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Rolling with It

Bill and Linda Mathews smiling together.

When Linda Matthews started having trouble standing up out of a chair, she wasn’t surprised. She was 70 years old, about the same age as her mother when she was diagnosed with inclusion body myositis (IBM).

“I was her caretaker, so I knew all about IBM,” Linda says. “As soon as I started showing symptoms, I went to my doctor and I said I’ve got IBM. They did the muscle biopsy and the bloodwork and the EMG. Within a week, it was definitely diagnosed.”

Often when someone is diagnosed with a disabling condition like IBM, they rely on their partner or a family member to care for them as the tasks of daily living become more difficult to manage alone. Linda, however, was already serving as caregiver for her husband Bill, who had been recently diagnosed with a different rare, autoimmune muscle disease: myasthenia gravis (MG).

In March of 2012, Bill had heart bypass surgery. About a month or so later during a routine eye exam, his ophthalmologist noticed his eyes looked a bit droopy. Linda’s brother, who had a friend with MG suggested he see a neurologist. When he did, it didn’t take long for the diagnosis to be confirmed. Bill says his neurologist thought it was the surgery that set it off.

Despite their disabling diagnoses, the Matthews do not despair. In fact, sharing similar diseases makes them more sensitive to each other’s needs. They have many of the same symptoms—muscle weakness, fatigue, mobility challenges—so they each understand, for example, when the other says they need to rest.

“Nothing is as important as getting the rest you need and taking care of yourself,” Linda says. “And it’s good that I know he’s got my back. I can say, ‘No, not right now,’ and I don’t have to justify it.”

“Sometimes you can tell when the care partner doesn’t understand that,” says Bill of other couples they’ve met. “And you can see how difficult it makes it for the person who’s got the disease. In a way, it’s to our advantage that we understand firsthand what the disease is doing to each other. We don’t have to have that fight.”

Having her mother pave the way for her has also made Linda’s journey just a bit easier. She knows what to expect, and she can plan accordingly as her muscle weakness progresses.

When she saw a Hoyer lift at an estate sale, for example, she scooped it up, knowing that one day it may come in handy if she starts having trouble moving from the bed to the chair. She has a motorized wheelchair that somebody gave her. It’s parked in the garage, though, since she still gets around fine with a rollator. The couple also recently traded their car for a wheelchair accessible van.

When they downsized their home earlier this year, Linda and Bill moved to a one-story (stairs had become a challenge) duplex next to their daughter’s family. Before they moved in, they remodeled the house to accommodate their future needs, adding a roll-in shower, raised toilets, grab bars in the bathroom, and wider doors that will accommodate a wheelchair.

The one thing that couldn’t be retrofitted for accessibility, though, was the kitchen. But Linda just winks and says, “Maybe by then I’ll teach Bill to cook, because I’m the one in the wheelchair.”

That’s not a problem for Bill. “We’ve been married for 56 years. We know how it’s done,” he says.

Linda and Bill feel very lucky to have the support of their family. They love living next to their daughter, son-in-law, and nine-year-old grandson and call on them when they need a hand. When they want to give their son-in-law a break—like when Bill needed to get to the hospital recently for cataract surgery—one of their two sons is always willing to step in and help out too.

Being part of a patient support community helps the couple in other ways. They belong to MG Texas, an MG support group in the Dallas/Fort Worth area. And they are active in Northeast Texas support group of The Myositis Association as well as on Facebook forums through Myositis Support and Understanding.

Getting together with others who share their disease helps Linda and Bill learn the medical ins and outs of both diseases, so they are better able to care for themselves and each other. It also gives them access to a whole pool of practical information that’s not readily available elsewhere: things like who is the best neurologist in a certain area, or tips and tricks people have used for managing certain challenges.

“The myositis Facebook site is wonderful, because people ask questions, and a lot of times it’s a question I have too,” Linda says. “And then in the MG group, we love it when we are able to go to the meetings. They’re always interesting, with new speakers and new ideas. It’s also nice getting to know other members who are in the same situation as we are.”

While living with conditions they know can cause significant health concerns, Linda and Bill remain upbeat.

“My mother was a wonderful example for me how to live my life with this disease,” Linda says. “You just roll with it and try to figure out another way of accomplishing your tasks.”

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Partnering with Patients to Solve the Mystery of Myositis

Christopher Stine headshot

When Lisa Christopher-Stine, MD, MPH and her colleagues formed the Myositis Center at Johns Hopkins University 13 years ago, they wanted to create a place where those affected by this collection of rare autoimmune muscle diseases could receive the very best care possible. The Center is a patient-centered, multidisciplinary clinic in which specialists in rheumatology, neurology, pulmonology, and rehabilitation come together to collaborate in the care of these very complex patients.

The Center also aims to better understanding myositis diseases and help develop new, more effective treatments. One way they are doing this is by partnering with patients. From the very beginning, the Center’s clinicians and researchers invited all of their patients to be part of a large, long-term registry, a research database that included blood samples, DNA, and clinical information acquired during clinic visits. This database now includes information from about 2,500 patients.

“That clinical care–research interface is an important way to think about rare diseases,” Dr. Christopher-Stine says. “You need lots of data points in order to see patterns that you just can’t see in caring for one, two, or three people. Especially when you follow people over time, you can look back and compare that data with their blood samples and DNA and find things that you weren’t even sure were true when you saw the patient in real time.”

