Introduction
Methods
Methods for qualitative patient interviews to develop a patient-centric conceptual model
Participants for qualitative interviews
Interview conduct
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“Could you please start by telling me about the first signs or symptoms of [participant’s term for RP] you noticed?
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“Does the [patient-reported sign/symptom] have any impact on your daily life? If so, how?”
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“Have there been any changes to your daily life because of [participant’s term for RP]? If so, can you please describe?”
Data handing and analysis
Methods for the Phase 2 study KPL-914-C001
Participants
Assessments
Procedure and analyses
Results
Qualitative patient interviews
Symptom or impact domain reported by participant | Descriptiona | Frequency of participant reportsb |
---|---|---|
(N = 10) | ||
n (%) | ||
Symptoms | ||
Chest pain | Described as sharp, stabbing, dull, or aching pain or pressure in the chest, which can radiate to the neck and shoulders | 10 (100.0%) |
Tiredness | Described as physical exhaustion lasting a few days, which may co-occur with shortness of breath and affect one’s activity level | 8 (80.0%) |
Shortness of breath | Described as difficulty breathing and losing breath quickly, similar to a feeling of suffocation | 7 (70.0%) |
Fever | Described as a low-grade fever that can include hot flashes or chills | 6 (60.0%) |
Heart palpitations | Described as the heart beating rapidly and arrhythmically and causing discomfort | 5 (50.0%) |
Chest pressure | Described as discomfort or heaviness in the chest | 3 (30.0%) |
Cough | Described as uncomfortable and painful sporadic coughing episodes triggered by a tickling feeling | 3 (30.0%) |
Swelling | Described as swollen feet and legs that feel tight and bloated, possibly associated with lack of circulation | 2 (20.0%) |
Abdominal pain | Described as intense pain above the navel | 1 (10.0%) |
Bone pain | Described as bearable pain in the bones, feeling like soreness in the upper back | 1 (10.0%) |
Difference in breathing | Described as difficulty breathing, both as difficulty inhaling deeply and breathing deeper and longer than usual | 1 (10.0%) |
Flutters | Described as an uncomfortable sensation of the heart beating rapidly | 1 (10.0%) |
Neck pain | Described as sharp nerve pain in the neck affecting neck mobility | 1 (10.0%) |
Impact domains | ||
Activities of daily living | Described in the following ways Inability to complete plans and daily activities Inability to begin or complete household tasks, such as cleaning, cooking, and/or yard work Impacts on driving (or fear of driving due to symptoms) Diet and lifestyle changes Inability to go shopping | 9 (90.0%) |
Physical impacts | Described in the following ways | 8 (80.0%) |
Difficulty exercising and restrictions on exercising | ||
Feeling dizzy (due to shortness of breath) | ||
Inability to lay down (due to pain and shortness of breath) | ||
Feeling the need to rest (due to heart palpitations) | ||
Psychological impacts | Described in the following ways Feeling scared because of symptoms Feeling depressed because of symptoms Feeling anxious, worried, or concerned because of chest pain Not feeling normal and wanting to feel normal (due to symptoms) Annoyance due to symptoms Feeling like a burden to others Feeling miserable due to chest pain | 8 (80.0%) |
Sleep impacts | Described in the following ways Waking up frequently or suddenly after falling asleep (associated with chest pain and shortness of breath) | 7 (70.0%) |
Social impacts | Described in the following ways Not being able to go out with friends or attend events | 6 (60.0%) |
Relationship impacts | Described in the following ways Not being able to go to family events Emotional distance or less activity with significant other Feeling distant from family Not being able to support family as much | 6 (60.0%) |
Work or school impacts | Described in the following ways Not being able to go to work Not being able to work as much or as effectively Saving up sick hour to take off when experiencing symptoms Feeling less comfortable with coworkers | 6 (60.0%) |
Hobbies or leisure impacts | Described in the following ways Inability to travel or go on vacations Inability to attend church | 4 (40.0%) |
Mobility impacts | Described in the following ways Impact on climbing stairs Difficulty writing due to chest pain experienced into the shoulder Shoes feeling uncomfortably tight due to swelling of feet | 3 (30.0%) |
