An Archer’s Dedication at Inhibitor Family Camp

Our inhibitor programs are all about pushing your boundaries to try something you may have never done before. Read about Evan Chung’s achievement at Inhibitor Family Camp at Victory Junction. This experience had such a profound effect on him, he wrote an essay about it! Way to go Evan-you demonstrate such determination! We are proud of you! -Janet Brewer

Published: Lifelines for Health Spring 2018

By: Evan Chung

I was running down a steep hill at 6:30pm. I was going to archery at Inhibitor Family Camp at Victory Junction. My dad and I came there together. My dad and I had to wait for a while. Finally, I got a spot. It was pretty windy there. I was feeling pretty excited because I just got a spot, then I tried reaching a bow, but I couldn’t reach one because I wasn’t tall, and I was only 6 3⁄4 years old. I tried many more times, but I couldn’t make it. Then my dad took one bow down, and I said I wanted a different bow. But he said it’s not my size and he also said the one he picked was the right size for me. Dad picked a blue bow.

Then came Larry the archery councilor at camp. He said to my dad “good!” I don’t know why. Then Larry gave me twelve arrows. Then he told me how to do archery. Then he said to try to get a bullseye which is when you get your arrow in the middle of your target. Once at a different camp called Camp Bold Eagle, there was archery too, but I never got a bullseye there. In fact, I never got a bullseye, ever. Anyway, I was really determined to even get one bullseye because my dad told me if I get one bullseye, I can get a gold medal from Larry.

Anyway, I shot my first arrow. Really surprising! I did better than last time! Then I shot my next arrow. Then I started going crazy because I just got my arrow in the red part of the target. My dad said, “calm down, Evan” so I calmed down, but in my thoughts, I just kept going crazy. That caused me to waste some arrows because I was going crazy. I only had three arrows left. I shot an arrow. Red part. I shot another arrow. Blue part. Worse. It was my last arrow... and I got the red part. I sighed. “Parent, pick-up!” shouted Larry. Then all of the parents went to pick up the arrows. They also pulled them off the targets.

It was almost lunch time, so I thought I’d better hurry up. But I didn’t waste any arrows. I did my first shot out of twelve arrows. Blue part, bad. Then I shot my second arrow. Red part, not that bad! Well now let’s skip to the ninth arrow. I got it in the yellow, but it was not a bullseye. I was on my second to last arrow. I took a deep breath, then set up and shot. Then... “twang!” It hit right in the middle of the bullseye! Then I started going really crazy. I shouted, “Larry! Larry! I got a bullseye!” Of course, he didn’t hear me because he was working with another kid. Then I shouted again. This time he heard me. He gave me a gold medal. It was the first medal I ever got. I felt really proud. “I did It!”, I thought. Dad said it was time for lunch. Then we ate lunch. I played with my medal. I felt really proud.

New Inhibitor Treatments: Weighing Risks and Benefits FEATURE

Published: Lifelines for Health Spring 2018

By: Dr. Jonathan Bernstein, Md

Over the past several years, hemophilia care has been turned on its head as new opportunities and ideas for the treatment of hemophilia have emerged. During this time, we have seen the greatest change in hemophilia care since Judith Graham Poole first helped to develop cryoprecipitate and observed that Factor VIII could be made cheaply from this product. These new changes include new factors, new bypass products that can be used subcutaneously, and gene therapy. Finally, we now may have two new ways to treat bleeding diseases that have no treatment at this time.

With these new treatments come choices where we must weigh the risks versus the benefits of these drugs.

Sometimes it is hard to know what the risk might be when a drug is first being used to treat patients. We can look at the research that has been done to test the new drug in a well-defined protocol and see if any significant adverse events were reported.

However, research protocols are strict and can have no variance. When a drug is available for use in the medical community, the way it is used by doctors and patients often differ from the strict rules for use that were specified by the research protocol. How many of you have not used your medication in the way you were instructed by your doctor? I know I have – but, of course, doctors are the worst patients. When patients and doctors vary in how they use a drug, this leads to increased risk of something bad happening, and possibly death. Because of our concerns when we first introduce a drug to our patients, we tend to be more conservative in prescribing these drugs. Companies are now required to do what is called “phase 4 research” to try and capture the changes in use over time of a drug or treatment.

Hemlibra

The first of these newer drugs, Hemlibra, had problems early on with one death, and the development of both microangiopathic hemolytic anemia and blood clots. These adverse events occurred when Hemlibra was used with a high dose of FEIBA. This combination of a drug that activates Factor Xa (Hemlibra) and a drug containing both Factor Xa and prothrombin (FEIBA), at high doses seems to activate compliment and cause increased clotting and the development of microangiopathic hemolytic anemia (which always seems to involve compliment activation). With further studies, Genentech, the company that developed and tested this drug, was able to eliminate these serious complications, by decreasing the dose of FEIBA, or by avoiding its use completely. Many of you know that 5 patients have died who received this drug off study under compassionate use. While it is stated that these patients’ deaths were not caused by Hemlibra, it is curious that so many deaths occurred in the Factor VIII inhibitor group, when so few were exposed to the drug. Little information has been released on the causes of deaths in these patients. We also have no data on whether we can tolerize patients while on these drugs. Without tolerance, these patients may never be allowed onto gene therapy protocols, as they might have a greater risk of negative outcomes.

