The Study Drug (or Introducing a "sister" blog -- Eating Gluten)

As I rounded the corner of my house on Friday, the UPS driver hopped out of his truck -- smiling and waving at me.  

(For YEARS, he delivered diapers to our door at a staggering rate.  He pushed them inside the front door for me, and once, when I clearly had my hands more than full with 4-week-old twins and a 2-year-old, he offered to unbox them for me.  We rewarded him with generous gift cards at the time, and while his deliveries have dropped off dramatically over the years, he still pulls up with a smile and a bounce in his step.)

I am sure I looked puzzled, because I was not expecting anything.

Which was silly, actually, for the clinical trial coordinator had sent me an e-mail on Thursday telling me that she would be "overnighting" me a "2-day supply of study meds," which, of course, meant that I should very much have been expecting some sort of delivery at some point in the day on Friday.

No offense to UPS, but IF had been expecting a delivery driver, I would have been looking for a FedEx truck (in my mind, "overnight" and FedEx are nearly interchangeable words...).

But, in all honesty, I had already forgotten about the delivery I should have been expecting on Friday.  To those who don't know me, this might make some question my commitment to the clinical trial, but let me assure anyone who might be concerned that while I am highly ambivalent about this whole journey, I am also absolutely committed.

While a huge part of me is still very much struggling with the fears I have that are associated with participating in something (anything) that is risky (I do, after all, have a husband and three children and a private practice as a social worker...and all sorts of volunteer activities), I still absolutely and completely understand how important it is that subjects in clinical trials remain in the clinical trial for the duration of it whenever possible.  Whatever happens, I'm going to see this through.

After the UPS driver left, I looked at the return address on the package.  Ahhh.  Yes.  Of course.  

Right.

The Study Drug.

I put the package on the marble table in our foyer.  It is the table for incoming and outgoing mail...and anything else that needs to go with us when we leave the house next.  Sometimes it is VERY cluttered, but on Friday, the table was empty.

Except for the package.

I left the package there.  I knew what was in it, and I knew that I did not need it until Sunday.

I had work to do.
I went back to my home office, but the package drew me down the stairs, back to the marble table.

What if there was something else in the package?
What if there was a note asking me to do something?
What if the study drug required special handling?
What if I had questions?

I opened the package and found a note with simple instructions, and a prescription bottle with my name labeled "Montelukast or Placebo (IRB 15-1413) 10 mg" with a big yellow sticker that read "FOR INVESTIGATIONAL USE ONLY."

Curious, I opened the bottle.  
I poured the (two) pills into my hand.

One dose for Sunday, one dose for Monday.
  (Gluten Challenge on Tuesday.)

The pills were sort of silly looking -- big, fat, puffy...RED.
I texted a picture to a friend who responded that they looked like "horse pills."
(She's the kind of person who can say this from experience, which gave me pause.)

This morning, I took that first pill.
But first, I hefted it in my hand.
It felt like a pretty regular pill.
Except that it was big.

And then I shook it.
Something rattled around inside.
Very interesting.

It wasn't until after I swallowed it that I realized I could have opened it.
But, I wouldn't have.
And I won't.

Because...curiosity killed the cat...and I don't want any sort of bad karma, even though I know full well that what is in the pill is said and done, and beyond my control.

Whatever happens, I'm going to see this through.


Since my current adventure has little to do with Eating Peanut, but it turns out lots of readers want to follow along, I started Eating Gluten last night.  If you want to follow along, you can find it at:  Eating Gluten.

My Walk in Susan's Shoes

In the spring of 2011, I was diagnosed with Celiac Disease.  The diagnosis was not obvious, as I did not have any of the more commonly known symptoms -- no gastrointestinal distress, no chronic diarrhea, no bloating, no vomiting.  (I now know these are just a few of the symptoms, and that these symptoms are actually the types of symptoms seen in children, not adults.)

The diagnosis followed a lot of tests, several of which considered things far more serious than Celiac Disease (think cardiac disease, blood borne cancers and more...).  

