Established Leukodystrophy Patients
How do I tell my friends, family and other children about leukodystrophy?
Caring for a loved with leukodystrophy will bring all sorts of expenses, how can we pay for the things we might need?
Why do I need to keep going to the hospital if there is no cure or treatment?
What do I do when I go to the ER?
When should I get in-home nursing care?
What if my loved one needs a feeding tube?
Why should I use hospice?
Should I send my child to school?
Why is newborn screening such an important issue for leukodystrophy families?
Is cannabis oil a useful treatment for managing leukodystrophy?
What is “Lorenzo’s Oil”?
What is respite care?
My hospital social worker told me to enroll for Medicaid, I have health insurance from my job: why do I need Medicaid?
I am so busy caring for my child with leukodystrophy, I worry that my unaffected children are getting neglected.
How do I tell my friends, family and other children about leukodystrophy?
It is emotionally draining and overwhelming to try to explain what leukodystrophy means to family and friends. It might be especially difficult to talk to young children. Recommending a book to family members, co-workers, and teachers can help them learn about the disease without you experiencing the trauma of discussing the diagnosis repeatedly. The Calliope Joy Foundation published a book titled “Loie’s Disease: A Book About Leukodystrophy.” You can order “Loie’s Disease” on-line at this link.
We also recommend Jill Kelly’s New York Times bestseller Without a Word: How a Boy’s Unspoken Love Changed Everything. This book chronicles Hall of Fame Quarterback Jim Kelly and his wife Jill’s life caring for their son, Hunter, who was diagnosed with globoid cell leukodystrophy.
One parent wrote a letter to email friends and family. The letter was a way for the parent to learn more about the diagnosis and share information with their network of friends and family and give them concrete suggestions about what the family needed. It can also be helpful to create a Caring Bridge or Facebook page (or have a close friend do so) to share information and update friends and family. This way, friends and family can check-in and not overwhelm you.
Caring for a loved with leukodystrophy will bring all sorts of expenses, how can we pay for the things we might need?
Many families find that fundraising to help with the challenges of care gives friends and family a way to help during uncertain times. Thanks to social media, there are many sites to choose from: Crowdrise.com, Gofundme.com, HelpHopeLive.org are just a few examples. If you are member of a faith community, your pastor, rabbi, or priest can also quietly reach out to donors who could help.
Keep in mind that raising money is much tougher than it looks, and it is good to create one fund so you don’t overwhelm friends and family resulting in “donor fatigue.”
It is also really important to make the first gift yourself. If you need $5000 to remodel a bathroom, the first donation to the fund should be from your own family. Also, be as clear and transparent about what the money will be used for. Less successful campaigns request funds for some vague and never-stated purpose. Sadly, we live in world where people commit fraud and exploit tragedy, so it is important to be responsible about the gifts well-meaning people provide. When one family lost power in their home and found themselves homeless with their sick child, a group of friends and neighbors raised $10,000 to pay for a generator so the family could shelter in-place and not worry about losing power to run medical equipment in the future. The very real need for generator and a real-life crisis mobilized the community.
You can check out this useful piece from Money on how to raise funds medical expenses.
Why do I need to keep going to the hospital if there is no cure or treatment?
It is important to follow up with your medical team. Like many diseases of the brain and central nervous system, leukodystrophy is an “all-hands on deck effort.” If your loved one seems to be having trouble holding down food, breathing, or you can’t manage symptoms on your own and your home based medical team has run out of ideas, ask your pediatrician or doctor to consult with a specialist in leukodystrophy. A doctor knowledgeable about leukodystrophies can offer guidance and support to your team. Ideally, a neurologist familiar with leukodystrophies should manage your loved one’s care and coordinate with specialists in pulmonary medicine, rehabilitation medicine, complex care medicine, gastrointestinal medicine, and palliative care. If you cannot travel to a hospital with specialized care for your loved one, try to have your home-based medical team seek advice and guidance from a specialist knowledgeable about leukodystrophies.