Hemlibra has also solved many of the problems that patients with Factor VIII inhibitors have. Patients on this drug are able to give themselves subcutaneous (SQ) shots once a week, with few if any breakthrough bleeds. Many patients who were bleeding once or twice a week are now going from a month to years without bleeding. This decreases the damage to and joints, improves quality improves compliance (much give a subcutaneous shot), and patients more independent. deciding whether to use this must make a choice weighing risks versus benefits. Patients who are doing well on current treatment with tolerance of their inhibitors, should probably avoid Hemlibra. However, for patients with higher titer inhibitors or frequent breakthrough bleeds, this drug’s benefits will most likely be greater than the risks as long as patients follow instructions for use.

Fitusiran

Another drug on the horizon is Alnylam’s/Sanofi’s Fitusiran. This drug increases the thrombin level in the blood by using small interfering RNA (siRNA) to silence the ability of the liver to produce Antithrombin III through RNA interference. It is similar to a process that the body uses to decrease production of certain proteins. This increases the thrombin level in the blood making up for the level lost when one is deficient in a factor. It can be used in patients with inhibitors of factors VIII and IX, as well as to treat patients with factor deficiencies and platelet disorders that can cause excessive bleeding. It is also given subcutaneously. It is currently being studied in a clinical trial protocol at this time. There has been one death in a patient with hemophilia A. This is thought to have occurred when the patient had a sinus venous thrombosis, which then hemorrhaged, resulting in the patient’s death. Protocols and education were put in place to decrease the chance that this would happen again. The decision to use this drug must be decided by both the patient and the physician after balancing risks against benefits to decide if the risks are greater than or less than the benefits. In patients with factor IX deficiency and an inhibitor, this would allow bypass and improve clotting greatly. In patients withwith no treatment other than antifibrinolytics, such as Factor V patients, as well as severe platelet disorders, such as Glanzmann’s Thrombasthenia and Bernard Soulier, Fitusiran may greatly improve quality of life and outcome.

Hemlibra and Fitusiran may also both make it hard to measure Factor VIII and inhibitors as they both bypass what we usually measure to determine levels. New methods must be discovered that will allow us to find the levels in patients on these drugs.

Gene Therapy

The next set of treatments on the horizon are gene therapies for Factor VIII and IX deficiency.
Both will soon be available. Studies are ongoing with increasing success. Gene therapy will only be offered to individuals above the age of 18 for the foreseeable future. This approach has been a long time coming. However, there are still substantial risks associated with gene therapy. In the past, patients (with disorders other than hemophilia) have developed cancers when treated with a retroviral vector (viral DNA that carries the gene into the cells). Genes carried by these vectors into the DNA often inserted themselves next to genes that allowed unrestrictive division, a hallmark of cancer. The improved treatments that were then developed avoid this problem by either not inserting into the genome or having more specific insertion. This is a vast improvement. One company estimated that only one in ten thousand would insert incorrectly. However, there may be millions of these vectors, that will have to be given. This might mean that several hundred could insert incorrectly. If this happens, it is possible that years later a patient could develop a malignancy – the most likely site of cancer would be the liver as these vectors insert the changes into liver cells. Next, although less worrisome, is that with time a patient who has undergone gene therapy may need to have boosts of the vector to maintain success. However, it is possible that patients might develop antibodies to the vector so that they are no longer candidates for repeat boosts of the vector.

Some patients may not be candidates for treatment with gene therapy. If one has an antibody to the vector the body may reject the treatment. Some companies are attempting to develop several vectors that would avoid this problem. If a hemophilia patient has an inhibitor it could either cause the inhibitor level to increase, or could allow the immune system to see a constant amount of factor and tolerize these patients. I believe that we will see both occur. Plasma-Derived Factor

Finally, a trial comparing plasma Factor VIII to Recombinant Factor VIII showed that plasma
Factor VIII was less likely to cause inhibitors than Recombinant Factor VIII. However, much has changed since the study was started. New factors, some of which had longer half-lives, became available. These factors were not compared to plasma. It is believed that humanized factor VIII or extended half-life Factor VIII may develop as few inhibitors as plasma does, and these may also be as good at tolerizing patients as plasma is. If studies prove this to be the case, we may find that more patients convert to these drugs.

Thus, we now have a wealth of new treatments available with many others on the horizon. There
will be risks and benefits, as is true of all drugs, even relatively benign drugs like acetaminophen or aspirin. When patients are given a chance to treat themselves with these drugs, they must weigh the risks against the benefits and decide whether to use the drug. They must also follow the instructions given to them by their doctors and the drug companies so that they can avoid side effects as much as possible.

