Summer 2011 Letter.pub - Oklahoma Hemophilia Foundation

HemOK news
Summer 2011
The OHF Annual Meeting will be held July 29 & 30 at the Tulsa Southern Hills
Hilton, 7900 S. Lewis Ave.
The meeting will start Friday at 4:00 PM with vendor displays, followed by dinner at 6:30. Camp highlights, scholarship, and award presentations will follow
dinner.
Saturday's meeting will include many national speakers, special teen programming, outside activities for children 7 to 12 years of age, on site programs for
kids 3 to 6 years old and childcare for children age 2 and under.
We are one of 6 chapters who have been selected to host LA Kelley Communication's "Pulse on the Road" insurance symposium, This informative presentation covers a variety of healthcare reform topics of urgent importance to families with bleeding disorders. Other Saturday presentations will include: Partnering with Your Child's School, Dad's in Action, NHF's Victory for Women, and
Stages in a Child's Bleeding Disorder.
Teen programming will include Transitions to Adulthood, Real Life Hemophilia, and a service project of preparing “care packages” and letters for Oklahoma
troops stationed in combat zones, followed by an afternoon at Big Splash. Children ages 7-12 will go to the Tulsa Air and Space Museum and Planetarium in
the morning and to Big Splash in the afternoon. On site programming will include arts and crafts, costume making, and other activities for children 3 to 6
years old.
INSIDE
This Issue
Patient Notification
Collaborative Drug
Study for Hemophilia
Camp Independence
Health Reform News
Alert
Genome Editing
Kid’s Corner
Medical IDs Save Lives
Hotel rooms will be provided for OHF members and their immediate family
who live more than 25 miles outside of the Tulsa metro area.
You can register by filling out the registration form that was attached to an email sent out on 6/28, or by returning the registration form that was sent out
via U.S. mail on 6/29. You can also register by phone by calling the OHF office
at (405) 463-6634.
We look forward to seeing you at the meeting.
Please note that due to space and room availability, no reservations for the
meeting will be accepted after July 21.
720 W. Wilshire Blvd.
Suite 101B
Oklahoma City, OK 73116
(405) 463-6634
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Patient Notification System
Board of Directors
Officers:
Nathan Holloway-President
Gabe Bass-Vice President
Kim Pearce-Treasurer
The industry-wide Patient Notification System informs people when a blood product is withdrawn or recalled.
Everyone should register with the Patient Notification System so they can be directly
notified when adverse conditions occur with blood products.
The system is being administered by the National Notification Center. The identity
of those individuals who have registered is completely confidential.
Participating companies involved in the system are:
• Alpha Therapeutics
Kari Monholland-Co-Secretary
Darla Williams-Co-Secretary
Directors:
• Baxter Healthcare
• Bayer Corporation
• CSL Behring
• Genetics Institute
• The American Red Cross
Kerri Crabtree
Elizabeth Oliver
Randy Tangco
Each person who registers with the Patient Notification System will select how they
wish to be notified—telephone, express delivery letter, fax or e-mail. All participants
will then receive a follow-up first-class letter back-up. Participants can also select
which products they wish to be notified about.
The system is available by telephone for updates and news at 800-UPDATE-U. There
is also a Web site at www.patientnotificationsystem.org.
Register with the Patient Notification System now!
• by phone: 888-873-2838 (888-UPDATE-U)
• online Enrollment: www.patientnotificationsystem.org
• fax registration forms to: 1-800-442-2906
• mail registration forms to:
Mission Statement
National Notification Center
20 N. Meridian Street, Suite 300
Indianapolis, IN 46204
The mission of the Oklahoma Hemophilia Foundation
is to improve today’s quality of life and tomorrow’s
opportunities for those affected by a bleeding disorder
through education, advocacy, and awareness..
Register now to be directly notified of a recall or withdrawal. Stay informed!
*reprinted from www.hemophilia.org
The Oklahoma Hemophilia Foundation is a
501 (c) (3) non-profit organization.
The OHF neither recommends nor endorses the
products in this publication and does not make recommendations concerning treatment regimens for
individuals. The OHF suggests that you consult your
physician or treatment center before pursuing any
course of treatment. This publication is for general
information only.
