Taha’s Moment: Finding “Normal”

By Taha Amir

Published: Lifelines for Health, Winter 2017

I am a 32-year-old male with severe hemophilia A. I moved to the United States, from Saudi Arabia when I was about 7 years old. Within that year, the hematologist told my parents that I had inhibitors - and they were lost, confused, and devastated. My parents knew what hemophilia was because my mom’s brothers also had it. But inhibitors were unheard of. The hematologist said that my body does not accept the “regular” factor, and I needed a bypassing agent called FEIBA. My brother was born in 1992, and he also has hemophilia, but not inhibitors, thankfully. To me, he was considered a “normal” person.

As a kid growing up with hemophilia with an inhibitor, I always stood out. I had bruises, swollen joints, and I walked with a limp. If I wasn’t walking, I was either using a wheelchair, crutches, cane, or a walker. I felt left out because the other kids were playing and walking better than me.

My pediatric hematologist suggested that I attend a hemophilia camp, which I did. The first time I went, I was nervous and scared because I wasn’t in my safety net and away from my parents for the first time. Even at the hemophilia camp, I stood out. I was still “different” from them because I had inhibitors. The “normal” hemophiliacs were still better than I was. Despite my struggles at camp, I attended a couple times after that because I was accepted. It felt like they knew me!

As a teen, I really didn’t attend any hemophilia events because it seemed like I was always bleeding. My younger brother, who also has hemophilia without inhibitors, was able to do more than I could. I felt as if I was the only one who had a different kind of hemophilia, which I did.

As I graduated high school, I decided to change my outlook on life. I attended a hemophilia conference, and met some amazing friends whom I still keep in contact with. At one of these events, I met the co-founder of CHES. She introduced herself and what CHES does for the hemophilia community - especially the inhibitor patients/community. One of the programs they offer is called Momentum, a men’s inhibitor retreat.

So far, I have attended Momentum twice. In July, I had started Immune Tolerance Therapy (ITT) in April 2017. By the time I went to the event, my inhibitor level was 0. Even though it had been 0 for a few months, the hematologists still hadn’t declared tolerization. I still have to do daily infusions of factor for the next few months.

Each time I have gone to the Momentum event, I have learned something new and met some amazing men who actually understand my struggles. The retreat is a three- day event over the weekend. The first day was dinner and a small informative talk by Dr. Dustin Sulak about the uses of medicinal cannabis for both acute and chronic pain. The second day started off with breakfast, a few more educational talks from treatment products to pain management, followed by lunch and more great information like infusion tips and tricks and practical career paths. One of the hot topics of this year’s retreat was health insurance and the current changes by NHF’s Michelle Rice. In the evening, the group went out for dinner and entertainment.

Most of the previous events that I’ve attended were primarily catered to “just normal hemophiliacs.” At Momentum, I feel accepted and better about myself. It has really changed the way I look at events for hemophilia with inhibitors.

What Inhibitor Family Camp Means to the Family

By  Cheryl Cook

Published: Lifelines for Health, Winter 2017

How do I even begin to tell anyone what Inhibitor Family Camp means to our family? How do you explain that it was a life-changing event?

My son is a mild hemophiliac, but we didn’t even know what an inhibitor was until he was diagnosed with one.
Our life changed in a minute. I heard about this camp from someone who had been there before, so I looked into it. We registered and “yeah!” we heard we were going!!

However, I was told by many that Jake should not go. His needle phobia required five (5) adults to hold him down for every infusion. We had health care companies that refused to work with us. We were giving infusions everyday. He was a mess; I was a mess. After much thought, I called to let CHES know we would not be coming to camp this year.

There was this voice on the other end of the phone, (I could barely hear because of the tears I was crying - saying we would not be coming.) It was another mom. The mom who said this camp was something she knew people needed; this was Janet Brewer (of CHES.) She told me to get Jake on that plane. She knew what I was going through and assured me that she would be there. She had survived through these same times and would help us get through them. She said she believed this would be something that Jake would never forget. She was so right. That weekend CHANGED Jake’s life!

When we got back from camp, none of his doctors or nurses believed this was the same kid. He was, but something had changed. I am still not sure what exactly happened to my 7-year-old that weekend, but he had
a different attitude and outlook. He was better able to accept this inhibitor diagnosis. He said it was because he was able to hang out with kids who “got” it. He said he felt that if they could do it, he could do it. We still have some stumbling blocks, but each time we go, it gets better.

The second year we were able to go, my son learned to self-infuse! Wow, what one year can do! The third year, he was excited about seeing his friends. He looks forward to hearing that registration has opened. He looks forward to seeing who is going to be there. He gets to go to Inhibitor Family Camp and be a kid; be with other kids, just like him, and no one is looking at his port. No one is wondering why he has it, or what is wrong with him? He’s able to just be a kid.

I asked him to describe what this camp means to him, and he turned around and said one word, ”JOY!”

All I can say is, “thank you!”

Pain and the Power to Heal - Winter 2017 Feature

By: Georgia Panopoulas, PhD, LP - Pain Psychologist and Janet Brewer, M.Ed

A few days ago, I came across an interesting article in the Minneapolis Star Tribune, and a very timely article, in retrospect. The author of the article titled “Opioids: Epidemic is the symptom, not the disease,” argued that “one of the reasons we have an opioid epidemic is because we have a pain epidemic.”

There is no arguing that pain, both acute and chronic, is prevalent in the United States and around the globe. Although trends in the prevalence of pain disorders are generally lacking, one study found that for chronic, debilitating low back pain (LBP), prevalence increased from 3.9% in 1992 to 10.2% in 2006. Increases were seen across all adult ages, for females and males, and across race. Reasons for the observed increase in prevalence rates were unclear, however, researchers suspected that increasing rates of obesity, changes in psychosocial and physical work demands, increase prevalence of depression, and increases in symptom awareness and reporting contributed to these increases in prevalence rates.

Pain in the Bleeding Disorders Community

Would these findings stand true within the bleeding disorders community? Witkop et al. (2017) argued that as life expectancy of persons with hemophilia (PWH) increases, pain and specifically joint pain is a major problem affecting adult PWH. Their study (the Hemophilia Experiences, Results and Opportunities study; HERO) found 85% of participants experienced pain during the 6-month period prior to completing the survey. Twenty percent of PWH (77% with Hemophilia A, 23% with Hemophilia B, and 9% with inhibitors, median age of 34 years) reported experiencing acute pain only, 34% reported experiencing chronic pain, and 32% reported experiencing both acute and chronic pain. Ankles (37%), knees (24%), and elbows (19%) were identified as the most painful joint. By comparison, 11.2 % of the general population in the United States experiences chronic pain, with LBP being the most common, followed by headache or migraine, neck pain, and facial pain.

