Episode 9: Personal experiences with clinical trials (English version)

Welcome to the MPN-train, your podcast about myeloproliferative neoplasms with a focus on essential thrombocythaemia, polycythaemia vera and myelofibrosis. Here, people affected, experts, patients and relatives talk openly and positively about MPN. 
With the kind support of AOP Health and Novartis

Welcome to the 9th episode of our MPN-Train. My name is Enrika, I'm a PV patient myself and I'd like to welcome you today and guide you through the programme. Today's topic is the participation of a member in a clinical trial, and that is our friend Peter who has made himself available. I would like to ask him a few questions. 
Hello Peter.

Hello, good afternoon. 

Please tell us a bit about your story, what is your illness?

At the beginning, I found out about it 9 years ago, by chance. My GP told me ‘you need to drink more, your blood is too thick’ and I did so, but it didn't get any better and then it turned out that the number of red blood cells among the thrombocytes was far too high. He then referred me to Linz, to the Elisabethinen Hospital in Linz, to the haematological oncology outpatient clinic and I have been undergoing treatment there ever since and am in very, very good hands, I have to say that upfront. I was diagnosed with polycythaemia vera. I was initially treated with a drug that is a mild chemotherapy, which didn't do me much good. I had a number of side effects, but then after a few years I developed some peculiarities in my abdomen, namely an enlargement of the spleen, which became noticeable and the doctor treating me thought it might be a good idea to take a bone marrow aspirate and it turned out through this bone marrow aspirate that the bone marrow had already become scarred and therefore the diagnosis has become myelofibrosis.

Enrika 
That means, sorry to interrupt, that polycythaemia vera was diagnosed at the beginning. Peter : and in the course of the following years Peter : this condition degenerated? Is that correct? (

Peter 
That’s how it was
Enrika 
And at what intervals were you regularly checked?

Peter Saler 
Relatively frequently at two-month intervals, but it did not show up in the blood values until the bone marrow aspiration, when it became apparent that something was wrong in the bone marrow.
Enrika 
And as for the general condition, i.e. the personal condition, how did you feel?

Peter 
Changeable. At the beginning of the illness, with the mild chemotherapy, I didn't feel well, I had swollen feet, I was mentally quite distressed because I was wondering where it would go from there. It got better with the interferon treatment because I had the feeling that it was working well and that it was good for me. After all, it's a treatment that aims to make the body react on its own, which was an idea I quite liked, and then came the big disappointment that it had become myelofibrosis and that we had to switch the medication again.

Enrika 
And did you notice the improvement immediately or any difference?

Peter 
I was on a too high dose at the beginning, which was then reduced, but somewhere along the line I got the impression that it wasn't helping properly and that was ultimately the case because myelofibrosis was diagnosed and the scarring was relatively advanced, which could not yet be determined based on the blood values.

 Enrika 
So, how long did the whole thing last?

Peter 
The total period was 9

Enrika 
And are you feeling better?
Peter 
Yes, it's hard to tell, I'm getting older too.

Enrika 
How has this affected your work and your activities?

Peter 
I had to learn to deal with restrictions, especially this exhaustion, which was new to me and which I actually wanted to fight by working against it, but I just realised one hour of gardening, and I had to lie down. Nevertheless, I was able to do some mountain hikes and cycling, swimming and so on - the sports I wanted to do, that all went quite well.
Enrika 
And how did it affect your diet? What about alcohol?


Peter 
So I've actually given up alcohol completely. I drink a maximum of 1/8 of wine, if I drink more I get a headache, but that hasn't caused me any major problems and apart from that I've tried to eat as healthily as possible, in the beginning it was no meat, especially no red meat, in the meantime it's different again, but I still have to be careful and try to eat as well as I can, not vegetarian but with less meat.

Enrika 
And how is the decision made, in the moment or is it a general plan to follow or on a whim?

Peter 
It's a gradual decision, especially depending on how you tolerate certain things. I realised that it's best to eat something with the medication so that you don't have the medication in your stomach just like that and I also had advice from a doctor who always said, ‘do everything that conventional medicine suggests and expects of you but you can also do something good for your body by taking something else additionally’.

