00:47:08
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[00:00:06] Dr. Christina Lampe: Dear colleagues, dear participants, welcome to this last module of the online course on the multidisciplinary team approach to MPS patients care treatment and support. It's today the last one, but not less important than the others. And I hope that it will be interesting to speak about the transition from childhood to adulthood practical advice for pediatricians. My name is Christina Lampe. I'm the director of the Rare Disease Center in Gießen. And I'm taking care of MPS patients since a long time. Just one housekeeping notes before we start the presentation. Please do not take any screenshots and do not reproduce any of the slides. The format will be around 30 minutes, probably I'm longer as usual. And at the end, we will have a lot of time for questions and answers. So whenever you have a question, please fill in the chat so that we can answer the questions after or discuss the questions after my talk. Thanks to the Institute of Excellence in Pediatrics to invite me again to perform this webinar.
[00:01:20] Dr. Christina Lampe: Also to the European Reference Network, MetaPRN, and MPS Europe, thank you all for inviting me and being part of this nice educational program. So what we are starting from is what is transition and why we need it. And actually, probably for everybody, it is clear what transition is. But there are also some definitions about it. Before we start with transition, actually, we are talking about not children, not adult patients, but we are talking about adolescents. And everybody who is in contact with adolescents know that it is in any case for all these young people a painful passage over a restless fluid. So it is extremely difficult to be an adolescent. And being an adolescent and having also a rare disease or a chronic disease means it is even more difficult. And actually, the transition is defined from the Society of Adolescent Medicine as a purposeful planned process to address the medical, psychosocial, and educational needs of adolescents with chronic physical and medical conditions as they move from child- centered to adult- oriented health care systems. Very technical, but we will see what this means. Who actually needs transition? Since we have around 800 metabolic diseases, this is not only for metabolic diseases, but since we are talking about MPS, I would like to focus on this. We have a lot of treatments available nowadays. And due to that, we have a lot of children with inborn errors of metabolism that are now growing up and getting adults. So it means also these so- called diseases of childhood are now also a topic for adult physicians. And the question is, are they aware of that? And what can we do to improve the awareness in adult medicine? So chronically, adolescents that need transitions are patients with long- term diseases, special medical needs, life- shortening diseases, and the need of complex medical care. Adolescents are defined as children between 10 and 19 years. It's a UN definition. And we have, in the early 10 to 13 years, to the 14 to 17 years, and late adolescence 18 to 21, very difficult physical, cognitive, and emotional behavioral changes. So this shows that we are not talking about rare diseases only and various individual diseases, but we are also talking about a wide range of different persons that have different needs and different status of development. Adolescent adults are called the older adolescents between 19 and 30 years. This is the phase of ongoing CNS involvement with myelination of the prefrontal cortex, cognitive flexibility, and so on. So at the end, we have, in the US, 18 million people aged between 18 and 21 that are moving into adulthood and will need to transition. So it's quite a big group that we are talking about. So what is our goal of transition? The goal is the culmination of a period of planned and coordinated transition care and guided by the choice of physical, emotional, and social maturity of young person. The major problem is how can we gap this bridge between pediatric care and adult care? The models of care are quite different if we look to how pediatricians work and also how adult care work. And if we see pediatric care, which is more pushing the patient, it's more family- centered, protective. It is more preemptive and focused on development and growth. And we have the adult care that is more independent emotionally, but also financially, autonomy for health, collaborative, and empowering. So it is more to pull the patient. And this is actually what we have to take care of. So when I checked the literature, I found some publications. And this is one of these publications, but there's only a few number of publications about transition, about the gaps regarding transition care. And this is a publication from, it's a little bit older, but it's a questionnaire. And 140 adolescents with a juvenile arthritis and neuromuscular disorders were asked. So it's very patient- centered. It's around 186 parents that answered and 19 providers, so pediatricians and adult physicians and nurses. And the questions were on provider- related characteristics, transitional care delivery process, physical and cultural environment of care services, and patient characteristics related to self- management of care. Sounds very difficult, but what we can see is that if we describe the gaps that the different parts of the group who is involved in transition is mentioning, we see that health care professionals in pediatric care, as well as in adult services, often have limited training in adolescent medicine. It means it's exactly the gap that I said, the pediatric is clear, the adult medicine is clear. But the group in between, which is a big one, is actually not covered properly in this or that system. The translational care delivery process sees that what is needed is an effective communication and coordination