I travel a lot, and so often miss the events that happen in hemophilia close to home. So I was thrilled to know I could attend the New England Hemophilia Association (NEHA)’s fundraising event this past weekend. And it wasn’t a golf tournament (never touched a club in my life) or a cooking class (disaster! Cooking, me?). It was murder.
Murder? Yes! A real-life game of Clue, based on the board game and movie. The movie is hysterical (thanks to actor Tim Curry) as our evening would prove to be!
Doug and I drove to a stunning mansion, just south of Boston, that on a cold dark night could really double as a creepy old haunted house. The massive entrance was crowned not with a statue of an angel, saint or cross, but of a devilish-looking, stone ram’s head, with blank, staring eyes. Inside, a wide-angle view of a spiraling, carpeted staircase, chandeliers, portraits of deceased notables, mounted hunting trophies… and the friendly NEHA staff ready to register us.
There was wine, beer, plenty of great company in seeing the families I’ve known for ages, and meeting some new ones. After a social hour, we had a delicious buffet as members of the cast started to appear, in character, sporting British accents. Professor Plum, Miss Scarlett, Mr. Green, Mrs. Peacock, my favorite—Colonel Mustard, Mrs. White, and the Detective from Scotland Yard. But let’s not forget Mr. Boddy, who unfortunately could not make dinner… because he was dead!
Who did it? That was our task. We had to search the mansion for clues to the murderer, and find the murder weapon: a pistol, lead pipe, candlestick, rope, wrench, or knife. It was fun to be with the cast while we prowled about looking under tables, behind curtains and up fireplaces! Everyone got into the act.
This was a fun fundraiser, but it’s also serious business. There are deep concerns in our community about sourcing funds to keep the nonprofits going. Budgets are tighter as companies wait on new drug developments to see how market share will be affected. At HFA in April, all the talk was about how funding was getting harder to secure. And I just noticed this week that NHF’s agenda for the October meeting has a lot of sessions on fundraising.
Well, Rich Pezzillo, executive director and person with hemophilia, and team did it right this time. They raised about $10,000 in one evening, all the while giving everyone a great time, a silent auction, and an arrest!
And something new: a couple at my table was unfamiliar to me so I asked if they had a child with hemophilia. No, they knew nothing about hemophilia. They saw a brochure for the event at their local bank, wanted a fun night out, and decided to attend. They were amazed at how much they learned about hemophilia. And they left with new friends and a silent auction item! (But those Red Sox tickets were mine)
So fun + prizes + game (+ murder) + out-of-the-box-thinking + inviting strangers = outstanding event and fundraiser.
Congratulations to Rich and his team for a great event. So what if it was a murder? It was a bloody good fundraiser and a bloody good time.
Who better to represent what it means to “Just B” with hemophilia than the people who are defining it in their daily lives? That’s why today, I’m excited to be one of the first to feature some of the new faces from our community who are joining the promotional campaign from Aptevo, makers of the factor IX treatment, IXINITY® [coagulation factor IX (recombinant)].
Keep reading to see if you can spot anyone you recognize, plus watch these community members share their inspiring stories at IXINITY.com.
This is a paid public announcement from Aptevo and does not constitute an endorsement of products or services. When you click on the links in this blog entry, you will be directed to the IXINITY website. LA Kelley Communications always advises you to be a savvy consumer when contacting any company; do not reveal identifying information against your will.
Just B: Because Life Is Bigger Than Hemophilia B
Our current promotional campaign is inspired by everyone redefining what it means to have hemophilia B by living life on your own terms and Just B-ing yourself.
And now, we couldn’t be more proud to introduce the next generation of community advocates sharing how they can Just B with IXINITY.
Watch these new patient stories and others at IXINITY.com, plus keep an eye out for even more fresh faces to be featured over the next few months.
Joe spent years struggling to find the bleed control he was searching for, until he heard about a clinical trial for a new recombinant product—which was eventually approved as IXINITY. Now he says, “Since switching to IXINITY, I have not yet had a breakthrough bleed. Overall, it really comes down to coverage for me. Coverage means that I have one less thing to worry as much about, that I can make plans, try different things, and know that I can rely on IXINITY when I need it.”
