Tag: myositis

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Building a Myositis Team in Texas

A neurology resident Building a Myositis Team in Texas

When Salman Bhai, MD and his wife were looking around for a place to settle into their careers last year, they decided Dallas was where they wanted to be.

“We were looking for a place that would support us,” he says. “Both of our families are here. We’re originally from Dallas, so this has always been home.”

Along with coming home, Dr. Bhai wanted to bring to the area his expertise as a neuromuscular specialist and a myositis researcher. His goal is to build a regional multidisciplinary clinic that specializes in myositis care. Such centers exist in other areas of the country—Baltimore, Boston, Pittsburgh, Kansas City, for example—but they are notably lacking in the South.

In Texas, Dr. Bhai was given the opportunity to create this specialty care clinic and be part of vibrant clinical and scientific communities. He serves as the Director of the Neuromuscular Center at the Institute for Exercise and Environmental Medicine, a joint institute between the University of Texas Southwestern and Texas Health.

“We are developing a myositis center where patients don’t have to travel so far to get the care that they need and deserve,” he says. “It will also be multidisciplinary, so they don’t have to keep coming back and forth to see different specialists. We will have everyone in one place.”

Dr. Bhai was fascinated by neurology when he was in medical school. He found the nervous system to be especially interesting because of its complexity, and he knew there was a lot more to be learned about its function and impact on people’s lives. As a neurology resident, he gravitated toward patients with neuromuscular challenges and did clinical fellowships with some of the country’s most renowned myositis experts in Boston.

 “Myositis patients have muscle disease as well as systemic involvement,” he says. “I especially enjoy speaking with patients and trying to understand the issues they have, because we can make a big difference. It requires multimodal care, and it’s a long-term relationship. These patients don’t just need medication. They need a good doctor-patient-caregiver relationship to help them achieve the best outcomes.”

Dr. Bhai is also an Assistant Professor of Neurology at the University of Texas Southwestern Medical Center where he cares for patients with a range of autoimmune neuromuscular conditions, including myasthenia gravis, chronic inflammatory demyelinating polyneuropathy (CIDP), and Guillain-Barré syndrome (GBS).

The myositis center will also be a research institute. Dr. Bhai is collaborating with other researchers at UT Southwestern on projects to better understand all forms of the disease and to develop new therapies to treat them.

Already there is a biobank in place where muscle biopsies and other de-identified patient specimens can be stored for research. The center will also serve as a site for clinical trials—Dr. Bhai currently serves as site investigator for nine clinical trials—where those with myositis can participate in testing of new therapies and diagnostics.

And because exercise is such an important part of the treatment plan for those with myositis, once COVID restrictions are no longer a problem, Dr. Bhai wants to include an exercise program in the mix. His hope is this will be a place where patients can come together not just for an exercise class, but to meet others who have their disease, share their experiences, learn from each other, and serve as a support network.

Raising awareness of myositis among new medical practitioners and trainees is also a priority.

“These patients are rare. You don’t see them every day,” Dr. Bhai says. “My hope is when trainees join me in clinic, they will see more of these cases than they might otherwise. Whether they’re trying to learn how to perform muscle biopsies or exercise testing, or they want to see what myositis looks like in a patient, I want to help support that.”

Dr. Bhai’s own research stems from his personal interest in exercise.

“I think muscle is quite beautiful and complicated, but we treat it too simply,” he says. “We think of it only as something that allows us to move. But in fact, it communicates with the rest of the body. Muscle tissue releases factors that influence your brain, your gut, and other muscles. Because of these factors, we know that exercise improves quality of life, it improves overall muscle health, and it improves mental health. Most people should exercise, even those who have a muscle disease like myositis.”

Dr. Bhai’s current research involves understanding why people with myositis experience exercise intolerance and fatigue, even when their disease is well-controlled. He’s trying to understand the biology of muscles, especially the mitochondria (tiny structures in the cell that generate energy). And he’s exploring genetic markers in the blood to see how these markers released by muscle tissue are different in those with myositis and how that produces the symptoms they feel.

“My goal is to create a partnership with a broad team,” Dr. Bhai says. “Not just with practitioners but also with the patient, the family, the caregivers, their pets, their support team. Everyone. I really want to have a patient-centered approach that brings change to those who live with this disease. That’s when patients get the best care.”

