Transcript: Lifelong care for NF1
Eric Legius, MD, and Ignacio Blanco, MD, PhD
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- [Eric] Hello. This podcast will be called "Lifelong care for NF1," meaning how to apply new evidence within the regional care models and support structures. I welcome again Professor Ignacio Blanco from the Germans Trias Hospital in Barcelona. And I'm Eric Legius, who's an emeritus professor from the Catholic University of Louvain in Belgium. So welcome, Professor Blanco. Which recent updates in NF1 evidence and guidelines do you consider most impactful? And how do they influence the management across the different age ranges of individuals with NF1 and also different care environments? So these are many questions in one question.
- [Ignacio] Yes. Okay. I will start recalling one thing that we were discussing in the previous podcast. I think, the first thing is that, we have changed our way to deal with patients with NF1. We moved from a reactive symptom driven interventions to a proactive way to manage patients. It means, we don't have to be at our hospital waiting for a patient to come with new symptoms. If not, we have to plan the way that we have to make the surveillance to the patients. Saying that, I think that at least to me, there are five important things that has changed the way that we have to in order to make the surveillance better for patients. First is the possibility to have medical treatment for patients. MEK inhibitors has changed the way that we will be able to manage some tumors. In order to decide if a patient has to receive these medical treatment or not, we need multidisciplinary teams. We need to discuss the overall patients, not only a tumor or symptom. That will be the first one. The second one has been the use of whole body MRI to know the tumor border of the patients. We know right now that this is important to classify patients according with the risk of malignant transformation or dependence of symptoms. Many of the tumors are visible, but we have internal organ tumor, and we need to know it. And I think that, right now, guidelines recommend the least once in life a whole body MRI in order to measure this internal tumor border. The first ones is that plexiform neurofibroma can become malignant. Malignant transformation of the tumor is something that we have to keep in mind. We will have to identify the risk of malignant transformation. The fourth, it will be that we have identified that women with NF1 have a higher risk of breast cancer. And patients women has to start the breast cancer screening earlier than the general population. And also, after 50 years old of age, NF1 women can have the similar screening that the general population. And finally is that cognitive and social problems of psychosocial problems of NF1 are very important and has to be addressed in order to improve quality of life of the patients. To me, these are the more important points to think in surveillance protocol.
- [Eric] Thank you. Thank you very much for that, Ignacio. And I think we are both old enough to remember that in the old days, it was such that you had to monitor the children, but you didn't have to do any surveillance in the adults. And you clearly showed us that also in adults with NF1, you need a proactive surveillance. And I think that's also a major difference with the 20, 30 years ago.
- [Ignacio] Yes, you're right. Completely right.
- [Eric] But when we now move to this landscape where guidelines change, national, international clinicians have to implement these guidelines in their routine practice, but also in a situation with different regional resources, regional pathways, and also infrastructure, that it can be very variable. Do you have any practical strategies how clinicians can get support to implement these changing guidelines?
- [Ignacio] Oh, it's difficult to have practical advice, but we have to be sure that we, right now we have very good guidelines that show us how we should monitor patients with NF1. Our problem is, as you mentioned, the heterogeneous regional infrastructure. We have to sit down with our CEOs, with our health ministers and to define the best way to apply these guidelines. Probably we need resources, general resources that can take care of the patients. I will say that the more common manifestation of patients with NF1, it will be nice to define regional or national hubs, reference centers to be able to solve very complex problems of these patients. And also, we have to connect with international centers. The European Reference Network has an idea that I think that we have to apply. The patients shouldn't move, and the knowledge has to go from one place to another place. When we are dealing with a complex case, we have to have this infrastructure to comment the patients with doctor specialist in other places and to make the plan in the local area. And for that, we have to ask for this, I will say three levels of organizations, a core where many of the manifestations can be solved, an enhanced level where we can discuss or treat complex cases, and perhaps, webs where we can discuss and make decision for very, very complex cases.
