Table 5

Outcome domains identified through phase 1 with lay definitions

ThemesOutcomeLay definitions
Fistula response to treatment (symptoms)≥50% tracts not draining on clinical examinationMore than half the openings on the bottom are dry and not oozing anything.
Closure of internal openingThe hole inside the bottom (as opposed to on the buttock skin surface) closes. This must be assessed by a doctor.
Rectal mucosal healingAn assessment of the last part of your intestine in clinic assessed by inserting a small probe into your bottom.
Complete fistula healing assessed clinicallyAn assessment of the bottom in clinic where all the opening/holes on the skin have closed on ≥2 consecutive clinic appointments (ie, assessed more than once).
Partial fistula healing assessed clinicallyAn assessment of the bottom in clinic where there is a decrease in the size/number of fistula and a reduction in drainage.
Closure of all the external openings on clinical examinationAn assessment of the bottom in clinic where all the opening/holes on the skin have closed on a single examination (ie, assessed once).
Clinical assessment of drainage either spontaneously or on gentle finger pressureAn assessment of the bottom in clinic where the doctors press around the openings on the bottom to look for discharge and also ask the patient about the drainage from their fistulae.
Local perianal inflammation/induration assessed clinicallyAn assessment of the bottom looking for acute inflammation around the fistula (swelling and redness) and chronic inflammation (scarring and shrinking of the anal opening).
A validated score to assess perianal disease activity, for example, Perianal Disease Activity IndexA scoring system used to assess whether the perianal disease is active and flaring up or stable.
Development of perianal features of Crohn’s disease (other than fistula)Developing skin tags, anal stenosis (narrowing), anal fissures, ulcers or cancer.
Patient-reported reduction in fistula drainageThe patient saying there has been a decrease in the oozing/draining from the openings on the bottom.
Direct impact of fistula on the patientIncontinence to windUnable to stop wind/flatulence/gas escaping from your bottom.
Mucus leakageUnable to stop mucus coming out from your bottom.
Recurrence of fistulaThe same fistula hole that closed opens up again (the hole inside the bottom or the hole on the buttock skin surface).
Development of a new fistulaA new perianal fistula develops in another place; that is, a new hole forms on the buttock skin surface or deeper inside the bottom after the intervention.
Perianal abscess on clinical assessment after interventionAn abscess (collection of pus/infection) or lumps in the bottom area that forms after treatment.
Wound infectionIncreasing pain, redness, swelling in the wound requiring antibiotics (without an abscess).
An incontinence scoreA scoring system used over time to assess change in continence/bowel motion (consistency/frequency).
Incontinence to liquid stoolUnable to stop liquid stool escaping from your bottom.
Incontinence to solid stoolUnable to stop stool/faeces escaping from your bottom.
Pads for continence/leakageNeeding to use pads inside underwear to soak up liquid discharge/oozing from the fistula.
Plug for continence/leakageNeeding to use anal plugs to soak up liquid discharge/oozing from the fistula.
#Discrimination between passing stool and gasUnable to know whether you have passed wind/flatulence/gas or whether you have passed faeces/stool.
Tenesmus or incomplete evacuationFeeling like you need to go to the toilet all the time (even if just been).
Anal bleedingBlood coming out of the bottom area (either from the fistula or the bowel).
Anal painPain around the bottom.
Increased frequency of loose stoolRunnier bowel motion and having to empty bowels more often than before.
Perianal related hospitalisationBeing admitted to hospital because of your perianal Crohn’s disease, such as an abscess.
Surgical reinterventionAnother operation is needed after the first treatment.
Faecal diversion or proctectomyOperation to remove the rectum (last part of the bowel) and/or having a stoma bag fitted.
Faecal urgencyInability to delay going to the toilet/defecation for 15 min.
Impact on the patient as a personLifestyle alterations (pain/restriction of activities)Change in lifestyle because of the fistula.
Limitation to moderate activitiesDifficulty performing tasks such as light housework.
Limitation to vigorous activities (eg, running, lifting heavy objects, participating in strenuous sports)Unable to run, lift heavy objects, participate in strenuous sports.
Change in general health—physicalChange in physical ability to do things.