between the different care systems. And this must be also in a written way so that everything is very standardized and clear. The physical and cultural environment is important. So the adult hospital environment should be more friendly, less threatening, and more personal. This is something that was mentioned, for sure, from the patient's point of view. And the patient characteristics related to managing self- care, it is not very common in adult care that the patients are coming to visits with the parents. But this should be granted because for these patients with chronic diseases and the parents that were always taking care of them are extremely important. So the key messages out of this publication is that it is extremely important to have a coordination and a communication between child and adult care. And this must have an improvement. The findings suggest the ambiguities concerning the roles and responsibility of adolescents, the parents, providers, in this translational care, which means everybody should know what is my role and what do I have to do and what is my responsibility as well. And actually, the whole group or the most people that answered the question has said that overall, most key elements of transitional care are generic. So it means it is actually quite positive. So this is just one publication about the gaps. But another publication that I want to show is the one, How Can We Improve Transition? And this is one qualitative study that was conducted between 2004 to 2007 in the Erasmus University Medical Center. And they had patients between 15 to 22 years, 24 patients, 24 patients, and 17 health care providers. And here, I don't want to go in detail, but what I would like to show is what are the differences between pediatric care and the adult care. This doesn't mean that pediatric care is only good and adult care is only bad. But it shows the differences. And also for a patient who is quite busy with the disease, with the managing of the daily life activities, has also to switch from a completely different system of medical care to another one. And this should be very smooth. And this is something we would like to work on in transition projects. So, for example, in the pediatric care, and I mentioned before some of them, but I would like to highlight a few of them. So, for example, in pediatric care, it is clear parents are involved. And the expertise, trusted providers' expertise as well. In adult medicine, we have sometimes that parents are less welcome because there's a kind of lack of respect for patients' and parents' expertise because the adult physicians are not used to it. It is not to say they are bad, it is just it is a different way of strategy to how to deal with patients. Also multidisciplinary teamwork or holistic approach is very typical for pediatric care, but not that much in adult care because we have a lot of specialists, a lot of specializations, and the patient goes to the specialist they need, for example. So pediatric care is sometimes, for sure, very childish and not age- appropriate. In adult care, this is, for sure, age- appropriate. It's for adults, but it is, for sure, not easy. Also, the fixed relationships in pediatric care, the child knows the physician, the pediatrician since birth, for example, for 18 years. And all of a sudden, he comes to an adult physician that never saw him before. So there are quite a lot of difficulties. So the key message of this publication was that transition is a very welcome and positive challenge despite inherited insecurities. Young adults and parents wish to be involved in that process, and this is important, not to do it from outside, but to have them as a partner, as in a collaboration to transfer them from pediatric to adult care. The key to successful transition is strengthening independency and encouraging self- management. This is something that I have to say, I'm an adult physician working with children and adults in a pediatric hospital. At the beginning, I was very strict with my patients, and I did not involve them very much, but for sure, I discussed on a medical level with my patients. But I was not, probably I'm very used to discuss also with parents to understand their emotions, feelings, and so on. So it means it is indeed a switch that has to be done. The key to successful transition is also gaining trust to each other and building new personal relations, because also in the development of a child, it is important to be able to manage the disease themselves, to take responsibility for themselves and their disease. So this is actually something that is extremely positive if they switch to adult care system. And actually, the key message is that transitional care is absolutely necessary, and so let's just do it. It is not only the pediatrician who has sometimes also some trouble to give his patient or let the patient go and to stop feeling responsible for a patient that he's taking care of for 18 years. It is also the adult physician who has some concerns. And there's one publication about the feeling of the internists, how they feel in taking care all of a sudden or being in the situation or condition of taking care of a child with a chronic disease and all the big binders of medical reports that the patient collected in the last 18 years. So the medical competency is something that the internists have concerns of. Also, the patient's psychosocial needs. Time in adult medicine is sometimes shorter than in pediatrics, so there's not that much time to take care about psychosocial needs. The patient's maturity, as we said at the beginning, adolescent is a big span of different children with different developmental steps. We have the system issues that are not clear. The family involvement is something internists are concerned of, and also how the transition coordination should work. So, this was the first part and the introduction to that, but what we would like to do in this