Lighten Up Program - Florida Hemophilia Association

The Official Newsletter of the Florida Hemophilia Association
Lighten Up Program
SUMMER 2013
INSIDE
Executively Speaking
Page 2
Community Corner
Page 3 - 4
NHF News
Page 7 - 8
FHA Resource Center
Page 11
“SAVE THE DATE”
30th Annual Family Education
Symposium
September 6-8
Amigos Dinner-Miami
September 15
NHF Annual Meeting
October 3-5
Educational Program-Ft. Lauderdale
October 22
Maintaining healthy
eating habits and an
active lifestyle is very
important to bleeding
disorder patients.
24th Annual
Swing for the Kids
Golf Tournament
December 9
Dr. Joanna Davis
Holiday Family Fun Day
December 15
HFA Symposium
March 27-29, 2014
4th Annual Walk
in the Jungle
May 3, 2014
eld on June 1, 2013 at the Art Institute
of Fort Lauderdale, the Lighten Up
program brought together community
members for a fun afternoon of cooking,
learning and best of all, eating! The day
started off with a talk by Dr. Joanna Davis
from the University of Miami HTC and
Sheah Rarback, Director of Nutrition at the
Miller School of Medicine. Sheah gave the
H
group great tips on healthy eating and how
easy it can be to integrate healthier foods into
daily life. Dr. Davis then discussed why
maintaining healthy eating habits and an
active lifestyle is very important to bleeding
disorder patients. Carrying extra weight can
place a heavy strain and cause damage to
joints when a patient has a bleed.
Continue Pg. 2
1
ExecutivelySpeaking
MensajeEjecutivo
It’s been a busy time for us here at the
Florida Hemophilia Association especially with the planning of our 30th
Annual Family Education Symposium
scheduled for September 6 - 8 at the
Embassy Suites in WPB. We’ve got a
terrific line-up of fun, education and
networking opportunities for the entire
family that’s surely not too be missed.
We also just hosted the last of our
three scheduled “Swim with Dolphins” outings in Key Largo.
Participants learned about marine life and even got to see and
hold some very rare creatures like the endangered “Queen
Conch” and sea urchins. You can see the photos of the dolphin
day and all of FHA outings in the Photo Gallery on our webpage.
Hemos estado muy ocupados en la Asociación de Hemofilia de
Florida, especialmente en la planificación de nuestro 30er.
Symposium Anual de Educación a la Familia , a llevarse a cabo del 6
al 8 de setiembre en el Embassy Suites de WPB. Tendremos gran
variedad de cosas divertidas por hacer, así como también actividades educativas y grupales para toda la familia que estamos seguros
no se perderán. También acabamos de auspiciar la última de las
tres actividades programadas “Swim with Dolphins” (Nadando con
los Delfines) en Key Largo. Los participantes aprendieron sobre la
vida marina y hasta pudieron ver y tocar algunas especies en peligro de extinción como la “Queen Conch” (Concha Reina) y erizos
de mar. Pueden ver las fotos de la actividad del día de los delfines y
de otras actividades de la FHA en la Galería de Fotos de nuestra
página web.
We’ve got a myriad of things up our sleeves for you in the coming months! You’ll want to make sure to open all of the emails
from us and also check out our website at www.floridahemophilia.org for new programming, upcoming educational dinners, holiday fun, fund-raising events and a whole lot more!
And finally a special request to all of you to help out your chapter
(FHA) by volunteering, fund-raising and or joining one of our
committees. With your help we can continue to provide some
great programs and outings for all of you to enjoy.
Tenemos infinidad de cosas que ofrecerles en los próximos meses!
Asegúrense de abrir todos los emails que les enviamos y también
chequear nuestro website www.floridahemophilia.org para estar
al tanto de la nueva programación, de las próximas cenas educativas, de las diversiones programadas para los feriados (holidays),
de los eventos para recaudar fondos y muchísimo más!
