Author: onenine

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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.

Categories
Patient communities

Finding Hope through Community

Larry and JoAnn smiling at CSI Pharmacy success

Larry Leisher had no idea what inclusion body myositis (IBM) was when he was diagnosed with this slowly progressive muscle disease 16 years ago. He’d been having trouble going up and down the stairs for a while before that. His legs were looking very thin, and he was falling too often for it to be normal. When the diagnosis came, it threw Larry and his wife JoAnn for a loop.

“The biggest problem was not having any idea where it came from or how it started,” Larry says. “I’d never really been a sick person, so to have this come upon me the way it did was really devastating for both JoAnn and me.”

The Leishers are not alone in feeling confused and overwhelmed. Being diagnosed with a rare, chronic condition, especially one like IBM that has no treatments, can be one of the most difficult and stressful life experiences, for both the patient and her or his partner. Suddenly, a spouse becomes a caregiver and the life they thought they were living has been upended. Plans for the future, work and social life, and relationships all have to be retooled to include medical interventions and disability. None of that is easy.

About a year into his diagnosis, Larry ran across an article in the local paper written by Lee Thames, the first person Larry ever heard of who had IBM. So he called him.

“Lee and I got together and decided to start a support group,” Larry says. “At first it was just he and I. Our first meeting was in February of 2007, and it’s been going ever since.”

Getting together with other people who also live with myositis can be a life saver. It’s a way to learn more about this condition that most people have never heard of before. And because no one else in your life really knows what you’re going through, sharing the story of your journey with someone else who understands the challenges of living with this life-changing disease can lighten the load, whether you’re the patient or the caregiver.

Over the years, the myositis support group Larry helped to start in Jacksonville has grown to include 20-25 people, both patients and their caregivers. Eventually they connected with The Myositis Association (TMA) and became an official part of the organization’s national network of support groups.

Before the pandemic ended in-person meetings, they would try to get together quarterly. Larry arranged for speakers to come in and talk with the group about different aspects of their disease. 

“A support group is a good thing to have,” Larry says. “It gives hope to those who have none. It gives people a chance to talk with others who have the same difficulties. We talk about things that get us through the day, things that you can’t just turn on the computer and find.”

But it’s not just about the meetings. Larry works hard to make sure members of his group are taken care of. He calls everyone every few weeks, just to check in. And pre-pandemic, he and another member, Jim Farrlley, would also get in the car and drive as far as 70 miles to visit members who were no longer able to make it to meetings.

These efforts have paid off. Before the pandemic, the group was so connected that, even after their loved one passed away, a couple of caregivers still attended in-person meetings. And more than one member continued with the group even after their doctor decided they didn’t have myositis after all. 

Like everything in our lives over the last year or so, however, the social distancing has made it difficult for members to be there for each other. While many events have moved online through video meeting apps like Zoom, that hasn’t been an option for the Jacksonville group. Too many of the members are unfamiliar and uncomfortable with this technology, and some don’t have access to a computer.

So Larry has been on the phone a lot. He calls members weekly or biweekly, just to let them know they’re still part of a supportive community that cares about them and to keep them abreast of what’s going on at TMA and in the myositis research world.

“It’s been hard since we can’t get out to visit people,” Larry says. “So I just try to keep the conversations going. A lot of people are disappointed because we’re no longer getting together. Things are getting more and more difficult for folks…problems at home and such.”

More than anything, Larry has an unwavering positive attitude. He is committed to his mission of passing on hope to folks who may be feeling even more lost and alone than in normal times.

Recently, for example, a report on arimoclomol, a new medication being tested for IBM, showed the drug had failed to meet research expectations in clinical trials. It was devastating news for IBM patients who had pinned all their hopes on this, the only drug anywhere close to being a possible therapy for their disease.

“I contacted our members who have IBM and let them know about the results,” Larry says. “But I also let them know that, although it failed, this was not a time to give up. We still have to have hope. We still have to support the research and TMA.”

In the end, this optimistic attitude and sharing it with others keeps him going too. He refuses to let this disease take him down.

