Infusions N E W S L E T T E R O F T H E H E M O P H I L I A F O U N D AT I O N O F N O R T H E R N C A L I F O R N I A
•
FA L L 2 0 1 5
Holiday Receptions Oakland, Madera and Modesto
H
FNC spread holiday cheer throughout Northern California in December, with holiday parties in Oakland, Madera and Modesto. Santa Claus made a special appearance at each luncheon, bringing presents and posing for photos with the families that attended.
OAKLAND
MADERA
At the Oakland event, HFNC’s annual award were given out in a variety of categories, recognizing the hard work done by members of the bleeding disorders community and industry in 2015. The Volunteer of the Year award was given to Sandy Puckett for her service in helping to organize and run HFNC events. The Special Project Award went to Michelle Higgins for creating and maintaining the new chapter website. Cathy Marquez was awarded youth of the year for her growing leadership role at Camp Hemotion. Chapter Recognition Award of Excellence was given to Marilyn August for her across the board support of HFNC’s mission. Karen Meier and Barbara Harvey each received the Step Up Volunteer award for taking on larger roles in the community over the past year. Sue Anderson Community Member of the Year award was given to Damia Dillard for her work in her role as social worker at the UC Davis HTC and helping the chapter in our new service area. For their work in starting up the Female Factor group, Ashley Gregory and Christa Parra were named Support Persons of the Year. The Dr. Marion Koerper Award for Medical Excellence went to Dan Peterson for his work supporting the infirmary at Camp Hemotion.
MODESTO 1
INTRODUCING
PROVEN PROPHYLAXIS + SIMPLE,* TWICE-WEEKLY DOSING SCHEDULE =
moments YOUR WAY 2
*ADYNOVATE allows you to infuse on the same 2 days every week.
ADYNOVATE [Antihemophilic Factor (Recombinant), PEGylated] Important Information Indication ADYNOVATE is used on-demand to control bleeding in patients 12 years of age and older with hemophilia A. ADYNOVATE can reduce the number of bleeding episodes when used regularly (prophylaxis). ADYNOVATE is not used to treat von Willebrand disease.
DETAILED IMPORTANT RISK INFORMATION You should not use ADYNOVATE if you: • Are allergic to mice or hamster protein • Are allergic to any ingredients in ADYNOVATE or ADVATE [Antihemophilic Factor (Recombinant)] Tell your healthcare provider if you are pregnant or breastfeeding because ADYNOVATE may not be right for you. You should tell your healthcare provider if you: • Have or have had any medical problems. • Take any medicines, including prescription and non-prescription medicines, such as over-the-counter medicines, supplements or herbal remedies. • Have any allergies, including allergies to mice or hamsters. • Have been told that you have inhibitors to factor VIII (because ADYNOVATE may not work for you). Your body may form inhibitors to Factor VIII. An inhibitor is part of the body’s normal defense system. If you form inhibitors, it may Baxalta, Advate, and Adynovate are trademarks of Baxalta Incorporated. USBS/MG159/15-0189
2
stop ADYNOVATE from working properly. Consult with your healthcare provider to make sure you are carefully monitored with blood tests for the development of inhibitors to Factor VIII. You can have an allergic reaction to ADYNOVATE. Call your healthcare provider right away and stop treatment if you get a rash or hives, itching, tightness of the throat, chest pain or tightness, difficulty breathing, lightheadedness, dizziness, nausea or fainting. The common side effects of ADYNOVATE are headache and nausea. Tell your healthcare provider about any side effects that bother you or do not go away. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088. Please see following page for ADYNOVATE Important Facts. For full Prescribing Information visit www.ADYNOVATE.com. Reference: 1. ADYNOVATE Prescribing Information. Westlake Village, CA: Baxalta US Inc.
How should I use ADYNOVATE? (cont’d) You may have to have blood tests done after getting ADYNOVATE to be sure that your blood level of factor VIII is high enough to clot your blood. Call your healthcare provider right away if your bleeding does not stop after taking ADYNOVATE.
Important facts about
What should I tell my healthcare provider before I use ADYNOVATE?
ADYNOVATE [Antihemophilic Factor (Recombinant), PEGylated]
You should tell your healthcare provider if you:
This leaflet summarizes important information about ADYNOVATE. Please read it carefully before using this medicine. This information does not take the place of talking with your healthcare provider, and it does not include all of the important information about ADYNOVATE. If you have any questions after reading this, ask your healthcare provider.
• Have or have had any medical problems.
What is the most important information I need to know about ADYNOVATE? Do not attempt to do an infusion to yourself unless you have been taught how by your healthcare provider or hemophilia center. You must carefully follow your healthcare provider’s instructions regarding the dose and schedule for infusing ADYNOVATE so that your treatment will work best for you.
• Take any medicines, including prescription and non-prescription medicines, such as over-the-counter medicines, supplements or herbal remedies. • Have any allergies, including allergies to mice or hamsters. • Are breastfeeding. It is not known if ADYNOVATE passes into your milk and if it can harm your baby. • Are pregnant or planning to become pregnant. It is not known if ADYNOVATE may harm your unborn baby. • Have been told that you have inhibitors to factor VIII (because ADYNOVATE may not work for you).
What are the possible side effects of ADYNOVATE? You can have an allergic reaction to ADYNOVATE.
What is ADYNOVATE? ADYNOVATE is an injectable medicine used to replace clotting factor (factor VIII or antihemophilic factor) that is missing in people with hemophilia A (also called “classic” hemophilia). Hemophilia A is an inherited bleeding disorder that prevents blood from clotting normally. ADYNOVATE is used on-demand to control bleeding in patients 12 years of age and older with hemophilia A. ADYNOVATE can reduce the number of bleeding episodes when used regularly (prophylaxis).
