In Treatment for PNH: Shane's Story

Leigh Clark:    Hi, everyone. It's Leigh Clark, director of patient services with the Aplastic Anemia and MDS International Foundation. Welcome to Podcasts for Patients. Before we get started, I'd like to recognize the generous support of our Diamond Sponsors, Alexion and Novartis, and the generosity of our patients, families, and caregivers who, without their support, this podcast would not be available. So, today I have the pleasure of speaking with Shane. So, welcome Shane, how are you?
Shane Pope:    Hi, Leigh, thanks for having me. I'm doing well, and how are you doing (laughs)?
Leigh Clark:    Doing great, thank you so much for asking. Tell us a little bit about yourself.
Shane Pope:    Yeah, hey folks, I'm Shane, like Leigh said, and I'm from Canada. I'm a 27-year-old, guy living in Canada. I'm an PNH patient. 
Leigh Clark:    Thanks, Shane. Tell me a little bit about your life before your diagnosis of PNH.
Shane Pope:    Yeah. Life was quite different, than it is now. But, life was, was good. I was working full-time. I'm a social worker by profession, so, you know, I was doing a lot of my passion work. That's how I like to call it. I was busy with work. I was going on trips, vacationing with friends, with family, my loved ones. Just really trying to enjoy the little bits of life outside of work and outside of the busyness, of life, and just trying to, you know, take care of myself, my loved ones. And I have two cats, who I adore.
    So, yeah, life was pretty good. And I would consider myself relatively healthy, and I didn't really, necessarily access healthcare much. I'm definitely not someone that goes to the gym or w- (laughing), you know, exercises much. But, I think I was relatively healthy and just kind of lived my life that way. I was someone who loved to dance, who loved just to have a good time when I was out with my friends. And little bit of innocence. I had a little bit of innocence in terms of my health and not knowing what would come next.
Leigh Clark:    What led you to seek medical attention?
Shane Pope:    I actually went down south to Dominican Republic, for my best friend's wedding---super exciting, this was back in April of 2024. Super pumped, first time being there, an all-inclusive resort, so, you know, that comes with its perks. And, the traveling there was fine. Once we got there, we had a really good time. I was actually MCing my best friend's wedding and it was just a lovely time. The weather was great, and, you know, I couldn't have asked for a better experience.
    And it's once I got back to Canada, and I got back into work, it was ab- about a week later after I got back from the trip, I started to have, symptoms that I've really never had before. They were very unfamiliar. And those symptoms were, I was having like abdominal pain that kinda just started randomly. I woke up one morning, I had it. Um, didn't really think much about it. I was like, "Maybe it's something else." Maybe I, I ate something, maybe I caught something from down south. You know, all those questions that are going through your head, but you're not trying to freak yourself out either, right? You're trying to just kind of go with the flow, see if it passes, over the next few days.
    And then it did not pass, and it actually progressively got worse. And then I started to have a fever that I just could not break. It was a fever for multiple days. That's what brought me to the emergency department the first time, was the abdominal pain and fever. I actually tried to access virtual care first, and virtual care actually wouldn't even see me, and they said, "You need to go to the emergency department." And so I went there, 
    and, waited a few hours, 'cause that's just how our healthcare system is here (laughing). But I was eventually seen by the doctor who ordered a CT scan of my abdomen. At that point, prior to even the scan, the things that we were considering were things that could be resolved pretty quickly, maybe acute issues. There was also a consideration of, you know, maybe I did catch something from Dominican.
    So at that point, it sounded like, you know, this might be what it is, and here is the solution to this. But I went for the CT scan and waited for the results. And the doctor came in, and he was like... He looked at me, and he said, "You're not so straightforward. We found blood clots in your liver and spleen." And I probably turned white as a ghost. I was panicked, I was freaked out. I was not expecting to hear that news. I really wanted it to be something else (laughing), that was a lot more easier to manage and, and figure out.
    He didn't have an answer as to why the blood clots were there. I can just hear the tone in his voice. He was concerned, which made me concerned (laughs) and I had my partner next to me, so, so grateful that I had support in that moment, that I can lean on, because there was a lotta questions and a lot of unknowns, and not enough answers at that point. Eventually I was referred to internal medicine, their team who came to see me that same night that I was in the emergency department.
    And they did an assessment. Essentially, after the assessment, they all came together, sat me down, and they were like, "Yeah, you have blood clots, and we don't know what caused these blood clots. These are unprovoked," or what they would consider to be unprovoked. And they said that, you know, the course of action here would, to start me on a, a blood thinner. They took a whole bunch of blood from me. I can't even count how many tubes of blood that they took. And they said they will follow up with me in two to three months with those results.
    I was like, "Okay. I guess that is the, that's what we do. That's how we are, that's how we're going to approach this." I was a little bit naïve at that point, because, again, I hadn't accessed healthcare much prior to that, right? So I thought, you know, I'll go along with what they're saying, and I left. I started the blood thinner. And two days later, I was back in the emergency department with, the pain had worsened significantly. I was still fevering, I was extremely weak, fatigued. I was nauseous. More symptoms had come, and I was like, "I don't know what this means. And I'm scared that I'm going to die." That's what was in my head. I was asking myself over and over, ruminating on it, of like, "What if this means I'm dying?" Right?
