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Treatment Survival Guide for AL Amyloidosis

The treatment for AL Amyloidosis varies and can include chemotherapy, a stem cell transplant, or immunotherapy. Each of these treatments comes with its own set of issues and side effects. If you are newly diagnosed and just beginning one of these treatments, it can be a scary and difficult time. The uncertainty of it all can be overwhelming. If you’re worried about what you’re about to face, here are some tips and tricks from patients who have been there. Hopefully, this treatment survival guide can help make the whole process just a little bit easier for you.

Preparing for treatment and having a plan in place (even if things don’t go according to that plan) can help the whole process go a little bit more smoothly. Having an idea of what to expect in terms of side effects helps you recognize and treat them immediately. Learning how to deal with the mental stresses can be as important as dealing with physical stressors. And living your healthiest lifestyle can help make the treatment as successful as possible.

 

Preparing for treatment

  • Ask your medical care team about what side effects you can expect, and what medications you can use to control them (such as anti-nausea medication, or mouthwash for mouth sores). Having a plan in place means you can treat symptoms early; knowing what to expect will lessen the shock of facing these side effects.
  • Create a support system: family, friends, doctors, homecare, neighbors, join a support group. This is a time to ask for help.
  • Make a list of things that family and friends can do to help you out. They can be a great supportive resource but often don’t know what to do. Ask them to walk the dog, pick up the kids, or drop off dinner.
  • Ask for help in the kitchen and plan for meals. Make and freeze your favorites so you don’t have to cook, and stock up on foods that are easy to prepare.
  • Pack a bag to bring with you to treatments. Include things such as snacks, water, books, headphones, mints or gum. Click here for a more complete list.
  • See your dentist before you start treatment (time permitting of course).
  • Do some research on what organizations are out there to help you. There are many reliable sources of information that can help answer your questions.  Check out this resource page from Mackenzie’s Mission for suggestions.
  • Seek out services that can give you some back up. Whether it is a meal delivery service, a cleaning company, or a car service to get you to your appointments, there are options out there to make your life easier. These will vary depending on your location, so ask around to find services close to you.

 

Dealing with side effects

  • Communicate openly with your health care team. Don’t suffer in silence. Be sure to report any side effects you face.
  • Keep a notebook to record any problems you have and when they occur. It can be difficult to remember all that happens between appointments.
  • Take your medication as directed to lessen any side effects. Timing can be important here, as is the case of anti-nausea meds, so follow directions closely.
  • Ask around! If you haven’t joined already, support groups are filled with people who have gone through the same or similar treatments; they may have suggestions as to what helped them deal with side effects. But be sure to check with your doctors before trying anything new.

 

Coping with the stress of it all

  • Battling physical symptoms seems obvious, but there is also a mental game involved. Keeping your spirits up through treatment is important but not always easy.
  • Many complicated emotions will come up during the treatment process: anxiety, grief, anger, denial, guilt. You don’t have to be strong 100% of the time. Talk about these feelings with a friend, family member, your doctor, or a therapist.
  • Create something that lifts your spirits, like a photo album of loved ones, an inspirational board, or a journal of your favorite quotes. Keep it close at hand for those tough times.
  • Focus on the things you can do, rather than the things you had to give up. Pick up some new low energy hobbies to help keep you occupied and your mind off your troubles.
  • Remember you are not alone. There are a lot of survivors out there willing to share their story, especially if it helps to make the life of someone newly diagnosed just a little bit easier.
  • Schedule time for fun! Make sure you spend some time doing things you enjoy. Now is the time to treat yourself well.

Living your healthiest lifestyle  

  • Ask your healthcare team what exercise is best for you. There may be times when it’s just not a possibility, but if you can get outside and take a short walk, do it! Just don’t push yourself too hard.
  • You will probably face a few dietary restrictions. Common ones are low sodium and fluid restriction, as well as avoidance of certain foods that can either conflict with medication or irritate an already sensitive stomach. Ask if your hospital has a dietician you can speak with who can create a plan tailored to your needs.
  • Follow those doctor’s orders! And communicate openly with your healthcare team. Keeping a journal of your treatment plan, medications you take, and side effects you experience, will help you keep it all straight.
  • Do what you can to prevent the spread of virus and infection: practice food safety, wash your hands, wear a mask, don’t visit with anyone who has a cold or virus.
  • Get plenty of rest. Try not to push yourself too hard. There will be times you’ll get frustrated with your limitations. Remember to give yourself a break, take it easy on yourself; you’ve got a lot going on.

 

Recognize that some days will be harder than others. Sometimes the best you’ll be able to do is to just breathe and get through the day.

Remember to reach out if you’re having a rough time. Sometimes just hearing that someone else has walked a similar path and come out the other side can give you the motivation you need to put one foot in front of the other and just keep going.