Call your healthcare provider right away and stop treatment if you get a rash or hives, itching, tightness of the throat, chest pain or tightness, difficulty breathing, lightheadedness, dizziness, nausea or fainting. The common side effects of ADYNOVATE are headache and nausea. Tell your healthcare provider about any side effects that bother you or do not go away. These are not all the possible side effects with ADYNOVATE. You can ask your healthcare provider for information that is written for healthcare professionals.
ADYNOVATE is not used to treat von Willebrand disease. What else should I know about ADYNOVATE and Hemophilia A? Who should not use ADYNOVATE? You should not use ADYNOVATE if you: • Are allergic to mice or hamster protein • Are allergic to any ingredients in ADYNOVATE or ADVATE Tell your healthcare provider if you are pregnant or breastfeeding because ADYNOVATE may not be right for you.
Your body may form inhibitors to Factor VIII. An inhibitor is part of the body’s normal defense system. If you form inhibitors, it may stop ADYNOVATE from working properly. Consult with your healthcare provider to make sure you are carefully monitored with blood tests for the development of inhibitors to Factor VIII. Medicines are sometimes prescribed for purposes other than those listed here. Do not use ADYNOVATE for a condition for which it is not prescribed. Do not share ADYNOVATE with other people, even if they have the same symptoms that you have.
How should I use ADYNOVATE? ADYNOVATE is given directly into the bloodstream. You may infuse ADYNOVATE at a hemophilia treatment center, at your healthcare provider’s office or in your home. You should be trained on how to do infusions by your healthcare provider or hemophilia treatment center. Many people with hemophilia A learn to infuse their ADYNOVATE by themselves or with the help of a family member.
The risk information provided here is not comprehensive. To learn more, talk with your health care provider or pharmacist about ADYNOVATE. The FDA approved product labeling can be found at www.ADYNOVATE.com or 855-4-ADYNOVATE. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.
Your healthcare provider will tell you how much ADYNOVATE to use based on your individual weight, level of physical activity, the severity of your hemophilia A, and where you are bleeding. Reconstituted product (after mixing dry product with wet diluent) must be used within 3 hours and cannot be stored or refrigerated. Discard any ADYNOVATE left in the vial at the end of your infusion as directed by your healthcare professional.
Baxalta US Inc. Westlake Village, CA 91362 USA U.S. License No. 2020 Issued 11/2015 15E001-ADY-US
NEWS
Hispanic Heritage Education Day
W
hat a great year we are closing out! Our programs give us the opportunity to provide education, support and much more, like for families to reunite and establish new friendships. Throughout the year we were lucky to find new families to our community, families that are already starting to know and to use the services we offer. We are growing! Family Camp, Crab Feed, Family Education Day, Camp Hemotion, Wine and Golf, and the most important program for Latinos: HHME. This year, the HHME, is proud to report that more than 220 people attended and participated in a full day of workshops, Mariachi, Aztecs Dancers, and much more. This beautiful program is becoming a tradition and is very much anticipated by our Hispanic community. Waiting for 2016!
EXECUTIVE STAFF AND BOARD OF DIRECTORS Dawn Pollard PRESIDENT
Peter Barbounis VICE-PRESIDENT
Pamela Morris SECRETARY
Victor Chao TREASURER
Suzanne Goldman Tina Lau Robert Toledo Iris Meadow Nicholas Self Susan Kuhn Cathy Mancuso McClelland Robert Seaton Vikas Meka BOARD MEMBERS
STAFF Patrick Dunlap EXECUTIVE DIRECTOR
Lonnie Wood EXECUTIVE ASSISTANT
CONTACT INFORMATION 66400 Hollis Street, Suite 6 Emeryville, CA 94608 E Office hours: Mon-Fri, O 88:30 a.m. to 5 p.m. www.hemofoundation.org w iinfo@hemofoundation.org
¡Q
ué año tan magnífico el que estamos cerrando! Nuestros programas nos dan la oportunidad de ofrecer educación, apoyo y más aun, para que familias se reen cuentren y establescan nuevas amistades. Durante todo el año tuvimos la suerte de encontrar familias nuevas a la communidad, familias que ya están empesando a conocer y a utilizar los servicios que ofrecemos. Estamos creciendo. ! Family Camp, Crab Feed, Family Education Day, Camp Hemotion, Wine and Golf,y el programa más importante para los Latinos: Hispanic Heritage Month Eevent (HHME). Este año estamos orgullosos de informar que más de 220 personas asistieron y participaron en el HHME en una jornada de talleres, Mariachi, Danzantes Aztecas y mucho más. Este hermoso programa se está convirtiendo en una tradición y es muy esperado por nuestra comunidad Hispana. ¡Esperando por el 2016!
