Taylor Coffman and Len Woodward are two public aHUS patient advocates who write about aHUS and the patient experience of living with it. Like many they have corresponded and collaborated and exchanged views on social media while oceans apart.
Recently during Taylor’s visit to the UK with her daughter July and husband Zach and his band The Dustbowl Revival ( on a UK tour) , they met in person for the first time. After catch up the two talked about aHUS and these blogs are about what was said.
Len: Taylor, I first became aware of you when I read an article that Zach wrote for a magazine about your encounter with aHUS. Soon after we were asked by Professor Fadi Fakhouri to provide a patient testimony of pregnancy associated with aHUS for a national USA conference. .We thought of you, asked you and you agreed and we left you with organisers to make a video.
Taylor: Thanks for asking me. I hope my story helped inform doctors about aHUS and give them real context to put to the clinical experience. Pregnancy aHUS can be tricky to diagnose.
Len : Yes it did. Then when Global Action were leading a USA patient partnership to have a Patient Listening session about aHUS with the US Food and Drugs Administration you joined in and we met many times by zoom despite the large time zone differences.
Taylor: That listening session was a great introduction to aHUS advocates. Always grateful to be involved to help advocate for patients.
Len: As we have this great opportunity to chat face to face I wanted to talk with you about what are the key aHUS issues of our time.
Taylor: Thanks Len! You have done so much for aHUS patients. A lot of what I know, I’ve learned from your articles.
Len: Thank you I guess we all learn from each other. Having recently listed them for an aHUS patient meeting , my key issue of our time would be
- The change of the name for aHUS
- How to achieve a rapid diagnosis of 7 days or less for all patients
- What is the optimal length of complement inhibitor treatment
What would yours be?
Taylor: For me:
- Awareness is crucial. When a disease has a treatment, awareness saves lives. It’s that simple. A new name is vital to this because any efforts we make now could be stifled by a new name.
- A rapid diagnosis through awareness or supporting scientific research to develop a diagnostic test.
- More treatment options – infusions should not be the end of the road. Infusions are challenging- from approvals, to a whole host of supplies, to needing a nurse or center to administer. I want treatment options the patient can self-administer for a better quality of life.
- Access to medication – because while there’s a treatment so many folks find barriers to actually getting the medicine even with a prescription.
Len: OK we are not far apart but lets start with Awareness and bring in name change with it as it is a hot topic at the moment. What do mean by it and what do you think needs to be done about it.
Taylor : For me, a name change could be a good thing IF (and that’s a big IF) the process is well-handled. A better name could help lead to a swifter diagnosis. But it’s not an easy process and needs great project management to assure smooth awareness to all systems and insurers.
Right now, we have vegetable soup. Good awareness efforts around a disease require funding and hard work – and it’s tough to put efforts into that when a name could change. As an advocate, I really want the entire community to get creative and get organized.
I wrote about this previously and in STAT but the National Kidney Foundation had a working group that essentially suggested a very sprawling fluid acronym system. You put their chart in an article.
I would be Pregnancy-Induced Complement-Mediated No Known Gene – Thrombotic Microangiopathy.
Kinda rough. Doesn’t exactly roll off the tongue.
I’m not sure their methodology took the practical life of a patient into account. That’s why our voices matter.
I also worry about doubling down on thrombotic microangiopathy or TMA (aka tiny blood clots) , possibly problematic and confusing. Lots of diseases feature TMA. Pregnancy induced-TMA could potentially complicate high-stakes situations like mine when there are multiple kinds of pregnancy TMAs already including HELLP Syndrome, thrombotic thrombocytopenic purpura (TTP), pregnancy-induced hypertension (PIH), and preeclampsia (PE).
To be fair, the disease is ultra-complex and rarely are two patients exactly the same in how the disease manifests. So it really does require some careful thought and creativity.
I hope people give some thought to the idea that this disease’s name could change because it will impact us all. And the Alliance’s project gives us a chance to use our voice in the process.