 

 

Lori Grover is a guest blogger for Mackenzie’s Mission. She was diagnosed with AL Amyloidosis in 2016 and writes to share experiences and lessons learned during her journey.  More wonderful blogs by Lori can be found on her page Amyloid Assassin.  When not writing, she is mostly a stay at home mom, florist, crafter, lover of books and food.

AL Amyloidosis: Age at Diagnosis Survey

Is the age at diagnosis of AL Amyloidosis changing?

According to a paper published in 1995 based on a study of 474 AL Amyloidosis patients, the median age of diagnosis for patients was 64 years.Being curious as to whether this number has been lowering since then, we went to the Amyloidosis patient community to conduct an informal survey in early 2019.

We heard from 251 wonderful patients from the Facebook Amyloidosis Support Group in response to our question as to how old they were when they received their diagnosis.

Here is what we learned.

 

  • 80% of patients were diagnosed between the ages of 40-69.
  • The largest group at 35% of patients surveyed, were diagnosed in their fifties.
  • The second largest group, at 24% of patients surveyed, were diagnosed in their sixties.
  • The third largest group, at 20% of patients surveyed, were diagnosed in their forties.
  • Just over 10% of patients were diagnosed before the age of 40.
  • The smallest group, at just over 8% of patients surveyed, were diagnosed over the age of 70.

We then organized the data by age to determine the occurrence rate by age range.

In answer to our question about the age at time of diagnosis, per our informal online survey of 251 AL Amyloid patients, the data indicated a mean (or average) age of 54.8 years and a median age of 56. The “mean” is the “average,” where you add up all the numbers and then divide by the number of numbers. The “median” is the “middle” value in the list of numbers. While not possible to definitively conclude, we acknowledge there may some age bias in our results towards younger ages due to the source being social media, and inaccuracies due to data being self-reported.

During the course of our research, we discovered other patient studies which we found informative. In 2015, an AL Amyloidosis patient experience survey was conducted online with 533 respondents.2  Their results indicated a median age at diagnosis of 57. A more recent study in 2017 of 341 AL Amyloidosis patients from across the U.S. found the average age to be 60 years.

We also posed the question “Do you think the median age upon diagnosis is lowering?” to doctors at some of the leading Amyloidosis centers in North America. Each of these doctors confirmed that the median is in the range of 57-65. However, one noted that “they are seeing far too many women in their 30’s and 40’s” in clinic. Another noted that they do seem to have an increase of younger patients, pointing out that this may indicate that the disease is being diagnosed in its earlier stages.

While ours was an informal online survey, the results are interesting and worth watching going forward. But we can all agree that this is what we want – that over time the age at diagnosis will lower. Not because the disease is spreading to a younger generation, but because it is being diagnosed in its earlier stages.

It is common for many Amyloid patients to suffer for years before they receive their diagnosis, delaying treatment while the disease progresses. If we can continue to spread awareness of the early warning signs to ensure that doctors across a multitude of disciplines have the latest information and diagnostic tools, hopefully, Amyloidosis will increasingly be diagnosed in its earlier stages. By doing so, treatment can begin sooner and, we believe, better patient outcomes can be achieved.

Mackenzie

 

 

 

 

 

A big shout out to Lori Grover for her collaboration on this piece. Lori is a guest blogger for Mackenzie’s Mission. She was diagnosed with AL Amyloidosis in 2016 and writes to share experiences and lessons learned during her journey.  More wonderful blogs by Lori can be found on her page Amyloid Assassin.  When not writing, she is mostly a stay at home mom, florist, crafter, lover of books and food.

 

(1) Kyle RA, Gertz MA. Primary systemic amyloidosis: clinical and laboratory features in 474 cases. Semin Hematol. 1995;32:45–59. [PubMed]

(2) Lousada I, Comenzo RL, Landau H, Guthrie S, Merlini G. Light Chain Amyloidosis: Patient Experience Survey from the Amyloidosis Research Consortium. Advances in Therapy. 2015;32(10):920-928. doi:10.1007/s12325-015-0250-0.

 (3) McCausland KL, White MK, Guthrie SD, et al. Light Chain (AL) Amyloidosis: The Journey to Diagnosis. Patient. 2017;11(2):207-216.

DOCTORS of Amyloidosis

Twelve of the most notable experts in the fight against this disease share, in their own unedited words, their views on the state of the disease. They voice what patients and the medical community need to do to push forward, and what lies ahead in the pipeline of potential treatment.

This unparalleled collection of messages from leading experts is a priceless read to understand the disease both today, and tomorrow.

We thank them for their words, and the passion and care they bring to their patients, in the fight against amyloidosis.

Thank you for taking the time to watch and read their stories.

Mackenzie

P.S. You can view the video, or for those preferring a larger font for easier reading, we have provided a transcript for download as well.

