients were followed up for ASCT-related complications, recovery of hematologic and immune system parameters, and disease outcomes. Conclusion. Intensive immunosuppression followed by ASCT resulted in sustained complete remission or marked improvement in 15 of 22 patients with progressive refractory JIA. The procedure, however, is associated with significant morbidity and risk of mortality due to prolonged and severe depression of T cell immunity. After fatal complications due to MAS were observed in some patients, the protocol was amended in 1999, to ensure less profound depletion of T cells, better control of systemic disease before transplantation, antiviral prophylaxis after transplantation, and slow tapering of corticosteroids. Following these protocol modifications, no additional ASCT-related deaths were observed among the 11 patients who received the modified treatment. Results. Reconstitution of hematologic values toJuvenile idiopathic arthritis (JIA) is a heterogeneous group of pediatric illnesses that share the Supported by grants from the Netherlands Organization for Scientific Research (940-37-004) and the National League against Rheumatism (NR 901).

In the first 2 years after bone tumor surgery, survivors improved significantly with respect to QoL, functional ability, and physical activity levels.

In children and adolescents who underwent surgery for a malignant tumor of the leg physical, functioning was significantly impaired as compared to healthy controls.

In rheumatic diseases the use of corticosteroids (CS), immobility, or the disease itself, may cause osteoporosis and growth retardation. We evaluated bone mineral density (BMD) by dual energy X-ray absorptiometry (DXA), growth and physical activity in 27 children with rheumatic disease, all treated with high dose CS for at least one year, in a cross-sectional design. BMD SDS was significantly lower than zero: -1.02 for total body and -1.49 for lumbar spine measurement. SDS for fat mass was higher than zero. In multiple regression analysis the Child Health Assessment Questionnaire score significantly correlated with BMD of the lumbar spine. There was no significant correlation with cumulative dose or duration of CS treatment. Height SDS decreased during treatment to -1.57 (p <0.001 compared to 0). In conclusion, BMD and body composition in children with rheumatic disease treated with CS are influenced by physical activity, as well as corticosteroid treatment and type of rheumatic disease.

One to 5 years after limb-salvage and ablative surgery due to a malignant bone tumor children and young adults do, apart from a few activities involving walking and climbing stairs, not differ with respect to overall functional ability and physical activity.

BackgroundCombination therapy with prednisone or etanercept may induce earlier and/or more improvement in disease activity in Disease Modifying Anti Rheumatic Drug (DMARD) naïve non-systemic Juvenile Idiopathic Arthritis (JIA) patients. Here we present three months clinical outcome of initial treatments of the BeSt-for-Kids study.MethodsIncluded patients were randomized to either: 1. initial DMARD-monotherapy (sulfasalazine (SSZ) or methotrexate (MTX)), 2. Initial MTX / prednisolone-bridging, 3. Initial combination MTX/etanercept. Percentage inactive disease, adjusted (a) ACR Pedi30, 50 and 70 and JADAS after 6 and 12 weeks of treatment (intention to treat analysis) and side effects are reported.Results94 patients (67% girls, 32 (arm 1), 32 (arm 2) and 30 (arm 3) with median (InterQuartileRange) age of 9.1 (4.7-12.9) years were included. 38% were ANA positive, 10 had oligo-articular disease, 68 polyarticular JIA and 16 psoriatic arthritis. Baseline median (IQR) ACRpedi-scores: VAS physician 49 (40-58) mm, VAS patient 54 (37-70) mm, ESR 6.5 (2-14.8)mm/hr, active joint count 8 (5-12), limited joint count 3 (1-5), CHAQ score 0.88 (0.63-1.5). In arm 1, 17 started with MTX, 15 with SSZ.After 3 months, aACR Pedi 50 was reached by 10/32 (31%), 12/32(38%) and 16/30 (53%) (p = 0.19) and aACR Pedi 70 was reached by 8/32 (25%), 6/32(19%) and 14/30(47%) in arms 1-3 (p = 0.04). Toxicity was similar. Few serious adverse events were reported.ConclusionAfter 3 months of treatment in a randomized trial, patients with recent-onset JIA achieved significantly more clinical improvement (aACRPedi70) on initial combination therapy with MTX / etanercept than on initial MTX or SSZ monotherapy.Trial registration NTR1574. Registered 3 December 2008.

The Bt-DUX was found to be a practical and valid instrument. Its added value compared with existing HRQoL measures needs to be further established.

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