Map out a treatment plan with your doctor
MAC lung disease is tough and can take time to treat and manage. That’s why it’s important to keep the lines of communication open with your doctor and establish a treatment plan.
Important things to discuss with your doctor:
Treatment goals: A goal of treatment is to test MAC-negative. Ask your doctor how you’ll track toward this goal
Possible side effects: It’s important to understand potential side effects of ARIKAYCE and ways to help manage them. Being prepared gives you a better chance of taking your treatment as prescribed
When you may begin testing MAC-negative: In the clinical study, people who tested MAC-negative were on treatment for 1-4 months before they received their first negative test
Length of treatment: The NTM Treatment Guidelines* recommend staying on NTM treatment for at least 12 months after your first MAC-negative test to help prevent the infection from coming back
ATS=American Thoracic Society; ERS=European Respiratory Society; ESCMID=European Society of Clinical Microbiology and Infectious Diseases; IDSA=Infectious Diseases Society of America; MAC=Mycobacterium avium complex; NTM=nontuberculous mycobacteria.
“ARIKAYCE made me feel hopeful that I could get this disease under control.”
–Lynn, a real ARIKAYCE patient
See how Lynn and her doctor created her ARIKAYCE treatment plan together
Lynn had long searched for help with her MAC lung disease. When she met Dr. Lapinel, she knew she had found a partner for her treatment journey.
My name is Lynn, I’m 67 years old and I live in Pearl River, Louisiana.
My name is Dr. Nicole Lapinel and I’ve been in pulmonary critical care for the last few years.
Lynn had had a long path—diagnosis, you know, many years before she had actually met me.
When the infectious disease doctor became aware that I had NTM, she told
me about a doctor who was involved in treating NTM, which is Dr. Lapinel.
I was very impressed the first time I met you, I was impressed with the
abundance of knowledge that you had about this illness.
That makes me feel very, very good to hear that. Very comforting for me as a physician.
My first visit was involved. I was a bit intimidated and overwhelmed at this point because it was just so much information for me to absorb.
During that first appointment, I always try to get as much of that
background history as I possibly can. So, all of that is important in terms of setting our pathway, setting our treatment regimen up and everything like that, getting more of that history. And I hope that even though it was overwhelming to kind of hear all that, that you had a sense that we are going to be taking this kind of step by step.
We had put you on a daily regimen and discontinued one of the antibiotics that you were on and then added two more antibiotics to that regimen on that very, very first visit.
I wasn’t excited about taking more medication, but I felt hopeful for the
first time in a long time of being treated properly.
When Dr. Lapinel told me that my sputum samples were still coming back
positive, I was disappointed. I had a lot of hope that I would see more of a result at this point.
Treatment can potentially not be successful for a variety of reasons. That is something that we also talk to our patients about in terms
of managing expectations.
I knew there was a possibility after six months if I was still
producing positive sputum samples, that we were going to add to the medication I was already taking.
So, it was actually six months into your regimen when cultures were
still positive that we actually decided to add a new medication, an inhaled antibiotic, which is called ARIKAYCE.
Knowing that ARIKAYCE was available for people like me, made me
feel hopeful that I could get this disease under control.
I felt confident that this was the appropriate addition to the regimen, because for the first in its time for MAC lung disease, we actually had an evidence-based approved medication that was also FDA approved.
Whenever we’re introducing new medications, we like to go over those side
effects because that’s…that’s part of handling the treatment. I always discuss cough because so many patients are already suffering with cough.
So, I was prepared for just what she said, the cough, the hoarseness, and the shortness of breath. But she also gave me ideas on what I could do to make it better.
I did keep track of my side effects. I took personal notes so that when
I did see you I could relay them to you and not leave anything out.
I enrolled in the Arikares Support Program. They have been very, very
helpful to me. Someone has come out to my house initially to educate
me on how to use the medication. If I had a problem all I had to do was
make a phone call and somebody was always there to help me immediately.
I’ve actually heard truthfully from many patients that they’re very satisfied with the support that they provide through the program.
We started to see that your sputum cultures started coming back negative
within that first six months of treatment on ARIKAYCE. And so that was a very welcoming sign that we were finally getting the effects, you know, that we had hoped for.
When I found out that my sputum sample was negative after taking ARIKAYCE, I was very excited, and I was very grateful.
You finally got to the point of actually seeing that progress from having
consistently positive cultures to turning to negative cultures.