“I don’t want to lose the group,” Larry says. “Even if it’s shrinking in size, I still feel that, between our group and TMA, there’s a lot of hope that can be passed around.”

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Advocacy Patient stories

Buyer Beware

Successful client photo

The cost of health insurance had been a struggle for Diane Steele for years. Even the plans offered by her husband’s employer were too expensive when they had children at home. For a while, the family had to go without insurance. When Diane learned about health share programs, she saw this as an affordable way to protect her family.

Health share programs (sometimes called medishare or healthcare sharing ministries) are faith-based programs in which members pay a monthly “share” (notably not called a premium), which is pooled together with other members to pay for approved medical expenses.

While health share programs have been around for decades, because they are significantly less expensive than traditional health insurance, their popularity has increased in recent years as healthcare costs and insurance rates have skyrocketed. Part of the reason they are less expensive is they are not actual health insurance.

Program guidelines for what will be covered are often limited and may be determined by religious beliefs rather than medical need. Some health shares, for example, specifically exclude things like birth control, addiction treatment, or conditions related to smoking or alcohol abuse. Health share programs may even require members to sign a statement of faith, and some require verification of regular church attendance.

Because these programs have a religious affiliation, they also are not subject to mandates imposed by the Affordable Care Act (ACA), such as coverage for preexisting conditions and no lifetime caps on coverage. Most also specifically exclude drug coverage, especially for long-term treatment of chronic conditions such as diabetes or heart disease. Instead, members are steered to prescription discount cards like GoodRx and NeedyMeds to help pay for insulin and high blood pressure medication.

“We went with the health share because of the cost, and because we liked the idea of helping other people,” Diane says. “The funds go into a universal plan, and as people need it and qualify for it, they cover your costs. The only thing is you have to get approval up front for any kind of major outlay.”

Having to get approval for major healthcare expenses seemed like a small concession for Diane and her family, one that seemed routine when her husband needed major open-heart surgery a few years ago. The health share covered all of the expenses of that hospitalization with little hassle.

When Diane was diagnosed with chronic inflammatory demyelinating polyneuropathy (CIDP) in 2017, however, she discovered limitations that were much more of a hassle. CIDP is a rare disease of the nerves and muscles in which inflammation causes disabling numbness, weakness, and pain in the legs and arms. It is a chronic condition that often requires a lifetime of treatment.

“I was so sick,” Diane says. “I couldn’t walk. I couldn’t drive. I was basically in a wheelchair most of the time. And I was gradually getting worse and worse.” 

Among the medications her neurologist prescribed for the condition was Gammagard, a form of intravenous immune globulin therapy (IVIG). This treatment, derived from donated human plasma, is very expensive, but it’s also very effective for a wide variety of autoimmune conditions like Diane’s. IVIG treatments did allow Diane to return to a more normal life, one that no longer includes a wheelchair, but the health share refused to pay for it.

“I worked and worked with the health share,” Diane says. “I talked to all kinds of people over there, and often had to ask for the supervisor’s supervisor. It was so complicated. They didn’t even know what Gammagard was.”

The health share required Diane to have a number of different tests to prove the diagnosis. They also required her to try other, less expensive medications (a process known as step therapy) to show that these treatments didn’t work for her condition. And they wouldn’t approve it just based on the doctor’s prescription. Her neurologist had to send them an extensive written report to justify the scientific basis for using IG for Diane’s disease.

To be fair, Diane’s experience with the health share program was not unique. Even traditional health insurance companies require preapproval for expensive treatments like IG therapy. Many also impose limitations on doses or frequency. There is often a large copay, and some will completely deny coverage for the treatment, especially if it is being prescribed off label (for a treatment that is not approved by the FDA for a particular condition).

But even after jumping through all these hoops, the health share only approved the treatment for forty-five days as a test to see if it would work. And the dose they allowed was also lower than what the doctor prescribed. Even when it did prove to be effective, the health share refused to cover the cost.