Financial impacts | Described in the following ways Increased co-pays due to the condition | 1 (10.0%) |
Romantic impacts | Described in the following ways Not feeling intimate with significant other as a result of symptoms | 1 (10.0%) |
PROMIS GH Items | Reported by patients during qualitative interviews | Representative patient quotes from N = 10 qualitative interviewsa |
---|---|---|
Item 1: In general, would you say your health is | ✓ | [INTERVIEWER]: … has this heart inflammation caused any psychological impacts or impacted the way you feel? |
[PATIENT]: I know it sounds crazy to say, but sometimes I just want to be normal. But what is normal?.… Because I think it's more of a hindrance. Like, I feel like my body's falling apart | ||
Item 2: In general, would you say your quality of life is | ✓ | [INTERVIEWER]: … has anything changed since … you first visited the doctor for those symptoms [sharp chest pain and trouble breathing]? |
[PATIENT]: Uh, my quality of life has.… I would do, like, walks. Um, I would walk three miles a day. And now I – sometimes I can't even get started when, um, when I have the symptoms | ||
Item 3: In general, how would you rate your physical health? | ✓ | [INTERVIEWER]: Are there any other impacts that you experience to your life? |
[PATIENT]: Um, I used to be able to ride a bike.… And that I can't do as much | ||
Item 4: In general, how would you rate your mental health, including your mood and your ability to think? | ✓ | [INTERVIEWER]: … how bad would you say it is [patient described symptoms as ‘sharp stabbing in the chest’] … if you had to describe, you know, how severe or bad it is? |
[PATIENT]: I mean, I'm pretty much just not the same person.… I guess I'm just a miserable person. Even my wife tells me I'm a miserable person to be around when – when I'm going through these episodes | ||
Item 5: In general, how would you rate your satisfaction with your social activities and relationships? | ✓ | [INTERVIEWER]: Is there anything else that you kind of changed, or, um, stopped doing, or do less now, because of the pericarditis? |
[PATIENT]: I used to go out, you know, with friends, stay out late at night. That I don't do anymore | ||
[INTERVIEWER]: … would you say that it [recurrent pericarditis] affects your ability to engage in social or leisure in your life? | ||
[PATIENT]: … it has stopped me from socializing with family gatherings. You know, I just – I'm too tired to even get ready and go out and do a 3-day weekend | ||
Item 6: In general, please rate how well you carry out your usual social activities and roles. (This includes activities at home, at work and in your community, and responsibilities as a parent, child, spouse, employee, friend, etc.) | ✓ | [INTERVIEWER]: are there specific examples that you're thinking of that, when you feel tired, you can't do as well? |
[PATIENT]: I just can't finish [mopping and sweeping]. Like, I'll do half the house, and then I'll just have to take – sit down | ||
[INTERVIEWER]: is there any other impact it [recurrent pericarditis] has on your daily life? | ||
[PATIENT]: I mean I can't be active with my grandkids | ||
Item 7: To what extent are you able to carry out your everyday physical activities such as walking, climbing stairs, carrying groceries, or moving a chair? | ✓ | [INTERVIEWER]: How severe would you say that [tiredness feeling] is, in general? |
[PATIENT]: I still kind of tried to work out, because I always tried to be as healthy as possible. And it – it was just too hard for me to do | ||
[INTERVIEWER]: Can you describe what that [fatigue or tiredness] feels like? | ||
[PATIENT]: Um, exhaustion.… I just I love to walk, well, that's eliminated. I don't do much walking. I'm just so tired | ||
Item 8: How often have you been bother by emotional problems such as feeling anxious, depressed, or irritable? | ✓ | [INTERVIEWER]: You mentioned fatigue. Um, can you go into a little bit more detail about that, how that feels – you know, how it affects your day? |
[PATIENT]: Well, when you have the acute episode, you know, you're going through all that pain, and all the other symptoms – palpitations and shortness of breath, and maybe a little low grade fever, and uh, that cough's coming off and on, the stabbing pains – all that is very taxing on your system, you know. And it's really denting. It's depressing to have all these symptoms. And you're fearful, you're extremely fearful | ||