presentation is actually to show a way what can we do to find a model or how can we find an idea to make transition successful for the patient. What do we know about transition in different countries? And here I would like to thank the MetabERN, the European Reference Network for Metabolic Diseases, that is supporting also these modules and the one from today. And we made a big questionnaire about transition and I will show you a little bit later some key messages out of this questionnaire, but just to explain you the MetabERN network, it's a European network. In 2017, we had 18 EU member states and 69 healthcare providers that were part of with 30, 000 to 40, 000 metabolic patients inside. And in 2019, we were grown to 23 European member states and 78 healthcare providers with 50, 000 to 60, 000 metabolic patients. So the network is growing, but this network also helps us and gives us the impact to change something for our patients and here in the MetabERN for the metabolic patients and so also for the MPS patients. To give you an impression how the division between pediatric patients and adult patients is, we can see in 2017, we had only 32% adult metabolic patients. And in 2019, so two years later, 51%. So it means the group of adult metabolic patients is growing and this is why we need good strategies and good ideas how to put these patients or have the best care for these patients in the best system that they deserve. I told already that we made a questionnaire and from this 69 healthcare providers that were asked to response, we had 63 healthcare providers from 20 European countries that answered to our questionnaire. And what we found out is that only 11% are adult metabolic physicians. 85 centers follow pediatric and adult physicians, which means that there are a lot of patients that have no transition or a lot of patients that are taken care by a pediatrician for a very long time, maybe in cooperation with adult physicians specialists, but nevertheless, they stay with the pediatrician. Most patients have a transition at the age of 18, but almost 20% have never any transition. So they stay indeed with the pediatrician, as I said before, or they were followed by pediatricians only lifelong. The reasons that healthcare providers think why a transition is not performed in some hospitals is historical. Adult metabolic patients have been taken care of pediatric hospital, that's it, and difficult to have more salaries for adult physicians. The need of a transition coordinator was mentioned in many, many cases in this from many, many healthcare providers. Also that still transition has no standardized transition protocol, but most have to share information with the adult physician. So there is an exchange of information between pediatric and adult medicine, but it is not standardized and not in a written protocol as it should be. There was also mentioned that there's a need of special training in adolescent metabolic patients. There's also mentioned a lack of financial support for transition in 90% of all cases. And when we asked the healthcare providers about the reason for not having transition programs, most physicians answered that is the lack of time, lack of adult metabolic physicians. So this gives us an impression in which direction we have to work on. So what would be needed is actually staff dedicated to transition, a coordinator, and training for adult physicians. So which models of transition exist, and is it that one model fits for all? Since we are talking about Europe, we have so many different healthcare systems, so many different payment systems, so many different political systems that it is difficult to establish one system that has to fit to each hospital or each country, especially in different countries. We have different regional rules, and we have also different rules in different hospitals. So at the end, what we would like to do is to find a way to offer some models, to offer some key messages that are important to perform a successful transition according to the system or according to the situation and condition you have in your hospital. So when we started with that, we started with the brainstorming about the major problem of establishing a transition program. And it seems to be the educational aspect of adult physicians, as we mentioned and we had the confirmation in our questionnaire before. We have the structural aspects, as I said, different political and health care systems. We have the financial aspect and also social aspects. And this is a busy slide, and I don't want to go in detail. But to the educational aspect of physician, what we would need to understand is how to engage adult physicians so that they really are interested and engaged to take care of our MPS patients, for example, or the other metabolic patients. The structural aspects, we would need to collect options to establish an adult multidisciplinary team and training programs. And also, MetabERN is working on these training programs. There's also the financial aspect. We would need to find ways to get adequate reimbursement for patient visits. For example, if a pediatrician is taking care of a patient and you invite an adult physician taking part of this visit with the patient together, who is getting the money at the end? So these are questions that have to be clarified. And there must be a kind of official way to take care of these patients with pediatricians and adult physicians. And we have, last but not least, the social aspect, which means how to encourage pediatricians and families and patients and adult physicians to cooperate with each other. So these are the questions. And if we're facing this five-fold challenge of transition care, we see that there's a lack of awareness of the challenges of adolescent transition care. We have a fragmentation of the transition care system set up between and within the European countries. We have an insufficient transition care