to let people know as soon as
possible, and maybe, by chance,
someone contacts us saying that
they know somebody who needs
This goes to show no volunteering
act is too small. We all have it
in us to spread awareness and can
start by looking for opportunities
in our own communities.
On October 9, 2014, Dr. Vidya
Sankar presented at the American
Dental Association’s (ADA)
Annual Meeting in San Antonio,
Texas. Her presentation, “The Dental
Detective: Investigating Autoimmunity,”
addressed the common
symptoms and referral patterns
for several different autoimmune
diseases, including PV and MMP.
This served as an excellent way to
provide continuing education on
these illnesses to the entire dental
“The oral health care professional
will commonly encounter
patients with an array of oral and
systemic health needs,” Dr. Sankar
said. “The ADA offered us a
platform to review some of these
lesser known conditions in order
to identify patients with potentially
undiagnosed needs and act as
a conduit to aid in diagnosis and
management. Additionally, linking
up the dental professionals
with professional societies such
as the IPPF will help to increase
awareness and access to care and
potentially increase our patients’
quality of life.”
The IPPF thanks Carlos, Dell,
and Dr. Sankar for their collaboration
on this project and their devotion
to raising awareness.
Together we can raise awareness
and promote early diagnosis
of PV and MMP. We all have ways
we can contribute. These are just
three of the many more tales of
awareness there are to share.
What’s yours? The IPPF encourages
our Community to get
involved with the Awareness
Campaign. If you are interested in
learning more about the campaign
or getting involved, please contact
Kate E. Frantz, MPH, CTTS, is
the Awareness Program Manager
at the IPPF living in Dixon,
CA. She is a contributor to
the Quarterly newsletter in her
“Awareness and You” column.
Kate can be reached at awareness@
. . .continued from AWARENESS, page 6
Vidya Sankar, DMD, MHS, University of
Texas Health Science Center, San Antonio
generous. It has been overwhelming and we have
done really well so far. The IPPF has helped us, too, by
providing promotional material that we handed out.
We also had T-shirts and a banner made, which have
definitely helped raise awareness of PV. Please check
out the IPPF Facebook page to see photographs of
Thanks entirely to the funds we raised, I received
my first rituximab infusion two weeks ago, and it went
well. I am due for my second infusion next week, and
while I know I have a long wait, I am feeling very positive
about the outcome.
I just hope and pray it will work for me. I have been
advised that it normally takes at least three months
to determine whether or not rituximab is effective,
and I understand it is likely I will need further infusions.
Over the next few weeks, I will hopefully be
reducing the steroids and immunosuppressents.
As you can imagine, trying to cope with this disease
over a number of years has taken its toll. I have gone
from being a very fit and healthy man to being overweight
with a painful, incurable disease. This whole
period has been a depressing and difficult time for
me and my whole family. The most difficult aspect of
living with this disease is the excruciating pain I have
had to endure, and therefore I am looking forward to
returning to a life free of pain where I can enjoy keeping
fit and healthy and return to my weekly cycling
event with my brothers.
. . .continued from ROAD TO REMISSION, page 13
Scott is an Operations/Call Center Manager
who lives and works in West Midlands, United
Kingdom. Scott is happy to raise P/P awareness
by telling his story in the Quarterly. Once he is
feeling better, he hopes to continue to raise
funds for other PV sufferers. If you would like
to support Scott’s treatment fund, please visit
a time . . . what else could I do?
In December one of the doctors
noted that along with the
predominantly oral presentation,
I had some skin symptoms, but it
wasn’t until July 2011 as a result of
my skin condition worsening with
blisters on my scalp and face that
I was referred to a dermatologist.