Look for us on the web
www.okhemophilia.org
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Brandeis University (MA)
June 23, 2011
Collaborative develops drug that may help hemophilia patients
Would free patients of many hassles and health risks
By Susan Chaityn Lebobvits
A razor nick in the shower? A fall on a slippery floor? The average person simply calls it bad karma, but for people with hemophilia such episodes can cause
prolonged blood loss.
Hemophilia is an inherited bleeding disorder in which a particular protein required for blood to clot is missing or is present at an insufficient level. Currently
there is no long-acting hemophilia agent, meaning that patients must either take an injection of the protein in reaction to a bleed, or have intravenous infusions —a method of delivering drugs — two to three times a week to maintain the clotting medication in the bloodstream.
But thanks to collaborative work involving the laboratory of Neil Simister, an associate professor of molecular biology at Brandeis, and doctors at Children’s
Hospital and Brigham and Women’s Hospital, hemophilia patients may soon have access to long-acting clotting medications that free them from the hassles of
frequent infusions and from worries that an internal bleed has gone unnoticed.
Simister’s laboratory, as well as labs at the University of Texas and Ohio State University, independently and simultaneously discovered that the receptor
FcRn protects antibodies from breaking down in the body. Building on that basic discovery has enabled the formulation of new hemophilia drugs.
One of them, Factor IX Fc fusion protein (rFIXFc), for hemophilia B, is expected to finish Phase III clinical trials by early 2012 then be filed for FDA approval.
The idea behind rFIXFc is genetic engineering of sorts. If you have a therapeutic protein that you want to last a long time in the blood — in this case a clotting
factor — you can fuse it to the part of the antibody (called Fc) that will bind to the protective receptor, FcRn.
Since antibodies last a long time in the blood relative to other proteins, this enables the therapeutic protein clotting factor to be protected by FcRn and, as Simister says, enjoy the same protection as an antibody.
Factor IX Fc fusion protein was created by Syntonix Pharmaceuticals, a company formed in 1998 based on breakthrough discoveries from the laboratories of
Simister, Dr. Wayne Lencer of Children’s Hospital and Dr. Richard S. Blumberg at Brigham and Women’s. The labs had been collaborating on the study of
FcRn as an antibody transporter in human cells. Biogen Idec bought Syntonix in 2007. It is the first company spun out of Brandeis to be acquired by a large
biotech firm.
Approximately one in 5,000 males born in the United States has hemophilia A, which is caused by mutations in the factor VIII gene. About one in 25,000 males
are born with hemophilia B, which is due to mutations in the factor IX gene.
Simister says that many of the problems that occur for people with hemophilia come from internal bleeds that just happen because of the rough and tumble of
life. Little bleeds that occur in muscles and joints stop very quickly for those not affected, but people with hemophilia are often unaware — say from a seemingly innocuous fall — and over time will develop muscle and joint damage.
“Because you don’t know that you’ve had a bleed you don’t know to take the clotting factor on demand,” says Simister. “Prophylaxis for people with hemophilia keeps a good protective level of these clotting factors in their blood.”
He adds that people are much more likely to be compliant with preventative therapy, which is now standard of care, if it’s only once a week.
“It’s probably considered one of the most important pipeline products at Biogen,” says Laur Blumberg ’83, one of the Syntonix founders and brother of Richard S. Blumberg, one of the researchers. “It’s contributing billions of market capital to the company.”
In early 2008, Biogen Idec filed an application to begin clinical trials of rFIXFc for the treatment of hemophilia B.
“What’s really exciting is that we’re getting to an ultimate moment for this little company’s [Syntonix's] technology,” says Blumberg. “For biotech companies
to make it to Phase III and near registration is very rare.”
Blumberg estimates that they are probably less than 12 months away from getting definitive Phase III results for both hemophilia A and B programs. The next
step would be filing for FDA approval.
“A lot of people have to do basic research for some fraction of the discoveries that they make to be useful in some way to people,” says Simister. “To be able to
see that progression, from something that was a series of basic discoveries in the lab going through to clinic, is great.”
