Let's talk about evidence

"We don't seek the painful experiences that hue our identity, but we seek our identity in the wake of painful experiences."

I don't know where this quote came from, only that I find so much truth in it. If whoever wrote it happens to read this, raise your hand so I can learn more from you.

Cystic Fibrosis sucks so bad. You can't usually see it, yet it forces you to act - restraining your kid for necessary blood work to make sure the medication we're taking to save his lungs isn't killing his liver. Childhood playtime after school is stolen from us as we sit for hours doing breathing treatments and airway clearance instead of going to the park without an agenda. A hug allows me to literally feel a mucus plug trapped deep in his airways, something I can loosen with a few aggressive beats on his chest and I do it without warning as he would deny my offer to help because of the pain it causes him while simultaneously offering some relief. And I sit on an airplane on my way to my second conference in two weeks, having received great news in the 4 days I was home, and yet I cry because I know that the relief I feel is temporary. Hopefully optimistic but not naive. Despite our best efforts this disease will progress. I can sit by the pool at my favorite hotel in Palo Alto so incredibly grateful for these people I've met because of this disease, and cry as I walk back to my room moments later cursing the world and wishing it all away in a trade for normalcy. I don't know normalcy though, I suppose. This is our normal. It's breathtaking to see the world in this way, a gift not many people get. And I love it and hate it equal parts.

Drew's lung function tanked this summer. We saw his doctors many times, and I used my personal network to crowd source ideas for what might be wrong. Early on, there were suggestions by my peers that a fungal infection might be the culprit. With no symptoms except for a rapidly declining FEV1, we spent countless hours troubleshooting - trying everything in our "evidence-based" bag of tricks. There's not significant evidence to suggest that fungus causes lung function decline in people with CF. Or I guess I should say that there's not peer-reviewed published scientific evidence. The "evidence" that I have, the experiences of the people living with cystic fibrosis who have lived through our same uncertain hell, is often discounted. And I get it, really. I am pro evidence-based medicine. I just can't help but think about the value of the individual human experience we are missing.

Despite there being no physical symptoms of exacerbation - data that I have based on our prior experiences, things that are in fact documented in his EHR - we opted for treating this like an exacerbation because we didn't know what else to do. The "we" I speak of is us and his care team. The first step was an oral antibiotic. Drew has never responded to oral antibiotics. I was skeptical of this plan but ultimately on board. After 10 days, there was actually increased decline. He has found benefit from steroids in the past due to the wonky nature of his airways, but I can identify with accuracy the situations that will be improved with steroids and this wasn't one of them. We tried anyway. The downside was outrageous mood swings. While medically induced, I still expect certain behaviors from my kids, and its absolutely heartbreaking to watch your 6yo, who feels like he's going to jump out of his skin and not understand why, sob in his bedroom for hours because, medication or not, hitting other people is not ok. The steroid may have slowed the decline, but his lung function continued to drop.

When we have to make the hard decisions about whether or not to try IV antibiotics, more frustration and disappointment came out of fear, from all of us, that yet another treatment, another procedure would render the same results and we would be left with even fewer options. It's so hard to balance these decisions. His health is a priority, but so is his life, and living means so much more than being alive. Can I take away the joy of the pool or the promised vacation with the hope that this could put us back on track? How could I convince him that this is the right thing to do when all of the preventative medicine he takes, the stuff we tell him we must do for hours every day to keep him healthy has left him so very sick. What the fuck kind of sick logic does this disease demand?

We opt for a combination of IV medications that we hope would target the bacteria that we suspect might be causing the problem, weighing dosing schedules against our ability to administer them from home because there's no way we are admitting him to the hospital when he looks and feels fine. We trade sleep for this decision and our whole world pays for it. Work commitments change and home routines suffer as patience wears thin. Tired and terrified while pretending to be neither is a bad combination. And yet we continue, a constant barrage of choices in our faces, very serious ones with real consequences.

They told us when he had his PICC placed that we might want to have a conversation with our team about a port given the increasing difficulty with placement of a central line due to the fact that he's had 9 in his 6 years of life and scar tissue is getting in the way. With some ativan to take the edge off, we both cried as they stuck him, then stuck him again. His fear has scars showing the distance he's come. I have fear too, but all of my scars are on the inside. I'm a little tougher to break, and each time we do this feels a little more routine, a little more scary.