It’s Easy! How WE Can Report an Adverse Event

by Jane Cotter Forbes

Published: Lifelines for Health Spring 2018

There are many medications for people with bleeding disorders in the marketplace today and our pipelines are bursting with new therapies and medications. Some medications have been available or many years while others are newly FDA approved. If you or your loved ones experience a serious adverse event (AE) while using a bleeding disorders medication, it is always a good idea to voluntarily report an AE to the Food and Drug Administration (FDA) MedWatch consumer voluntary reporting program. AE’s should be reported as soon as possible to MedWatch using Form FDA 3500B by those who are consumers and medical personnel for the following situations: death, life-threatening medical events (e.g., anaphylactic reactions), AEs that require hospitalization (short or long term), disability or permanent damage, congenital anomaly and/or birth defect, and any required medical intervention to prevent permanent impairment (e.g., hemorrhage). If you are not sure if the AE is related to the medications you are using, it still is a good idea to voluntarily submit a report to MedWatch just in case. Mandatory reporting is required by law by user- facilities, importers, distributors, and manufacturers. AE’s for these organizations need to be reported to MedWatch within 24-48 hours. In considering the filing of a voluntary report for an AE to the FDA, here are some points for guidance!

IT IS EASY. First you go directly to the FDA MedWatch website [https://www.accessdata.fda. gov/scripts/medwatch/index.cfm?action=reporting. home] and click on form 3500B. Then you start filling in the blanks. When you are done, you hit send. In particular, the website asks for a lot number, an NDC number, the strength, the units, and the quantity (how many doses or vials of the medication were used).

Most of this information can be found from the container of the medication and/or on the prescription label. The website asks for a detailed description of the AE or serious problem as well any and all medical reports (if available). You will need to include a list of all other medications in use. This website also suggests not throwing the medication away because the FDA may request what remains for further inspection.

In addition, medication that comes with faulty equipment or devices such as needle breaking or ineffective transfer device to be reported. If you are not able to use the website addressed herein, you are free to call the FDA at 1-800-FDA-1088 in order to file a report. “The FDA encourages patients to report AEs as soon as possible. If the AE team gets a cluster of reports about the same drug in a short period of time, it will be able to respond more quickly.” IT TAKES ONLY A FEW MINUTES. Depending on the details of what you submit and the explanation of the AE, it will mostly likely take only 10 to 15 minutes to complete.

IT IS IMPORTANT. It is the fundamental way by which American people can alert the FDA of any
and all AEs after having taken a pharmaceutical medication. This information is gathered, reviewed, analyzed, and a determination is readily made if further action is needed. All AE reports are kept in the FDA database.

IT IS APPRECIATED. The FDA truly welcomes this information, for it provides ready and immediate feedback on medications which they have approved. It is an essential feedback mechanism. The FDA MedWatch system also allows for consumers or patients to report voluntarily and independently from their doctors. The FDA “understands that for a variety of reasons, you may not wish to have the form filled out by your health care provider, or your health care provider may choose not to complete the form.

IT IS REWARDING. There is a genuine sense of personal fulfillment in having participated in this safety program for not only the American people, but for people worldwide, who might also have access to such medications. It is a tangible means by which we can protect ourselves from harmful medications.

IT IS FOR EVERYONE. All Americans using prescription drugs approved by the FDA are encouraged to report all adverse events or serious problems.

SO WHAT HAPPENS AFTER AN AE REPORT IS FILED?

The FDA reviews all of the submitted MedWatch reports for safety signals - similar adverse events

occurring with a particular drug. Some examples include:

• A new serious AE which is not listed in the drug’s package insert

• An increase in the reporting rate of an event already occurring with a drug

• A new report of how an FDA approved drug interacts with other medications.

• A product’s name, packaging, or labeling leading to an AE needs to be adjusted, relabeled, and added safety information needs to be included.

Depending on the information found during the FDA’s review of the safety signals and the severity of the AE, the FDA may decide to:

Update the drug’s package insert or labeling

• Place restrictions on the drug’s distribution

• Release a drug safety communication to inform the public

• Withdraw the drug from the market

A Mitigating WHAT?

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

Published: Lifelines for Health Spring 2018

Child Find

Child Find is part of the 504 law in which a school district is responsible for evaluating students who have a suspected disability to determine if they need related services and accommodations under a 504 plan. This extends to students in private schools and who are homeschooled. The district is responsible for evaluating and offering appropriate services and accommodations in the public school setting if a private school student is identified as having a disability. Parents whose children don’t attend public schools may not understand what kind of help and services are available.

Section 504 of the Rehabilitation Act of 1973

Section 504 was initially enacted as one part of the American’s with Disabilities Act. It PROTECTS students with a disability and requires public institutions who receive federal funding. A plan is developed to facilitate EQUAL access to a disabled student as a non-disabled student.

Section 504 was enacted to protect qualified individuals from discrimination based solely on their disability. Whether a particular student is protected under Section 504 requires a determination that the student is an
“individual with a disability” and that the student is “qualified” under Section 504. A student is “disabled” under the Section 504 regulations if the student meets any one of the three criteria listed in 34 CFR §104.3(j)(1). An individual is considered disabled under Section 504 if the individual:

1. has a physical or mental impairment which substantially limits one or more major life activities (SEE table 1 for “Major Life Activities”),

2. has a record of such an impairment, or

3. is regarded as having such an impairment.