    I was 26 at the time, and, you know, as a 26-year-old, have to ask myself that you know, and try to prepare myself for that potential newsit was pretty scary. And ended up going back to the emergency department, my partner took me again, and within 20 minutes, I was seen. It was a different doctor who, his response to my symptoms and what I was experiencing was a lot more, I guess, he was a lot more concerned. He was like, "Okay, we're gonna figure out what's going on. You shouldn't have been sent home the first time. You should still be here, and I'm glad that you came back."
    Again, the initial reaction was I had caught some sort of infection from Dominican Republic. They started me on IV antibiotics, thinking that would help with the fever, which it did, luckily. Also strangely, that it did help with that. 
Leigh Clark:    How did the diagnosis of PNH happen?
Shane Pope:    I was, admitted to hospital, after I went back the second time to the emergency department. And, they kinda looked at me as this kind of, you know, "You're a case to be solved," right? At one point, a resident came in and called me, like, the million-dollar workup, because they had no idea what was going on with me, and how to explain. There was just no answers. And I was admitted to hospital for two weeks. And within that span of two weeks that's when I received my PNH diagnosis.
    Leading up to that it was a lot of blood work, daily blood work--- more tests, more tests. My symptoms were starting to reduce, which was nice. But again, we still didn't know what was happening. And I had another resident who came to see me, who eventually ended up, part of my healthcare team my whole entire stay in the hospital. He came to me and wanted to kinda get a little bit more information about what was happening prior to me going to Dominican. 'Cause at that point, the theory was still that I had an infection.
    And he wanted to know more about what, what was things like months prior to that, right? And it was a, a light-bulb moment for me, it kinda came back to me. And I was like, "Well, I did have very dark urine, months before my trip." And I can't tell you the exact time, but I remember having very dark urine. It was early, early in the morning, I was waking up. And I called my partner in, actually, and I was like, "I don't know what this is. It's really dark. I've never seen this before." We kinda go to this mindset that, you know, maybe I'm dehydrated. Maybe I drank too much tea the day before. All those questions, right?
    And I just kinda let it go. And it really didn't happen... It, it took a few days for my urine to get back to normal. I was drinking a lotta water, staying hydrated, it never really happened again. And when I told the resident that, he was like, "Okay. We're gonna test you for something that's called PNH." He’s like, "It's a longer name." He's like, "I'm not gonna try to butcher it, but it's PNH." He's like, "Google it." And I was like (laughs), "Okay." And he's like, "We're gonna test you for it."
    And so I Googled it, which probably is not the best idea, in hindsight. You know, I was reading the symptoms, and I was like, this, you know, the abdominal pain, the dark urine, the, you know, fatigue, the weakness, it was all just kinda adding up in my head. And I was like, "This sounds about right." And, it was actually a Friday afternoon, a late Friday afternoon that someone from my healthcare team came to me and said, you know, at that point they hadn't received the PNH result. And they were like, you know, "You're doing better," you know, "we might send you home." "You would just be waiting here to get results. And we don't think that's productive for you."
    And so they were actually getting the paperwork signed to send me, to discharge me home. Until I said, "I wanna make sure..." and I looked at the, you know, the person from my healthcare team, and I was like, "You know, I'm okay to go home, 'cause that's where I would rather be (laughs), to be honest. But I wanna make sure that we're not doing this prematurely. I feel like there is something that we need to figure out first." And they were totally understandable, and then 15 minutes later, the result for PNH came back and it was positive.
Leigh Clark:    And then, what did the healthcare team talk to you about what your treatment options were going to be? And did you experience any challenges getting access to treatments?
Shane Pope:    So, it was about an hour after the result came back that a hematologist came to see me in hospital. And I was in my bed with my partner next to me, emotional, of course, because I didn't know what this meant for me. She was a very lovely woman, who's still my hematologist to this day. And I'm so grateful for her. She broke it down for me in very simple language, that, "Hey, Shane, this is what's happening. This is what we've found, or this is what we're seeing. And this is what this means," right?
    And she was so comforting in that moment and made me feel like, "Okay, everything is going to be okay." And it was when she said, you know, "The prognosis is good, the outlook is good, there's treatment for this, we're gonna get you on treatment. We're gonna figure out how to get you on treatment," 'cause where I live in Canada, very, very, very few cases of PNH, very rare. And this is still something that when it pops up for doctors, they are, "What do we do with this?" Right? Because it is so rare. And I actually had another doctor come see me who said that, you know, he's like, "You're one in a million." He was like, "This is something we learn about in school, but in practice, we rarely ever see."
    And I just, you know, I was like, very scared, very worried. I was comforted by the idea or the thought that there's treatment for this. But it was the road there, the journey there. How are we gonna get there? How am I going to afford it? What does it cost? You know, am I going to have to make these changes in my life so I can afford and get treatment, right? And all these what-ifs, and unknowns, and uncertainties. But, you know, they were reassuring that we would get treatment.