I was honestly relieved by the diagnosis -- which was suggested by a blood test and confirmed with the "Gold Standard" of diagnostics -- an upper endoscopy to look for inflammation and to take a number of biopsies of my small intestines followed by three months of gluten-avoidance and then a second upper endoscopy to confirm evidence of healing.

I know there are some who will doubt me when I say that I was not even particularly daunted by the diagnosis, but I wasn't.  Having mostly mastered the art of managing Susan's food allergies (and having become accustomed to spending a lot of my time assuring that there was not even the possibility of a trace amount of peanut in anything she might eat), Celiac Disease -- avoiding gluten -- seemed like a relatively manageable thing to me.  And it was a lot better than some of the other things doctors had considered along the way to the diagnosis.

I still remember one friend who reacted with horror -- telling me that I would never be able to avoid gluten on top of being vegetarian (by choice) and keeping a strictly nut-free and very-limited-soy household.  

I remember another friend who mourned the end of my baking (a hobby I loved).  I remember that friend wondering aloud if I could get away with "cheating."  When I gave her a funny look, she explained that she thought I might be able to get away with "cheating" since I had never had any clear symptoms of Celiac Disease.  I remember patiently explaining to her that if living gluten-free would rid me of my debilitating fatigue, the threat of fainting every time I stood up and the frighteningly regular irregular heartbeats I had grown accustomed to, there was NO way I would ever cheat.

And I meant it.
I have never "cheated" on my gluten-free diet.

(That's not to say I haven't been "gluten-ed," for I have.  And that's no fun, for sure.)

But, why would I cheat?

What message would THAT send to Susan, for whom a "cheat" could result in a life-ending anaphylactic reaction.

I never felt that Susan needed me as an example, but once I found myself in a position where I had to scrupulously check and sometimes avoid foods for my own health, there was no way I was going to do anything but exercise extreme caution when it came to food.

Despite my friends' dire predictions, avoiding gluten in our mostly-free-of-prepackaged foods household was not really all that hard.  

Despite my children's fears, I did not make our whole household gluten-free.  I found suitable substitutions for some foods and simply live without others.  (I recognize that I am fortunate to have been diagnosed with Celiac Disease in 2011 instead of 2001 or even 1991...as times have changed, for sure.)  I gave up my baking hobby and stopped regularly baking for others, although I never stopped baking for Susan.  As the years have passed, I have begun baking some, although I try to limit my use of flour, as I have come to understand how it swirls through one's kitchen, microscopic traces of it lingering in the air, hanging on the drawer handles and cabinet pulls, "dirtying" the countertops that look clean.

Despite my friends' certainty that my life was going to change for the worse, I embraced my new gluten-free lifestyle, made healthy choices and moved on.  Feeling good, healthy -- strong -- was all the reward I needed for my efforts.

I can honestly say that within a few months, I got to the point that I really didn't think that much about life with Celiac Disease.  It was just a part of who I was, and how I ate. 

Celiac Disease did not define me.  
Celiac Disease did not limit me.  

I did not feel in some way like I had somehow brought Celiac Disease upon myself.

And despite some people's certainty, I never felt that there was a clear connection between the fact that I had Celiac Disease and Susan's food allergies.

I always carried things I could eat with me, but, again -- that was no different than what I had always done for Susan.  

I did not worry at the diagnosis, wondering why I had Celiac Disease...I simply lived with it.

Life went on.
And really, life was just fine.


So -- when I got an e-mail from the University of Chicago earlier this summer, seeking volunteers for a clinical trial for Celiac Disease -- studying the efficacy of Singular (a drug I have come to know well -- and love -- because of it's role in Susan's OIT) on tolerance of gluten by those with biopsy-diagnosed Celiac Disease, I paused. 

I thought about it.
I thought some more about it.
I started to delete the e-mail.

I am busy.  
My husband is still recovering from his illness earlier this year.
Susan is still undergoing OIT for her peanut allergy.