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IG Therapy

FDA Approves First Proven Treatment for Dermatomyositis

CSI Pharmacy Director of Nursing Roxanne Marlar

Until recently, those who live with dermatomyositis (DM), have relied on off-label medications to treat this rare autoimmune disease of the muscles. On July 16, however, the Food and Drug Administration (FDA) approved an intravenous immune globulin (IVIG) therapy called Octagam10% for use by adults with DM.

“This is fantastic news for the myositis community,” says Dr. Rohit Aggarwal. “Octagam is the first proven FDA approved treatment for a myositis indication. We’ve had drugs in the past, but this is the first one that has the kind of scientific evidence—a Phase 3 clinical trial that is randomized, double blind, and placebo controlled—that is required for FDA approval.”

Dr. Aggarwal, an internationally recognized myositis expert, was the principal investigator for the Phase 3 clinical trial called ProDERM that demonstrated that Octagam 10% caused significant improvement in DM skin and muscle symptoms as well as other disease criteria.

Immune globulin is a concentrated solution of antibodies derived from donated human plasma. While immune globulin has been used to treat a variety of autoimmune diseases for more than 30 years, it’s use in myositis has been “off-label.” In fact, with a few grandfathered-in exceptions, all treatments for myositis diseases are used without an FDA labeled indication (approval) for this condition.

For a rare disease like myositis, however, it’s often difficult to convince a pharmaceutical company to invest in the research necessary to attain FDA approval. Fortunately, Octapharma, a privately owned pharma company based in Switzerland, chose to take that leap with Octagam.

“We are very responsive to patient and clinician requests,” says Eric Pluckhorn, US director of sales for Octapharma. “This now gives us an entree into the autoimmune market that up until now we really have not had for the Octagam brand.”

Several years ago when the company started this project, DM represented a huge unmet need. At the time, Dr. Aggarwal and his colleagues had recently developed response criteria that would provide the kind of measurable data needed to clearly demonstrate the drug’s benefit for patients. The success of the trial is good news for both DM patients and the providers who care for them.

For patients, off-label use often makes accessing the medication a challenge. IVIG is very effective, but it’s also very expensive. Health insurance companies, in an effort to cut costs, often don’t want to pay for such costly treatments. So they create roadblocks for patients, such as requiring them to first try a number of other treatments without success before they allow IVIG. This practice is called “step therapy.” If the drug is prescribed off-label, many companies simply refuse to pay for it.

Practitioners are also enthusiastic. “Empirically,we always knew IVIG works,” Dr. Aggarwal says, “but we were hesitant to give it, partly because of not having Phase 3 clinical trial-level of evidence and partly due to the insurance issues. Now we have an approved drug that we can give to the patients, and insurance companies can no longer deny it because it’s off-label.”

Perhaps more importantly, Dr. Aggarwal suggests this approval sets a precedent for future drug development in myositis.

“Once you have a drug approved through the FDA based on a rigorous process and a valid set of criteria, other drug companies will look at that as a pathway for novel drug clinical trials in myositis,” he says. “The ProDERM study sets a precedence for future drug approval in myositis.”

Within the next five to ten years, Dr. Aggarwal predicts there will be a number of novel therapies approved for myositis diseases, including not only dermatomyositis, but also polymyositis and necrotizing myopathy.

To address the other side of the access equation—that is, the expense—Octapharma plans to offer financial assistance to help cover the cost of the treatment. Eligible patients with commercial health insurance will have access to the company’s Copay Assistance Program that will cover out-of-pocket costs, such as coinsurance, copay expenses, and deductibles, up to $2,500 per year. This assistance is provided without regard for ability to pay. (By law in the US, the company is not able to offer this coverage to those who have government plans such as Medicare, Medicaid, or Tricare.) Patients who don’t have insurance or have lost their insurance may also get assistance through a compassionate use program.

“The FDA approval of Octagam 10% as a safe, tolerable and efficacious treatment for dermatomyositis in adults is exciting news for patients who previously relied on unapproved treatments,” said Octapharma USA President Flemming Nielsen. “Octapharma is committed to providing life-saving and life-enhancing therapies for patients with rare diseases. We look forward to partnering with patient organizations and the medical community to develop educational and other support programs that will serve dermatomyositis patients.”

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Health Information

Sleep on It

sleeping peacefully with peace of mind that CSI Pharmacy is doing all they can
Photo by bruce mars on Unsplash

Getting a good night’s sleep does more than just keep you alert during the day. Proper sleep is critical to good mental and physical health. Of course sleep helps you concentrate and think more clearly. But research shows that a good night’s sleep is also associated with other positive health outcomes.