education and training for patients, families, and caretakers. We have a lack of research into the particular aspect of rare disease management and a lack of coherent budget plans for national strategies. So actually, what do we need? There is a need of a personalized and flexible transition process tailored to patient-specific needs, or do we have one model fits all? There are some very successful transition programs in the EU, but also in the US. And here's just a list of different publications about that, or models of transition. There's one from Germany in Berlin. We have one from France that I will explain a little bit more in detail as an example. We have some, actually quite a lot from Spain. I see four, at least, different publications about that. We have Belgium, Ireland, Italy. So there are quite a lot of people thinking how we can improve with the transition of care. I mentioned already that we have also in the US. And one example from another European reference network is from the ERKNet. This is Kidney, and they had current management of transition of young people affected by rare renal conditions in their network. So they have an idea already. So the conclusion is we really need to revise, implement, merge, adopt such programs, and provide a consensus document. And this is what the transition working group of MetabERN is doing. We started with a questionnaire. No, we started with a brainstorming. We started with a questionnaire to understand the situation in Europe about transition. The next step will be to publish the data that we have, so the current situation of metabolic patients and transition in Europe. And the next step will be to give some recommendations how transition programs could be established, how it could work, and what we can do to establish them in our hospital. Since not one model is fitting in all, it means it is very variable. But it gives some key messages. This is what we try to do, is to give some key messages that you. You can pick these things that are necessary, needed in your hospital to prepare a transition program that is fitting to your patient and your hospital or your situation. And actually, the last part is that we would like, as MetabERN, Transition Working Group, going to the European Commission and showing this data, showing our need of improvement in this transition care. We need financial support. We need structural support. And this is actually the step that we want to do. But now back to the models. I promised to show some examples how transition models could look like. We have here one from Washington that they start at the age of 12 years. And the integration to the adult care is finalized at the age of 23 to 26 years. So there's a startup with the transition policy, then the planning of the transition at the age of 14. With 16, they prepare and discuss the transfer. With 18, there is a transition to the adult model of care. And then the transfer takes from 18 to 22. So it means it's a long process. And this is also what we have to take into consideration or into account when we speak about transition. We cannot say, now you are 18, now you are in a different system. But we have to go this way with our patients and to bring them to the right physician in the other system. And this US model that I showed, the transition is made in preliminary steps, in six steps. So securing senior leadership support, the forming the transition improvement team, defining transition process for improvement, and dedicating time to implement transition.
[00:28:19] Dr. Christina Lampe: And this means that the pediatric health and the adult health, at the end, has to follow six core elements, like the transition policy, the registry of transitioning, the preparation that they do together, the transition planning, also incorporation between pediatrics and adult medicine, the transition and transfer of care. And then the transition completion. So this is actually the steps. And it's a very nice system. I see it. So now I lost my... Ah, here it is. So another example from Europe is I've chosen the one from France that is called Jump. And it is quite similar, but adapted to the country. And you see here, the first step is to enroll the patient. And I will show you some patient numbers that were enrolled in that system. The second step is the pre- jump, so the review of the pediatric medical notes, identify patient's principal interest and medical issues, and then to arrange the jump day. The jump is then to introduce patients to the adult hospital, therapeutic education, cultivate patient's life project, and tailored consultations in a multidisciplinary team. The post- jump, then, is implementing the recommendations made by the jump team and the exit of the jump, the program length determined by the patient. So it is not done by the age, but by the patient when the patient is ready. And you see here that they started in this program 111 patients. And you see that a lot of consultations were done, not only by medical specialists, but also by allied healthcare professionals. Disease evolution is there. And also assess the disease complications that were mentioned here. So it's quite a nice project. So now, since we are here in the MPS, multidisciplinary team approach module, what is about MPS? Our major problem in MPS is that we have two different groups of patients. We have the patients without cognitive involvement that are of normal intelligence. They can speak for themselves. They can make their mind. They can say what they want. And we have the patients, and they are getting adults as well, that have a cognitive involvement and cannot explain what they want to say. Nevertheless, since we are talking about here, it was Hunter's disease, but this is also valid for all MPSs, we have the need of a continuing monitoring treatment of the somatic symptoms. Since we are talking about a progressive disease, symptoms worsen over time. So it means