I told myself I did not need to take anything else on.

And yet, I didn't delete the e-mail, but...I also did not respond immediately.


And then I texted Susan, who was at the skating rink:

Hey -- think I should do a clinical trial for Celiac Disease?  It is a double-blinded study with a 1:3 chance of being asked to eat gluten (bread) while receiving placebo...

Susan:  I think you should do it!

Somehow...I knew you would say that!


And then I texted a friend, who said essentially the same thing.  And then I took a very long walk and thought about it...and when I got back, I sent an e-mail.

Over the next few days, I exchanged several e-mails with the clinical trial coordinator and had a lengthy conversation with her.  We talked about blood draws, upper endoscopies, fecal samples, and gluten challenges.  We talked about Singular and the placebo.  We talked about commitment.

Commitment.  
It is a big word, and an even bigger responsibility.
The clinical trial coordinator was clearly trying to assess my level of commitment.  And I think she liked what she heard when I shared a bit about Susan's clinical trial experience with her.  

I know first-hand what placebo looks like in a clinical trial.

I understand all too well how what a protocol says might not actually be the way things look in the end.

I understand about extra visits and unexpected blood draws.

I understand about the unknown.

The clinical trial coordinator e-mailed me the study protocol.  I reviewed it.  My husband reviewed it.  I signed it, scanned it, and e-mailed it back.  A few days later, I executed a release allowing the doctors at the University of Chicago Medical Center to obtain the medical records pertaining to my original diagnosis.

(For those who are curious, this clinical trial is for research purposes only.  When I get done, I will go back to my gluten-free diet, and hope that what the researchers learn as the result of this clinical trial will move them one step closer to a treatment.)


The wheels were in motion...and I was sort of terrified at the prospect.

While I am fully committed to this clinical trial, if I am honest with myself, I will admit that a BIG part of me -- a reallyreallyreally big part of me was hoping that somehow, for some reason, I would not be a good candidate for the clinical trial.  

Of course, one reason a clinical trial candidate might be disqualified from this clinical trial (most, I assume, actually) is unrelated health issues.
And I certainly did not want an unrelated health issue to come to light during the course of my evaluation.

And, of course, another reason a clinical trial candidate might be disqualified from this clinical trial is uncontrolled Celiac Disease.
And given my commitment to a gluten-free diet, I certainly did not want to learn that my Celiac Disease was not controlled.

And so, as much as I hoped -- really, reallyreally, truly hoped -- I would not be a good candidate for the clinical trial, at the same time, I also hoped -- really, truly hoped -- that nothing would come to light that would disqualify me from the clinical trial.


Days passed.
Weeks passed. 
I thought "phew!" 
I thought "I'm not a good candidate based on my original diagnosis."
I thought "I bet they don't have funding yet (been there, done that)."


And then I got an e-mail.
The medical records pertaining to my original diagnosis looked good. 
They had funding.
They were ready to go.
Could I come in mid-to late August for a blood draw?
Could they tentatively schedule an upper endoscopy for the last week of August?

Ummm.
Sure.

And so I went for that first blood draw.
I was SO nervous -- not about the blood draw, or the protocol review, or the papers I had to sign, or the intake questionnaire.

I was sososo nervous because...I am embarking on something medically unnecessary, with unknown consequences, and an unknown outcome.  

I do not have to take this risk.

It isn't that I am afraid I will receive the placebo -- I have no control over that.  My clinical trial identification number was assigned long ago, and attached to it was the randomization for the clinical trial.  While I do not know whether I will be given Singular or the placebo, that part of it is already done.

I am nervous because I do not have to take this risk.

The wheels have rolled quickly since that first blood draw.

Lab results.
Check.

Fecal sample.
Check.
(And can I just say -- best to get such a thing done while all member's of one's household are sleeping or out?!?)

Upper endoscopy.
Check.
(And if I was nervous about the blood draw, I was positively terrified about the upper endoscopy -- an easy, simple procedure I had already undergone twice.)