“Substantially limits” is measured against their same age, non-disabled peers in the general population without the benefit of medication or other mitigating measures such as accommodations.

Table 1 (Major Life Activites)

“Major life activities, as defined in the Section 504 regulations at 34 C.F.R. 104.3(j)(2)(ii), include functions such as caring for one’s self, performing manual tasks, walking, seeing, hearing, speaking, breathing, learning, and working. This list is not exhaustive. Other functions can be major life activities for purposes of Section 504. In the Amendments Act (2008), Congress provided additional examples of general activities that are major life activities, including eating, sleeping, standing, lifting, bending, reading, concentrating, thinking, and communicating. Congress also provided a non-exhaustive list of examples of “major bodily functions” that are major life activities, such as the functions of the immune system, bleeding disorders, normal cell growth, digestive, bowel, bladder, neurological, brain, respiratory, circulatory, endocrine, and reproductive functions.”

Children that have bleeding disorders have the right to a “free and appropriate public education (FAPE)” regardless of the type of disability or the severity. A 504 plan lasts for one school year and MUST be reviewed/ updated annually. A 504 plan protects your child and ensures that the school and school staff are implementing all accommodations your child requires!

What should be in a 504 plan?

The plan should outline what accommodations a student needs
to access academics (instructional supports), the physical building space, transportation, testing (standardized and regular classroom), behavior etc. This is to ensure that students with
a disability have equal access to all education as their non-disabled peers. For students with bleeding disorders, it is imperative that part of the 504 plan addresses self-management of health conditions in the school setting or school-sponsored events (field trips, dances, field day etc.). Additionally,
if self-management is unattainable, the 504 plan should address how the student will have access to health management and by whom. It is also important that the plan address how the plan will be monitored and who the school contact person is.

Many districts across the United States find it easier to say to a family that their child with bleeding disorders
should not participate in physical education or recess. This is a civil rights violation! Your child has
access to all the activities that their non-disabled peers have access to. It is recommended that a 504 plan
specifically addresses physical activity as one part of the plan. For example, a student with a bleeding disorder might consider joining a swim team but not a football team, or can play golf but not soccer. It’s imperative that the 504 plan be a specific as possible!

The 504 Plan would define the signs and symptoms of your child’s bleeding disorder (i.e. external bleeding, swelling, tingling etc.). Additionally, it should address an emergency care plan for the school about what should happen and who is responsible in the case of a bleed. There should be a coordinated plan between home and school regarding specificity around what to do with a bleed. There should be listed contact numbers for Hemophilia Treatment Center’s (if used) and/or pediatrician, hematologist and parents along with any other pertinent members of the Team. It should discuss any accommodations necessary for the student to access the building (wheel chair accessible, elevators etc.) and who is responsible for keeping teachers and staff (including substitutes) up-to-date on the students’ 504 plan and/or health care plan. Often times, a 504 and health care plan overlap. Some districts insert the health care plan into their 504 document and vis a versa.

Mitigating Circumstances

The Americans with Disabilities Amendment Act of 2008 updated eligibility based on positive mitigating circumstances. They ruled that the determination of a disability must focus on whether or not the student would be substantially limited in performing a major life activity REGARDLESS of what the mitigating circumstances are. Mitigating circumstances are things like medication (i.e. Factor), medical equipment/devices, mobility devices etc. For comparison, refer to table 1 on pg. 30. The law also notes that it can take into consideration the negative mitigating circumstances which is considering the side-effect or burdens of the mitigating circumstances and whether there remains a need for the accommodation.

Post-Secondary 504 Information

ADA rights carry into the college setting. However,
it is the responsibility of the student to self-identify and request appropriate services to staff. It stays as confidential information and can not be used against the student. Generally, there is a Disability Services office on all college campuses. One thing to keep in mind, is that unlike 504 plans in public schools, there is not a written plan. The college student needs to advocate for himself/herself and provide documentation that can help the disability services center provide the accommodations needed. Although it is not a written plan per se, students have the rights to the accommodations needed!

The Access to Marketplace Insurance Act: Allowing Charities to Be Charitable

by: PSI, Mandy Herbert & Jim Romano

Published: Lifelines for Health Spring 2018

Patient Services Incorporated (PSI) is leading the effort to pass H.R. 3976, The Access to Marketplace Insurance Act, and protect the valued safety net of patient assistance. This legislation is in response to a regulation issued in 2014, during the implementation of the Affordable Care Act (ACA), that allowed Marketplace insurance companies to deny charitable third-party premium and cost-sharing assistance. The rule essentially provided a permission slip to health insurance providers to prohibit assistance by charities looking to help patients in need in order to shift sicker patients off their plans.

Since 2014, the impact of this regulation has been considerable, with approximately 90 insurance plans in 43 states having implemented the prohibition. This rule allows insurers to evade the reforms of the ACA removing the pre-existing condition exclusions and provides an open route to discriminate against
patients with higher healthcare costs simply because they receive assistance. Taking these discriminatory practices, a step further, health insurance providers have also attempted to expand this prohibition into other markets including the Medicare Supplemental Insurance Market.

