Fistula

The prospective validation study recruited patients at the investigating centre, a university hospital serving a population of approximately 330 000 people with an established secondary care IBD service providing outpatient and inpatient medical and surgical care.23 (link) Patients were recruited during routine visits to the outpatient clinics prior to consultation with the doctor or specialist nurse, during other treatment-related visits (eg, azathioprine monitoring clinic or infusion visit for biologics) or at the time of admission for inpatient care. Inclusion criteria specified a confirmed diagnosis of IBD on the basis of clinical, endoscopic, radiological and/or histological criteria with disease duration of at least 6 months. Exclusion criteria specified non-English speaking subjects, cognitive impairment or serious active psychiatric disease.
After informed consent, the patients completed the IBD-Control questionnaire and were then asked to complete a questionnaire pack comprising a disease-specific QoL questionnaire (the UK-IBD-QoL),16 (link)
17 (link) a generic health status instrument (EuroQol, EQ-5D-3L),24 (link) and the Hospital Anxiety and Depression Scale.25 (link) The research team undertook a simultaneous assessment of current disease activity, using the Harvey-Bradshaw Index (HBI) for CD 5 (link) and the Simple Clinical Colitis Activity Index (SCCAI) for UC.6 (link) The research team and clinicians were blinded to the results of patient-completed questionnaires.
Where the study visit was taking place at a scheduled clinical review (eg, outpatient attendance), the treating clinician or specialist nurse was asked to complete a questionnaire at the end of the consultation to indicate the current state of IBD (Global Physician Assessment) using a categorical scale (remission, mild, moderate or severe), blinded to patient surveys. All treatment decisions were recorded, capturing whether new therapies were started, existing drug doses changed, therapies discontinued or surgery recommended. The research team reviewed the hospital case records and clinical information systems to extract background clinical information regarding diagnosis, duration of disease, previous hospitalisation and surgery, disease extent, presence of stoma or perianal fistulae, major comorbid illness and current therapy for their IBD.

The primary endpoint was the achievement of Hidradenitis Clinical Response (HiSCR) at week 16 (defined as a ≥ 50% reduction in AN count with no increase in abscess count and no increase in draining fistula count relative to baseline [14 (link)]). Ranked secondary endpoints included the achievement of ≥ 30% reduction and ≥ 1 unit reduction from baseline in Numerical Rating Scale (NRS30) in Patient’s Global Assessment of Skin Pain (PGA Skin Pain) at week 8 or at week 16 among patients with baseline NRS scores ≥ 3; an experience of ≥ 25% increase in AN counts with a minimum increase of 2 relative to baseline during the double-blind period; change from baseline to week 16 in the Dermatology Life Quality Index (DLQI); and change from baseline to week 16 in HS-related swelling, HS-related odor, and HS-related worst drainage assessed based on Hidradenitis Suppurativa Symptom Assessment (HSSA). Additional efficacy endpoints included change from baseline to week 16 in lesion count by lesion type (AN count, abscess, draining fistula, and inflammatory nodule) and the achievement of a DLQI score of 0/1 (indicating disease has no or almost no effect on patient’s quality of life) at week 16.
Safety assessments in the two study periods included monitoring of treatment-emergent adverse events (TEAEs) during the double-blind period (in all patients who were randomized and received at least 1 dose of study drug from baseline to week 16) and during the open-label period (in all patients who received at least 1 dose of study drug from week 16 through the time of study termination).