In a clinical trial, 71% of bleeding episodes were resolved with 1 infusion and 13% were resolved with 2 infusions. Individual response may vary.1
This information is based on Joe’s experience. Different patients may have different results. Talk to your doctor about whether IXINITY may be right for you.
With a history of breakthrough bleeds, Nathan felt like he couldn’t live the life he wanted while managing his hemophilia B. After doing his research and talking to his doctor about IXINITY, he says, “Since switching, I’m more willing to just jump in and go because I’m confident that I’m covered with IXINITY. I’m even more open about having hemophilia because I’m not feeling held back by it.”
In a clinical trial, 71% of bleeding episodes were resolved with 1 infusion and 13% were resolved with 2 infusions. Individual response may vary.1
This information is based on Nathan’s experience. Different patients may have different results. Talk to your doctor about whether IXINITY® may be right for you.
IXINITY INDICATIONS AND IMPORTANT SAFETY INFORMATIONWhat is IXINITY®?
IXINITY [coagulation factor IX (recombinant)] is a medicine used to replace clotting factor (factor IX) that is missing in adults and children at least 12 years of age with hemophilia B. Hemophilia B is also called congenital factor IX deficiency or Christmas disease. Hemophilia B is an inherited bleeding disorder that prevents clotting. Your healthcare provider may give you IXINITY to control and prevent bleeding episodes or when you have surgery.IXINITY is not indicated for induction of immune tolerance in patients with hemophilia B.IMPORTANT SAFETY INFORMATION for IXINITY®
You should not use IXINITY if you are allergic to hamsters or any ingredients in IXINITY.
You should tell your healthcare provider if you have or have had 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 hamsters, are nursing, are pregnant or planning to become pregnant, or have been told that you have inhibitors to factor IX.
You can experience an allergic reaction to IXINITY. Contact your healthcare provider or get emergency treatment right away if you develop a rash or hives, itching, tightness of the throat, chest pain, or tightness, difficulty breathing, lightheadedness, dizziness, nausea, or fainting.
Your body may form inhibitors to IXINITY. An inhibitor is part of the body’s defense system. If you develop inhibitors, it may prevent IXINITY from working properly. Consult with your healthcare provider to make sure you are carefully monitored with blood tests for development of inhibitors to IXINITY.
If you have risk factors for developing blood clots, the use of IXINITY may increase the risk of abnormal blood clots.
Call your healthcare provider right away about any side effects that bother you or do not go away, or if your bleeding does not stop after taking IXINITY.
The most common side effect that was reported with IXINITY during clinical trials was headache.
These are not all the side effects possible with IXINITY. You can ask your healthcare provider for information that is written for healthcare professionals.
For more information about IXINITY, please see full Prescribing Information, including Important Patient Information.
You are encouraged to report side effects of prescription drugs to the Food and Drug Administration. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.
Reference: 1. IXINITY [coagulation factor IX (recombinant)] prescribing information. Berwyn, PA: Aptevo BioTherapeutics LLC; April 2018.
Aptevo BioTherapeutics LLC, Berwyn, PA 19312IXINITY [coagulation factor IX (recombinant)] and any and all Aptevo BioTherapeutics LLC brand, product, service and feature names, logos, and slogans are trademarks or registered trademarks of Aptevo BioTherapeutics LLC in the United States and/or other countries.
This week is a very special: it’s the 100th anniversary of the death of the last Russian royal family, the end of the Romanovs. Special because not only did it lead to a massive change in world history, but it was also the death of the little prince with hemophilia, Alexis.
Hemophilia appears to have played a vital role in the story of the Bolshevik takeover of Russia, and subsequent spread of Communism. In Nicolas and Alexandra by Robert K. Massie* Tsar Nicholas II, though not a very clever leader to begin with, is so distracted by his son’s suffering that he unwisely allows the “mad monk” and faith healer Rasputin to gain access to the inner circles of the royal family, while at the same time ignoring and not heeding the needs of his people for food and better quality of life. Rasputin is able to influence the course of events as Russia is battling the first World War, and yet also appears to have a calming effect on Alexis during his terrible bleeds.