*reprinted from NHF News Briefs June 24-30 2011
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Camp Independence- A Hero's Journey
62 kids from OHF and 5 foreign students from Southern Nazarene University had a great time at this year's Camp Independence, which was held from
June 7-11 at Camp Bond in Tishomingo, OK.
Each camper was individually announced , introduced, applauded and welcomed by the entire camp staff upon their arrival into the great hall. Who
would have believed that we soon would have all of the Camp counselors, assistant counselors, junior counselors and all of the campers up and dancing
to the “Cupid Shuffle” to kick off the activities? Or that another great starting activity would be diving into a mud pit for a game of kickball?
The wide range of activities throughout camp included: fishing, archery, arts and crafts, musical sessions, Knight's of the Roundtable, GaGa dodge ball,
swimming in the pool and creek, medical instruction and infusion lessons, broom ball, human chess, an outstanding service project, a 20 mile bike ride,
Native American dancers and a talent show at the Hero's ball.
Catch and release was practiced at the fishing pond where large numbers of sunfish, bass and catfish were caught by the campers.
A special GaGa pit was built on site and the game was introduced to the campers. The game quickly became a favorite activity for many of the campers.
The campers showed off their artistic skills creating ceramics and painting at arts and crafts. They also had the opportunity to help paint and recreate a
mosaic of Van Gogh's famous Starry Night painting, which will be displayed at this year's Annual meeting. The mosaic will also be used as an auction
item at a future event.
Several campers caught on to the archery lessons very quickly and proved to be excellent archers.
Musical activities included harmonica lessons, guitar lessons and a session called “Stomp” where the campers filled the great hall with the rhythmic
sounds of drums.
Continued: See Camp on page 6
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Health Reform News Alert – June 29, 2011
Bipartisan appeals panel upholds federal mandate that everyone buy health insurance
A three-judge panel for the Sixth U.S. Circuit Court of Appeals in Ohio became the first federal appellate court today to uphold the
constitutionality of the Affordable Care Act mandate that all Americans purchase health insurance coverage.
The 2-1 decision is particularly significant as it was joined by Judge Jeffrey Sutton, a conservative jurist appointed by President
George W. Bush who has previously stoked controversy for rulings that narrowed federal protections available to persons with
disabilities. Judge Sutton rejected the argument that the purchase of health insurance constitutes “inactivity” that cannot be regulated by the federal government. Instead, he became the first Republican-appointed federal judge to conclude that self-insuring is
economic activity that is within the bounds of Congress’ historically-broad power to regulate interstate commerce.
Judge Sutton’s opinion followed the decision written by Judge Boyce Martin, who was appointed by President Carter. The majority
decision held that “the unique aspects of health care…make all individuals active in this market” and that an individual mandate
was a constitutional mechanism to prevent “free riders” who can afford to purchase health insurance but instead force taxpayers to
subsidize their health care when they inevitability get sick or injured. However, Judge Sutton included the caveat that the court
could revisit the issue once the full impact of the individual mandate becomes clear after its 2014 implementation.
Judge Michael Graham, a Reagan appointee, was the lone dissenter. He argued that upholding the individual mandate would effectively make Congress’ interstate commerce power boundless.
The case was brought by the conservative Thomas More Law Center and four individuals, who can now appeal to the full Sixth
Circuit and ultimately the U.S. Supreme Court. Three-judge panels for the Fourth and 11th Circuits are expected to render decisions
in July.
Source: PSI - Government Relations
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Camp Independence—A Hero’s Journey
Continued from page 4
A meal of Turkey legs and potatoes was served without any silverware, around a roundtable setting, complete with “candlelight” and goblets during
the Knights of the Roundtable. Campers cheered on their favorites during a realistic mock sword fight between the Blue Knight and Red Knight. The
two knights saluted each other and embraced as friends at the end of the program.
Each cabin carried their torch to the outside stage where they were honored to witness an amazing presentation by Native American Fancy Dancers.
Valuable lessons were shared about the history and meaning of each dance by the narrator. At the end of the presentation, the campers and staff
joined the dancers for a dance around the camp fire.
Sixteen teens, junior counselors, assistant counselors and counselors braved the heat to participate in a 20 mile bike ride. Congratulations to all of
those who participated and especially to those who finished this difficult event.