Para concluir, quisiera pedirles a todos ustedes un favor especial:
que apoyen a su capítulo (FHA) ya sea como voluntarios, en la
recaudación de fondos y/o formando parte de uno de nuestros
comités. Con su ayuda podremos seguir brindándoles los excelentes programas y actividades especialmente diseñados para el
deleite de todos y cada uno de ustedes.
I look forward to seeing you soon!
Ansiando verlos muy pronto!
Debbi Adamkin
FHA Executive Director
Debbi Adamkin
Directora Ejecutiva de FHA
Lighten Up Program Continued ...
After the discussion, the group made their way into the kitchen
to cook with culinary instructors and students. The recipes
prepared were submitted prior to the event to be “lightened up”
by the chefs. Some of the dishes prepared included: oven-fried
chicken, macaroni-and-cheese, mofongo, braised collard greens and
pecan pie.
We would also like to thank the culinary instructors and students
of the Art Institute of Fort Lauderdale for their time
and teaching us how to cook a delicious and light meal!
This program was made possible through a grant
from the Hemophilia Alliance Foundation. We’d also
like to acknowledge Grifols for being a Program
Sponsor.
2
SUMMER 2013 • LIFELINE
Mother's Educational
Focus Luncheon
Submitted by Amy Hook
On May 18, 2013, my mother and I attended the Mother’s
Educational Focus Luncheon at Brio Tuscan Grille in Gulfstream
Park. I just wanted to let you know how much my mother and I
appreciated the luncheon. It was the second time we attended.
The other mothers that were there were really nice. The
atmosphere was so laid back and relaxing. We all shared our
stories and I truly felt we were
able to connect with each other.
Although I would never wish
hemophilia on anyone, it was so
comforting to know that we are
not alone. When I told my
husband what an enlightening
experience I had, he got
jealous. He wants to know
when the Father’s Luncheon
will be! I look forward to next
year’s luncheon.
This program was made possible through grants provided by: Baxter, Bayer and Kedrion Biopharma
SUMMER 2013 • LIFELINE
3
SWIMMING WITH THE DOLPHINS
Submitted by O
mar
Submitted by
Ayden
I had an amazing
time at dolphin camp
this year. This was my
third year going to
Island Dolphin Care.
irt had a
This year was very special because Squ
born in August of
was
baby dolphin. His name is Tashi and he
with Squirt and
swim
to
last year. I was lucky enough to be able
old and he can
year
a
than
baby Tashi. Tashi is amazing he is less
has so many
IDC
).
d
moo
already do all kinds of tricks (if he’s in the
tank named
h
touc
the
in
al
cool things to see. They had a new anim
h is a
whic
hare
sea
a
d
Squishy. He is a very weird animal calle
to kiss
ce
chan
a
had
also
I
!
type of mollusk. He was very squishy!
a
had
I
e.
som
awe
was
the sea cucumber for good luck. The food
was
day
the
of
part
rite
favo
cheeseburger and it was delicious. My
hins. It was so much fun.
being able to go swimming with the dolp
with Squirt and Tashi. The
It made me so happy to be able to swim
Squirt. I had so much fun
best part was when I got to kiss and hug
and Island Dolphin Care.
at dolphin camp this year. Thank you FHA
"I had a very grea
t experience in
2nd. This was th
Key Largo on Fr
e first time I sw
iday, August
am with dolphins
three dolphins na
. I swam with
med BB, Figi, an
d Ding . At first
nervous but the
, I was a bit
instructors kept
te
lling me that the
very friendly an
dolphins were
d that I was goin
g to have the be
My instructors
st experience.
were extremely
nice in guiding
how not to plac
me on how to an
e my hands on
d
each dolphin. Th
e best part
was when I was
up and close
and kissed the do
lphin.
It was great to le
arn
about facts abou
t fish and
dolphins which
are
mammals. I actu
ally kissed
the sea cucumbe
r , 7 years
of good luck. M
uah!!!! Lol
I also enjoyed
painting on my
shirt and enjoyi
ng
my day with my
mom and cousin
s. Thanks FHA."