After the forty-five-day trial period, Diane worked with her specialty pharmacy to access the life-saving treatments she needed. They helped her apply for a special patient assistance program through the manufacturer that provided the medication for free. The specialty pharmacy then provided the nursing services and supplies at no cost. They also helped her apply for Social Security Disability, which would allow her to enroll in the more comprehensive benefits available through Medicare.

Diane’s story is a cautionary tale that underscores the need to know what you’re buying when you purchase healthcare coverage. All plans have restrictions and limitation. But when a plan is so inexpensive compared to the norm, you have to be especially careful to understand what they will pay for and be sure it covers the care you need.

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Advocacy IG Therapy

Don’t Take No for an Answer

The agency licenses, permits, and oversees pharmacists and pharmacies, as well as the distribution system where there is the sale, delivery, or distribution

Karen has struggled for many years with intense pain and muscle weakness, symptoms of chronic inflammatory demyelinating polyneuropathy (CIDP). This rare nerve disease causes gradually increasing loss of sensation, numbness and tingling, intense pain, and weakness in the extremities. For the last two years, though, Karen has been doing well with monthly infusions of intravenous immune globulin (IVIG) therapy.

Now, however, Karen is terrified that this life-saving treatment will be yanked away from her, not by her doctors, but by her health insurance company.

Karen receives IVIG infusions at home through an independent specialty pharmacy. But after every monthly treatment, she receives a letter from her insurance company’s pharmacy benefits manager (PBM) telling her that her plan requires that she obtain this medication from their preferred specialty pharmacy.

“We’ve just been living with this every month,” Karen’s husband David says. “It’s so nerve racking. We’re always afraid that they will deny coverage and we will get a bill for thousands of dollars.”

This scenario is happening more often lately, according to James Sheets, CEO of CSI Pharmacy, a small, independent specialty pharmacy that caters to patients who use IVIG. Between five and ten percent of new referrals to his pharmacy are rejected by the patient’s insurance plan because of preferred pharmacy limitations.

“The thing is, this is not always true,” James says. “Even when the claim is denied, we have to do some investigating to see what the truth really is. Often, we are, in fact, able to provide services to these patients.”

So far, Karen is still able to get her treatments through her pharmacy of choice. But last month the PBM, a middleman in servicing pharmacy benefits for health insurance companies, stepped up the pressure. In a letter she received, they stated that there was a nationwide shortage of IVIG and again stated Karen was required to switch to their preferred pharmacy.

Someone from the preferred pharmacy even followed up with a phone call out of the blue, insisting that her health plan would not pay for the treatment if she didn’t get her medication through their company, a large, nationwide chain that is financially linked to both the insurance company and the PBM.

“IVIG is extremely expensive,” Karen says. “There’s no way we can pay for it ourselves. They’ve been writing these letters and now they’re calling me. I’m terrified! These people have no concern for my diagnosis or my medical care.”

While plasma donations decreased last year as a result of pandemic restrictions and many expect some immune globulin (IG) products to be in short supply, an IG shortage has not yet materialized. In fact, the only agency authorized to make a declaration of a drug shortage is the Food and Drug Administration (FDA), and to date no such declaration has been issued for immune globulin.

“It’s just wrong that there is an IG shortage,” James says. “Even if there were, the pharmacy in question would not be the only place that has availability of IG products. In fact, patients at small pharmacies like CSI Pharmacy would be more likely to be protected than those at a large national chain pharmacy.”

For patients like Karen who might be feeling more pressure to change to a new pharmacy for specialty medications like IVIG, James has a few suggestions. For starters, if you are already receiving services that you are happy with, don’t automatically agree to change.

“Be an advocate for yourself,” James says. “Tell your plan, look, I have been stable with my current pharmacy. I’m happy with the services they provide. I have a good relationship with my infusion nurse. I do not want all that change, and I would like to opt out and continue to use my current provider.”

It’s also a good idea to call your specialty pharmacy’s patient advocate and let them know what’s going on with the insurance company. You have a relationship with the people at your pharmacy, and they can do the necessary investigating to see if anything has changed with your coverage.