Recently, this database facilitated one of the most significant discoveries in myositis. For many years, Dr. Christopher-Stine and her colleagues heard from patients that their muscle weakness and fatigue came on after they started taking statin medications, a widely used drug to lower cholesterol and prevent heart disease. The weakness didn’t go away after they stopped taking the drug, and the cardiologists who prescribed it said that meant their symptoms were unrelated to the statin. Scientists at the Center proved that wasn’t true.

“It’s a great example of how patients drive what we do,” Dr. Christopher-Stine says. “After a while, you hear that story enough times and you say that’s really curious.”

She remembers vividly the evening a young research assistant came up to her after clinic and said, “This is an unusual antibody here. What do you think this is?”

The research assistant, Grace Hong, had been working with Dr. Christopher-Stine in concert with the Myositis Center team, including Dr. Livia Casciola-Rosen PhD, an expert on autoantibodies, to understand how autoimmune diseases work in the body.

What Grace had first noticed turned out to be a new myositis-specific autoantibody that had not been described before. After comparing a number of the patient samples from the Center’s database, it became clear that many of the patients who said their weakness started after taking statins were the same people who had those antibodies. Now we know that that antibody—Anti-HMG-CoA reductase (HMGCR)—is associated with a form of myositis called necrotizing autoimmune myopathy (NAM), which causes muscle cells to die.

This was a valuable discovery, but there is still much more to learn about myositis diseases and how we can help improve patients’ lives. Among the first tasks that must be achieved, says Dr. Christopher-Stine, is to get more drugs approved by the Food and Drug Administration (FDA) for treatment of myositis diseases.

While a number of medications are very effective in treating myositis, most of these are used “off-label,” meaning outside the official approved indications. Insurance companies often challenge these uses, causing delays in treatment as patients and providers fight for access.

Along with this goal is finding an effective way to treat inclusion body myositis (IBM). Currently, the only treatment available for this chronically debilitating form of muscle disease is exercise, which only serves to slow the progress of disability. Those who live with IBM are understandably desperate for any therapy that can improve their condition.

Besides new therapies, a consistent treatment protocol is needed that has been scientifically verified, rather than based on “what we’ve always done.” Currently, there is no such standardized formula for deciding which drugs to try first when a patient is diagnosed with dermatomyositis (DM), for example. Providers differ widely on how they use corticosteroids and other treatments, how they evaluate effectiveness, and when they add to or change the regimen. Patients often suffer prolonged or worsening symptoms because of ineffective protocols.

Dr. Christopher-Stine also suggests that even the way providers refer to these diseases is confusing and not based on the science. Specifically, she challenges the term polymyositis (PM), calling it a diagnosis of exclusion. When myositis diseases were first classified more than 40 years ago, someone with the typical pattern of myositis muscle weakness but without the rash associated with DM was identified as PM. Modern science has refined the picture of all forms of myositis, yet old terminology remains, causing confusion and possibly hindering further progress in understanding these diseases.

“We need to put people into the right category so that they’re studied properly,” Dr. Christopher-Stine says. “The way the disease works is very different between NAM, DM and PM. If you put too many people in one box who have entirely different disease states, you’re going to bias the results.”

If a drug company has a new drug, for example, they need to test it on a fairly similar group of patients so they can tell if it is effective. If the group they study includes both DM and PM patients, the results may be mixed rather than showing a strong positive effect. This may mean that a treatment that worked well for, say, DM patients shows statistically that it isn’t effective because it didn’t work well for PM patients. 

None of these challenges are insurmountable, however. The myositis research community is one of the most collegial communities in academic medicine. Myositis experts from the Johns Hopkins Myositis Center are working together with colleagues from around the world to solve these and other questions with the goal of improving the lives of those who live with myositis diseases.

“I dream that one day we can take care of people with targeted therapies that are personalized just for them,” says Dr. Christopher-Stine. “When I retire, I want to leave the field knowing that I and others made a significant contribution to this personalized approach for all myositis patients.”

With the collaboration of the myositis research community along with data from patient registries like the one established at the Center, Dr. Christopher-Stine is optimistic they will achieve this goal.

The Johns Hopkins Myositis Center is one of the most highly respected centers in the country. It brings together a wide range of clinical expertise in rheumatology, neurology, pulmonology, and physical medicine rehabilitation along with basic science research. Patients with a suspected or confirmed diagnosis of myositis from across the country can be evaluated at the Center, with follow-up consultations with local practitioners.

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Patient stories

Service Dog Brings More than Self-sufficiency

Terry and Bitsy with their service dog Maya at a Fidos for Freedom, Inc. event.

Bitsy Anderson was literally teetering on the edge of a cliff when she started taking seriously the muscle weakness she had been feeling off and on for a while. She and her husband Terry were hiking in Utah, and she was stuck. She had lost her sense of balance, kept tripping over nothing, and needed help to get down off that mountain. In hindsight, symptoms like these had been creeping up on her for years.

That was twelve years ago, and Terry had just retired. The couple was planning to spend their golden years traveling. They had just bought a pair of matching bicycles. They had taken some paddling lessons and were about to buy a couple kayaks. But when they returned home after the hiking trip Bitsy was diagnosed with a rare muscle disease called inclusion body myositis (IBM), a disease that causes disability through increasing muscle loss in one’s legs and arms, difficulty grasping objects, and often trouble swallowing.

“IBM has pretty much changed our whole life,” says Bitsy, who leads the Maryland/Delaware/DC/Northern Virginia support group for The Myositis Association.

“It’s had an impact on both of us,” Terry says. “But it’s an impact we share, and we’re doing the best we can.”