Submitted by Genesis
ifer
Submitted by Jenn
others with
r and I have two br
My name is Jennife
through on a daily
what my brothers go
ficult it is for
hemophilia. Seeing
n’t imagine how dif
ca
I
.
me
to
ing
pir
life. They’re
basis is very ins
ilia take over their
ph
mo
he
let
n’t
do
Debbi and
them. But they
careful. Thanks to
be
to
ve
ha
d
an
ks
aware of the ris
n to experience so
mily and I have gotte
. It was an
all her friends, my fa
ming with dolphins
im
sw
e
lik
s
ing
th
l
We got to swim
many wonderfu
unforgettable day!
It was beyond
with Bella and Bob.
! Every time
amazing for all of us
Education
we go to the Family
a lot about
rn
lea
Symposium, we
fun at the
hemophilia and have
u to everyone
same time. Thank yo
possible.
who has made this
On July 12th, I visited for the very first
time Island Dolphin
Care in Key Largo. The Island Dolphin
care staff was so caring,
friendly and patient towards us. Kim told
us about a lot of
invertebrates like the sea urchins, star
fish etc. The food was
great. After we ate, Eli taught
us about the dolphins and
then we swam with them! This
experience was just amazing.
The dolphins were so friendly
and I felt like I was in the
dolphin show at Seaworld! It
was just awesome and I want
to thank the FL Hemophilia
Association and Island
Dolphin Care for allowing me
to experience this.
Submitted by Sharella Flowers
Island Dolphin Care. They enjoyed
The kids had a wonderful experience at
vertebrate and invertebrate animals that
the touch tank and learning about the
d begin able to interact with the
live in the ocean. The kids especially love
day they asked if they could come back
dolphins in the water. At the end of the
in
we have the opportunity to participate
tomorrow and do it all again. We hope
this adventure again in the future.
4
SUMMER 2013 • LIFELINE
This program was made possible through grants provided by: Bayer and Grifols.
To view more pictures of “Swimming with
the Dolphins”, visit us at www.floridahemophilia.org
SUMMER 2013 • LIFELINE
5
NHF News • August 2013
Preparing Your Daughter for Her First Period
By Leslie Quander Wooldridge
M
ichigan attorney Mindy Buurma, 36, is the mother of four hildren under
age 10. She has type III von Willebrand disease (VWD), and her two
daughters (ages 5 and 3) have type I. She plans to tell her daughters about
menstruation when they are 8 or 9. “I want to make sure the information is coming
from us and the doctors rather than from school or other children,” she says.
Buurma has the right approach, say experts. Although girls can start their periods
at 8 years old, the American College of Obstetricians and Gynecology (ACOG) says
90% experience their first period, or menarche, between 12 and 14. For young
women with bleeding disorders, their menstrual flows can be especially heavy, long
and painful, a condition called menorrhagia. Typically, their periods continue longer
than seven days—the top range of what is considered normal.
Impending menstruation can cause nervousness in young women. Girls and
teens worry the blood will leak, staining their clothes, says Danna Merritt, MSW,
LMSW, a social worker at the hemophilia treatment center at the Children’s Hospital
of Michigan in Detroit. That’s why it’s important to prepare young women early on.
They need to know what to expect and what to do if they later have a heavy flow,
severe cramps or other complications.
Starting the Discussion
Mothers should have ongoing discussions about puberty and bodily changes with
their daughters. They should avoid one long talk, says Lisa Perriera, MD, MPH,
assistant professor of obstetrics and gynecology at University Hospitals in Cleveland,
who treats young women with bleeding disorders. Experts suggest having the talk
when your daughter is 8–12 years old. The initial discussion should cover what
menstruation is, what happens in the body, what products to use and how to handle
cramps, moods and fatigue. Chances are your daughter may already have had a
previous discussion in her physical education or health class at school. And some
tampon and pad manufacturers sell first-period kits for mothers to use when they
“have the talk” with their daughters.