I appreciate you, Dr. Lapinel. You made sure that I got all of the
proper treatments. You didn’t give up when one of them or three
of them didn’t work, you added to it to make sure that we continue to try to fight this illness.
I appreciate you, the commitment, how seriously you’ve taken this. And just overall being just a wonderful, agreeable person to, to kind
of go with on this journey.
I think that all of the work that we’ve done, all of the treatments that she has recommended and put me on, everything through all of this time has been so successful.
My advice for someone just starting ARIKAYCE is to be positive and take one day at a time. As of today, I’m still MAC-negative.
I feel good about the future.
In the Know Program: Taking ARIKAYCE
MAC lung disease specialist, Dr. Gwenn Huitt and Elisse, a real ARIKAYCE patient, discuss topics such as talking to your doctor about adding ARIKAYCE to your multidrug treatment regimen.
Hi everyone, and welcome to today's program. Thank you for joining us for In The Know: Let's Talk About ARIKAYCE. Today's program will focus on taking ARIKAYCE amikacin liposome inhalation suspension, and you'll hear from a leading physician expert and a patient who's taking ARIKAYCE. So before we begin, I did wanna take a moment to share some important information that you should know about ARIKAYCE. ARIKAYCE is a prescription medicine used to treat adults with refractory, difficult to treat, mycobacterium avium complex, MAC lung disease, as part of a combination antibacterial drug treatment plan or regimen. This product was approved by the FDA using the limited population pathway. This means that the FDA has approved this drug for a limited and a specific patient population, and studies on the drug may have only answered focused questions about its safety and its effectiveness. ARIKAYCE is associated with risk of increased respiratory adverse reactions, including allergic inflammation of lungs, coughing up blood, severe breathing problems, and worsening of COPD.
Now, I would like to introduce Dr. Huitt. She's a physician with 29 years of experience, specializing in Infectious Disease at National Jewish Medical and Research Center and NTM Center of Excellence. She's also a professor in the Department of Medicine, Director of The Adult Infectious Disease unit, chair of the Medical Executive Committee and chair of the Micro-bacterial Consult Service at National Jewish Health in Denver, Colorado. Her research mainly focuses on infectious disease, pneumonia and germ theory of disease. Dr. Huitt is a distinguished physician, having published many articles on NTM. She's also the recipient of many awards, such as America's Top Doctor from Castle Connolly Medical limited. We are delighted and honored to have her here with us today as our expert for this program. Now over to you, Dr. Huitt.
Well thank you, Nicole, I really appreciate the opportunity to participate in this program this afternoon, I'd like to welcome everyone here for this program. I'm Dr. Gwen Huitt and I really look forward to the next hour because I think we're going to really get into NTM MAC lung disease and treatment options for this, including ARIKAYCE. I would also like to introduce today's special guest, Elise, who is an ARIKAYCE patient. And Elise is going to be sharing her thoughts and experiences with us today throughout the course of today's program. And so right now I'd like to turn it over to Elise and say, Elise, why don't you tell us a little bit about yourself to start out with.
Thank you, Dr. Huitt. My name is Elise, I live in Florida was my husband. We have two children, a son and a daughter, and we also have a roofing business, which keeps us quite busy. I'm a cat person, I admit this. [chuckle] All our cats are rescues, to be quite honest. But I'm looking forward to sharing my story with everyone, and thank you for inviting me. Back to you Dr. Huitt.
Great. Thanks so much. And we're gonna be tossing this back and forth throughout the next hour between us as we go through this program, so I'll be back to you shortly.
So let's look at today's agenda for the program, and this is the program focusing on taking ARIKAYCE. And a few things that we're going to cover are an overview of MAC lung disease and treatment, what is ARIKAYCE and where does it fit in, talking to your doctor about ARIKAYCE and maybe some scripting we can talk about, getting started on ARIKAYCE. And in the end, we are going to focus on a brief summary of what we've talked about. So let's delve right into what is MAC lung disease and who might be at risk for this? Well, MAC lung disease is a serious lung disease that can certainly get worse over time and cause lung damage if left untreated, it's caused by a group of bacteria commonly found in the environment, in water and soil, particularly in large numbers in those areas. And MAC is the most common form of non-tuberculous mycobacteria, or we shortened that long word to say NTM. And over 80% of NTM are MAC, the cases of NTM are attributed to MAC.