DOCTORS of Amyloidosis transcript (download)

 

 

 

 

VOICES of Amyloidosis

According to the Mayo Clinic, Amyloidosis is a rare disease that occurs when a substance called amyloid builds up in your organs. Amyloid is an abnormal protein that is produced in your bone marrow and can be deposited in any tissue or organ. Amyloidosis can affect different organs in different people, and there are different types of amyloid. This disease frequently affects the heart, kidneys, liver, spleen, nervous system and digestive tract. Severe amyloidosis can lead to life-threatening organ failure.

VOICES of Amyloidosis is a five-part campaign intended to give voice to those impacted by the disease. Each person in VOICES, which includes patients, caregivers, and expert doctors, has a story to tell in their own unedited words. You will feel their emotions and share their hope for the future.

Thank you for reading and sharing far and wide, as the more people that know about this disease, the better the odds are to advance the timing of diagnosis, find answers for improved treatments, and ultimately develop a cure.

This is their message.

Please scroll down to see each of the five videos.

Part 1 of 5

Part 2 of 5

 

Part 3 of 5

Part 4 of 5

 

Part 5 of 5

 

2018: Thanks for a Great Year!

Thank you for your support in 2018!

Let me begin by saying THANK YOU for supporting Mackenzie’s Mission last year.

You may have donated cash or an auction item, bought a sponsorship, participated in one of our raising awareness campaigns, or played in our Play FORE The Cure charity golf tournament. You may have donated your time volunteering for the tournament, Liked/Shared our Facebook posts, or taken the time to read our blogs to learn about amyloidosis. Whether you did one of these or many, you helped us push forward our fight against this disease and we thank you.

How Did We Do In 2018?

This was our first full year of operation, busy and loaded with lots of activities to advance our mission — to make a difference in the fight against Amyloidosis. We worked to make a difference in two ways.

  • Raise awareness about Amyloidosis, which can lead to earlier diagnosis and better outcomes.
  • Support medical research on Amyloidosis, seeking the cause of the disease and more effective treatments to improve and extend lives.

Raising Awareness

The feedback I have received from this outreach has been both touching and heartwarming. While being an inspiration to others is wonderful, I am most moved by the conversations I have had with others affected by this disease. We share a common bond with uncommon experiences. Knowing we are not alone in this fight gives us all strength.

Supporting Medical Research

As I have said over and over, nothing happens in research without money. And knowing the NIH currently funds only 11% of its applications, this leaves a heavy burden on private foundations and individuals to help close the shortfall gap. So our work to raise money matters.

  • Held our first annual Play FORE The Cure golf tournament
  • Raised Over $164,000!  Donated 100% of all donations and net tournament proceeds to the Mayo Clinic’s Amyloidosis Research Fund. According to Dr. Morie Gertz at the Mayo Clinic, our monies go towards activities including:
    • Outcomes research.
    • Clinical trials research.
    • Valuable tissue repository and cell bank, where serum and cells from thousands of amyloid patients can be used for research as new understanding comes forward.
    • Sixteen (16) research publications from Mayo’s amyloid research group published in 2018.
    • Supporting one clinical trial coordinator and one research fellow for one year.

What Are Our Goals For 2019?

Raising Awareness

  • Launch VOICES of Amyloidosis, our second major campaign. This project, with submission from patients, caregivers, and expert physicians, will be a series of videos conveying what they want the public to know about amyloidosis and how this disease has affected their life. VOICES of Amyloidosis promises to be moving and informational.
  • Continue to publish educational blogs and amyloidosis news.
  • Pursue speaking opportunities, both large and small, spreading the word on the importance of early diagnosis.

Supporting Medical Research

  • On September 9, 2019 we will sponsor our second annual Play FORE The Cure charity golf tournament at the prestigious Robert Trent Jones Golf Club in the Washington D.C. area.  Mark your calendars and come join us!
  • Raise $175,000. We hope we can count on your continued support!
  • Donate 100% of our donations and net tournament proceeds to the Mayo Clinic’s Amyloidosis Research Fund, one of the worldwide leaders in research to understand the disease, develop improved therapies to treat and potentially reverse the damage of the disease, and ultimately find a cure. We are fortunate to have a benefactor paying 100% of our operational expenses, which enables us to maximize your donated dollars.

I am encouraged by the impact Mackenzie’s Mission is already making. There is much work to be done, but with your help I know we can win this fight!

 

An Update On Me

In December 2018, I moved back to the Washington D.C. area after finishing a nearly two-year term as a research associate at Harvard Medical School. I learned an incredible amount about scientific research, its value, and the translation of the work back into the clinic. It has continued to energize me as I prepare to apply to medical school in the coming year. While in Northern Virginia, I shadow an orthopedic trauma surgeon at Inova Fairfax Hospital, where I’ve been shadowing on and off for over six years. In my spare time, I continue coaching youth hockey, something I have come to truly love, and volunteer as an assistant coach for a U14 girls ice hockey team. I continue to feel great and remain disease free.