Len Yes awareness is a multi faceted issue. There would be no rare disease organization which does not have raising awareness of its disease as its major purpose. As you say awareness impacts on diagnosis which we will come to that . I think that name change will also have an impact on prognosis and treatment will be more tailored, which we will also come to also. But aHUS and other conditions are potentially facing an unprecedented overhaul of nomenclature which will affect everything to do with having and living with the disease. Too big a change for it to happen in one go. And a transitional period could last a long time.
The early 21st century has been about raising awareness of “aHUS” which changed in meaning as the century progressed but between now and the beginning of the mid 21st century awareness will be about both the old and new names which will be a major challenge. You gave a very good synopsis of the current name change debate. You make the point that this is going to need exceptional good project management to bring it about and there will be risks that will have to be mitigated.
It is right as you point out that for the first time patients are being given the chance to speak about it all. And what they say needs to be carefully considered. Patients need a “line in the sand” moment like clinicians did three years ago with their name change report.
Before I ask you what name you would propose to replace aHUS . Mine would be cTMAHUS during the transition, can you say what you can foresee as the main risks that patients are going to face in the next five to ten years as implementation begins? Between now and when they will be sitting down with their doctors and their doctors say “you used to have aHUS but now you have “X” and the implication is “Y””.
Taylor: We will need robust and clear ways to communicate the change – funding would help.
And if you ask me about a new name idea, I actually think we need to let go of TMA. It’s a symptom yes, but 100,000 people could have TMA, and only 2000 have aHUS truly.
Maybe it’s simpler. I’m just throwing out ideas but something like Complement Dysregulation Vasculopathy (aHUS). Because it’s the only disease in my research that is triggered by the complement system regulators AND causes vasculopathy, which just means disease of the blood vessels. The combo of the too differentiate it from other similar diseases.
I’d keep aHUS in the second part of the name because I think it will help in the transition. Maybe we can say it in a way that’s easy. C-Dys or C-DysV for short! Might be memorable for an awareness campaign- like C-Dys or SEE DIS new name for aHUS!. Sorry for the puns. I’m just trying to think out of the box. A doctor should fact check me – but I think we should stretch creativity a bit more- throw out ideas .
Len: That is certainly a creative effort and emphasises complement as the reason. The name vasculopathy seems just the sort of term a rare disease should have. And we would join another range of diseases with vasculopathy at its core too. I suppose as well it can have a name that is different to the official name as aHUS has always been and it did not matter it was not official.
You make a very good point about the relative incidence (per annum) and prevalence ( living at any time) of aHUS among TMAs . If for aHUS it is globally 4000 and 30,000 respectively then TMAs would be 200,000 and 1,500,000. At those sorts of levels for TMA and the fact it is a medical emergency, is not TMA something that doctors could be aware of like heart attacks or strokes.
“Think kidney- Think TMA- Think aHUS” is a sequence to get to it at the end . The name cTMA will not be associated entirely with kidneys, in some it primarily affects another organ not the kidneys. That is why doctors came up with cTMA. Given that HUS was actually plural the name and HUS is the official name , cTMAHUS is consistent with both the doctors suggestion and official dome. Advocacy could even emphasise the shared A in red font.
Although aHUS, even if it is inconsistent with upper case and lower case use in disease name, has a bigger claim to the A then the two word term “thrombotic microangiopathy”.
You see communication and adequate funding as the key risks of this name change project. It could be said that as far as patients are concerned communication has not been sufficient already. Despite the efforts of patient advocates to make good the communication deficit. And funding, who is going to provide the funds? Pharmaceutical Companies?
In the aHUS Awareness Day campaign this year some awareness of name change has been achieved and the capturing of views of patients/relatives who made them will provide insights of what this means to them. Because that will be where the impact falls.
We can leave awareness and name change there, and move on to your next key issue of a rapid diagnosis through awareness or supporting scientific research to develop a diagnostic test.
The conversation about diagnosis will be in the next blog article. Look out for it