Well, when we... As a clinician... I'm a physician, and so when I see a patient who I've diagnosed and deemed that they have MAC NTM lung disease, we need to come up with a treatment regimen. And the initial treatment regimen is called a multi-drug treatment approach, and we sometimes refer to it as multi-drug therapy or multi-drug treatment. But it involves taking more than one antibiotic at the same time, and usually it's three oral medications as the initial treatment regimen. And the important thing is, is we always wanna use more than one. So at a minimum two, and usually it's three, sometimes it's more than three. But the reason we use multiple antibiotics at the same time is that we want to prevent the bacteria from developing resistance to any of the antibiotics, so we sort of come at... Try to attack the germ from a variety of directions with each of these different antibiotics that act in slightly different ways, so we can not only attack the bacteria and hopefully kill it, but also at the same time prevent drug resistance from developing. Well, the 2020 NTM treatment guidelines strongly recommend adding ARIKAYCE and specifically...
Together and said, Okay, if we diagnose somebody with MAC lung disease, how is the clinician to approach this, and the initial treatment is with this multi-drug regimen, usually at least three antibiotics after the patient is diagnosed as having true lung disease due to this group of organisms, in addition, we recommended or the physicians recommended that the patients have sputum tests every one to two months, and the purpose of getting the repeated sputum test is to see, are you positive or are you negative on the sputum cultures, and obviously, the ultimate goal is to make you sputum culture negative, and if after taking the multi-drug regimen of the at least the three different medications that you are starting with, if by month six, we have not been able to... On your sputum cultures find that you have attained culture negativity, then the 2020 NTM treatment guidelines recommend adding ARIKAYCE at that six-month time period if you still are continuing to be positive for the MAC bacteria.
And it's important to remember in addition to this recommendation, and many other recommendations, but specifically the guidelines, continue to recommend that a patient must stay on treatment for a minimum of 12 months after testing culture negative on their sputum culture, and this is really just to ensure that the MAC bacteria is cleared from the lungs. Well, so as a patient is taking this multi-drug regimen treatment course, it works in most people, and yet there is a portion or proportion of folks that have been taking all the medicines they were supposed to at least three medicines up to the six month time period and they have been unable to get a sputum culture that is culture negative, so it doesn't really work for everyone, the multi-drug treatment regimen, so it's then that the guidelines strongly recommend the adding ARIKAYCE to this multi-drug regimen. And it's important to say, "We're gonna continue those drugs, we started you on, but the ARIKAYCE is now recommended to be added as an additional drug to that multi-drug regimen if you are continuing to test positive at that six-month time period."
And at this point, it's very important that you start this dialogue with your physician to say, Gosh, I've been doing everything I'm supposed to do, what's the next step? And what's the best way to move forward so that we can achieve the ultimate goal of getting to that 12 months of culture negativity." Well, Elise you kinda walked this walk, so I think everyone would really like the benefit of telling us what your walk down the MAC road initially with diagnosis and then maybe what your doc might have suggested at that point in time.
Thank you Dr. Huitt. For two years, I was going for my annual check-ups to my doctor, and I complained of a cough and it was not debilitating or anything, but it was a bother-some cough and she just ignored it and said it was probably allergies. Year two, same thing, probably allergies. Then I changed doctors. Something wasn't right. So my other doctor, my new one, listened to my lungs, she decided to send me for a chest X-ray where she found that the bottom part of my lungs were damaged probably from pneumonia, I may have had in years before I didn't know about, then she sent me to the pulmonologist who tested me and diagnosed me with the MAC infection. He started out with three antibiotics right away because of the condition of my lungs it was not a wait and see kind of thing. He told me that they may not work, he said that it was very, very hard to... hard to fight infection, it was very persistent and that the medications might not work, but I was willing to take a chance and get started on treatment as soon as possible, I needed to do something. When I finished the treatment, I was okay for a while. Then I started coughing again I went back to the pulmonologist who diagnosed me again with the MAC infection, I was disappointed but I really wasn't surprised. At that point, he added ARIKAYCE to my treatment plan and I was ready to fight again with something new. Thank you, Doctor.
Great, thank you. And I think this story, I must have heard a thousand times in my career, where people start with a cough that maybe slowly progresses over months to years, honestly, before they finally go through treatment options, saying it's allergies, "Oh, let's give you inhalers, let's try this, let's try that," until you finally get a sputum culture and some radiograph, some X-rays or CAT scans and say, "Gosh, this may indeed be NTM MAC lung infections," and then get started on treatment. Well, we'll come back to you in a bit Elise, thanks so much for that input 'cause that's very illustrative. Well, we've been... And part of the program is to talk about not only the multi-drug treatment approach that we do as standard starting place, but what is ARIKAYCE and where might it fit into a treatment plan for MAC lung disease? Well, ARIKAYCE is the first and only FDA-approved treatment option that is designed specifically for adults who do not respond to the multi-drug treatment regimen alone after six months of treatment. ARIKAYCE is an add-on to an existing multi-drug therapy regimen, and I just want to really hone in on that it is not to be taken by itself, it's always to be added on to a baseline treatment regimen.