In September 2011, as my dermatologist
had suspected, my
PV diagnosis was confirmed and
refined as pemphigus with predominant
involvement. Unfortunately, my
dermatologist moved to another
country and I was referred to a
new dermatologist. I was now being
seen by both an oral consultant
and a dermatologist. I continued
a variety of different treatments
(Dermovate, Protopic ointment,
Elidel, Betnesol nasal spray, and
prednisolone mouth wash) and
drugs (prednisolone, azathioprine,
mycophenolate). None of them
were particularly effective.
During this time, the erosions
had spread all over my face and
nose and were particularly severe
on the top of my head. I was then,
and I still am, obese due to the
large amount of steroids. The disease
is extremely painful; so much
so it affected my sleep because I
cannot rest my head on the pillow
at night. I remember visiting
the hospital one day, when my lesions
were particularly bad and the
nurse thought I had been in a car
Every time I washed my hair
or face I would lose a piece of
skin. Every time I ate anything my
mouth would start to bleed. I can
honestly say at this point I was a
broken man with little quality of
life. I would hide the pain and discomfort
from my family. Many
times I said I was okay, but inside I
felt so depressed and despondent:
there did not seem to be any light
at the end of the tunnel. It felt like
no one could help me and there
was nowhere to go.
In an attempt to seek help and
get yet another opinion, my dermatologist
arranged for me to
see a world expert in dermatology
at Guys Hospital who determined
that my high daily steroid
requirement could not continue.
He recommended a drug called
rituximab and initiated a request
for funding via National Health
Service (NHS) of England. Unfortunately,
the funding request was
declined. I was devastated.
My wife wrote to our local member
of Parliament hoping his support
would sway NHS to reconsider
their decision, but it didn’t. My
wife and family decided that we
had no alternative but to try and
raise the money ourselves, and as
a family we began our mission.
In preparation to receive rituximab,
I was advised to start weaning
off the drugs I had taken for so
long. This had disastrous consequences
and led to a massive flare
up — the worst I have ever had. I
had to be taken into hospital as an
emergency case, where for over
four days I received three pulsed
doses of IV methylprednisoloine
and IVIG infusions.
This made an enormous improvement
to my pemphigus and
for the first time in years I felt so
happy and such a sense of relief as
the pain had largely subsided.
I was aware IVIG is meant to be
an interim treatment and a temporary
solution. Generally patients
remain lesion-free for up to 30
days. In my case only two weeks
passed. I continued to receive IVIG
every two to four weeks, and it
seemed to stop disease progression.
Because I had to be hospitalized,
my consultant submitted
additional clinical information to
the NHS explaining my new circumstances.
Sadly, this renewed
request for funding of rituximab
was declined again.
One of my family’s first fundraising
events was a 100-mile
bicycle ride. I am so proud of my
son, brothers, and friends who
took part. Everyone who donated
tween the winter holidays and early January is the
most busy time of the year for mental health professionals,
in part because it is difficult during these
times of reflection not to become depressed, if that
is a struggle.
It is important for those who are affected in this way
to make a special effort to practice positive thinking
(mindfulness) and reflect not only on the unpleasant,
but also pleasant memories. I’ve suggested imagining
that you’re putting negatives into a box and taping it
up; then seeing yourself locking that box into a trunk;
then imagining wrapping chains around the trunk
and leaving it for a later date. You will know where the
trunk is and how to retrieve what is inside, but you
can also choose to keep all or most of the contents
locked up for a while. At this point you may be willing
to accept offers of help from others, which is among
the healthiest of escapes.
The holidays are also a good time to reach out to
others. We’ve all heard that it is truly more satisfying
to give than to receive. Inviting lonely people to an
event or volunteering at a nursing home or other facility,
for instance, will have a positive effect on your
mood. As a way to track your progress through the
difficult holiday period, keep a record of your mood
and activities or lack of activities. As regularly as you
can, rate your mood on a 1-to-10 scale with 1 being
the worst and 10 being the best. Also track your activities
or lack of them as a record of the patterns and
both positive and negative triggers. Everyone is different,
and how you are in this world and during this
time of year will not be the same as for others. Hold
onto necessary traditions, but allow yourself to have
new experiences. You may surprise yourself.
