One of the best things patient communities can do is to share experiences that aren’t showing up in medical journals, so other patients know what to expect in various situations.
There’s something I’ve been hearing from patients the last couple of years, and to a minor extent I experienced myself, which isn’t highlighted in the literature about burosumab, namely that for some patients — nowhere near all, probably not even a majority, but still a significant number — this new treatment can make them feel worse temporarily before things get better. In all but one person that I’ve spoken to, it did get better eventually, but the beginning was rough. And what made it even worse was that the patients weren’t prepared for it.
It can be a real rollercoaster of emotions, when a patient who has experienced a lifetime of health challenges without any effective relief is suddenly given a new treatment that promises to fix the underlying problem, and instead of feeling better right away, you feel worse! It’s easy to fall into negative thoughts then, to believe you were given false hope, or that you’re among the very tiny percentage of patients who don’t respond well to the treatment. Perhaps you’ve had your hopes dashed too many times before when doctors claimed to have answers but didn’t, and now you’re ready to declare everyone in the medical community to be a charlatan.
It can be really tempting to say, “never mind. I was coping before, I’ll cope again without this new treatment.”
But please wait. And know that temporary step backwards when starting burosumab seems to happen a lot. Perhaps more than is reported even among patients, definitely more than is mentioned in published literature. My experience was pretty mild — five days after my first dose of burosumab, right when my blood levels were hitting normal, I developed a major back spasm that took about a week of bed rest to resolve. I’d had that kind of spasm before, quite frequently, maybe three or four times a week, but only for a few seconds at a time, but this post-burosumab one was more severe and didn’t go away in a few seconds. Fortunately, it did improve after a week, and I was fine by the time I got my second dose, and I never again had any increased pain after subsequent doses. I did continue to have back spasms several times a week for a while, but just the kind I’d had before burosumab, not the one-time worsening of them. And then, after about 18 months, I felt significantly better in all sorts of ways, and suddenly realized that it had been several months since the last time I’d had even a small spasm. I’d gotten so used to not having them that it had taken a while for me to even notice they were gone!
I’ve heard from other patients who had other types of pain than spinal spasms after the first dose, generally widespread bone pain. In some patients, it continued for several months, so I was lucky to have it happen just once. But in all but one person, who ultimately stopped treatment after a few months, the pain did go away, and the patients felt better and were able to do more than before starting burosumab.
It actually makes sense that the first few doses — up to six months at least, maybe even a year — could make things worse. They’re definitely a shock to your system! It takes a year or more for a broken bone to fully heal, and our bones are all essentially broken, all in desperate need of phosphorus to heal, and our muscles have been starved for our whole lifetimes. So especially for patients who are adults, there’s thirty, forty, even sixty or seventy years of damage from living with insufficient phosphorus. Perhaps you had supplement treatment before burosumab, and in that case your systems were in a constant state of flux, going in and out of phosphorus-shortages. And your body got used to the feast or famine of phosphorus supplies. Didn’t like it, but accommodated it, anticipated it, established workarounds to deal with it. Then, all of a sudden, your body is flooded with phosphorus, the building block of bones and fuel for muscles. All of a sudden, your bones are drinking up plentiful phosphorus, healing old wounds, and your muscles are having a party with their new energy drink. Your whole body is on a sort of drunken phosphorus binge, and it needs time to adjust.
Basically, everything, from bones to muscles, is being rebuilt from scratch, and your body is being pulled in all sorts of directions. It’s not surprising that some patients feel worse at first. What’s surprising is that any patients are fortunate enough to avoid this feeling-worse phase!
I hope more patients will speak out on this issue and how they coped with either a temporary worsening or the impatience of waiting for noticeable improvement. There may be some reluctance to say anything less than glowing about a treatment that is life-changing and good overall, but we also need to prepare each other for the process, both good and bad. We’re strong, and we can deal with some short-term worsening in order to get to the longer-term improvement. Because there is emphatic improvement for most XLHers on burosumab, both objectively measurable and subjectively experienced. It’s hard to explain, but after a year or so, I felt stronger in my bones, like they could support me better than ever before, and I was more grounded somehow. I felt more solid, less frail, if that makes sense. Not heavier per se, just solid. Plus, we know from x-rays and other objective measurements that our bones are, indeed, stronger than ever before, which has long-term benefits that still need to be studied, but may include less calcification of soft tissue, which is a significant factor in disability.
I suspect there are things that clinicians can do to ameliorate the initial worsening in those patients who experience it, but more research needs to be done to see, first of all, how often it’s an issue, and then what can be done about it. It’s possible that older patients in particular, the ones with a lifetime of accumulated damage, especially things like spinal stenosis, or even younger patients with particularly severe symptoms, should perhaps start with a lower dosage than is standard. Or is it better to start with the standard dosage for all patients and then reduce it or space it out more than four weeks apart if the patient reports a worsening? No one knows right now, and it will likely be years before there are solid answers.
For now, it’s important that patients, especially adult patients, who are just starting burosumab be prepared with the expectation that 1) positive results can take time to be noticeable by the patient — months, maybe even a year or more, not days or weeks, but 2) temporary adverse results can appear within a few days and repeat with each of several doses until the body adjusts to this huge change in its metabolism.
We’ve learned to cope with a variety of challenges, and we can cope with this one too, as long as we know it’s a possibility. For me, it was a shock to feel worse when I thought I’d feel better, and that disappointment was almost worse than the spasms themselves. I got over it and am glad I did, since I feel so much better now than I did five years ago when I started on burosumab.
The key is to establish reasonable expectations. If you expect to feel better and immediately feel worse, it’s a huge disappointment, made worse perhaps by feeling betrayed, possibly after a lifetime of other medical betrayals. On the other hand, if you expect to experience some worsening, and you don’t, woohoo! And if you do have some worsening, it’s not as big a deal if you’re prepared for it, and you can cope with it on the way to getting to the time when you’ll feel better. And in my experience, that day will come if you can just be patient while your body adjusts.
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Please note that the author of this blog is a well-read patient, not a doctor, and is not offering medical or legal advice.
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