Improved brain function is the most obvious benefit of getting enough sleep. In addition to helping you focus better and think more clearly, studies show that those who sleep well are more productive and perform better on mental tasks. Memory improves with adequate sleep, which helps you learn better, improves problem-solving, and allows you to form new insights better.

Healthy sleep patterns, especially during middle age, may also help you avoid dementia as you get older. Mental health issues, such as depression and even thoughts of suicide, are associated with poor sleep quality and sleep disorders, such as sleep apnea and insomnia.

The benefits of bedtime are not limited to the brain, though. Studies show that those who sleep well perform better on physical activities as well as mental ones. Athletic performance, for example, was shown to improve with more sleep. In contrast, functional limitations, such as walking, grip strength, and ability to perform independent activities, was worse for older women who slept less well.

Good quality sleep is essential for other factors associated with chronic illness, too. For example, not getting enough sleep is one of the strongest risk factors for obesity. Adequate sleep helps regulate hormones and other factors that affect appetite and blood sugar (an important factor for those with diabetes), which helps you maintain better weight control.

Getting enough sleep also improves immune function and decreases inflammation. So sleep may be an important part of your therapy if you have an autoimmune disease like myositis, CIDP, myasthenia, and others. Better sleep may also lower your risk for developing serious conditions like heart disease and stroke.

So how do you get a good night’s sleep? Good sleep hygiene is all about putting yourself in the best position to fall asleep easily and sleep well throughout the night. Here are some tips for making that happen:

  • Stick to a consistent sleep schedule. Go to bed at the same time every night, and wake up at the same time every morning, even on weekends. Make sure this schedule allows for 7-9 hours of shuteye.
  • Unwind with a relaxing bedtime routine. Give yourself 30-60 minutes before tucking yourself in to leave behind the busyness of your day. Take a warm bath, meditate, write in your journal, read…anything that helps you de-stress and feel drowsy. Anything, that is, except activities that involve a blue light-emitting screen.
  • Make your bed a device-free zone. Phones, computers, and e-reader devices emit a blue light that reduces melatonin levels in your body, making it more difficult to fall asleep. When you use them before bedtime, they can keep your brain more alert, which also makes it harder to fall asleep. Your phone’s dings, pings, and rings and the light that pops on when notifications come through are also disturbing to your sleep. Best to turn the phone off at night.
  • Exercise every day. Thirty minutes of exercise a day not only improves your health and physical functioning, but it can improve sleep quality as well. Being active outside, where you are exposed to natural light, can also help to regulate your sleep cycle. It’s best, however, to avoid vigorous exercise within an hour or two of bedtime.  
  • Limit caffeine late in the day. Caffeine’s effects can last up to seven hours after your last cup of coffee, making it more difficult to fall asleep.
  • Make your sleep space cool, comfy, dark, and quiet. Bedroom temperatures are better when they’re lower than the rest of the house (between 60°F and 67°F). A comfortable mattress, pillows, and bed linens also go a long way in helping you fall asleep and stay asleep. And keep the room dark and quiet (use earplugs if you must) to keep you from waking up.
  • Manage your stress before you go to bed. Try not to bring your worries to bed with you. Write down what’s bothering you or make a to-do list for tomorrow so these concerns don’t keep you up at night. Meditation or other relaxation techniques can also calm your mind.
  • Save your bed for sleep and sex only. It’s best to avoid working, talking on the phone, watching TV, or other activities when you’re in bed. That way your brain is conditioned to expect to fall asleep when you climb under the covers. Even reading can keep your brain on alert, so it’s better to read in another comfy spot before slipping into bed.
  • Avoid tossing and turning. If you have trouble falling asleep, especially if you wake up in the middle of the night, don’t just lie there. If it takes more than 20 minutes to fall asleep, try getting up and doing something relaxing—drink a cup of herbal tea, read a book (on the couch, not in bed), meditate—until you feel tired.
  • Limit daytime naps. Napping during the day can make it harder to fall asleep and stay asleep during the night, especially for older people. If you must nap, don’t do it late in the day, and limit it to 30 minutes or less.
  • Prioritize sleep. While it’s tempting to work, study, socialize, or exercise late into the night, burning the midnight oil is not a good idea. It’s important to treat sleep as a priority in the same way you do eating three healthy meals every day.
  • Avoid these things: consuming alcohol in large quantities, eating a large meal late in the day, or smoking at any time. All of these activities cause sleep disruption.