As Karen and David found out when they called their specialty pharmacy, the insurer was continuing to cover the cost of Karen’s infusions, despite the threatening letters they were sending to her. And her pharmacy had no intention of billing her for anything more than her copay.

If you have Medicare and your IVIG is covered under the Medicare Part D drug plan, you are protected against this practice. Language in the Medicare legislation specifies that patients can choose to get their medications from “any willing provider.” So by law, insurers are not allowed to force patients to a certain pharmacy.

“I believe that patients deserve to have the power to make their own health care decisions,” James says. “That’s when the best care takes place. I hate to see patients being forced, against their will, to use specific pharmacies simply as a business decision. The problem with that is there’s no incentive for the big chain pharmacies to do a great job, because when patients don’t have a choice, they never have to make patients happy in order to keep their business.”

CSI Pharmacy would like to know if this sort of pressure to change pharmacies is happening to others. If you’ve received communication from your insurance plan or their pharmacy benefits manager urging you to move your care to their preferred specialty pharmacy, please drop us a note at [email protected].

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Health Information Patient communities

COVID Vaccine and Our Patient Communities

COVID-19 Vaccine Bottle that says Injections Only
Photo by Artem Podrez from Pexels

Across the country we are starting to see the light at the end of the tunnel as the rollout of COVID-19 vaccines has begun. For many of our patients with primary immunodeficiency diseases or autoimmune conditions, however, getting vaccinated is not a straightforward decision. They have lots of questions and must weigh carefully a number of considerations.

Immunologist and rheumatologist Dr. Terry Harville has some concerns, too. “There are questions in my mind that haven’t been fully answered,” he says about the new vaccines that have recently been granted emergency use authorization by the Food and Drug Administration (FDA). “And I’m worried that there will be unintended consequences.”

The fact that we have only two months’ worth of safety data from the clinical trials testing these new vaccines from Pfizer/BioNTech and Moderna leaves many medical professionals worried about longer-term effects. Others are unsure about how the vaccines will react in patients with rare autoimmune and primary immunodeficiency diseases, because testing did not include people who live with these conditions.

Despite these concerns, Dr. Harville—along with most other healthcare practitioners—stresses that most people should still get the vaccine. Because when hundreds of thousands of people get COVID and one or two percent of them die, the statistics overwhelmingly support COVID-19 vaccination.

Dr. Harville, who is a professor of medicine at the University of Arkansas for Medical Sciences and an expert in primary immunodeficiency disorders, offers some guidelines for those who are trying to make a decision about the vaccine and might be at greater risk for complications from it.

If you have a primary immunodeficiency (PI) disease, you probably already know that you need to be careful about taking immunizations, especially if they are made with live virus. The COVID-19 vaccines that are currently being administered, however, are not made with live virus. They are created from a man-made part of the virus’s genetic code called mRNA. This means they are safer for those who are unable to take live virus vaccines.

With a few exceptions, Dr. Harville says, “It is the general consensus that we don’t see any reason to not vaccinate those with primary immune deficiency diseases.”

One exception is interferonopathies, IDs associated with dysfunction in components of the immune system called interferons. Another exception is CTLA4 deficiency. These are conditions in which patients tend to develop autoimmune disorders. Dr. Harville recommends that individuals with these types of ID should probably not get the currently available COVID vaccines. While there is no data to support this at this time, there is concern among immunologists that vaccination in this group of patients may trigger an overly aggressive immune response that would be harmful. As more data becomes available, better recommendations will be forthcoming.

If you use immune globulin therapy, there appears to be no reason you shouldn’t get the vaccine. In fact, even if you get the disease, you may actually be better off than others.

“What’s interesting is,” Dr. Harville says, “if you look at SARS-CoV-2 infection in patients with antibody deficiencies—whether that’s X-linked agammaglobulinemia (XLA) or common variable immunodeficiency (CVID)—when these patients are receiving appropriate immunoglobulin (IG) replacement therapy, they tend to have milder disease.”