Our junior counselors participated in a service project by shopping for much needed school supplies for the Mill Creek school district. After purchasing everything from copy paper, Kleenex, markers and even basketball nets, the junior counselors went to the school and presented the supplies to
the school superintendent and some of the teachers. Our young adults were warmly received and all were very well-spoken when the superintendent
asked each individual about their bleeding disorder.
As always, Lah-Lah and medical instruction by the Hemophilia Treatment staff was an integral part of camp. Thanks to the medical staff for their
involvement in all of the camp activities and congratulations to the campers who learned to self-infuse.
The last night highlight was a the Hero's Ball. Campers had to earn their invitations to the ball by performing good deeds and unselfish acts. Each
cabin did at least one skit during the talent show at the Hero's Ball, and several campers also performed individual skits. The skits were creative, fun,
and appreciated by fellow campers who enthusiastically supported and applauded each skit.
Our camp continues to grow and we look forward to another great camp next year. We cannot express enough gratitude for the unselfish commitment of time and effort by all of the people who work so hard to make camp special for the participants. Thanks to our planning committee, counselors, activity leaders, medical staff and the Camp Bond staff. A special thank you to our junior counselors, Michael Lacey, Paige Budder, Megan
Hagerdon, Joseph Driver and Scott Driver and our assistant counselors, Robert Driver, Micah Morgan and Koura Saul and Kaleb Lewis. The efforts of
these future leaders of the OHF community were greatly appreciated and we look forward to expanding their roles next year.
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Genome Editing, a Next Step in Genetic Therapy, Corrects Hemophilia in Animals
Released: 6/24/2011 2:45 PM EDT
Embargo expired: 6/26/2011 1:00 PM EDT
Source: Children's Hospital of Philadelphia--Children’s Hospital of Philadelphia Study Advances New Strategy for Gene Therapy--
Newswise — Philadelphia, June 26, 2011 – Using an innovative gene therapy technique called genome editing that hones in on the precise location
of mutated DNA, scientists have treated the blood clotting disorder hemophilia in mice. This is the first time that genome editing, which precisely
targets and repairs a genetic defect, has been done in a living animal and achieved clinically meaningful results.
As such, it represents an important step forward in the decades-long scientific progression of gene therapy—developing treatments by correcting a
disease-causing DNA sequence. In this new study, researchers used two versions of a genetically engineered virus (adeno-associated virus, or AAV)
—one carrying enzymes that cut DNA in an exact spot and one carrying a replacement gene to be copied into the DNA sequence. All of this occurred in the liver cells of living mice.
“Our research raises the possibility that genome editing can correct a genetic defect at a clinically meaningful level after in vivo delivery of the zinc
finger nucleases,” said the study leader, Katherine A. High, M.D., a hematologist and gene therapy expert at The Children’s Hospital of Philadelphia. High, a Howard Hughes Medical Institute Investigator, directs the Center for Cellular and Molecular Therapeutics at Children’s Hospital, and
has investigated gene therapy for hemophilia for more than a decade.
The study appeared online today in Nature.
High’s research, a collaboration with scientists at Sangamo BioSciences, Inc., makes use of genetically engineered enzymes called zinc finger nucleases (ZFNs) that act as molecular word processors, editing mutated sequences of DNA. Scientists have learned how to design ZFNs custommatched to a specific gene location. ZFNs specific for the factor 9 gene (F9) were designed and used in conjunction with a DNA sequence that restored normal gene function lost in hemophilia.
By precisely targeting a specific site along a chromosome, ZFNs have an advantage over conventional gene therapy techniques that may randomly
deliver a replacement gene into an unfavorable location, bypassing normal biological regulatory components controlling the gene. This imprecise
targeting carries a risk of “insertional mutagenesis,” in which the corrective gene causes an unexpected alteration, such as triggering leukemia.
In hemophilia, an inherited single-gene mutation impairs a patient’s ability to produce a blood-clotting protein, leading to spontaneous, sometimes
life-threatening bleeding episodes. The two major forms of the disease, which occurs almost solely in males, are hemophilia A and hemophilia B,
caused respectively by a lack of clotting factor VIII and clotting factor IX. Patients are treated with frequent infusions of clotting proteins, which are
expensive and sometimes stimulate the body to produce antibodies that negate the benefits of treatment.
