Why EGPA should be included in more ANCA vasculitis clinical trials
Too often, the door slams shut on EGPA patients before we even knock
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Every time a new ANCA vasculitis clinical trial is announced, I do the same little dance. I read the headline, feel a flicker of hope, and then scroll down to the eligibility criteria looking for one acronym: EGPA, which stands for “eosinophilic granulomatosis with polyangiitis.” Most of the time, it isn’t there.
If you don’t live in the EGPA world, as I do, here’s the quick version: ANCA vasculitis comes in three forms: granulomatosis with polyangiitis (GPA), microscopic polyangiitis (MPA), and EGPA. These conditions are like three siblings of the same family. But when clinical trials come around, only two of them get invited to the table and the third gets left standing in the hallway. GPA and MPA are almost always in. EGPA is almost always out.
I’ve mostly made my peace with the reason why: We’re the smallest of the three. We’re harder to recruit, harder to standardize, and — let’s be honest — probably more expensive to include. I understand the math. A trial has a budget and a timeline, and adding a smaller, messier group can blow up both. And trials aren’t cheap. I’m grateful for those investments. Nobody’s being cruel. It’s a spreadsheet decision.
But I’ll tell you the truth, because that’s the name of this column: It still feels bad.
We have plenty to offer researchers
It feels bad to be an asterisk. To read “ANCA-associated vasculitis” in a study title and realize they didn’t actually mean all of us. It feels bad to watch good science — science about the exact problems I live with — get built in a room those of us with EGPA are not allowed into.
And here’s the part that really gets under my skin: The two areas where clinical trials matter most to me are relapses and steroid sparing. Getting off prednisone, which quietly takes a toll on your bones, your eyes, your moods, and your face, is important to all of us. Those are the studies I most yearn for. And those are exactly the type of studies EGPA tends to be left out of.
It’s also ironic, because relapse is practically our signature. We come back, over and over. One large study recently found that nearly 40% of people with EGPA relapsed within a single year, and our type is defined in part by frequent and sometimes life-threatening flares, and a stubborn dependence on steroids. Our relapses come in two flavors: the vasculitis flares everyone studies, and the asthma and sinus flares that quietly keep so many of us tethered to prednisone.
If you wanted a group that could teach you something about relapse, you’d want us at the table. If you wanted to know whether a drug could hold someone steady off prednisone, you’d want the people who flare the easiest. Leaving us out of relapse and steroid-sparing research isn’t just a loss for EGPA. It’s a missed opportunity for the entire field. It’s a loss in terms of understanding the nasty little guest that now permeates our lives.
Now let me say another true thing, because gratitude and frustration can live in the same body — I’d know.
There are trials that do focus on EGPA, and I don’t take a single one of them for granted. GSK (formerly GlaxoSmithKline) and AstraZeneca have put real money (hundreds of millions of dollars) and real attention into our type — GSK developed Nucala (mepolizumab), and AstraZeneca developed Fasenra (benralizumab), both of which were studied specifically in EGPA. That matters more than I can say.
When you’ve spent years being a footnote, having companies decide that your disease is worth studying feels like being seen. What a gift to those of us with EGPA. Thank you. I mean that. Those investments have changed lives, mine included.
So this isn’t a complaint about being forgotten. It’s a question about being chosen — or not.
What I keep coming back to is this: We don’t always need our own clinical trial. Sometimes we just need to be part of the sample, such as a few EGPA participants folded into a broader ANCA vasculitis study; a relapse endpoint that doesn’t quietly screen us out; or a steroid-sparing question that includes the people most likely to answer it.
I come from the world of healthcare business, and I know the reality of budgets. That said, here’s what gets lost in the budget conversation: Behind every exclusion criterion is a person reading it — someone like me, hoping that maybe this will be the study for us, that this will be the door that finally opens, only to find it closed before we even knock.
We are part of this family. We relapse the most. We have the most to teach about the questions everyone says they care about. All I’m asking is for us to be let into the room.
Note: ANCA Vasculitis News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those of ANCA Vasculitis News or its parent company, Bionews, and are intended to spark discussion about issues pertaining to ANCA vasculitis.
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