4
Espanol E Alex Mateos A SPANISH BILINGUAL COORDINATOR
espanol@hemofoundation.org 510-658-3287 (510) 658-3324 phone (510) 658-3384 fax
CALENDAR
2016 Save the Date Blood Brotherhood - pending Asian Infusion - pending HTC Camp Pizza parties - pending NHF Victory for Women, webinar series -TBD January 8-9 11 15-17 26-28 29-31 30
Baseball Clinic - pending Hemophilia Community Medical Reception - pending HTC - Family Days, Symposiums - pending Educational Dinners TBD- pending
NHF CA Chapter meeting Board Meeting - in person Family Camp Walk Training NACCHO Female Factor - Victory for Women-San Jose
February 6 Female Factor - Blood Sisterhood-Bay Area 8 Board Meeting - call 13 Crab Feed, Oakland CA 24-26 NHF Washington Days 27 Taylor Family Foundation Camp Directors Meeting March Volunteer Orientation- pending 14 Board Meeting - in person 12 or 19 Family Education Day- pending 12 or 19 Female Factor - Steps for Living-North of Santa Rosa 31to HFA Symposium – Las Vegas, NV April 2nd April 4 8-10 16 11-14 17 24-25 23 30
Board Meeting - call BLeaders - Bay Area - pending Dads in Action, Bay Area - Oakland A’s Game/ Tailgate BBQ NHF Leadership Seminar - New Orleans, LA World Hemophilia Day Bi-Regional HTC Conference, Scottsdale, AZ Female Factor - Blood Sisterhood-Sacramento Walk
May 9 9-10 11 15 28
Board Meeting - in person HCC Future Leaders - Sacramento Leg Day Sacramento Spanish Education Day- Biogen, Fresno - pending Female Factor -Victory for Women-Fresno
June 4 6 11-18 25
Dads in Action, Golfland Sunsplash Waterpark, Roseville Board Meeting - call Camp Hemotion Female Factor - Blood Sisterhood-Santa Rosa
July 9 11 21-23 24-28 August 1 7-8 13 14-20 27
Female Factor - Steps for Living-San Jose Board Meeting - in person NHF Annual Meeting WFH World Congress, Orlando, FL HFNC Scholarships due Board Meeting - call Vines Hops and Golf Female Factor - Blood Sisterhood-Bay Area California Coastal Ride- HCC Advocacy Summit - Hemophilia Council of California, Manhattan Beach
September 10 Dads in Action, Alcatraz Island Tour, San Francisco 12 Board Meeting - in person 24 Female Factor - Victory for Women-North of Santa Rosa October 10 Board Meeting - call 15 Female Factor - Blood Sisterhood-Sacramento Hispanic Heritage Day- Baxalata pending Gettin’ In the Game, Junior National Championship,CSL, Phoenix Arizona November 5 Dads in Action, Fresno, TBD 12 Female Factor - Steps for Living-Fresno 14 Board Meeting - in person December 5 Female Factor - Blood Sisterhood-Santa Rosa 4 Oakland Holiday Reception 10 Madera Holiday Reception 11 Modesto Holiday Reception
Future Dates NHF Annual Meeting August 24-26, 2017; Chicago, IL October 11-13, 2018; Orlando, FL October 3-5, 2019; Anaheim, CA
HFNC Special Events May 6, 2017; Walk August 13-14, 2017; Vines, Hops and Golf, Sonoma, CA
Programs TBD Parent Empowering Parents, Sacramento, CA
5
NEWS
Federal 340B Drug Pricing Program in the News November 25, 2015
H
emophilia treatment centers (HTCs) participating in the 340B Drug Pricing Program have been busy over the past several months analyzing and responding to proposed guidance issued by the Department of Health and Human Services’ Health Resources and Services Administration (HRSA). The guidance proposes major changes to how federal grantees, such as the HTCs, and hospitals will participate in the drug discount program. The proposed guidance was released in late August and was open for public comment through October 27, 2015. Of greatest concern to the HTCs were some of the proposed changes related to the definition of an eligible patient, the audit requirements, and issues related to how federal grantees and sub-grantees access and are listed as participants in the program. The guidance is not likely to be finalized until next year and an implementation date of its provisions is uncertain. As background, the Veterans Health Care Act of 1992 designated federally-funded HTCs as covered entities eligible to participate in the 340B Drug Pricing Program. HTCs were included as covered entities to stretch their federal grant funding to provide comprehensive services to all patients served by the center. Before the passage of the Affordable Care Act, access to discounted drugs also prevented patients from exceeding their insurance plans’ lifetime and annual
6
limits. Today, approximately 100 of the 140 HTCs have elected to participate in the 340B program. As a condition of their federal grant, HTCs must invest all revenues from the 340B program back into patient services, care coordination, research and other programs that directly benefit patients. NHF issued a policy statement this year in support of HTC participation in the 340B Program. In addition, NHF submitted a comment letter on the proposed guidance in support of many of the concerns raised by the HTCs and The Hemophilia Alliance. NHF believes that HTCs with 340B drug programs are an important option that should be available to all patients, regardless of their health insurance coverage. Whether HRSA will revise its proposed guidance recognizing the concerns raised by the HTCs is uncertain. There is also the possibility that Congress will act and changes will be made to the 340B Program through legislation. The outcome is uncertain at this point, but it will likely be an issue for HTCs and the bleeding disorders community in 2016. NHF’s public policy team will keep you informed of the outcome of any changes to the 340B Drug Pricing Program and how they might affect you and your family.
NEWS
NHF Statement on FDA Change of Donor Deferral Policies December 22, 2015
O
n December 21, 2015, the Food and Drug Administration (FDA) finalized its revision of blood donor deferral policies for men who have sex with men (MSM). In addition, FDA continued implementation of a national blood surveillance monitoring system, and changed its deferral policies for people with hemophilia and their sexual partners. FDA’s final guidance on donor deferral policies reflects a number of priorities advocated for by NHF and APLUS, the coalition of patient advocacy organizations representing those who use blood, plasma-derived and recombinant products.
deferral policy was suboptimal. It supports the adoption of donor deferral policies that are less discriminatory, as long as the overall risk to end users is not increased.
Beyond changing the MSM lifetime donor deferral to a one-year deferral, the FDA’s guidance includes changes to deferral policies for people with hemophilia and their partners. People with hemophilia (PWH) are no longer deferred because of a risk of transmitting HIV; therefore, their sexual partners will no longer be deferred for this reason. Under prior donor policies, partners of PWH were deferred for a year. However, PWH will be deferred due to the risk of being injured by the large needles used during the donation process.
Disabled Adventure Outfitters
Yesterday’s FDA announcement also discusses the implementation of a national blood surveillance system that will help the agency monitor the effect of this policy change and further help to ensure the continued safety of the blood supply. This monitoring system is a joint project of the FDA and the National Heart, Lung, and Blood Institute (NHLBI) at the National Institutes of Health (NIH). The creation of the blood surveillance system has been a top priority of NHF and APLUS. NHF has advocated that the complex blood safety policies, such as those related to donor deferral, should be based on science. NHF has recognized that the prior lifetime MSM
NHF will work with the FDA, stakeholders in the blood and plasma collection communities, and its colleagues in APLUS as these new donor deferral policies are implemented and the blood surveillance monitoring system continues over the coming months and years.
