• Pediatric programs may offer easier access to multidisciplinary specialist care for young adults with spinal muscular atrophy (SMA).
• Transitioning to an adult care setting may provide support around adult issues like work and relationships.
• Comfort, rapport with a familiar provider, and medical fragility or complexity can influence the decision to stay in pediatric care or transition to an adult setting.

Adolescents with SMA will face a decision about whether to remain in the care of their pediatric neurologist or transition to a specialist in adult neuromuscular disorders. New medications such as Zolgensma (onasemnogene abeparvovec-xioi), Spinraza (nusinersen), and Evrysdi (risdiplam) are expected to improve longevity. More children with SMA could reach adulthood and still require continuing specialist care and treatment.

Dr. Jonathan Strober spoke with mySMAteam about the challenges and benefits of the transition to an adult care practice. Dr. Strober is a specialist in childhood disorders of the nerves and muscles. He directs the Neuromuscular Clinic at UCSF Benioff Children’s Hospitals.

What’s the biggest issue facing older teens or young adults with SMA who might want to transition to adult-centered care?

Ease of care is the biggest issue for a lot of my patients. There are very few programs in the “adult world,” as I call it, that are like the pediatric programs. Even in big institutions like ours, the adult side is all spread out, whereas in pediatrics there are multidisciplinary clinics with a neuromuscular clinic that has a physical therapist, an occupational therapist, a respiratory therapist, a social worker, nurse case manager, sometimes a cardiologist, pulmonologist, gastroenterologist, endocrinologist, and genetic counselors. We’re all there together on specific days.

People would rather come on one day every so often, especially when you have lots of needs and equipment you have to pack up, and travel is difficult. People drive hours to their clinics. If you receive care at a multidisciplinary clinic, you don’t have to miss work or school to see five different people on five different days, every so often. That’s a lot to ask of somebody.

Are there any multidisciplinary clinic programs being organized to treat adults with SMA?

We’re discussing it at our institution. We — the neurologists — all want to do transitional care. The problem is administrative. You have to find a pulmonologist, cardiologist, and other specialists who have an interest in neuromuscular disease and who are willing to devote the time to a clinic schedule. For our pediatric program, I’ve slowly been adding specialists over the years.

Are some patients more comfortable staying under the care of their pediatric specialist?

Sure, there’s a comfort level. I know your medical history. I know who you are. Sometimes the patient doesn’t want to leave because they’re comfortable with the care, apart from the idea of having to move and coordinate the care for themselves. And the pediatric specialist may have trouble giving up that patient. I’ve cared for these people for 20-plus years.

Adult specialists can also have a different personality than people who take care of kids. During my neurology training, one of my attendings once said, “The patients really like you. What’s your secret?” I said, “I just treat everyone like a kid.” We’re used to doing that in pediatrics.

When do you start talking to your patients about taking more responsibility for their care decisions?

We start when we hit the teenage years. We start to talk about treatments and medicines. Do you know what your medications are and when you take them? Do you know what your doses are? Do you know what the potential side effects are and when to let somebody know if you experience any? How often are you using your cough assist? What are your equipment needs? How do you communicate your needs and advocate for yourself?

At what age does the question of transition from pediatric care arise?

I definitely think it starts earlier than 21. I knew when I was a senior in high school that I was ready to leave my pediatrician. I wanted to feel like an adult with my provider and not feel like a child anymore.

We try to start those talks about responsibility for care when they’re teenagers, so they feel comfortable talking about transition later. Sometimes we say, “Let’s graduate college and then we’ll talk.” Or sometimes they’re moving somewhere else to go to college, and that’s a natural transition to a specialist in adult care. Hopefully by that point, they’re feeling comfortable enough that they can go see the next physician and just settle in.

How do you handle transition when a patient requests a referral to a specialist in adult care?

I want to feel certain they’re going to get good care when they transition. I’m on a transitions task force, and we’re trying to figure out how best we can do it. Right now, I have a conversation with the patient and recommend a specialist. Then I’ll contact that specialist, and they’ll agree to take on the patient. We are hoping to have the specialist on the adult side come to the clinic with me to meet the patient, and then I will go to the adult clinic with them the first time. That way there’s a familiar face, and they can slowly become comfortable.

What are the potential downsides for an adult with SMA who remains in the care of their pediatric specialist?

I am less comfortable talking about sex and sexual relationships, adult concerns about life once they graduate from college and get a job. How are you going to make money? Find a partner? What is that adult life going to look like in somebody who has SMA? Those are all difficult questions that physicians who care for adults handle all the time. There are pediatricians who are very good at it. I’m trying to get better about it.

What’s the benefit in the transition to adult care?

I think the benefit is feeling like an adult. You want to get a job. You want to have a home, maybe you want to have a family. If you’re stuck in the world of pediatric care, I think it’s a harder perspective to come from. Being in the adult care world, you may feel more like the adult life you want is possible.

When might it be most beneficial to stay in pediatric care?

An adult care practice may not be for everybody. It’s not uncommon, especially for patients who are a little bit more fragile, or whose care is a little bit more nuanced, and we’ve been dealing with this together forever. Fragility is a big deciding factor. You have to decide what’s right for you. If your pediatric specialist is willing to keep you, and you have a good relationship, and you don’t mind being the adult in a pediatric world, then great. I’m not pushing you out the door. That's what I tell my patients.

How do you see your role as your patients reach adulthood?

It’s my job as a physician to help people along their journey. It’s about their journey, and what they want. What’s the point of all of the treatment and care, if you’re not going to enjoy your life and live the life that you want?