What do I do when I go to the ER or have to be admitted to the hospital?
A visit to the ER/in-patient is always stressful, but when caring for a loved one with leukodystrophy, the experience can be terrifying. Here are some pieces of advice that will help you in preparing for such visits.
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Have a go-bag with a list of medications and formula, diapers, doctors’ names and phone numbers, change of clothes, and a blanket so you don’t have to assemble these items in a rush to the hospital.
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Stay calm, it is stressful and emotional to be at the hospital, but you need to listen and pay attention and work with the doctors and nurses and help them care for your loved one. Losing your temper makes it harder for you to get the care you need.
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Create a plan for cold and flu season or recurrent problems like urinary tract infections so you can help guide your team at the hospital.
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With your medical team, review the situations that require hospitalizations (respiratory distress, dehydration, high fever that fails to respond to over the counter medications etc.) You should not be at home wondering when you have to go to the hospital. But if you are in doubt, call and seek guidance.
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Be sure your local hospital’s team has an updated medical history and a care plan for common, recurring problems prepared by specialists. If your loved one has a DNR (or doesn’t) be sure to have a plan in place on how much treatment you would like before an emergency arises.
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Sometimes a doctor familiar with your loved one’s case can meet you at the ER and help work with your family and guide your medical team. If your specialist is not at your local hospital, be sure to have phone numbers with you so your medical team can call your specialists and seek guidance to treat your loved one.
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Don’t doubt yourself. As a patient advocate, you are the expert in your loved one’s care so you should work with the team and offer your knowledge and expertise. Don’t be shy about asking questions and seeking explanations.
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The ICU is not a bad thing. When you hear you are headed to the ICU you might be frightened. However, in the ICU nurses and doctors have fewer patients under their care so patients receive high quality and specialized care. As soon as your loved one is stabilized, you will be moved back to a regular floor.
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It might be useful to have photos and video of your loved one when they are “okay” so that the medical team has a clear sense of what normal looks like.
When should I get in-home nursing care?
Home based nursing care is provided to many leukodystrophy patients through Medicaid insurance programs. The way such programs work is that a company (such as Bayada) will seek to find nurses and aides to work with your families. A doctor’s letter of necessity will help the insurer determine how many hours of skilled nursing and aides your loved one requires. Nurses and aides are trained and overseen by the agency, but then come in to work in your home with you and your loved one. Finding a nurse to provide care for your family member takes time. But, it is important to take advantage of the help if you can. No matter how devoted you are to caring for your loved one, care-giving should not be the only thing you do. You may have a full-time job, and it is also important to spend time with friends and family or even take a vacation. In time, your nursing care team will be an integral part of your life. When nursing care works, it can make all the difference. Many leukodystrophy families come view their nurses and aides as members of their families.
You can watch this video to get a sense of how skilled nursing works with the Carr family.
What if my loved one needs a feeding tube?
There is a great deal of anxiety over feeding tubes. Some devices require surgery and others do not. It is quite difficult to come to a point in your loved one’s care when you can’t rely on feeding them by mouth. That said, a significant number of leukodystrophy patients use feeding tubes and they are a recommended (prophylactic) feature of care. While some families worry the feeding tube may diminish the quality of life, most families find it a very useful tool in providing the best care. The use of a feeding tube can relieve a great deal of stress and anxiety since A feeding tube can reduce the worry about whether a loved one gets enough nutrition and relieve concerns about choking. In fact, a feeding tube can save you visits to the hospital because a feeding tube can manage the risk of dehydration and guarantee that a patient can receive medications. Once your loved one receives a feeding tube, take the time to work with a gastrointestinal (GI) specialist and a nutritionist experienced with leukodystrophy so that your feeding plan can be tailored to the needs of your loved one and your family.
Why should I use hospice or palliative care?