#A quality of life score, for example, Short Form Survey 36A scoring system that patients fill out to assess the various aspects of a person’ s life.
Change in general health—psychologicalChange in thought and feelings.
Lethargy and fatigueExhausted, tired because of the fistula.
Social interaction avoidanceStop leaving the house unless you have to (going to work and medical appointments but not going to see friends or going to parties/celebrations).
Anxiety and worriesAnxious or worried about impact of the fistula.
Feeling depressed and down, and hopelessnessFeeling depressed and down, and hopelessness.
Irritable, frustrated and angryFeeling irritable, frustrated and angry.
Concern over further interventionConcerns over needing more treatment (having just had one type of treatment).
Sleep disturbanceHaving to get up at night due to toileting needs, soiling sheets, underwear change and so on.
Modifying how you walk, sit or stand because of your fistula*Sitting on one buttock rather than both, standing as sitting is too painful or having to walking with your legs wider apart.
Modifying travel*Choosing modes of transport depending on access to the toilet (eg, train) or planning car journeys around toilet stop-offs.
Body detachment*Feeling ‘medicalised’ and that rather than yourself you are a ‘body’ on which medical treatment is performed.
A feeling of being unhygienic*Feeling unclean, dirty and unhygienic (rather than actual being unclean, dirty and unhygienic).
Concerns about and impact on fertility, birth, parenthood and family*Worried about getting pregnant in the first place, about actually going through labour, keeping up with busy children and inability to give them everything because of limitation of the disease (eg, not being able to go for long walks).
Restriction of sexual activityUnable to have sex or be physically close to someone.
Physically restricted in self careUnable to wash, get dressed or look after yourself without help from someone else because of the fistula.
Patient perception of continence compared with others or baselineA difference/change in continence compared with other people or how you used to before the fistula.
Tolerability of treatmentAn overall assessment of how ‘acceptable’ a treatment is; for example, does it have so many side effects that you want to stop it?
Decisional regret of treatment choiceA measure of regret of choosing a specific treatment option whether (medical or surgical).
Avoidance of intimacy*Avoiding getting too close with another person (hugging, sitting next to each other and so on) due to fears that you smell or they might know that you have a fistula. This includes getting into new relationships.
Unable attend school/workCannot go to school/college/university or do your usual job because of perianal fistula due to need to be off at short notice.
Change in lifestyle based on toileting needsGo out less or only go to places where you know there is a clean toilet and washing facilities because of perianal fistula. Or take spare clean underwear and wipes with you when you go out.
Restricted in what you wear*Being unable to wear tight clothing and wearing baggy clothes to reduce pain, conceal bulging pads or bulky gauze inside underwear. Also wearing dark clothing to conceal stains.
Embarrassment and feeling isolated*Feeling conscious of the fistula, which subsequently affects the way I walk/behave/interact with the world, which is obvious to others and leaves me embarrassed so that I alter what I do.
Assessment with imaging techniques (scans)#MRI assessment of fistula volumeCalculating the size of fistula on an MRI scan to generate a number, and then comparing the size/number over time.
Fistula response on endoanal ultrasoundA rectal probe is inserted into the bottom to look for and assess the fistula using an ultrasound machine to see if the fistula is better/worse/the same.
Abscess on MRI following treatmentA collection of pus on MRI scan.
T1 enhancement on MRIA specific way to assess inflammation within a fistula on a MRI (it adds time to a normal MRI but allows doctors to better decide what is fistula and what is blood vessel).
Hyperintensity on T2-weighted MRIA specific way to assess inflammation within a fistula on an MRI—this is standard care.
MRI assessment of rectum (proctitis)Looking for inflammation of the rectum (last part of the intestine/bowel) on MRI.
Fistula response on MRI imagingThe fistula looks ‘better’ or ‘about the same’ or ‘worse’ on MRI.
#An activity based MRI score, for example, Van Assche ScoreA scoring system used by radiologists (MRI doctors) to assess whether a fistula is ‘active’, that is, acute inflammation or nearer to the other end of the scale of healing.
Fistula response to treatment (tests)Time to loss of response to medical treatmentsThe length of time taken before you develop resistance to a medication (biologicals/anti-TNF).
Objective blood markers of inflammationBlood tests looking for inflammation.