Perhaps the hardest change for Bitsy was adapting to the loss of her independence. As her disease progresses, she’s had to give up driving, has trouble putting on her shoes and managing the housework, and she is always dropping things. She does not like having to constantly call on Terry just to get through the day. Fortunately, a fair-haired helper recently arrived.

Maya, an English Labrador retriever and Bitsy’s service dog, officially arrived in January of this year. Now it is her job to bring in the newspaper, help with the laundry, open the freezer, find things Bitsy has dropped, and dozens of other little tasks that make Bitsy more self-sufficient. She even helps Bitsy on with her jacket and off with her shoes.

“I don’t think she could have come to our home at a better time,” Bitsy says. “Maya makes it possible for me to maintain my independence and also adds an element of fun. If she hears the click of the brakes on my walker, in two seconds she is right by my side to see what she can do. She is very devoted.”

Aside from the help she provides for Bitsy, Maya has brought an added dimension of joy to the Anderson’s lives. Both Terry and Bitsy love her like a family member.

“We love to play with her,” Terry says. “We take her out to the backyard. I will throw the tennis ball, and she always brings it back to Bitsy. It is a good opportunity for us to be outside and enjoy each other. It’s good exercise for her, too.”

“She loves to have her belly rubbed, but I can’t get down on the floor anymore,” Bitsy says. “So, when Terry comes along, she jumps all over him, so he’ll scratch her belly.”

Maya came to Bitsy from Fidos for Freedom, a local volunteer organization that trains service, hearing, and therapy dogs. Fidos is a member of Assistance Dogs International, Inc., a worldwide coalition of nonprofit programs that creates quality standards for assistance dog acquisition, training, and partnership. Fidos also educates the public about individuals with disabilities and about the benefits of assistance dogs and therapy dogs.

As Bitsy and Terry learned, getting a service animal is a big commitment. And the process is far from simple…or easy! Years of training are involved, and not just for the dog. Maya spent a year with a puppy trainer, becoming socialized and learning basic commands before coming to Fidos to find a partner.

Bitsy was accepted into the program in September 2017 and spent more than two years participating in twice-weekly training sessions before she was matched with Maya and allowed to bring her home. Even now Maya’s stay is probationary for a year before her adoption is final, allowing Bitsy and Terry to be certain the arrangement is working out. And even when their probation is over, Bitsy can still consult with a trainer to help her figure out how to help Maya adapt to her needs.

“The program is intense,” Bitsy says. “There was a time I almost quit. There were days when I came home from training saying, I am not doing this anymore. But you must have perseverance. You have to hang in there.”

Terry agrees. “You have to go into the program with your eyes wide open, knowing that there is a commitment there.”

Both Terry and Bitsy are thrilled to have Maya in their lives now and cannot even imagine giving her back at the end of the year. And once the limitations of the coronavirus pandemic have passed, they look forward to getting out into the world again. While they may not be traveling internationally again, they do hope to flee to Florida for winters again and to explore some or our beautiful National Parks. And they will have Maya right beside them.

“This has been a great experience,” Bitsy says. “You just have to go with the idea that I’m going to do this no matter what it takes, that it’s going to be a benefit. It really is well worthwhile.”

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Patient stories

When You Need a Little Help from Your Friends

Maddie Cure, a young female advocate.

Madison Davis had been planning her career in the military since she was seven or eight years old. Living near the Naval Academy in Annapolis, Maryland, she joined the Naval Sea Cadets and was intent on moving up through the ranks. She strategized her high school resume to help her get accepted into the Coast Guard Academy, as a soccer player, if possible.

“She was obsessed with the Naval Academy,” says her mom, Christine Davis. “She always wanted to visit there and wear the uniform and work on advancing her ranks. She was a big creator, too, a lego girl, up until she was 13 or so. She always talked about wanting to work on Lear Jets, fixing planes. She always knew what she wanted to do, and she was excited.”

In May 2017, however, gazing down at her daughter lying limply in a hospital bed as doctors gave their diagnosis, a strange, rare disease that seven weeks earlier had weakened Maddie’s muscles, given her a rash over large portions of her body, and paralyzed her into the worst pain she’d ever experienced, Chris cried. Not only was her daughter suffering, but she knew all Maddie’s dreams had just been evaporated by an autoimmune disease they couldn’t even pronounce: juvenile dermatomyositis (JDM).

“I was so numb to everything in the hospital. It really didn’t hit me,” Maddie says. “I wasn’t actually facing all these things I had just lost. I was just on to the next thing. Like, okay, so what do I do now? What schools do I apply to now? I didn’t really give myself a lot of time to deal with it in the moment. And believe it or not, I felt like I was handling everything very well.”

The next few years were difficult for Maddie. She is fortunate that she lives near one of the world’s leading Myositis Centers at Johns Hopkins University, so the medical treatment she received, including prednisone, methotrexate, and intravenous venous immune globulin (IVIG) therapy, was excellent and effective. Her mental health, however, was a different story.

Maddie missed out on much of the excitement of her senior year of high school because she was too weak to participate in many of the activities. Not only was her athletic strength gone, but the prednisone, a steroid medication, made her gain weight and gave her what she calls “’roid rage,” making her emotionally erratic. She lost friendships, because she missed a lot of school and couldn’t go out on weekends, and her friends just didn’t understand why she was behaving so strangely.

“She suffered a lot in many different ways,” Chris says. “We were just trying to survive it every day and trying to seek answers to make it better.”