There are no spoilers: everyone knows how this story ends. The entire family—Nicolas, wife Alexandra, daughters Olga, Tatiana, Anastasia and Maria, and son Alexis—was cloistered in Ekaterinburg under house arrest while the Bolsheviks take over. Although they are promised safe passage to England, where Nicholas’s cousin and doppleganger George V is on the throne, the family was instead slaughtered in the early morning hours of July 17, 1918. It was a horrific scene; the princesses did not die at once as they had about 18 pounds of royal jewels sewn into their clothing, to take to England with them.
Their bodies were eventually tossed down a mine shaft, which was not discovered until 1979, and even then not revealed until 1989, when the Russian archives were officially opened.
On August 15, 2000, the Russian Orthodox Church announced the canonization of the royal family. Alexis, the prince with hemophilia, would now be known as Saint Alexis.
And DNA tests would later reveal that the royal family, all the way back Queen Victoria, Alexis’s great-grandmother, had factor IX deficiency.
You can read more about this fascinating story hat changed word history by reading Nicolas and Alexandra, or one of many other books and on-line resources. I am happy to say that we are currently revising our children’s storybook called Alexis: The Prince Who Had Hemophilia, with new illustrations, done by a Romanian with hemophilia!
If you have hemophilia, this is a story you must know.
*The author is father of Bob Massie, currently running for governor of Massachusetts, who was born with hemophilia!
Five years sure went by fast. That was the last time I attended the Dominican Republic hemophilia camp “Yo Sí Puedo!” (Yes, I Can!). I don’t understand how the time slipped by, especially when I have such a deep attachment to the DR, which is only a 4-hour plane ride away. The DR was the first developing country I ever worked with, back in 1998, when there was no camp, and no factor. While Project SHARE supplied factor when we could, together with the fledgling Fundación Apoyo al Hemoílico (FAHEM) we created the DR’s first hemophilia camp. We were all new at this, so the first camp was a bit wild… but magical. While the camp grounds were trashed by Hurricane Georges, no one much minded when Red Sox pitcher Pedro Martinez, Houston Astros Moises Alou and Toronto Blue Jay Tony Fernandez showed up to autograph baseballs and play ball with the kids!
Those children I first met at camp are grown now, and many are serving as camp counselors. In fact, the one who was the quietest at that first camp, Brahian Valdez, is now the most extroverted, and has become Camp Director!
It was a joyous reunion in the parking lot of the Robert Reid Cabral Hospital in Santo Domingo. The teens were men, the little boys were teens, and a new generation of youngsters with hemophilia were present, wondering who this “gringa” was.
And then it felt like no time had passed. This was my ninth camp, so I knew everyone pretty well, except for the smallest ones and of course the newcomers. One of the joys of camp is that you get to see children grow and hopefully flourish through the years. While we have lost some to bleeds through the years, the majority are here, and have become best friends.
The theme was “Super Heroes,” perfect for this group. I could easily see during the four days of camp in the Lomas Lindas area outside of Santo Domingo that the older guys hobbled about on stiffened ankles, arthritic knees, and misaligned hips. The younger ones had no joint problems and dashed about madly, enjoying the excitement of camp and the freedom from their parents. After working with developing countries for 20 years now, this amazed me. Children without joint problems. Here, in the Dominican Republic.
Camp is now a highly structured program that mixes plenty of activities with quiet time and lectures for the 60 campers with hemophilia. Each day starts around 7; by 7:30 everyone is on the grass doing exercises, led by Cuchito, the coach. Blowing his whistle, Cuchito has the children stretch, do light exercises and then march in ever tightening circles, which ends in a crush and everyone cheering. Breakfast follows, which is always delicious. The food in the DR is superb! Then activities: it might be swimming in the pool, arts and crafts, or a game of ball that serves as teambuilding.