The IV meds didn't help either. With our lung function creeping closer and closer to 70%, we re-group for another team meeting. I want to know all of the options. We decide on a bronchoscopy, something that will allow them to get a reasonable sample of what might be in his lungs and enable us to target our therapy. Why didn't we start here?  Everything comes with its own risks, and the care team was hopeful that we could address the problem more easily. I trust them and agreed with their plan. I also asked about the risks of treating with an anti-fungal medication. After all, we just followed the script for oral and IV antibiotics and also a steroid, each with its own risks, especially given that they seemed to be the wrong choice as they showed no clinical benefit and the risks of antibiotic resistance in this population is enormous. There is not evidence to support a fungal infection causing a decline in lung function, or "published, peer-reviewed, scientific evidence" anyway. There is a surplus of anecdotal experiential evidence.

With a family vacation just a day away, we go to the hospital for this outpatient procedure. I do my best distraction song and dance while they give him sleepy air. My husband and I don't even go to these together anymore because we've been through them so many times, and life and work don't pause for this disease. I anxiously wait to be called back to see the doctor in what I can only describe to be the purgatory that is the same day surgery waiting room. Her words have so much power to change my life and I'm terrified. But all looks good! His lungs look as good on the inside as they sounds on the outside and we are left wondering. We have to wait 5 long days to see what grows on the culture, to see what explanation we might get from another piece of data. I've convinced that I lose about a day of my life in this unavoidable worry that comes from this unavoidable waiting.

By now, his doctor has given me a prescription for the anti-fungal medication I've been asking for. I promised not to use it until after the bronch, We agree that we have nothing to lose by starting it while we go on our vacation and wait for our results. When the results do come back and show that he has a fungal infection, we exchange emails with his care team about their lack of optimism that the anti-fungal will have an impact given the type of fungus he has, a more common household name I'm told, but without other options it seems to be the last resort and we go forward.

After 4 weeks on the treatment, we wake up early, get our treatments done before the sun comes up, and head into the office for an assessment. I have a preference for the 7:30am  Monday morning appointment as I believe it lowers the cross-infection risk, something that no evidence exists for. It gives me some resolve to have this time slot and I'm grateful to the team that recognizes that and works to accommodate me. He's up to 84! It seems to be working! He's re-gained more 10pts of that lost lung functions and we are elated! We decide to stay the course and after another 4 weeks he is up to 91. I express my joy to his care team who is equally delighted, admitting their early skepticism about this being the cause of the problem and acknowledging the treatment as the reason for our success. I certainly didn't set out to prove anyone wrong, only to make him well, but it's nice to hear that they are learning along with me.

This is a happy story, but it's not the end of the story. We will have treatment decisions to make again next month - stay the course? Make a change? What changes will happen that will be out of our control? Will the next culture show a bug that's completely resistant? Is there anything I can do to prevent that? To protect him?

The past two courses of IV antibiotics that Drew has had have not been needed. We treated a bad case of acid reflux and a fungal infection with hard core antibiotics. We followed the guidelines and used decision trees and made the decisions that we felt we had to make, and we were wrong. Drew has been on inhaled antibiotics - 2 different medications alternated in 15 day cycles, inhaled 3x a day, everyday for the past 3 years. In those 3 years he has not had a bacterial infection in his lungs, an "exacerbation". In 3 years he has not gotten sick. He's been on IV and oral antibiotics that he hasn't needed, and been sedated countless times. He has been admitted for central line infections for central lines he hasn't needed, all because we followed the rules. I don't think we made a mistake, we did what we knew how to do. But now that we know better we need to do better. I wholeheartedly believe there has to be a better way. We might talk about the risks of exploring medications or procedures that lack "evidence", but I also want to talk about the risks of evidence based medications and treatments that we use when we don't need them. How might we get the right thing to the right person at the right time, every time? How might we improve the mental health of our patients and caregivers who are rightfully distraught over the lack of answers to what should be straightforward questions? Why is there still so much uncertainty in medicine? What responsibility should we put on people to advocate for themselves, tracking their own outcomes and then sharing them with the rest of the healthcare system to enable personalization of treatments, and then machine learning to aggregate all of these N of 1's, improving population health through the spread of personalized solutions.

