What drives the decision to optimise biological treatment in children and youngsters with juvenile idiopathic arthritis? A discrete-choice experiment.

OBJECTIVE: To analyse factors involved in the decision to optimise biologics in juvenile idiopathic arthritis. METHODS: A "discrete-choice" methodology was used. In a nominal group meeting, factors which may influence physicians' decisions to optimise biological dose were identified, together with decision nodes. 1000Minds® was used to create multiple fictitious clinical scenarios based on the factors identified, and to deploy surveys that were sent to a panel of experts. These experts decided for each item which of two clinical scenarios prompted them to optimise the dose of biologic. A conjoint analysis was carried out, and the partial-value functions and the weights of relative importance calculated. RESULTS: In the nominal group, three decision nodes were identified: (1) time to decide; (2) to maintain/reduce or prolong interval; (3) what drug to reduce. The factors elicited were different for each node and included patient and drug attributes. The presence of macrophage activation syndrome (MAS), systemic involvement, or subclinical inflammation made the decision easier (highest weights). The presence of joints of difficult control and year of debut influenced the decision in some but not all, and in different directions. Immunogenicity, adherence, and concomitant treatments were also aspects taken into account. CONCLUSIONS: The decision to optimise the dose of biological therapy in children and youngster can be divided into several nodes, and the factors, both patient and therapy-related, leading to the decision can be detailed. These decisions taken by experts may be transported to practice, study designs, and guidelines.

Biological therapy in refractory chronic nonbacterial osteomyelitis: A case series of 19 patients.

INTRODUCTION: Chronic non-bacterial osteomyelitis (CNO) represents an autoinflammatory bone disorder. Currently there are no standardized diagnostic or treatment guidelines. The objective of the study is to describe our experience with biological therapy in children with the disease. METHODS: Retrospective chart review of patients with CNO treated with biological therapy followed at two tertiary hospitals from January 2007 to April 2020. Biologicals were started in most patients due to persistent disease activity after receiving standard therapy with at least 2 drugs (NSAIDs and corticosteroids and/or pamidronate). RESULTS: Twenty-five patients were diagnosed with CNO. Out of those, 19 patients (15 females) failed conventional therapy. The mean age at diagnosis was 8.8±2.9 years and the mean diagnostic delay was 6.9±8.3 months. All patients presented with bone pain and 6/19 also had fever. The most frequently affected bones were femur (9 patients), followed by clavicle, tibia and vertebrae (6, 6 and 5 patients respectively). Nine children had skin lesions. C-reactive protein was elevated in 13/19 patients (mean 20.2mg/L±11.7) and ESR in 16/19 (mean 48mm/h±29). All patients received nonsteroidal anti-inflammatory drugs, 15/19 pamidronate, 10/19 corticosteroids and 19 anti-TNF-therapy. At the last follow-up visit, 10/19 patients were still on biological therapy (8 adalimumab, 2 infliximab) and 18 out of 19 remained asymptomatic. In regards to adverse effects, one patient receiving infliximab developed S. aureus osteomyelitis and another cutaneous leishmaniosis. CONCLUSIONS: This research emphasizes that anti-TNF-therapy represents an effective and safe alternative for patients with CNO refractory to conventional treatments.