    And so, so the journey started, to get treatment, to get me started on treatment. It took about three, almost four months post-diagnosis to get confirmation that I had funding for treatment. We started a week later after my diagnosis with the paperwork, with looking at different insurances, getting connected with patient support programs to see if they can help. We were on the ball, and for some reason we had such a difficult time securing funding for treatment. This treatment, I'm on Ultomiris... Originally, we were trying for Soliris. And this treatment is almost 700, over $700,000.
    I can't afford that, right? And we were looking at different avenues to get funding, and it was, consistent declines. No. No. No. And it was like passing the buck to see who else would, wanna cover it. And we were unsuccessful with Soliris. We tried Ultomiris. And in that process, too, I was really advocating for myself. And, you know, I was doing a lot of research on my own. I wasn't only putting this in the hands of my healthcare team, 'cause I'm part of my healthcare team, right? And I get to make decisions, and I have a voice, and I have a say.
    And I was doing my research and, you know, even during the process of trying to find funding for Soliris, I was like, "Well, there's this other drug that's approved in Canada, Ultomiris. Why are we not trying to get funding for that?" Right? And it wasn't until we were unsuccessful with Soliris that they came back and said, "Oh yeah, Shane, you know, we are gonna try Ultomiris." And so it was a lot of self-advocacy. My healthcare team is amazing. It was more so, external folks that were on my case that were helping, that it was just taking a little longer, the process. And, and I under- I understand that there's process to everything (laughs), and, and that's important. But also recognizing that my life is hanging on a thread here, that it's in the hands of other people. And that was out of my control, and that felt very unsettling for me.
Leigh Clark:    And how, how have you been doing on treatment?
Shane Pope:    So, treatment is going great. I started treatment, back in September. And I've had three treatments so far. So, I get them every eight weeks. And they're going incredible. I had my third one, my third treatment about two weeks ago, and I had no side effects at all, which was a huge change compared to the first time I got it (laughs),but I've been feeling really well. Treatment's going really well. I was getting blood work, I was going for my follow-up appointments at the hematology clinic.
    You know, I was doing everything that I could do, to ensure that I was getting the care that I need. I was staying on top of things, doing my own research. And I think because of that, in part, I'm doing well, and I'm doing better. Actually, as of recently, my blood work has improved so much that my hematologist has, instead of meeting monthly, our appointments are now every two months. 
Leigh Clark:    Awesome.
Shane Pope:    ... instead of weekly blood work, I get monthly blood work. And I actually went for a follow-up CT scan not too long ago, just to kinda see how things were going. And I actually got news last week that my blood clots have dissolved.
Leigh Clark:    That's awesome.
Shane Pope:    Yeah.
Leigh Clark:    Wonderful, wonderful news.
Shane Pope:    Yeah.
Leigh Clark:    So, throughout this process, what have you learned about yourself? And what do you want other PNH patients to know?
Shane Pope:    Yeah, I've learned that I am capable. I learned that, you know, I am my own advocate. I'm the, I'm my best advocate. It takes time, it, you know, you're having to insert yourself in conversations. It takes energy, energy that, as a PNH patient, especially prior to starting treatment, I just did not have a- as much of, and I was having to put my physical and mental health on the line here to have a voice. And it was tough, but it was so worth it. And I'm so glad that I was able to advocate for myself, because I don't think I would be where I am today, a few months later.
    Treatment is doing its job. It's working. But to heal, and to recover, and to bounce back from such a, an experience, it takes a lot more than just treatment, right? And it takes, instilling this confidence in yourself to know your body, right, and when your body is telling you you're not okay. And listening to your body when you're not okay. And keep pushing forward, keep moving forward, so you can get the answers you need. because it's not always about people's intentions, but it's really a lot about people's impact, right? When you're feeling like you're not heard, that impact is great, right?
    And I want other PNH patients to know that it's okay to speak up, it's okay to ask questions, to get clarification. It's okay to want care, and to want the best care for yourself, and that life goes on, right? It looks different, right? It is different. But it's different for all the good reasons. For all the best reasons. I'm know myself a lot more now, and I'm a lot more confident in how I move forward in conversations with my healthcare team. And, you know, I feel like I've built that respect, and I only feel like this is just the start of something really great. And I just want people to know that your life can improve and will improve. You need to be in the driver's seat.
Leigh Clark:    Thank you so much, Shane, for sharing your story with everyone. And it will have great impact, to encourage and inspire other PNH patients. So, thank you so much of sharing, of yourself. And if you'd like more information about PNH, and the treatments, and how to find a second opinion, for yourself, which I'm sure Shane will say saved his life (laughs), 
Shane Pope:    Yeah. Yeah.
Leigh Clark:    ... you can find that information on our website, which is aamds.org. Thank you so much, Shane. We really appreciate it.
Shane Pope:    Yeah, thank you for having me, Leigh.