When the nurse asked why I was so nervous, I explained:  I am nervous because I do not have to take this risk.

She heard me, and said something about how she was certain I would be fine, because I am not doing this for myself.

Of course, everything was fine that day.
And everything IS fine.
So fine, in fact, that I've been scheduled for my first gluten challenge.

Yep.
Susan grinned when I told her.
I grinned back, but deep down inside I felt (and feel, even as I type this) fear.

I do not have to take this risk.

I am nervous because I do not have to take this risk.

But this is a risk I am going to take -- for Susan, and for everyone in this world who has to think about absolutely everything they put in their mouth.

How to Stay Alive (in Three Seconds)

In May of 2016, the U. S. Food and Drug Administration (FDA) released new guidelines for use in the administration of EpiPens, reducing the hold time after injection from ten seconds to three seconds.  

I am so proud of Susan, who revised her video to reflect the new guidelines.  The revised version of her video is entitled How to Stay Alive (in Three Seconds).  Here is the link:  https://www.youtube.com/watch?v=a7OROfMnJXo 

Please, take the time to watch this video, which runs just over 7 minutes.  As Susan said, some day, it just might save someone's life.

(Why) I Am Not Mad

I am not mad.
I am not angry.
I am not upset...
     (although I know a lot of people who think I should be...)

I do not blame anyone who cared for Carl on Monday and Tuesday for any of what transpired during what we believe was an anaphylactic reaction to a bite or sting.

I am not mad, angry, or upset with any of them because I believe each and every person involved in Carl's care did what they thought they should be doing at the time.

I am not mad, angry, or upset with any of them because I believe each and every person truly cared for Carl as they would have cared for their own child.

I am not mad, angry, or upset with any of them because I remember full well the times when I did not administer epinephrine to Susan and I should have.


I remember the first time.
We were on an airplane.
Susan was wheezing, coughing, gasping for breath.
Clearly -- one major symptom.

I remember the first time.
We were new to anaphylaxis.
I thought it was impossible...
  for Susan to have an anaphylactic reaction without eating something.
There was so much we did not know.

I remember the first time.
A medical doctor -- surely more informed than I
   advised against administering epinephrine, cautioning me on how dangerous it could be.
   (she could not have been more wrong)

I remember the first time.
There was so much we did not know.
We got lucky that day.
Soso lucky.


I remember the second time.
We were in a movie theater.
Susan was wheezing, coughing, gasping for breath.
   I later discovered that she had a dusting of hives on her chest.
Clearly -- one major symptom..and one minor symptom.

I remember the second time.
We were still new to anaphylaxis.
I still thought it was impossible...
  for Susan to have an anaphylactic reaction without eating something.
There was so much we did not know.

I remember the second time.
I was afraid to administer epinephrine.
I was afraid...of the epinephrine.
I was afraid to give it if Susan did not really, truly need it.
   I was afraid...to label what was happening anaphylaxis...
   I was afraid to call it an emergency.
I was afraid...of...so many things.
I wasn't thinking about what should have been my biggest fear that day.

I remember the second time.
There was so much we did not know.
We got lucky that day.
Soso lucky.


With more than ten years of managing Susan's food allergies, and a bit of a crash course in anaphylaxis during the clinical trial, I now believe learning to recognize and manage anaphylaxis is, for most, a skill that develops over time.  It comes with experience, and matures with the understanding that epinephrine works best before someone is in significant distress.  It comes with a matured understanding of the risks of anaphylaxis -- with the understanding that what one can see is only a small part of the picture...one cannot see lungs filling with fluid, a throat that is swelling shut, a slowing heart rate, dropping blood pressure...all of which render the body increasingly less able to do its job.


And I venture to say that some...if not many...if not actually most...parents of children with life-threatening food allergies would admit -- even if only in the deepest recess of their mind, the place they rarely go -- that there once was a time when...(now, on the safety of the other-side-of-the-reaction) -- they themselves should have administered epinephrine, but did not...


