Sunday, September 25, 2016

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 Or I guess I should say that there's not peer-reviewed published scientific evidence.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.



Thursday, September 8, 2016

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?

Saturday, August 6, 2016

Research is Hope

“Hope” is the thing with feathers - 
That perches in the soul - 
And sings the tune without the words - 
And never stops - at all"

I am vulnerable. I have a child who is sick, like really sick.  I go to the doctor with the desperate hope that someone - anyone - will be able to help me to keep him well, to save him. And in that vulnerability, I am asked to do things I often don't fully understand. 

I had to give my baby to strangers when he was two days old, signing papers to waive liability if something happened to him when he was under the care of someone I had never even met.  This is the story of most chronic illness caregivers.  As time goes on, our comfort level grows, relationships build with our doctors and care teams.  But with chronic illness, or CF, anyway, in our case, there is always something new, always something scary, always something we don't understand. Then researchers come in - knocking on the door after a long 3 hr appointment, or while we are in the surgery holding area waiting to go back and wondering how the results of whatever we're having done will forever change our reality, yet holding it together for our kid who is equally scared - trying to explain what the researcher whose interested in a sample of our tissue or some of our thoughts (neither of which we're giving with a clear mind) is trying to do, what they're hoping to learn. But in that instant it sounds promising so we go with it, signing their paper since the risks seem low, collecting our gift card for participation, or just their thanks, in many cases, and the promise of learning or better connection with our care team, whatever it is that their trying to do. Hope is really what their offering us, and we sign up. 

Sometimes there's further interaction, but many times not. Then months later, long after we've signed their papers, something hits us and we remember that time we signed up for that thing, and a bit of us wonders what ever came of it. And, if we search just a little bit, we find out that the study didn't recruit, or that it didn't meet the appropriate end points, or that the institution supporting it didn't see the value in it and it's done. Those researchers move on to their next theory to test and our contribution has been tossed aside. Or maybe it went on and that researcher learned many great things that they wrote about and got credit for and published their findings in some fancy journal that we don't have access to because, after all, we're just the patient. Forget about what the patient thought, what they learned, that brief glimmer of hope. 

It feels abusive, really, to approach the sick and offer them hope - to take something from them, physical or otherwise, something that's so sacred - and then to just end it because it didn't meet your goals. When do my goals matter?

Why are we allowed to just throw that all away? Why is that ok? Shouldn't there be, if nothing else, a moral or ethical obligation to say, "I'm sorry, I tried."  Shouldn't we be giving patients the opportunity to take what's been learned from the work they contributed to - the work that couldn't have been done without them - and start from a new baseline of learning? Why aren't we sharing what we are learning in research, successful or failed, in a way that allows folks to build upon it? This is among the greatest failures of modern medicine. 

I want to ask researchers what they're actually trying to do. If the answer isn't that you are trying to, wholeheartedly, help patients, then please stop wasting our time. 

A research project I had been a part of recently shut down and I'm so...I guess sad is the right word. I wasn't a participant, but rather a member of the study team so I can't help but feel a bit of responsibility. My frustrations are for those families who joined this project, sharing with us their precious time, the intimate details of their health and lives. People who were given hope only to be shown another dead end, and for no good reason. My participation and contributions felt uncomfortable at times. It was frustrating to have my ideas and suggestions for a more patient-centric approach limited by both institutional research regulations, and the discomfort or fear or I'm not exactly sure what of trying to do something different than it had ever been done previously. I should have pushed harder. 

Helping patients means doing things differently than they're being done right now.  I do believe researchers are  trying to learn, to help us. But to be honest, it's moving at a devastatingly slow pace and it's too often letting us down. 

Give me back what you've taken from me and encourage me to share it, to build upon it, to figure out what else we can learn together. My participation in research and the view I have of the participation of my peers has left me feeling exposed and uncomfortable, not wanting to say yes to future engagement. This is a problem that we need to fix! This is how we will really start to learn and grow and change and improve. Stop picking on the weaker man. 

Research is Hope

“Hope” is the thing with feathers - 
That perches in the soul - 
And sings the tune without the words - 

And never stops - at all"

I am vulnerable. I have a child who is sick, like really sick.  I go to the doctor with the desperate hope that someone - anyone - will be able to help me to keep him well, to save him. And in that vulnerability, I am asked to do things I often don't fully understand. 

I had to give my baby to strangers when he was two days old, signing papers to waive liability if something happened to him when he was under the care of someone I had never even met.  This is the story of most chronic illness caregivers.  As time goes on, our comfort level grows, relationships build with our doctors and care teams.  But with chronic illness, or CF, anyway, in our case, there is always something new, always something scary, always something we don't understand. Then researchers come in - knocking on the door after a long 3 hr appointment, or while we are in the surgery holding area waiting to go back and wondering how the results of whatever we're having done will forever change our reality, yet holding it together for our kid who is equally scared - trying to explain what the researcher whose interested in a sample of our tissue or some of our thoughts (neither of which we're giving with a clear mind) is trying to do, what they're hoping to learn. But in that instant it sounds promising so we go with it, signing their paper since the risks seem low, collecting our gift card for participation, or just their thanks, in many cases, and the promise of learning or better connection with our care team, whatever it is that their trying to do. Hope is really what their offering us, and we sign up. 