Falling asleep and staying asleep can be a problem for some, especially when you’re taking certain medications, such as prednisone. If you’ve tried these tips but sleep continues to be a problem, discuss this with your doctor. He or she can help you figure out whether an underlying medical condition is causing your sleep issue and work with you to solve it.

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Patient communities

Friends Who Get It

Being with people who understand what you’re going through can be a life line for those with a rare disease.

“It’s like explaining how chocolate tastes to someone who’s never had chocolate,” Rose Mary Istre says of trying to explain dermatomyositis to her friends and family. “They don’t get it.”

When she was diagnosed more than twenty years ago, even doctors didn’t have a good understanding of this autoimmune muscle disease or how to treat it. Which made Rose Mary feel very much alone and anxious. This is also what made her want to find other people with myositis, people who had tasted this sort of chocolate and knew what it was like.

She contacted The Myositis Association who gave her a list of people in the Houston area who had some form of the disease. In those pre-internet days, Rose Mary sent everyone on the list a survey with a self-addressed, stamped envelope, asking about their interest in getting together for some sort of support group. Shortly after that, 48 of the 50 people on that list showed up at Rose Mary’s house for their first meeting, including one woman who literally crawled in the front door because she was too weak to walk.

“That’s how desperate people were to find someone else who got it,” Rose Mary says. “But the best part was, we realized we weren’t alone. There was somebody else out there struggling with the same issues we had. Even if we thought we just couldn’t cope with it, someone else was out there coping. It gave us hope. So we held hands and became friends and formed a support group, a community.”

When Lekiha Morgan was also diagnosed with dermatomyositis five or six years ago, she too realized she needed to talk to someone. Rose Mary’s group in Houston was almost an hour away from her home in Galveston, but knew she needed to get herself there.

“People around me kept saying you don’t look sick, but my body was catching hell,” she says. “It was great to be around people I could relate to. They also knew more about the disease and taught me things that I could bring home and share with my family and my kids to help them to better understand what I was facing.”

Feeling less alone and having words to express what you’re dealing with are only a few of the ways support groups help those who live with rare diseases like myositis. Simply being able to talk about your feelings and share the frustrations of living with a disease that leaves you unable to pick up your baby or walk up the stairs can be very therapeutic. Even caregivers can benefit from this form of social support, developing improved coping, lower stress levels, less anxiety, and finding hope and purpose in life.

Each support group has their own way of doing things, but they often involve opportunities to both share personal experiences and learn from expert speakers. Rose Mary, for example, has tapped contacts in the medical community, including her own treating physician, infusion nurses, a clinical psychologist, art therapists, a tai chi instructor, and even members of the group who have a special talent they can share.

Among the more popular speakers are medical professionals who are experienced in treating myositis. Members are eager for their answers to questions about everything from treatments and side effects to research and “am I ever going to get better.”

Group members don’t give medical advice, but they are able to talk about what worked for them and to guide people on how to navigate where they are with the disease. Still, this personal experience is often more informed than some physicians who have little experience working with a person with myositis.

“I believe we actually have saved lives at our support group,” Rose Mary says. “We’ve had people who wander into meetings who are going to a medical clinic or seeing a doctor who is not following what we have learned to be a really strong protocol in combating this condition. So our group is a good resource to let people know what’s out there, what’s available in the medical community, and who to contact.”

Lekiha is one of those people who was struggling with treatments that were not working well to control her symptoms and were causing complications. At the Houston support group, she learned about intravenous immune globulin (IVIG) therapy, which had been very effective for several people, including Rose Mary.

“I was having a hard time getting around, and I couldn’t work anymore,” Lekiha says. “But the doctor I was seeing at the time said I wasn’t affected to the degree that she felt I needed IVIG. After talking with people at the support group, I ended up going to see a different neurologist who said I was a perfect candidate for IVIG. She said they don’t want you to get to the point where you’re completely immobile and they can’t bring you out of it. Now IVIG is the only medication that I’m on. IVIG changed my life.”

For many people who live with rare diseases like myositis, having a support group to depend on is a life-saving experience.

“I’m so glad and grateful for this group,” Lekiha says. “It’s a scary disease to face by yourself, especially when nobody you know knows anything about it, even doctors. I’m so thankful for these support groups. I don’t think I would be here without them, honestly.”