NEWS
DAO is happy to announce the dates for their 2016 program, which involves whitewater rafting while camping out on the bank of the Trinity River in Northern California. This year’s teen camp will be held July 8-14. This year will also see the return of our Vets Camp for individuals with bleeding disorders over the age of 21. This weekend trip will take place July 15-17 at the same location as the teen trip. Search for our Facebook page and keep an eye on our website at specialadventures.org <http://specialadventures.org> for further details and applications, which will be available in the coming months. Feel free to contact DAO Board President Ben Martin at 650-773-6551 or Program Director Donald “Ziggy” Douglas at 619-787-1763 with questions.
7
THE FIRST FACTOR VIII WITH A PROLONGED HALF-LIFE Learn how a prolonged half-life may affect your infusion schedule
Meet your CoRe Manager Marilyn August E: marilyn.august@biogen.com T: 925-864-0547
Indications ELOCTATE [Antihemophilic Factor (Recombinant), Fc Fusion Protein] is a recombinant DNA derived, antihemophilic factor indicated in adults and children with Hemophilia A (congenital Factor VIII deficiency) for: control and prevention of bleeding episodes, perioperative management (surgical prophylaxis), and routine prophylaxis to prevent or reduce the frequency of bleeding episodes. ELOCTATE is not indicated for the treatment of von Willebrand disease.
Important Safety Information Do not use ELOCTATE if you have had an allergic reaction to it in the past. Tell your healthcare provider if you have or have had any medical problems, take any medicines, including prescription and non-prescription medicines, supplements, or herbal medicines, have any allergies, are breastfeeding, are pregnant or planning to become pregnant, or have been told you have inhibitors (antibodies) to Factor VIII. Allergic reactions may occur with ELOCTATE. Call your healthcare provider or get emergency treatment right away if you have any of the following symptoms: difficulty breathing, chest tightness, swelling of the face, rash, or hives. Your body can also make antibodies called, “inhibitors,” against ELOCTATE, which may stop ELOCTATE from working properly. Common side effects of ELOCTATE are joint pain and general discomfort. These are not all the possible side effects of ELOCTATE. Talk to your healthcare provider right away about any side effect that bothers you or that does not go away, and if bleeding is not controlled after using ELOCTATE. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.
Please see Brief Summary of full Prescribing Information on the next page.
This information is not intended to replace discussions with your healthcare provider. © 2015 Biogen. All rights reserved. Printed in U.S.A. ELO-US-0492 12/15
8
FDA-Approved Patient Labeling Patient Information ELOCTATE™ /el’ ok’ tate/ [Antihemophilic Factor (Recombinant), Fc Fusion Protein] Please read this Patient Information carefully before using ELOCTATE and each time you get a refill, as there may be new information. This Patient Information does not take the place of talking with your healthcare provider about your medical condition or your treatment. What is ELOCTATE? ELOCTATE is an injectable medicine that is used to help control and prevent bleeding in people with Hemophilia A (congenital Factor VIII deficiency). Your healthcare provider may give you ELOCTATE when you have surgery. Who should not use ELOCTATE? You should not use ELOCTATE if you had an allergic reaction to it in the past. What should I tell my healthcare provider before using ELOCTATE? Talk to your healthcare provider about: • Any medical problems that you have or had. • All prescription and non-prescription medicines that you take, including over-the-counter medicines, supplements or herbal medicines. • Pregnancy or if you are planning to become pregnant. It is not known if ELOCTATE may harm your unborn baby. • Breastfeeding. It is not known if ELOCTATE passes into the milk and if it can harm your baby. How should I use ELOCTATE? You get ELOCTATE as an infusion into your vein. Your healthcare provider will instruct you on how to do infusions on your own, and may watch you give yourself the first dose of ELOCTATE. Contact your healthcare provider right away if bleeding is not controlled after using ELOCTATE. What are the possible side effects of ELOCTATE? Common side effects of ELOCTATE are joint pain and general discomfort. Allergic reactions may occur. Call your healthcare provider or emergency department right away if you have any of the following symptoms: difficulty breathing, chest tightness, swelling of the face, rash or hives. Your body can also make antibodies called, “inhibitors,” against ELOCTATE, which may stop ELOCTATE from working properly. Your healthcare provider may give you blood tests to check for inhibitors.
How should I store ELOCTATE? • Keep ELOCTATE in its original package. • Protect it from light. • Do not freeze. • Store refrigerated (2°C to 8°C or 36°F to 46°F) or at room temperature [not to exceed 30°C (86°F)], for up to six months. • When storing at room temperature: • Note on the carton the date on which the product is removed from refrigeration. • Use the product before the end of this 6 month period or discard it. • Do not return the product to the refrigerator. Do not use ELOCTATE after the expiration date printed on the vial or, if you removed it from the refrigerator, after the date that was noted on the carton, whichever is earlier. After reconstitution (mixing with the diluent): • Do not use ELOCTATE if the reconstituted solution is not clear to slightly opalescent and colorless. • Use reconstituted product as soon as possible • You may store reconstituted solution at room temperature, not to exceed 30°C (86°F), for up to three hours. Protect the reconstituted product from direct sunlight. Discard any product not used within three hours. What else should I know about ELOCTATE? Medicines are sometimes prescribed for purposes other than those listed here. Do not use ELOCTATE for a condition for which it was not prescribed. Do not share ELOCTATE with other people, even if they have the same symptoms that you have. Manufactured by: Biogen Idec Inc. 14 Cambridge Center, Cambridge, MA 02142 USA U.S. License # 1697 44279-01 ELOCTATE™ is a trademark of Biogen Idec. Issued June 2014
9
NEWS
Vines, Hops & Golf
August 13-14, 2017, Sonoma, CA
TH
NEWS
BLeaders SAVE THE DATE April 8-10th, 2016 BLeaders is a 3-day leadership and empowerment retreat for teens ages 14-18 in the bleeding disorders community of the HFNC. BLeaders was established in 2008 in order to address the lack of programming available for transitional aged youth within the bleeding disorders community. This retreat serves teens who are no longer camper age for Camp Hemotion, HFNCâ&#x20AC;&#x2122;s one-week residential camping program for children with bleeding disorders and their siblings. At BLeaders, we create a safe environment for teens with bleeding disorders to discuss with their peers the ways in which bleeding disorders affects their lives as teens. Participants engage in community building exercises, educational sessions and personal development activities in order to strengthen their bonds to the community and with one another. In past years, we have taken the youth to challenge courses, camping trips, goal setting workshops, and college campus tours. Past participants have also taken part in service projects such as creating an informational video about hemophilia and how being active members of the community has helped them to live healthier lives.
