QuestionWhich is the best strategy to achieve (drug-free) inactive disease in juvenile idiopathic arthritis (JIA)?MethodsIn a randomised, single-blinded, study in disease-modifying anti-rheumatic drug (DMARD)-naive patients with JIA, three treatment-strategies were compared: (1) sequential DMARD-monotherapy (sulfasalazine or methotrexate (MTX)), (2) combination therapy MTX + 6 weeks prednisolone and (3) combination therapy MTX +etanercept. Treatment-to-target entailed 3-monthly DMARD/biological adjustments in case of persistent disease activity, with drug tapering to nil in case of inactive disease.After 24 months, primary outcomes were time-to-inactive-disease and time-to-flare after DMARD discontinuation. Secondary outcomes were adapted ACRPedi30/50/70/90 scores, functional ability and adverse events.Results94 children (67 % girls) aged median (IQR) 9.1 (4.6–12.9) years were enrolled: 32 in arms 1 and 2, 30 in arm 3. At baseline visual analogue scale (VAS) physician was mean 49 (SD 16) mm, VAS patient 53 (22) mm, erythrocyte sedimentation rate 12.8 (14.7), active joints median 8 (5–12), limited joints 2.5 (1–4.8) and Childhood Health Assessment Questionnaire score mean 1.0 (0.6).After 24 months, 71% (arm 1), 70% (arm 2) and 72% (arm 3) of patients had inactive disease and 45% (arm 1), 31% (arm 2) and 41% (arm 3) had drug-free inactive disease. Time-to-inactive-disease was median 9.0 (5.3–15.0) months in arm 1, 9.0 (6.0–12.8) months in arm 2 and 9.0 (6.0–12.0) months in arm 3 (p=0.30). Time-to-flare was not significantly different (overall 3.0 (3.0–6.8) months, p=0.7). Adapted ACR pedi-scores were comparably high between arms. Adverse events were similar.ConclusionRegardless of initial specific treatments, after 24 months of treatment-to-target aimed at drug-free inactive disease, 71% of recent-onset patients with JIA had inactive disease (median onset 9 months) and 39% were drug free. Tightly controlled treatment-to-target is feasible.Trial registration number1574.
The purpose of this study was to translate the English bone tumour DUX (Bt-DUX-Eng) questionnaire for lower extremity bone tumour patients, a disease-specific quality of life (QoL) instrument, into Italian and then examine the validity of the Italian version of Bt-DUX (Bt-DUX-It). The adaptation and translation process included forward translation, back-translation, and a review of the back-translation by an expert committee. The Bt-DUX-It was validated in a sample of adolescents treated for lower extremity osteosarcoma in Italy. Assessments included the Bt-DUX, the Toronto Extremity Salvage Score (TESS), and the European Organization for Research and Treatment Core Quality of Life Questionnaire of Cancer Patients (EORTC QLQ-C30). Fifty-one patients with a median age of 20 years (range: 15–25) completed the questionnaires. The mean Bt-DUX score was 70 (range: 16.30–100). The internal consistency of the overall score and that of the Bt-DUX-It was good: Cronbach’s α was 0.95. Spearman’s correlation coefficient between the Bt-DUX (total and domain scores) and EORTC QLQ C30 and TESS were overall moderate to good, reaching a p-value <0.01 in all cases. The Bt-DUX-It version is a useful tool for measuring QoL in patients with bone tumour and has similar internal consistency, construct validity, and discrimination as those of the Dutch and English versions.