Enrika 
And what did she recommend? Chocolate?

Peter 
No, unfortunately not. Yes, that might be good for the soul.
Enrika 
Yes, the soul needs something too. (Peter: The soul needs something too)

Peter 
And that's coffee for me, I always drink coffee in the morning, it's actually not bad for me, I just need it so that I feel a bit fresher and then I feel a bit better too. In other respects, there are trace elements that you should also supplement if there is a deficiency, but this changes depending on the time, the disease and the season. In winter, for example, I always take vitamin D.

Enrika 
And how did the whole thing affect your body weight?

Peter 
No way at all, actually. I've always been the same weight since I was a teenager.


Enrika 
Well, and the whole thing then led to something where you personally took a decision. I want to take part in a study. How did this happen? How did this come about?

Peter 
Yes, it came about because I read the results of the bone marrow aspiration from one time to the next with horror and my doctor told me that she didn't want to scare me of course but that there was a slight deterioration, but of course I could figure out how things would go if the medication stopped again and wasn’t just reduced, it would continue without me being able to do anything about it. But I was very lucky that my doctor was a scientist at the same time.
She works at the university in Linz and she told me several times about studies and then always said that it must be suitable for me and once I was close to taking part in a study but then informed myself about the side effects and then flinched on the last day and didn't do it. And then another 1-2 months passed and then she said to me ‘there is another medication that would be suitable for you’ and she was kind enough to send me information from study 2.  

Enrika 
And I suppose you must fill in a few things when you do this. There must be questionnaires that you have to fill in.

Peter 
Yes, there is a lot to fill in. You must be suitable for the study, i.e. the degree of illness has to be such that it guarantees, how should I put it, scientific success for the people who commission the study, in the sense that you say the person has such and such symptoms and we look at whether the drug works this way or that. This meant that I had to indicate the level of my pain, on a scale of 0-10 and how exhausted I felt, how the pressure in my ears and stomach was, etc. There was a questionnaire of several pages to fill in, but I didn't have to fill it in on my own - there's a study nurse at the hospital in Linz who also accompanies you with these forms that you must complete.

Enrika 
That means you also have to understand the questions.

Peter 
You have to understand the questions, of course you can also ask back, but it's good if you ask a lot.
Enrika and Peter in agreement
YES

Peter 
Because ultimately your own body is affected and it is a study, a scientific experiment.

Enrika 
And I can only say from my own experience that it is so difficult to define pain.

Peter 
Very difficult, very difficult, my attending physician encouraged me and said ‘don't be so cautious in the scale assessment of the pain, otherwise you won't even make it into the study. And that encouraged me to make the definitions a bit more bluntly than I wanted to. I'm a bit accustomed as a person to endure pain and in this case, you have to say quite honestly, yes, I can feel something and I'll admit it.

Enrika
In other words, the study involved daily queries via mobile phone?
Peter 
Yes, via my own mobile phone and from about 5 o'clock in the afternoon the scale that you had or have to fill in (as I'm still doing it) was activated for the day.

Enrika 
What are the requirements for such a study? Do you have to have a certain amount of knowledge, or can you really start as an absolute layman?

Peter 
I am also a layman, but I have tried to inform myself, I would advise anyone who wants to get involved in a study to inform themselves, especially about the effects in the study.

 Enrika 
Could you explain a bit about Study 1, Study 2, Study 3?

16:26 Peter 
Scientifically unfortunately not really, but only as I (16:21 Enrika: from your own experience) so I can't say anything about study 1, I don't even know if it's done with people. In study 2 only 300 people worldwide were included in my medication, although they were varied in terms of age, gender and origin. 300 is not a lot, but at least it was established that there are such and such side effects, and I did find out more because I am sensitive in the digestive area - gastrointestinal issues and so on - and actually everything was stated as tolerable in study 2 and the cases that were reported did not occur in my case, I must honestly say. So, the side effects in the trial I'm currently on are very minimal, lucky for me.


