Summary Background Janus kinase (JAK)‐mediated cytokine signalling contributes to local and systemic inflammation in hidradenitis suppurativa (HS). Objectives To describe the safety and efficacy results from two multicentre phase II trials of the JAK1 inhibitor INCB054707 in patients with moderate‐to‐severe HS. Methods Patients received open‐label INCB054707 15 mg once daily (QD; Study 1) or were randomized to INCB054707 30, 60 or 90 mg QD or placebo (3 : 1 within each cohort; Study 2) for 8 weeks. Eligible patients were aged 18–75 years and had moderate‐to‐severe HS (Hurley stage II/III disease), lesions present in at least two anatomical locations, and a total abscess and inflammatory nodule count ≥ 3. The primary endpoint for both studies was safety and tolerability. Secondary endpoints included HS Clinical Response (HiSCR) and other efficacy measures. Results Ten patients were enrolled in Study 1 (15 mg INCB054707) and 35 in Study 2 (INCB054707: 30 mg, n = 9; 60 mg, n = 9; 90 mg, n = 8; placebo, n = 9). Overall, 70% of patients in Study 1 and 81% of patients receiving INCB054707 in Study 2 experienced at least one treatment‐emergent adverse event; 30% and 42% of patients, respectively, had at least one treatment‐related adverse event. Among the evaluable patients, three (43%) in Study 1 and 17 (65% overall: 30 mg, 56%; 60 mg, 56%; 90 mg, 88%) receiving INCB054707 vs. 4 patients (57%) receiving placebo in Study 2 achieved HiSCR at week 8. Conclusions INCB054707 was well tolerated, with responses observed in patients with moderate‐to‐severe HS. The safety and efficacy findings from these studies demonstrate proof of concept for JAK1 inhibition in HS. The studies are registered on http://clinicaltrials.gov (NCT03569371 and NCT03607487).

Although many reports in the literature describe the use of endoscopic retrograde cholangiopancreatography (ERCP) in children, few mention the therapeutic application of the technique in treating pediatric patients with pancreatic or biliary pathology. We report here on our two-year experience, consisting of 42 ERCPs conducted in 25 patients for a variety of pancreaticobiliary indications. Ages ranged from 22 months to 19 years. Five patients had normal studies; the remainder were diagnosed with a variety of pancreatic and or biliary anomalies. Seventeen patients had therapeutic interventions consisting of sphincterotomy (n = 12), dilation (n = 6), or stent placement (n = 9), with many of these procedures being done in combination. Five patients had mild complaints of abdominal pain or vomiting immediately after ERCP, but no major technical or medical complications were encountered. We find that therapeutic ERCP can be safely performed in children, but only by highly experienced endoscopists familiar with these specialized procedures.

BackgroundTV-1106 (Teva Pharmaceuticals) is a genetically fused recombinant protein of human GH (hGH) and human serum albumin, in development for treatment of GH deficiency (GHD). TV-1106 is expected to have an extended duration of action compared to daily GH treatment and may enable a reduction in the frequency of injections and improve compliance and quality of life for adults and children requiring GHD therapy.ObjectiveTo assess the safety, local tolerability, pharmacokinetics and pharmacodynamics of TV-1106 following single s.c. injections in healthy male volunteers.MethodsSubjects (n=56) were assigned to one of seven ascending dose groups (3–100 mg) and received either a single dose of TV-1106 (n=6) or placebo (n=2) by s.c. injection.ResultsEighteen subjects reported 43 adverse effects (AEs), which were mild to moderate; no serious AEs (SAEs) occurred. In 50, 70 and 100 mg groups there were mild to moderate increases in heart rate and systolic blood pressure that significantly correlated with higher levels of IGF1. TV-1106 showed pharmacokinetic characteristics of a long-acting hGH as demonstrated by a terminal elimination half-life of 23–35 h, delayed time of peak concentration, and systemic levels seen up to 7 days after dosing. IGF1 levels increased in a dose-dependent manner, before reaching a plateau, with levels above baseline extending beyond 7 days post dose.ConclusionSingle administration of TV-1106 up to 100 mg was safe in healthy volunteers. Pharmacokinetics and pharmacodynamics support once-weekly administration in patients with GHD.

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We tested a new patient-reported outcome tool, the Hidradenitis Suppurativa Quality of Life (HiSQoL) questionnaire, which is designed to measure quality of life (QoL) in adults with hidradenitis suppurativa (HS). The HiSQoL demonstrated validity and reliability, and may be a useful tool for measuring QoL, especially in clinical trials testing new drugs in HS.