Besides these mindfulness approaches and techniques
related to reaching out to others, there are
a number of physical solutions that may help with
dealing with SADS. Vitamin D supplementation has
been found to be useful. As well, daylight spectrum
lamps (or just special bulbs put into existing lamps)
can also help counteract the seasonal lack of natural
sunlight. Still, many people will need different medications
or increased doses of their current medications.
Checking with your primary care physician or
having an evaluation by a psychiatrist or psychologist
can be helpful and may greatly increase the quality of
your holiday season.
After eight months of oral symptoms and appointments
with doctors and dental specialists, I received
my own PV diagnosis right before Thanksgiving. One
of the first posts I read on the P/P online discussion
group forum was from someone (Hi, Skip!) who recounted
feeling lucky to swallow mashed potatoes in
his first symptom-filled year. That really helped me
to put things in perspective.
I focused on what I could do, not what I could not
do. Unless you’re having a flare or going through a
particularly rough time physically, get out and do
things. Happiness is a choice, and all humans have
the ability to be happy.
Happy holidays and my best wishes for a terrific
- of COL17 to test the effect of BP (human) patient-derived
antibodies. Generally, mice are a great experimental
model for studying the human immune system
since the mouse and human systems have been
found to be mechanistically very similar.
The authors genetically removed C3 from the humanized
COL17 mice and showed that indeed, they
lack the complement system. The authors also isolated
four different autoantibodies from four different
BP patients and found they vary in the degree
to which they activate the complement system. All
of the BP antibodies could induce skin detachment
(characteristic of blisters) when injected into either
the normal mice or in the complement-deficient
mice, demonstrating the complement system is likely
not at play in BP blister formation.
They next developed new antibodies that recognize
the exact same portion of COL17 and found a
correlation between the level of COL17 recognized
by the autoantibodies and blister formation. Recent
studies have shown COL17 antibodies not only recognize
and bind COL17 but also deplete it from cultured
cells. Ujiie and colleagues repeated that result show it
is complement-independent. As well, they find the
same effect of COL17 depletion in the COL17-humanized
mice - the antibodies caused blisters and
simultaneously reduced the amount of COL17.
Finally, the authors found that this was due to an
induction of the ubiquitin-proteasome system, the
machinery of cells that acts as a garbage disposal for
unwanted proteins. In this case, the COL17 autoantibodies
somehow mark the otherwise normal COL17
for destruction, possibly setting the stage for BP
symptoms and disease.
Several mechanisms may still be at play to mediate
the effects of COL17 autoantibodies generated
by BP patients (see figure). These include a degradation
system, as suggested from the current work or
COL17 may be internalized into cells upon binding of
the autoantibodies, as has been seen in studies from
other labs. It is also possible that COL17 gets internalized
first and then the intracellular proteasome
system degrades it. In any case, COL17 targeting by
BP autoantibodies is a probably occurring by a more
direct mechanism than if it involved the complement
It is possible that BP shares a mechanistic basis with
other autoimmune mucocutaneous diseases such as
pemphigus vulgaris, where autoantibodies recognize
desmoglein proteins Dsg1 and Dsg3 in keratinocytes
of the epidermis. Therefore, understanding the underlying
mechanisms at play in blister formation in
the various P/P diseases will be applicable to all patients
- What it Means to be a Citizen of the IPPF
- It’s a pleasure to be here with you all today, and an honor to take the helm, along with our CEO Will Zrnchik, at what I believe is an inflection point in the Foundation’s growth. I wanted to share some sentiments that I hope will guide and enrich your experience this weekend. For some time now, I have been thinking about what it means to be a citizen of the IPPF.