In 2014, the Food and Drug Administration (FDA) sponsored a public forum titled “Patient-Focused Drug Development for Hemophilia A, Hemophilia B, von Willebrand Disease, and Other Heritable Bleeding Disorders” in an effort to learn from those affected about their treatment options and treatment concerns. A major topic of concern was pain management (both acute and chronic).

Definition of Pain

For the purpose of this article, pain is defined by the International Association for the Study of Pain (IASP) as: “An unpleasant sensory and emotional experience associated with actual or potential tissue damage, or described in terms of such damage.” IASP also added that pain is a subjective experience. Acute pain typically lasts less than 3 to 6 months. In PWH, acute pain is common during joint or muscle bleeds.

Chronic pain is defined as pain that persists longer than 1 month beyond the normal healing period or that is associated with a pathological process (e.g., arthropathy) that causes continuous or recurrent pain over months or years.

Chronic Pain Syndrome (CPS) effects approximately 25% of people with chronic pain. CPS occurs when people have symptoms beyond pain alone, like depression, anxiety, sleep disturbance, which interfere with daily functioning.

Why is pain so difficult to treat? The pain experience is subjective by nature. Contrary to 17th century thought that pain was either physical or psychic in origin, physical and mental aspects of pain as inseparable was the focus of medical research by the early part of the 19th century. By the 20th century, the Gate Control Theory of Pain emphasized the role of sensation, affect, and cognition “interacting with one another to provide perceptual information that ultimately influences the motor mechanisms characterizing pain,” (Olson, 2013). Further, as pain evolves from acute to chronic, it alters neural pathways, impacting mood and interfering with sleep.

Pain Management Requires a Variety of Approaches

As such, treatment requires an interdisciplinary approach focusing on physical, cognitive and emotional aspects of pain, in addition to focusing on pain and its impact on relationships. Opioids alone cannot successfully treat pain. In fact, successful treatment with opioids is considered to be 40-50% relief in pain (according to a scale of pain from 0-10, 0 representing “no pain,” and 10 representing “the worst pain imaginable”). Thus, successful treatment using opioids alone would result in a decrease of pain from 10/10 to 5-6/10. Opioids cannot eradicate the pain, much less “treat” the cognitive, emotional, and relational aspects of pain. Additionally, opioids are not without their negative side effects which include sedation, dizziness, nausea, vomiting, constipation, physical dependence, tolerance and respiratory depression. Long term use can result in allodynia and hyperalgesia (thus pain increases over time). From a psychological perspective, opioids and marijuana are dissociative drugs, numbing physical and emotional distress, making it more difficult for individuals to stay connected with loved ones and to find purpose and meaning in life.

Managing Physical Aspects of Pain

Managing the physical aspects of pain may require use of factor (in the case of acute pain, or chronic pain with acute exacerbation, or flare), RICE (rest, ice, compression, elevation), breathing exercises, relaxation exercises, gentle stretching, etc., in addition to certain medications. The goal is to manage the physiological arousal that often results from pain, but that only serves to feed right back into the pain experience. Pain is perceived by the brain as a potential threat or danger to the system. As such, pain activates the sympathetic nervous system (SNS), the fight or flight response, resulting in increases in blood pressure, heart rate, fuel availability, adrenaline, oxygen circulation to the organs, blood clotting, pupil size, while decreasing fuel storage, digestion, and salivation. Rest, which is essential for healing, becomes more difficult. Activation of the parasympathetic nervous system (PNS) is essential to counteracting the effects of the SNS. Activation of the PNS can be accomplished through meditation, massage, deep breathing, being in nature, playing with children or animals, yoga, tai chi, mindful movement. Finding the balance between relaxation and reactivation can strengthen the relaxation response and serve as an immediate defense against the effects of pain.

Cognition and Pain

Managing the cognitive aspects of pain may at first seem trivial, unimportant. Can pain management be as simple as mind over mood? Of course not. And in fact, we discussed previously that it’s not mind over mood, but mind and mood. Our pain is based on our perceptions, our perceptions are based on what we know, and what we know is interpreted based on what we have experienced in the past. Not knowing if our pain is a result of a bleed can be very disconcerting. Do we treat? Do we ignore the pain and push through? Are we thinking realistically about the situation or are we engaging in distorted, unrealistic, or irrational thoughts like:

*(Adapted from Margaret Caudiull’s Managing Pain Before It Manages You)

If you recognize any of these thought patterns, you are not alone. But once you recognize them, try to change them, not by thinking positively (for positive thinking can be as distorted as our negative thinking), but by thinking realistically/ rationally/with the evidence.

Emotional Well-being

The importance of managing emotional aspects of pain cannot be overstated. The prevalence rates of depression and anxiety in PWH are indeed higher than what we would expect in the general population and higher and/ or comparable to rates of emotional distress in persons with other chronic pain conditions (e.g., lower back pain, headache/migraine, neck pain, facial pain). Sadness, anger, fear are real and justifiable emotional responses to chronic illness and chronic pain, but can be managed. First identify what you are feeling. I like having a cheat sheet of “feeling words.”

Ultimately, however, our feelings fall into one of four categories: mad, glad, sad, and scared. What are you feeling? What do you attribute the feelings to? Something that just happened or something that happened in the past? Can you do anything about the situation that is contributing to how you feel? If not, can you allow yourself to feel without trying to change the feeling (i.e., mindfulness)? Can you allow yourself to feel anger, sadness, grief and loss, forgiveness toward yourself and others, and to accept these feelings as they are, real and valid? Or do we too quickly assign judgment to these feelings?

Keep in mind that feelings of grief, sadness, loss apply to both the parent/caregiver and the person with pain.
Guilt can be a source of great pain for mothers given that hemophilia is a x-linked chromosomal disorder. “It is my fault my son suffers because I gave it to him”. Or, as the person in pain, “I am a burden to my wife/children because I can’t do the things I once did which falls on them.”

Pain and Relationships

As much as we would like to think that pain only impacts the person with pain, we know otherwise. Pain impacts those we love deeply. Family and friends want to help, they want to “fix it.” They can’t. They become frustrated and, at times, it seems as if they are frustrated with the person who has pain. Their response to the person with pain can be perceived as “punishing.” Or maybe overly “helpful.” The research suggests that these responses are common, but not helpful especially when attempting to manage chronic pain. So, what are family members to do? “Distracting” responses can be most helpful. What does that mean? Offer understanding, validation. But more than that, I encourage family and friends to ask, “What do we need to do to get through this.” The “we” refers to the person in pain along with the family member or friend. This, of course, is a work in progress and requires communication, willingness to try new things, and reflection. When it all gets overwhelming, there is the propensity to shut down and pull away. Parents watching their children withdraw is painful. Trying to help seems to make it worse because the child may not want to talk about it. Ultimately, we may seek counseling and/or use mood enhancing medications to get us through. As much as we’d like to put the bleed, or the flare, or the crisis behind us, it would serve us better if we talked about how we got through, what helped, what didn’t, and what we might want to do different next time, because there will always be a next time.

The Power to Heal

In 2016, I attended the NHF Inhibitor Summits. Dr. Tyler Buckner and I presented several sessions on pain management (an overview of strategies for managing acute and chronic pain). Dr. Buckner presented information about various classes of medications including but not limited to opioids and medical cannabis. The utility of these medications, as indicated previously, was variable at best.But the participants, those living with inhibitors, shared their favorite pain coping strategies with the group and, once again, I left the Summits truly believing that I learned more from the Summit participants than they learned from me. The following is a list of pain coping strategies identified by participants (including PWH, parents/ caregivers, siblings, significant others):

Assistive Devices

• Poke program*check out on our website in reference section

• Smart phone apps, i.e. - Healing Buddies Comfort Kit

  http://www.healingbuddiescomfort.org/

• Virtual reality (see related articles in what’s new on page 42)

• Buzzy (see related articles in what’s new on page 46)

Distraction-Passive and Active

• Blow bubbles

• Listen to or create music

• Play games

• Read/Listen to Stories

• Watch movies

• Utilize social media

Emotional Strategies

• Calming Techniques

• Change your thoughts-
  positive self-talk and reinforcement

• Coping

• Counseling

• Grief and loss acceptance

• Humor

• Pain Education

• Set realistic goals/expectation

Movement

• Balance, coordination, ball therapy

• Change position

• Dance

• Exercise/Gentle stretching

• Mini Breaks

• Tai Chi

• Walk/pace/rocking

• Yoga

Mindfulness

• Attention Management

• Breathing

• Guided imagery

• Meditation

• Progress muscle relaxation/body scan

• Self-hypnosis

• Set realistic expectations of self/others

Physical Comfort Measures

• Acupuncture

• Acupressure

• Beverages

• Biofeedback

• Contrast baths-Epsom salts

• Cuddle

• Dim lighting, noise reduction

• Essential oils, salves, rubs/ aromatherapy

• Food

• Hot/Cold packs/pads

• Loose, soft clothing

• Massage

• RICE

• Sleep

• TENS (Transcutaneous electrical nerve stimulation)

• Management-anti-inflammatory, gluten, soy, soda, sugar free diet

  This list is far from exhaustive! But these strategies are tried and true. Will we eradicate the pain? Can we eradicate the pain? Should we eradicate the pain? Remember, pain is not all in our head, but that’s where pain messages are processed and where we can begin to alter neural pathways and change the suffering.

Managing Inhibitors in Hemophilia Treatment: The Importance of Bypassing Agents

Bypassing Agents are a Factor option with a long history of use in treating hemophilia patients with inhibitors

By: Mike Denne

Published: Lifelines for Health, Winter 2017

Treatment of inhibitors is one of the greatest challenges in hemophilia today

Up to 33% of people with severe hemophilia A develop an inhibitor, or antibody, to treatment, while 3% to 13% of patients with moderate to mild hemophilia develop an inhibitor.1,2 (Inhibitors are less common among people with hemophilia B, affecting only 1% to 6% of individuals.1) These inhibitors stop, or block, Factor VIII (FVIII) or Factor IX (FIX) function in your blood-clotting process. Bypassing Agents offer an alternative treatment approach because they go around, or bypass, the need for FVIII or FIX.3

Bypassing Agents supplement other Factors in the blood-clotting process

This chart is a simplified visualization of Bypassing Agent activity in the blood-clotting process.

REFERENCES:

1. DiMichele DM. Inhibitor treatment in haemophilias A and B: inhibitor diagnosis. Haemophilia. 2006;12(suppl 6):37-42.

2. Hay CRM. Factor VIII inhibitors in mild and moderate-severity haemophilia A. Haemophilia. 1998;4(4):558-563.

3. DiMichele DM. World Federation of Hemophilia website. Inhibitors in hemophilia: a primer.

   http://www1.wfh.org/publication/files/pdf-1122.pdf. Published 2008. Accessed October 5, 2017.

4. Lenting PJ, van Mourik JA, Mertens K. The life cycle of coagulation factor VIII in view of its structure and function. Blood. 1998;92(11):3983-3996.

5. Berg JM, Tymoczko JL, Stryer L. Many enzymes are activated by specific proteolytic cleavage. In: Biochemistry.

   5th ed. New York, NY: WH Freeman; 2002. https://www.ncbi.nlm.nih.gov/books/NBK22589/. Accessed September 19, 2017.

6. Johari V, Loke C. Brief overview of the coagulation cascade. Dis Mon. 2012;58:421-423.

7. Retzios AD. The new and improved (?) activated factor VII molecules. Bay Clinical R&D Services. 2001:1-41.

8. Shapiro AD, Hedner U. Advances in bypassing agent therapy for hemophilia patients with inhibitors to close care gaps and improve outcomes. Ther Adv Drug Saf. 2011;2(5):213-225.

9. Ananyeva NM, Lee TK, Jain N, Shima M, Saenko EL. Inhibitors in hemophilia A: advances in elucidation of inhibitory mechanisms and in inhibitor management with bypassing agents. Semin Thromb Hemost. 2009;35(8):735-751.

10. Leissinger CA. Prevention of bleeds in hemophilia patients with inhibitors: emerging data and clinical direction. Am J Hematol. 2004;77(2):187-193.

11. Wolberg AS. Plasma and cellular contributions to fibrin network formation, structure and stability. Haemophilia. 2010;16(suppl 3):7-12.

12. Colvin BT. Physiology of haemostasis. Vox Sang. 2004;87(suppl 1):S43-S46.

13. Hoffman M, Monroe DM. A cell-based model of hemostasis. Thromb Haemost. 2001;85:958-965.

14. Hoffman M, Dargaud Y. Mechanisms and monitoring of bypassing agent therapy. J Thromb Haemost. 2012;10:1478-1485.

15. Astermark J, Donfield SM, DiMichele DM, et al. A randomized comparison of bypassing agents in hemophilia complicated by an inhibitor: the FENOC Study. Blood. 2007;109(2):546-551.

16. Peyvandi F, Garagiola I, Young G. The past and future of haemophilia: diagnosis, treatments, and its complications. Lancet. 2016;388:187-197.

17. Hedner U. Recombinant activated factor VII: 30 years of research and innovation. Blood Rev. 2015;29(suppl1):S4-S8.

18. Hemophilia Federation of America. Treatment of hemophilia. http://www.hemophiliafed.org/bleeding-disorders/ hemophilia/treatment/. Accessed September 20, 2017.

19. Santagostino E, Escobar M, Ozelo M, et al. Recombinant activated factor VII in the treatment of bleeds and for the prevention of surgery-related bleeding in congenital haemophilia with inhibitors. Blood Rev. 2015;29(suppl1):S9-S18.

20. Mehta R, Parameswaran R, Shapiro AD. An overview of the history, clinical practice concerns, comparative studies and strategies to optimize therapy of bypassing agents. Haemophilia. 2006;12(suppl 6):54-61.

21. National Hemophilia Foundation. Medical and Scientific Advisory Council (MASAC) recommendation regarding the use of bypassing agents in patients with hemophilia A or B and inhibitors. MASAC document #167. 2006.

22. NIH Clinical Trials Registry. Ongoing and complete clinical trials using bypassing agents in hemophilia patients with inhibitors. https://clinicaltrials.gov/ct2/results/ details?term=hemophilia+a+with+inhibitors%2C+hemophilia+b+with+inhibitors&recr=Closed&cond=hemophilia +a+with+inhibitors%2C+hemophilia+b+with+inhibitors. Accessed September 19, 2017.

©2017 Shire US Inc., Lexington, MA 02421.
All rights reserved. 1-800-828-2088.
SHIRE and the Shire Logo are registered trademarks of Shire Pharmaceutical Holdings Ireland Limited or its affiliates. S34496 10/17

New Sub-Q Treatment is FDA-Approved for Inhibitors

by Eric Lowe

On November 11th 2017, Genentech announced that the study drug, widely known as ACE910 (emicizumab), had been FDA-approved for prophylaxis treatment

in children and adults with hemophilia A and inhibitors to prevent or lessen bleeding episodes. This approval snapped a streak of nearly 20 years for the inhibitor population on newly-approved treatment products.

Almost 1 in 3 hemophilia A patients that are treated with factor VIII replacement therapy tend to develop inhibitors against factor VIII, which can lead to a higher risk for bleeds and long-term joint damage. The FDA-approved drug, now named Hemlibra, bypasses that problem as it is a bispecific factor IXa- and factor X-directed antibody. This means its use can bring together the two proteins, which will activate the natural clotting cascade. Hemlibra also requires only once a week use as an injection under the skin.1

Hemlibra, has had positive results from two studies, HAVEN 1 (NCT02622321) for patients 12 years of age or older with hemophilia A with inhibitors, and HAVEN 2 (NCT02795767) for patients younger than 12 years with hemophilia A with inhibitors. Results indicated that Hemlibra significantly reduced bleeds in both adult and pediatric patients with hemophilia A.

“People with hemophilia A who develop inhibitors face significant challenges preventing and controlling bleeds and typically require infusions of medicine multiple times a week, which can be especially difficult for young children and their families,” Dr. Guy Young, professor of pediatrics at University of Southern California Keck School of Medicine, said in a press release. “This new medicine has been shown to reduce the frequency of bleeds compared to the currently available medicines and only needs to be injected once a week. This could make a meaningful difference for these children.”1

Results

Phase 3 of the HAVEN 1 study (in patients 12 or older) showed an 87% bleed-reduction when using Hemlibra compared to those who did not treat prophylactically, and a 79% bleed-reduction was reported for those who did treat prophylactically with a different bypassing agent (BPA) before switching to Hemlibra.

Similarly, the HAVEN 2 study (in patients younger than 12) also showed an 87% bleed-reduction when beginning prophylactic treatment with Hemlibra, and a 99% bleed- reduction when changing their prophylactic treatments from a BPA to Hemlibra. Common side effects of treatment were injection site reactions, headache, and joint pain (arthralgia).1

One death has occurred in the HAVEN 1 study back in February 2017. The patient received bypassing agents (BPA) after experiencing a serious rectal hemorrhage.2 The patient’s repeated use of the BPA in combination with the remaining residual amounts of Hemlibra in the patient’s system caused threat of serious thrombotic events. After discontinuation of the (BPA), the patient showed improvement. But Roche said the hemorrhage therapy was complicated because the patient declined blood transfusions. The trial investigator determined that the cause of death was the rectal hemorrhage, not emicizumab.2 You may read more details on this using the #2 resources link below.

Looking Ahead

Hemlibra’s availability in the US is currently emerging and evolving.

HAVEN 3 and HAVEN 4 studies are currently underway to evaluate the treatment’s efficacy and safety at other doses and schedules. HAVEN 3 (NCT02847637) is being conducted in patients 12 and older for those with hemophilia A without inhibitors with doses given once every 1-2 weeks.

HAVEN 4 (NCT03020160) is being conducted in patients 12 years or older, with or without inhibitors, with doses given every four weeks after an initial one-month “loading” period.

1. https://hemophilianewstoday. com/2017/11/17/fda-approves-hemlibra- by-genentech-to-treat-hemophilia-a- patients-with-factor-viii-inhibitors/?utm_ source=Hemophilia+News&utm_ campaign=58c1c52d86-RSS_EMAIL_ CAMPAIGN&utm_medium=email&utm_ term=0_ab10fdd11a-58c1c52d86-72167405

2. https://hemophilianewstoday.com/2017/02/24/ death-of-hemophiliac-in-clinical-trail-raises- more-concern-about-emicizumab/

When Someone You Love Hurts How Can You HELP?

by Dr. Gary McClain, PhD

Published: Lifelines for Health, Winter 2017

A while back, a politician said something to the effect of, “I feel your pain.”

These days, that line is generally used in a humorous sense. But if you have a partner or child who is living with pain, I suspect that line conjures up all kinds of thoughts and feelings for you. Most of them not very funny.

That’s because watching someone you love struggle with pain is anything but humorous. As a partner, as a parent. You know how hard things are for them, you can see it in their face, the way they move, and in the way they speak.

You want to say something to make them feel better. You might find yourself saying to your partner: “You’re gonna be just fine. And soon.” Or to your child: “”Where does it hurt? Let me make it better for you.”

And the way in which your loved one responded may not have been so gratifying. They may felt frustrated, or angry, because you were promising something you clearly couldn’t deliver on. Sure, you were just trying to be helpful. But it didn’t feel that way to them.

I think it is not an overstatement to say that having a loved one with chronic pain can really make you feel helpless.

Of course, you can’t take their pain away. Even if that’s what you wish you could do. But between doing absolutely nothing, and ridding them of their pain, there are actually a lot of things you can do to help a partner or child living with chronic pain.

Here are some ideas to consider:

Get Informed Together

Make it known that you are on stand-by. Let your partner or child know that you are standing by to help in not only gathering information, but making sense of it, whenever they are ready. Kids are especially in need of reassurance, so feel free to repeat as needed.

Do your own information-gathering. It’s not realistic to remain uninformed even if your loved one is locking you out of this process. Find information on your own so that you can educate yourself on how to be a better healthcare partner. And active information-gathering is a great way to help you cope with your own helpless feelings.

Let your loved one know you are getting informed. You don’t need to surf the Web in secret. Gently inform your loved one that you want to be as educated as possible and are doing your own research.

Offer to share your information. As you get educated, offer to share what you are learning. Use statements like: “I just found out about an interesting new treatment. Do you want to know about it?” or “I saw a list of foods that might help you. Any interest?” If you are a parent, your information-sharing may be needed to help explain any limits you need to set.

Encourage Adherence

Look for teachable moments. The long lectures get old after awhile. You might instead be alert for moments when you can gently point out your loved one’s lack of adherence. For example, if you notice they have missed a medication dosage, you can quietly remind them and ask if they would like you to help them remember. Parents may find using teachable moments especially helpful because younger kids and teens can be especially resistant to feeling lectured.

Use some “patient” education. If you find yourself scolding your loved one, or getting angry, or giving orders, you may also find that you’re not making a whole lot of progress. Instead, assume good intention on their part rather than a desire to cause you frustration and annoyance. Who knows? Your loved one may get the overall concept of self-care but not be sure how to actually make it happen. Kids often need additional guidance in establishing self-care routines. How about asking if you can make a suggestion before you launch in with one? And then, focus on what they could have done and not what they didn’t do.

Choose your battles. As you have probably learned already, when people feel pushed into doing something, they become more resistant. That’s true for your partner and it’s true for your children. So if, out of your own desire to help, you are turning everything into a battle of wills, your partner or kids may put up a wall. Be mindful of what is most important – like taking medication on schedule – versus what be less important. Use your judgment here.

When You Don’t Know What To Do...

Ask. It’s sad to me that so many people don’t take the time to ask questions. They assume to know already. Or they just don’t think about what their loved one needs. The simple question – “What can I do for you?” – shows how much you care. By asking it, you’re already one step toward helping your loved one feel more supported.

Listen. With an open mind. You might be surprised at what you hear from your loved one. Maybe you’ll learn that a simple gesture would help him/her have a better day. But you may also learn that your partner or child needs a lot more from you than you expected. You might hear about how overwhelmed they feel at times, or a lot of the time. A few disappointments or resentments might come up. Be open to what your loved one has to say.

Get specific

I have found that both caregivers and patients avoid talking about the support process itself, and what they are expecting of each other. Family members are often afraid that if they bring up the subject of support, their partner or child may assume that they are feeling burdened with their support tasks, or that they don’t want to help them anymore and are trying to find a way to break the news. In turn, your loved one may fear burdening you, but also doesn’t want to be made to feel incompetent by receiving too much care. Keep in mind that grown-ups, younger children, and teens can all feel disempowered because “hovering” over them can be interpreted as not trusting them to do what they can do to take care of themselves. Furthermore, when needs and expectations are not clear, this can result in missteps that can lead to tension.

Figure out a path forward. Talk about how you see your role — what you can and want to give, and what you think they need — as well as your partner’s or child’s expectations. While defining your role is going to be a work in progress, open communications can help you to build a solid foundation for moving forward, and for protecting your own health and well-being. Keep talking!

Watch the Silver Lining Talk

Don’t be a positive thinking police bully. Being told to “think positive” or to “stop feeling that way” is a tactic of the positive thinking police. Jumping in with a “yes but” and showering your loved one in rainbows and puppy dogs is another tactic. Denying feelings doesn’t make them go away.

Your loved one has the right to feel bad. Be sensitive to what your loved one needs to hear and doesn’t need to hear. They are living with this chronic pain. Some days are going to be harder than others. On a bad day, go into asking questions and listening mode. Keep in mind, you may be asked to step aside and just be quiet. When your partner says, “I’ll be okay, honey,” or your child says, “please leave me alone for awhile,” keep an eye out but back off.

Let your loved one know he/she doesn’t have to sit all alone with negative feelings. Be someone who can listen without telling them how they should be feeling or judging them. Encourage them to vent! When you release feelings into the light of day, they lose their power over you.

Give Your Loved One Their Space

Individuals living with chronic pain are facing a wide range of emotions. Many of these feelings are uncomfortable — a feeling, like fear or anger, may be so uncomfortable that they may not be able to even acknowledge feeling this way, let alone begin to express these feelings to someone else. Your loved one may feel so emotionally overwhelmed that they may shut down. And that’s scary for you to witness. Gently ask your partner or child how he or she is feeling, not only physically but emotionally, let them know you are here to listen. Remind them as needed, but don’t push.

Be willing to step aside. Keep in mind that, at least initially, some individuals are more comfortable opening up to people who are not their family members, and with whom they are less involved on a daily basis, like
a counselor or support group member. Don’t take this personally. Your partner may feel the need to protect you from his or her feelings, and so might your child. Give your loved one space to cope in a way that works for them.

Take Care of Yourself

Take ownership of your own helplessness. Human beings love being in control, and we love it so much that we tell ourselves we have control even when it is obvious that we don’t. And in a caregiving situation, that need to be in control can result in running ourselves into the ground trying to meet every possible need of someone we love to the point that we are running on empty. What parent hasn’t felt this way, right? In the process of depleting ourselves, we also risk alienating the people we care about by taking their own sense of control away from them.

Find your own support system. If you totally deplete yourself, you aren’t going to be helpful to anyone. Yes, I know you’re superhuman, but you’re still human. Find a safe place to talk about your own emotions — your fears and frustrations — and to get feedback and advice. Don’t be afraid to ask for help in coping with being a caregiver. And don’t be afraid to talk about how you feel, even the feelings that you aren’t so comfortable with, like anger. A trusted friend, a family member, a counselor, or a member of the clergy can be beneficial.

Express your own emotions. You may not feel comfortable admitting to emotions like fear, out of concern that they may come across as having a negative or pessimistic attitude. While it is realistic not to sound alarms, expressing your own concerns can help to create an atmosphere of honesty. If fear, for example, has become the “elephant in the room,” getting it out in the open can relieve the tension that results from talking around the emotions that are most likely on everybody’s mind. While you want to encourage and support your partner or child, he or she most likely wants to do the same thing for you. Open the door for both of you.

And recognize where you don’t have control. Here’s the hardest one of all. When you love someone, you also want the best for them. But nobody likes to be told what to do. Even if they probably need to be told what to do. Be a support, a cheerleader, and throw in some tough love when you need to. What you can do is be a supportive partner or parent. But you can’t make the pain go away. So focus on the goal of helping to motivate your partner or child to do everything they need to do to take the best possible care of him/ herself. Be a team!

You and your loved one. Here is some final advice that is guaranteed to make every day a better one. Begin and end the day with three words: “I love you.”

The Individualized Healthcare Plan: What’s It All About?

by Ann Marie M. Minichiello

Published: Lifelines for Health, Winter 2017

The Individualized Healthcare Plan

The Individualized Healthcare Plan (also known as IHP or IHCP) is a common tool used for students with special health needs. When you send your child off to school, you want to know that his or her medical needs will be met while there, and an IHP is just the place to start. A child with a bleeding disorder should have this plan in place and it should be developed by your school or district nurse. According to the National Council of State Boards of Nursing (2005), the development of an IHP must be the responsibility of a licensed nurse. However, in the absence of a school nurse, it is recommended that there still be a plan developed by caregivers, student, healthcare providers and designated school personnel to ensure continuity of care in the school setting.

The IHP is a document based on the nursing process and in essence, is a variation of a nursing care plan. It includes a nursing assessment, diagnosis, interventions, and student outcomes. It is meant to outline the health services that your child will receive in school. It should include how, when and where these services will be provided at school. It should be reviewed and updated annually or as often as the student’s health status changes. An IHP is considered a standard for good nursing practice.

Although an Individualized Healthcare Plan is a standard of school nursing care and should be in place for any student with mild to severe health care needs, there is no law that states your child has the right to this plan.

It is designed to address only medical and nursing issues that do not impact student learning. It does not address access to education in any way; it is strictly a formalized plan to address the medical issues of a student. It does however, provide documentation of the nursing process and can be used in a legal proceeding, should the school nurse’s conduct or performance ever result in such a case. That being said, if a legally protected plan is what you want for your child, then an IEP or 504 Plan would be the appropriate and necessary route to pursue.

If it is determined that your child is eligible for special education services, then an IHP can be included in the health portion of an IEP. An IHP should also be part of a 504 plan that your child qualifies for due to a medically related health condition. When the Americans with Disabilities Act of 1990 (ADA) was first written, it was considered to be fairly strict, but certain health conditions were not always viewed as disabling. A health condition has not always guaranteed qualification as having a disability under the 1990 Americans with Disabilities Act. For instance, it could have been argued that an individual with a bleeding disorder who successfully treated prophylactically might not have been considered to have a disability because his or her medical condition did not substantially limit a major life activity. In 2008, the ADA Amendments Act sought to expand the meaning of a disability and make the law more inclusive of those who may not have qualified previously as having a disability. As a result of this legislation, a school district must consider the effect of the disability without the use of “mitigating measures” such as medication (i.e. worst case scenario). As a result of this change, those with a medically related health condition have the right to a legally binding agreement with the school by having a 504 plan which should include an IHP for specific nursing care procedures.

Emergency Plan:

Just as an IHP can be added to, or part of a 504 Plan, so too can (and should) an Emergency Care Plan (ECP) be part of the equation. An ECP is a document that requires a plan for the worst-case scenario - the emergency situations - that none of us like to think about, but that we always have in the back of our minds as parents of children with special health needs. Unlike the IHP which should be written in “nursing language,” the ECP should be clear and succinct so it is can be read quickly and easily in an emergency situation. It should be in layman’s terms, because it is not just for the school nurse but for any school staff member to implement in an emergency. It can even be formatted like a basic chart:

An IHP and an ECP are both plans that strictly address the medical issues that may arise for the student during the school day. Unlike an IEP or 504 Plan, the IHP and ECP do not have a standardized format. The format may vary by school district or even by school. While considered standard, good nursing practice, you may not find that this type of plan is the “norm” in your school district or with your school nurse. In such a case, it is important for parents to advocate for this plan for your child at school. Your healthcare provider can assist you by acting as a liaison with the school or district nurse or by giving you a plan that you can discuss with the district’s health team. In the end, an ECP is the bare minimum of what should be in place for any child with special health needs. An IHP is a more lengthy and detailed plan that focuses on nursing procedures for your child and can incorporate an ECP. However, if you feel that your child requires more accommodations (and a legally binding agreement) that a nursing care plan does not offer, then you should seek out an evaluation for services provided under a 504 Plan.

The Basics of a Well-Developed IHP:

• should always include up to date emergency contact information

• should include a review date

• should always be signed by a parent/guardian and school nurse

• should include six components

  1. Assessment: gives the background information on the student/health issue

  2. Nursing Diagnosis: the school nurse’s analysis/summary of the health issue and how the nurse can contribute for care of the student

  3. Goals/Outcomes: clear, concise outcomes for student healthcare plan

  4. Nursing Interventions: details how the nurse will meet the goals

  5. Implementation: put the plan in practice

  6. Evaluation: reviewing student goals and whether they have been attained; review interventions and adjust as needed based on status of the student’s health

• The Emergency Care Plan should be part of the IHP - it must include what emergent scenarios could arise as a result of the student’s health condition and what actions must be taken in response - for any member of the school staff (not just the school nurse)

  Remember, there is no standardized format for the IHP. You may see many different versions of this type of document. Your medical provider must submit written orders for any medical treatment that may be performed at school.

References and additional resources:

http://oley.org/?page=IHP_IEP_Difference

https://www2.ed.gov/about/offices/list/ocr/504faq.html

A Review of Inhibitor Family Camp at Victory Junction

Making Life-Long-Distance Friendships

By: Ashley Davis

Published: Lifelines for Health Spring 2017

This was our first year attending Inhibitor Family Camp at Victory Junction. We were beyond excited.

We really didn’t know what to expect. We knew there would be rap sessions and fun activities, but we had no idea the dynamic relationships we would create.

Upon arriving, we met our awesome crew chief who stayed with us all weekend and helped us out with anything we needed. Our six year old son warmed up to her pretty quickly and was never torn about having to leave us to go hang out with her and the other children. He normally does not want to leave our side if he’s around people he doesn’t know well, so we weren’t sure how he was going to feel when we headed off to rap sessions and he had to go a separate way with the kids. However, our crew chief was so good with him, and he had no hesitation about going off to have some fun without mom and dad! This was great for us, since we hardly ever leave his side at home. We needed this time to talk and empathize with other parents dealing with the same things.

The emotional and physical aspect of being a caregiver is often very overwhelming. Understandably, nobody back home – even those closest to us – really knows what it feels like on a daily basis. Being able to meet other parents, and for me other mothers, was extremely humbling. For once, we were able to be around people who really, truly get it. This was the biggest takeaway for me, personally. I needed desperately to find that connection. I was able to make lifelong friendships and find people to connect with, even if hundreds of miles away.

In the same way, our son was able to finally meet other boys going through the same things he does each day. He was able to do fun activities and participate in things we wouldn’t normally do back home. All of the boys were so welcoming and made him feel like they had always been friends.