In response to this misguided policy, PSI created the Marketplace Access Program (MAP) coalition to bring together leading patient advocacy groups and patient assistance organizations dedicated to protecting charitable assistance for individuals suffering from chronic and life-threatening illnesses. The focal point for our coalition’s work is H.R. 3976, federal legislation that would require insurers to accept assistance from non-profits, places of worship, and local civic organizations. PSI has worked closely with H.R. 3976’s sponsor, Congressman Kevin Cramer (R- ND), to develop this legislation and stop this harmful policy from continuing.

We have made great progress on the bill, garnering over 130 cosponsors since introduction in October.

PSI would welcome any assistance from readers who would like to get involved. If interested, please contact PSI at jromano@uneedpsi.org or (804) 521-7908 for more information.

Clinical Trials: For Inhibitor Patients

by Ann Marie M. Minichiello

Published: Lifelines for Health Spring 2018

As research and medical advances progress, it is important to recognize that this momentum is not possible without the involvement of patients. Without clinical trials, new drugs and treatment options cannot be made available to those who would benefit from them. Presently, there are six clinical trials recruiting patients with hemophilia and inhibitors. The following studies are all “interventional” (clinical trials) with the primary purpose of treatment. The information on each of the six trials begins with its official title and drug being used.

1. Individualized ITI Based on Fviii(ATE) Protection by VWF (INITIATE) with Wilate® began on June 1, 2017 and is estimated to be completed by December 1, 2022. Requiring 120 participants, the primary goal of this trial is to compare the outcome of individualized lot selection to random lot selection using Wilate® for immune tolerance induction (ITI). The principal investigation site for this study is the University of California, Davis. (Sponsor: UC Davis)

2. A Non-controlled, Open-label, Multicenter,Study of Efficacy of rFVIIIFc for Immune Tolerance Induction (ITI) in Severe Hemophilia A Subjects With Inhibitors Undergoing the First ITI Treatment , utilizing Biological: rFVIIIFc, began June 30, 2017 and is estimated to conclude December 2020. There are 30 participants needed for this study to describe the time to tolerization using rFVIIIFc within 12 months of ITI. There are 9 sites recruiting. (Sponsor: Bioverativ Therapeutics, Inc.)

3. A Phase III Study on the Safety, Pharmacokinetics and Efficacy of Coagulation Factor VIIa (Recombinant) in Congenital Hemophilia A or B Pediatric Patients From Birth to <12 Years Old With Inhibitors to Factor VIII or IX: PerSept 2, using Biological: Coagulation FVIIa (Recombinant), began in December 2015 and was estimated to conclude August 30, 2017. Requiring 24 participants, the goal of this study is to assess the safety, efficacy and pharmacokinetics of 2 separate dose regimens of Coagulation Factor VIIa (Recombinant) for the treatment of bleeding episodes. The principal investigation site is the University of Colorado, Denver, with 2 additional sites. (Sponsor: LFB USA, Inc.)

4. ATLAS-INH: A Phase 3 Study to Evaluate the Efficacy and Safety of Fitusiran in Patients With Hemophilia A or B, With Inhibitory Antibodies to Factor VIII or IX is utilizing Fitusiran, recombinant Factor VIIa and FEIBA. This study began January 8, 2018 and is estimated to

   be completed in July 2019. Requiring 54 participants, the goal is to determine the frequency of bleeding episodes in patients receiving Fitusiran for prophylaxis, compared with patients who continue with their regular medication. There are 5 sites recruiting. (Sponsor: Alnylam Pharmaceuticals)

5. A Phase IV, Multicenter, Single-Arm, Open-Label Study of Emicizumab (Hemlibra) Prophylaxis in Patients With Hemophilia A and Inhibitors Undergoing Minor Surgical Procedures with Emicizumab began March 31, 2018 and is estimated to conclude June 28, 2019. 30 participants are needed for this study to evaluate whether participants receiving Emicizumab prophylaxis can safely undergo minor surgical procedures without prophylactic bypassing agents. There are 3 sites presently recruiting. (Sponsor: Genentech, Inc.)

6. A Phase III, Multicenter, Open-label Study of the Efficacy and Safety of B-Domain Deleted Recombinant Porcine Factor VIII (BAX 802) in Subjects With Congenital Hemophilia A With Factor VIII Inhibitors Undergoing Surgical or Other Invasive Procedures with Biological: Antihemophilic Factor (Recombinant), Porcine Sequence (BAX 802), began December 22, 2016 with an estimated completion date of June 1, 2018. There are 15 participants needed for this study to evaluate the efficacy and safety of BAX 802 in patients who are undergoing surgical or other invasive procedures. There is 1 site recruiting in the U.S. (Sponsor: Baxalta, part of Shire)

The safety and validity of a study is the responsibility of the sponsor and investigators. As with any clinical trial, please consult your healthcare provider (HTC) before participating. For more information, please visit www.clinicaltrials.gov.

PTSD and Chronic Health Conditions

by Krystyn Strother

Being diagnosed with a medical problem can be distressing, even traumatic. The experience of trauma is entirely subjective but it can lead to post-traumaticstress disorder (PTSD). Let’s first look at the definition of PTSD and Trauma:

Trauma is defined by the American Psychological Association (APA) as an emotional response to a terrible event.1 That’s a pretty broad definition and can be entirely subjective.