the pulmonary, cardiological, neurological complications have to be monitored and followed up and also taken care of in all these patients. So the life- threatening disease complications. But also continuous monitoring of and treatment of symptoms is necessary in the symptoms that are quality of life reducing disease complications like orthopedic complications, psychological complications, pain management, visual and hearing loss. So this is very typical for MPS and it's for sure a specialist for MPS. But whenever an MPS patient is getting adult, they have the common diseases of adulthood as well, like diabetes, hypertension, cancer, regular gynecological check-ups and so on that are needed. So they need also adult physicians who are taking care of them. If we see the cognitive assessments of psychological problems in MPS patients, a severe affected patient, he cannot express his thoughts, but he's very sensitive to changes. So whenever he knows he's going to the hospital, there's always the same voice, the same doctor that is taking care of. He feels safe and protected. But if he is all of a sudden in a new environment, it can be quite a big shock and very difficult for cognitive involved patients. So at the end, it's the parent's decision how to manage adulthood. They have the same somatic complications as the non-cognitive involved patients. And you have also to take care of the end-of-life planning in these patients. In the patients without cognitive involvement, one sentence of my patient that you see on the picture was, I'm different, but I want to have a normal life. So the problems of daily life are a little bit different. It's the independency while assistance is needed. There's the work, there's the family life and family planning, genetic counselings, and also the end-of-life planning at a certain point. So what I would like to highlight is that in any case, you need a lot of specialists. So a multidisciplinary team that is taking care of MPS patients. And this is what you heard in the different modules before as well. But in any case, you need a coordinator or you need a metabolic specialist who knows about the disease and who knows how at the end to make a good decision together with the patient or the parents. About transition and MPS patients, we made a publication last year. And what we did actually, since we don't have one model fits all, we presented in this publication four different models of transition. And we showed how we manage our transition in Germany, in Spain, Russia, and UK. UK has a fantastic program for transition as well. So at the end, we tried to give some input how a transition could work on. And the key steps are actually in the early planning of transition, the education and engagement, also to bring pediatricians and adult physicians together and to work together. And the transition documentation, as we said before, we need a clear standardized documentation of this transition. We need clinical expertise, for sure, and programs to get this clinical expertise, so educational programs. We need a coordination or someone who is organizing this transition and is taking care of this transition program that it is really working. We need family involvement, for sure. If a family does not want to take part of a transition, it's extremely difficult to find a way. And even if we have a good model of transition, we need quite a lot of flexibility and individualization due to the fact that we have such a heterogeneity of disease severity in our patients. So, at the end, the conclusion of our paper of transition was that each patient manifests with so variable symptoms, rates of progressions, capabilities, and interaction with their carers. While a structured transition progress is required, the plans must be really individualized for each patient and remain flexible throughout to ensure that each patient moves through adolescence, changes in their independence and support needs are really reflected in their progress toward disease self-management and adult care. So it means even if we think, or it is my personal opinion, transition is needed, at the end we have really to see and check the patient's personal individual situation to make the best model for him. So to summarize transition recommendations, and I'm quite keen on having a lot of questions and discussion points in that because there's no real clear way of transition so far in the world. So to summarize that, young people should be given the opportunity to be seen without their parents. So even if there's no transition program, children at a certain age should have the chance to speak with the pediatrician or the physician alone to ask questions that they don't want to ask when the parents are inside, like fertility or family planning and so on. Some children don't want to scare their parents and they don't ask questions when they're in the room because they know the parents are very concerned about the disease and they don't want to ask these questions. So please give the children a short time of asking questions alone. I start this personally at the age of six for a few minutes, and at the age of 12 in a longer talk without the parents. I send them to have a coffee and I speak with the children, and I'm quite astonished how much questions they ask without parents inside the room. We also have to establish multidisciplinary teams. We have to take care of an optimal cooperation between the adult and pediatric services. We have to find a coordinator. This must not be always a physician. This could also be a nurse or a manager, someone who is bringing all the information together, collecting the information, and then making, having a holistic picture. And in the multidisciplinary team, decisions can be made in collaboration with the patients or the parents. So there must be someone who is taking care also of the data. Young people should be encouraged to take part in transition programs.