During infusion time, our son was able to build up enough courage to stick his dad for the first time. Some
of the older boys rallied around him and gave him their support, which was so cool to watch. These kids shouldn’t have to worry about things like this, but they embrace it so well and run with it, and then encourage the others to run with it as well. Amazing.

We are so grateful for the opportunity that CHES provided us to attend Inhibitor Family Camp. Without their generosity and willingness to host, we would not be able to do something like this. They made it such a special weekend for us. We each had our own personal takeaways, as well as family experiences to take home. Thank you CHES!

The Timeline of Education: A New and Uncertain Turn?

by Lisa Cosseboom, M.Ed. & C.A.G.S School Psychologist & Special Education Evaluation Team Chairperson

Published: Lifelines for Health Spring 2017

Important Events in Public Education:

April 11, 1965: President Lyndon B. Johnson signs the Elementary and Secondary Education Act (ESEA) which expanded the federal role in k-12 education. Title 1 was implemented that enabled the Federal Government to assist with providing funding to school districts to help disadvantaged students.

1968: Congress expands on ESEA to provide programs for immigrant children, neglected children and passed the Bilingual act.

1973: The Rehabilitation Act becomes law in which the Section 504 ensures civil rights for people with disabilities requiring school districts to accommodate for students with disabilities to access buildings, programs and activities.

1974: Equal Educational Opportunities Act passes. This Act requires school districts to take action and overcome barriers which would provide equal protection for students.

1975: The Education of All Handicapped Children Act (PL-94-142) becomes Federal Law. This law provided that handicapped children and adults ages 3-21 be educated in the “least restrictive environment” to the maximum extent appropriate, meaning that they are educated with children who are not handicapped and that special classes, separate schools or other removal of children from their regular educational environment, occurs only when the severity of the handicap is such that education in regular classes cannot be achieved.

1978: President Jimmy Carter reauthorized ESEA and changed Title 1 rules allowing school- wide Title 1 programs when 75% or more of the students are low-income.

1979: President Ronald Regan reauthorized ESEA and changed funding into one block grant and reduced regulatory requirements by states.

1988: Student testing and accountability takes hold and regulations require districts to test annually and to create improvement plans.

Recent Education Bills Passed:

House Joint Resolution 58: Passed House on 2/7/17, Senate on 3/8/2017: Rejected a Department of Education regulation that imposed a new federal standard for the education and preparation of teachers which linked teacher preparation to eligibility for federal grants.

House Joint Resolution 57: Passed House on 2/7/2017, Senate on 3/9/2017: This resolution overturns a regulation by the Department of Education that placed federal restrictions on systems developed by states to hold schools accountable to parents and taxpayers for their performance.

New Bills introduced:

What is H.R. 610?

Termed “Choices in Education Act of 2017” according to Congress.gov, this Act “Repeals the Elementary and Secondary Education Act of 1965 (ESEA) and limits the authority of the Department of Education” to nothing but the power to award block grants to qualified states.

The repeal of ESEA would essentially eliminate every education act noted in the timeline...

including Every Student Succeeds Act (ESSA) under Obama and No Child Left Behind under Bush. The more recent ESSA promotes equality in education and provides federal protections for disadvantaged and disabled students. The block grants would distribute federal funding to eligible states to award in the form of vouchers for eligible students to use in school choice. Additionally, this Act repeals

the rules surrounding established nutrition standards (availability of fruits, vegetables, reduction of sodium etc.) for the national school lunch and breakfast programs. This Act was introduced to the House Committee on Education and the Workforce on 1/23/2017.

Thinking behind H.R. 610:

Proponents behind the Choices in Education Act believe that this Act would provide better competition between public and private schools and therefore increase the quality of public education through the spirit of competition. They feel that disadvantaged students would have access to private or religious schools through the voucher system where they would not have had access freely because of their poverty levels. Families who tend to be financially well-off, tend to live in identified communities that are known for quality education, or can afford to send their children to private schools.

Nuts & Bolts of Choices in Education Act:

Title 1 is federal funding provided to public school systems based on the number of disadvantaged students that are enrolled in their districts. The funds are distributed in public schools to assist in educating the disadvantaged. H.R. 610 would remove Title 1 funds to public schools and transform this funding into the voucher system that would follow an eligible child to whatever school they are attending (including religious, private, charter schools or students being home-schooled). Many states have laws in place separating church from state and the voucher system could allow for public funding to be funneled to religious organizations.

The proposal is assuming that states would contribute approximately 110 billion dollars into the voucher system which would ultimately provide approximately $12,000 per year to each student who qualifies for the voucher system. Additionally, the vouchers are geared towards students whose family fall in the poverty level and may not cover the complete cost of private schools and would require the families to pay the difference. The very families that have met poverty levels. There are a lot of proposals on how this voucher system would work, but none seem very clear. States hold local control over education policy and regulation, and would need to “buy-in”, literally and figuratively, with the federal proposal. The Act appears to be the beginning of an attempt to privatize education and dismantle public education. Essentially, it reallocates federal funding leaving public school districts scrambling to make cuts and find funding.

Voucher programs have existed on a smaller scale in this country for a long time. Some of the research points to inconclusive or contradictory results. Some states have reported better success of student on vouchers in testing and graduation and some have noted no increase in testing scores or graduation.

The second component of H.R. 610 introduces the “No Hungry Kids Act.” While on the surface this seems like a positive Act, it is actually removing the previous “Healthy, Hunger-Free Kids Act which purpose was to improve child nutrition through the school lunch and breakfast programs. The Healthy Act was requiring the schools to increase availability of fruits, vegetables, whole-grains, low-fat milk and reduce levels of sodium, trans-fats and saturated fats in school breakfast/lunch The prosed act would prohibit the USDA from rationing calories to children and remove the previous Healthy act requirements.

Effect of H.R. 610 Special Needs Students:

The Elementary and Secondary Education Act of 1965 has been reauthorized every 5 years since its inception and has changed names several times. Whatever name it was at a given time did not matter to special education students, as it always provided protection. The ESEA provided that schools who receive federal funding must provide support for students with disabilities. Under the new proposal, federal funding in the form of grants, would not require the schools receiving the vouchered students to provide services. Private and religious schools are not required to provide special education services. Removing funding from public schools that offer a spectrum of services and place it in the hands of private or religious schools further impacts public education and decreases funding for special education services.

Summary:

If H.R. 610 passes, essentially it removes federal funding to public education and repeals the ESEA which protects programs for special education students, students in poverty, gifted students, ESL programs, rural education and school safety. Providing a free and appropriate education to all and ensuring special education students access to the curriculum and accommodations will cease to exist. The goal of H.R. 610 is to privatize education and defund public education. To dismantle fifty years of progress in education and rely on inconsistent research of school vouchers is a dangerous path and may leave many disabled, under-privileged and middle-class students abandoned.