With warm regards for a wonderful 2019,

Mackenzie

 

Clinical Trials 101

Clinical research is simply medical research involving people. There are two types, clinical studies (aka observational studies) and clinical trials. In this blog, we explore clinical trials and the basics of what you need to know.

 

WHAT ARE CLINICAL TRIALS?

According to the National Institutes of Health (NIH), clinical trials are research studies performed on people that are aimed at evaluating a medical, surgical, or behavioral intervention. Clinical trials are the primary way that researchers find out if a new treatment, like a new drug or medical device (e.g., a pacemaker) is safe and effective in people. Often a clinical trial is used to learn if a new treatment is more effective and/or has less harmful side effects than the standard treatment. Other clinical trials test ways to find a disease early, sometimes before there are symptoms. Still, others test ways to prevent a health problem before it begins. A clinical trial may also look at how to make life better for people living with a life-threatening disease or a chronic health problem.

 

WHY CLINICAL TRIALS ARE IMPORTANT

Clinical trials permit researchers to test the safety and effectiveness of new therapies. They also allow for a rigorous evaluation through patient participation. Bottom line: it is only after the extensive evaluation and testing from a clinical trial that the FDA will approve the widespread use of any new therapy.

According to Dr. Morie A. Gertz at the Mayo Clinic:

Advancing the medical care for all patients requires participation in clinical trials. Only through clinical trials can we further improve the available therapy options for current and all future patients. Clinical trials seek to answer questions about the natural history and biology of the disease as well as important questions regarding outcomes was all available new therapies. All current treatments received by our amyloid community were derived through others participation in clinical trials. Clinical trials are not only an opportunity to get the cutting edge therapy but is a way to “pay it forward“ for future generations of Patients.

 

WHAT ARE THE PHASES OF CLINICAL TRIALS?

All clinical trials must be approved by the U.S. Food and Drug Administration (FDA) before they can begin. Prior to that decision, scientists perform laboratory tests and studies in animals to test a potential therapy’s safety and efficacy. Assuming favorable outcomes, the FDA then gives approval for a clinical trial involving humans.

Clinical trials are comprised of four phases to test a treatment, find appropriate dosages, and detect side effects. If following the completion of the first three phases, researchers find the drug or intervention to be safe and effective, the FDA approves it for clinical use and continues to monitor its effects. Overall, the duration of a clinical trial spans years.

  • Phase I trial tests an experimental treatment on a small group of often healthy people (20 to 80) to judge its safety and side effects and to find the correct drug dosage.
  • Phase II trial uses more people (100 to 300). While the emphasis in Phase I is on safety, the emphasis in Phase II is on effectiveness. This phase aims to obtain preliminary data on whether the drug works in people who have a certain disease or condition. These trials also continue to study safety, including short-term side effects. This phase can last several years.
  • Phase III trial gathers more information about safety and effectiveness, studying different populations and different dosages, using the drug in combination with other drugs. The number of subjects usually ranges from several hundred to about 3,000 people. If the FDA agrees that the trial results are positive, it will approve the experimental drug or device.
  • Phase IV trial for drugs or devices takes place after the FDA approves their use. A device or drug’s effectiveness and safety are monitored in large, diverse populations. Sometimes, the side effects of a drug may not become clear until more people have taken it over a longer period of time.

 

WHY PEOPLE CHOOSE TO JOIN A CLINICAL TRIAL

There are many reasons why people choose to join a clinical trial. Some join a trial because the treatments they have tried for their health problem did not work. Others participate because there is no treatment for their health problem. Some studies are designed for, or include, people who are healthy but want to help find ways to prevent a disease that may be common in their family. By being part of a clinical trial, participants may access new treatments before they are widely available. Especially with a rare disease such as amyloidosis, clinical trials may offer a meaningful impact to a patient’s quality of life.

In addition, people may feel that participating in a clinical trial allows them to play a more active role in their own health care. Participants may receive more frequent health check-ups and closer monitoring through the clinical trial. Other people say they want to help researchers learn more about certain health problems. Whatever the motivation, when choosing to participate in a clinical trial, one becomes a partner in scientific discovery. This can also help future generations lead healthier lives. Major medical breakthroughs could not happen without the generosity of clinical trial participants—young and old.

In the words of Dr. Vaishali Sanchorawala at The Amyloidosis Center at Boston University School of Medicine and Boston Medical Center:

Clinical trials allow researchers and physicians to test the safety and effectiveness of new, promising drugs. Before any drug can be approved, it must be rigorously tested in clinical trials. Without the participation of patients, new treatments and cures will never happen. In addition, participating in a clinical trial may be a great way for patients to access new treatments before they become available. Especially in a rare disease such as amyloidosis, clinical trials can be a vital resource for the care of patients.