In the current study, the researchers used genetic engineering to produce mice with hemophilia B, modeling the disease in people. Before treatment,
the mice had no detectable levels of clotting factor IX.
Previous studies by other researchers had shown that ZFNs could accomplish genome editing in cultured stem cells that were then injected into
mice to treat sickle cell disease. However, this ex vivo approach is not feasible for many human genetic diseases, which affect whole organ systems.
Therefore the current study tested whether genome editing was effective when directly performed in vivo (in a living animal).
High and colleagues designed two versions of a vector, or gene delivery vehicle, using adeno-associated virus (AAV). One AAV vector carried
ZFNs to perform the editing, the other delivered a correctly functioning version of the F9 gene. Because different mutations in the same gene may
cause hemophilia, the process replaced seven different coding sequences, covering 95 percent of the disease-carrying mutations in hemophilia B.
Continued: see Genome on page 15
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Medical IDs Could Save Your Life
Can Medical IDs be functional and fashionable?
By Kadesha Thomas | 05.23.2011
For Robyn Salk, Saturday night is a time to dress up—perhaps a sequined blouse paired with slick, knee-high boots wrapped around a pair of skinny jeans.
The penchant for fashion didn’t change when Salk, 38, from Boca Raton, Florida, decided to purchase a medical identification bracelet.
Salk was diagnosed with mild hemophilia B in 2003. She says no one had told her she should be wearing medical identification for emergencies until two
years ago at a Florida Hemophilia Association symposium in Deerfield Beach. The bracelet she ordered had black and silver beads—perfect for any outfit, she
thought.
But during an emergency room visit, her husband, Jonathan, tapped a nearby emergency medical technician (EMT) on the shoulder. Raising Salk’s wrist, he
asked, “Would you know this was a medical ID bracelet?” “Probably not,” the EMT said, waving his colleague over. Both agreed they would have ignored the
fancy bracelet as just a fashion accessory.
“That was an eye opener,” Salk says. “I realized my husband was right. I need to not worry about being fashionable, but about being alive.” Days later, her
new bracelet—a silver chain with her medical information engraved on a heart-shaped tag—arrived in the mail.
Guidelines for Wearing Medical IDs
Saving lives during medical emergencies is exactly why the National Hemophilia Foundation (NHF) encourages everyone who has a bleeding disorder to
wear some form of medical ID. In a car accident or other medical emergency, medical personnel need to know if an unconscious injured person has a bleeding
disorder.
“It changes the response to treatment in a lot of ways,” explains Jocelyn Bessette Gorlin, RN, CPNP, a nurse practitioner at Children’s Hospitals and Clinics of
Minnesota in Minneapolis. “Ideally, the person with the bleeding disorder might get the first response, and the hospital would know to give factor to stop the
bleeding before doing a CT scan. This is especially important if the person needs surgery and particularly important with head injuries.”
Despite the importance, Gorlin discovered troubling results during a survey of nearly 100 nurses she completed in 2009 on the use of medical IDs. She found
that no official guidelines existed and that parents of children with bleeding disorders rarely used medical IDs for their infants. Many of the nurses and EMTs
surveyed had already encountered situations in which a person who had been shot was taken in for surgery without factor or someone died after being infused with the wrong factor.
Gorlin and NHF’s Nursing Working Group developed a set of clinical guidelines that the NHF Board adopted in November 2010 as MASAC document #201,
“Recommendation on Use of Emergency Medical Identification Devices for Children.” The guidelines recommend that medical IDs be worn on the person’s
body, displaying an easily visible and familiar medical symbol, such as the caduceus, which has two serpents intertwined around a winged staff, or the staff
of Asclepius, which has one serpent wrapped around a staff. The Nursing Working Group recommends medical IDs display the person’s diagnosis, factor
deficiency and emergency contact.