Intravenous immunoglobulin (IVIG) has even been used, in some cases, to treat COVID-19 infection. While clinical trials to prove effectiveness are still ongoing, some physicians believe IVIG does decrease the risk of severe complications. IG manufactured from the plasma of people who have recovered from COVID-19 infection is also being tested as a treatment.

If you have had a severe reaction to immunizations in the past, this may be a reason to opt out of getting the COVID vaccine. This includes those who have a primary immunodeficiency disorder and developed the infection after receiving a vaccine. It also includes autoimmune disease patients who have experienced a severe flare of their symptoms after a vaccination.

In the 1976, there was a rise in cases of the autoimmune condition known as Guillain-Barré syndrome (GBS) that was thought to be triggered by the swine flu vaccine given that year. This was an inactivated virus vaccine, not the type of mRNA vaccine we now have with COVID. Nevertheless, if you’ve had GBS in the past, be sure to let your healthcare provider know this, as it may be a consideration for whether or not you should get the COVID vaccine.

During the COVID vaccine clinical trials, a very small number of participants who received the Pfizer vaccine developed a severe, life-threatening allergic reaction. Since then, others have also had what is called an anaphylactic response soon after getting vaccinated. If you have ever experienced shortness of breath, tightening in the throat, swollen lips or face, or other severe allergic symptoms, you should probably not get this vaccine in particular. (Other vaccines using whole virus are currently in development and will likely be safer.) Anyone who does get a COVID vaccine should be observed for at least 15 minutes and be sure the healthcare personnel administering it have emergency equipment needed to respond to anaphylaxis.

Regardless of your disease, everyone can reduce their own risk. Dr. Harville stresses that, even if you get vaccinated, it’s still important to wear a mask, wash your hands, and maintain physical distance from others.

“Ultimately whether you take or do not take the vaccine is an individual patient decision,” Dr. Harville says. “This should be made after weighing all the risks and benefits and based on a discussion with your physician.”

We highly recommend that you learn more about the risks and benefits of COVID-19 vaccination as it relates to your particular situation. The Centers for Disease Control and Prevention (CDC) offers these guidelines for COVID-19 vaccination for those with underlying medical conditions. In addition, a number of disease organizations offer educational programming about the impact of the virus and the vaccine on specific disease conditions.

Please also see disease-specific statements about COVID-19 vaccines from these patient organizations:

Guillain-Barré syndrome – GBS|CIDP Foundation

Immune Deficiency Foundation

Myasthenia Gravis Foundation of America

The Myositis Association

Myositis Support and Understanding

Platelet Disorder Support Association

Categories
Advocacy IG Therapy Patient stories

IG Shortage is Coming

Bonnie and Walter Joslin smiling in front of their home door.

Walter “Kip” Caro started noticing tingling, numbness, and pain in his feet back in 2007. Gradually these symptoms progressed up his legs and into his body to the point where he had to be hospitalized because his torso was affected and he couldn’t breathe. It took three years for doctors to diagnose him with chronic inflammatory demyelinating polyneuropathy (CIDP). In that time, Kip suffered permanent nerve damage in his
feet.

For the last 11 years, however, Kip has been treated with intravenous immune globulin (IVIG), which has worked like a charm. He never has to worry about his symptoms, unless he can’t get his treatments every three weeks. If he doesn’t, as happened last June when there was a shortage and his infusion center couldn’t get immune globulin (IG) products from their distributor, his disease starts to relapse.

“It was scary,” he says of this experience.

“Over the years, we’ve met people said they could make a change to their treatment schedule, and their symptoms would not be affected,” says Kip’s wife Bonnie Joslin. “But we’ve learned, for Kip, his body just can’t do that.”

Kip feels lucky that Bonnie is such an advocate for him. By the time his infusion center called and told him they weren’t able to get his medication, Bonnie had already been reading about other patients’ experiences
online. She reached out Facebook and was put in touch with CSI Pharmacy, which was able to get Kip hooked up
with home infusions within a week.