 

POTENTIAL RISKS OF A CLINICAL TRIAL

There are no guarantees of success from a clinical trial, and there are risks. For starters, there may be serious side effects. Also, the therapy may not improve upon current treatment, or may not even work at all. Finally, as a clinical trial participant, you may be part of the control group, which means either standard treatment or no-treatment placebo. In other words, there are no assurances you would receive the new therapy.

 

FINDING A CLINICAL TRIAL

Thanks to the internet, folks can find lots of information regarding the wide array of open clinical trials. So much so that it may be overwhelming. Particularly with regards to amyloidosis, casting such a wide net may not be the most productive approach. Since finding an appropriate clinical trial is not as easy for rare diseases such as amyloidosis, here are a few excellent places to start.

  • My Amyloidosis Pathfinder (MAP). Developed by the Amyloidosis Research Consortium (ARC), MAP helps patients discover and learn about amyloidosis-related clinical trials. After answering a short questionnaire, MAP matches patients to trials specific to their condition and ones for which they may be eligible.
  • Boston University / Boston Medical Center. A recognized Center of Excellence for amyloidosis, BU has an ongoing robust array of clinical trials for different types of amyloidosis.
  • Mayo Clinic. A recognized Center of Excellence for amyloidosis, Mayo Clinic has an extensive clinical trial program in the area of amyloidosis.
  • ClinicalTrials.gov. This resource, provided by the U.S. National Library of Medicine, is a database of over 250,000 privately and publicly funded clinical studies conducted around the world (in all 50 states in the U.S. and 204 countries).

 

INFORMED CONSENT PROCESS

The informed consent process is a key part of the safeguard of a clinical trial. Before joining a clinical trial, each participant will be told what to expect (e.g., treatments, tests) and what might happen (e.g., benefits and risks, including possible side effects). It is also the point where participants should ask ample questions about the trial, which the clinical trial coordinator should be more than willing to answer.

Below is a list of questions compiled from sources, including the NIH and The Clinical Study Center, recommending what patients should consider asking before consenting to participation in a clinical trial.

  • What is the purpose of the study?
  • Who is sponsoring the study, and who has reviewed and approved it?
  • Who will be in charge of my care?
  • What treatment or tests will I have? Will they hurt?
  • What are the chances I will get the experimental treatment?
  • What are the possible risks, side effects, and benefits of the study treatment compared to my current treatment?
  • How will I know if the treatment is working?
  • How will you protect my health while I am in the study?
  • What happens if my health problem gets worse during the study?
  • How will the study affect my everyday life?
  • How long will the clinical trial last?
  • What will happen after the conclusion of the study?
  • Where will the study take place? Will I have to stay in the hospital?
  • Will you provide a way for me to get to the study site if I need it?
  • Will being in the study cost me anything (e.g., treatment, tests, travel)? If so, will I be reimbursed for all expenses (including other charges such as child care)? Will my insurance cover my costs?
  • Can I take my regular medicines while in the trial?
  • Who will be in charge of my care while I am in the study? Will I be able to see my own doctor?
  • Will you follow up on my health after the end of the study?
  • Will you tell me the results of the study?
  • Whom do I call if I have more questions?
  • How will you keep my doctor informed about my participation in the trial?
  • Does the study compare standard and experimental treatments?
  • If I withdraw, will this affect my normal care?
  • What are the chances that I will receive a placebo?
  • What steps ensure my privacy?

 

Taking part in a clinical trial is solely the decision of the participant, although they may want to discuss it with their medical team prior to finalizing a decision. If one decides to join the trial, they will be required to sign an informed consent form that presents the key facts of the study and indicates they have been told all of the details and want to be part of the study. Importantly, the informed consent form is NOT a contract. Participants can leave the trial at any time and for any reason without being judged or put in a difficult position regarding medical care. Researchers much keep health and personal information private. Also, during the trial, participants have the right to learn about new risks or findings that emerge.If researchers learn that a treatment harms any of the participants, they’ll be removed from the study.

 

PARTICIPANT PROTECTION & SAFETY

Before committing to participate in a clinical trial, it is important to understand participant safety. Congress has put laws in place to protect against abuses, and today every clinical investigator is required to monitor and make sure that every participant is safe. These safeguards are enforced by the Federal Government. Every clinical trial follows a protocol that describes what the researchers will do. The principal investigator, or head researcher, is responsible for making sure that the protocol is followed.

In addition, there are multiple scientific oversight groups to aid in the control of clinical trials.