Rose Mary, too, is grateful for the folks who have shown up at her meetings.

“I don’t know how I would have survived,” she says. “I feel like I needed them more than they needed me. I felt like I finally had purpose in helping somebody else. So many people benefited, and helping them gave me real courage to carry on. I shudder to think where I would be had I not had that support group.”

Resources

CSI Pharmacy provides a resources page with information that may be helpful for all of our patient communities.

The IVIG/SCIG Patient & Provider Support Group is a private Facebook group for those who use immune globulin therapies.

Cure JM Foundation seeks to find a cure and better treatments for Juvenile Myositis (JM) and improve the lives of families affected by JM.

The Myositis Association (TMA) is the leading international organization committed to the global community of people living with myositis, their care partners, family members, and the medical community.

Myositis Support and Understanding (MSU) is a patient-centered, all-volunteer, 501(c)(3) nonprofit organization empowering the myositis community through education, support, awareness, advocacy, access to research, and need-based financial assistance.

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Health Information IG Therapy Patient communities

Choices: IVIG versus SCIG

Good. Better. Best.

Many patients with autoimmune disorders and primary immune deficiency diseases depend on regular infusions of immune globulin (IG) to keep them healthy. For most of the nearly four decades since immune globulin therapies have been available, patients have had only one viable option for how this treatment was given. It was only available as an intravenous (IV) infusion.

Since 2006, however, when the first IG product was approved for subcutaneous (SC) administration, patients have had a choice about how they received their treatments. Both products are considered equivalent in terms of efficacy, but there are lots of other factors that may make one preferable over the other. Providers usually have their own sense about how IG should be administered, but we asked IG users for their thoughts on the pros and cons of each option.

Convenience is the biggest factor in which route patients prefer. Ironically, both IV and SC users think their choice is most convenient.

Rebecca, for example, has been getting IVIG for 12 years after being diagnosed with common variable immunodeficiency (CVID). She speaks for many when she says, “I like that I only sacrifice one day every three weeks for treatment.”

The convenience of once-a-month infusions with IVIG comes at the expense of independence, though. IVIG poses higher risks, because it goes directly into the vein rather than under the skin. So it must be given under a nurse’s supervision, whether that is in the hospital, an infusion center, or at home. This means it also has to take place on a schedule that may not always be convenient.

Those who use SCIG usually take their infusions once a week rather than once every three to four weeks or so. Still they prefer the control they have over when they infuse, because they do it themselves. As Brandina, who has myasthenia gravis, says, “I love that I can administer it myself. The treatment days are flexible, and I can take the medication with me, so I don’t have to plan my vacation around treatments.”

Infusing once a week is also inconvenient for some SCIG users, but for most this is a minor drawback. As Jen, who has specific antibody deficiency, says, “I absolutely love SCIG. There are so many more pros that I could list and only this one con.”

Getting infusions at home, whether it is IV or SC, is also a convenience. This has become especially important since the COVID-19 pandemic has made it less desirable to go to a healthcare clinic. Brynne, whose six-year-old daughter uses IVIG for juvenile dermatomyositis (JDM), was grateful when her overnight hospital infusions were changed to in-home infusions because of coronavirus restrictions.

Making the most of infusion time is something IVIG users have worked into their lives. Sitting in an infusion center or even hanging out at home with a nurse for six to eight hours or more can be a huge inconvenience, but it doesn’t have to be wasted time. Dana, who has dermatomyositis, likes IVIG, because it forces her to take time for herself and relax. And Robin, who has CVID, uses the time to crochet.

Mary, whose husband has myasthenia gravis (MG), prefers to get his IVIG at the hospital infusion center for other self-care reasons. “He loves the heated, vibrating recliner,” she says. “And they provide snacks and lunch.”

Adverse effects can be more of a problem with IVIG. In fact, this is often the reason patients switch to SCIG, which has far fewer reactions. Symptoms can range from fatigue, fever, flushing, chills, and ‘‘flu-like’’ symptoms to more life-threatening reactions like anaphylaxis (severe allergic reaction) and blood clots.

The most frequent side effect is headache, which can last several days and be more severe than a migraine. Some, like Lola, who has Sjögren’s syndrome, even get aseptic meningitis (inflammation of the membrane covering the brain) after infusions. This causes debilitating headaches, dizziness, and other symptoms.

