Table 3 Example responses during concept elicitation, in patients’ own words

Headache

I got there [to maintenance], after 2 weeks then they finally subsided and I just, I told my doctor that was my mercy cry. I just could not force myself to do it again. I was just done. The headaches were debilitating. I couldn’t function. I couldn’t think straight. I just didn’t want to do anything. And then when you add in the extremity pain, it was just… you were this big ball of pain that just, you just didn’t want to do anything but try to zone out and do the best you could to cope. It took all my energy to cope with the side effects. (Patient 2)

Super mild. It’s also associated with while I’m at work […] But yeah, very tolerable with Tylenol. More of like…I would be able to tolerate it without Tylenol. I just need my brain to be at its best while I’m working, so I just take it anyway. (Patient 12)

Jaw pain

That usually comes with the first bite or drink of something and it’s real sharp and it hurts but once it fades down, it goes away. And it usually only happens that first bite. […] When it’s happening, you think it’s severe because it hurts that bad. But it only lasts for a few seconds really, so I think it’s more bothersome than severe. I mean, it’s not by any means, but it is what it is and it doesn’t last. I can tolerate that. (Patient 20)

Nausea

That seemed to start right when I started the medication feeling kind of sick to my stomach, and I think it happened after the first titration, I felt it a little bit more. […] And then I went up and the medication again and it felt a little bit more nauseated. I wasn’t throwing up, but just feeling like overall yuck. And like it subsided after a few months. (Patient 17)

Diarrhea

When it hits when I’m about to go out, it makes me late. And then I got to find a toilet while I’m out in the street. And it’s kind of embarrassing being in the bathroom and stinking it up. And they know it’s you, because it’s only 2 stalls or something like that, or it’s one stall, and if somebody’s trying to get in that 1 stall but you’re there stuck. It’s going out in public and actually have to go and race to toilets, so you learn where the toilets and the clean bathrooms are with this disease. You can’t be one of them people that don’t use public washrooms or you’ll be stuck in the house. (Patient 4)

Muscle pain

I started noticing more muscle pain right after I started doing the titration, I guess. Just sore, achy muscles. My calves. […] It was pretty constant, the achy muscles and joints during this whole process, and I still have achy muscles and joints. And there’s…sometimes I’d get muscle cramps in my calves, sometimes my hamstring. I get a muscle cramp. […] More severe during titration than now. It’s just nagging muscle aches now. […] It’s not an everyday thing. It’s more of a nagging. Like everything else, just a nagging. If it gets really sore, I’ll just take some Tylenol. (Patient 1)

Flushing

It’s just like, I feel like I’ll get red in the face or my arms mostly and sometimes in my chest. Like hot, it feels hot… and I start sweating. It’s just uncomfortable. (Patient 18)

Pain in the extremities

I think every time I’d titrate up it was 1 or 2 days of some leg pain and so I’d put my legs up for maybe 20 min and then I was fine. After those 2 days, there was nothing. (Patient 5)

Joint pain

Sometimes I’ll just wake up in the middle of the night and all of my major joints—my shoulders, my hips, elbows, everything—all my major joints are…I have to take Tramadol. I don’t take medication for fun but I have to take an actual opioid, which I don’t love, because it’s that painful and Tylenol doesn’t…PAH patients can’t take NSAIDs, so it’s Tylenol or an opioid pain medication. So that sucks. (Patient 7)

Nasopharyngitis

So at first, you’re very stuffy. It’s hard to explain that. But it’s like you have a really bad head cold, without any of the…snot, I guess is the not-so-nice way to say that. To the point that I remember having to get up in the middle of the night and go just sit in a recliner and try to sleep that way. Because I just couldn’t breathe. (Patient 3)

Pretreatment discussion with healthcare provider

My doctor talked about possible headaches, leg pain, nausea, throwing up, possible diarrhea, all of these different things. (Patient 5)