Parents should answer all questions truthfully, says Perriera. Because young
women can be shy or embarrassed to talk about their bodies around their parents,
they should be encouraged to talk to their gynecologist or primary care physician
privately. Merritt notes that doctors, nurses and social workers at your hemophilia
treatment center also can talk to girls about menstruation and guide parents on what
to say at home.
Complications and Treatments
Unlike Buurma’s older sisters, who have type I VWD—the most common and
the mildest type—she has the most severe type. “We all knew that it would be a
problem when I started menstruating,” she says. “I bled for about two months
straight and was hospitalized for about three and a half weeks.”
Knowing what’s normal or out of the ordinary is important for a young woman
with a bleeding disorder during her first few menstrual cycles. “It’s not normal to be
soaking a pad in an hour,” Perriera says. Other complications that require a
physician’s attention include severe cramps that interfere with school or activities,
and bleeding between periods.
To reduce heavy bleeding, hormonal medications may be needed. But
hormonal medications are also prescribed as birth control for sexually active
women, which can sometimes present religious, cultural and other concerns for
parents. When she was younger, Buurma’s doctors prescribed “the pill” as
continuous hormonal medication to relieve her symptoms. “We are Catholic. In our
religion, birth control is not an acceptable practice,” she explains. But her parents
made it clear that taking the pills wasn’t against church doctrine because she was not
using them to prevent pregnancy. Other hormonal options include certain
intrauterine devices (IUDs) or hormonal implants.
“I wish hormonal medicines weren’t marketed as birth control. They are period
control. They are bleeding control,” says Perriera. Still, she realizes some parents
have concerns and may request nonhormonal treatments first. That’s when
hematologists can help.
Because each girl with a bleeding disorder experiences a different degree of
severity with menstrual bleeding, it may be wise to meet with her hematologist to
discuss treatments. “For example, women with von Willebrand disease can be
prescribed a hormone called DDAVP in the form of a nasal spray during menses to
decrease the flow of bleeding,” says Sanjay Ahuja, MD, MS, a pediatric
hematologist and oncologist who works with Perriera. “Women with type III von
Willebrand disease may need von Willebrand factor replacement during menses.”
Further, physicians will also combine hormonal and nonhormonal medications in
patients with severe bleeding, he says.
No young woman should suffer in silence. “I think the messages ‘you’re going
to be OK’ and ‘we will deal with any issues together’ are huge,” Merritt adds.
Source: HemAware-April 2013
Cómo preparar a su hija para su primera menstruación
By Leslie Quander Wooldridge
Mindy Buurma, abogada de 36 años de Michigan, es la madre de
cuatro niños menores de 10 años. Ella padece de la enfermedad de von
Willebrand (VWD) de tipo III y sus dos hijas (de 5 y 3 años de edad) padecen del
tipo I. Ella piensa explicarles a sus hijas lo que es la menstruación cuando tengan
alrededor de 8 o 9 años. “Quiero asegurarme de que reciban la información de
nosotros y de los médicos en lugar de hacerlo en la escuela o de parte de otros
niños”, dice ella.
La Sra. Buurma lo está enfocando de la manera correcta, dicen los expertos.
Aunque las niñas pueden empezar con sus menstruaciones a los 8 años de edad,
el Colegio Estadounidense de Obstetricia y Ginecología (ACOG, por sus siglas
en inglés) indica que el 90% de ellas tiene su primera menstruación, o menarquia,
entre los 12 y los 14 años. Para las jovencitas que padecen de trastornos
hemorrágicos, sus flujos menstruales pueden ser especialmente abundantes,
prolongados y dolorosos, lo que se conoce como menorragia. Típicamente, sus
menstruaciones duran más de siete días, que es el límite superior de lo que se
considera normal.
La inminente llegada de la menstruación puede producir nerviosismo en las
jóvenes. Las niñas y las adolescentes se preocupan de que la sangre se vaya a
pasar y les manche la ropa, comenta Danna Merritt, MSW, LMSW, una
trabajadora social del centro para el tratamiento de la hemofilia en el Hospital
Infantil de Michigan en Detroit. Por eso es importante preparar a las jóvenes con
tiempo. Ellas necesitan saber qué esperar y qué hacer si más adelante tienen un
flujo abundante, cólicos intensos u otras complicaciones.
