Quality of life after bone sarcoma surgery around the knee: A long-term follow-up study.

It remains unclear if quality of life (QoL) improvements could be expected in young patients after malignant bone tumour surgery after 2 years. To assess the course of QoL over time during a long-term follow-up, malignant bone tumour survivors of a previous short-term study were included. Assessments were done at least 5 years after surgery. QoL was measured with Short-form (SF)-36, TNO-AZL Questionnaire for Adult's Quality of Life (TAAQOL) and Bone tumour (Bt)-DUX. QoL throughout the follow-up was analysed by linear mixed model analysis. From the original cohort of 44 patients; 20 patients were included for this study, 10 males; mean age at surgery 15.1 years and mean follow-up 7.2 years. Twenty-one patients of the initial cohort (47%) deceased. Fifteen patients (75%) underwent limb-salvage and five (25%) ablative surgery. QoL improved significantly during follow-up at Physical Component Summary Scale scale of the SF-36 and TAAQOL and all subscales of the Bt-DUX (p < .01). No significant differences were found between current evaluations and previous evaluations at 2 years after surgery (p = .41-.98). Significant advantages after limb-salvage were seen at the PCS scale of the SF-36 (MD 13.7, p = .05) and the cosmetic scale of the Bt-DUX (MD 17.7, p = .04).

A comparison of three treatment strategies in recent onset non-systemic Juvenile Idiopathic Arthritis: initial 3-months results of the BeSt for Kids-study.

BACKGROUND: Combination 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. METHODS: Included 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. RESULTS: 94 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. CONCLUSION: After 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.

Autologous stem cell transplantation in children with severe progressive systemic or polyarticular juvenile idiopathic arthritis: long-term follow-up of a prospective clinical trial.

OBJECTIVE: To assess the safety and efficacy of intensive immunosuppression followed by T cell-depleted 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 low-dose total body irradiation. Patients were followed up for ASCT-related complications, recovery of hematologic and immune system parameters, and disease outcomes. RESULTS: Reconstitution of hematologic values to normal range was rapid. Recovery of immune system parameters, especially normalization of CD4+, CD45RA+ naive T cells, was delayed, occurring at >/=6 months after ASCT. The prolonged period of immune deficiency resulted in a large number of viral infections and may have contributed to the development of macrophage activation syndrome (MAS), leading to death, in 2 patients. After ASCT, 8 of the 20 evaluable patients reached complete clinical remission of their JIA, 7 were partial responders, and 5 experienced a relapse of their disease (occurring 7 years after ASCT in 1 patient). Later during followup, 2 of the patients whose disease relapsed died from infections that developed after restarting immunosuppressive medication. 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.

Bone mineral density and body composition and influencing factors in children with rheumatic diseases treated with corticosteroids.

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.

The relationship between impairments in joint function and disabilities in independent function in children with systemic juvenile idiopathic arthritis.

OBJECTIVE: Knowledge about the impact of joint impairment on functional ability is needed in planning care and setting treatment goals in children with juvenile idiopathic arthritis (JIA). We investigated the relationship between joint impairments and upper and lower limb function. METHODS: Twenty-one children with systemic JIA with an average age of 9.2 years and a mean disease duration of 4.8 years participated in this study. Joint impairments were assessed by the following variables: joint counts on swollen (JCS) and tender (JCT) joints and the loss of joint motion as determined by the Joint Alignment and Motion scale (JAM). Functional performance and functional ability were determined by the Juvenile Arthritis Functional Assessment Scale (JAFAS) and Childhood Health Assessment Questionnaire (CHAQ), respectively. The relationship between impairments and functional disabilities was studied at the level of (1) the complete instruments, (2) upper and lower limb function separately, and (3) the individual joints and items. RESULTS: Regarding complete instruments, the Spearman rank correlation between functional disabilities and loss of joint motion was moderate to good (JAM/CHAQ rs = 0.66, JAM/JAFAS rs = 0.77). A fair correlation was found between functional disabilities and the joint count on swollen joints (JCS/CHAQ rs = 0.45, JCS/JAFAS rs = 0.52), but no significant relationship was found with the number of tender joints (JCT/CHAQ rs = 0.02, p > 0.05, and JCT/JAFAS rs = 0.14, p > 0.05). At the extremity level (upper and lower limb function), the relationship between functional disabilities and the loss of joint motion appeared to be stronger in the leg than in the arm. At the level of the individual joints and questionnaire items, loss of joint motion in hip or shoulder joint appeared to be the most important factor in predicting limitation in leg or arm function. CONCLUSION: Our study shows that with respect to joint impairments, loss of joint motion is the strongest indicator of functional disability in children with systemic JIA. Loss of joint motion has a greater effect on lower limb function.