So if you start with the three drug regimen, this will the fourth drug added into that regimen. And the other thing about ARIKAYCE is that it is an inhaled antibiotic and you take that particular preparation with a nebulizer device, a specific nebulizer device that we're gonna talk about later. Well, how does ARIKAYCE work? Well, it's quite unique in the way it works. Its active ingredient is amikacin, but that amikacin is contained inside tiny particles called liposomes, and when you inhale the ARIKAYCE medication, the liposomes containing the medicine travel into your lungs, and then once the medicine is inside your lungs, the medicine is released from the liposome to fight the bacteria at the site of the infection, which is inside the lung, and on this slide, there's one of these QR codes that you can point your phone camera at and take a picture of, and when the program is done, you can go back to arikayce.com and see exactly how ARIKAYCE works in the lung, and I think that's always illustrative for folks to look at.
Well, ARIKAYCE can cause some serious side effects including allergic inflammation of the lungs, coughing up blood, and that's called hemoptysis in medical jargon, severe breathing problems, worsening of chronic obstructive pulmonary disease, or we shorten that to say COPD, and it can also cause serious allergic reactions. While using ARIKAYCE, these side effects may become serious enough that treatment in a hospital is needed, call your health care provider or get medical help right away if you have any of these serious side effects while taking ARIKAYCE, your health care provider may ask you to stop using ARIKAYCE for a short period of time or completely stop using ARIKAYCE. Do not use ARIKAYCE if you are allergic to any aminoglycoside or any of the ingredients contained in ARIKAYCE.
Well, when any new medication is proposed for use in humans, it has to be studied, and so the clinical study that was the cornerstone study for ARIKAYCE involved 224 patients, and in the study patients received ARIKAYCE plus the multi-drug regimen in 224 patients with MAC lung disease, who either had stopped responding or never responded to their previous treatment. A key goal was culture conversion, which means testing MAC negative by month six, patients continued ARIKAYCE treatment for 12 months after testing MAC negative. Additional measures evaluated in the ARIKAYCE study included improvement in the distance walked after six minutes, as well as improvement in clinical health, daily life, as well as perceived well-being in patients with lung disease. Well, this is really, really important. So adding ARIKAYCE help patients test MAC negative for 12 months and beyond, and as you can see from this graph, the purple... dark purple bar is the group that had ARIKAYCE plus the multi-drug regimen, and the gray bar is the multi-drug regimen alone without ARIKAYCE, and what you see is a ARIKAYCE helped three times as many patients test MAC negative at six months, than those who were just in the multi-drug regimen alone. ARIKAYCE helped seven times as many patients test MAC negative at 12 months as well.
And additionally, only those patients that were on ARIKAYCE tested MAC negative three months after completing the treatment. Additionally, for those who successfully tested MAC negative ARIKAYCE helped more patients stay culture negative ARIKAYCE helped two times as many patients test MAC negative at 12 months, and that would be in the purple bar as well. Only people taking the ARIKAYCE tested MAC negative at three months after completing treatment, and lastly, only people taking ARIKAYCE tested MAC negative at 12 months after completing treatment, so you see this had a durable effect going forward when the ARIKAYCE was included in the MAC regimen that had the multi-drug regimen plus the ARIKAYCE. Additional study results that were looked at in this ARIKAYCE clinical study show that there was no improvement in the six-minute walk test and the St. George's Respiratory Questionnaire measurements at the end of six months.
Well, now, it's really important for patients to feel empowered so that they have areas that they can get more information about any product, let alone ARIKAYCE. So fortunately, ARIKAYCE does have a program that you can learn more about it, and it's called The ARIKAYCE Knowledge Moves Program, and this program provides information, educational resources, patient stories, and tips on how to have conversations with your physician or health care practitioner should ARIKAYCE be entertained as a treatment option for your MAC lung disease. And you can learn more about the ARIKAYCE Knowledge Moves Program by going to arikayce.com to enroll. Elise, I'd like to hear what your thoughts were and what your experience with ARIKAYCE was once you guys started on it?