M
Cómo empezar la conversación
Las madres deberían conversar frecuentemente con sus hijas acerca de la
pubertad y de los cambios corporales. Deberían evitar una sola conversación
prolongada, dice la Dra. Lisa Perriera, MPH, profesora agregada de obstetricia
y ginecología en University Hospitals en Cleveland, quien trata a jóvenes con
trastornos hemorrágicos. Los expertos sugieren hablar con su hija cuando tenga
entre 8 y 12 años de edad. La conversación inicial debe tratar sobre lo qué es la
menstruación, qué ocurre en el organismo, qué productos usar y cómo manejar
los cólicos, los cambios de humor y la fatiga. Lo más posible es que ya su hija
haya hablado anteriormente sobre esto en la clase de educación física o de salud
en la escuela. Algunos fabricantes de tampones y toallas sanitarias venden kits
para la primera menstruación para que las madres los usen cuando tengan “esa
conversación” con sus hijas.
Los padres deben contestar todas las preguntas con sinceridad, dice la Dra.
Perriera. Se debe animar a las jovencitas a hablar en privado con su ginecólogo
o médico de cabecera ya que pueden tener timidez o sentir vergüenza de hablar
sobre sus cuerpos en frente de sus padres. La Sra. Merritt indica que los
médicos, enfermeras y trabajadores sociales en su centro para el tratamiento de
la hemofilia también pueden hablar con las niñas sobre la menstruación y
orientar a los padres sobre lo que tienen que decir en casa.
Complicaciones y tratamientos
A diferencia de las hermanas mayores de la Sra. Buurma, quienes padecen
del tipo I de la VWD (el tipo más frecuente y leve), ella padece el tipo más
SUMMER 2013 • LIFELINE
7
NHF News • August 2013
grave. “Todos sabíamos que iba a ser un problema cuando yo comenzara a
menstruar”, dice ella. “Sangré durante casi dos meses seguidos y estuve
hospitalizada por alrededor de tres semanas y medias”.
Es importante que una joven que padece de un trastorno hemorrágico sepa
lo que es normal y lo que no durante sus primeros ciclos menstruales. “No es
normal empapar completamente una toalla sanitaria en una hora”, dice la Dra.
Perriera. Entre las otras complicaciones que requieren atención médica se
encuentran los cólicos intensos que interfieren con la escuela o las actividades,
y el sangrado entre una menstruación y otra.
Para disminuir el sangrado abundante, es posible que se necesite tratamiento
hormonal. Pero los medicamentos para tratamiento hormonal también se indican
como métodos de anticoncepción para las mujeres sexualmente activas, lo que
algunas veces puede representar una inquietud de tipo religioso, cultural o de otro
tipo para los padres. Cuando era más joven, los médicos de la Sra. Buurma le
recetaron “la píldora” como medicamento hormonal continuo para aliviar sus
síntomas. “Nosotros somos católicos. En nuestra religión, el uso de
anticonceptivos no es aceptable”, explica ella. Pero sus padres le aclararon que
tomar la píldora no estaba en contra de la doctrina de la iglesia ya que ella no la
estaba usando para evitar un embarazo. Otras opciones hormonales incluyen
ciertos dispositivos intrauterinos (DIU) o los implantes hormonales.
Experimental Hemophilia
B Gene Therapy Tricks
the Immune System
I
n July, researchers at The Children’s Hospital of Philadelphia (CHOP)
published the results of a study in which they used bioengineered decoys as
a ruse to foil the immune system. This allowed for the successful delivery of gene
therapy in mice with hemophilia B, or FIX deficiency. The lead author of the
study was Katherine High, MD, director of CHOP’s Center for Cellular and
Molecular Therapeutics.