There is a great deal of fear and misunderstanding about hospice. Hospice care for pediatric patients is different than adult hospice. Hospice is best understood as a program that provides care, medical supplies and support to families caring for a loved one with a life-limiting condition. Many leukodystrophy families receive hospice care for years, and hospice nursing provides medicines, medical equipment, home-visits, respite care, and family support with social workers, child life specialists, and art and music therapy. Hospice programs vary from region to region. And the recommendation of hospice for leukodystrophy patients does not necessarily mean death is imminent.
Reframing Hope: Pediatric Palliative Care
Watch the video “Reframing Hope” to learn more about pediatric palliative care.
Should I send my child to school?
As the parent of a child with leukodystrophy, it is your right to send your child to school. And many families find the rhythms of taking the bus in the morning and interacting with classmates and teachers a very positive experience. However, for many children with leukodystrophy, school puts great strain on parents and care teams managing children with complex medical needs. Because many children with leukodystrophy suffer from paralysis, seizures, respiratory problems, and are nonverbal, the decision to send a child to school is one that should be taken with care and input from a medical team. Attending school elevates the risk of infection that can lead to hospitalizations. In the end, you will know what is best for your family and your child. Seek the advice of experts and advocates to determine the path that is right for your family.
Why is newborn screening such an important issue for leukodystrophy families?
Newborn screening for leukodystrophy is a very important issue in the leukodystrophy community. And many parent advocates work passionately to lobby elected officials for newborn screening. All babies are given heel prick test in the hospital, and the blood samples are then analyzed and tested for several dozen disorders. States and countries vary on the number of disorders screened for at birth. At present, in the US, only one form of leukodystrophy is part of the Recommended Uniform Screening Panel (or RUSP) and that is ALD (or adrenoeleukodystrophy). In the US, seven states currently screen for Krabbe disease or globoid cell leukodystrophy, however, Krabbe disease is not part of the federal RUSP newborn disorders. Newborn screening is crucial for leukodystrophy since promising therapies (namely bone marrow transplants, gene therapies and other treatments) are most effective before the children get sick, when children are presymptomatic. No treatment exists to reverse the damage to the brain’s white matter, but new therapies could prevent the disease from damaging the brain. Newborn screening is a hotly debated issue because states and governments must fund and implement testing and there are serious challenges in creating an effective (and cost-effective) heel prick test that could be administered to millions of infants born annually. Until recently, leukodystrophies were not considered diseases that would be served through newborn screening. The good news is that recent breakthroughs mean there is new hope for children caught before symptoms develop and we hope to see more leukodystrophies included in newborn screening in the US and abroad.
Is cannabis oil a useful treatment for managing leukodystrophy?
Despite the national attention surrounding medical marijuana and cannabis oil, there is still a great deal to learn about how to use such treatments. While there is some sound medical research to suggest that medical marijuana might be useful in treating some forms of epilepsy, there is no reviewed medical research showing that leukodystrophy patients respond to such treatments. Clinical trials are working to assess the effectiveness of cannabis oil for a variety of neurological disorders. Be assured, we here at the Leukodystrophy Family Forum will be on the watch for new findings. The other concern doctors express is that since the medical marijuana industry is not well regulated, the chemical content varies across different brands and manufacturers. Doctors are trying to learn how cannabis oil interacts with other drugs that leukodystrophy patients use. So, while some families have become staunch advocates for medical marijuana and there is growing interest from the Child Neurology Foundation and other medical professionals, at this time, we do not have research determining if such treatments benefit patients with leukodystrophy.
What is “Lorenzo’s Oil”?