Maddie and Chris did find answers, mostly from others who live with rare diseases and know what Maddie was going through. On her first outing after spending three weeks in the hospital followed by two weeks in rehab to relearn how to walk, Maddie and Chris attended a local support group meeting of The Myositis Association (TMA). The speakers at that meeting happened to be three members of the CSI Pharmacy team, talking about immune globulin (IG) therapy and home infusion.

During the presentation, a representative from CSI Pharmacy shared her own journey with myasthenia gravis—another rare autoimmune disease—and IG therapy. In her talk, she described how her disease had ended her career as a Navy nurse, and how hard that was to deal with.

Maddie remembers glancing at her mother and whispering, incredulous, “Did she just say she was in the Navy?”

After the talk, Maddie and Chris approached the speaker and shared Maddie’s similar regrets about losing her own dream career in the military. After that, The two not only became good friends, but she also became Maddie’s home infusion nurse, allowing Maddie to receive her IVIG treatments at home rather than in the hospital infusion center. Later, when Maddie went off to York College of Pennsylvania, her new friend helped her learn how to give her own IG treatments subcutaneously (SCIG, under the skin, rather than in the vein).

The Davises also found out about Cure JM Foundation, a group more focused on children with myositis. They attended the Cure JM annual patient conference, and there Maddie befriended a group of other teens who also lived with myositis. It didn’t take long for Maddie to become a leader in this organization, one of the “older kids” who, the following year, helped lead the panels she had learned so much from the previous year.

Maddie discovered a passion for helping others through the rough spots of living with JDM. She had a Facebook page and website, Maddie Strong, where she talked about her challenges with her disease. Once she even posted a video demonstrating how she gives herself SCIG treatments.

Maddie may have powered through at the beginning of her disease, but eventually the walls started crumbling. During the summer before college, she started looking around and realizing how much had been taken away from her.

“That’s when I started dealing with depression,” she says. “I was thrown so much off course. It was really, really hard. I know everything happens for a reason and I’m really I glad to be where I am. I’m glad I’m at York. I’m actually really happy I’m not playing soccer. But it took a lot out of me.”

It’s the friends she met along the way, the other kids who share this disease and understand the journey, who have helped her get through that dark time.

“They have always been the first families that we’ve reached out to,” Chris says. “If she was feeling really low or like life isn’t worth living anymore or the medicine is just too much, she always had people to turn to who could walk her through it, because they’ve already been there.”

Now as she prepares to start her junior year of college, Maddie is strong. She’s an education major, making the dean’s list, works with K through eighth graders at the York City schools, and is an education technology entrepreneur with a fun teaching app for kids she’s hoping to market. Best of all, her disease is in remission.

Still, kids who are struggling with coming to terms with the life-altering experience of a disabling rare disease diagnosis like JDM are never far from this young advocate’s mind.

“The one thing that I wish I could tell parents is, for kids who are going through this, the mental health aspects are a really big deal,” Maddie says. “I would say focus your attention more on the fact that your kid is going through a lot of mental stuff right now. They are confused and they don’t understand and its really scary. Just talk about what’s going on.”

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How to Reengage with Less Risk

We’re now four months into a global pandemic, and lots of us are just tired of staying home. Most of the folks in our patient communities, however, have underlying medical conditions that put them at higher risk for developing severe COVID-19 infections. So while others consider reemerging into the world again as states start lifting social distancing restrictions, our patients might be reluctant to take this step.

Still, completely avoiding contact with others for an indefinite period of time is not a viable option either. Human beings need physical and social contact with other human beings, if only to stay sane. The social isolation we’ve been enduring since shutdowns in March has caused serious anxiety and depression for a significant number of people, especially those who live alone.

Fortunately, public health experts say the decision doesn’t have to be either/or. While staying home is still the lowest-risk option for avoiding infection, there are lots of ways to socialize that don’t involve crowded indoor spaces where no one is wearing a face mask (the highest risk option). Weighing the risks of contact with others against the possible benefits to your health can help make the decision-making process easier. Here are some things to think about:

Consider the risk for you and your immediate contacts. If you or someone you live with is especially vulnerable to infection, this is an important factor in weighing how much you want to risk coming in contact with someone who might give you COVID-19.

It’s also important to think about those outside your household with whom you may want to socialize and how much exposure they might have had too. You may want to consider creating a pandemic “pod,” a small cohort of friends or relatives with whom you choose to interact somewhat normally but who agree to stay socially distant outside the pod.

Keep tabs on how the virus is spreading in your community. The number of new cases, hospitalizations, and deaths varies widely in different areas of the country. Check local health department reports or find out here whether these numbers are increasing or decreasing where you live. If they are climbing or remain high, you may want to rethink whether going to a salon for a haircut is a good idea, even if restrictions have been lifted.

Think about how risky the activity is. Most people know the basics of how to stay safe from coronavirus infection. Keep these ideas in mind as you make decisions about venturing out in public. These include:

  • Wearing face covering when out in public significantly reduces transmission, both for the wearer and the ones they are with.
  • Maintaining a distance of at least six feet from others decreases the chances of contact with infected droplets.
  • Large gatherings, especially if they are indoors and especially if others are not wearing face covering, significantly increase the risk that someone will pass on the virus.
  • Encounters in the outdoors are safer than those in close indoor spaces.
  • Limiting the time you spend among others, especially if it is indoors, reduces the chances of encountering the virus.
  • Bringing your own (BYO) food and drink means a lower risk of transmitting the virus through touching a contaminated surface. Bringing your own chairs or picnic blankets also helps you keep your distance from others in an outdoors group.
  • Handwashing with soap and water or using hand sanitizer is still the most effective way to prevent transmission of coronavirus and other disease-causing organisms.

And finally, here are additional tips from NYC Health to keep yourself and your loved ones safe and healthy as pandemic restrictions start to lift. Be careful out there!

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Patient communities Patient stories

Meeting Patients Where They Are

Like most neurologists, Dr. Charlene Hafer-Macko treats patients with a variety of neuromuscular conditions. Her focus, though, is myasthenia gravis (MG). It’s MG patients who provide her with the intellectual challenge she loves.

“I really like this population,” she says. “There are so many things you can do to help them stay in control of their disease. And this is a group that really uses the information they have to help themselves. Helping people through the journey is the part I find the most fun.”

As an associate professor of neurology at the University of Maryland School of Medicine, Dr. Hafer-Macko serves as director of the university’s multidisciplinary Myasthenia Gravis Center. This clinic pulls together all the services an MG patient needs in one coordinated package—including an infusion suite that provides intravenous immune globulin (IVIG), plasmapheresis service, and thoracic surgeons that focus care for thymectomy for myasthenia gravis.

“Our team is very well versed in myasthenia,” Hafer-Macko says. “So not only are they providing care, but they’re also monitoring for side effects and providing education and support at the same time.”

For Dr. Hafer-Macko, it’s the education and support part of working with MG patients that she finds most satisfying. Several years into her career, she realized that she wasn’t feeling fully fulfilled by her interactions with patients. She would assess their weakness and check their blood work and tweak their medications, but these exchanges with mostly stable patients felt flat. She needed something more.

She discovered that something more in the stories her patients told about their daily experiences. They reported, for example, that even when their double vision was controlled or they were back to walking normally, they still had trouble reading or watching TV, and they felt exhausted after a trip to the grocery store or just walking across the room.

“Even when many patients are well controlled, fatigue is an element that just stays with them,” Hafer-Macko says. “Fatigue is such a tricky thing. It’s something that is not often addressed effectively. So really understanding what’s driving that fatigue was something that I got very interested in.”

She teamed up with occupational, physical, and respiratory therapists to develop a better understanding of fatigue and the needs of MG patients. Together with this team, Hafer-Macko developed a toolbox of techniques for helping patients avoid or overcome fatigue and other challenges.

Listening to her patients’ stories has also helped Hafer-Macko become a better doctor.

“I learned how to ask questions differently, questions that gave me better data,” she says. “And then once I’d ask them differently, I could coach individuals on how to give me better information.”

When she would ask a question like, “Are you better,” for example, she found the patient’s response—“Yes, I’m better now”—didn’t provide much in terms of measurable outcomes. If, however, she asks about how long the patient can read before their eye symptoms make them put the book down, she has a benchmark that she can compare to a previous exam. It’s data that shows a meaningful response to treatment.

Stories of her patients’ fatigue also inspired Hafer-Macko’s research. She is part of a group at the Baltimore Veterans Association Medical Center that is exploring exercise, nutrition, and fitness in older adults. One of the things they have learned is that, because of their weakness, those with MG must work extra hard to accomplish even minor tasks like walking to the bathroom. This leaves far less energy for all other activities.

“It’s like every time they walk to the bathroom, they’re running a marathon,” Hafer-Macko says. “They have very little reserve. They’re just working very hard because of that weakness.”

Dr. Hafer-Macko has been recognized by the Myasthenia Gravis Foundation of America (MGFA) for her outstanding work with the MG community. She has served on the board of directors for the organization and currently serves on two of their committees.

Ironically, Hafer-Macko’s greatest inspiration is not a patient at all. It’s her mother. At 82, Charlotte Hafer still teaches dance—these days remotely by zoom. In 41 years of teaching elementary school during the day and dance at night, she never took a sick day. She continues to work as a math and reading specialist by day and teaches dance at night. As a devoted theater fan, her mother saved up her sick leave compensation so she could go to shows in New York City and in the Pennsylvania/Maryland/DC area to see shows. This year, Charlotte engaged the brave new world of Facebook to win a contest in which she was named Broadway’s Biggest Fan.

“She’s actually my inspiration,” Hafer-Macko says. “To deal with my mom and her medical hang ups, I’ve learned so much about taking care of people. You’ve got to meet folks where they’re coming from. They’ve got nuances, and that makes such a difference in working with a patient to find a care plan that will work well for them.” 

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Advocacy Health Information

Tips and Tricks for Managing MG

We recently spoke with neurologist Charlene Hafer-Macko, MD from the University of Maryland’s Myasthenia Gravis Center about ways myasthenia gravis (MG) patients can keep themselves healthy and stay out of crisis. Here are some of her tips and tricks:

Communicating about your condition with healthcare providers, especially in an emergency situation, can be a challenge.

  • Wear a medical ID bracelet, such as the MedicAlert, that identifies you as having myasthenia gravis.
  • Know what medications MG patients need to avoid, and carry a list of them in your wallet for easy reference.
  • Download the MyMG app from the Myasthenia Gravis Foundation of America. The app also has a list of medications to be avoided with MG.
  • Document your symptoms, treatments, and how they affect your daily life so you can have these data readily available when your doctor asks, “How have you been feeling?”
  • Always ask your healthcare provider or pharmacist if a newly prescribed medication is on the list of drugs MG patients need to avoid.
  • Use online resources to look up conditions and medications. Just be sure the source is reliable and informed by science.