PTSD or Post Traumatic Stress Disorder, is described as an intense response to trauma and can create severe distress and can limit one’s ability to function in life.2 PTSD from chronic pain or illness is very real and if you think about it, makes a lot of sense.3 Chronic illness and pain are all at once traumatic to the body, brain and emotions, and the level to which people experience symptoms are vast. Some folks dowell, while others struggle to get out of bed each day.

One of the tools that we possess to cope with trauma is to work directly with the brain using mindfulness. Science has shown us, through a huge number of studies, that the brain does not become static once it reaches maturity. It continues to change based on our experiences and actions by way of neuroplasticity. They gray matter in our brain can get bigger or smaller; neural connections can become stronger or weaker. Every time you learn a new dance step you are creating a new neural pathway.

We often hear about neuroplasticity in relation to using techniques to heal from injury, cope with chronic pain, deal with stress and anxiety. While creating new neural pathways does help us deal with these things, it can also work against us when we have negative experiences. When trauma is encountered, the brain creates new pathways and changes its structure in response to the event in a way that does not serve us well.4

There is a significant amount of data that supports using mindfulness as treatment for those affected by trauma and PTSD.5 Practicing mindfulness reverses the neurological patterns taking place with trauma and PTSD by decreasing areas of the brain that are more active in stressful situations and increasing activity in areas of the brain that regulate our emotion and decision making.6 Do a google search for “mindfulness brain scan” to see side by side images of brain function before and after practicing mindfulness.

It is important to realize and reiterate that fear or anxiety around procedures, infusions, or other pokes and sticks is nothing to be ashamed of, and trying to get yourself or your child to accept an “it’s no big deal” attitude is often not the answer, especially if there is trauma and PTSD involved. Fortunately, there are tools that we can use to help our kids, and ourselves, become more comfortable. Practicing together as a family will help those with fear, anxiety, or trauma feel less alone and supported.

1. Progressive relaxation

   A. Tense the muscles of your body (arms, upper body, legs) and hold for a count of 10.

   B. Release the tension and relax to the count of 20.

2. Belly breath

A. Relax your face and place one hand on your belly. Take long, slow, deep breaths in through your nose and out through your mouth. Feel the belly rise as you breathe in and gently drop as you breathe out. Don’t force the breath, let it be easy. Do this for 5 or more breaths.

3. Fear wave

a. This is helpful for those that may have a fear of needles or experience anxiety around pokes and sticks. This can also be used as a template for other procedures.

We begin by noting what it is that makes us the most anxious. Rather than tackle the big stick first we can start with small things. Use the chart below and have your kiddo rank what scares them the most. This one is filled out as an example.

Begin with the least difficult situation. You can visualize this situation as an island out at sea. You slowly paddle out in your boat or surfboard (your kid’s watercraft of choice) toward this point. If anxiety begins, hold where you are in the water and try a few rounds of progressive relaxation or belly breath. The anxiety doesn’t have to go away completely but it will begin to lessen and become more manageable. Continue moving towards the feared situation. Each bout of anxiety or fear is like a wave. It builds and intensifies and then it will gradually reduce. Staying with the fear allows us to see how anxiety makes us feel and shows us that it is only temporary. Ride the waves of the sea until you find yourself at the shore of this particular island.

When your child feels confident with one situation, move onto the next. Eventually, with visualization practice, there will be smooth sailing towards these situations. This practice changes the way the brain reacts to the actual fear inducing situations when they arise. Overcoming your fear will take some time and practice, but in the long run, it will make life less stressful and you will feel less anxious.

Rewarding Surrender

by Stephanie Shea

Published: Lifelines for Health Fall 2019

When I first heard about the MUMS event (Moms’ ‘Uninhibited Meeting), I made up a zillion reasons why I wouldn’t be able to go. The dates weren’t right with tentative summer plans we had already made, it was too close to other events we had already committed to, my son might need me (his port had been acting up), a friend had mentioned she may potentially come to town that weekend....yada, yada, yada. Excuses. All of them.

The truth was, I was scared to leave my 5-year-old son. My husband is amazing and completely capable of taking care of our two boys, daily factor infusions and all. But if something did happen to my boy, I would want to BE there - to reassure and hug and comfort and do all those things that us mamas do. But it’s often all of those things that us mamas do that can lead to burnout and feeling depleted. And when we’re exhausted and
not taking care of ourselves, it’s often the case that we don’t have anything left to give to those we love around us.

Being a caregiver for someone with a chronic condition such as hemophilia can be frustrating
at times and exhausting. It’s often emotionally, physically, and mentally draining. It may limit your social life and can cause financial problems. Burnout occurs when the stress and burden from these negative effects become overwhelming, negatively affecting your life and health.

When I received the email about the MUM event,
I was definitely experiencing burnout. Before I had children, I was a professional counselor/therapist,
so I was very aware of the signs and symptoms. Yet I still had a hard time agreeing to the trip. My husband finally -lovingly- nudged me to commit to it (and I’m so grateful he did!)