“He was already starting to relapse,” Bonnie says. “But CSI pushed it through, and Natalie Edwards in the insurance department worked to get approval. I know it wasn’t easy, but it was like a miracle
happened.”

By early 2021, however, nearly all IG manufacturers expect to see shortages again. This time it’s because pandemic shutdowns earlier in the spring have decreased plasma donations. Plasma is the raw material from which IG and other protein therapies are made. The manufacturing process takes seven to nine months to create IG from donated plasma, so unlike last year’s shortage, this time we know it’s coming and can be prepared.

When IG products are in short supply, rationing does happen. Certain sites of care may stop treating outpatients. If they do treat outpatients, they may limit treatment to primary immune deficiency, Kawasaki disease, and transplant patients.”

That’s because supplies of IG are not universally distributed. Hospitals and infusion centers have contracts with
certain manufacturers or distributors, so they can only get specific brands. Last year, patients like Kip who were served by these sites of care sometimes found their treatments delayed or dropped completely.

Specialty pharmacies like CSI Pharmacy that offer home infusion services, however, have access to more brands
of IVIG as well as subcutaneous immune globulin (SCIG). In 2019, CSI Pharmacy was able to work with patients, their healthcare providers, and their insurance plans to allow them to continue IG treatment in the home.

At times this meant switching to a different brand of IVIG when certain brands were not available. Some patients even switched to subcutaneous (meaning under the skin) infusion after being on the intravenous (IV) form.

While changing to a different product can be scary for patients who have confidence in a brand of IG that is working well for them, please be assured that  a switch can be relatively comfortable if you
work closely with your doctor and pharmacist. The pharmacist can find a brand that is close in formulation to your current brand, for example. If side effects become a problem, the infusion rate can be reduced
and pre-medications prescribed. 

If you depend on IG therapy, now is the time to prepare. We suggest you have a conversation with your provider and current site of care. 

  • Ask your provider what other brands are good options if your brand is in short supply, and ask your care site if they are able to obtain one of these brands if needed.
  • Ask the site of care if they will prioritize patients if there is a shortage. Will it be based on disease, IgG levels, and/or rationing product by the number of grams or frequency?
  • Ask how the site of care handled past shortages.

If it turns out that your site of care does not prioritize patients with your disease or they are unable to obtain your brand of IG, you may want to consider how important is it to you that you continue IG therapy. (Some patients feel comfortable skipping their treatment for a month or two if it means they don’t have to make changes.)

If you’re like Kip, however, and can’t function without regular IG infusions, you’ll need to think about what options are available to enable you to access this treatment. Are you willing to switch brands? Are you willing to move to home infusion? Are you willing to switch to SCIG?

If home infusion becomes your alternative, we urge patients to make that move now. Specialty
pharmacies are more flexible and were less affected during the last shortage, but that may not be the case this time. 

If you are a patient of a specialty pharmacy when this crisis hits, you may be able to get access to IG since they do not ration based on disease and have access to more brands. However, we do not know how the shortage will affect sites of care. 

At CSI, we want to see all patients have access to all brands in their preferred site of care. But we just don’t know what the impact is going to be. We expect this shortage will be across all manufacturers. Patients should understand that this is not going to be a perfect scenario. You may still run into hurdles like access to your preferred brand.

CSI Pharmacy’s patient advocates are available to discuss your options related to IG therapy, regardless of whether or not you are our patient. If we can’t service your needs, we will gladly help you find other resources. No patient should suffer alone.

Categories
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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Patient communities

Stumbling onto Rare Disease

Susan Foley

Susan Foley used to brag that if she told you she forgot, she was lying. For most of her life, she remembered everything. In the last ten years, however, her mind is more like someone with a traumatic brain injury. She loses her train of thought, and sometimes she can’t get the right words to come out when she talks.

Susan has Hashimoto’s encephalopathy (HE), a type of autoimmune encephalitis (AE) that causes swelling in the brain. When this happens, patients experience an odd array of symptoms. They can say strange things, act like they have no feelings, and stumble, as Susan says, like a drunken sailor. Too often, family members and even doctors think these people are just behaving badly. So getting an accurate diagnosis for this rare disease can be a challenge.

