the official newsletter of the florida hemophilia...

The Official Newsletter of the Florida Hemophilia Association

Executively SpeakingPage 2

Community CornerPage 6 - 8

NHF News6

FHA Resource CenterPage 19

FALL 2014

INSIDE

Advocacy Training DinnerJanuary 14

Educational ProgramFort LauderdaleFebruary 8

NHF Washington DaysFebruary 25-27

HFA Symposium - St. LouisMarch 26-28

5th Annual Walk in the JungleMay 2

“SAVE THE DATE”

n Monday, November 17, 2014, the Florida Hemophilia Association (FHA)hosted its 25th Annual Swing for the Kids Golf Tournament at Lago Mar CountryClub in Plantation. After a rainy and overcast morning, the sun came out and it was abeautiful day for golf ! At the start of the day, golfers checked in and ate lunch,generously donated by Corporate Caterers, in the clubhouse. Then it was off to thedriving range where golf pro Perry Parker conducted a free video swing analysis andmini golf clinic. After a welcome and announcements from Executive Director DebbiAdamkin, the golfers hit the course for the afternoon. There were a number ofcontests that the golfers could participate in during the day. On Hole #14, for a smalldonation golfers got the chance to challenge golf pro Perry Parker in “Beat the Pro”.The contest raised an additional $660 for the tournament. Thank you to Perry and theCSL Behring team for putting the contest together!

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Continue...

ExecutivelySpeaking

It’s been incredibly busy for us here at FHA andwith the year winding down, I want to take aminute to thank all of our supporters, volunteersand donors that were instrumental in helping usachieve our goals and for providing theirassistance to make our programs, events and

various outings a success. With that said, we’ve already got a headstart on 2015 as we have several new things up our sleeve. Pleasestay connected by checking out the website and our Facebook pagefor up-to-date information.

One of our main focuses in 2014 was the launch of the BleedingDisorder Coalition of Florida (BDCF). The coalition hosted its firstState Advocacy Day on March 20, 2014 at our State Capitol, whichproved to be very successful with over 20 scheduled visits withFlorida legislators and staff. BDCF and its volunteers will continue todevelop an important dialogue with the key Florida decision makers.If you have a passion for advocacy and are interested in gettinginvolved, please contact us at [email protected]

Finally, we are always looking for volunteers to serve on the followingcommittees: 5th Annual Walk in the Jungle and the 32nd AnnualFamily Education Symposium. If you are interested or have someideas that you’d like to share, please contact me directly [email protected]

I look forward to seeing you in 2015!

Debbi AdamkinFHA Executive Director

MensajeEjecutivo

Este año ha sido increíblemente movido para nosotros aquí en la FHA ycomo ya va a finalizarse, quiero tomar un momento para agradecerles atodos nuestros partidarios, voluntarios y donantes que fueroninstrumentales para ayudarnos a alcanzar nuestros objetivos y porproporcionar su asistencia y hacer que nuestros programas, eventos ydiversas salidas fueran un éxito. Dicho esto, ya tenemos una ventaja en2015 ya que tenemos varias cosas nuevas planeadas. Por favor,manténganse informados de nuestra página web y nuestra página deFacebook para obtener la información mas actualizada.

En el 2014 el enfoque principal fue el lanzamiento de la Coalición deEnfermedades Sanguíneas de la Florida (Bleeding Disorder Coalition ofFlorida, BDCF). La coalición organizó su primera Día de Abogacía Estatal el20 de marzo de 2014 en nuestro Capitolio de la Florida el cual resultó serun gran éxito con más de 20 citas con los legisladores y el personal de laFlorida. BDCF y sus voluntarios continuarán desarrollando un diálogoimportante con personas claves al hacer decisiones en la Florida. Si ustedes apasionado por la abogacía y está interesado en participar, por favorpóngase en contacto con nosotros en [email protected]

Por último, siempre estamos buscando voluntarios para servir en lossiguientes comité: 5ta Caminata Anual en la Selva y el Simposio 32 Anualde Educación Familiar. Si usted está interesado o tiene algunas ideas quele gustaría compartir, por favor póngase en contacto conmigodirectamente en [email protected]

Espero verlos en el 2015!