Thank you, Doctor. Well, when I went to my pulmonologist the second time, and he told me he had the bad news and the good news, and I knew what the bad news was, so I said, "Just skip to the good news, I know what the bad news is already." He told me that the good news was that there was a medication now called ARIKAYCE that would take the antibiotic straight to my lungs through a nebulizer. I was thrilled, I was hopeful, I was all on board and ready to start the fight with ARIKAYCE now, he said it was made for people just like myself who had already fought MAC infection and unsuccessfully. So when I got the study results, I was very impressed, I was very encouraged, and when I got my first negative sputum test after starting the ARIKAYCE program, I was thrilled I thought, this is great. So far so good. Thank you, doctor.
Well, thanks so much. And each time you're sharing these little snippets of your journey with us, I think this is probably gonna sound very familiar to many people at least, depending what part of the journey that they're on. But hearing it straight from a patient who's been interacting with their physician about this is really important, and hopefully physicians will be able to view this program as well, so they can learn from patients about the best way to interact with the patient when we're talking about ARIKAYCE and how to move forward.
So in that arena, I think it's important to empower our patients as to how do you broach the subject about, "Gosh, doc, I've been taking these medicines and three drugs for six months and I haven't converted my sputum to negative yet. I heard about this drug called ARIKAYCE, and how can we have that discussion to see whether I'm a candidate for this medication, and should it be added to my regimen?" So one of the things that I think is really important that patients need to know is you need to know, "How will I know if the treatment with all the medications is working after six months?" And that is generally several different ways, but for sure, we're going to get ongoing sputum cultures every one to two months, because again, we're watching...
That clock is ticking. Are we getting to culture negativity? And any of you who have MAC lung disease know that these cultures, the sputum cultures, take anywhere from six to eight weeks to get back, so you're always just waiting for these cultures to come back. And I think that must be one of the hardest things is to... It seems like... It's just you're in the waiting game all the time, but unfortunately the germs grow very slowly, so that's what we're left with right now. But if the multi-drug treatment is or isn't working after six months, perhaps we need to think about ARIKAYCE if it isn't working, and is ARIKAYCE right for me? And if I start taking ARIKAYCE, how will I know if I'm getting better? Is my cough gonna go away? Am I gonna produce less sputum? Just what are we expecting to see that might change? Next, how long is the treatment going to be? How many months or weeks am I gonna be taking ARIKAYCE, and along that course, how are we gonna track that? And again, our goal... Remember the 2020 NTM guidelines instruct us that we're really shooting for 12 months of culture negativity on your sputums.
And how do we track our progress? Well, obviously sputum cultures is the first and foremost thing. As the patient, you certainly need to ask us, "Well, gosh, if I'm taking any medicine, ARIKAYCE or anything else, what should I be aware of? What problems have you heard that might occur with this medication? What should I be looking for?" So ARIKAYCE may cause serious side effects, including hearing loss or ringing in the ears, and we call that ototoxicity, worsening kidney problems, and we call that nephrotoxicity, as well as worsening muscle weakness or something called neuromuscular blockade. So certainly you and your physicians need to be aware of these particular side effects. In the clinical study that was done, 19.7% of patients taking ARIKAYCE plus a multi-drug regimen experienced serious side effects compared to 16.1% of patients taking only the multi-drug regimen alone. The most common side effects of ARIKAYCE are listed, but cough during or after a dose of ARIKAYCE was reported in most patients during the first month of treatment. And I would say I certainly have experienced that with my patients and interacting with them, when they have been taking ARIKAYCE.
These are not all of the possible side effects associated with ARIKAYCE. Be sure to call your doctor or pharmacist for medical advice about side effects. You may report side effects to the FDA at 1-800-332-1088. Also tell your healthcare provider about all the medicines you take. This is huge, and I tell all my patients to always have an up-to-date list of the medicines you take, including prescription medicines, as well as over-the-counter medicines and really, for sure, vitamins and herbal supplements, because we don't know some of the interactions between medicine, so we need a comprehensive list of all the medications you're taking. And keep a list of the medicines you take and show it to your physician each time you meet with them and/or the pharmacist when you get a new medication. This is really, really important. Back to that clinical study. Most patients who experienced side effects did not stop treatment, so they were easily manageable. 81% of the patients taking ARIKAYCE plus the multi-drug regimen did not stop treatment, even though they might have had at least one side effect.