- [Eric] Yes, because there is this potential risk that different medical specialties will implement these guidelines at a different speed or in a different way. And also specific regions are not as fast as implementing these than others. And this might create a lack of consistency within a country or within a continent. But I suppose that the hubs and networks that you just mentioned are very important in this, but also maybe the multidisciplinary teams. Can you elaborate a little bit on this, to keep all the different specialties on the same part?
- [Ignacio] Yes, you're completely right. When many different specialties are taking care of a patient, there is the possibility that everyone followed the guidelines in a different speed. And we can lose some follow-up, specific follow-up. For example, in our experience, what has been successful for us has been to clearly define standard operational procedures. We have to be sure what has to be done, who has to perform something, and how we can be sure that the procedure has been performed correctly. We have to define responsibilities, and this has to be shared with all members of the medical team that follow the patients. Everyone has to rely in the responsibility of the other one. And for that, for us, the standardized operational procedures are very important. Defining who and when has to be performed, everything. And we have to also be sure that we will be able to meet together virtual meetings for doctors that deal with patients at least once a year. And also defining, we have to be able to follow all steps and defining KPIs in order to be sure that the procedure is accomplished.
- [Eric] I think that's very important to have standard operational procedures, SOPs and KPIs and everything. But there is this network, the regional NF centers and the dissemination of new evidence to get this into new standard operating procedures. But if you look at the specific country, is this something that every country has to develop in its own setting, every region, or maybe at a level of Europe, or United States, or Asia? How do you see the different levels there?
- [Ignacio] That's complicated because, as you say, we define Europe and United States, but if we look for Europe, every country is different. For example, in our country, we have 17 different health system in a small country like Spain. If we want to, as you mentioned, translate the guidelines, the ways that we should follow the patients, we have to look carefully to our structure and define what's the best way. Here, for example, in Spain, in order to harmonize the way that patients are followed in Spain, we are trying to first centralize translation. Spanish government has identified reference centers. They are the ones that has to lead the translation of the new evidence to all centers. We have to identify these reference centers. We have to provide them with all the possibilities to use as a translational central healthcare provider. Then, it's very important to organize, I will say, cross-regional forums where all doctors that works in a regional area dealing with patients are able to connect to, for example, virtual MDT case conferences where they can hear what are the actual evidence. We have to, as we mentioned before, we have to revise and actualize standard operational procedures from the local point to the reference center. We have to share our standard operations procedures from the different regions and to look how to improve them. And finally, we have to audit all the procedures. We have to identify this, I will say, the PKI, in order to see how we can improve that. It's a process of communication and trust to each other. We cannot build a reference center, and say, okay, I'm the best one and you have to follow my rules. We have to hear the local or the regional doctor that deal with patients and trying to harmonize the best way to be sure to provide a healthcare and the care of our patients. I don't believe in a familiar--
- [Eric] More horizontal but, yeah, less vertical.
- [Ignacio] Yes, less vertical, more horizontal to here. And also, as I mentioned, when we start talking, we have to also to introduce the psychosocial area of the patients with NF1. As you just mentioned, we trust only in specialties. We can have the best surgeon, the best neurologist, the best neurosurgeon, but our patient can have very poor quality of life because they have problems with the self-image, with the burden of the social problems, lack of capacities to work. We have to introduce that in the treatment, and we have to relate in each other. The surgeon is not better than the neurologist and not better than the psychologist. Everyone is taking part of the care of the patient.
- [Eric] Thank you very much, Ignacio, for showing how you solve this issue in Spain, in your own country. And I believe that every country will have its own complexity and every country probably has to find their own solution to provide a consistent healthcare for NF1 individuals. And also thank you for mentioning how the patients feel because I also think that patients have or might have a role in this dissemination of standards, and they also probably should be involved. How can clinicians inform patients and their families about these changing guidelines in the regional healthcare system? And also what is the role of the patients to make sure that they will get the best healthcare? What's your opinion about this?