BackgroundIn rheumatoid arthritis treatment, targeted treatment has shown to improve disease outcomes including the option of drug tapering and discontinuation. In non-systemic juvenile idiopathic arthritis (nsJIA) this has not been tried in a trial.ObjectivesTo investigate which of three treatment strategies, targeting at drug-free inactive disease, is most effective and safe in recent onset DMARD-naive nsJIA.MethodsWe conducted a randomised, multicenter, treatment strategy study with 24 months of follow up. Patients, 2–16 years old with symptom duration <18 months were randomised to 1)Sequential DMARD-monotherapy (sulfasalazine (SSZ) or methotrexate (MTX), 2)Combination therapy MTX +6 weeks prednisolone, 3)Combination therapy MTX +etanercept. Treatment to target entailed three-monthly treatment intensifications in case of persistent disease activity. DMARDs were tapered to nil in case of inactive disease for at least 3 (in oligoarticular) or 6 (in polyarticular) months. After 24 months, primary outcomes were time-to-inactive-disease and time-to-flare after DMARD discontinuation. Secondary outcomes were adapted ACRPedi30/50/70/90scores, functional ability and toxicity. Missing data were imputed.Results94 children (67% girls) with a median (InterQuartile Range) age of 9.1 (4.6–12.9) years were enrolled: 32 in arms 1 and 2, 30 in arm 3. Eleven had oligo-articular JIA, n=73 polyarticular JIA and n=8 juvenile psoriatic arthritis, 37% were ANA positive. At baseline VAS physician was median (IQR) 5039–58 mm, VAS patient 5437–70 mm, ESR 6 (2–14) mm/hr, active joints 8,5–12 limited joints 2.5 (1–5), and CHAQ score 0.9 (0.6–1.5).After 24 months 61% (arm 1), 63% (arm 2) and 61% (arm 3) of patients had inactive disease and 45% (arm 1) 31% (arm 2) and 38% (arm 3) had stopped all DMARD(s). Time to inactive disease (median 9.0 (6.0–12.0) months) was not significantly different between arms, nor was time to flare (18.0 (15.0–21.0) months). Adapted ACRpedi-scores were comparably high between arms. Functional ability improved and remained almost normal. Toxicity reports showed mild events in similar rates across all arms.Arm 1n=31Arm 2n=32Arm 3n=29 ACRPedi50 (%)(CI)85.5(72.4–98.6)83.8(70.1–97.4)93.1(83.7–102.4)ACRPedi70 (%)(CI)69.0(52.1–85.9)68.8(51.6–85.9)82.8(68.8–96.8)ACRPedi90(%)(CI)58.4(40.6–76.1)55.3(37.3–73.3)69.0(51.8–86.1)Inactive disease (%)61.0(39.7–82.3)63.1(43.6–82.7)61.0(40.9–81.2)Results after 24 months by GEE.ConclusionsTreatment to target drug free inactive disease is feasible in recent onset non-systemic JIA, resulting, regardless of initial treatment, in over 60% of patients in inactive disease and 38% drug free.Disclosure of InterestNone declared
BackgroundIn the BeSt for Kids (a three-armed treatment strategy study in juvenile idiopathic arthritis (JIA)) time to inactive disease, time to flare after Disease Modifying Anti Rheumatic Drug (DMARD) discontinuation, ACRpedi scores, functional ability, safety, and radiological damage are compared. Follow up will be 2 years. Here we present the 3 months clinical outcomes of initial treatments.MethodsRecent onset, DMARD naïve JIA patients (Rheumatoid Factor-negative polyarticular, oligoarticular or with psoriasis (ILAR-criteria), <18 months symptom duration) were randomized (per center, variable blocks) to 3 treatment strategies: 1. sequential DMARD-monotherapy (starting with sulfasalazine (SSZ) 50mg/kg/day or methotrexate (MTX) 10mg/m2/week, based on physician's preference), 2. combination therapy: MTX 10mg/m2/week and 4 weeks prednisolone-bridging 0.5mg/kg/day, in 2 weeks tapered to nil, and 3. combination therapy with MTX 10mg/m2/week and etanercept 0.8mg/kg/wk.NSAIDs and intra-articular steroids are permitted in all patients. MTX was combined with folic acid 5mg/week. Treatment adjustments were made every 3 months after assessment by a trained, blinded examiner. Target was ACRpedi50 after 3 months and inactive disease from 6 months and onwards.ResultsFrom October 2009 to April 2014, 95 children, of which 33% boys, were enrolled: 32 in arm 1, 32 in arm 2 and 31 in arm 3. Baseline median (IQR) age was 8,6 (4,5-12,9) years. 38% were ANA positive, 12 patients had oligoarticular disease, 68 patients oligoextended/polyarticular JIA and 15 patients had JIA with psoriasis. 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, number of active joints 8 (5-12), number of joints with limited range of motion 3 (1-5), CHAQ score 0,88 (0,63-1,5). In arm 1, 17/32 had started with MTX, the others with SSZ. One patient in arm 1 (lost to FU) and 1 in arm 3 (uveitis) dropped out before 3 months.By 3 months ACRpedi50 was reached by 10/32 (31%),12/32 (38%) and 16/30 (53%) (p=0,19) and ACRpedi70 was reached by 8/32 (25%), 6/32 (19%) and 14/30 (47%) in arms 1-3, respectively (p=0.04).Toxicity (mainly gastrointestinal complaints) was similar in the treatment arms. Three short hospital admissions (serious adverse events) were reported. In 2/32, 1/32 and 2/30 patients MTX dose was reduced or switched to subcutaneous and 3/15 patients stopped SSZ after 6 weeks because of nausea, malaise, headache.ConclusionsAfter 3 months of initial treatment in a three-armed strategy trial, patients with recent onset JIA achieve more clinical improvement (significantly more ACRpedi70) on initial combination therapy with MTX and etanercept than on initial MTX or SSZ monotherapy. Numerically, response to initial treatment with MTX and prednisolone bridging seems more effective than monotherapy and less effective than combination with etanercept, but these differences were not statistically significant.Disclosure of InterestP. Hissink Muller Grant/research support from: Pfizer, D. Brinkman: ...
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