The event was held at a beautiful hotel in downtown Santa Fe. When I first arrived and checked in to my room, I felt at a loss. What do I do with myself when I don’t have two little humans to look after?! I realized my predicament and committed to sinking into that uncomfortable space for the weekend in hopes of not only recharging my mama batteries but rekindling my relationship with myself.

The event started with a welcome session before dinner as everyone began trickling in from different places all over the U.S. Since I was one of the first to arrive, I took the time to chat with some of the other moms. And then I had a very happy surprise when I met someone with whom I had only ever connected with online. She was my first ever hemophilia mom friend and she reached out to me online through social media mere days after my son was first diagnosed (when my head was still spinning from the shock). She was a huge support to me then and I was thrilled to finally meet her in person. And I had no idea she was going to be there, such an awesome surprise!

Connecting with other moms at the event was definitely the highlight of the weekend for me. Hemophilia can be such an isolating condition to live with and when you throw an inhibitor into the mix, it can feel even more isolating within an already isolated community. I feel fortunate because I do have a wonderful group of friends and tons of support outside of the hemophilia community. Yet, sometimes I still feel isolated. When we’re going through a lot of hemophilia related issues, it tends to be forefront on my mind. And if I don’t talk about it to the people who I spend my time with, I don’t feel authentic or as if I’m able to truly connect. And although I have friends who will listen and provide support, there’s a part of me that knows they will never truly understand what I’m experiencing.

That is not the case with hemophilia moms and especially other mamas of kiddos with inhibitors. I often feel an automatic connection, a knowingness that we truly get what it’s like to walk in the others’ shoes. I also often learn more from other members of the community than I do from medical professionals and this weekend was no exception. Connecting with other mothers has been so incredibly healing for me and the MUM event was invaluable for that.

We had a “rap” session that was a bit of a free for all in terms of subject matter. We talked about the things that we felt were necessary to talk about. We talked about the biggest challenges that we face in raising a child with intense medical issues. We discussed new treatments and how we feel about them. We spoke about how we make decisions for our children and how we advocate for them. We talked about how we manage (or don’t manage) the stress we face in our lives. We spoke about anything and everything related to mothering a child with an inhibitor and it was very satisfying and therapeutic.

After dinner on the first night, most women walked a few blocks into downtown Santa Fe and checked out the sights. There was live music and wine-tasting and art and antique cars and all sorts of interesting things to see and explore. But the best part of the night? And the best part of the entire event? Simply being with other mothers who know what it’s like to have the extra challenges of raising a child with hemophilia and inhibitors. Thank you CHES for allowing me this opportunity!

Taking Time On

by Donovan Guerrero

Published: Lifelines for Health Fall 2019

Action. The willpower that it requires to take risks is vital to growing as a person. Sometimes I struggle with this. I think it is imperative for young adults like myself to do this to gain independence.

This past September I was blessed with an opportunity to apply for a program called LeverageSM. I had heard very little about this camp specially designed for adults with inhibitors. My mother actually filled out part of the application without my knowledge. I was shocked to get a call from Eric at CHES, never having met him, asking if I’m still interested in the program. I was unfamiliar with the two facilitating organizations, GutMonkey and CHES. Within three weeks of that phone call, I was packing my gear and preparing to go to Portland, Oregon.

The first lesson that I learned was the elephant-rider- path-theory (a concept that explains the conflicting struggles within all of us. Developed by the Heath brothers in a book called Switch, it’s the idea that everyone has a figurative elephant along with a rider inside him/herself. The elephant (relative to our emotions) is often controlled by the rider (our thought of logic.) But at times, our elephant (or emotions) can derail from its path by outside factors. And at this point, it’s our rider’s responsibility to motive our elephant to return to its path.) This lesson resided with me the most. There was not one significant moment that I could point to as being a pivotal moment during this program. I think it was more of an accumulation of different challenges and tasks that I can focus on. To name a few in particular, would have to be the rock-climbing tower, building didgeridoos, and the high ropes course. I felt as though these were the biggest barriers I overcame. From the burning of my forearms on the tower, frustration of my didgeridoo cracking all over, and recovering from the splits on the stirrups obstacle on the high ropes course. My elephant wanted to give up – tell me to stop climbing, stop trying, stop caring. But the elephant-rider-path-theory really pushed me to succeed.

Personally, I think about when I do great things or go to amazing places, I always end up asking myself the same question when I return home: “What’s next?” For example,
I like setting goals. One goal I made a few months prior was to get my boating license so I can operate a boat that has 25 horsepower or more. I’ve almost completed the course currently and hoping to pass the test soon after. My current boat does not require a boating license since it only has 7.5 horsepower. I did it to learn the “rules of the road” so to speak and so I’m already ahead of the game when I get a big boat in the future. When the time comes, I’m sure I will evaluate my decision and think to myself, “Yay, you passed the test!” Then when I buy a nice boat, I will think, “Well, now I guess I should take a fishing trip to Alaska for salmon, or Florida for smallmouth bass, or even the Great Lakes for perch and crappie.”