Sometimes there's further interaction, but many times not. Then months later, long after we've signed their papers, something hits us and we remember that time we signed up for that thing, and a bit of us wonders what ever came of it. And, if we search just a little bit, we find out that the study didn't recruit, or that it didn't meet the appropriate end points, or that the institution supporting it didn't see the value in it and it's done. Those researchers move on to their next theory to test and our contribution has been tossed aside. Or maybe it went on and that researcher learned many great things that they wrote about and got credit for and published their findings in some fancy journal that we don't have access to because, after all, we're just the patient. Forget about what the patient thought, what they learned, that brief glimmer of hope. 

It feels abusive, really, to approach the sick and offer them hope - to take something from them, physical or otherwise, something that's so sacred - and then to just end it because it didn't meet your goals. When do my goals matter?

Why are we allowed to just throw that all away? Why is that ok? Shouldn't there be, if nothing else, a moral or ethical obligation to say, "I'm sorry, I tried."  Shouldn't we be giving patients the opportunity to take what's been learned from the work they contributed to - the work that couldn't have been done without them - and start from a new baseline of learning? Why aren't we sharing what we are learning in research, successful or failed, in a way that allows folks to build upon it? This is among the greatest failures of modern medicine. 

I want to ask researchers what they're actually trying to do. If the answer isn't that you are trying to, wholeheartedly, help patients, then please stop wasting our time. 

A research project I had been a part of recently shut down and I'm so...I guess sad is the right word. I wasn't a participant, but rather a member of the study team so I can't help but feel a bit of responsibility. My frustrations are for those families who joined this project, sharing with us their precious time, the intimate details of their health and lives. People who were given hope only to be shown another dead end, and for no good reason. My participation and contributions felt uncomfortable at times. It was frustrating to have my ideas and suggestions for a more patient-centric approach limited by both institutional research regulations, and the discomfort or fear or I'm not exactly sure what of trying to do something different than it had ever been done previously. I should have pushed harder. 

Helping patients means doing things differently than they're being done right now.  I do believe researchers are  trying to learn, to help us. But to be honest, it's moving at a devastatingly slow pace and it's too often letting us down. 

Give me back what you've taken from me and encourage me to share it, to build upon it, to figure out what else we can learn together. My participation in research and the view I have of the participation of my peers has left me feeling exposed and uncomfortable, not wanting to say yes to future engagement. This is a problem that we need to fix! This is how we will really start to learn and grow and change and improve. Stop picking on the weaker man. 

Friday, July 29, 2016

Vacation FTW!

This vacation.... You guys, this vacation was the most vacation-y vacation I think I've ever been on. Yes, the kids were there, and yes it was hotter than just about anywhere we've ever been, but the "wake up when you want" and the "walk to the beach in 5" and the "getting in the pool a moment after someone's says 'Hey does anyone wanna go in the pool'"....and it being 85ish in there when you jump in. And I haven't even mentioned the shrimp and grits yet, or the oysters and fried chicken. A day at the spa and having someone rub my feet for an hour. RUB MY FEET FOR AN HOUR!! We're doing puzzles and playing euchre, coloring and crafting and moving at a pace that everyone should try. Clear minds here. There's not a schedule. If you wanna go sightseeing you just do it. And if you wanna go out for breakfast you just go. There's no "we need to get home for..." and there's no answering emails or solving problems because there aren't any problems. I'm reading a book instead of a Facebook feed and checking for the best spot to set up on the beach more than I'm checking email. This is freaking vacation, and I needed this from the bottom of those rubbed feet to the tip of my soul. I'm tan! Like not tan mom tan, but I've definitely been whiter, so this counts. This is cornier than I get, but the refreshment that this week away is bringing to our family after a month of problem solving and worrying and making excuses and trying and hoping - the kind of stress that exacerbates back pain and turns a typically happy family into a tired and confused family - the relief of this vacation has alleviated all of that. We didn't just pack up that worry and stick it on our doorstep to wait for our return, we let it go. We got our answers before we left and we decided to forget about life for a while. There's still treatments and medicines down here, but there's comfort in knowing that his lungs are ok. He's doing ok and we're doing ok. Actually we are doing freaking awesome. Charleston for the win!

That dropping FEV1 is just a number - as he proclaimed with frustration over the weeks of medications and appointments and attempts to get that number to stop dropping and start improving prior to our departure for this magical vacation - and  when we got the results from the bronch and found no bacteria - not even the achromobacter that we've been treating for the past few years - we moved to the other side of worry. As predicted, fungus seems to be the culprit, and an oral anti-fungal medication for the next few months will hopefully help to shift that dial back toward a higher number, but he is well. When we return we will talk to our doctors and make a plan and keep on swimming. For now, I'm putting in a vote for more vacation.

Thursday, July 28, 2016

Your vote counts

This post is an ask for your vote for me, as President of the....wait, wrong topic.

I'm darn proud of the work that I do and I'm even prouder to share that I've been nominated for it as a leader in the Health Activist category by Health 2.0 Conference, the leading showcase of cutting edge innovation that's transforming health and healthcare. I'm listed among many of great ePatient leaders that I'm also proud to call my friends and know that whoever is chosen to represent the collective ePatient voice will do us proud. I'd also feel quite proud if I was chosen as that leader. Voting ends this Sunday, so if you have a moment to spare, your vote for me, Erin Moore, under the Patient Activist category, would be most appreciated.

Vote Here!