I am not a patient. I am a family member. I am involved with this Foundation because I care. I don’t want anyone to go through the pain and suffering I saw my mother go through. I know from seeing her and other patients that physical discomfort and pain are significant, but even worse is the loss of confidence that comes from feeling like you are a shadow of your former self, feeling alone and uncertain, and finding that even people who you thought could help – like your family doctor -- may not be familiar with the disease. This foundation can help. Remember the people sitting next to you: they can help. And when you feel less than yourself, don’t give in.
Once again look around: these people can help. The foundation can give you a community of patients, caregivers, doctors and researchers. As you get control of physical pain and psychological stress, you will see a light at the end of the tunnel and eventually make it outside, and although the world will be different than what you had previously known, it is bright and it is happy. Now you’ve earned ... your badge of courage, your stripes if you will, but to develop as a citizen of the IPPF community, you now need to look back and help others in need who still only see darkness. Shine the light down the tunnel, offer words of encouragement from a soothing voice and lend a strong hand to pull your fellow traveler from the tunnel and let them see the bright of day. Show them what remission is like and how they might get there. Even more, be a catalyst for positive change at the community level whether it is fostering research, helping raise funds, or educating doctors and patients.
I see all of the activities of the foundation coming down to four imperatives:
The foundation is about these four imperatives. Help us fulfill them for yourself and for all those around you.
If we come together and help each other, we can make an undeniably meaningful difference in patients’ lives. The foundation is a vehicle for your efforts. The foundation gives embodiment to our collective aspiration to help our patients and their families. I hope you will get involved.
My mother is a success story. Though she has to be vigilant, she is living a good life. So should all of our patients. For those recently diagnosed, stay strong – you will get through this. For those in remission, lend a helping hand to those still dealing with active disease.
For all of you, think about how you can catalyze positive change for the entire community. As you approach this weekend, ask yourself what you can do to be a true citizen of the IPPF. I wish you all the very best.
- We are trying to improve a patient’s quality of life -- skin care, eye drops, puréed foods, emotional support.
- We are trying to reduce the length of time it takes to diagnose patients. This comes from educating dentists, doctors and nurses.
- If we can support research on disease flares, we might lay the groundwork for an academic institution or company to develop tests to predict emergence of flares. In fact, this may be a more pragmatic approach in the near term than going for a cure.
- Ultimately we can support research and product development efforts that will someday bring us to a cure and make this picture disappear.
Vanguard Health is the latest company that's signed up with the Dossia Consortium to offer its employees electronic health records as a work benefit.
But when will it become mainstream for any and all patients to access their health records electronically, rather than it being a rare job perk for some?
The HITECH federal stimulus legislation signed into law in February not only offers healthcare providers financial rewards starting in 2011 for their meaningful use of e-medical record and other health IT systems. The law also gives patients the right to access to their personal health information electronically, too.
That might seem like a given-- of course it makes sense for patients to have access to their medical data-- after all, it's information about their health, right? But with all the challenges involved with hospitals and doctors making the transition from paper to electronic records, and them figuring out how to share patient data (and many aren't anxious to do that for competitive and other reasons,) you have to wonder how much effort they'll spend--at least initially--in their IT project plans to provide patients with access to their digitized data.
How much of this information should they show patients? Could the data be misunderstood or taken out of context by patients? What about security and privacy worries? Those are just a few of the many concerns some healthcare providers have about giving patients an electronic window into their health data.
While healthcare providers figure that all out, some employers--like Dossia member companies-- have already decided to get a jump on providing patients access to at least some of their health data in the hopes that it will help employees better manage chronic and other medical problems they and their dependents face. So far, much of the data for Dossia e-health records comes from the payers, health plans, pharmacies that provide various services to Dossia employees via their health benefit packages. The data for the most part isn't coming directly from the actual doctors and hospitals caring for these patients.
Since so many Americans get their health coverage from their jobs, it's not all that surprising that many patients who also have electronic access to their health data are able to do so because their employers are providing e-health records as a work perk.
But the goal is that eventually for more patients to see their health data because their doctors and hospitals are providing secure access to that information, too.