[00:39:13] Dr. Christina Lampe: Sometimes pediatricians don't want to, as I said at the beginning, let the patients go. So it is important to let them go and to encourage them to go new ways and to think, to be more independent. The involvement of adult physicians prior to transfer, so that they get used to the patients, that they have a chance to know the patient and to understand already the situation of the patient before the adult physician has to take care of the patients completely. Then, for sure, we have to take care of the social, psychological, emotional problems, which require assistance. So we need not only physicians, but also dieticians. We need physiotherapists. We need psychologists. We need a lot of people around to make this transition process smooth. And actually, the transition services must undergo continued evaluation. And this is why there is no one model fits all. But it must be quite individualized, but standardized. So our final goal is the optimal health care is then achieved when every person at every age receives health care that is medically and developmentally appropriate. Thank you so much. We are working on that in the MetanERN transition group, I told you already. Thank you for your attention. Here is also my email address whenever you have some personal comments, some discussion points, or some questions. But now I would like to open the chat. And I'm very happy to receive your questions to discuss with you. Thank you so much. This was from my point of view. And now let's start the chat. OK, there's the first question. How do you decide on different aspects of transition when you know the patient's condition will deteriorate in the medium to long term? How can your future prove the transition process? Actually, I don't understand the question directly. And I need glasses because it is too small to read it. Well, it is extremely difficult. This is what I said, how to decide. I mean, important is in patients that you think they will deteriorate very soon with their disease to work together with adult teams. So what I'm usually doing, since I don't have an adult team in my hospital that is taking over my patients completely, I'm an adult physician working in a pediatric hospital. And what I do, I have a lot of adult specialists that are taking care of my patients. So I send them to an adult cardiologist, I send them to an adult neurologist so that they are part of the adult medicine, but I'm the one who is coordinating or yeah, coordinating and taking care at the end of the patients. Does one have to wait for an adolescent to turn 18 before referring to an internist, even if the adolescent looks like an adult already in terms of his physique? Actually, this is what I said. There are some countries that start strictly with 18 because from the reimbursement systems, most systems allow pediatricians to take care of patients up to the age of 18. Sometimes it's prolonged to 21 years of age, but then they have to switch to the adult physicians. In patients that are much more mature or look more mature than expected, I would still think that they are part of the pediatric care, but you can start the transition process earlier so that you have a longer time of overlap between pediatric and adult care. Who is best placed to head up the transition, the pediatric and adolescent or adult care teams who takes the lead? So far in the models that we have are the pediatricians, the ones who are taking the lead. This is actually due to the fact that they have the patients first and they want to transfer the patients to adult system. So actually it is mainly the pediatricians that are switching the patients. In most transition programs, it is led by the pediatricians. But I have to say, if we would have adult physicians that would be willing to coordinate this transition and would lead this, it would be fantastic. And actually, I think I made a mistake. I think in UK, there's indeed an adult physician who is really the one who is taking care of the transition and is transitioning the patients from the pediatrician to the adult medicine. But this is a physician that is in between. Any further questions?What should I do as a pediatrician when the support care is not what I would expect once the transition is underway? Should and how can I continue to play a supportive role? I think that, or this is what I also see in my patients, that whenever they are more linked to the adult care system, at the end, the one who knows them best, it's me. And I think that there's always a way, and this is why the communication and the work together with the pediatric and adult physicians is so important is indeed to play a role, to support the family, to find a way with the adult physicians, where I'm usually trying really to be in close contact to the adult physicians, to let them understand how the patients are feeling, how they are, what the disease is, so that the patients feel more comfortable.
[00:46:08] Dr. Christina Lampe: For sure, it is difficult if the process started and the patient don't want to switch and there's no way back. There's only the physician or pediatrician who can support. But I think this support is extremely necessary, needed for the patient, the parents, but also for the adult physicians. And I think with meetings together, visits together, this should be possible to have a good transition at the end. Thank you so much for listening, for the questions. And this was the last module of the multidisciplinary team approach in MPS. I hope you enjoyed these webinars. I enjoyed very much. I was part of the first and now the last one. Thank you so much and have a nice day. Bye-bye.