The risk of choking is one of the main reasons parents often do not use medical IDs for their infants. The guidelines encourage parents to avoid medical IDs
with beads, charms or other detachable parts that a child could easily swallow. Gorlin also says that not only can the more fashionable medical IDs be overlooked, but they also tend to break or come off during regular activity. For children younger than 1, the guidelines recommend placing the emergency ID on
the child’s ankle.
Consumers Demand More Flair in Products
In the early 1990s, consumers began demanding medical identification styles with more flair than the plain metal bracelet and information tag, says
Ramesh Srinivasan, MedicAlert Foundationｮ’s senior vice president of marketing and business development. Jewelry companies entered the market, designing bracelets, anklets, sports bands and dog tags. “There’s always a fine balance to strike between style and functionality,” Srinivasan
says. “But you don’t want vanity to prevail.”
Continued: see Medical IDs on page 14
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Go Paperless
Want to reduce the amount of clutter in your
life and help the environment? Consider going
paperless by receiving your copy of the HemOKNews via email. Interested? Send your
email address to the OHF office at bgoodley@okhemophilia.org and start receiving your
newsletters and other OHF information electronically.
Interested in volunteering??? Call
the OHF office at (405) 463-6634
to find out how you can help!!
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Medical IDs Could Save Your Life
Continued from page 12
Most of MedicAlert’s bracelet and necklace styles feature a stainless steel, gold or silver chain and information plate with lettering in myriad colors. Other styles
include colorful beaded bracelets, sports bands and watches. “We’ve come a long way, but what has remained consistent is the emblem and the quality of the
information engraved,” Srinivasan says. The metal plate on all of MedicAlert’s products has the staff of Asclepius flanked by the words “Medic” and “Alert” on
the front.
MedicAlert requires all customers to register as members, so that clinical staff can review their medical information before it’s engraved on the metal plate. In an
emergency, the first responder can call a live emergency response service, 24/7, for more specific information on the member. The member’s family contacts are
also notified of the incident.
Medical ID Jewelry Designs Gaining in Popularity
The metal plate with one of the two medical symbols is also a mainstay on the designs offered by American Medical ID.ｮ The Texas-based company’s offerings
include mostly metal bracelets, necklace pendants, sports bands and dog tags. Some of the dog tags even double as USB drives. These portable devices can be
connected to most computers to save and transfer important medical information.
Toni Bissell, president and founder of the medical jewelry company N-Style ID,™ says she often does what Salk’s husband did—she takes new designs to her
local emergency medical personnel to ask their opinion. A creative design, however, is still important. “If people don’t like them, especially children, they will
not wear them,” Bissell says. “When children and teens have a medical condition, they may already feel like an outcast. They want to wear something that everyone else might want. That actually makes them feel proud. I’ve had parents say, ‘I bought this for my child, and now the other one wants one.’”
The Right Medical ID Style for You
Joe Mileson, 15, a high school sophomore from Spokane, Washington, opted for a cotton sports band to identify his diagnosis, severe hemophilia A. His band is
decorated with music notes, a nod to his love of singing. He once tried a necklace, but says it felt weird and he would take it off to go swimming. His next style,
a watch, pinched his arm.
Mileson knows other teenagers with chronic conditions who have gone as far as having their diagnoses tattooed on their bodies. People with bleeding disorders
who are considering a tattoo should discuss the issue with their hemophilia treatment center first. Beyond any health concerns, tattoos may not be that effective.
“The tattoo would have to be very large to put the necessary information, and that information might change,” says Gorlin. “And the tattoo might fade over
time.”
Mileson agrees tattooing is not necessary, especially with so many styles of medical IDs available. “There’s a style for everyone,” he says. He eventually settled
on the sports band because it is versatile enough to be worn during choir practice and lifeguard training drills without coming off.
Medical IDs for Small Children
While some teens and adults choose the medical IDs based on style, parents with small children may be more concerned about making sure it survives their
child’s curiosity. Tiffany Intal’s daughter Annika, 2, was diagnosed at birth with severe factor VII deficiency. “I saw the medical ID jewelry as a form of insurance,” says Intal, a medical assistant in Daly City, California, and hemaware.org’s Diary of a HemoMom blogger. “You buy insurance for your house, for your
car and for all the other ‘what ifs.’ If I’m unconscious during an emergency, or if someone can’t speak on her behalf, then she’ll have the medical ID jewelry.”