Two academic centers did a study via phone with 26 patients that had been prescribed ARIKAYCE during a two-month period, and this was in the United States, and this... Insmed was not involved with the conceptualization, development, conduct, or analysis of this study, and it's not included in the ARIKAYCE full prescribing information. But they did provide writing assistance when the authors were publishing this through a grant through Insmed Incorporated. Well, this slide is a compilation of four of the major side effects that people commonly complain about in management strategies that we might use to help alleviate any of these symptoms. So in the first column it talks about increased cough, the next column is listed as dysphonia, and basically that just means a change in your voice or change in your voice quality.
The next column is dyspnea, and that just as a fancy word for shortness of breath, and then the last one is showing increased sputum production. And so starting in column number one, there are a lot of choices of management strategies that you and your doctor can go over, and they're listed on the left, but it might be using a bronchodilator or the puffer medicine that you may or may not already be on, sometimes just changing the ARIKAYCE administration from morning to evening will help decrease cough 'cause it lets your vocal cords rest a little bit. You might talk with your doctor about the coughing or change in your voice and your doctor may say, "Well, let's hold off on your ARIKAYCE for a few days and see if this improves just by holding the ARIKAYCE."
Additionally, you can take some cough medicines or throat lozenges, or do some warm water, or warm salt water gargles. And those may be quite beneficial, something just as simple as drinking some warm tea or warm water may help either the increased cough or voice changes. When it gets to the column of dyspnea, or shortness of breath, we may need to use some additional things, we still may need to go back to using that bronchodilator medicine or puffer medicine, and additionally, we may need to stop the ARIKAYCE for a little while, but yeah, sometimes it takes limiting your physical activity, so slowing down a little bit for a couple of days, maybe not running, or having a really robust exercise regimen for a couple of days, and lastly, if you are already on supplemental oxygen, you may need to increase your oxygen use, but again, you're going to be discussing all these issues with your physician and going through with them what your symptoms are, so they can work and partner with you about the best approach to handle any of these certain side effects that might be appearing.
Well, if the treatment plan is established, and you and your doc have talked about your goals and how you're gonna track your progress, you need to establish your new treatment plan that includes ARIKAYCE, and as we've just alluded to in the prior slides, we're going to ask the question, "Well, doc how long do you think I'm gonna be taking ARIKAYCE so that I can feel prepared for what's ahead of me?" Understand your goals of treatment, so you know how quickly you may start to see results, and again, "How frequently am I gonna be checking my sputum cultures with you? How often am I gonna be seeing you in clinic?" Things such as that, and then asking your doc how are you gonna track your progress? And if you have questions that are coming up along the way, "Do you have a patient portal access that I can talk to you or do I go through the nurse in the office or just the best way that I can get a hold of you if I'm experiencing any side effects or benefits for that matter, and I need to communicate with you?"
And then for sure, you're gonna wanna keep track of any side effects that you are experiencing, and what I suggest to everyone is make sure that your time and your date, when said side effect or a thing that we're trying to figure out what might be going on. Give me a time and a date and where was it in relationship to when you might have taken any medication. So keep track of what time you take all your meds on, and not only just a new medicine that might be added to your regimen. Well, so back to Elise, can you share with us how you and your doctor strategized regarding some of these issues?
Sure doctor. I discussed with my doctor the issues of the side effects, and it was quite a conversation because there was a lot to speak about. But when I got my ARIKAYCE materials in the mail, that answered really, all my questions, everything was answered. They answered questions I didn't even think I had, because I didn't understand anything about it to begin with, so it was very, very informative. After reading everything, I was willing to take a chance. I went ahead and got started. After a while, I did get the hoarseness for a while, and I did lose my voice at one point for two days, and then at another point for five days. But everybody's different, everybody has different reactions to every different medication. So if something is going on, then you need to really call your doctor and speak to them about that directly. That's what I did, I called my doctor with questions about anything I didn't understand or anything I was experiencing, and they gave me advice. So that's how I handled it. Thank you.
I think what you've just said is so very important, and I think that if I hear a complaint from patients is, "Doc, I can never get a hold of you in a timely fashion," and so I think as important for physicians to know about new medications that we might be using in treatment plans for our patients that we educate our staff, honestly, our nursing staff, our medical assistants to the best of their ability to... Their knowledge base, because they can actually be very helpful to you, and they're hugely important to us, physicians as extensions of helping our patients manage any kind of side effects that they might be having, so I think establishing, how am I gonna get a hold of you in a timely fashion and get some feedback from you or your office staff regarding my complaints or concerns even, is hugely important, and I think that will give you success for sure. And so it sounds like you had a very good interaction with your physician and office staff to help you manage these speed bumps, as I call them.
