I’ve been thinking about a couple of related topics lately, without coming to any sort of solid conclusions or action plan, so today I’m throwing them out for your consideration.
The first topic is the how chronic disorders (with no cure) are different from acute ones: there are just so many opportunities (not all opportunities are good) to not get adequate treatment. The big ones are: 1. diagnosis delays (and related lack of access to a qualified clinician), 2. the transition from pediatric to adult treatment, and 3. the transition from adult treatment to retirement-age treatment. (Many thanks to Joyce Inman for pointing out to me that the challenges faced by senior patients coalesce into a second transition period, different but as life-changing as the transition from child to adult.) And that’s all without even considering the smaller opportunities to fall off treatment—things like changing insurance plans (or going from Medicaid to private insurance or private insurance to Medicare), political changes (access to insurance generally, or the end of telehealth for Medicare patients), life changes (moving from one state to another, or getting married/divorced), career changes (getting/changing jobs, becoming unemployed, retiring), health care provider changes (their retirement or changing jobs), and so on.
The barriers I’ve listed are all fairly large, and can require a significant effort for the patient to return to a new normal for treatment. They’re also barriers that we have little to no control over, so at least in the immediate aftermath of bumping into one of them—a new insurer denies coverage, an experienced clinician retires without anyone taking over their XLH patients’ care, or the patient moves to a new state where there is no known qualified clinician—we can end up feeling totally out of control of our lives. Oftentimes when we run into one of these barriers, there’s other stress in our lives too, which leaves us susceptible to thinking, “The hell with it, I’m done. Might as well just stop treatment.”
Not all of the barriers are that large. The big things happen infrequently, but there are constant stupid little things. Like the person who does the monthly injections calls in sick the day of the injection, and there’s no one else who’s qualified to do it, and the next available date is a week away, and you’re already feeling the pain and fatigue that come with being at the end of the phosphorus cycle. Or the pharmacist forgot to order the dose, and it’ll be a week before a new appointment can be scheduled. Or your usual date for the injection is a holiday, and there’s no one available the day before or after. Normally, we just suck it up, rant to our friends, and live in pain and exhaustion for that week. But if it’s the final straw in a bad few days (or we’re out of spoons, as that theory goes), it’s so easy to do the nose-spiting cutting thing and think, “Eff it, if the universe doesn’t want me to stay on treatment, then I might as well just give up and stop struggling.”
Anyway, the first issue I’m looking at is just how often it is that we’re confronted with having to make the decision to stay on treatment. It’s not like, say, a broken leg, where you know that the cast will come off in a month, or even some kind of radiation or antibiotics, where you know that after a certain number of doses, it will be done, so all you have to do is grit your teeth and get through that month of hobbling or the specified number of doses, and you won’t have to deal with treatment any longer. With a chronic disorder, though, you’re dealing with the treatment FOREVER. And we can’t ever entirely go on automatic pilot. Thinking about it—did I schedule the appointment or the dose delivery, is my clinician going to retire, is this the month my insurer is going to decide I don’t really need treatment?—is exhausting. And if we give in to the exhaustion and the frustration, go on hiatus for a bit, we’re viewed as bad patients who are non-compliant. Which, for someone like me, who tends to take that kind of criticism as a dare, just makes us LESS likely to make the effort to continue the treatment!
Okay, so I’m wandering off topic, even more than I’d planned. I did tell you I’m thinking out loud here, without any clear message. So let’s move on to the second, related issue. Which is that XLH doesn’t have just two basic manifestations (pediatric and adult), but three (pediatric, adult, and senior), and the transition from adult to senior is yet another opportunity to fall off treatment.
I’ve long advocated for better understanding of the adult manifestations of XLH (it was only in 2019, when the Burden of Disease Study was published by Ultragenyx, when anyone other than the handful of top experts started to realize there were serious lifelong symptoms), and I think we’ve made significant progress there. But I’m beginning to think that there’s still a sub group of adults (age 60 or 65 and up) whose experiences are as much different from the middle group (around age 30 to 60) as the middle group is from the pediatric-to-young-adult group, and it’s seriously misunderstood both by us patients and by clinicians.
I’m not sure even I understood how different my experiences are as a senior until recently, and I’ve been in that category for a while now! I only started to pay attention after my recent comments on the ninth anniversary of burosumab treatment generated more feedback than I’m used to, and all from patients in the 65+ demographic.
One bit of feedback (quoted with permission) is from Sue:
I too am an oldie, 68 yrs and on Crysvita only one year. Our experiences are remarkably parallel, what with symptoms and trying to know which are related to xlh and which are simply old age.
Interesting to read of how you noticed the pain when it came roaring back after a delayed dose. WOW, I LIVED WITH THIS FOR DECADES????? Yup, I get it.
And then there’s Judy (also quoted with permission), who told me:
I am 73 years old. I started on burosumab in 2018. My phosphorus level is consistent at 2.1. Better than 1.9 or lower. … My other issue is RLS. Not much helps with that. I still get bone pain. I think the shots keep me walking.
I am one of 7 siblings out of 9 who were diagnosed with “inherited rickets” at a very young age. My mom had the disease and was actually experimented on at a young age, including surgeries in wrong places. … We went through so much for this. I was happy to get the Crysvita shots. I deal with the side effects.
I’ve also talked to other senior patients frustrated with the changes that retirement brings to their health care. I mean, if you think it’s endemic that we’re not listened to when we’re in our thirties, forties, and fifties, try getting health care providers to listen when they’re mostly focused on testing you for memory issues and insisting you need a Dexa scan that they’re not qualified to interpret! And then there’s the switch from private insurance to Medicare, which can bring a change in providers and different rules and regulations for attaining that care (who can do self-injection and who can access telehealth). There’s the progressive nature of XLH, which means that the mobility challenges of senior XLHers may be accelerating, but it’s hard to tell how much is age and how much is XLH, and the tendency to think it’s too late for any treatment. There are also mental/emotional issues around whether, as a retired person who isn’t contributing to society (the conflation of self-worth and career productivity is so harmful), we deserve to get treatment, especially given its cost. Plus, there are at least some folks who think treatment in senior adulthood isn’t medically necessary (as in Italy, where the health service won’t pay for burosumab once patients turn sixty-five).
As noted, I’m not really sure where I’m going with these thoughts, so I’m throwing it out to you. First, what are the “opportunities” for dropping off of treatment that you’ve encountered, and how did you get past them (if you did)? Second, what challenges to staying on treatment have you encountered (or seen in family members) upon reaching retirement age (65-ish)? I’m especially interested in any challenges that senior patients experience that are somehow different from the challenges of the middle age group (30 to 60). I’m hoping that hearing from you will help to focus my thoughts and perhaps lead to some sort of useful resource (like the Weak Bones, Strong Wills book, but focused exclusively on senior patients) for others who are making the second transition for XLHers (and probably all chronic disorder patients), the one from adulthood to senior adulthood.
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Please note that the author is a well-read patient, not a doctor, and is not offering medical or legal advice.
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