10
Skilled Pharmacists. Experienced Infusion Nurses. Dedicated Patient Experience Team. Soleo Health provides a team approach to bleeding disorder therapy management. To learn more about programs in Northern California, call Brent Mascorro at 866.665.1121
or 916.340.5660. www.soleohealth.com
Now Approved!
Antihemophilic Factor (Recombinant)
Available EARLY 2016
11
NEWS
NHF and CDC Collaborate on Screening Patients for Inhibitors November 24, 2015
T
he development of an inhibitor (an antibody to infused clotting factor) is one of the most devastating complications of hemophilia, since it prevents factor treatment from stopping bleeding. The Centers for Disease Control and Prevention (CDC) estimates up to 1 in 5 people with hemophilia will develop an antibody (inhibitor) to their infused factor product at some point in their lifetime. Inhibitors have a huge impact on the families they affect and are an important public health concern as well. As a result, both the CDC and the National Hemophilia Foundation (NHF) have undertaken a number of projects to better understand inhibitors with a goal of ultimately developing strategies to prevent their occurrence. The CDC’s Division of Blood Disorders has done significant surveillance and research work in the last 10 years to better understand inhibitors and their impact on patient health. For example, the CDC’s Hemophilia Inhibitor Research Study (HIRS) involved 17 US hemophilia treatment centers (HTCs) and was a public-private partnership with support from Pfizer and Baxter Healthcare (now Baxalta) through the
12
CDC Foundation. The study had two key findings: all people with hemophilia are at risk of developing an inhibitor, even those with mild disease; and many individuals with an inhibitor may not have clinical signs, so inhibitor testing is critical for the diagnosis. In response to these findings, the CDC has worked to reduce barriers to inhibitor screening. For instance, it has revised its hemophilia surveillance system, “Community Counts,” to include inhibitor screening and is offering annual inhibitor screening for patients who are part of this surveillance system at no cost to HTCs or patients. Further, the CDC has improved the inhibitor test itself, making it more accurate and reproducible. For example, heat treatment of specimens precludes the need to “wash-out” or stop factor treatment before drawing the sample. Based on these CDC efforts, NHF’s Board of Directors and its Medical and Scientific Advisory Council (MASAC) recently took a major step by recommending that people with hemophilia be tested for inhibitors at least annually and more frequently if clinically indicated.
On October 6, 2015, NHF released MASAC Recommendation on Standardized Testing and Surveillance for Inhibitors in Patients with Hemophilia A and B (#236). NHF’s MASAC relied heavily on research and surveillance supported by the CDC’s Division of Blood Disorders over the past 10 years in making this recommendation. NHF looks forward to continued collaboration with the CDC and the National Network of HTCs to ensure that annual inhibitor testing is implemented across the US. This is why NHF advocates for funding for the federal hemophilia programs at the CDC and at the Maternal and Child Health Bureau during its annual Washington Days advocacy event. These funds enable the CDC to work directly with HTCs to collect important information on inhibitors. They also allow the CDC to collaborate with NHF to develop consumer and provider education materials that emphasize the importance of regular screening as part of national prevention efforts. NHF will continue to advocate for continued CDC funding for research and surveillance to increase knowledge of the risk factors for inhibitors and to develop inhibitor prevention strategies.
Benefiting the Bleeding Disorder Community
HFNC th Annual ALL YOU CAN EAT Community Crab Feed
Saturday, February 13th, 2016
4:00 pm – 8:00 pm $45 per person
COLOMBO CLUB
5321 Claremont Ave, Oakland, CA 94608
NEWS
Researchers Learn More about FVIII Origins
I
n breakthrough research, Rice University (RU) scientists have uncovered more about the cellular origins of factor VIII (FVIII), a protein that plays a critical role in the blood clotting process. The study paper was co-authored by research biochemist Nancy A. Turner, BA, and hematologist Joel L. Moake, MD, at RU’s Department of Bioengineering.
With her expertise in biochemistry, Turner first conducted a series of lab tests to verify the presence of FVIII in HUVECs and GMVECs. Follow-up research confirmed that FVIII is not only synthesized in HUVECs and GMVECs, but is also stored in and secreted from Weibel-Palade bodies (WPBs) within these cells. WPBs are specialized organelles (part of a cell with a specific function) that also contain von Willebrand factor (VWF), another critical protein that binds to FVIII during the clotting cascade. VWF works as a carrier for FVIII as it circulates in the bloodstream.