Clinical Trials: Optimistic Caution

Published: Lifelines for Health Spring 2017

It is an incredibly hopeful time in the bleeding disorders community! Longer acting products, gene therapy, and subcutaneous injections all seem to be within our reach. The plethora of new treatments truly boggle the mind. Keeping up with the changes of names for manufactures alone is bewildering! Baxter became Baxalta, which then became Shire. Biogen became Biaverativ, Emergent became Aptevo. While multiple new companies are offering emerging therapies in our community such as Spark, Dimension and uniQure. Deep hope has been ignited again for the first time since recombinant product became available. Treatment promises that may yield fewer infusions per month, subcutaneous injections could make self-infusion and ports a thing of the past. Gene therapy has significantly prolonged FIX activity levels, which substantially increases the length of time one may be infusion-free. Each advancement comes with the opportunity to participate in a clinical trial. This decision however, comes with excitement, responsibility, uncertainty and at times - false hope.

Hemophilia has been in my family for over 7 decades and I have heard “a cure in our lifetime” since 1970. I have personally seen the excitement and uncertainty as we seek new treatment that will make our lives more predictable, better, maybe even.... normal? When carrying my first son in 1988, knowing that I was a carrier, all
I knew was that I was carrying a son and he had a 50/50 chance of having hemophilia. Like every newborn, he was miraculous! A preciously beautiful son who looked normal in every way. I dared to hope that he didn’t have hemophilia. Three days postpartum, cord blood results indicated that he did in fact have severe FVIII deficiency. As a family member of one affected by hemophilia, at least I had some knowledge of what life with a chronic condition might look like. Nothing prepared me for being the mom of a child with a bleeding disorder.

In 1992, I was anticipating the arrival of another beautiful child who would be blessed with hemophilia. Prenatal testing had evolved by that point, that via amniocentesis, we were prepared. Once I learned that he was a boy, I knew in my heart he had it. In truth, I was grateful to have two (2) sons that shared the same disorder. Boundaries and rules would be the same for each. There wouldn’t be one who could pummel the other, while the other one couldn’t pummel them back. My salvation was that I was the one who would infuse them! “Choices have consequences” was our family motto.

In 1991, while pregnant with my second son, I was approached about participating in a clinical trial for recombinant factor. He was the last previously untreated patient (PUP) in the world to be enrolled. Looking back at that time, I realized I knew very little about what adverse events could be anticipated. As a community, we were most worried about HIV and Hepatitis. The scramble to be sure that factor products held no human element of plasma was the goal. My first son was tested for HIV every year until his 6th birthday. The anxiety waiting for those results would reach a crescendo.

With a family member impacted by HIV/Hep C, my first concern was that my sons would also be affected. Signing up for a clinical trial seemed like the best possible choice I could make. Decreased risk of HIV/Hep C was paramount. The trial lasted for five years. It required multiple blood tests at a HTC nearly 2 hours from our home. I look back on that time making those long trips, multiple pokes, the fabulous nurse who bribed him with trucks-one for each hand with a sense of pride that we were doing our part to contribute to science and ground breaking research that would benefit others.

Fast forward to 2002, my second son has developed an inhibitor at the age of 10. THIS was a totally different diagnosis than “plain old hemophilia”. An inhibitor was life changing for our family. Our family was now coping with the challenges of an adverse event. In under 50 years we have seen the development of factor in 1970, (a huge improvement over fresh frozen plasma or cryoprecipitate). Factor then evolved from being plasma derived to recombinant, and then multi-generational factors that eliminated any element of human blood. In that time, our community lost thousands of lives to AIDS and Hepatitis C. Those who survived, now manage as many as three chronic illnesses-hemophilia, HIV and Hep C. Recombinant factor saved my sons from HIV/Hep C, which I will be forever grateful for. Yet, in the last 25+ years, we have seen a 25% or higher rise in inhibitors affecting those with hemophilia A and B from mild to severe. Since 2012, inhibitors are the biggest threat to our community today; finally, surpassing HIV/Hep C.

Now in 2017, there are multiple new treatments in clinical trials that heightens our feelings of hope that finally, THIS might be the one to change our lives. It is both an exciting and perplexing time in the bleeding disorders community. On average it takes 14 years and billions of dollars for a new product to be developed. Choosing to participate in a clinical trial can be a challenging decision. At the 2016 Inhibitor Summits, Dr. Tarantino’s and Dr. Kruse- Jaress’ presented on the “Knowns and Unknowns” of current therapies in clinical trials. There is still so much we do not know when it comes to choosing to participate in a clinical trial. Choices have consequences, with positive and negative effects. If you read the adverse effects of aspirin or acetaminophen, the list is lengthy. When participating in a clinical trial,every symptom is reported. On a trial and have a headache? It will be reported. The headache may have nothing to do with being on a trial, but it could be, so it must be documented. It is probably fair to say that every medication has side effects. This is a big decision, one that may not affect just you. As a parent, you are deciding for your child something that will affect them for the rest of their lives. As an individual, you may have loved ones who could be affected by your decision. Only you can decide what is best for yourself or your family. It is all about making the most informed choice you can possibly make, and your ability to trust in it. So, what can you do as an informed potential participant?

Answer: Arm yourself with as much knowledge as you possibly can.

Clinical trial thoughts to examine:

1. What phase trial am I participating in?

2. What is the inclusive/exclusion criteria?

3. By participating in this trial, does it preclude me from ever participating in another?

4. Carefully read the Informed Consent Form that contains:

   1. Purpose

   2. Details

   3. Duration

   4. Required procedures such as lab work and how often needed

   5. Key contacts

5. Is there a patient advisory committee included with this trial and may I participate?

6. What are the risk/benefits?

7. Is my current HTC an Investigational Site? If not, is there still a way I could enroll?

8. Will I be reimbursed for travel if participating in an Investigational Site far from my home?

9. Will study drugs be included free of charge? For how long? Will my insurance company pick up remaining costs of lab draws, office visits, etc.?

10. What is the purpose behind wanting to participate in this trial?

    1. Quality of Life?

    2. A feeling of duty/determination to further research for others?

11. What are your feelings on taking risks?

12. Avail yourself of resources such as clinicaltrials.gov, PubMed https://www.ncbi.nlm.nih.gov/pubmed/, or Wiley Online Library wiley.com. Trial abstracts are available on those sites and if you would like to read the entire journal articles, ask your HTC provider for them or purchase if available.

    13. Read.

    14. Talk to friends, family, clergy.

    15. Write down your questions before visiting your health care professional. They want you to make a well- informed decision as well.

    16. Take your time. There are plenty of opportunities in the pipeline.

    17. Trust your gut.

    18. Read some more.

    19. Be prepared that this may or may not produce the desired affects you were anticipating.