Enrika 
So, the questionnaire had to be filled out meticulously with help. You were given a special mobile phone (Peter: Yes) 
You must answer the questions every day (Peter: Yes) and how long have you been doing this?

Peter 
I have to say something in advance: before the study even starts, a comprehensive examination is carried out. This doesn't just mean blood and bone marrow, but also ECG and MR, i.e. magnetic resonance of the whole body, of the internal organs. A detailed examination is carried out to ensure that nothing else is present or that there is no overlap with another illness, etc. This is also a requirement before entering a study.

Enrika 
Experience shows that the liver is always affected. Is that also the case for you?

Peter 
No, thank God it's not. Of course, I take special care with my food, too. I also take things here and there to detoxify my liver - milk thistle, harmless extracts.

Enrika 
Does this mean that you are within the normal range for the entire examination?

Peter 
Yes, the spleen is very enlarged, and it is also pressing and nothing can really be done with medication.


Enrika 
So you are receiving adequate medical and scientific care in your current condition?

Peter 
I feel very well looked after; I have to say that I was very lucky to have an expert as a doctor. Of course, I also put up with a lot, I travel one hour by train to the hospital and have weekly check-ups during the study, I have to add. Of course, you also have to wait for hours in the hospital until you get your turn, until the blood is analysed etc. So you have to accept these inconveniences.

Enrika 
And do you also have contact with your attending physician or is this taking place through medical staff?

Peter 
I always have contact with the attending doctor or her deputy. It can easily happen that she's not there one day, but I feel well looked after. Of course, I also have to bear in mind that her time is limited and the time I can talk to her is 10 -15 minutes at the most and I have to write down everything I want to ask her beforehand, I do it now with a cheat sheet (Enrika: yes, that's good), not that I then go home and then say ahh, you forgot to ask that again.

Enrika 
I have another question - there are a lot of appointments and examinations that you have to do. What does it look like financially, do you have to shoulder the everything?

Peter 
Before I answer the question, you need to know that I'm retired and I'm in the fortunate position of being able to manage this in terms of time. If someone is working, it can be difficult to find the time. That much about time. Financially, I have to say that the company bears all the costs. In terms of the study, that is certainly a lot of money and of course it is also expected that the results be scientifically acceptable. So, I can't complain financially, I also get vouchers for the journeys I have to make, I get my medication allocated to the exact day. I always have to take the empty box back with me, which means they check if I've taken all my medication. In the first 2 weeks, I even had to state what time I took the medication, which was a bit difficult for me because I don't eat breakfast regularly and always at the same time, for example, but you have to indicate the time.

Enrika 
And what happens when the trial is over and the whole thing works and you feel better, do you continue to receive this medication?

 Peter 
Yes, I have been promised that I will continue to receive the medication, even if it is not yet licensed. If you commit to it, then you also have the right to continue to receive the medication

Enrika 
Yes, that's wonderful and I think that in you it has also reached a person who already has some inherent system and is predestined for such a study, because you stand by your disease, you stand by the guidelines that you receive and that you adhere to the rules. So, I think you are the perfect candidate. One thing fits the other

Peter 
I don't want to praise myself, but if someone gets involved in a study, then they have to take it upon themselves. It has to be clear to them from the outset: I want to do it the way it's supposed to be done, otherwise it makes no sense neither for me nor for science. Maybe a few words about the emotions that are associated with it, that all sounds so rational and so great, but in reality, there are also worries and fears involved – how will it be going forward, how will I tolerate it, who can I tell if I feel something, does it have anything to do with the medication or not? So, it takes a lot of courage and optimism when you embark on a trial.

Enrika 
And hope.

Peter 
And hope, of course.

08:20 Enrika 
Could you maybe tell us, how was the beginning of this study, from a personal aspect, the first contact, how did it take place?

 Peter 
Yes, that was when I said OK to it. I was ordered to an appointment, I absolutely had to keep the appointment, I also had a negative personal experience that day because I had a cycling accident, but I kept the appointment, went to the hospital and had to take the first medication in the presence of the doctor. It's a strange feeling when you take a tablet that doesn't have a name, is unknown and you don't know what effect it would have. That feeling then wears off over the course of a few days.