[INTERVIEWER]: …what did the shortness of breath feel like? | ||
[PATIENT]: It's frightening, it's scary. You think you're going to smother. And – and you think you're going to die. It's like you're – you're underwater, and you can't get oxygen | ||
Item 9: How would you rate your fatigue on average? | ✓ | [INTERVIEWER]: which one would you say is the most bothersome symptom and why? |
[PATIENT]: Hmm, the most that bothers me – I would say palpitations. But actually, I don't get those as often as how I'm feeling fatigue. Do you know what I mean? I've gotten to the point where I'm so fatigued that I – I really can't get out of bed | ||
Item 10: How would you rate your pain on average? | ✓ | [INTERVIEWER]: I just wanted to talk to you a bit about your … the sharp, stabbing chest pain you talked about before.… I was just hoping you could describe it a little bit more |
[PATIENT]: I would say sharp pain more like, um, like if, like if a elephant's sitting on me so I'm suffocating | ||
[INTERVIEWER]: I'd like to talk a little bit more in detail about, um, each of these things that you've mentioned … so, you said the chest pain, um, sort of feels like a stabbing? | ||
[PATIENT]: Yes, stabbing pain.… Like needles |
Phase 2 study rilonacept
Characteristic | Active recurrence (A-RP) | Not symptomatic, Corticosteroid-dependent (CSD-RP) |
---|---|---|
N = 16 | N = 9 | |
Age (years) (Mean ± SD [range]) | 39.8 ± 10.52 (26–58) | 48.2 ± 8.56 (36–62) |
Gender (% female [n]) | 75.0% (n = 12) | 33.3% (n = 3) |
Race (% white [n]) | 81.3% (n = 13) | 100% (n = 9) |
BMI (kg/m2) (Mean ± SD [range]) | 31.99 ± 7.51 (23.4–52.7) | 28.97 ± 4.68 (22.5–34.3) |
Duration of disease (years) (Mean ± SD [range]) | 2.6 ± 2.13 (0.2–7.9) | 1.4 ± 0.97 (0.6–3.4) |
Number of prior recurrences (median, [range]) | 2 (1–8) | 3 (2–5) |
Baseline NRS Pain Rating 0–10 (Mean ± SD [range]) | 4.6 ± 1.82 (2–8) | 1.4 ± 1.51 (0–5) |
Baseline CRP values (mg/dL) (Mean ± SD [range]) | 3.8 ± 5.30 (0.09–19.84) | 0.19 ± 0.11 (0.05–0.36) |
Concomitant medications at baseline | ||
Aspirin (n [%]) | 0 (0%) | 2 (22.2%) |
NSAID (n [%]) | 7 (43.8%) | 5 (55.6%) |
Colchicine (n [%]) | 12 (75.0%) | 8 (88.9%) |
CS (n [%]) | 6 (37.5%)a | 9 (100.0%)b |
PROMIS GH item/ domaina | Active recurrence (A-RP) | Not symptomatic, Corticosteroid-dependent (CSD-RP) | ||||||
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Baseline (n = 16) | End of TP visit (n = 15) | End of EP visit (n = 15) | ES (95%CI)b | Baseline (n = 7) | End of TP visit (n = 9) | End of EP visit (n = 8) | ES (95%CI)b | |
GPH | 39.94 ± 8.94 | 51.35 ± 7.96 | 51.32 ± 6.56 | 1.44 (0.65 to 2.23) | 43.30 ± 5.31 | 45.09 ± 4.06 | 46.81 ± 9.27 | 0.46 (− 0.57 to 1.48) |
Item 3: physical health | 2.6 ± 0.96 | 3.2 ± 1.01 | 3.5 ± 0.83 | 1.00 (0.25 to 1.75) | 2.8 ± 0.46 | 3.1 ± 0.33 | 3.0 ± 0.93 | 0.27 (− 0.75 to 1.29) |
Item 7: physical activities | 3.3 ± 1.39 | 4.4 ± 1.06 | 4.1 ± 1.03 | 0.65 (− 0.07 to 1.37) | 3.4 ± 0.74 | 3.3 ± 0.87 | 3.8 ± 1.04 | 0.44 (− 0.59 to 1.46) |
Item 9: fatigue | 3.1 ± 0.96 | 3.7 ± 0.49 | 3.7 ± 0.82 | 0.67 (− 0.05 to 1.39) | 3.1 ± 0.69 | 3.2 ± 0.44 | 3.4 ± 1.06 | 0.33 (− 0.69 to 1.35) |
Item 10: pain | 4.8 ± 1.88 | 0.6 ± 1.18 | 0.5 ± 1.13 | -2.69 (− 3.66 to − 1.72) | 1.7 ± 1.60 | 1.0 ± 1.32 | 1.4 ± 2.50 | -0.14 (− 1.16 to 0.87) |
GMH | 44.50 ± 10.48 | 50.13 ± 11.33 | 50.54 ± 11.00 | 0.56 (− 0.16 to 1.28) | 46.49 ± 7.77 | 47.91 ± 5.51 | 50.66 ± 6.30 | 0.59 (− 0.44 to 1.63) |
Item 2: quality of life | 3.0 ± 1.03 | 3.6 ± 1.06 | 4.0 ± 1.00 | 0.98 (0.24 to 1.73) | 3.3 ± 1.04 | 3.6 ± 0.73 | 3.4 ± 0.74 | 0.11 (− 0.90 to 1.13) |
Item 4: mental health | 3.3 ± 1.13 | 3.7 ± 1.23 | 3.6 ± 1.12 | 0.27 (− 0.44 to 0.97) | 3.4 ± 0.74 | 3.6 ± 0.73 | 3.9 ± 0.83 | 0.63 (− 0.41 to 1.67) |
Item 5: social activities and relation-ships | 3.1 ± 1.34 | 3.7 ± 1.18 | 3.6 ± 1.12 | 0.40 (− 0.31 to 1.12) | 3.1 ± 0.83 | 3.3 ± 0.50 | 3.6 ± 0.92 | 0.57 (− 0.47 to 1.60) |
Item 8: emotional problems | 3.1 ± 1.41 | 3.5 ± 1.36 | 3.4 ± 1.12 | 0.23 (− 0.47 to 0.94) | 3.4 ± 0.98 | 3.3 ± 0.71 | 4.0 ± 0.53 | 0.78 (− 0.27 to 1.83) |
Items that are not included in above domains | ||||||||
Item 1: general health | 2.9 ± 0.72 | 3.5 ± 0.83 | 3.6 ± 0.91 | 0.82 (0.12 to 1.59) | 2.9 ± 0.64 | 3.1 ± 0.33 | 3.1 ± 0.64 | 0.31 (− 0.71 to 1.33) |
Item 6: social activities and roles | 3.1 ± 1.09 | 3.5 ± 1.25 | 3.5 ± 1.13 | 0.36 (− 0.35 to 1.07) | 2.9 ± 0.99 | 3.1 ± 0.93 | 3.5 ± 0.93 | 0.63 (− 0.41 to 1.67) |