Sandra

Age at interview: 46

Brief Outline:

One year after her daughter's diagnosis, Sandra was invited to enrol her daughter in a ten year trial to follow her daughter's progress. Sandra believes her daughter is still in the trial, although she was unsure.

Background:

Sandra aged 46 years is a full time housewife. She has five children. One of her daughters was diagnosed with juvenile dermatomyositis, a rare condition that affects children under the age of 18 years.

More about me...

 

Sandra aged 46 years is a full time housewife. She has five children. One of her daughters was diagnosed with juvenile dermatomyositis at the age of four years; she is now nine years of age. Juvenile dermatomyositis (JDM) is a rare condition that affects children under the age of 18 years and affects approximately two children per million.   It is an autoimmune disease where the body’s own immune system attacks the body, skin, muscles, and internal organs and the cause of JDM is unknown.

 

When her daughter was aged five years Sandra was invited to enrol her in a trial. Sandra was informed the trial was for ten years and would involve following her daughter’s progress and keeping medical records to help get a better understanding of the condition and its prognosis. They would take blood tests and completing a Childhood Myositis Assessment Scale (CMAS) to measure physical function, muscle strength and endurance, and disease activity. Although at the time of being invited she was given plenty of information to read, it was quite a lot to take in. However, she was reassured that her daughter’s information would be kept confidential and was happy to take part if it could help her daughter and provides information to help understand the condition better; thus helping other children in the future.

 

Sandra believes her daughter is still in the trial, although she was unsure.  She says she would like some more feedback and a bit more information about the progress of the trial. However, her daughter is doing well and she is pleased with her daughter’s treatment and taking part in the trial.

 

Sandra hadn't come across clinical trials or medical research before, but she wanted to take part in the study to help advance knowledge about juvenile dermatomyositis and would consider other research and clinical trials in the future.

If it had been a change of, a different treatment, or, wanting to try a different treatment for her, would you still have been quite as keen?

 

Yes, I think so. I totally trusted the doctors and, you know I believed that they knew what was best for my daughter and they were all for getting her well. So if they’d, you know, recommended, I mean she did end up having another treatment as well, which was, I’m not sure, immunoglobulins or something or other, which was a pretty new treatment, tested on children with JDM (juvenile dermatomyositis). She had that as well. And then she also had another treatment, which was for side effects of the steroids, because it started to affect her bones, thinning bones. So she had Promiginate, which they give to old ladies for osteoporosis. But I guess I just trusted them and let them give what I thought was best for [daughter], you know. Because they’re the best people to help, aren’t they?

 

I mean would you, you know, did you ever think, “Well, actually I don’t want to do it” at all?

 

What, the trial? No, no, never, no. It was just a instinct, gut reaction, “Yes, I’m happy for that.” Because I knew that no bad would come of it, you know. It was all confidential. And I thought even some good may, may come of it. There’s a good chance that some good may come of it. Because in the medical profession, and science and stuff like that, they’re learning things every day, aren’t they? So they’re bound to learn something from those, these results, bound to. And to me that’s good, you know. Anything they can learn about JDM is brilliant, you know, because it’s going to help kids in the future who get it.

 

Do you, do you support clinical trials in general?

 

It’s not something that I’ve ever thought about, clinical trials, at all. Because I’ve got five children, [daughter] is my fourth, and none of my other kids have ever been ill or anything like that. And, you know, I’m just a housewife. So clinical trials had not even entered my radar, you know, until my daughter.

 

What do you know about clinical, if you were to explain it, how would you explain your understanding of clinical trials, as that was your first experience?

 

I don’t, don’t really know. A lab, a whole load of blood results, a whole load of CMAS scores. And, you know, I really don’t know. I don’t have a clue. I don’t understand how it works. I, I didn’t even think of that. I just thought if it can help, then that’s it.

 

Juvenile dermatomyositis is a rare condition and the researchers need every child with the condition on board. Sandra was happy for her daughter to join the trial to help the researchers and help her daughter get better.

 

I was approached because my daughter was such a severe case of juvenile dermatomyositis. And basically her CMAS (Childhood Myositis Assessment Scale) scores, which is a thing they do to test muscle strength, all sorts of different, you know, counting how long she could hold her head up and how long she could lift her arms up in the air, her feet off the floor and stuff like that, go on all fours and actually get down to the floor, pick up a, and get back up without touching anything, you know. And the score is up to 54. That’s the highest you could get. When [daughter] was recovering, and this was when she was recovering, she got a score of 4 out of 54. So she was a very bad case. So the CMAS score is part of the trial, to try and understand the different severity of juvenile dermatomyositis. And also the bloods as well to, to record, you know, like all the different levels, how it affects, you know, the inflammation in the body. So all that’s shown in the bloods. So they take the bloods and they’re sent to the study, but with no names. Everything is kept private.