I am not mad, angry, or upset with any of the individuals who cared for Carl because once, as hard as it is for me to believe now, there was a time when I might have made a similar series of decisions.  

My original blog post was a call to action, a call for greater awareness -- a call for education on the signs and symptoms of anaphylaxis and a call for more uniform availability of epinephrine in emergencies.  Please, let this be the same.

Anaphylaxis, or We Got Really Lucky Yesterday

We got really lucky yesterday. 

(I’m going to start there, but hope people will read on, because our story could have had a terrible ending, and education is the key…)

While my blog, Eating Peanut, is about my daughter's life-threatening food allergies and her journey through an Oral Immunotherapy (OIT) clinical trial, I am going to share this story about my son, Carl, here.  It is relevant because, without our experiences eating peanut over the last 2 1/2 years, I might not have understood the significance of what was happening, either.

It started with a phone call from the scoutmaster Carl is attending overnight camp (nearly 5 hours away) with this week -- at 11:15 am.  I knew it couldn’t be good.

But I wasn’t ready for how very bad it was.

The scoutmaster explained that Carl had been bitten or stung by something (bee, spider, who knows?  We might NEVER know) at camp on Monday.  Because the bite/sting was very painful, Carl had seen the nurse at camp.  She gave him an ointment and he went back to being the very happy Boy Scout camper that he is.

By yesterday morning, the bite/sting had (according to Carl, 11, later) swollen “times a million.”  Unlikely, but I got the point.

The scoutmaster took Carl back to the nurse, who gave him Benadryl and told him to return if anything else happened. 

All good, right?

Right.

But then, things changed.

He vomited.

The scoutmaster immediately took Carl to the camp nurse, who assessed.  The site of the bite/sting was even more swollen.  Carl’s blood pressure was elevated and his pulse ox was 94.  He was wheezing, and complaining that his mouth and throat did not feel right. 

The scoutmaster told me they had called an ambulance, and were transporting him to the local hospital.  (I now know that the camp started the summer with three undesignated EpiPens, and had already used one when yesterday rolled around.  As this unfolded, I didn't know that...)

I told her I knew a few things about allergic reactions.

I told her I thought it sounded like an anaphylactic reaction.

I told her I wanted them to give epinephrine.

She told me to hold on…

My kid with no known allergies appeared to be having an anaphylactic reaction.

I was barely breathing when the EMT got on the phone.

She told me her name, but I don’t remember it.

She told me Carl was having an anaphylactic reaction.

She reviewed the symptoms, and told me that the swelling in his throat had doubled since she arrived.

My kid with no known allergies appeared to be having an anaphylactic reaction.

Fighting tears, I asked her to administer epinephrine.

She explained she was an EMT, and could not administer epinephrine.

I tried another tact:  “I am his mother, and I am telling you to administer epinephrine.”

She explained respectfully that she did not have epinephrine.

She told me she had to go, as talking to me was delaying transport to the ER.

GO!

The EMT turned the scoutmaster's phone back over to her.  The scoutmaster told me she would follow the ambulance.  I told her I was on my way -- knowing full well that there was nothing I could do in the minutes that mattered, but knowing still that I had to be there for the after -- hoping, praying that the after would not be what I feared.

I was panicking as I tried to gather the papers I was in the process of submitting at the school district office to prove Carl's (and his twin sister's) residency for the following year…

I got in the car and started driving north.  I didn’t know where I was going, but I knew I needed to go north.  North.  North.  Fast.  But not so fast I got a ticket.  NORTH!  I called my husband, and fighting panic explained the situation.  I had him figure out the nearest ER to the camp and nearly vomited when I realized it was thirty minutes away from the campground.

My kid with no known allergies appeared to be having an anaphylactic reaction.

Fighting for calm, trying not to cry, I raced northward. 

Helpless doesn’t even begin to describe how I was feeling.