Weak muscles can make breathing difficult, but there are things you can do to make breathing more efficient.

  • Use pursed-lip breathing, a technique in which you breathe out through puckered lips. See this demonstration.
  • Learn how to breathe into your belly by lowering your diaphragm. Learn how here.
  • Explore mindfulness practices that focus on breathing. This is also helpful for stress reduction. Find a guided exercise here.

MG symptoms tend to get worse with heat for many people. To avoid melting when it’s warm:

  • Take tepid showers. If you really like a hot shower, finish it off with a cold splash.
  • Avoid being out in the sun for long periods of time.
  • When the weather is hot, plan big activities for the cooler part of the day, and take advantage of air conditioning as much as possible. Ask your electric power company for a form that will ensure you are a higher priority for power when the electric goes out a storm.
  • Consider using a cooling vest if, for example, you want to sit in the hot sun for hours at the baseball game. Here is a sample of some available models.
  • Sporting goods stores also sell cooling towels and reusable, freezable gel packs.

Not getting enough sleep will make anybody more fatigued. For those with MG, insomnia can be related to corticosteroid use, anxiety, stress, and other effects of chronic illness. Good sleep habits can help. Here are some tips:

  • If you take prednisone, do so early in the day.
  • Schedule your bedtime so you get at least 7-8 hours of sleep.
  • Make a habit of going to bed at the same time every night and getting up at the same time each morning, even on the weekends.
  • Keep your bedroom quiet, dark, relaxing, and at a cool temperature.
  • Create a relaxing bedtime routine.
  • Ban electronic devices, such as TVs, computers, and smart phones, from the bedroom, and avoid using screens during the hour before you go to sleep.
  • Avoid large meals, caffeine, and alcohol before bedtime.
  • Exercise regularly to help you fall asleep at night.

And finally, Hafer-Macko advises that those living with MG watch their bodies, learn how their treatments affect them, and plan activities accordingly. For example, if you (or those you live with) notice that you start to slow down as you get closer to your next pyridostigmine (Mestinon) dose or IVIG infusion, that may not be the best time to schedule a big day with the kids that will use up a lot of your energy.

Similarly, if you take pyridostigmine, notice how it affects you. If it starts to wear off too soon, mention it to your doctor; you might need to adjust the dose. Also, be aware of scheduling high-energy activities like shopping or cleaning during times when your meds are wearing off.

The following organizations offer additional resources, including support groups, education, and research:

Categories
Advocacy Patient communities Patient stories

Being There for Those in Need

Rebekah Dorr never set out to become a myasthenia gravis patient advocate. It started with her Facebook page, Myasthenia Gravis Unmasked, and just sort of evolved. That’s where, in 2014, Rebekah first shared the story of her own harrowing journey with myasthenia gravis (MG) and some of what she learned along the way. She wanted to bring hope to those who live with the disease by raising awareness about how it affects individuals and correct some of the misconceptions even the medical community still endorses.

When someone messaged her on the page asking for help, she wasn’t sure what she could do. “But I’d advocated for myself,” she says. “So I thought, let me see if I can help this person. I think she was indebted to me, because she turned around and started sharing about me in other groups.”

Since that time, Rebekah has lived on her phone. She posts educational content about living with MG and provides a platform for the personal stories that give voice to the challenges myasthenics face every day. She also responds to every comment and private message—sometimes dozens a day—from those with questions or who need her help to get the care they need.

“I was frustrated with what was available at the time,” Rebekah says of the MG support system. “There was research, there were support groups, and that was it. There was this huge no man’s land for what was happening for the patients. And I was like, who’s taking care of patients the way I needed to be taken care of? So that ended up becoming my passion.”

At least part of this passion for walking with patients in their time of need comes from her own experience. She knows what it’s like to be alone and afraid, not understanding what was happening to her, and not trusting the medical community to make the right decisions for her care.

Rebekah’s symptoms began one summer when, out of the blue, she started feeling really tired, like she had the flu. Very quickly, however, those symptoms escalated to significant shortness of breath. She had trouble chewing and swallowing, and her legs became so weak she couldn’t walk. It seemed like one minute she was playing on the beach with her cousins and the next she was unable to get out of bed.

The next two years was a terrifying odyssey that included countless ER visits, hospitalizations—including several stays in ICU and more than one time when she had to be resuscitated—lab tests, scans, surgeries, spinal taps, and specialist consultations. It was a time when, looking back, she wished she’d had someone she could have called upon to help her know what to do.

Doctors refused to believe that a woman of 22 could develop MG, despite the fact that her grandmother also has MG and other evidence to support the diagnosis. Instead, they said she was faking the fact that she couldn’t walk and couldn’t breathe, and diagnosed her with somatic conversion disorder (meaning she was mentally ill, making it up). This label, together with the disrespect with which she was treated, did more damage to her health and spirit than MG ever could. It made her question her own truth and made her terrified to seek the care she desperately needed.

“I didn’t know anything,” Rebekah says. “I didn’t know blood tests for antibodies had to be sent to a special reference lab. I didn’t know my shortness of breath wouldn’t necessarily make my oxygen saturation go down. They didn’t explain the drugs to me. I had no idea I was being overdosed. I had no idea what any of it was.”

But she learned. Having people who depended on her for answers forced Rebekah to dig into the research and understand all she could about MG. She quizzed her own neurologist, listened closely to conversations she heard in hospital hallways, and read everything she could get her hands on. She also listened to the stories of patients. And she became the expert others needed.