It’s that drive that I have to learn and share. For me, I want to keep setting goals and I don’t want to meet them,
I want to surpass them. To go above and beyond. Like the difference from getting an “A” or a “B” on a test. I’m at a point in my life where I graduated high school and I’m not attending college this year. I want to go next year but I wanted to take time off and become a more mature person, learn more on my own and gain independence, and work to save up for college. All of these facets would contribute to working on my mental health as well. What I really want to do is take the remainder of the year head on. Take a “gap year” on. Leverage definitely gave me the support and the confidence to do that. I have big plans ahead of me. Taking action to put them in motion is the crux of the issue. I want to be a leader in some fashion.

Thank you to Takeda pharmaceuticals for making this event possible. Thank you to CHES and GutMonkey for putting on a great Leverage 2019.

Camp Fosters Healing, Friendship, & Play

by Stephanie Shea

Published: Lifelines for Health Fall 2019

In June, we had the opportunity to attend CHES' After the ShockSM inhibitor camp. It was a hard decision for my family as it happened to fall on the same weekend as our local chapter’s annual camp. Although my 5-year-old son Dakoda’s inhibitor isn’t “active”, we decided to go to After the Shock as we felt we might relate more with this community. And I’m so glad we did. One of the biggest blessings that we have found on our hemophilia journey has been the community....and the love and support we get from them. At times, we have felt isolated among the “normal” hemophilia population. Not all, but many that I’ve connected with, seem to infuse factor prophylactically a couple to a few times a week with no issues and they have been fortunate enough not to experience any major traumas.

Those of us that have experienced inhibitors know that it’s an entirely different ballgame for us. Although Dakoda’s inhibitor has been considered partially tolerized for almost 2 years, his half-life is very short, so he still needs daily infusions, aka, needle pokes. Daily infusions can definitely take their toll on a family, not to mention the task of emotionally healing from past traumas (head bleeds, prolonged bleeding from ports, joint bleeds, etc.). Most families with inhibitors know this path all too well.

On the first day of camp, we learned that every family was assigned a camp “buddy” who would stay with the kids for most of the day, even while parents went to educational classes. On the first day, my husband and I were on the tennis court, watching Dakoda and his little brother Phoenix “play” tennis. We started talking with a father who we’ve met before at other events. Dakoda and Phoenix wanted to run to the upper fields to play soccer with their camp buddy.

The other father we were talking with could clearly see the anxiety on our faces (as we have very rarely let Dakoda be with anyone other than us or one or two very trusted family members). The father of the boy was several years older than Dakoda and he shared his own experiences with anxiety. He encouraged us to let go and trust that Dakoda would be ok without us. During the conversation, my husband and I both teared up and were greatly touched. It set the stage for us to loosen our grip for the rest of the weekend and it felt like a turning point in both of our personal healing journeys.

Everyone in the inhibitor community seems to just “get it”. They get that we’ve all seen our children in pain, we’ve all seen them suffer and we’ve all been through a lot of trauma, which often results in fear and anxiety.

Aside from the immense emotional value we received from talking and sharing with other parents, the camp itself was incredible. Set on a lake in the

Pocono mountains in Pennsylvania, the scenery of Camp Zeke was breathtaking. We were surrounded by lush green trees and grass and it was so nice to be immersed in nature. We have attended many national and local events in the hemophilia and inhibitor community, and they have all taken place indoors, mainly in small windowless classrooms. The difference at After the Shock was staggering. The kids were able to be how kids are meant to be - running carefree and playing in nature. Even when the parents were in educational sessions or talks, the rooms had huge windows overlooking the lake and giant trees, giving us the sense that we were in a forest.

There's so much to do at Camp Zeke, that there was never a dull moment. There was the lake, of course and
all the activities that came with it (kayaking, fishing, swimming) with an outdoor pool, nature trails, a full theater, art studios, and so much more!

The educational sessions were great too! In addition to Current Treatment of Inhibitors and separate “rap sessions” for the Moms and Dads, there was also Aqua Therapy and Yoga for Relaxation. My personal favorite though was

The Bio-psychosocial Effects of a Chronic Condition. The speaker was knowledgeable about trauma as well as the intensity and daily challenges that come with managing a chronic condition. I feel that both my husband and I greatly benefited from this session. We took home tools and techniques that have enhanced our lives, especially in times of stress and anxiety.

When recalling the weekend, we spent at After the Shock, there was a special incident that stood out for me. Dakoda wanted to play basketball, but a group of older kids were in the middle of a game on the court. Dakoda just hopped on and started “playing”. Instead of having him move off the court and wait for a turn like many kids their age would, they slowed down the pace of their game and included him. When I witnessed this, my heart expanded, and I teared up again. I know that most kids with hemophilia and inhibitors have probably been excluded at some point in their lives and instead of doing the same thing to a younger kiddo, they made the point to include him (despite it diminishing the speed and flow of their game!) Every child at camp seemed to exhibit such kindness and compassion and it made me so grateful to be a part of this community.

The final night was special, with a talent show and a wooden wish boat launch to close out the weekend. My boys have already asked me when we can go back to the camp in Pennsylvania. And they’re not the only ones, I can’t wait until next year!