Earlier studies established that FVIII is produced in endothelial cells that line the walls of blood vessels in organs such as the heart, liver and intestines. RU investigators have delved further by looking for the specific source of FVIII generation and deployThis discovery has future potential therapeutic significance for ment from within different types of endothelial cells. people with bleeding disorders. “Now that we recognize that factor VIII is normally synthesized in endothelial cells and Turner and Moake’s experiments focused on human umbilical vein endothelial cells (HUVECs)’ which are found in large veins, stored in Weibel-Palade bodies, those become the precise, most effective physiological targets for gene delivery,” concluded and glomerular microvascular endothelial cells (GMVECs), Moake. which are located in the smallest capillaries of the kidneys. Although the presence of FVIII in these types of cells had not The article, “Factor VIII Is Synthesized in Human Endothelial been previously confirmed, investigators had recognized them, Cells, Packaged in Weibel-Palade Bodies and Secreted Bound to particularly HUVECs, as a viable focus of research for several ULVWF Strings,” was published online October 16, 2015, in the reasons. journal PLOS ONE. “HUVECs are the generic human endothelial cells that (biological researchers) use the first time they do anything,” Turner said. Source: Rice University news release dated November 2, 2015 “They’re cheap. They’re easy to work with, and they’ve been the model for endothelial cells for, I don’t know, at least 50 years.”
8
13
THE LARGEST
INTERNATIONAL
MEETING FOR
THE GLOBAL
BLEEDING DISORDERS
COMMUNITY Deadline for early registration and abstract submission: JANUARY 22, 2016 Hosted by:
Organized by:
www.wfh.org/congress
8th Annual Bay Area Hemophilia Walk
April 30th, 2016
8th Annual Bay Area Hemophilia Walkth Annual Bay Area Hemophilia Walk
April 30th, 2016
HELP US RAISE FUNDS FOR OUR COMMUNITY PROGRAMS 14
COMING
SOON KOVALTRY
TM
Antihemophilic factor (recombinant)
Register for updates at www.KOVALTRY.com Bayer and the Bayer Cross are registered trademarks of Bayer. KOVALTRY is a trademark of Bayer. © 2015 Bayer HealthCare Pharmaceuticals Inc. All rights reserved. Printed in USA 08/15 PP-675-US-0006
¿Qué usted puede recibir de nosotros?
What can you expect from us? We are here 24 hours a day, 365 a year. Hemophilia doesn’t take a break and neither do we.
We coordinate nursing services that come to you, on your schedule.
We provide personalized services to match your family’s unique needs.
We offer all factor brands with supplies delivered on time to your doorstep.
We have reimbursement specialists that track, navigate, and handle all your insurances issues; one less thing for you to worry about.
We are community advocates, educators, volunteers, and your child’s mentor.
(877) 276-4248 | www.brothershealthcare.com
Estamos aquí las 24 horas al día, 365 días al año. Hemofilia no toma descanso, nosotros tampoco.
Nosotros coordinamos los servicios de enfermera(o), en la cual lo visitarán cuando usted este disponible.
Nosotros proveemos un servicio personalizado y único para la necesidad especial de cada familia.
Ofrecemos todo tipos de factor con todas las herramientas medicinales que necesite, directamente entregado a usted, en sus manos, y en su casa.
Tenemos especialistas de rembolso que se encargan de navegar, lidiar, y aclarecer todos los problemas y compilación de la seguranza medica; otra área en donde no tiene que preocuparse.
Somos abogados por nuestra comunidad, educadores, voluntarios, y hasta un consejero para su hijo(a).
Se Habla Español (866) 591-3955 | www.brothershealthcare.com
15
NEWS
SIPPET Results Presented at ASH 2015 December 17, 2015
T
he results of the SIPPET (Survey of Inhibitors in Plasma-Products Exposed Toddlers) study were presented at a plenary session of the recent American Society of Hematology (ASH) conference. SIPPET was a randomized study which took place between January 2010 and December 2014 that collected data on 251 children <6 years of age with severe hemophilia A from 14 countries in Africa, North and South America, Asia and Europe. The purpose of the study was to compare the incidence of factor VIII (FVIII) inhibitors in minimally treated and previously untreated patients (PUPs) treated with von Willebrand (VWF)-containing plasma-derived (pd) FVIII concentrates versus those treated with recombinant FVIII concentrates. Following are a few important features of the study: • The ASH presentation suggested that in PUPs, the combined risk of developing a high or low titer inhibitor within the first 50 exposure days (EDs) when using recombinant factors was 1.87-fold higher than when using VWF-containing plasmaderived factor concentrates. For high-titer inhibitors only, the risk was 1.7 fold higher for those using the recombinant class of products, but this difference did not reach statistical significance. • SIPPET compared classes of products, not specific treatment products within or between classes. The comparison was between intermediate-purity pdFVIII products that contain VWF, not all pdFVIII products, vs. first through third generation recombinant products only. Newer products that have been brought to the market since the study began in 2010 were not included within SIPPET. • The study only looked at the first 50 EDs and did not address the issue of product switching after 50 EDs.
16
While this is potentially an important study, the data are incomplete at this time, and it will be critical to review the full study report when it is published. We do not yet know when or where the study will be published. There are numerous other studies that have been published or are ongoing that address the issue of inhibitor development in PUPS. The field is constantly evolving as is the availability of new treatment products that were not part of this study. Other on-going studies may prove to have additional relevance in determining which product to begin treatment with for PUPS. In addition to product type, other factors of importance in determining inhibitor risk include whether there is a family history of inhibitors and whether the patient’s genotype is one that is associated with a higher risk of inhibitor development. All of these factors need to be taken into account when choosing a product for initiation of factor replacement in PUPS. At this time, MASAC is recommending that families of minimally treated or previously untreated patients discuss the issues surrounding product selection with their hemophilia treater. At its upcoming meeting in February, MASAC will carefully review the full study report, if published, as well as reports from other studies that have been published, before deciding whether to issue a revised recommendation on the use of recombinant products in PUPS.
HFA’s Symposium is an annual community-centered educational event that draws together hundreds of members from the bleeding disorders community to share information, learn new advancements, and build a network of support! HFA recognizes that bleeding disorders are not a one size fits all medical condition. Dealing with, or caring for a loved one with a bleeding disorder is a lifelong journey requiring diligence and flexibility in handling the challenges along the way. Symposium offers community members the opportunity to learn and grow personally and collectively in becoming strong self-advocates! During our powerful sessions, community members can connect on a personal level with our presenters. This year, we are proud to offer special tracks for families treating inhibitors, and Spanish-speaking families.