I called the ER of the hospital closest to the camp.  I spoke with a very kind woman whose name I don’t remember.  I think she was the ER charge nurse.  I was crying and I didn’t know whether to be relieved or scared when she told me they knew he was on his way, that the EMTs were transporting him quickly, that the doctors and nurses were waiting for him, that they were ready with epinephrine for him.

My kid with no known allergies appeared to be having an anaphylactic reaction.

I told the ER charge nurse that I had a lot of experience with anaphylaxis.

I told the ER charge nurse that I wanted the EMT to administer epinephrine.

I told her the epinephrine would not hurt, but that waiting might result in a terrible outcome.

She told me she knew that, but that the EMT did not have epinephrine, but that they would administer it when Carl arrived at the hospital.

I was panicky, and felt incredibly helpless, trapped in a set of circumstances beyond my control.  My kid with no known allergies appeared to be having an anaphylactic reaction.

What I didn’t know at the time was that the swelling around the site of the bite/sting was continuing to swell.  I am glad I did not know.

What I didn’t know at the time was that all they could do for Carl en route to the hospital was give him oxygen.  I am glad I did not know.

When Carl arrived at the hospital, they gave an injection of a fast-acting steroid, gave another dose of Benadryl, drew blood and hung a bag of IV antibiotics -- covering both an allergic reaction and an infection, as they weren’t sure what was happening.

The site of the bite/sting continued to swell at first, and they traced a second line around it, in purple (the EMT’s line -- from about 11:20 am -- is in black, and is fainter in the photo).  The purple line was done in the ER at 12:45 pm.

(Photo taken at about 5:00 pm, hours later.)

As I drove north, I talked to the ER nurse, and to the scoutmaster, and to the ER doctor, and to the very kind woman who I think was the charge nurse.  And I talked to Susan’s allergist, and later, when I knew more, I talked to Susan’s allergist’s receptionist, who kindly took down a detailed accounting of all I knew.  As I got further north, I had to turn the phone calls over to my husband, as cell service was spotty at best.  He talked to Susan’s allergist -- who is now Carl’s allergist.  He talked at length to our pediatrician (conveniently, Susan had her annual physical as our pediatrician’s last appointment of the day).

By the time I was that far north, it sounded like Carl was stable.  I was less panicked, but still scared.  I know the risks of biphasic reactions are greater when administration of epinephrine is delayed.  I suspected the risks were even greater when epinephrine was not administered.

I put my husband in charge of making a plan.

I stopped to go to the bathroom.

I breathed. 

I sent my thankfulness and relief out into the universe.

I have no words for how terrible it was to be trying to educate the person responsible for transporting my son to the hospital on the signs and symptoms of anaphylaxis.  The moment of crisis is not the moment.

It is simple:

Administer epinephrine with any two symptoms.

Carl had five:  wheezing, drop in blood pressure, swelling at site, nausea/vomiting, throat closing

Administer epinephrine with any one major symptom.

Carl had three:  wheezing, drop in blood pressure, throat closing

Strung together like that, I find myself wondering if really that is just one major symptom.  No matter how you count the symptoms, a case for epinephrine was there.

Carl is fine.

We got lucky.

Sososososolucky I feel sick just thinking about it.

Carl’s pediatrician feels strongly that what happened yesterday was an allergic reaction to whatever the venom was.

Carl’s new allergist wants to test for hypersensitivity to a panel of venoms.

Carl is the (proud?) new owner of two EpiPens.  I am (fortunately), confident he knows what to do with them.  I believe -- because of all he has learned by being Susan’s brother -- that he would (and could) self-administer epinephrine if he needed to.  Fortunately, two EpiPens fit nicely inside the cargo-pocket of his Boy Scout uniform shorts. 

In the light of day, I cannot think too much about how lucky we got as I begin working out in my mind the advocacy and education that needs to take place.  I am thinking about Annie LeGere’s mother, who is tirelessly working to be sure all emergency responders have access to epinephrine.

There is so much work to be done.

So much education to do.

What started as a simple journey -- with the goal of protecting Susan from accidental exposure to peanut -- has turned into a lifetime of work.