Well, now let's move to getting started on ARIKAYCE. So as I mentioned earlier, it's an inhaled antibiotic, and it's taken once daily with a nebulizer that you can see illustrated in the slide. As the patient who's taking this medication, you are going to be responsible for cleaning and disinfecting your Lamira handset and aerosol head before using them for the first time, as well as after every time that you use them. You're going to administer this medication once a day in your own home or, quite honestly, anywhere that has a clean, flat surface. The ARIKAYCE administration time is about 14-20 minutes, depending on your ability to take a deep breath, and you're going to inhale once daily dose of ARIKAYCE around the same time each day. And I think this is important for the physician and the staff who is gonna be helping you on this journey, it's important for us to know that you're taking the ARIKAYCE at the same time each day, so that we can try to pinpoint a particular issue, how it may be related to ARIKAYCE, or maybe related to something entirely different. So if you can stay consistent on when you take the ARIKAYCE, that's very helpful for your physician. And keep the ARIKAYCE, of course, as well as all medicines, out of the reach of children.
Well, what should you expect once a decision is made to start you on ARIKAYCE as part of your antibiotic regimen? Well, when it's time for your shipment to arrive, you're gonna be contacted by your specialty pharmacy to schedule your shipment, and you will need to be home to sign for the shipment so that's very, very important. And you also may need some assistance handling these boxes, which may be heavy. At the start of your treatment, you're gonna receive two shipments from the specialty pharmacy, and that's gonna include the first 28-day supply of ARIKAYCE as well as the one-time shipment of the Lamira nebulizer system. And then going forward, you're gonna receive monthly shipments of the 28-day supply of ARIKAYCE, and that's illustrated on this slide.
Well, there is another support program associated with ARIKAYCE, and it's called the Arikares Support Program, and once you're prescribed ARIKAYCE, the Arikares Support Program provides additional information and support, and you will be assigned a care team dedicated to helping you along this journey. And I can just say from personal experience, every one of my patients who have started on ARIKAYCE have nothing but positive things to say about the Arikares Support Program, 'cause they help them navigate the insurance issues as well as... Just every step along the way. They're there to help you. And so the number to contact to get started in this program is 1-833-ARIKARE, A-R-I-K-A-R-E, and there's somebody there to help you start the enrollment process over the phone. And once you're enrolled in the Arikares Support Program, you're then going to be assigned your Arikares coordinator, your point person, to help you with your journey and to answer questions that you have as you proceed with starting to take ARIKAYCE.
So they're going to help schedule an optional device training with your Arikares trainer, and I would say most people really benefit from this as well. They will work with your specialty pharmacy to verify your insurance coverage, as well as coordinate medication shipments with your specialty pharmacy. They also serve as your primary point of contact throughout your treatment journey, and they provide information about potential savings and financial support options, which is hugely important to most patients as well. Once you are enrolled, you can request an Arikares trainer, and your Arikares trainer is a trained nurse or a therapist who will conduct an in-home training session to show you how to take your first dose of medication and make it part of your daily routine. They will also provide voluntary in-home or virtual device training, and this is important in the time of COVID, I must say. They will also provide ongoing clinical support and information throughout your treatment journey.
Well, a lot of folks that I've seen over the years are always searching for ways to connect with other people who have MAC lung disease, and there are three groups and sites here that you can certainly avail yourself of, NTMir, BRONK, and NTM-360 social, as well as Inspire, and all of these three social supports are ways to connect with other people who are going through the same journey, or certainly a similar journey that you are. Arikayce.com also provides a variety of resources that are listed here, such as important questions that you will need to ask your doctor about ARIKAYCE and how to make the most out of your next appointment. So you can write your questions down and just go bang, bang, bang through your questions with your doc. Tools to help locate a doctor who specializes in lung disease, if you don't have a pulmonologist or a pulmonologist who knows much about MAC lung disease.
They also have real patient and physician stories about MAC lung disease as well as ARIKAYCE, and a printable brochure that provides an overview of ARIKAYCE so you can read and reread it. Also they provide you with ways to connect with others who have MAC lung disease through online communities and support groups. And lastly, they provide information about how to take ARIKAYCE, including the important how-to video, and all these resources are available on arikayce.com. Well, Elise, you, once again, went through this journey and did you avail yourself of any of these support groups or information resources, and did you have any issues with receipt of ARIKAYCE or anything of that nature?
