If you need more information
on a particular meeting or are interested in hosting a meeting
in your state, please contact Lelia
Brougher.
My husband Paul and would like to thank everyone who came to the
In-Touch
meeting at our house in early June. Your enthusiastic response
made the
meeting more valuable to us all. A group of nine patients and seven
of
their fathers and husbands had a chance to talk and share information,
and
to ask questions of Dr. Neville Pimstone, an APF Scientific Advisory
Board
Sponsor who gave generously of his time and expertise.
Most of
the patients attending were women, and all have or suspect one
of
the acute porphyrias. The one man who attended has EPP. Our youngest
attendee, a teenager, has been horribly sick for a year. She
has been
unable to keep down any food or fluids and lost a lot of weight
over
several months before her doctor put her on total parenteral
nutrition
(TPN). Getting enough calories is a big issue for porphyria patients:
fasting can bring on an attack, just as harmful drugs can. It
can also
worsen an attack in progress.
Patients at the meeting talked a
lot about difficulty obtaining treatment.
Nearly everyone has run into problems with some central aspect
of their
care, whether that's getting diagnostic tests performed properly,
or
finding a doctor who will both pay attention to treatment protocols
and
work with the patient to figure out what her/his disease course
is and
adjust treatments accordingly. For many of us, these problems
stem from
relying on doctors who have no interest in the porphyrias; are
overworked
with other, and often more familiar, illnesses; or are overwhelmed
by the
sea of questions that come up when treating a rare and highly
variable
disease.
All of our problems with medical care so far point to
the desperate need
for a porphyria center in southern California -- both to give
porphyria
patients in the area a place to go for comprehensive care, and
to provide
young doctors an opportunity to train with the current generation
of
porphyria specialists, many of whom are retired from practice
or nearing
retirement. If you want to help make such a southern California
porphyria
center a reality, call the APF office to find out how.
If you
want a chance to talk to a specialist on your own, holding your
own
meeting and asking the APF to help is a great option. So is participating
in a drug or other clinical study—you get to meet and talk
with a
specialist while advancing medical knowledge about porphyria.
Looking back on my participation in the recent Porphozym trial
and its
negative result, I have no regrets. I found it a worthwhile use
of the
time and travel (which included one two-day hospitalization and
five
follow-up visits, all a plane-ride from home). I say this even
though it
seems to be taking my frequent attack cycle a while to settle
down from
the physical challenge of all that air-travel. I met Dr. Herbert
Bonkovsky, an APF board Sponsor with decades of experience treating
the
acute porphyrias and an extremely kind and compassionate doctor,
six times
during this study. I had the chance to ask his advice, go home
and try it
out, and then discuss and revise treatment plans with him. Dr.
Bonkovsky
also took the time to investigate my symptoms thoroughly and
give my local
doctors feedback on my care. So that's the short-term outlook—my
care is better now than it was a year ago because I participated
in this study.
The longer-term outlook is that I was able to help the scientists
learn
more about how the disease works, which is vital to managing
it and may
mean care improvements in my lifetime.
Some important announcements
from the meeting:
