Spring 2017 7th Annual Walk in the Walk in the ungle...

The Official Newsletter of the Florida Hemophilia Association

Spring 2017

Teen and Young Adult RetreatBoca Raton, FloridaJune 29 - July 2

Dolphin Day CampKey Largo, FloridaJuly 14

PEP en EspañolSouth FloridaJuly 14-16

34th Annual Family EducationSymposiumWest Palm Beach, FloridaAugust 3-6

Dolphin Day CampKey Largo, FloridaAugust 11

NHF 69th Annual MeetingChicago, IllinoisAugust 24-26

“SAVE THE DATE”

Executively SpeakingPage 2

Community CornerPage 8, 9 - 18

NHF News3, 17 - 18

FHA Resource CenterPage 19

INSIDE

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Walk in the Jungle Walk in the Jungle 7th Annual

n April 30, 2017, members andsupporters of the bleeding disorders

community came together at Jungle Island forthe 7th Annual Walk in the Jungle. It was aclear, sunny day with only a touch of rain.

This year, more than 700 walkers united toraise critical funds and awareness for thebleeding disorder community. The AnnualWalk in the Jungle has become such animportant event for the Florida HemophiliaAssociation. Each year, we raise the level ofawareness for hemophilia and other inherited

bleeding disorders. FHA’s main goal isto educate people, not just thoseaffected, but their family, caregivers,

friends, and the general public as well.

The funds from this year’s Walk will gotoward programs like the Teen andYoung Adult Retreat and the Annual

Family Education Symposium, amongnumerous others. Funds will also go towardadvocacy efforts, like Legislative Days, wheremembers of the community go to Washingtonto speak with their representatives about issuesthat affect them and their families; towardfinancial assistance for families in need throughour Compassionate Care initiative; and finally,toward research.

At this year’s Walk, we raised over $88,000!!!The money that we raise is instrumental tocontinuing all of the wonderful things that wedo here at FHA to support the bleedingdisorders community and their families. Weare so close to reaching our $100,000 goal, andwe hope you can help. Proceeds from the Walkwill go towards our compassionate careprogram, college scholarship program, campprograms, family services, and groundbreakingscientific research.

O

Summertime in Florida and at FHA isalways filled with fun! We’ve scheduled amyriad of activities for you and your familyto choose from such as: Dolphin DayCamp, FES, Teen and Young Adult Retreat,Bowling, and more! Don’t miss out on allof these great events and sign up today.All of the upcoming programs/events are

posted on our website www.floridahemophilia.org so be sureto check it out along with all of the invitations we send out toyou frequently through Constant Contact.

Check out the past events articles and photos in this issue,including the 7th Annual Walk in the Jungle, and I lookforward to seeing you all soon!

¡Es la temporada de verano en Florida y en la FHA siempre estállena de diversión! Hemos programado un sinnúmero deactividades para que usted y su familia puedan elegir, talescomo: Campamento diurno Dolphin (Dolphin Day Camp), FES,Retiro para adolescentes y adultos jóvenes (Teen and YoungAdult Retreat), Boliche (Bowling) y mucho más. No se pierdatodas estas excelentes actividades e inscríbase hoy mismo. Ennuestro sitio web, www.floridahemophilia.org, encontrará todoslos próximos programas y actividades, así es que asegúrese devisitarlo y estar al pendiente de todas las invitaciones que leenviamos con frecuencia a través de Constant Contact.

En este número, vea artículos y fotos sobre actividadesanteriores, entre ellas la 7a Caminata anual en la selva (7thAnnual Walk in the Jungle). ¡Espero verlos a todos muy pronto!

2 SPRING 2017 • LIFELINE

ExecutivelySpeaking MensajeEjecutivo

Please consider making an additional contribution and also lettingeveryone know how close we are to meeting our goal. The Walkpage www.hemophiliawalk.donordrive.com/event/floridawill remain open until we meet or exceed our goal.

Thank you to everyone who participated at this year’s Walk,including Honorary Chair Lorena Estrada, WSVN Anchor andReporter. You are all greatly appreciated by FHA and thefamilies we serve through the state of Florida! A special thankyou to all of our wonderful volunteers, who gave their precioustime to ensure that the Walk went smoothly! And finally, thankyou to our sponsors, whose generous contributions helped makethe Walk even more successful!

We’re looking forward to seeing you all at the 8th Annual Walkin the Jungle next year!!!

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7th AnnualWalk in the Jungle continued...

Debbi AdamkinFHA Executive Director

Debbi AdamkinDirectora Ejecutiva de FHA

3SPRING 2017 • LIFELINE

NATIONAL SPONSORS• Shire• Novo Nordisk• Bayer• Bioverativ• CVS Specialty• Grifols• Hema Biologics• Octapharma• Pfizer• Accredo

LOCAL SPONSORS

Gold Sponsors• Bayer• CSL Behring• Genentech• Novo Nordisk• Octapharma

Silver Sponsors• Clover Systems LLC

Bronze Sponsors• BioTek reMEDys• Broward Health• Bullet Line LLC• Diplomat Specialty Infusion• Embassy Suites• Matrix• Soleo Health• Thrivent Financial Photo Booth Sponsor• Kidz Medical Services

Supporter Sponsor• Bioverativ

Kilometer Sponsors• All Accounting Services II• Group M Advertising & Design• Skyemed Pharmacy• TML Florida Holdings

Breakfast Sponsor• Cottrill’s SP

In-Kind Sponsor

Top FundraiserRobyn Salk

Top Ten Teams1. FHA Blood Drops

2. Team Alex Dones

3. Carly’s Angels

4. MF’s Friends

5. Team JAIZER the “Spartans”

6. We Are CSL

7. Abraham Lincoln School FriendsLima Peru

8. J Walkers

9. Mothers in the Jungle

10. Team Bioverativ

Most Creative Team T-Shirt1. Matrix Health Group

2. Team JAIZER the “Spartans”

3. Team Alex Dones* After May 1st, donations have continued to come in. Amounts may vary.

Thank youto all of our Sponsors

Congratulationsto all of our Teams

• Best Buy• BJ’s Restaurant &

Brewhouse• Del Monte Fresh

Produce• Dunkin’ Donuts• Florida Panthers• Jungle Island• Kennesaw Juice• KenographyOne

Photographic

• Krispy Kreme• MC Alex• Miami Marlins• NuGo Nutrition• Panera Bread• The Photobooth Bros.• Sr. Ceviche• Starbucks• Zalez Photo

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Team Jaizer the Spartans

Team Alex Dones

7th Annual

Walk in the Junglecontinued...


7th Annual Walk in the Jungle, continued...

This year, MORE THAN 700 WALKERS united to raisecritical funds and awareness

for the bleeding disordercommunity.

FHA staff and Walk volunteers!


THURSDAY, AUGUST 3 TO SUNDAY, AUGUST 6, 2017

EMBASSY SUITES BY HILTON WEST PALM BEACH CENTRAL

1601 Belvedere Rd., West Palm Beach, FL 33406

SPACE STILL AVAILABLE! REGISTER TODAY!Visit www.floridahemophilia.org


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WorldHemophilia

Day

n April 17, the bleeding disorders community gatheredat the Hyatt Regency Miami in celebration of World

Hemophilia Day. Established in 1989, the purpose of WorldHemophilia Day is to raise awareness about inherited bleedingdisorders and the 400,000 people it affects worldwide.

O

April 17thMiami Tower

with Red lights


FHA community members connected over a delicious dinner and beverages. Theevening’s tagline was “A night of learning, imagination and inspiration.” Kidsand adults alike had a blast during the hands-on activities, one of which showcasedthe wonders of kinetic energy. The activities showed us what we can accomplish whenwe come together. Afterwards, everyone assembled outside to take in the beautifulMiami Tower, which was lit up in red in recognition of World Hemophilia Day.

This event was sponsored by Bioverativ.

NHF • eNews 2017

BloodCenter of Wisconsin Announces New Test for VWDNHF News Story April, 2017

he BloodCenter of Wisconsin (BCW) recently announcedthe launch of a new and more sensitive test for von

Willebrand disease (VWD), a genetic disorder characterizedby either a qualitative or quantitative flaw in vonWillebrand factor (VWF). Milwaukee-based BCW is a not-for-profit organization that specializes in blood services,organ, tissue and marrow donation, diagnostic testing,medical services and research.

Depending on the specific type, individuals with VWD canexperience bleeding-related symptoms that range from mildto severe. Despite being the most common bleeding disorder

VWD is historically a more challenging condition to screenfor, often necessitating a battery of tests to pinpoint adiagnosis.

The “VWF GPIbM Activity” test is designed to uncoverqualitative VWF defects to reduce variability and provide“more precise, reliable and sensitive test results,” accordingto a BCW press release. The availability of the test could bea boon to clinicians, particularly hematologistsencountering potential cases of VWD.

“As a physician caring for individuals with inheritedbleeding disorders, this development is an excitingadvancement in von Willebrand disease diagnostics,” saidJonathan Roberts, MD, Associate Medical Director,Bleeding & Clotting Disorders Institute, Peoria, IL. “Thisassay will reduce some of the diagnostic challenges in caringfor individuals with von Willebrand disease.”

Source: BloodCenter of Wisconsin press release dated April 4, 2017


T

BloodCenter of Wisconsin anuncia un nuevoanálisis para la enfermedad de Von WillebrandNHF News Story Abril de 2017

l BloodCenter of Wisconsin (BCW) anunciórecientemente la introducción al mercado de un

análisis nuevo y más sensible para la enfermedad de VonWillebrand (EVW), una enfermedad genética que secaracteriza por un fallo cualitativo o cuantitativo en el factorde Von Willebrand (FVW). El BCW es una organización sinfines de lucro con sede en Milwaukee que se especializa enservicios relacionados con la sangre; donación de órganos,tejidos y médula ósea; pruebas diagnósticas; serviciosmédicos e investigaciones.

Según el tipo específico, las personas con la EVW puedenpresentar síntomas hemorrágicos que van de leves aintensos. A pesar de ser el trastorno hemorrágico másfrecuente, la EVW siempre ha sido una enfermedad difícilde detectar y con frecuencia se necesita una serie de pruebaspara precisar el diagnóstico.

El análisis de la “actividad del GPIbM en la EVW” estádiseñado para descubrir defectos cualitativos del FVW parareducir la variabilidad y brindar “resultados analíticos másprecisos, fiables y sensibles”, según el comunicado de prensadel BCW. Tener a su disposición este análisis puede ser degran ayuda para los médicos, en especial para loshematólogos que se enfrentan a posibles casos de la EVW.

“Como médico que atiende a personas con trastornoshemorrágicos hereditarios, esta innovación es un fascinanteavance en las técnicas diagnósticas para la enfermedad deVon Willebrand”, expresó el Dr. Jonathan Roberts, DirectorMédico Adjunto del Bleeding & Clotting Disorders Instituteen Peoria, IL. “Este análisis aminorará algunas de lasdificultades diagnósticas que se presentan en la atención delas personas con la enfermedad de Von Willebrand”.

Fuente: comunicado de prensa BloodCenter of Wisconsin de fecha 4 de abril de 2017

E

NHF • eNews 2017

Beams and Bubbles Deliver Gene Therapy to Mice with Hemophilia BNHF Newsletter Story April 2017

cientists from the Center for Cardiovascular Research atthe University of Hawaii (UH) have developed a novel

approach to hemophilia gene therapy that employsmicrobubbles and an ultrasonic beam to deliver the treatment.

The study was led by Dr. Cindy Anderson and Dr. Chad Walton,who were both studying at UH’s John A. Burns School ofMedicine (JABSOM) during the breakthrough phase ofresearch. Anderson at that time was a graduate student in theCell & Molecular Biology Department at JABSOM and is nowan Assistant Professor at Georgetown University. Walton was anAssistant Professor of Medicine at JABSOM and is now anassistant to the Vice-Chancellor for Research at UH Mānoa.Their study was funded by the American Heart Association andthe U.S. National Institutes of Health.

The UH team took genetic material that can elicit theproduction of factor IX (FIX) and placed it within minisculemicrobubbles composed of lipid molecules that do not dissolvein water. The therapy was then injected into the bloodstreamof mice with hemophilia B. As the microbubbles passedthrough the bloodstream they were hit by a beam ofultrasound that dispersed the genetic “payload” and depositedit into liver cells targeted for their ability to stimulate the

production of FIX. The technique is known as ultrasound-targeted microbubble destruction (UTMD).

The animals subsequently showed both reductions in clottingtime and increased FIX levels. An additional set of mice weretreated via UTMD and evaluated for long-term effects, theresults of which demonstrated a persistent reduction in averageclotting time 160 days after one administration of the therapy.

“We were able to improve clotting in mice for months after asingle treatment,” said Ralph Shohet, MD, Professor ofMedicine and Director of the Center for CardiovascularResearch. Shohet sees unique advantages in UTMD, as it isboth non-viral and minimally invasive.

“Hemophilia is a chronic debilitating disease. If we can treat itsimply, cheaply, and non-invasively with gene therapy we willhave helped to fulfill the promise of the modern medical era.”added Shohet.

The study, “Ultrasound-Targeted Hepatic Delivery of FactorIX in Hemophiliac Mice,” was published in April, 2016, in thejournal Gene Therapy

Source: JABSOM news release dated March 10, 2017


S

Haces y burbujas proporcionan Genoterapia a ratones con Hemofilia BNHF Newsletter Story April 2017

nvestigadores del Centro para InvestigacionesCardiovasculares de la Universidad de Hawái (UH)

han desarrollado un novedoso método en la genoterapiapara la hemofilia que emplea microburbujas y un hazultrasónico para proporcionar el tratamiento.

El estudio estuvo dirigido por la Dra. Cindy Anderson y elDr. Chad Walton, los cuales se encontraban estudiando enla John A. Burns School of Medicine (JABSOM) de la UHdurante la fase decisiva de la investigación. En esemomento, Anderson era estudiante de posgrado en elDepartamento de Biología Celular y Molecular deJABSOM y actualmente es profesora adjunta enGeorgetown University. Walton fue profesor adjunto de

Medicina en JABSOM y actualmente se desempeña comoasistente del Vicerrector para Investigaciones de UHMānoa. La Asociación Estadounidense del Corazón(American Heart Association) y los Institutos Nacionales dela Salud de los EE. UU. (U.S. National Institutes of Health)financiaron el estudio.

El equipo de la UH tomó material genético con capacidadde desencadenar la producción del factor IX (FIX) y locolocó dentro de minúsculas microburbujas compuestas demoléculas de lípidos que no se disuelven en agua. Luego seinyectó la terapia en el torrente circulatorio de ratones conhemofilia B. A medida que las microburbujas pasaban através del torrente circulatorio, eran golpeadas por un haz

I

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Haces y burbujas proporcionan Genoterapia a ratones con Hemofilia BNHF Newsletter Story April 2017 (...Continued)

de ultrasonido que dispersaba la “carga útil” genética y ladepositaba de manera selectiva en las células hepáticas dadasu capacidad para estimular la producción del FIX. Estatécnica se conoce como destrucción a través demicroburbujas dirigida por ultrasonido (ultrasound-targetedmicrobubble destruction o UTMD).

Posteriormente, los animales mostraron reducciones en eltiempo de coagulación y concentraciones más altas del FIX.Se trató a otro grupo de ratones mediante UTMD y se lesevaluó para determinar los efectos a largo plazo; losresultados demostraron una reducción persistente en eltiempo promedio de coagulación 160 días después de unaadministración de la terapia.

“Pudimos mejorar la coagulación en los ratones durantemeses después de un solo tratamiento”, explicó el Dr. RalphShohet, Profesor de Medicina y Director del Centro deInvestigaciones Cardiovasculares. Shohet ve extraordinariasventajas en la UTMD, ya que es una terapia no viral ymínimamente invasiva.

“La hemofilia es una enfermedad crónica y debilitante. Sipudiéramos tratarla de manera sencilla, a bajo costo y noinvasiva con la genoterapia, habremos ayudado a cumplir lapromesa de la era moderna de la medicina”, agregó Shohet.

El estudio, “Ultrasound-Targeted Hepatic Delivery ofFactor IX in Hemophiliac Mice”, se publicó en abril de2016 en la revista Gene Therapy.

Fuente: comunicado de prensa de JABSOM de fecha 10 de marzo de 2017

LADIES DAY OUTAT PAINTING WITH A TWIST

This program was sponsored by Octapharma

NHF • eNews 2017

CENTERS FOR BLEEDING DISORDERS*Hemophilia Treatment Center (HTC)

ABOUT THIS PUBLICATION

Florida Hemophilia

Association Office

(888) 880-8330

Executive Director

Debbi Adamkin(305) [email protected]

National Hemophilia

Foundation

(800) 424-2634

Hemophilia Federation of America

(800) 230-9797

LA Kelley

Communications, Inc.

Free resource material onHemophilia(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

Johns Hopkins All Childen's

Outpatient Care Clinic

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

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

Nemours Children’s

Clinic, Jacksonville

Department of PediatricHematology/Oncology807 Children’s WayJacksonville, FL 32207Phone: (904) 697-3789(904) 697-3600www.nemours.com*HTC

Nicklaus Children’s

Hospital

Kidz Medical ServicesDivision ofHematology/Oncology3100 SW 62 AveSuite 121Miami, Florida 33155Phone: (305) 662-8360

University of Florida

Hemophilia Treatment CenterDivision of Pediatric Hematology/Oncology1600 SW Archer RoadGainesville, FL 32610Phone: (352) 273-9120www.peds.ufl.edu/divisions/hemonc/*HTC

Nemours Children’s

Clinic, Orlando

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

Sacred Heart Pediatric

Hemophilia Program,PensacolaPhone: (850) 416-7712

St. Joseph’s Children’s

Hospital

Pediatric HematologyOncology Out-patientClinic3001 W. Dr. MartinLuther King Jr. Blvd.Tampa, FL 33607Phone: (813) 554-8294or (813) 321-6820*HTC

The University of Miami

Hemophilia Treatment

Center Pediatrics and Adults

University of Miami Miller

School of Medicine

Department of Pediatrics Div of Hematology/OncologyLocator Code D8201601 NW 12th Ave., Room 5019Miami, FL 33136Phone: (305) 243-0834www.htcextras.org*HTC

USF Adult Hemophilia

Center

Hematology 4th Floor13220 Laurel DriveTampa, FL 33612Phone: (813) 974-3725*HTC

LIFE LINE is the officialNewsletter of the FloridaHemophilia Association.It is produced quarterly anddistributed free of charge torequesting members of thebleeding disorder community.

Florida Hemophilia

Association Headquarters

915 Middle River Drive, Suite 421Ft. Lauderdale, FL 33304Toll Free: (888) 880-8330www.floridahemophilia.org

Translation:

Roxanna Delgado

Design and Production:

Group M, Advertising & Design305-235-2538


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

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