High and her team used capsids, the protein shell that encapsulates a virus, as
the decoy. They also used adeno-associated viruses (AAVs) as delivery vehicles, or
vectors, to carry the actual genetic material that triggers the production of factor
IX. AAVs have recently become the “vector of choice” for researchers because
they can deliver the genetic material into living cells to sustain therapeutic effect
without causing disease. In addition, they can be targeted to liver cells, which
manufacture FIX.
However, one drawback in using injected AAVs is the body’s natural immune
response. In some cases, antibodies are released, neutralizing the AAVs. To
remedy that, investigators injected both empty capsids and genetically altered
AAVs into the mice. The empty capsids effectively drew antibodies to them and
away from the gene therapy.
“This decoy strategy could be individualized to patients and could greatly
expand the population of patients who may benefit from gene therapy,” said High.
“Right now, 30 to 60 percent of adult patients develop antibodies that block the
ability of an intravenously infused vector to reach the target cells in the liver. This
approach holds the promise of overcoming this roadblock--pre-existing
antibodies--and allowing successful intravenous gene therapy in virtually all adult
patients.”
This technique also proved effective in follow-up studies performed in rhesus
macaque monkeys. The therapy initiated higher levels of factor IX production, with no
adverse events reported. Although additional studies, including clinical trials in
humans, will be necessary before such a therapy becomes a reality, the authors are
encouraged by these preliminary findings.
“Our results, which held up over a range of doses, suggest that in clinical
studies, it will be feasible to adjust the ratio of empty capsids to gene vector doses,
depending on an individual's pre-existing level of neutralizing antibodies. That
means we could personalize gene therapy to make it more efficient for each
patient,” concluded High. “This work should make it possible to bring effective
gene therapy to most adults with severe hemophilia B. Each patient would receive
a personalized final formulation that contains just the right amount of empty
capsid to neutralize any pre-existing antibody, and allow the gene-expressing
vector to reach the liver.”
The study, “Overcoming Pre-existing Humoral Immunity to AAV Using
Capsid Decoys,” was published in the July 2013 online issue of Science
Translational Medicine.
Source: ScienceDaily, July 17, 2013
8
SUMMER 2013 • LIFELINE
“Desearía que los tratamientos hormonales no se comercializaran como
anticonceptivos. Son para controlar la menstruación. Son para controlar el
sangrado”, dice la Dra. Perriera. Sin embargo, ella reconoce que algunos padres
tienen ciertas preocupaciones y pueden solicitar que se usen primero
tratamientos no hormonales. Ahí es cuando los hematólogos pueden ayudar.
Puede ser una buena idea visitar al hematólogo para considerar los
tratamientos, ya que cada niña que padece de un trastorno de sangrado presenta
un grado diferente de intensidad con el sangrado menstrual. “Por ejemplo, a las
mujeres con la enfermedad de von Willebrand les pueden recetar una hormona
llamada DDAVP que viene en forma de un spray nasal para usar durante las
menstruaciones para disminuir el flujo del sangrado”, dice el Dr. Sanjay Ahuja,
MS, un pediatra hematólogo y oncólogo que trabaja con la Dra. Perriera. “Es
posible que las mujeres con la enfermedad de von Willebrand de tipo III
necesiten remplazo del factor durante las menstruaciones”. Además, en las
pacientes con sangrado fuerte, los médicos también combinarán medicamentos
hormonales y no hormonales, expresa él.
Ninguna jovencita debería sufrir en silencio. “Creo que los mensajes de
‘vas a estar bien’ y ‘solucionaremos cualquier problema juntas’ son muy
importantes”, agrega la Sra. Merritt.
Terapia génica experimental
para la hemofilia B “engaña
al sistema inmunitario
E
n julio, los investigadores del The Children’s Hospital of Philadelphia (CHOP)
publicaron los resultados de un estudio en el que usaron señuelos creados
mediante bioingeniería como una treta para desconcertar al sistema inmunitario. Esto permitió
la distribución exitosa de la terapia génica en ratones con hemofilia B o con deficiencia del
factor IX. El autor principal del estudio fue la Dra. Katherine High, directora del Centro
para Tratamientos Celulares y Moleculares del CHOP.