In the film 1992 Lorenzo’s Oil starring Nick Nolte and Susan Sarandon, Augosto Odone, played by Nolte, invents an oil to treat adrenoleukodystrophy. The Odones were remarkable parent advocates and the legacy of that work lives on today through the efforts of their foundation, The Myelin Project. However, Lorenzo’s Oil is not a cure for leukodystrophy. The oil, if started early in boys with adrenoleukodystrophy (ALD) showing no symptoms might have some benefit in preventing the childhood cerebral form of adrenoleukodystrophy by normalizing the levels certain chemicals called very long-chain fatty acids. Lorenzo’s Oil might help prevent some of this build-up. The build-up of these fatty acids is thought to cause many serious problems throughout the brain and body. Lorenzo’s Oil is specific to ALD, does not repair myelin, and does not have any known effect on other demyelinating disorders, such as multiple sclerosis or any other forms of leukodystrophy.
What is respite care?
No matter how devoted you are to the care of your loved one, this cannot be the only thing you do. A caregiver who takes time for him or herself is not being selfish, it is crucial for the well-being of the entire family for the primary caregiver to take time to do other things that bring him or her pleasure.
Respite care provides short-term relief for primary caregivers. This is different from the nursing care provided by Medicaid to make it possible for a family member to work and support a family Respite care is for a caregiver to take time for him or herself to go on a trip, workout at the gym, or even just take a walk. It can be arranged for just an afternoon or for several days or weeks. Care can be provided at home, in a healthcare facility, or a day care center.
Respite services charge by the hour or by the number of days or weeks that services are provided. Most insurance plans do not cover these costs. You must pay all costs not covered by insurance or other funding sources. Respite care varies by states, but some states will cover most of the cost of up to 5 days in a row of respite care in a hospital or skilled nursing facility for a person receiving hospice care. Medicaid also may offer assistance.
My hospital social worker told me to enroll for Medicaid, I have health insurance from my job: why do I need Medicaid?
While Medicaid does offer health insurance to low-income families, it also provides supplementary health insurance to cover the costs of caring for special needs and medically complex children. A leukodystrophy diagnosis will often make your child eligible for Medicaid. Medicaid serves as a supplementary insurance to pick up the costs of care that even very good, private insurers will not cover (EXPENSES such as medical equipment, adaptive strollers, skilled nursing and aides etc.). Medicaid varies greatly by states (and is often described as essentially 50 different insurance companies operating in the 50 states). Some states offer more expansive and generous benefits, while others are more difficult to work with and require hiring an attorney or benefits specialists.
As you navigate the Medicaid journey it can be useful to work with a complex care pediatrician or primary care pediatrician who works with medically complex children to help file the proper paperwork. To learn more about advocating for your child and Medicaid, you might find it useful to go to Family Voices and learn more about resources in your state. Be prepared to be diligent, applying for Medicaid is not going to be easy.
Since each state has different policies and procedures for applying to Medicaid, check out the website Kid Waivers to learn about the guidelines for your home state so you can get started.
States purposely make it challenging to enroll in Medicaid as a policy to discourage applications and save money. It may take months to get enrolled. Some families may even consider moving to a different state to get better coverage, this is not uncommon. You can learn more about Medicaid by watching this video produced by Vox tilted "Why it's worse to be sick in certain states?"
It is also true a leukodystrophy diagnosis may make you eligible for SSI (or Social Security), however, your household family income would have to meet a certain level. Again, you should work with your state officials (welfare office) doctors, and case workers to determine your eligibility.
Many leukodystrophy families, of all political persuasions, have been engaged in the public debates about ACA (Obamacare) and Medicaid. This essay explains why: "Why I'm Inviting Members of Congress to Meet My Daughter." If you want to work with other parent advocates committed to supporting care for medically complex kids, check out the work of the Little Lobbyists, a bipartisan group of families who work to champion for health care for special needs and medically complex children.
I am so busy caring for my child with leukodystrophy, I worry that my unaffected children are getting neglected.
The reality of caring for a child with leukodystrophy means that it is inevitable your time and attention will go to your medically complex child over your healthy, unaffected children. Frequent hospitalizations and the demands of your loved one's medical needs may mean that vacations, sporting events, school activities, and even a family night out for dinner become difficult if not impossible to do. It is comforting to imagine that our "superhero" siblings sacrifice their childhoods to these circumstances without complaint or harm. However, it is important to remember some key issues when considering the health and well-being of the “unaffected” siblings.