  • Institutional Review Board (IRB): Comprised of doctors, scientists, statisticians, and lay people, IRBs provide scientific oversight for all clinical trials in the United States. IRB members regularly review studies and their results, making sure risks (or potential harm) are minimized.
  • Office for Human Research Protections (OHRP): The U.S. Department of Health and Human Services’ (HHS) Office for Human Research Protections (OHRP) oversees all research done or supported by HHS. The OHRP helps protect the rights, welfare, and well-being of research participants. They provide guidance and oversight to the IRBs, develop educational programs and materials, and offer advice on research-related issues.
  • Data Safety Monitoring Board (DSMB): Comprised of research and study topic experts, this board is required for every NIH phase III clinical trial. Their role is to review data from a clinical trial for safety problems or differences in results among different groups of relevant studies. If they find that the experimental therapy is not working or is harming participants, they will halt the trial right away.
  • Food and Drug Administration (FDA): In the United States, the FDA provides oversight for clinical trials that are testing new medicines or medical devices. They review applications before any testing on humans is done, checking to ensure a proposed clinical trial has proper informed consent (see earlier) and protection for human subjects. In addition, the FDA provides oversight and guidance at various stages throughout the trial.

 

Scientific oversight informs decisions about a trial while it’s underway. For example, some trials are stopped early if benefits from a strategy or treatment are obvious. That way, wider access to the new strategy can occur sooner. Sponsors also may stop a trial, or part of a trial, early if the strategy or treatment is having harmful effects. Protecting the safety of people who take part in clinical trials is a high priority for all involved. Each trial has scientific oversight, and patients also have rights that help protect them.

 

DECIDING WHO PARTICIPATES IN CLINICAL TRIALS

After signing the informed consent form, the clinical staff will screen the candidate against the clinical trial criteria. The screening may involve cognitive and physical tests. Inclusion criteria for a trial might include age, stage of disease, gender, genetic profile, family history, and whether or not the candidate has a study partner who can accompany them to future visits. Exclusion criteria might include factors such as specific health conditions or medications that could interfere with the treatment being tested. Generally, individuals can participate in only one trial or study at a time. Different trials have different criteria, so being excluded from one trial does not necessarily mean exclusion from another.

Clinical trials need numbers … many volunteers must be screened to find enough people for a study, and with rare diseases such as amyloidosis, important trials are often significantly delayed due to a lack of participants. This can seriously slow down the rate at which new drugs are discovered, tested, and made available to patients.

 

CONCLUSION

Not all clinical trials have successful outcomes. However, every disease-related drug and therapy treatment prescribed today is the result of clinical research. Clinical trials are absolutely necessary to determine that a treatment is safe and that it has a real positive effect on a particular disease, better than that observed by a placebo or the current standard of care.

Final thoughts from Isabelle Lousada, founder and CEO of Amyloidosis Research Consortium:

Clinical trials play a critical role in evaluating novel therapies, establishing the best treatment pathways, and increasing our knowledge about amyloidosis.

 

SOURCES

Amyloidosis Research Consortium

Boston University / Boston Medical Center, Amyloidosis Center

The Clinical Study Center

Mayo Clinic

National Institute on Aging

National Institutes of Health

U.S. National Library of Medicine

 

Amyloidosis ICD Diagnosis Codes

What are diagnosis codes and what are they used for? Here’s a brief summary.

ICD, which stands for International Classification of Diseases, is a coding system implemented by the World Health Organization (WHO) to represent diagnoses. It was originally developed by the WHO in the 1970s, and periodically new revisions are released. On October 1, 2015, ICD-10 (meaning the 10th revision) became effective and is used in almost every country worldwide, except the United States.

 

ICD-10 Codes in the United States

The National Center for Health Statistics (NCHS) is responsible for ICD-10 use in the United States. With the permission of the WHO, the NCHS has developed a modification of ICD-10 used only in the United States called ICD-10-CM, with the CM part standing for clinical modification.

 

Who Uses The Codes in the United States?

These codes are used by physicians, insurance companies, and other healthcare providers to classify and code all diagnoses, symptoms, and procedures in conjunction with hospital care in the United States. Every disease, disorder, injury, infection, and symptom has its own ICD-10-CM code.

 

What Are The Codes Used For?

These codes are used for everything from processing health insurance claims to tracking disease epidemics and compiling worldwide mortality statistics. According to AAPC, the more granular codes in ICD-10-CM provides multiple benefits, such as the following.

  • Better coordinate a patient’s care, both across providers and over time.
  • Improve the quality of measurement and reporting.
  • Facilitate the detection and prevention of fraud, waste, and abuse.
  • Lead to greater accuracy of reimbursement for medical services.
  • Improve data capture and analytics for:
    • public health surveillance and reporting
    • national quality reporting
    • research and data analysis
    • provide detailed data to enhance healthcare delivery

 

What Are The Codes for Amyloidosis?

ICD-10-CM allows for approximately 69,000 codes, up from approximately 13,600 in ICD-9-CM. All codes are alphanumeric, beginning with a letter and with a mix of numbers and letters thereafter. Valid codes may have three, four, five, six or seven digits. Specific to amyloidosis, here are the codes.

The first three characters define the category of the disease, disorder, infection or symptom. For example, codes starting with M00-M99 are for diseases of the musculoskeletal system and connective tissue (like rheumatoid arthritis), while codes starting with J00-J99 are for diseases of the respiratory system.