La Dra. High y su equipo utilizaron cápsides (la envoltura proteica que encapsula a un
virus) como señuelo. También usaron virus adenoasociados (AAV, por sus siglas en inglés)
como vehículos transportadores, o vectores, para llevar el material genético que desencadena
la producción de factor IX. Los AAV se han convertido recientemente en el “vector de
preferencia” para los investigadores ya que estos pueden llevar el material genético dentro de
las células vivas para mantener el efecto terapéutico sin producir enfermedad. Además, es
posible dirigir a estos virus para que tengan como objetivo las células hepáticas, las cuales
producen el factor IX.
No obstante, un inconveniente al usar AAV inyectados es la respuesta inmunitaria
natural del organismo. En algunos casos, se liberan anticuerpos que neutralizan los AAV.
Para remediar esto, los investigadores les inyectaron a los ratones tanto cápsides vacías como
AAV alterados genéticamente. Las cápsides vacías atrajeron hacia ellas, de manera eficaz, los
anticuerpos, alejándolos así de la terapia génica.
“Esta estrategia con señuelos podría individualizarse para cada paciente y podría
aumentar en gran manera la población de pacientes que pueden beneficiarse de la terapia
génica”, dijo la Dra. High. “En la actualidad, del 30% al 60% de los pacientes adultos
producen anticuerpos que bloquean la capacidad de un vector que se infunde por vía
intravenosa de alcanzar las células que se tienen como objetivo en el hígado. Este enfoque
promete superar este obstáculo (los anticuerpos preexistentes) y permitir una terapia génica
intravenosa exitosa en prácticamente todos los pacientes adultos”.
Esta técnica también demostró ser eficaz en los estudios de seguimiento realizados en
monos macacos de la India. La terapia puso en marcha un aumento en la producción del factor
IX, sin que se comunicaran eventos adversos. Aunque serán necesarios otros estudios, entre
ellos ensayos clínicos en seres humanos, antes de que dicha terapia sea una realidad, los autores
se encuentran alentados por estos hallazgos preliminares.
“Nuestros resultados, los que se mantuvieron con diferentes dosis, permiten suponer que
en los estudios clínicos será factible ajustar la proporción de cápsides vacías a dosis de los
vectores de genes, en función de la concentración preexistente de anticuerpos neutralizantes
en una persona. Eso significa que podríamos personalizar la terapia génica para hacerla más
eficaz para cada paciente”, concluyó la Dra. High. “Este trabajo debería hacer posible que
se pueda ofrecer una terapia génica eficaz a la mayoría de los adultos que padecen de
hemofilia B grave. Cada paciente recibiría una formulación final personalizada que contenga
solo la cantidad apropiada de cápsides vacías para neutralizar cualquier anticuerpo
preexistente, y que permita que el vector que expresa el gen llegue al hígado”.
El estudio, “Overcoming Pre-existing Humoral Immunity to AAV Using Capsid Decoys”
(Vencer la inmunidad humoral preexistente contra los AAV mediante el uso de cápsides
“señuelos”), se publicó en la edición en línea de Science Translational Medicine de julio de 2013.
Fuente: ScienceDaily, 17 de julio de 2013
SUMMER 2013 • LIFELINE
9
FHA ResourceCenter
MISSION STATEMENT
CONTACT NUMBERS
The Florida Hemophilia Association, Inc (FHA) is a Not-for-Profit organization that is
dedicated to enhancing the quality of life in the bleeding disorders community by creating
programs and services that provide education, emotional support and advocacy. We are
contributing toward research to ultimately find a cure.