Some new opportunities have recently come my way to improve things within the healthcare system. My goals remain the same - right person, right solution, right time, every time. I think success is more than just improved outcomes just as living is more than just being alive. Success for me is influence. When more people believe that this is possible, when they challenge the status quo and try out of the box solutions, that feels like success. I know I'm making a difference. We are farther in this culture change than we were 5 years ago when I realized my purpose in all of this. As I fly to another healthcare conference, my second in two weeks - a conference where patients and caregivers are being introduced to the world as a symbol of action and influence, a conference where our participation is being fully financially supported - I know that my role is to honestly and vulnerably share these stories and ideas for change. I've figured out how to fold the worst narrative of my life into triumph, and that for me is how I measure success.

Patients Included. But are we respected?

This post originally appeared on my blog in October of 2013.

"Any man or institution that tries to rob me of my dignity will lose because I will not part with it at any price or under any pressure." - Nelson Mandela, Long Walk to Freedom

A few weeks ago, I was asked to be a part of a Cystic Fibrosis work group at a hospital on the east coast under a grant from the Patient Centered Outcomes Research institute, or PCORI. A colleague had recommended me for the position, knowing my passion for collaborative efforts in improving outcomes and the value of the patient voice in research.  Below is an excerpt from the email that I received:

PCORI was founded under the ACA to fund comparative effectiveness research that improves the information available to patients in a meaningful way. Specifically, PCORI has two goals: further engage patients in research design, and reduce the gap between research results and patient knowledge. The CDRN opportunity grants institutions money to develop a large multi-million patient data warehouse for researchers (from around the country) to conduct research - both prospectively and retrospectively. PCORI will fund seven of these networks around the country to form one national network. If awarded the grant, our team would like to offer you a role as a Cystic Fibrosis Community Workgroup member.

My interest peaked, I wrote back and a day later had an interview and was submitting my biosketch and a letter of support. I made them aware of the other work I'm currently involved with to ensure there would be no conflict of interest, and expressed the need to weigh the time commitment and opportunity for compensation with these other projects. Then, as if to define how they value my knowledge, experience, and expertise, I received this reply:

While this particular position will likely be unpaid, this is an incredible opportunity to directly influence the research efforts around CF and will provide you with access to cutting edge researchers in our city.

Is my time and participation not valuable enough to compensate?

Later that week, I packed my bags and headed to MedicineX in Stanford, California, a conference that positions itself as a catalyst for new ideas about the future of medicine and emerging technologies. At this conference, there were patients and clinicians, caregivers and technologists, entrepreneurs and researchers and others from the industry. It’s difficult to explain the experience of being at a conference so full of energy, excitement and hope for better care of rare diseases and chronic conditions, better interactions with the healthcare system, and how the only way to improve is collaboration and teamwork, valuing and trusting one another, in every single possible way.

During the first day of this conference, PCORI -- the organization offering the grant mentioned above -- was presenting a panel on the importance of involving patients in research. I wondered about the offer I had received: Was it the institution that could not offer to compensate me for my time? Or PCORI that isn't accommodating patients who participate in the research by appropriately and fairly compensating them? Or maybe just not all patients? Some view the amount that I work to improve health and care in Cystic Fibrosis as remarkable. I view it as necessary. If I had endless capacity, I would do it all. But remember, I have a seriously and chronically ill child that I am caring for, in addition to 3 others, and a husband, and a couple of fish. Unlike many researchers, I don’t have an institution or grants paying me a full time salary to participate in such research.

My reaction: Don't take advantage of the knowledge and experience that I have that you want and need to create a system that works for me. Appreciate me, and embrace me just as you would any other member of your team, and just watch how far we can go together.

A huge barrier to true patient-centered care is the failure to see patients as equals to the other contributors in this system. Let's not forget than in medicine, the patient is the only one who is expert in her experience. Should the hospital that offered me the position in their workgroup receive the grant from PCORI at the end of the year, I will participate and my participation will not be in vain. I said yes, regardless of compensation status, because I know and appreciate the value of this project. I have a unique perspective to bring that I believe will add value to this research and benefit others in the community. I said yes, because so many other people who have expertise and passion and value to add don't have the time or the voice or the energy to do it. I believe that our story, in the end, will be one of success. But to quote an incredible patient and advocate that I had the great pleasure to meet in person this weekend, "Not every medicine story can have a happy ending. It's not the ending that makes it a success."

How much farther and faster could this patient-centered research go if it weren't so hard to be a part of it?