Symposium Highlights: x
NEW! Spanish-language track.
x
Special track for families with inhibitors.
x
Advocacy tools and strategies.
x
Updates on therapies, policies, and more!
Register 7oday! http://www.hemophiliafed.org What Participants Say About Symposium: “HFA Symposium gave us the chance to discuss topics in depth with knowledgeable speakers and participants with shared experiences.” “Knowing about other families' life experience and learning to solve problems and advocate for ourselves.” “At Symposium, we learned that one family, one person can make a difference. People are listening–we all have something to say.”
17
Introducing…
Tap into My Access to help manage your condition—or your loved one’s—in a cost-effective manner to help you cover the out-ofpocket costs associated with your therapy
through the My Source™ program
for reenrollment every year
For more information or to enroll, call the My Source Hotline at 1-800-676-4266 to speak with a My Source Care Coordinator.
Visit www.MySourceCSL.com. www.facebook.com/vonWillebrandandU My Source™ is a trademark of CSL Behring LLC. ©2014 CSL Behring LLC 1020 First Avenue, PO Box 61501, King of Prussia, PA 19406-0901 USA www.CSLBehring-us.com www.MySourceCSL.com COA13-11-0083 11/2013
18
Half the volume
Twice the factor*
ALPHANATE® (antihemophilic factor/von Willebrand factor complex [human]) is now available in a 2000 IU FVIII vial with a reconstitution volume of only 10 mL. * That’s TWICE the amount of factor of the largest vial available for other FVIII/VWF products,1-4 so patients may require:
Learn more at alphanate.com
• Less volume • Less time • Fewer syringes
Isn’t it time you tried ALPHANATE? Indications ALPHANATE® (antihemophilic factor/von Willebrand factor complex [human]) is indicated for: • Control and prevention of bleeding in patients with hemophilia A • Surgical and/or invasive procedures in adult and pediatric patients with von Willebrand disease (VWD) in whom desmopressin (DDAVP®) is either ineffective or contraindicated. It is not indicated for patients with severe VWD (Type 3) undergoing major surgery
Important Safety Information ALPHANATE is contraindicated in patients who have manifested life-threatening immediate hypersensitivity reactions, including anaphylaxis, to the product or its components. Anaphylaxis and severe hypersensitivity reactions are possible. Should symptoms occur, treatment with ALPHANATE should be discontinued, and emergency treatment should be sought. Development of activity-neutralizing antibodies has been detected in patients receiving FVIII containing products. Development of alloantibodies to VWF in Type 3 von Willebrand disease (VWD) patients has been occasionally reported in the literature. Thromboembolic events may be associated with AHF/VWF Complex (Human) in VWD patients, especially in the setting of known risk factors. Intravascular hemolysis may be associated with infusion of massive doses of AHF/VWF Complex (Human). Rapid administration of a FVIII concentrate may result in vasomotor reactions. Plasma products carry a risk of transmitting infectious agents, such as viruses, and theoretically, the Creutzfeldt-Jakob disease (CJD) agent, despite steps designed to reduce this risk. The most frequent adverse events reported with ALPHANATE in >5% of patients are respiratory distress, pruritus, rash, urticaria, face edema, paresthesia, pain, fever, chills, joint pain, and fatigue.
Please see brief summary of ALPHANATE full Prescribing Information on adjacent page. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088. References: 1. ALPHANATE® (antihemophilic factor/von Willebrand factor complex [human]) Prescribing Information. Grifols. 2. CSL Behring. Humate P Package Insert. August 2013; 3. Octapharma. Wilate Package Insert. January 2012; 4. Kedrion. Koate-DVI Package Insert. August 2012. For more information: Grifols Biologicals Inc. Tel. 888-GRIFOLS (888-474-3657)
Grifols Biologicals Inc. 5555 Valley Boulevard, Los Angeles, 90032 CA - USA
© 2014 Grifols Inc.
All rights reserved.
Printed in USA.
July 2014
A817-0714
www.grifols.com Tel. 888-GRIFOLS (888 474 3657)
www.grifols.com
19
Ready to help you define your IXperience™ Your IXINITY® Product Specialist, Leo Tellez
Many families I’ve worked with have shared stories of perseverance, challenge, and dedication. I am moved by their experiences and want to do more to help the rare blood disorder community. — Leo Tellez
Let’s talk about IXINITY and how you can get the most out of Emergent-sponsored programs, including the Generation IX Project and the B More™ Scholarship Program.
Contact Leo at 503.314.7298 or ltellez@ebsi.com
IXINITY.com Manufactured by Cangene Corporation, a subsidiary of Emergent BioSolutions Inc. and distributed by Cangene bioPharma, Inc., a subsidiary of Emergent BioSolutions Inc. IXINITY [Coagulation Factor IX (Recombinant)], IXperience, B More, and any and all Emergent BioSolutions Inc. brand, product, service and feature names, logos, slogans are trademarks or registered trademarks of Emergent BioSolutions Inc. or its subsidiaries in the United States or other countries. All rights reserved. © 2015 Emergent BioSolutions Inc.
20
IXI 136-0915
NEWS
Congratulations to the 2015 HFNC Scholarship Winners! The Brett Leach Memorial Scholarship Fund Brett’s commitment to helping others did not stop when he left work. Brett was an active volunteer of his own time, serving in leadership capacities at both the Central California and Northern California Hemophilia Foundations, and as a co-director of “Camp Hemotion,” the annual summer camp for children with bleeding disorders in Northern California. Provides a scholarship in the amount of $500.00 to an individual with a bleeding disorder seeking to continue or further their education.