 Enrika 
Well and how long will this go on, how long will this study last?

 Peter 
Yes, I had a bone marrow aspiration at the end of November and with a bone marrow aspiration you can always determine how high the degree of scarring of the bone marrow is, and to my great joy the degree of scarring has decreased. So, I was very happy when I found out. I'll have my next consultation and examination at the hospital next week, when I'll find out whether the dose of the medication needs to be changed, what the next steps are and for how long and so on. I wanted to say something about the dosage. The purpose of study 3 is also to determine the dose of medication and this also varies from person to person. In my case, the dose had to be changed more often because the number of thrombocytes decreased. It's an effect of this medication that you had to reduce the dose. I was afraid, if I took too little, would it work at all? but that fear was unfounded, because it turned out that it did work, but you also have problems with adjusting the dose and you always have to have your doctor check on you.

 Enrika 
Yes, it is a delicate work. (Peter: Yes) And it's the medication on the one hand, but it's also the mental aspect, isn't it? (Peter Yes) You can move mountains (Peter Yes) and I also think the relationship, the relationship that you build up with the doctor looking after you, that you put yourself and your hope completely in their experience, in their hands, don't you? Especially the hope of how it would go on, can the disease be passed on and these are all factors that I think are important when you make yourself available for a study.

Peter 
Yes, that's right. Right at the beginning of my illness I enquired whether this condition is hereditary, which was also very important for my children to know, and I learnt that it is not hereditary. I hope that's true. Another interesting fact was that I was 65 when I was diagnosed with the disease. At the time I was told ‘you are too old for a stem cell transplant’ and I have since learnt that they tend to go by biological age, so if I had done it back then I could still have met the age to have the operation. But I don't regret it anyway because I know what complications a stem cell transplant is associated with. One more thing about hope. I have also built myself up mentally, I have to say, and I dare to say that I am a believer and have also put a lot in God's hands.

Enrika
Yes, and I think it's always important that you always see the glass half full, that you undertake activities that are nice. Personally, I discovered music and that helps a lot too, that you mentally engage with things that are beautiful and that helps you, helps everything. (Peter : Yes) It helps you, it helps your environment (Peter Saler: Yes) if you are positive yourself, then you also meet positive people ( Peter Yes) and that is very, very important but of course does not exclude the fact that you have to undergo therapy, that is really, really important and you can only thank the doctors who spend many, many hours of their whole lives on it, especially in haematology, which is incredibly large as a field and changes every hour, new findings are constantly being added and that is actually what gives us hope that we can continue to live normally. How do you see it?

Peter 
Yes, that's exactly how I see it and what you described as the uplifting element with the music might be a forest walk for me. I walk through the forest with my Nordic walking poles three times a week, if I can, if I don't have an infection or something, for an hour or so and it gives me a lot of strength and I have the feeling that it's good for me.

Enrika 
So are there any other things you would like to share with us to give us all courage? So far, I can only say thank you for the positive experiences and messages you gave us and I hope we keep on getting further positive messages. Is there anything else you would like to share with us?

Peter 
Yes, I have said a lot already, I think that everyone has to decide for themselves whether they have the courage to take part in a trial and that they have to think carefully and clarify a few things for themselves beforehand to see whether they can go through with it, whether they have the courage, whether they trust themselves and also whether they have the confidence in the doctor treating them.

Enrika 
Yes, dear Peter, I would really like to thank you very, very much; the train has now arrived at the main station, we can all get off again and look forward to the next episode. Thank you very much, see you soon.

00:28 Peter Saler 
You’re welcome, speak to you soon.

00:27 Outro- Musik

00:25
That was the MPN train, your podcast about myeloproliferative neoplasms with a focus on essential thrombocythaemia, polycythaemia vera and myelofibrosis. Here, people affected, experts, patients and relatives talk openly and positively about MPN. 
With the kind support of AOP Health and Novartis.