Debbi AdamkinDirectora Ejecutiva de FHA

2 FALL 2014 • LIFELINE

Maylen De La Fuente

In Memory of ...

In November, we unfortunately lost a member of our“family”. Maylen de la Fuente was an active memberof the Florida bleeding disorders community. She wasespecially involved in the Walk in the Jungle, when sheand her beloved husband Johnny lead Team Matrix toraising over $7,000 in the past two years. Maylen andher beautiful smile will be missed.

3FALL 2014 • LIFELINE

Congratulations to all the winners! (Continued...)

Left to right: Jim Sherratt, Joe D'Amico, Greg Rapplean, Gerry Rapplean

Left to right: Ron Hoffman, Greg Thomas, Ricky Thomas, Marty Martinez

Left to right:Pablo Mejia, Joey Robinson, Robert Fernandez, Chuck Mercer

2nd PLACE and Crystal Cup Sponsor

1st PLACE

3rd PLACE

Continue...

Following an exciting round ofgolf, everyone gathered in the clubhousefor the dinner program, whereUniversity of Miami and FloridaPanthers announcer Jay Rokeach servedas the emcee and auctioneer for theevening. During dinner, everyone watched apresentation and video that showed the daily lifeof a person with hemophilia. A special thank you goes out to the familieswho contributed photos and videos to the presentation. It was awonderful way to show why we do what we do at FHA.

Thank you to everyone who helped to make this year’s golftournament another success! Special thanks to our golf committee and allthe volunteers who helped us on the day of the tournament. It truly takesa team to put on an event ofthis caliber and it wouldn’thave happened withoutyour commitment anddedication. Finally, we’dlike to thank ourparticipants and oursponsors for their generousdonations. FHA is sograteful for all that you doto support ourorganization!


(Continued...)

Thank you to everyone who helped to make

this year’s golf tournamentanother success!

TITLE SPONSORBaxter International Inc.

MAJOR SPONSORSBayer Healthcare PharmaceuticalsCSL Behring

Corporate SponsorsNovo Nordisk OctapharmaPfizer

Crystal Cup SponsorEmergent BioSolutionsWalgreen’s Infusion ServicesThe Wingmen Foundation

Par SponsorCommunity Bank of BrowardZuni Transportation

Hole SponsorsAlma FoundationBiogen IdecBioRxGrifolsMasters Title, Inc.Morgan-Weiss LLCThe Cesta FamilyThe Ziccardi Family

Hole In One SponsorMassey Yardley

Lunch SponsorCorporate Caterers

In-Kind SponsorsCostcoLago Mar Country ClubPerry Ellis International

Embroidery SponsorLevy Advertising

THANK YOUSPONSORS!

Debbi Adamkin — Event ChairJon Salk — Committee ChairElise Boston, Sheila Brooks, Chad

Brown, Yamida Garcia, Lily Gomez,

Jim Harrison, Justin Lindhorst,

Kathy Naughton, Chris Ricci

Auction/Raffle Sponsors and Other Donations

Thank You To Our Golf Committee

Alf’s Golf ShopBest BuyBoomersBradford PortraitsCantina LaredoCheesecake FactoryDr. Tory R. Lindh Dolphins PlusDowntown BicyclesEdwin WattsFlamingo Beach Resort & SpaFlorida PanthersFunky Buddha BreweryGallery One Fort LauderdaleGG’s Waterfront Bar and GrillGolf GalaxyHome DepotHumberto CapeloHyatt Regency JacksonvilleInternational Links MiamiItalio KitchenJ. Alexander’s

Jaguar Hospitality GroupJay and Joan EichelJohanna E. La RosaKerry’s By The SeaLatif’s JewelryLifestream Wellness SpaLilac and LiliesMaggiano’s Little Italy Mama Mia Italian RistoranteMarriott Boca RatonMarriott Harbor Beach Resort &

SpaMiami SeaquariumMr. M’s SubsNovecentoOceans 234Ocean Properties Ltd. Olive GardenP.F. Chang’sPadrino’s Cuban CuisinePhllyis Lazarow SalonPlantation Preserve Golf Course

Presidente SupermarketRamada Downtown HollywoodRed BullRed Door SpaRenaissance Tampa International

PlazaROK:BRGRSpecialty Restaurants Corporation Tel Aviv TexacoThe Club at Emerald HillsThe Fresh MarketThe Tower ClubTrattoria RomanaTropical Sailing Catamaran

ChartersTruluck’s Fort LauderdaleUmberto’s Pizzeria

Barbie Arrebola, Valerie Barnhart, Jesus Benitez-Soto, Mary Bosco, Jeanette Cesta, Ken Crandall, Jan Espinosa, Robin Potter, Lily Gomez, Myriam Lagomasino, Ginny Leibick, Lisa Leventhal, Ivan Sada, Justin Lindhorst, Barbara Matias, Laura Robbins,Heather Stewart, Jeanine Schmidt, Linda Thomas

Volunteers


Guy’s DAY OUT

n Sunday, November 16,

men, dads and their sons

gathered at Lago Mar Country

Club for an awesome

afternoon! After introductions,

the guys went out to the

driving range and putting

green for some a mini golf

clinic and some friendly

competition. When they came

back to the clubhouse, the guys

broke out into groups for rap

sessions. Thank you to Perry

Parker, Jeff Salantai and Ivan

Sada for facilitating the

program and CSL Behring for

sponsoring the program.

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Women’sRETREAT

n Saturday, November 8, we hosted a program for Women with Bleeding Disorders at the Sheraton Ft Lauderdale. The featuredspeaker was Dr. Diane Dimon of Matters of the Mind. Immediately after the introductions and an icebreaker, Diane had the

group of ladies grab a beach towel and then escorted them all out to the beach where they all gathered in a circle. While basking in thebeautiful Florida sunshine, they all participated in Diane’s very interactive presentation “Sharing and Exploring the Power ofGratitude”. Afterwards the ladies went back inside and had a delicious brunch while listening to the second half of Diane’s presentation“Mediation for Anxiety and Stress Reduction”. Everyone took away something from her talks and maybe even learned some relaxationexercises to deal with everyday stress.

Special Thanks to Diane for providing the incredible presentations and CSL Behring for sponsoring the program.

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Submitted byEdward

First of all, thankyou for choosing meto represent the Florida Hemophilia Association in Phoenix.

I had a great time just to let you know! I learned a lotabout playing golf from the best Perry Parker, and now I loveit. Of course, I made some friends. At first I was kind of shybecause I didn’t know who everybody was. Then, I started totalk to them and wasn’t that shy anymore because I wasgetting to know them. My best NEW friend was Percy. Hewas so much fun! We always played in the game room and Ihave got to say, he does excellent at playing golf. He wasone of the best golfers that I've ever seen. I also had anotherfriend, Omar. I have known him for 3 years now! I have seenhim at other activities like the Symposium. Also, thevolunteers were excellent with helping us get better. JanellBadami was my caddy on Saturday and she was great.

P.S I ABSOLUTEY LOVE GOLF! IT’S THE MOST EXCITINGSPORT EVER.

Submitted by Aron, Age 9

I would first like to say thank you for picking me to play

golf at JNC. I really liked the golfing it was cool. I met

someone there that played with Tiger Woods. I hope I can go

again next year.

It's really neat that we have this opportunity.

Thank you!

JUNIOR NATIONALCHAMPIONSHIP

Save the Date

Lace Up Your Sneakers for the

5th ANNUAL WALK IN THE JUNGLESaturday, May 2, 2015 Jungle Island

It’s time to start thinking about forming your teamand some creative ways to fundraise for the Walk!

The 2015 website will be up and running shortly!

We are excited to announce that we are once againaccepting applications for the

Daniel L. Carlin Memorial Scholarship!

SCHOLARSHIP FUNDS

FHA will be awarding a total of three (3) $1,000scholarships for the 2015-2016 academic year.

Two (2) of these scholarships are designated forpeople who are diagnosed with a bleeding disorder.

One (1) of these scholarships is designated for aparent of a child diagnosed with a bleeding disorder.Awards will be dispersed directly to the institution.

For more information, please contact Debbi at

[email protected]

for the application packet.

My NHF Annual Meeting Experience By: Sara Workman

n September I had the fortunate experience of attending the NationalHemophilia Foundation’s (NHF) Annual Meeting in Washington D.C.As the chair of the Bleeding Disorder Coalition of Florida, I am not

only passionate about learning effective ways to advocate on behalf of thecommunity, but also about meeting others in the community and hearingabout their struggles and successes. Attending an annual meeting allows notonly adult patients and caregivers to learn and make connections, but alsoprovides a way for children - whether they have a bleeding disorder or havea family member affected by a bleeding disorder - to learn and makeconnections with others their age in the community.

The annual meeting is three days long and offers multiple sessions to attend.It is such a wonderful opportunity for patients and families to learn how toeffectively advocate for themselves and for their community. Whether youchoose to attend a session on how to communicate with your healthcareproviders, participate in one of their multiple roundtable discussions, orlearn how to address your insurance claims and the importance of advocacy,you are sure to leave the meeting feeling empowered and educated. Witheach session, you are made aware of the tools available to ensure that youand your family are able to deal with the multiple challenges that living witha bleeding disorder - some of which we aren’t always aware of.

In addition to the annual meeting this year, the Food and DrugAdministration (FDA) held a public meeting on Patient-Focused Drug

Development for Hemophilia A, Hemophilia B, von Willebrand Disease,and other heritable bleeding disorders such as other factor deficiencies(including I, V, VII, X, XI), and platelet disorders that I was able to attend.The focus of this meeting was to get input on symptoms and other impactsof life with a bleeding disorder, as well as patient perspectives on currentand future therapies to treat these disorders. There were several brave menand women that sat on two panels to address the FDA about their lives andtheir hopes for the future, and I am honored to have been able to watchand listen to them educate government officials on what having a bleedingdisorder really means.

If you haven’t had the chance to attend an NHF Annual Meeting, I wouldencourage you to consider attending. NHF offers scholarships for first-timeattendees and announced that they are increasing the amount of totalscholarships to $100,000/year, allowing more families to attend. I wouldencourage you to get your applications in early if you are interested inapplying, as many families are hoping to go. Please check the NHF websitefor more information and details regarding deadlines for submission.

If you are interested in becoming involved with the Bleeding DisorderCoalition of Florida, please contact Debbi Adamkin [email protected] or 305.235.0717.

I


Feast in the Park aka Holiday DuoS U N D A Y , N O V E M B E R 2 3 A T M A R K H A M P A R K

THANK YOUSPONSORS!

NHF News • FALL 2014

Gene Therapy Study Still Succeeding Three Years Later

t has now been three years since a group of patients with severehemophilia B, or factor IX (FIX) deficiency, in London received a single

dose of gene therapy as part of a new clinical trial. Early results of the trialwere positive as these patients began to generate FIX levels ranging from1%-6%. Prior to the study, they produced little to none of the crucialclotting factor protein.

This seemingly modest boost in FIX “expression” is important. The increasein FIX essentially transforms a patient symptomatically, from severe to mild,with the end result a significant, even dramatic, reduction in bleeds. Resultsdescribed in a new article indicate that the initial breakthrough results havebeen sustained during the three years since the study began in 2011.

The report, “Long-Term Safety and Efficacy of Factor IX Gene Therapy inHemophilia B,” was published in the November 20, 2014, issue of The NewEngland Journal of Medicine. The lead author of the update was AndrewDavidoff, MD, St. Jude Children’s Research Hospital in Memphis, TN.Davidoff has collaborated for more than a decade with a strong team ofresearchers, including coauthor Amit Nathwani, MD, PhD, at the UniversityCollege London. “I believe that, scientifically, this is ready for prime time,”said Davidoff.

The gene therapy trial employed an adeno-associated virus serotype 8(AAV8), a small virus that does not cause disease and produces mildimmune responses, as a vector (delivery vehicles) to introduce a functioningFIX gene into the liver cells of subjects with severe hemophilia B. The goalof the trial was to trigger viable, long-term FIX protein productionthrough a single administration of the therapy.

Overall, 10 subjects with severe hemophilia B participated in the study, sixof whom received high doses of AAV8 and reached average FIX levels of5.1%. According to investigators, this “resulted in a reduction of morethan 90% in both bleeding episodes and the use of prophylactic factor IXconcentrate.” Also, no toxic effects were reported.

“I think it’s going to have a big impact. The study showed both safety andefficacy, and the side effects were minimal,” said Timothy Nichols, MD,who heads the Francis Owen Blood Research Laboratory at the Universityof North Carolina at Chapel Hill. He was not involved in the study. “Thisis a single shot of medicine given to patients who are treating themselvestwo or three times a week,” he told Reuters Health over the phone.“Suddenly, they don't have to take the medicine anymore.”

Source: Reuters, November 19, 2014

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Estudio de genoterapia continúa con éxito tres años más tarde

a han pasado tres años desde que un grupo de pacientes conhemofilia B grave, o deficiencia del factor IX (FIX), recibieron una

sola dosis de genoterapia como parte de un nuevo ensayo clínico en Londres.Los primeros resultados del ensayo fueron positivos ya que estos pacientescomenzaron a producir concentraciones del FIX que oscilaban entre un 1%y un 6%. Antes del estudio, estas personas producían muy poco o nada deeste importantísimo factor de la coagulación. Este aparentemente modestoincremento en la “expresión” del FIX es importante. El aumento en lacantidad del FIX básicamente transforma los síntomas de un paciente degraves a leves, y el resultado final de esto es una reducción importante, eincluso impresionante, de las hemorragias. Los resultados que se describenen este nuevo artículo indican que las extraordinarias respuestas iniciales sehan mantenido durante los tres años transcurridos desde que inició elestudio en el 2011.

El informe, titulado “Long-Term Safety and Efficacy of Factor IX GeneTherapy in Hemophilia B” (Seguridad y eficacia a largo plazo de lagenoterapia para el factor IX en la hemofilia B), se publicó en la edición del20 de noviembre de 2014 de The New England Journal of Medicine. Elautor principal de la actualización fue el Dr. Andrew Davidoff del St. JudeChildren’s Research Hospital en Memphis, TN. El Dr. Davidoff hacolaborado durante más de una década con un sólido equipo deinvestigadores, entre los que se encuentra el coautor Dr. Amit Nathwani,PhD, de University College London. “Creo que, a nivel científico, esto estálisto para salir a la luz”, expresó Davidoff.

El ensayo de genoterapia utilizó un virus adenoasociado de serotipo 8(AAV8) —un pequeño virus que no produce ninguna enfermedad yprovoca respuestas inmunitarias leves— como vector (vehículotransportador) con el fin de introducir un gen activo del FIX en loshepatocitos de personas con hemofilia B grave. El objetivo del ensayo fueestimular una producción viable y a largo plazo de la proteína del FIX pormedio de una sola administración de la genoterapia.

En total, en el estudio participaron 10 pacientes con hemofilia B grave, 6de los cuales recibieron altas dosis del AAV8 y lograron concentracionespromedio del FIX del 5.1%. Según los investigadores, esto “dio lugar a unareducción de más del 90% tanto en los episodios hemorrágicos como en eluso del concentrado del factor profiláctico”. Además, no se informaronefectos adversos.

“Creo que esto va a tener un gran impacto. El estudio demostró tanto laseguridad como la eficacia, y los efectos secundarios fueron mínimos”, dijoel Dr. Timothy Nichols, quien dirige el Francis Owen Blood ResearchLaboratory en la Universidad de Carolina del Norte en Chapel Hill. El Dr.Nichols no estuvo involucrado en el estudio. “Esto es una sola inyección demedicamento que se aplica a pacientes que normalmente se inyectan doso tres veces a la semana”, le explicó a Reuters Health por vía telefónica. “Yde pronto, ya no tienen que seguir utilizando más el medicamento”.

Fuente: Reuters, 19 de noviembre de 2014

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HEMOPHILIA CLINICS

All Children’s Outpatient

Care Clinic

Pediatric Cancer and Blood Disorders Center601 5th Street South, Third FloorSt. Petersburg, FL 33701Phone: (727) 767-4931www.allkids.org

Center for Children’s

Cancer and Blood

Disorders at Arnold

Palmer Hospital

for Children

92 West Miller St., MP 318Orlando, FL 32806Phone: (321) 841-8588www.orlandohealth.com

Joe DiMaggio

Children’s Hospital

Pediatric Specialty Center1150 N. 35th Ave.Suite 520Hollywood, FL 33021Phone: (954) 986-2234

Lee Memorial Hospital

Department of Pediatric Hematology & Oncology9981 So. Healthpark Dr. Suite 156Ft. Myers, FL 33908Phone: (239) 332-1111

Nemours Children’s

Clinic, Jacksonville

Department of Pediatric Hematology/Oncology807 Children’s Way Jacksonville, FL 32207Phone: (904) 697-3789(904) 697-3600 www.nemours.com

University of Florida

Department of Medicine Adult HemophiliaP.O. Box 100277 Gainesville, FL 32610Phone: (352) 265-0725

University of Florida

Pediatric Hematology/OncologyP.O. Box 100296Gainesville, FL 32610Phone: (352) 273-9120www.peds.ufl.edu/divisions/hemonc/

Nemours Children’s

Clinic, Orlando

Department of Pediatric Hematology/Oncology13535 Nemours ParkwayOrlando, FL 32827Phone: (904) 697-3789, (904) 697-3600

P

Sacred Heart Pediatric

Hemophilia Program,

ensacola

Phone: (850) 416-7712

St. Joseph’s Children’s

Hospital

Pediatric HematologyOncology Out-patient Clinic3001 W. Dr. Martin Luther King Jr. Blvd. Tampa, FL 33607Phone: (813) 554-8294 or (813) 321-6820

The University of Miami

Hemophilia Treatment

Center

Pediatric Clinic LocationAlex’s Place at Sylvester1475 NW 12th AvenueSuite C103Miami, FL 33136Phone: (305) 689-7210

Adult Clinic LocationACCW-3A1611 NW 12th AvenueMiami, FL 33136Phone: (305) 243-6925www.htcextras.org

USF Adult Hemophilia

Center

Department of Internal Medicine12901 Bruce B. Downs Boulevard, MDC 19Tampa, FL 33612Phone: (813) 974-1325

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 421Ft. Lauderdale, FL 33304Toll Free: (888) 880-8330www.floridahemophilia.org

Newsletter Committee:

Debbi Adamkin, Maria Rubin,

Translation:

Roxana Ambrosini, Roxanna Delgado

Denise Schacher, Linda Thomas

Design and Production:

Group M, Advertising & Design305-235-2538

Florida Hemophilia Association Office(888) 880-8330

PresidentJon Salk

Executive DirectorDebbi Adamkin(305) [email protected]

National Hemophilia Foundation(800) 424-2634

Hemophilia Federation

(800) 230-9797

LA KelleyCommunications, Inc.Free resource material on Hemophilia(978) 352-7657

CONTACT NUMBERSMISSION STATEMENT

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


FHA ResourceCenterThe Florida Hemophilia Association, Inc (FHA) is a Not-for-Profit organization that isdedicated to enhancing the quality of life in the bleeding disorders community by creatingprograms and services that provide education, emotional support and advocacy. We arecontributing toward research to ultimately find a cure.

Florida Hemophilia Association(Formerly known as Florida Chapter, NHF)915 Middle River Drive, Suite 421Ft. Lauderdale, FL 33304

If you would like to contribute to Community Corner, please submit your stories to

Debbi at [email protected]

www.facebook.com/FloridaHemophilia

@FLHemoAssoc

www.youtube.com/user/FloridaHemophilia

FHA is online! Visit www.floridahemophilia.org

for upcoming programs, event photos and more!

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