For amyloidosis, the disease is categorized E85 within the E00 – E89 group.

E00 – E89: Endocrine, nutritional and metabolic diseases

E85: Amyloidosis

Characters in positions 4-6 define the body site, severity of the problem, cause of the injury or disease, and other clinical details. A seventh character is an extension character used for varied purposes such as defining whether this is the initial encounter for this problem, a subsequent encounter, or sequela arising as a result of another condition.

E85.81   Light chain (AL) amyloidosis

E85.82   Wild-type transthyretin-related (ATTR) amyloidosis

E85.89   Other amyloidosis

 

From medical records to ICD-10-CM Codes to Usable Information

According to Very Well Health, medical coders read medical records, extract the diagnoses from those records, and translate the diagnoses into ICD-10-CM codes. While most coders have software to help them, the process can also be done by hand using books and coding manuals and can vary from health care system to health care system. Whether the medical coder uses software or a book, coding a medical record correctly requires education in the myriad rules used to choose and apply the ICD-10 codes, as well as close attention to detail.

Once the medical record has been coded by the coder, the data can be used in a number of ways.

  • The medical biller can send the coded claim to the health insurance company for processing.
  • Insurers may use the data to help predict future health care expenditures.
  • Researchers may use the data to determine disease prevalence across geographic areas, ages, or in conjunction with other diseases.

In the end, having richer and more granular data provides a number of benefits and brings value to the analysis, processing, and forecasting within the healthcare community.

 

Now if we can just get amyloidosis diagnosed earlier …

 

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Sources: AAPC, Very Well Health, World Health Organization, Centers for Disease Control and Prevention.

Building a Caregiver Team

BUILDING A CAREGIVER TEAM

There are times when those afflicted with amyloidosis need assistance — a caregiver. In some instances, such as a stem cell transplant (SCT), the treatment is so extensive that securing a caregiver is required before treatment commences. It is for these situations that we thought we’d share some thoughts about how to build a caregiver team.

 

CONSIDER YOUR NEEDS

The role of a caregiver can be intensive and draining. In particular, SCTs performed on an outpatient basis, while deemed to be good for patients, shift a significant amount of work to the caregiver (see https://mm713.org/transplant-inpatient-vs-outpatient/).

Begin by itemizing the different aspects surrounding your caregiver roles, such as the following:

  • Expected duration of treatment
  • Location
  • Meals (e.g., cooking, shopping)
  • Transportation
  • Basic medical monitoring and care (e.g., drug administration and tracking, food and liquid intake tracking, vitals monitoring)
  • Administration (e.g., insurance)
  • Care for kids and/or pets
  • House sitting

 

WHO IS AVAILABLE TO HELP

Family members are often the first call to offer caregiver support. Neighbors and good friends often stand at the ready to help as well. Embrace and accept their help.

 

HOW TO ASSEMBLE THE TEAM

Particularly if the expected duration is long, caregivers need breaks. The caregiving process itself can be intensive and draining, and they have their own daily needs to attend to as well.

It is not uncommon to have multiple sequenced (one after another) caregivers during a four to six-week SCT treatment. However, another way to think about assembling your caregiver team is to line them up to work in parallel and together. You may be able to extend the duration of each caregiver if their tasks are narrower in scope. In addition, assign tasks consistent with each caregiver’s skills.

In my situation, I needed to prepare for having a SCT on an outpatient basis. I was extraordinarily fortunate to have four family members live with me throughout the entire treatment process. We figured it would be most productive to allocate responsibilities based on skills and that we would build in breaks from the intensity. Here’s how my caregiver team looked:

  • My Dad. He was in charge of room and transportation. We needed a place to stay for five people for up to six weeks (we found a fantastic transplant-friendly house through HomeAway near the Mayo Clinic in Rochester). We also needed transportation to get all of us to/from Rochester, and determine how best to get around during our stay (a combination of fly and drive so that we had a car).  Four of us were coming from the Washington D.C. area, and one from the Los Angeles area. Lots of logistics to consider.

 

  • My Mom. With her strong organizational skills and squeamishness towards all things medical, she was in charge of all things administrative and insurance-related. She was in charge of making the list of things we needed to bring or buy to set up the household and kitchen. She brought favorite recipes so we could more easily muster grocery lists. In addition, she would collect receipts, track mileage, and made sure there was good coordination between our insurance provider and Mayo Clinic. This is a procedure that requires advance authorization and is expensive, so attending to the financial aspect is important.

 

  • My Brother. He is three years younger than me (20, and I was 23), studying pre-med with a keen interest in becoming a physician, in addition to a strong interest in sports and nutrition. His role was three-fold: exercise for all of us (myself included), nutrition, and assist in monitoring my vitals and food/liquid intake. Our rental house neighborhood was flat and had sidewalks, offering an easy way for all of us to get some fresh air even if just for 15 minutes at a time. We also found a local fitness club that we could join for a month-to-month low cost, offering another way for the caregivers to rotate to work up a sweat and work off stress.