C reactive proteinA specific blood test looking for inflammation.
Non-inflammatory blood markers (related biologicals) Blood tests looking for other things, such as anaemia, B12 levels and so on.
Instability of weight (assessed by body mass index (BMI))Putting on weight or losing weight, assessed using BMI (a score based on weight and height).
FeverFeeling ‘hot’ and feverish/getting ‘the chills’ with a high temperature.
Safety implications related to treatmentNausea or vomitingFeeling sick or vomiting after treatment.
DeathDeath as a result of a treatment.
Rash or other skin/hair problemsChanges in the skin (rash, dryness, acne and so on), hair thinning or hair loss.
Allergic reactionA reaction to a treatment (ranging from a rash to swelling of the throat).
Cardiorespiratory complicationsComplications of treatment related to cardiovascular system—heart attacks and abnormal heart rhythms.
Neurological complicationsComplications of treatment related to nervous system—visual symptoms, headaches and nerve damage.
Urinary complicationsComplications of treatment related to urinary system—waterworks infections, damage to the any of the anatomical structures during surgery.
Grading system for surgical complications, for example, Clavien-DindoA generic validated grading system of all complications related to surgery (covers all systems—cardiovascular, neurological and so on).
Safety (adverse events) and toxicityMeasuring how safe and tolerable a given treatment is.
Surgical complications specific to the surgical procedure (eg, plug extrusion)Specific surgical complications, for example, plug extrusion (following anal plug insertion).
#Medical complications specific to the immunosuppression (eg, opportunistic infections and cancers)Increased risk of cancer (eg, lymphoma) and an increased risk of any infection as a result of being on an immunosuppressive medication (anti-TNF/biologicals/thiopurines).
Impact on the patient over timeDuration of healing/improvementHow long the treatment helped you to feel better for.
Biological-free remissionPeriod of time not needing to take biologicals/anti-TNFs (eg, infliximab/remicade, humira/adalimumab).
Cost-effectivenessA measure of how effective the treatment has been but also factoring in the cost of this treatment.
Increasing analgesia*Needing more painkillers to get through the day.
Antibiotic-free remission*Period of time not needing to take antibiotics.
Use antidiarrhoeal drugsTaking medication to make the stool less runny (eg, loperamide).
Recovery time after interventionLength of time off work/study after treatment intervention.
Financial implications*Financial hardship including loss of income, career stagnation, extra expenses, for example, buying pads, clean underwear and so on.
OriginFrom SR (n=76)
Added from patients (n=11)*
#Added from SMG (n=5)
Excluded by the SMG (n=14)Faecal calprotectinNot specific to perianal Crohn’s disease and more to luminal disease.
Steroid-free remissionNot specific to perianal Crohn’s disease and more to luminal disease.
Abdominal discomfortNot specific to perianal Crohn’s disease and more to luminal disease.
Dietary supplements as oral intake lowNot specific to perianal Crohn’s disease and more to luminal disease.
A validated Crohn’s disease activity scoreNot specific to perianal Crohn’s disease and more to luminal disease.
Remission of Crohn’s diseaseNot specific to perianal Crohn’s disease and more to luminal disease.
Identification of endoscopic signs suggestive of failure of treatment (relating to systemic/overall Crohn’s disease)This relates to luminal relapse, and we have included assessment of the rectal mucosa separately.
MRI features (luminal)Not specific to perianal Crohn’s disease and more to luminal disease.
Abdominal massNot specific to perianal Crohn’s disease and more to luminal disease.
Extraintestinal manifestations of Crohn’s diseaseNot specific to perianal Crohn’s disease and more to luminal disease.
Comparison with other individuals (luminal)Not specific to perianal Crohn’s disease and more to luminal disease.
HaemorrhageNot specific to perianal Crohn’s disease and more to luminal disease.
Feasibility of techniqueNot specific to perianal Crohn’s disease and more to luminal disease.
Decrease in size of fistulaDefinition unclear and not defined within the study. Likely to have covered through other forms of clinical assessment (consensus to remove).
  • *Added from patients (n=11).

  • #Added from SMG (n=5).

  • SMR, study management group; SR, systematic review; TNF, tumour necrosis factor.