The pool is the favorite unstructured activity. Though they live on an island in the Caribbean, most of the children never get to spend time in water. The surf near the beaches in the capital is too rough. And the children come from very humble homes, where there is no access to a pool. Camp gives them a chance to enjoy water, which almost every child loves.
Camp founder Haydée de Garcia, who has an adult son with hemophilia, realized this and arranged for top-notch, young swim instructors to visit, and they gave each child swim lessons which mostly consisted of overcoming a fear of putting your head under water. Once that fear was conquered, the children enjoyed practicing floating and strokes.
The favorite activity, judging by the cheers and participation level, is wiffle ball. You must know that baseball is almost a religion in the DR! The DR sends more ballplayers to the US than any their country, and about 25% of all ball players in the leagues are from the DR. While we don’t use baseballs or softballs for safety reasons at camp, the kids and adults attack the wiffle ball as if it were! The games are exciting, loud, fun, funny and tiring!
This year we even had a bonfire just outside the camp facility, where we roasted marshmallows and everyone participated in some singalongs that ranged from traditional Spanish songs to rap!
A great presentation was made by Fendi, who was at the original camp in 1999. Wearing a Spider-Man mask, he talked about how the boys may have developed hemophilia: inherited, like WonderWoman; a mutation, like Wolverine; or acquired, like Storm. Very clever! And he stressed the need for community, like the X-Men, and proposed a program called Hermanos X (Brothers X), to keep this community strong.
The beauty of camp is abundant factor. Prior to about 2016 camp might be touch and go; you cannot have a camp if there is no factor, and the DR has struggled to get factor. SHARE always tries to assist and we stockpile factor starting in January for camp. A medical ward is set up before camp ever begins where everyone gets factor. This year? Everyone got factor prophylactically. How does that happen?
Well, we had our own superhero at camp this year. Bioverativ CEO John Cox visited the camp with his wife Ivette (who is Dominican) and his four children, to experience hemophilia camp in a developing country, and to see the results of his visionary idea (along with other visionaries; read about it here) to donate one billion IU of factor to the World Federation of Hemophilia (WFH) over 10 years. With this strategy, knowing there will be a steady flow of factor that doctors can count on, hundreds of children around the world in developing counties are now on prophy for the first time ever. These include “my” boys, here in the DR, here in camp. As I watched the older boys still hobble about, I watched the younger ones race and jump and climb, without fear, without arthropathy. It seems like a miracle, but it is in reality this vision come true—that with proper treatment, children with hemophilia can live full and active lives, in our countries and theirs.
The World Federation of Hemophilia, at Bioverativ’s request, donated all the factor to camp this year. No child had to worry about running out, which allowed them to run about!
And for the first time, WFH attended camp. Miquel Izquierdo, WFH regional director, arrived from Mexico to enjoy the camp—he himself has hemophilia! He fit right in and was beloved by all. He witnessed the great results of the factor donation, and also of Save One Life as about 16 enrolled beneficiaries attended camp, all looking great and doing well!
We all loved having outside visitors, and next year, for our 20th anniversary, I am hoping to entice more volunteers from the hemophilia community to attend, to see the needs, the progress, to make connections. Despite the overwhelming success of camp, we can do more. Yes, we can… and we will!
From John Cox:
“I want to thank you and Presidenta Haydée Benoit de Garcia for including my family in hemophilia camp in the Dominican Republic. We were so impressed with the camp and honored to be there. The Dominican boys were learning to deal with hemophilia in a poor country, and they were having fun too (the lively whiffle ball game was our favorite activity). It was clear that this camp was something these boys looked forward to all year. What struck me most was that Haydée and her counselors were teaching the boys to live a values-driven life no matter their circumstances. Haydee’s leadership and her love for these boys — as well as yours, Laurie — is clearly impacting generations of Dominicans living with hemophilia. My children walked away believing in the camp theme “Yes You Can!” (¡Yo Sí Puedo!). Thank you for all you do, Laurie, and for the role of Save One Life in impacting lives in the Dominican Republic and around the world.”