HEMOPHILIA CLINICS
All Children’s Outpatient
Care Clinic
Pediatric Cancer and
Blood Disorders Center
601 5th Street South,
Third Floor
St. Petersburg, FL 33701
Phone: (727) 767-4931
www.allkids.org
Center for Children’s
Cancer and Blood
Disorders at Arnold
Palmer Hospital
for Children
92 West Miller St., MP 318
Orlando, FL 32806
Phone: (321) 841-8588
www.orlandohealth.com
Joe DiMaggio
Children’s Hospital
Pediatric Specialty Center
1150 N. 35th Ave.
Suite 520
Hollywood, FL 33021
Phone: (954) 986-2234
Lee Memorial Hospital
Department of Pediatric
Hematology & Oncology
9981 So. Healthpark Dr.
Suite 156
Ft. Myers, FL 33908
Phone: (239) 332-1111
Nemours Children’s
Clinic, Jacksonville
Department of Pediatric
Hematology/Oncology
807 Children’s Way
Jacksonville, FL 32207
Phone: (904) 697-3789
(904) 697-3600
www.nemours.com
University of Florida
Department of Medicine
Adult Hemophilia
P.O. Box 100277
Gainesville, FL 32610
Phone: (352) 265-0725
University of Florida
Pediatric Hematology/
Oncology
P.O. Box 100296
Gainesville, FL 32610
Phone: (352) 273-9120
www.peds.ufl.edu/
divisions/hemonc/
Nemours Children’s
Clinic, Orlando
Department of Pediatric
Hematology/Oncology
13535 Nemours Parkway
Orlando, FL 32827
Phone: (904) 697-3789,
(904) 697-3600
Sacred Heart Pediatric
Hemophilia Program,
Pensacola
Phone: (850) 416-7712
St. Joseph’s Children’s
Hospital
Pediatric Hematology
Oncology Out-patient
Clinic
3001 W. Dr. Martin
Luther King Jr. Blvd.
Tampa, FL 33607
Phone: (813) 554-8294
or (813) 321-6820
The University of Miami
Hemophilia Treatment
Center
Pediatric Clinic Location
Alex’s Place at Sylvester
1475 NW 12th Avenue
Suite C103
Miami, FL 33136
Phone: (305) 689-7210
Adult Clinic Location
ACCW-3A
1611 NW 12th Avenue
Miami, FL 33136
Phone: (305) 243-6925
www.htcextras.org
USF Adult Hemophilia
Center
Department of Internal
Medicine
12901 Bruce B. Downs
Boulevard, MDC 19
Tampa, FL 33612
Phone: (813) 974-1325
Do the 5
1 Get an annual comprehensive check-up at a hemophilia treatment
center. 2 Get vaccinated – Hepatitis A and B are preventable.
3 Treat bleeds early and adequately. 4 Exercise to protect your
joints. 5 Get tested regularly for blood-borne infections
Florida Hemophilia
Association Office
(888) 880-8330
President
Jon Salk
Executive Director
Debbi Adamkin
(305) 235-0717
dadamkin@floridahemophilia.org
National Hemophilia
Foundation
(800) 424-2634
Hemophilia Federation
(800) 230-9797
LA Kelley
Communications, Inc.
Free resource material on
Hemophilia
(978) 352-7657
ABOUT THIS PUBLICATION
LIFE LINE is the official
Newsletter of the Florida
Hemophilia Association.
It is produced quarterly and
distributed free of charge to
requesting members of the
bleeding disorder community.
Florida Hemophilia
Association Headquarters
915 Middle River Drive, Suite 421
Ft. Lauderdale, FL 33304
Toll Free: (888) 880-8330
www.floridahemophilia.org
Newsletter Committee:
Debbi Adamkin, Maria Rubin,
Denise Schacher, Linda Thomas
Design and Production:
Group M, Advertising & Design
305-235-2538
SUMMER 2013 • LIFELINE
11
Florida Hemophilia Association
(Formerly known as Florida Chapter, NHF)
915 Middle River Drive, Suite 421
Ft. Lauderdale, FL 33304
SAVE THE DATE • SAVE THE DATE • SAVE THE DATE • SAVE THE DATE • SAVE THE DATE • SAVE THE DATE
Monday, December 9, 2013
Lago Mar Country Club
500 NW 127th Avenue, Plantation, FL 33325
Golf Tournament
Consider joining a committee! We need help with:
Auction and Raffle Items, Golfers and Volunteers.
Please email info@floridahemophilia.org
if you interested in helping us out!