The Mark Helm Memorial Scholarship Fund
Perseus Patel
Mark’s involvement in the bleeding disorders community has been both personal and professional. He started Tri Medical Inc. in 1988, a small business designed to meet the needs of patients and families living with hemophilia. Quantum Health Resource bought Ti Medical in 1993, and Mark continued as Branch Manager for several years. After taking a short break to spend time with his family, he returned to the community creating Herndon Healthcare, Inc., a company dedicated to serving the needs of people with bleeding disorders. He spent many years serving on the CCHF and HFNC boards as well as the scholarship committees. Mark and Julie, his wife of 24 years, adopted two beautiful children, Hugo (17) and Rebecca (11). Hugo has moderate hemophilia. Provides a scholarship in the amount of $500.00 to an individual with a bleeding disorder seeking to continue or further their education.
The Sue Anderson Memorial Scholarship Fund Sue is well known in the Central Valley for founding the Fresno Hemophilia Auxiliary. With the help of her dear friends, Mark Helm, Susan Kuhn, and Marta Terray, Sue and the Fresno Auxiliary have been able to reach a large percentage of families affected by hemophilia in the Central Valley and help them manage this disease with professional and community support. In honor of Sue and her heartfelt community and volunteer spirit, the Anderson Family is requesting that the recipient of the Sue Anderson Memorial Scholarship Fund donate a small portion of their time by volunteering with the HFNC at two events of your choice. This will not only help the hemophilia community, but will also instill a sense of community involvement in the recipient of this scholarship. Provides a scholarship in the amount of $500.00 to an individual with a bleeding disorder seeking to continue or further their education.
Molly Selfridge The Daniel Gallagher Memorial Scholarship Fund Daniel Gallagher was born September 19, 1949, in San Jose, California. Daniel was born with severe hemophilia, which was not diagnosed until he was four years old. At the same time his brother Patrick, then 6 years old was also diagnosed. Daniel studied Business at Hayward State University and Law at Santa Clara University. Daniel and his law partner had a successful law practice in Los Gatos. His law partner continues to run the business today. Daniel inspired to be a judge one day. He enjoyed collecting antiques, old cars and teaching legal courses at West Valley College. However, his pride and joy was his family.
Peter Skorokhodov
Daniel met his true love, Michelle Chiechi-Gallagher, when they were 16 years old. Later they married and had two wonderful children. Unfortunately, Daniel’s children, Matthew and Caitlin, were only one and three years old at the time of his death. They have lived life only knowing how special their father was by stories told by their mother, family members, and numerous pictures. Education was a very important part of Daniel’s life. Daniel would take great pride to know that the Daniel Gallagher Scholarship would help further education for those with bleeding disorders. Provides a scholarship in the amount of $500.00 to an individual with a bleeding disorder seeking to continue or further their education.
21
CAREERS
New HFNC Bilingual Spanish Coordinator My name is Alex Mateos I am the new Spanish bilingual coordinator for the hemophilia foundation. I have had the privilege of being an active member in this community for many years. I have participated in camps, information events, and helped many people with different things related to the community. I am here to assist the Spanish speaking community and make an impact toward our mission goal. I have gone through most of the problems the hemophilia community is going through and with the help of the community I was able to overcome all these things and gain new family members from the community who I trust and care for along the way. Feel free to outreach to me with any questions or assistance.
22
Mi nombre es Alex Mateos Yo soy el Nuevo Español bilingue cordinador para la fudación de hemophilia. Yo tengo el privilegio de ser un miembro activo dentro de la comunidad por muchos años. Yo participe en campamentos, eventos de información, y ayude muchas personas con differentes cosas de la comunidad. Yo estoy aqui para assistir la comunidad quienes hablen español y acer un impacto con dirección a nuestro objetivo meta. Yo e pasado por muchas de la problemas que la comunidad tiene y con la ayuda de la comunidad yo pude sobresalir todas estas cosas y ahora yo tengo nuevos miembros de familia de la comunidad en quienes yo puedo confiar y me importan mucho. Por favor con confianza avisen me de cual quier pregunta o assistencia.
For more information, contact your Baxalta representative today: Aaron Hermann Phone: (916) 801-0562 E-mail: aaron.hermann@baxalta.com To learn more, visit www.RIXUBIS.com. Baxalta and Rixubis are trademarks of Baxalta Incorporated. USBS/MG45/13-0004a(3)
23
The Hemophilia Foundation of Northern California (HFNC) does not endorse any particular pharmaceutical manufacturer or home care company. PLEASE NOTE: The companies whose advertisements are listed herein have purchased this space, and are NEVER provided with members’ names, addresses or any other personal details. Paid advertisements and paid inserts should not be interpreted as a recommendation from HFNC, nor do we accept responsibility for the accuracy of any claims made by paid advertisements or paid inserts. Since we do not engage in the practice of medicine, we always recommend that you consult a physician before pursuing any course of treatment. Information and opinions expressed in this publication are not necessarily, those of the Hemophilia Foundation of Northern California, or those of the editorial staff. MATERIAL PRINTED IN THIS PUBLICATION MAY BE REPRINTED WITH THE EXPRESS PRIOR WRITTEN PERMISSION FROM THE EXECUTIVE DIRECTOR. SPRING, SUMMER, FALL OR WINTER WITH YEAR MUST BE INCLUDED Hemophilia Foundation of Northern California is a 501(c)(3) non-profit. Donations are tax-deductible, much needed and appreciated. The Infusions Newsletter is underwritten yearly by generous grants from Baxter and Bayer.
DONATE YOUR VEHICLE TO SUPPORT NHF AND HFNC Donate your car, boat, motorcycle, truck or other vehicle to NHF’s Vehicle Donation Program. Visit NHF at www.hemophilia. org and click the “vehicle donation program” link, or call 1 (855) NHF-4-CAR. A representative will explain all of the details — including how to schedule free pick-up service that’s convenient for you.
Visit our website at www.hemofoundation.org for important information. If you would like to advertise in the next issue, please contact the foundation. 24