- [Ignacio] I think that you give the answer. If we really believe that the patient has to be the center of the care of them, we have to rely on that. And patient has to be in the discussions of how to organize healthcare, has to be participating in the discussion about the organization. And also it's very important to hear patients about how the field that has been treated. For example, the Spanish association of NF1 patients many times call us or send an email saying, "Okay, this is not working properly. Patient have to wait in the entrance at the hospital." I remember last Friday they call me, "Okay, it's so difficult to park a car in your hospital that patient gets worried." We need to hear them because, if not, we won't be able to give a good healthcare to them. I always think, like, in a picture, patient is in the center, we can have care plans that has to involve patients. Also, we have to provide material information and the possibilities to have all the information for them. And also, I think that now we have to think in another way to tracking everything. And I believe that we can start using the new technologies in order to have this fluid communication with patient and doctors. We have to introduce apps. We have to be more, with a higher proximity to patients and doctors. And the communication is, I think, that is a key point in all this structure.
- [Eric] Yeah, thank you, because this brings me immediately to the next question. It was a very nice end of your answer, yeah, because do you see any innovative care models? And I'm thinking about care models that are specifically interested in patient empowerment and so on, or digital tools that you mentioned already, apps and so on, but there might be also an important place here for central medical files and so on that everywhere where the patient goes, all his or her medical files are available to the local team there in supporting this more consistent and evidence-based NF surveillance and managements across different age categories, different regions in the country and so on and so on. So which innovative care models and specific digital tools do you see that look promising?
- [Ignacio] Oh, it's very tough question. I will say that, you mentioned one important point. Imagine a patient that is living in Barcelona, has to move to Madrid, or to Brussels or Germany. All the information that this patient has gathered for a long time in Barcelona, have to move to Madrid or Brussels or wherever, Paris. We have to be able to communicate the electronic healthcare systems from different hospitals, different countries. We have to keep the safety of the privacy of the patient. But we have to allow to share the information with different health system. Also, we have to improve the communication of the patient with the health team. And I think that we can build a digital bridge. I mean, right now, for example, we are using a new application in our hospital where patients, every month, every two months, are getting an information from the hospital asking how they feel, if they have new symptoms. And the experience of our patient is very good because they say, "Okay, someone is thinking of me." And that's very easy to make. And now, patients that receive this communication from an app from our hospital are getting more use to communicate them with us. Today, I'm having headache, I have pain in my leg. And now we can use that information to improve the healthcare. I mean, we have to have better communication about healthcare providers, and we have to have better communication with patients and families. For me, these are the two main areas where we have to apply new technologies to improve the healthcare system.
- [Eric] So there is a need at the local level between healthcare providers and patients of a very interactive communication, preferably using digital tools. The same, let's say within a specific country and healthcare system. You need to be able to exchange data from one place to another, also at the international level. And maybe the patient could have his own medical file somewhere, and could grant access to this medical file to different people that need it. But also, Ignacio, if you have a specific issue as an expert, how can you get advice from your peers? Is there a way of internationally have a virtual, multidisciplinary teams and so on?
- [Ignacio] Yes. For example, here in Europe, we have established a European Reference Network for dealing with rare diseases. Specifically, we have the GENTURIS European Reference Network, and we have established twice a month a virtual meeting to discuss patients. We have an application, a safe applications, where we can discuss patients. This is what we call CPMS. That's very important. We have to have this way to discuss specific patients with peers or with patient, with doctors that has a lot of information. One thing that I forgot before to talk about, as you mentioned before, as we are moving from taking care only for children and also for adults, sometimes we believe that our patient, they have a good knowledge of these new applications. And the reality is that not all of them are able to use these applications.
- [Eric] The digital tools.
- [Ignacio] Yes, the digital tools. We need to be sure that the patient has able to use this because, if not, it is another problem for them. I remember, I think that was last month that I was trying to get in touch with a patient in another area of Barcelona. And I say, "Okay, we can have a video conference and we can solve the problem," but the patient has no email and was not able to use a phone. I say, "Okay, we can make a telephone call." But we have to keep in mind that not all people are able to use the same tools that other can.
- [Eric] I think that's a very important remark, Ignacio. So we always have to put the patient at position one and organize everything so that it fits very well with the patient we are dealing with. And with this, I would like to end this podcast. And I want to thank you very much for all your input on the "Lifelong care for NF1." Thank you very much, and thank you all for listening.
- [Ignacio] Thank you very much, Eric. Thanks.
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