Medical pathways of children with juvenile idiopathic arthritis before referral to pediatric rheumatology centers.

OBJECTIVE: A better understanding about the referral pathway of patients suffering from juvenile idiopathic arthritis (JIA) is required The aim of this study was to describe and analyze time from onset of symptoms to first pediatric rheumatology (PR) visit and the referral pathway of children with incident JIA in two French competence centers. METHODS: From October 2009 to October 2017, new JIA patients were registered in the "Auvergne-Loire cohort on JIA". We collected referral pathway, symptom onset, biological and clinical data at first assessment in PR department. RESULTS: In all, 111 children were included. Median time to first PR visit was 3.3 months [interquartile range (IQR) 1.3, 10.7] with a significant difference between JIA subtypes. After exclusion of systemic JIA, older age at onset of symptoms, and presence of enthesitis or joint pain were significantly associated with a longer time to first PR visit, while joint swelling or limping, abnormal ESR or CRP were associated with a shorter time. The median number of health care practitioners met was 3 [IQR 3, 4]. Orthopedists referred children to a PR center in 64% of cases, pediatricians in 50%, emergency care practitioners in 27% and general practitioners in 25%. Although non-systemic JIAs are not an emergency, 45% were referred to the emergency room. CONCLUSION: Time to first PR visit is rather short compared to other countries but remains too long. Pediatric rheumatologists should offer primary care providers basic training on JIA and fast direct access to PR departments if JIA is suspected.

Challenges of the management of severe hemophilia A with inhibitors: two case reports emphasizing the potential interest of a high-purity human Factor VIII/von Willebrand factor concentrate and individually tailored prophylaxis guided by thrombin-generation test.

Severe hemophilia A is an X-linked bleeding disorder. Immune tolerance induction (ITI) is the best strategy of treatment when patients develop inhibitors. The objective is to illustrate the benefit of a high-purity human factor VIII/von Willebrand factor (VWF) concentrate (Octanate) in the management of ITI. We also wanted to raise the potential interest of laboratory assays such as thrombin-generation test (TGT) and epitope mapping. Two patients were treated during ITI, first with a recombinant FVIII and then with plasma-derived factor VIII without success, and, finally, with Octanate. Bypassing agents were used based on the results of TGT. Epitope mapping was performed during ITI therapy. These observations suggest the potential contribution of Octanate in the management of ITI in difficult cases. The use of bypassing agents can be necessary in prophylaxis or to treat bleedings, and may be guided by TGT results. Epitope mapping is used to describe the inhibitor. This article shows a decrease of the inhibitor directed against the C2 domain after initiation of Octanate. A high-purity human factor VIII/von Willebrand factor concentrate (Octanate) may be a valuable therapeutical option for ITI therapy. TGT and epitope mapping could be of help in the management of ITI.