Statement about Fitusiran from New England Chapters

The Connecticut Hemophilia Society (CHS), the Hemophilia Alliance of Maine (HAM), and the New England Hemophilia Association (NEHA), work to ensure that people affected by bleeding disorders and their families living in New England remain informed about important medical information that affects the community.

On Sept. 7, 2017, Alnylam Pharmaceuticals announced that it recently became aware of a fatal serious adverse event (SAE) that occurred in a patient with hemophilia A who was receiving fitusiran in the Phase 2 OLE study (clinical drug trial). As a result, Alnylam suspended dosing in all ongoing fitusiran studies pending further review of the safety event, and development of a risk mitigation strategy.  You can read a full press release here. Additionally, through conversations with Alynylam, we’ve been made aware that the SAE was an embolism (blood clot) in the brain. It is not yet known if this event is directly related to the patient’s treatment with fitusiran.

It is important to note that this product is not a ‘gene therapy’ medication, but rather modifies the expression of people’s healthy antithrombin, which causes thrombin to be increased by a subcutaneous (subQ) injection.

Times like these call our attention to the need and importance for community wide advocacy. We must be strong advocates for ourselves, and our communities.  We rely heavily on our therapy partners for future medical advancements and commend Alnylam on their transparency during this process. We take this moment to call upon our care providers and ask them to be diligent in their understanding and administration of drug trials as we all continue together towards a cure.  

If you have questions about this situation, please contact your Hemophilia Treatment Center (HTC), or your primary health care provider. Alnylam also has a hotline for patients to ask questions at: 1-866-330-0326. CHS, HAM, and NEHA will continue to monitor this case and will provide updates as they become available.   

Read the National Hemophilia Foundation’s (NHF) statement here.

 

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Travel Tips for Families Affected by Bleeding Disorders by Lianne Lapierre

As I take a look at my different social media outlets, I’m beginning to see that the time of year for travel, is upon us!! As the snow in Maine TRIES to melt, many of my friends and family have started posting pictures of airports, sand, sun, and THEME PARKS!

Having a child with Hemophilia, or any type of bleeding disorder, sometimes makes the word “travel” seem a little… scary. And then you throw in “theme park,” and our minds can sometimes spin into a whirlwind of anxiety. But have no fear!! DisneyHemoMom (shamelessly inserting my Twitter handle here, haha!) is here!!

We started flying (and visiting those incredible theme parks in Florida) when our son, who has severe Hemophilia, was about two years old, and honestly, we haven’t stopped since! And while some things have changed in the way we do things (especially now that he has some target joints), we’ve picked up some great tips about traveling, along the way.10

 

Air Travel Tips

  • Prior to travel, request a medical information travel letter from your Hemophilia Treatment Center or hematologist (if you don’t use an HTC) that you can bring with you. This letter typically lists your diagnosis along with a brief explanation of your condition, as well as an explanation that the vials of clotting factor are sterile and cannot be opened for inspection by TSA. Our HTC will also include a list of the local hospitals and HTC’s in the area that we will be traveling to, which is handy to have, just in case a trip to the Emergency Room unexpectedly gets added to your agenda!
  • Keep your medication and all supplies needed with you in your carry-on bag. If you put it in your checked luggage, there’s always a chance that your luggage will be lost, or delayed – and you definitely don’t want to be stuck on vacation without your medication!
  • Speaking of carry-on bags, one thing that we invested in years ago, was a medical travel bag JUST for our son’s clotting medication and supplies. Our son has had a port-a-cath since he was 14 months old, and we use this bag all the TIME! Even just for weekend road trips. Yes, it counts as one carry on item on the plane, but it’s nice to have everything all in one place. And it makes it super easy when TSA has to open it up to check what’s inside (apparently lots of vials of medication, syringes of saline and heparin, along with a ton of needles, seems to look a bit “suspicious” on the X-ray machine.)
  • Most airlines offer pre-boarding for families traveling with children or for passengers that need special assistance. If you are traveling with medical equipment, or small children, this perk is for YOU! We loved being able to pre-board when our son was smaller – especially during those early years, when we had his medical bag, diaper bag, and carry-ons to get situated before we could even sit down!
  • One thing we have learned (on our son’s very first flight EVER) was that instant ice packs and bandaids are something we will never travel without! It’s so much easier to grab that instant ice pack out of your bag if joint pain starts in mid-air, than to try to fit some ice in one of those throw-up bags in the seat pocket in front of you…
  • When it comes to flying on an airplane, one thing I have to say is to remember to STAY HYDRATED. Flying is known to cause dehydration, which often is accompanied by headaches (and in my case, migraines!) Try to make it a habit to grab a bottle of water on the plane, and in between flights. Nobody likes dealing with a headache or dehydration when they’ve finally reached their amazing destination! Another reason to keep the water flowing?? Kiddos with bleeding disorders sometimes struggle with nosebleeds… which can be worsened by dehydration.

 

Hotel Tips

  • If you or someone you are caring for will be using a wheelchair during your stay, most hotels have Handicap or Wheelchair Accessible rooms available. They are limited, so make sure you reserve or request ahead of time. It’s important to note, however, that typically, these types of rooms often have a roll-in shower instead of a bath tub, lower countertops, and wider doorways.
  • Just like instant ice packs are good for airplanes, they are also great for hotel rooms! You can even pack a few Ziplock bags in your suitcase, just in case you need to make a run to the hotel ice machine and want to have something leak-proof to keep the ice in while you ice a joint or muscle bleed.5
  • Many hotels, especially those in the Orlando, Florida area, have complimentary transportation to/from the theme parks – just make sure you find out before you go, as this could determine whether or not you rent a car during your stay. All Disney Resorts have either bus, boat or monorail transportation to all four of the Disney theme parks, including Disney Springs, so renting a car doesn’t have to be in your budget, if you don’t think you’ll be leaving Disney property. We always rent a car, as we have family in the area, and like to travel outside of the Disney parks (shhh… don’t tell Mickey!), but we have been known to use Disney transportation, too! Expect there to be a wait, especially for the buses, so give yourself about 45 minutes to an hour or so each way, especially if your Disney resort is further from the parks (like their Value Resorts). Universal Orlando Resorts also have complimentary transportation to all three of their theme parks (Universal Studios, Islands of Adventure, and Volcano Bay – opening in May 2017), however, Universal transportation does NOT service Disney parks, and vice versa. So if you’re looking to visit both Disney and Universal during your visit, you might want to look at renting a car, or staying in a hotel that offers transportation to ALL of the parks.
  • If you’re lucky enough to stay in a hotel that offers an outdoor pool, then make sure you find time to enjoy it! Often, we plan SO much during our vacation that it’s easy to forget that we need a day or two to slow down sometimes, and regroup. Taking advantage of your hotel pool area is a great way to do just that! Many of the Disney and Universal resorts offer daily poolside activities for the kids, water slides and lazy rivers – which can be a full day of fun just in itself! Walking (sometimes running, if you’re late for a FastPass+ time or a dining reservation!) around theme parks all day can be tough on joints, so taking time to just relax, while still enjoying your vacation, is a must!

 

Theme Park Tips

    • If you know you’ll be spending time at a theme park, which usually involves a TON of walking, make sure you talk to your HTC or medical provider about your treatment plan. Some doctors are fine with their bleeding disorder patients keeping their current treatment schedule, and others prefer to change it up (in dosing amount or frequency), depending on the days that a lot of walking (or roller coasters – eek!) will be involved. When in doubt, ask your doctor – as every patient is different, and treatment plans are different for everyone!
    • If you aren’t relying on resort transportation to get you to and from the Theme Parks, both Walt Disney World and Universal Studios have preferred parking available for Guests. It costs a little bit more, but you will get a parking space closer to the entrances – which for the Universal Parks, is extremely nice. Walt Disney World, however, does have trams that run through the parking lot, and there’s never a long wait for one. Take note, however, that if you DO arrive first thing in the morning before park opening (and the big crowds arrive), you may be fine with just purchasing the standard parking.1

 

  • Wheelchair rentals are available both on and off site. There are several different companies in the Orlando/Kissimmee area, and many of them deliver right to your hotel. If you’re staying at a Disney Resort hotel, you can find a list of those companies here. If you are not bringing your own wheelchair, all Disney handicapped parking lots have complimentary wheelchairs to use for park entry, however, you will need to transfer out of the parking lot wheelchairs once you arrive at the front of the theme park. You also have the option of renting wheelchairs once you are inside the theme park (adult-sized only), however, these wheelchairs are on a first-come first-serve basis, and there is a daily rental fee of $12. As an aside – up until a few years ago, we didn’t need a wheelchair, as he was able to fit into a stroller. We brought our own a few times, but once he grew out of the stroller at home, we just rented a double-stroller once inside the theme parks, and that worked just fine. He was around the age of six or so when he finally grew out of the double-stroller.

 

  • All theme parks have a First Aid Station – which is a great place to store any medication you bring into the park with you that needs to be refrigerated, or medication that cannot exceed a certain temperature (like clotting Factor). If you’re unsure of where to go, you can make a quick stop at Guest Services, and ask them to direct you to their main First Aid Station. Once there, you should be able to fill out a form, have your bag of medication and supplies tagged, letting them know if it needs to be refrigerated or not, and off you go! Just remember to pick up your medication before you leave the park for the day, as they won’t be able to hold it overnight.

 

  • Most of the larger theme parks (i.e. Disney, Universal, Sea World, etc) have a Guide for Guests with Disabilities (GGD) brochure, in addition to their normal park brochures. The GGD brochures give detailed information about the different services and facilities each park offers for their guests with disabilities, including rides that allow guests to remain in their wheelchair, or that are transfer-accessible. This is also where you can find out which rides have flashing/strobe effects (for those with seizure disorders), and other medical related warnings.

 

    • Walt Disney World now has the Disability Access Service Card (DAS), which can be helpful, especially on those days that joint and muscle pain rears their ugly heads. It’s designed specifically for Guests that aren’t able to wait in a conventional line, due to a disability. You can stop in at Guest Services any time during your visit for more information! Universal Studios and Sea World also have similar passes available.4

 

  • Staying hydrated is something that’s very important to do, but so easy to forget! If you’re visiting a theme park, it’s probably somewhere warm – which means dehydration can come fast! If you’re looking to save a little money, most theme parks offer free cups of ice (tap) water at their restaurants – all you have to do is ask! There are also some great water bottles out there that come with filters, which can make the taste of tap water a little better.
  • If you have a “picky eater” like I do, or especially if you or your child has a food allergy, you can definitely ask for meal accommodations. We have found that the restaurants at both Walt Disney World and Universal try their best to make you happy when it comes to your dining experience – so don’t be afraid to ask for a grilled cheese! Seriously – if they serve hamburgers and cheeseburgers, odds are, they can whip up a grilled cheese for your picky eater!

 

  • Taking breaks to rest during the day are SO important, not just for those with bleeding disorders, but for EVERYONE! The average person isn’t used to walking around a theme park all day, and you definitely don’t want to overdo it on your first or second day there! We like to save some of the longer shows that are offered at the parks for after lunch. Not only is this a good way to take a much needed break, without having to leave the park, but this time of day is usually the hottest. So take advantage of that indoor air conditioning! Another great way to take a break (or take a nap!) is to hop on the Monorail, if you’re at Walt Disney World! There’s a Resort Loop that you can take right from either EPCOT or Magic Kingdom, which takes you around to some of Disney’s amazing Deluxe Resorts, and the views are amazing! It’s air conditioned, fairly quiet, and the noise of the Monorail can be pretty relaxing!7

 

Most importantly, remember to HAVE FUN!!! For us, a family vacation to Walt Disney World or the Universal Theme Parks, is something we look forward to at least every other year (or more, if I can convince my husband to go more often!). It’s a place where we can go to forget our troubles, we can leave our worries behind, a place where it feels like nothing else matters. And if medical issues DO arise for our son while we’re there, it’s okay – because we have a plan (and usually a hotel with a super awesome swimming pool to make it all better!).

 

Thank you for letting me share some of our travel tips we’ve picked up along the way! And if you have some of your own to add, let us know!!!

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Summer 2017 Newsletter

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Portland Sea Dogs

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Looking for something FUN to do this summer??? How about attend a Portland Sea Dogs baseball game!! Hemophilia Alliance of Maine is selling Sea Dogs books of four tickets for $20 – a savings of $12!! Good for nearly any home game this season!! Support HAM and take your friends and family to a baseball game! Click the donate button below to purchase YOUR Portland Sea Dog ticket booklet!




And if that’s not exciting enough, this year, HAM’s Summer Social is moving to Portland!

Join us August 2nd, at Portland Sea Dogs’ Hadlock Field! Limited Space Available, RSVP, ASAP. Thanks!

Event Schedule:

10:30 AM: Sports + Bleeding Disorders with Surprise Guest!
11:30 AM: Lunch!
1:00 PM: Let The Game Begin!

RSVP via Eventbrite! Click here!

Visit our Facebook page for additional information!


 

Summer Social 2015 is sponsored by American Homecare Federation and CSL Behring. Many Thanks!

 

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The Way A Bleeder's Life Should Be, Ayuh!