 

So the trial that you were invited to, tell me about, about that, what, what the trial is and what, what the purpose is.

 

It’s the JDM (juvenile dermatomyositis) study and it’s basically to get records of children. Because it’s so rare they need really every child on board so that they can get a better understanding of the condition, because it’s so rare. So they like, you know, they want to do the, the muscle test and the blood test because they’re the two most important things with JDM. That’s the, obviously the abbreviation for it. I can actually find it.

 

They asked me, you know, would I be happy for them to use my daughter’s information? It would all be confidential. And I said, “Yes, fine” because anything that helps her. Because it’s only in recent years, if she had have been born a decade, a decade earlier, then I don’t think she’d have been here because, and it’s only, a lot of children died from it, and still do. Because obviously once it, it, depending on what, how it affects the body, if it attacks the heart and the lungs, then, you know, and also the swallow, because she started to choke and, you know, it can go into the lungs instead. And, you know, it’s a horrible, horrible nasty condition. It really is. It’s awful.

 

Sandra recalls being given lots of information to take home and read and had time to make a decision. She says the hospital were 'fantastic' and she was reassured that everything would be kept confidential.

 

They did give me some written information but I can’t remember exactly what it entailed. At the time it was, it was obviously a difficult time. But even when she was allowed home it was still, you know, I still had a disabled child to look after. Do you know what I mean? I also had my son as well, who was badly affected by what happened to my daughter, and still is now. He suffers from selective mutism and with, and separation anxiety he did at that time. And we think that all kind of stems around what happened to her. So it was a very difficult time. So I can’t remember exactly. But they did give me some information and basically explained it was to do with the, the blood tests and the CMAS score, what they call it.

 

Did you have to sign anything there and then or could you go away and read it?

 

I think I could go away and read it, and I think I did take the information home with me. But I was quite happy to sign it. Do you know what I mean? The hospital are absolutely fantastic and they told me it was all confidential. So I just, I just signed it basically. They just wanted to do it to help other children, and to me that’s a good thing because it’s such a bad illness.

 

I was going to say, what, what made you sort of decide to do it really?

 

To help future children with JDM, you know, they need all the help they can get. They really do. Even with all the expertise of the doctors, sometimes it’s not enough. So it’s a good thing. Anything they find out that can help, you know, is massive as far as I’m concerned. It’s such a good thing. So any way I could help, that’s, you know, what I wanted to do. And I’m sure any parent who’s had a child with JDM would do the same thing, you know. Because, as I say, it’s confidential anyway, isn’t it? So only good can come of it.

 

And did you, when you sort of signed up to take part, did you have any questions at all you wanted to ask or did ask

 

I didn’t, no. Because they just told me it was confidential and explained to me that, you know, it would help into research into JDM. I was, you know, I was happy with that. It’s a complicated subject, so I don’t really think that I could think of anything to ask. Do you know what I mean? I didn’t know; I know a lot more about JDM now than I did then, because I didn’t have a computer at the time. So, and I’m actually glad I didn’t, because if I’d have gone on there and read the stuff, I would have been awful, absolutely. Because the computer’s not always a good thing to have, the Internet, you know, in, in cases like that. And I’m so glad I didn’t have it. Because I actually managed to Google her symptoms, I, when I got a computer about two years later, and it come up with her condition. And I was just like, “Wow.” But you can Google headache and it’ll give you a brain tumour. So it’s not reliable. But it’s all the stuff you see on there about the condition; you know, how, the things it can do. Not nice.

 

So it can be quite frightening, can’t it the Internet?

 

Very frightening, yes, yes. I mean it was a frightening time anyway for, for all of us, you know. My daughter doesn’t even remember it. So I suppose in a way that’s a good thing, isn’t it? I certainly remember.

 

 

 

 

It is a 10 year study and Sandra and her daughter have been in the study for 6, but they would like to know if the information has helped in any way and what they have discovered about the condition.

 

But in terms of how her data is being used, would you want, do you want to know that…?

 

Well, I suppose it would be interesting to know, you know. And whether anything’s come of it as well. I mean I think it’s a ten-year study. So, I’m sure they said it was a ten-year study, so maybe, I mean we’re nearly six years on, maybe I will find out something in ten years. Whether the information helped in any way, and whether they actually discovered anything from the study. It would be nice to know. As a parent, you know, of a child who’s suffered from JDM, it would be nice to know if they actually got anything back from the study.

 

Nice for your daughter to know as well.

 

Yes, definitely.