About a year before she was diagnosed, Susan started having trouble walking up steps, noticed tremors in her hands, suffered debilitating headaches, and couldn’t make her bed in the morning without needing to take a nap. She blamed these symptoms on her stressful job. When she began having absence seizures (a type of epilepsy in which the person blanks out for a few seconds, staring off into space) it scared her. She knew she needed to get help.

“I had so many different symptoms that the doctors had no idea what was going on,” Susan says. “My primary doctor kept telling me it was stress related, but I knew there was something more going on. Finally, I asked her to send me to a neurologist.”

Still, it took almost a year of every imaginable diagnostic test and a trip to see a specialist at a large teaching hospital for the neurologist to put all the pieces together. Among the tests the specialist did was a blood test for thyroid antibodies. The results were off the charts. This was the clue the neurologist needed.

“HE has nothing to do with your thyroid,” Susan says. But doctors have noticed that this form of encephalitis is associated with elevated thyroid antibodies. As in Susan’s case, this is often how physicians identify the disease.

Having a diagnosis is one thing but figuring out what this means for your life is a whole different process. When Susan went to the internet in search of accurate information about HE, all she found was a single article. It took a lot more digging through online forums and support groups to find even one other person who had the disease.

That one other person turned out to be Nicola Nelson, a Chicago attorney who had also been recently diagnosed with HE. The two became good friends and, when Susan told Nicola about her idea to write a book, she wholeheartedly agreed.

“I had big dreams, you know?” Susan says. “I thought, people who are diagnosed with HE have no information. They need to know from other patients what to expect, what that patient has gone through.”

Hashimoto’s Encephalitis: A Guide for Patients, Families, and Caregivers, now in its second edition, is still the only book about HE available. It includes stories of patient experiences along with interviews with prominent physicians who specialize in the disease.

In addition to the book, Susan and Nicola started a nonprofit patient organization to provide ongoing support and information for those affected by HE and other autoimmune encephalopathies. HESA (Hashimoto’s Encephalopathy/SREAT Alliance), provides both scientific and experiential information on living with HE (also known as steroid-responsive encephalopathy associated with thyroiditis [SREAT]; encephalopathy associated with autoimmune thyroid disease [EAATD]; and nonvasculitic autoimmune inflammatory meningoencephalitis [NAIM]).

HESA raises awareness and advocates for the HE community and raises funds to support research that patients hope will one day shed more light on the underlying cause of HE/AE and create new, more effective therapies. Last year, HESA joined together with other AE organizations to host a symposium to educate physicians and other healthcare professionals. (Recordings of these and other presentations are available on the HESA website.)

HESA’s support groups include two private Facebook pages, one specifically for teens. Susan and her medical advisory board also consult with individuals privately on everything from physician referrals to how to get insurance to approve IVIG therapy to sensitive issues such as incontinence.

Despite the fact that Susan’s career in business ended when she became sick, she’s busier now than she’s ever been. HESA is her labor of love.

“I will tell you, when one door closes, another door opens,” she says. “HESA has been a life saver to me. I still feel like I’ve got something to give, and I tell people when they’re feeling sorry for themselves, they need to find a purpose. Do not feel sorry for yourself. Do something. It will give you a reason to get up in the morning.”

One of the most effective therapies for autoimmune encephalopathy is IVIG. CSI Pharmacy is excited to welcome HESA and those with AEs into our family of care.

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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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Health Information

Clinical Trials for Rare Diseases

Test Tubes and microscope

The idea of clinical trials is much in the news these days as medical researchers work hard to identify new treatments and vaccines for COVID-19. A clinical trial is a formal research study that, among other things, tests new therapies to see how well they work and if they are safe to use in humans. It’s an essential step in the Food and Drug Administration’s (FDA) process for approving new therapies.