Feeling like they need to be perfect. Siblings of individuals with special needs know how hard their families work to ensure all of their sibling’s needs are met, and often see their parents struggle to meet these needs. Many siblings feel like they can’t make mistakes because that would add to their parents’ burden, so they believe they must be perfect at all times.
Feeling like they can’t express their feelings. Most typically developing children love their sibling with special needs. Yet they may also resent how much of their parents’ time is taken up by caring for their sibling or feel embarrassed about their siblings’ behavior or appearance. They feel tremendous shame and guilt over such feelings.
Having a different idea of family and home. For most children the concept of family is based in togetherness. But when a sibling has special needs, family quality time may look different. In some cases, the presence of caregivers and therapists may redefine what a family home is for children. The presence of nurses and aides and medical equipment makes some siblings feel that places outside the home, such as school or a relative’s house, are more of a refuge for them than their home.
Feeling as though their problems are minimized. Sometimes a sibling with special needs has complex and even life-threatening problems. An issue faced by a typically developing sibling, whether it is a problem with a friend or an academic struggle, may seem small compared to having limited mobility, learning difficulties or sensory issues that require intensive care or prevent a child from attending the neighborhood school.
Feeling isolated. Typically developing siblings may be lonely because they don’t have peers who have siblings with special needs. So they feel different when their friends ask “what’s wrong with your sister?” Some children also feel self-conscious about their sibling with special needs, and aren’t sure when or how to tell their friends about him. Others feel uncomfortable inviting friends over because they are unsure of how their friend or sibling will react.
Dealing with intolerance early and often. Children learn early that there is not universal acceptance for individuals with special needs, and that their sibling is not welcome everywhere that typically developing children are. This can be deeply disappointing to typically developing children who want to have shared experiences with their sibling. They regularly encounter individuals who refuse to move from seats designated for individuals with disabilities, and those who make unkind comments about other accommodations their sister needs. These early lessons in intolerance, and even hate, can affect their worldview and make them cynical or resentful of the limitations placed on their sibling and themselves as a result.
Feeling like they are asked to help too much. Some typically developing children are expected to help care for their sibling with special needs from a young age, even if that sibling is older. One girl said that she felt like the “attention police” at home since her mother was constantly telling her that she had to pay attention to her sibling with special needs. Others are expected to push wheelchairs, participate in therapy sessions, or attend to their sibling’s personal care needs by feeding them or helping to get them dressed. Many are told early on that they will be expected to care for their sibling when their parents are no longer able to do so. This puts enormous pressure on them.
Feeling like they must grow up quickly. Because of the sum of their experiences, from feeling as though they are on their own to handle their problems to feeling pressure to be perfect to being given responsibility for their brother or sister, some siblings of children with special needs feel as though they are forced to grow up too quickly.
Most typically developing children love their siblings with special needs beyond measure and are close to them. To better understand and support them, it’s important to acknowledge the struggles of typically developing siblings. One mother explained, “After six years of living with leukodystrophy, I realized I needed to acknowledge to my older children that I was not the parent they deserved. I apologized for not being as engaged in their lives as I should have been or wanted to be. I think it was important to let them know I had missed out on their lives too, and that we could all be angry about the situation, and even be angry with me, but I did not want them blame their sister.”
There is a need for more information about the experience of growing up with a sibling with special needs. We here at the Leukodystrophy Family Forum are working on a new children’s book to explore the feelings of siblings. Many hospitals now run support groups for siblings of children with complex medical needs.
Read Jamie Davis Smith’s Washington Post piece here.
Read Perri Klass’ NY Times essay here.
Read Norman Berlinger’s NY Times Magazine story here.
You can watch this video produced by the Courageous Family Network on the perspective of being a sibling to a child with leukodystrophy here.