I absolutely enrolled in the Arikares Support Program. It was great. They answered questions that I had along the way. I had a delivery problem once. They got that resolved for me with great detail. It was far above and beyond what I expected. They had answered any questions that I had about the equipment or the delivery or the prescription. It was wonderful. I had someone in my corner to help me every step along the way, and I would suggest it to anybody that is undergoing this treatment. The online support also was very good, 'cause you can connect with other patients. No one else knows what you're going through except for someone else that also has it. And it's not in your everyday life, in my case anyway, that I've ever found anybody else that has this. So it's very important to connect with other people, to give them tips on how to navigate things and to give tips on how to navigate things and to just get some support and a backup. So it was great. Thank you.
Well, I think that patient support groups and any information that a patient can avail themselves of is only beneficial. Now, obviously, the information is only as good as it can be, and I think that's one of the negatives perhaps about some support groups, because it's important to know that everybody's MAC lung disease disease may be different, little differences from one person to the next person. People may have a set of side effects. One person may have a set of side effects, but the other person has no problems, or they may have coexisting medical conditions. So it's very important for every patient to remember you are a unique individual, and it's very important that you have this ongoing relationship with your physician as you go on this road. And these extra perks, as I call them, with the information access are just that, and you should definitely participate in those. But understand that each one of us is very unique in the disease and our journey treatments for the disease. So let's summarize what we've just talked about here. So MAC lung disease is a serious condition. It can certainly get worse over time, and this is generally over months to years, not days or weeks. The standard approach to treating MAC is with multiple antibiotics.
So again, we're trying to get at that germ from different directions and try to kill it, coming at it from several different directions, but at the same time, we're giving multiple drugs because we're trying to prevent antibiotic resistance from developing. If a patient still tests positive for MAC after six months of taking the initial multi-drug treatment, the 2020 NTM treatment guidelines strongly recommend adding ARIKAYCE to their regimen. And ARIKAYCE is the first and only FDA-approved treatment option that's designed specifically for adults who do not respond to a multi-drug treatment regimen alone after six months. The inhaled liposomal formulation of ARIKAYCE delivers the medication directly to the lungs at the site where the MAC bacteria infection is. Adding ARIKAYCE is proven to help patients test MAC negative for 12 months and beyond. ARIKAYCE is associated with risk of increasing respiratory adverse reactions, including allergic inflammation of the lungs, coughing up blood, severe breathing problems and worsening of COPD. It's very important to talk to your doctor about establishing an ARIKAYCE treatment plan and planning for any side effects. Once prescribed ARIKAYCE, the Arikares Support Program provides additional information and support. Visit arikayce.com for more information and support.
Well, now that we've come through the summary, Elise, what type of closing thoughts might you have to anyone who's out there and thinking, "Gosh, maybe ARIKAYCE is something I need to discuss with my physician, or I've already made a decision, and I'm getting ready to start, or I've already started?"
Well, in my case, I think that if I was diagnosed earlier, it would have been very helpful. I would have started treatment earlier. Maybe the disease wouldn't have gone to both my lungs or been as prolific as it became. So I would suggest to people to be advocates for themselves. To advocate to your doctor, push them and get the answers to your questions and repeat the questions until you get the answers you need. And when you do start treatment, don't skip. Be persistent. Put it on a schedule. Do it the same time every day. It's much easier that way. That way, you don't skip and you... It's in your routine. That's very important, I believe. For me, I would do it after dinner or before bed, in that time period there. And if you're not getting results after six months of the multi-drug treatment, I would suggest for anyone to ask their doctor about ARIKAYCE. Thank you.
Thank you, Elise. And I tell my folks, the squeaking wheel does get the grease. And so your point of being your own advocate is huge, and that's certainly one of the main reasons forums like this are out there, to allow patients to be the squeaky wheel and get the grease, which is the answers that are certainly out there. Whether it's a patient support group or directly from your physician's office, your physician themselves or the staff in the physician's office, we need to empower you to be able to move forward in your treatment of MAC lung disease. So thanks so much for sharing your journey with us 'cause it's so important. So I guess this closes the formal presentation, and I guess I'm gonna turn it back to our moderator right now.
So this concludes our presentation this evening. Thank you again so much to our special guests, and for all of you who joined us today. We have additional programs coming up in the future so we hope to see you again. Thank you.
- See tips to establish a routine and fit ARIKAYCE into your daily life
- Use this doctor’s visit checklist to prepare for a discussion about your ARIKAYCE treatment