For those who live with rare diseases, though, clinical drug trials have a more direct impact on their day-to-day lives. Many rare disease patients have few or no options when it comes to effective treatment of their symptoms. Even when medications with confirmed effectiveness are prescribed, they often are used without FDA approval (a practice known as off-label use), which sometimes makes it difficult to get insurance to cover the cost.

Getting new medications to market requires extensive testing in order to attain FDA approval. These studies need to have a sizable number of participants in order to show accurate results. That’s not a problem for common diseases like heart disease and cancer. But with so few people with diseases like myositis, myasthenia gravis, or pemphigus, it can be difficult to get enough qualified participants to show clear results.

That’s why many of the patient organizations that we work with encourage their members to consider taking part in a clinical trial if at all possible. This can be a big decision, one that you should discuss with family members and your doctor to be sure they are comfortable with your participation and the role they may need to play in the process. But it’s a great way feel like you are contributing to a cure.

Clinical trials are not limited to testing medications, but here we focus on clinical trials testing safety and effectiveness of new drugs. We’ve outlined answers to some common questions that can help you understand the general process of participation in such trials. Specific questions about a particular trial can be answered by the study coordinator of that trial.

How does a clinical trial work? Each clinical trial follows a strict set of rules outlining who can participate, what processes will be done, how participants are protected against risks, how long the trial is expected to last, and more. This protocol is designed to clearly answer specific research questions about the treatment. These protocols are outlined for all medications being tested in the US on the NIH website ClinicalTrials.gov. Here you can search for a trial based on disease, drug, location, or other criteria. Each trial also has contact information for additional information.

What is informed consent? All research participants have the right to know exactly what they are getting into. Informed consent is the process of providing you with essential information about the study before you decide to take part. Members of the research team will provide written and verbal explanation of the details of the study, including its purpose, how long it’s expected to last, tests or procedures that will be done as part of the research, and who to contact for further information. All known risks and potential benefits are also explained and included in the document. Before you decide to sign this document (which means you agree to participate) you should ask questions and be sure you understand everything included in it. It’s important to note that taking part in a clinical trial is voluntary, and you can leave the study at any time. 

Why do some patients not receive the treatment? Clinical trial protocols often involve comparing outcomes of patients who receive the new drug or treatment with those who are given a placebo (a non-active substitute). Most times neither the researcher nor the participant knows whether the drug or a placebo is being given. Participants are usually randomly assigned to the treatment or the placebo group. In this way, researchers can clearly see that the medication was responsible for the effect and not some other cause, such as participants getting better on their own.

What are “phases” of clinical trials? New medications and other treatments take place using a step-by-step process that begins in the laboratory (preclinical phases) then moves on to testing in humans. Each of these phases seeks answers to different questions.

These are the questions posed during each of the phases:

  • Phase 1 – Different doses of the drug are tested for the first time in a small group of healthy people—usually less than 100.
    • What are the side effects?
    • Is this drug safe to continue testing?
  • Phase 2 – The drug is tested in a larger group of people who have the disease—ideally a few hundred (but usually fewer in rare diseases).
    • What are the side effects in this population?
    • Does the drug work as treatment for this specific disease?
  • Phase 3 – The drug is tested in a much larger group of people who have the disease—ideally several hundred to several thousand.
    • What are the side effects?
    • How well does the drug work to treat the disease?
    • How much of the drug should people take?
    • How does the drug compare to currently available treatments?
  • Phase 4 – This phase happens after the drug is approved by the FDA and is being used by patients.
    • What are the long-term effects of the drug?
    • What is the best way to use this drug as treatment?
    • Are there other risks to using this drug?
    • Are there other benefits not previously identified?

How safe are these experiments? While all medical interventions carry some amount of risk, those who participate in clinical trials are not “guinea pigs.” The FDA requires researchers to include in their protocols certain measures to ensure the study is ethical, that the rights and welfare of participants are protected, and that the risks are reasonable when compared to potential benefits. And all research organizations require any protocol that involves human subjects to be reviewed, monitored, and approved by an independent Institutional Review Board (IRB) of scientists and ethicists.

You can learn more about clinical trials at the National Institutes of Health (NIH) website.