 

  • My Aunt. Being an oncology pharmacist, she was the obvious pick to administer my drugs, which were extensive, and monitor my vitals and food/liquid intake. In addition to the required drugs, there was also the as-needed drugs for pain, nausea, fever, etc. that she would collaborate with the Mayo medical team. Her expertise was priceless, particularly during those days when things were most difficult.

For our two pups, we found an extraordinarily loving “free range” place that lets the pups play with other like-sized pups all day, and then boards them at night. It gave us great peace of mind knowing they weren’t caged all day and night for a month. In addition, we arranged for them to send us weekly pictures, which quickly became something to look forward to.

Once we began the SCT process, I worried about nothing other than myself. I left it to my caregiver team to do all of the worrying, errands, purchasing, preparing, communicating with Mayo, etc.  I loved having my family around, as I found it extraordinarily comforting and appreciated their attempts at distracting (for example, we must have played Monopoly 20 times).

 

BE CREATIVE AND PLAN AHEAD

While everyone has a different situation, my biggest suggestion is to be creative, mindful of who and how many caregivers you assemble for your team, and don’t underestimate all that is required of your caregiver(s). Think about your needs, as well as their needs, expertise, and time availability to commit. Listen to any guidance provided by your healthcare team. There is no question there is a lot to plan for, but the more thought you put into it will no doubt pay off in your experience. After all, while you are going through treatment this will be the last thing you are interested in or able to focus on. Advance planning is critical.

 

Play FORE The Cure 2018

On August 6, 2018 we held our first charity golf tournament Play FORE The Cure. It was a smashing success!

Play FORE The Cure was designed to provide the ultimate player experience, from tip to toe. It was held at the world-class Robert Trent Jones Golf Club, just outside of Washington D.C. in Gainesville, VA. Renown PGA Tour Professional and Maryland native Fred Funk joined the event, playing a hole with every foursome and conducted a short game clinic for the players. Callaway brought their latest clubs for the players to test on the range, and take home a custom-fit Callaway wedge as one of their gifts. Fantastic contests with prizes including 5-day cruises, $10,000 cash, $50,000 cash, $100,000 cash and a 2018 Audi Q5 gave the players plenty of motivation. We invite you to view hundreds of great photos on our photo sharing site. A silent auction, thoughtfully curated with items including a beautiful handmade mahogany Gentlemen’s jewelry box, special six-bottle vertical cabernet sauvignon from Euclid Wines, 7-day Italian culinary experience for ten at a Tuscan villa, fine jewelry, memorabilia from the 2018 Stanley Cup Champion Washington Capitals, and a night in the Presidential suite at Ritz-Carlton added to the success. The evening program, emceed by my father Mark, included my keynote speech (see below), where I shared the background behind Play FORE The Cure and why it’s so important.

The objective of the fundraiser was two-fold:

  • Raise Money
  • Raise Awareness

Raising money is central to every fundraiser. We maximized the monies raised through player registrations, sponsorships, donations, silent auction, 50/50 raffle, and mulligans. We amplified the net proceeds through the judicious management of expenses and generous donations of items and services.

In the end, we raised over $135,000 for Mackenzie’s Mission!!!!

Net proceeds will be donated to the Amyloidosis Research Fund at the Mayo Clinic, one of the Centers of Excellence for this disease.

In addition, our efforts to raise awareness about amyloidosis took shape through easy-to-read pamphlets in player and volunteer gift bags, our FACES of Amyloidosis video, and sharing the story of my journey and the disease. Over 80 people have now been introduced to amyloidosis, a rare and incurable disease.

We are grateful for the contributions of our supporters, which include sponsors, auction donors, cash donors, and wonderful volunteers. Our Thank You Supporters! video highlights the names and logos of these individuals and organizations, and we invite you to take a few minutes to view the extensive list. And of course, a big thank you to our players who enjoyed the full day of activities!

We look forward to seeing you next year.

With my warmest regards,

Mackenzie

FACES of Amyloidosis 2018

Each person in this video is affected by amyloidosis. Each has their own story to tell the world, which begins, but doesn’t end, here.

Amyloidosis is a rare and incurable disease, affecting people in different ways. As a result, this often slows the diagnosis time, during which the disease continues to advance. Accurate diagnosis as to the type of amyloidosis is critical, as it directly determines the path of treatment.

Amyloidosis knows no boundaries. In the words of Jill Johnson, the last face in the video, “… amyloidosis does not discriminate. It will take the young, the old, male, female and all races and socioeconomic levels.”

A huge thank you to those that participated, for they are the true fighters.

The push to raise awareness is intensifying.

The fight to find a cure is ON.

A special thank you to Andra Day. Her song “Rise Up” and amazing voice brought these stories to life.

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