“Word of mouth was spreading about me,” she says. “I don’t think the word advocacy was ever used, but it was just, hey, contact this woman, she’ll help you. And so I started getting flooded with messages. It became a job for me. It became my life.”

Rebekah now has clients all over the world, some of whom she works with for months or years at a time, sharing knowledge and awareness. More often, however, she’s there with patients—in-person for local clients, but by phone for most—when they need to go to the ER or are admitted to the hospital to help them navigate a system that often doesn’t understand this rare disease.

By 2016, however, Rebekah realized she was not receiving the kind of respect she needed from the healthcare community. She didn’t have credentials or the backing of some authority that would make medical professionals take her seriously. So she started her own nonprofit organization: The Myasthenia Gravis Hope Foundation.

“Our whole focus is advocacy,” she says of the Foundation. “I define that as clinical advocacy. We’re not just doing awareness or education. We’re actually coming in for the patient when they are most vulnerable to challenge the stigmas and misconceptions about MG that severely affect how they are perceived and treated.”

Beyond Rebekah’s lifesaving advocacy, MG Hope also provides funds for patients to travel for care and to cover the cost of critical medication until they can get enrolled with manufacturers’ assistance programs. The organization also helps patients access medical and specialty care and emergency medications.

For Rebekah this work—none of which she is paid for—is all about helping others avoid the hell she went through. She remembers sitting in a tiny closet of a hospital room which she had occupied for thirty-five days. She’d gone in for a thymectomy, but never got it. Instead, she experienced anaphylactic shock as a reaction to blood products, endured two resuscitation codes, went through cholinergic crisis because of titration mistakes, and so much more.

She remembers thinking if only somebody had educated her about these possibilities, she could have prevented nearly all of them. As a person of faith, that’s when she vowed to be the one to help others overcome or avoid these challenges. Now as the founder and CEO—and the only active member of the staff—of the MG Hope Foundation, she’s doing that work.

“I’m passionate about focusing on the patient experience,” Rebekah says. “I think that honesty and vulnerability is where we have the power to transform things, to actually step into somebody’s life and to maybe change it for the better. Whether they need emotional support or education, I want to show up for them in whatever way I can. That’s just where my heart is.”

Rebekah’s grandmother, Doris (95) was diagnosed with MG more than 60 years ago. This photo of Doris and Rebekah was part of MG Hope Foundation’s project called The Humanity Behind MG, designed to capture the essence of the human experience of those who live with the disease.

Categories
Advocacy

Standards Still Apply

Recently, a patient we will call Angela posted a note on a private Facebook page describing a troubling situation she encountered with her home IVIG infusions. She wrote to the group of fellow immune globulin (IG) patients that her infusion company decided that the nurse no longer needed to stay with her for the duration of her infusion.

Because of coronavirus infection risk, some infusion companies are apparently trying to limit the time the nurse spends in the patient’s home. The nurse is instructed to set up the infusion and stay until it had been increased to the scheduled drip rate, then she is to leave, for the rest of the day. Another patient in the group posted that her nurse didn’t leave, but she spent most of the time of the infusion sitting in her car outside the house.

“My infusions take basically eight hours, and she will be here for two of them,” Angela posted. “She’s going to teach my husband how to draw and administer diphenhydramine [an antihistamine used to counteract an allergic reaction, also known as Benadryl] in case of an emergency. I am nervous for sure. What will we do if air gets in the line? What will we do if something goes wrong?”

Angela’s concerns are not unfounded. Leaving the patient during an infusion can be dangerous and violates strict standards of care established by the Immune Globulin National Society (IgNS), an organization of Ig therapy professionals.

“As nurses, our duty is to provide safe and effective nursing care,” says Brittany Isaacs, RN, IgCN, Director of Nursing at CSI Pharmacy. “Our nursing judgement should not be clouded by situations that place a patient or their safety in jeopardy. Our duty is to do no harm, so we need to protect both the patient and ourselves during any encounter. Ensuring proper personal protective equipment is donned to keep everyone safe and following the guidelines outlined by the CDC, WHO, IgNS, and the Infusion Nurses Society allows a nurse to continue to provide safe and effective nursing care during home infusions.”

While COVID-19 has caused many changes in healthcare protocols, patient safety should always be the ultimate guiding principle. The following guidelines are drawn from IgNS’s Immune Globulin Standards of Practice and COVID-19 Resource Guide and FAQ.

During the COVID-19 pandemic, patients receiving in-home immune globulin therapy can expect the following from their specialty pharmacy or home infusion company:

  1. Pharmacy personnel will wear personal protective equipment while packing the medications and supplies that are sent to the patient’s home.
  2. The home infusion nurse will be screened by their company for COVID-19 symptoms to ensure they will not carry infection into the patient’s home.
  3. Patients will be screened to ensure they do not have COVID-19 symptoms before being infused.
  4. Nurses will wear personal protective equipment, including masks, gown, gloves, and face protection, while in the home.
  5. Social distancing should be maintained to the extent possible, except when providing direct patient care.
  6. Patients should wear a mask or face covering while the nurse is in the home.
  7. Patients can request that their specialty pharmacy include masks, gloves, and hand sanitizer in their IG shipment.

The following practices are not acceptable, even during COVID-19 restrictions:

  1. Neither the patient nor a family member should be taught to self-infuse IVIG or to remove the IV after the infusion is completed.
  2. The infusion nurse should never leave the home for any reason while the infusion is in process. This includes sitting in her/his car outside of the home during the infusion.
  3. Family members should not be asked to leave the home during the infusion.
  4. Nurses should never refuse to wear personal protective equipment.