Scar tissue and knots of fluid under the skin from subcutaneous infusions was a drawback for those using SCIG. These knots usually disappear within a few hours, though, and any redness or swelling at the injection site usually decreases over time.

Pain from being stuck with needles is not an insignificant side effect, regardless of whether it’s IV or SC. Whether it’s having to stick oneself multiple times or whether it’s having difficult-to-access veins, nobody likes to feel like a pincushion.

This can be especially challenging for children. Nancy’s nine-year-old daughter has JDM and receives IVIG at a pediatric infusion center. She says having ultrasound to find and insert the IV needle makes a world of difference for her daughter. Being spoiled by the nurses also takes some of the sting out of the whole ordeal.

Fluctuations in therapeutic effect is another reason many people switch to SCIG. An IG dose is mostly metabolized by the body over about 22 days, whether it’s given IV or SC. With IV infusion the dose reaches its peak immediately and dissipates over the next three to four weeks. This means that some patients will feel their symptoms returning as IG levels in the blood go down.

“As I got closer to my next treatment date, I would start to feel the effects of needing my next treatment,” says Karon, who has MG. “After I received it, I could tell I had just received a boost and had more energy.”

Giving IG under the skin makes the blood levels rise more slowly. And because SCIG is given more frequently—usually weekly—IG levels in the bloodstream fluctuate far less, so patients don’t feel that fatigue and other symptoms returning.

Whatever you decide about IG therapy, Lea, who has used IVIG for 22 years to treat CVID, offers this important advice: “You have to listen to your body and watch how it reacts to everything and try things until they work for you.”

For those who would like to learn more about IVIG or SCIG, please contact the CSI Pharmacy advocacy team at [email protected].

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Health Information

Get Your Flu Shot Now

Dale wearing blue gloves giving a flu shot to a patient.

For some, getting a flu shot is just part of the fall routine every year. Immunization against the virus that causes influenza prevents millions of people from getting the disease, makes the disease less severe if you do get it, and can prevent death from severe disease and its complications. According to the Centers for Disease Control and Prevention (CDC), influenza causes an estimated 12,000 to 61,000 deaths every year in this country.

This year, because of the COVID-19 pandemic, physicians and public health officials say it’s even more important that everyone over the age of six months get a flu shot. This is especially important if you have an underlying health condition that makes you more at risk for developing complications if you get the flu. While the influenza vaccine will not prevent you from getting COVID-19 (researchers are still working on a vaccine for this different virus), it will help you avoid getting seriously sick with or—heaven forbid—dying from the flu. And the fewer people who get the flu, the more it saves healthcare resources that are still urgently needed for treating COVID-19 patients.

Those who are at high risk for flu complications are also at greater risk for getting COVID-19 and having serious outcomes. If you have an autoimmune condition or immune deficiency disease, such as myositis, myasthenia gravis, pemphigus and pemphigoid, chronic inflammatory demyelinating polyneuropathy (CIDP), and primary immunodeficiency (PI), this means you. You are at a much greater risk for getting sick with the flu and at greater risk for developing complications like pneumonia if you do. If you have heart disease, cancer, or diabetes, getting the flu can also make these conditions worse.

Getting the flu vaccine, however, is not a straightforward decision for some who have these conditions. For example, some people with a history of Guillain-Barré Syndrome (GBS) may have developed this form of muscle paralysis after receiving an influenza immunization. According to the GBS|CIDP Foundation International, the association between GBS and flu vaccines is inconclusive, but they suggest these individuals avoid the vaccine in the future. If you have had GBS, especially if it developed four to six weeks after getting a flu shot, you should talk with your doctor about the risks and benefits of getting the vaccine again.

Another consideration is the aerosol form of the flu vaccine. This is a live attenuated (weakened) influenza vaccine that is given through the nose. While the injected vaccines are made with inactivated virus, the nasal spray is made with live organisms that have been weakened but are still able to activate the body’s immune response against the disease.

The intranasal vaccine is not recommended for those younger than two, older than 50, or those who have a weakened immune system, including some patients who take immune suppressing medications. If you care for or live with someone who is immune compromised, you should also avoid the nasal spray. And if you have an underlying medical condition that puts you at risk for developing severe complications from the flu (such as chronic lung disease, heart disease, kidney disease, liver disorders, neurologic and neuromuscular disorders, blood disorders, and diabetes), it’s important to check with your physician before taking the nasal flu vaccine.