A necklace or bracelet was out of the question, because Annika probably would have tried to chew them off. So, at the NHF Annual Meeting in 2009, Intal purchased a blue beaded anklet for Annika. At first, it sent Annika into a fit when Intal tried to put it on. But in the past year, the little girl has become accustomed
to wearing it, although she still fiddles with it and manages to kick it off during tantrums.
Intal says most clinicians notice the anklet, but the most common reaction from strangers, is, “That’s so pretty.” Intal uses such comments as an opportunity to
educate and to clarify myths about bleeding disorders, such as the belief that little girls cannot have them. “People usually seem very receptive to the information.”
Now, Intal’s two older boys, almost ages 8 and 6, who do not have a bleeding disorder, want medical IDs, too.
*reprinted from May 2011 HemAware
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MASAC Document #201
The following recommendations were approved by the Medical and Scientific Advisory Council (MASAC) of the National Hemophilia Foundation on
November 13, 2010, and adopted by the NHF Board of Directors on November 14, 2010.
MASAC RECOMMENDATION ON USE OF EMERGENCY MEDICAL IDENTIFICATION DEVICES FOR CHILDREN
The Nursing Working Group (NWG) conducted a survey of use of Emergency Medical Identification (EMI) devices available for children and found
that there are no devices appropriate for infants and young children under 3 years of age. In addition, the number of children wearing these devices is
low in all age groups. Based on their study, the NWG has established the following guidelines for use of EMI devices in children:
•
bag.
child.
For all age groups, EMI should be worn on the body instead of externally such as in a wallet, on a car seat, or in a caregiver’s purse or diaper
An emergency medical identification card placed in one of those locations can accompany the child, but this should not replace EMI on the
•
The EMI should be identified with a medical symbol (i.e. Caduceus) and look like an EMI so it will not be mistaken for a piece of jewelry.
•
The EMI should be fastened securely but not tightly and should be assessed frequently to insure that the fit is snug but not tight.
•
Presently there is no ideal EMI for infants under 1 year of age.
•
All EMIs placed on a child<3 years old which have emblems or clasps <1 3/4 inches should carry the warning: “Not intended for children < 3
years old due to choking hazard.”
•
These are general guideline to be adapted to meet the needs of each child.
NHF does not endorse any specific brand of EMI, however being affiliated with a 24- hour on call nonprofit program is encouraged.
Genome Editing, a Next Step in Genetic Therapy, Corrects Hemophilia in Animals
Continued from page 9
The researchers injected mice with the gene therapy vector, which was designed to travel to the liver—where clotting factors are produced. The mice
that received the ZFN/gene combination then produced enough clotting factor to reduce blood clotting times to nearly normal levels. Control mice
receiving vectors lacking the ZFNs or the F9 minigene had no significant improvements in circulating factor or in clotting times.
The improvements persisted over the eight months of the study, and showed no toxic effects on growth, weight gain or liver function, clues that the
treatment was well-tolerated.
“We established a proof of concept that we can perform genome editing in vivo, to produce stable and clinically meaningful results,” said High. “We
need to perform further studies to translate this finding into safe, effective treatments for hemophilia and other single-gene diseases in humans, but
this is a promising strategy for gene therapy.” She continued, “The clinical translation of genetic therapies from mouse models to humans has been a
lengthy process, nearly two decades, but we are now seeing positive results in a range of diseases from inherited retinal disorders to hemophilia. In
vivo genome editing will require time to mature as a therapeutic, but it represents the next goal in the development of genetic therapies.”
Support for this work came from the National Institutes of Health and the Howard Hughes Medical Institute. High’s co-authors were from The Children’s Hospital of Philadelphia, the University of Pennsylvania, and Sangamo BioSciences, Inc. of Richmond, Calif.
“In vivo genome editing restores hemostasis in a mouse model of hemophilia,”Nature, published online June 26, 2011. doi: 10.1038/nature10177
Oklahoma Hemophilia Foundation
720 Wilshire Blvd. Suite 101B
Oklahoma City, OK 73116