If you are uncomfortable or do not feel safe with your infusion company’s changes in protocol, please do not stop treatment! Staying on therapy is vital. If your company is unwilling to adhere to these standards of care, you may want to consider changing companies. If you need help with this, CSI Pharmacy’s patient advocates can help, even if you are not our patient.

Additional resources can be found here:

Infusion therapy standards of practice. Journal of Infusion Nursing

Immune Globulin National Society – Standards and guides

The role of an IG infusion nurse. IG Living Magazine. August/September 2013

National Home Infusion Association

IDF guide for nurses: Immunoglobulin therapy for primary immunodeficiency diseases Immune Deficiency Foundation

Categories
Advocacy Patient communities Patient stories

Paying It Forward

Helping other people has helped us heal from what we’ve lost.

Ten years ago, Karon Faught started slurring her words and having trouble lifting her arms to blow dry her hair. She was only in her thirties, but some months later while at work, she couldn’t find the right words she wanted to say. She thought she was having a stroke. An MRI disproved this possibility, but it did nothing to alleviate the overwhelming fear she and her husband Jerry felt about what was causing these symptoms.

When she went to her neurologist, he had a list of conditions he wanted to test her for. Among them was not myasthenia gravis (MG).

“He said he was going to test me for MS and ALS and a couple other things,” Karon recalls. But nobody gets myasthenia gravis, the neurologist said, so I’m not even going to test for that.

Ironically, MG was the thing Karon’s primary care provider specifically suggested she be tested for. Also ironically, while she was in the office, the neurologist agreed to give Karon a magnesium infusion, a treatment she’d had before to treat her chronic migraine headaches.

What Karon and Jerry didn’t know at the time was that magnesium is one of a laundry list of drugs that makes myasthenia symptoms worse. That infusion persuaded the neurologist to do the blood test, because there in his office, Karon lost her ability to speak and move as a result of it. Three weeks later, the tests came back positive for anti-acetylcholine receptor antibody-positive MG (AChR MG).

This was just the beginning of a harrowing ride for Karon and Jerry, one they are grateful to the MG community in their home state of Texas for helping them survive.

“Ten years ago, there was really no good information about MG,” Jerry says. “Even at the top hospital in the region, one of the only hospitals in the country that was doing an MG clinical trial, people didn’t understand it. Their nurses were still giving IV Benadryl to MG patients, and their medical school was still teaching that kids couldn’t get MG. When we came into this, the best knowledge base was those that came before us.”

So when Karon was facing surgery to remove her thymus (a procedure that is often part of treating MG), she wanted to talk with others who had the disease and could help her understand what to expect from this major chest surgery. She and Jerry attended their first Myasthenia Gravis Foundation of America (MGFA) national conference that year and connected with the only MG support group in Texas, which was in Lubbock, about 300 miles from their home north of Dallas.

The group was led by Coleen Shinn. She and others from the group took the couple under their wing. And when Karon was in the hospital after surgery, they were a lifeline for Jerry who guarded his wife’s treatment from healthcare professionals who repeatedly tried to treat Karon with medications on that laundry list of drugs that make MG worse.

“After my surgery, Jerry was on the phone with Coleen almost 24/7, asking her questions and gathering information,” Karon says. “When the doctors wanted to give me a medicine, he would call Coleen and say, is this okay? And she would say yes or no. A lot of times it was no, don’t give her that medication, because she’ll go into crisis.”

Jerry chokes up when he remembers this time. “I owe a huge debt of gratitude to Coleen and Marvin and Lowell and Margaret and all the people in Lubbock who took us in. There’s no way I can explain how they helped us. There are just no words that can describe it.”

After Karon’s surgery, she and Jerry continued to attend support group meetings in Lubbock. They needed that companionship and the knowledge of others who lived with MG. But it was a five-hour trek, and they knew there had to be other MG patients closer to home. So armed with contacts they met at their second MGFA conference, they pulled together a support group in the Dallas-Fort Worth area.

Since then, the couple has created a nonprofit support network, MG Texas, whose only mission is to empower people by sharing knowledge about this rare neuromuscular disease. The organization now supports honorary Texans all over the world, and through the work of many others, there are now support groups in Dallas, Lubbock, Austin, San Antonio, Corpus Christi, San Angelo, and Beaumont. And because they live only 20 miles from the Oklahoma border, it was easy enough to restart the groups in Oklahoma City and Tulsa after the MGFA reorganization. They’ve also started, MGKids.com, the only nationwide MG support organization for children with MG.

“There are so many fears when a person comes into this,” Jerry says. “If we can alleviate any one of those fears to help them get to the next stage of the healing cycle, then that’s really what drives us.”

This support group network has also been part of the healing cycle for Karon and Jerry.

“The support group has helped us get through this,” Karon says. “Helping other people has helped us heal from what we’ve lost. Having a diagnosis like MG, you have to look at life a little bit differently and make some adjustments. But when you help other people, you don’t think so much about what you’re going through.”

“I’ll be honest with you, at the beginning, I was angry, Jerry says. “And the only way to battle this disease that we can’t fight is to educate so that it doesn’t hurt others. That was the why we started the support group in Dallas. The bonus was, if we get more people in the group, it will help us.”

But healing the fear and anger is only a part of Jerry’s motivation.

“There’s a debt we have to pay forward,” Jerry says. “We’re going to have it for a long time. And that’s really what drives me.”