Thanks to John Cox and family for spending family and vacation time with us, to the WFH and Bioverativ for the factor donation, to uniQure for gifts for the children, to Haydée and team, for another great camp!
This article was originally published in PEN, August 2002, Vol. 12, issue 3
Summer is in full force and with it, trips to theme and amusement parks. I’m a huge lover of them, including the thrill rides, like roller coasters. In 2000-2002 there were many stories and studies in the news about the potential for brain bleeds, and these were about people without bleeding disorders. Last year came the first reported case of symptomatic bilateral subdural hematoma associated with riding a centrifugal motion simulator ride.
What does this mean for our kids (and big kids–adults!) who love to ride roller coasters and simulator rides? Have you asked your hematologist for his or her opinion? I decided to rerun one of my favorite articles from PEN 2002 about risks for kids with hemophilia at theme parks, written by Dr. Richard Lipton.
Summer is here, and millions of families will seek adventure and thrills at theme parks like Six Flags, Disney World or Universal Studios. What a wonderful day a family can have at a theme park! Kids and parents love the rides, water slides, entertainment and general excitement. Are there any special safety concerns or precautions for families with a child with hemophilia? Yes!
Think of a theme park as a very big playground, but with an atmosphere favoring less parental control—a setting that can lead to impulsive behavior by children.
Imagine yourself at a typical theme park. You’ll have general health concerns. Parks can be crowded, hot and sunny—so apply sunblock and drink plenty of water.
Theme parks have paved surfaces, harder than public playgrounds, and filled with children running. Your child needs to wear appropriate footwear. Flip-flops or Tevas might be suitable for water activities, but sneakers are safer for walking and running.
You’ll also have concerns specific to the theme park you visit. “Mind Eraser,” “Shockwave,” and “Nitro”—what about these special high-end rollercoaster rides? Riders are frequently subjected to changing speeds that result in “G-forces” similar to those experienced by trained, appropriately suited and restrained combat fighter pilots. Your child becomes “Top Gun” in shorts and a T-shirt! Parents should remember that any person can experience head trauma on these specialized rides.
How is the head affected by a ride like the Mind Eraser? Recall that your brain is surrounded by fluid; it is floating inside your skull. This arrangement cushions the brain, and reduces movement, protecting your brain from direct trauma and sudden shifts in skull position. It works quite well in our daily activities, and in automobiles (as long as we’re wearing a seatbeat). Now imagine speeding over the crest in a roller coaster. All of a sudden you’re weight- less, like an astronaut—this is called a Negative G- force. (You’ll have no trouble recognizing this moment, because every- body screams!) Then, after the coaster speeds down and resumes its climb, you feel your backside being pushed into the seat. It feels like the force of gravity has suddenly increased. This is a Positive G- force. Although your body is restrained, high G-forces could exceed the protective cushioning of fluid surrounding the brain, and could cause injury.
Interestingly, this year New Jersey became the first state to seek legal restrictions on the maximum allowable G-forces on amusement park rides. The regulations result from concerns raised by physicians about the association between neurologic damage and high G-forces on these rides. Certainly, such injuries occur very infrequently, but serve as a cautionary note to all riders of high-end roller coasters—with hemophilia, or without.
My advice? Take some precautions. Level the playing field by giving your child a prophylactic infusion of factor the morning of your visit to a theme park. Yesterday’s dose is not sufficient! Don’t wait until your child reports the symptoms of a bleed—it may be too late. Besides, your child is not going to report the flop he took running to the haunted house until you’re stuck in traffic on the long, long ride home.
Infuse first, then have a great time!
Dr. Richard Lipton was the physician in charge of the hemophilia treatment center at the Long Island Jewish Medical Center. As a United States Air Force Physician (1966–1968), Dr. Lipton knew several fighter pilots, who took him on “joy rides” (with lots of G-forces) that more than fulfilled his childhood fantasies. He is now retired.
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