According to the Immune Deficiency Foundation, those with certain forms of immune deficiency (common variable immune deficiency [CVID], severe combined immune deficiency [SCID] or Bruton’s agammaglobulinemia) are unable to develop protective immunity following vaccination. This means their bodies don’t have the infrastructure to develop the immunity needed to keep them from getting sick, so vaccines will not do them any good. In fact, for individuals with these forms of PI, live vaccines—including the influenza nasal spray—may put them in danger of developing severe disease.

The only reason not to get a flu shot is if you have a severe, life-threatening allergy to the vaccine or any of its ingredients. This might include gelatin, certain antibiotics, or other ingredients. If you get hives when you eat eggs, studies have shown that most times you can still get the flu shot. If you have a more serious reaction to eggs or you are worried about this, you should of course talk with your doctor. Egg-free alternative vaccines are available.

The CDC has more information about seasonal influenza and how to prevent it.

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Advocacy Patient stories

Disability Benefits in Danger

Andrea Williams

Andrea Williams had a panic attack when she opened her mail earlier this year. Her hands were shaking even before she tore into the envelope from the Social Security Administration (SSA). The letter inside informed her that, according to SSA, she was no longer disabled and would no longer receive disability benefits, including Social Security Disability Income (SSDI) and Medicare.

Andrea is one of thousands of American citizens who are unable to work because of severe, chronic health problems who can expect to receive these notices in the coming year. This is because SSA wants to cut $2.6 billion dollars from SSDI and its sister program Supplemental Security Income (SSI) by changing the rules they use to terminate people like Andrea, who have depended on the disability safety net for years.

Six years ago, Andrea was diagnosed with a rare, debilitating disease of the muscles called myositis. She had trouble lifting her head, she couldn’t pick up her newborn baby, she couldn’t climb stairs, she couldn’t even lift her arms enough to wash her hair. For months she went back and forth to the doctor saying, “I feel like I’m dying.” It wasn’t until she was referred to a specialist and ended up in the hospital for a week that she was finally diagnosed.

Myositis causes chronic pain, disabling weakness, and extreme fatigue. Treatment has helped Andrea, but she has lost too much muscle tissue that she will never regain. She can’t stand for any length of time, she drops things, and the brain fog from her medications makes it difficult for her to think. Her doctor told her she would never work again. When she applied for SSDI benefits in 2016, her application was accepted on the first try, which almost never happens.

“I’m scared to death,” she says. “My biggest fear is not having the medications, and I already can’t afford the doctors.”

An “Explanation of Determination” letter like the one Andrea received is a notice an SSDI or SSI recipient gets when they have been identified by SSA for “Continuing Disability Review” (CDR). This is the agency’s review process to see if beneficiaries are still medically eligible for the program. After this review, if SSA believes the person no longer meets their criteria for disability, their benefits are terminated.

A medical CDR is done at least once every three years, unless the SSA expects your medical condition to improve sooner. Those who have a medical condition that is not expected to improve undergo a CDR every seven years.

A rules change that took effect earlier this year, however, dramatically increases how often a person must undergo a CDR review. This change adds a new category for those whose condition is “likely to improve.” Hundreds of thousands of people now in less frequent CDR categories will be moved into this new category to be reviewed every two years.

This new rules change is especially disturbing because it targets people like Andrea who have chronic conditions that flare up unpredictably. Those who are approaching retirement age and those with mental illness are some of the other people this rules change aims to remove from benefits.

If you get one of these letters, the first thing you need to do is notify Social Security that you want to appeal this decision. To appeal, you need to submit a Request for Reconsideration form within 60 days of receiving the notice of denial of benefits. If you want to continue receiving benefits while your case is being decided, you will need to submit this form withing 10 days of the denial, and you need to specifically ask that benefits continue. Be aware, however, that if your case is unsuccessful, SSA can require you to return the overpaid benefits.

Andrea has followed this advice and is awaiting a response from SSA. She is hopeful that, with the support of her doctors and her patient advocate, her appeal will be granted and she will once again be able to sleep at night.  

For others like her, Andrea offers this advice:

“You have to reach out and get some help from someone like a patient advocate,” she says. “You have to have your medicine. You have to see your doctors. You can’t take no for an answer.”

CSI Pharmacy has patient advocates who can help you navigate this and other health care access challenges. We offer this service regardless of whether you are a CSI Pharmacy patient.

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Patient stories

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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Patient communities Patient stories

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.”