Our patient speakers at the Amyloidosis Speakers Bureau are powerful educators and offer compelling insights.
Have a listen to this brief clip from Darlene with thoughts on giving a devastating diagnosis.
Our patient speakers at the Amyloidosis Speakers Bureau are powerful educators and offer compelling insights.
Have a listen to this brief clip from Darlene with thoughts on giving a devastating diagnosis.
There is no cure for Amyloidosis.
There are, however, an increasing number of treatment alternatives that can significantly reduce, if not eliminate, the disease and put the patient into remission. The most aggressive treatment is a stem cell transplant (SCT); sometimes referred to as a bone marrow transplant.
Stem cells are cells in the bone marrow from which all blood cells develop. This treatment aims to eradicate, typically through high-dose chemotherapy (e.g., melphalan), the faulty plasma cells which make the amyloid light chains. Once eradicated, fresh cells, harvested from the patient themselves (autologous), a donor (allogeneic), or an identical twin (syngeneic), are infused into the patient. This will help to recreate a healthy bone marrow and hopefully stop further production of the amyloid protein.
This complex treatment typically takes four to six weeks and is performed on an inpatient, outpatient, or some combination, depending on the hospital. There are meaningful differences that are important to know and incorporate into each patient’s personal situation in order to make an informed decision.
From the Healthcare Perspective
Across the country, there are multiple hospitals that perform SCTs to treat amyloidosis. While hard data is elusive, the tally of transplants at each facility, we know, is not spread evenly. We do know that Mayo Clinic (Mayo) and Boston University (BU) dominate the list and perform the majority of transplants. It may not be a surprise, then, that these two hospitals are considered amyloidosis Centers of Excellence in the U.S. They see a high volume of cases, have extensive depth and breadth of expertise, and have sophisticated diagnostic equipment. They are also the two hospitals who have pioneered performing outpatient transplants. The good news is this is evolving, with more centers across the country expanding their transplant program to treat amyloidosis.
Everyone would agree that hospitals are germ and bacteria magnets, which can be dangerous for transplant patients with low to no immune systems. BU and Mayo, for example, found patients were better able to withstand the everyday germs outside of the hospital better than the more potent ones within hospitals. This provides a strong incentive for hospitals to consider outpatient, or if they choose the inpatient route, must be ever super mindful of this reality.
There are risks with SCT, and patient safety is key. Having a patient in-house during the treatment affords the hospital maximum control during the process, while being outpatient transfers some responsibility to the caregiver, such as monitoring the patient’s temperature, food, and fluid intake. Being inpatient also affords the quickest access to experts, equipment, and drugs in the event things go awry, which does happen. Mayo has found that a meaningful percentage (38% according to Dr. Morie Gertz) of patients never need hospitalization during the SCT process; however, on the occasions where it is necessary the duration averages a handful of days.
Treating patients on an outpatient basis requires hospitals to alter their process and training, and rely on the patient and caregiver to assume a more engaged role. Without question, hospitals benefit significantly from the experience of performing high volumes of outpatient transplants. Mayo, according to Dr. Morie Gertz, performed their first SCT in March 1996, and their first outpatient SCT in September 1998. In total, they have performed 744 SCTs and currently average about 33 transplants per year. According to Dr. Vaishali Sanchorawala, BU performed their first SCT in July 1994, and their first outpatient SCT in October 1996. In total, they have performed roughly 675 SCTs for AL Amyloidosis, with an annual run rate ranging between 25 and 50. Together, these institutions have over two decades of valuable experience. According to experts, small volume and the resultant lack of experience is likely the key driver behind why hospitals elect to perform SCTs on an inpatient basis.
From the Caregiver Perspective
Caregivers play a critical role in the SCT process, working closely with the healthcare team to ensure the patient is progressing appropriately. They are so critical, in fact, that regardless of inpatient or outpatient, hospitals will not proceed with a SCT unless they are confident the patient has capable and continuous caregiver support.
The role of a caregiver varies greatly between an inpatient and outpatient process. When inpatient, the caregiver provides important emotional support, as being confined to a hospital for weeks on end can be draining and discouraging. This can range from just being present, to chatting, to light activities. Caregivers also assist in the physical need for exercise, helping and encouraging the patient to walk whenever and however many steps possible. The caregiver role may be filled by one or more persons, often impacted by the distance the hospital is from home.
Outpatient SCT procedures are significantly more demanding of caregivers. For the duration of treatment, the hospital will require the patient and caregiver(s) to be proximal to the hospital. Mayo, for example, requires patients to be within ten minutes of the hospital. Fortunately, there are many hotels, motels, inns, and homes for rent (HomeAway, VRBO) that are transplant-friendly and reasonably priced. It is 24/7 support, monitoring the patient’s key indicators, administering and monitoring meds, transporting the patient to/from the hospital daily, securing meds, shopping and preparing food, maintaining the household (e.g., laundry, sanitizing, etc.), and on and on. The list is extensive and exhaustive. Arranging for such intensive support can be a challenge. Some patients assemble a series of caregivers who rotate in/out for periods of time, others are able to secure one dedicated caregiver for the entire time, and in rare instances, the patient is able to have a team of caregivers for the duration.
Whichever caregiver structure is chosen, it is important to also consider self-care for the caregiver. Mini breaks can go a long way to help sustain their ability to meet the needs of the patient and the requirements set forth by the hospital.
From the Patient Perspective
For patients, it is all about getting through this treatment and hopefully arriving at a successful outcome. Time distills down to weeks, then days, and then when things are their most difficult, just getting through the next hour is the focus.
Having a good and capable caregiver(s) in place can help the patient focus only on themselves, knowing the caregiver will take care of everything else.
Side effects of the SCT can be multiple and vary from patient to patient. The list of effects can include fatigue, fever, diarrhea, nausea/vomiting, loss of appetite, mucositis, and hair loss. Fortunately, the healthcare team can be very helpful in mitigating these effects.
Exercise is important to ward off muscular atrophy and does improve recovery. Every step matters. Both Mayo and BU find patients do better and are home quicker if they spend less time in bed and more time moving around. In addition, patients tend to benefit from the required additional movement needed when living away from the hospital.
Emotionally, a SCT is tough. No way around that. But having distractions, whether provided by the caregiver, getting out of bed to exercise or being out and about via outpatient does contribute to an improved psyche. Having any sense of normalcy is welcome.
Cost differs greatly between inpatient and outpatient treatment, with outpatient coming in meaningfully less expensive. Anecdotal information has outpatient transplants at roughly 50% off the cost of inpatient transplants. Yet regardless of the approach, SCTs are extraordinarily expensive, and most likely patients need their insurance to sign off before treatment can begin. One of the considerations by insurance companies is which hospital the patient is proposing for treatment. During our personal experience, where we dealt with two national insurance companies, both informed us that having treatment at a Center of Excellence made a difference.
Finally, what is it really like? While situations vary widely from patient to patient, as may treatments and outcomes, hearing about a SCT straight from a patient who has been there is helpful. Having had an outpatient stem cell transplant in July 2017, hear Mackenzie’s perspective while fresh post-Mayo. Additionally, preparing for an outpatient SCT is more involved for the patient and caregiver; we have provided SCT and Post-Chemo Tips on the Resources page of our website which others may find helpful.
Closing Thoughts
There is strong evidence over many years and many transplants that patient outcomes are better when performed on an outpatient basis. There are, however, notable implications for the healthcare providers, patients and caregivers, depending on which approach is chosen. Inpatient, outpatient and hybrid approaches can provide successful outcomes, but knowing these differences in advance is helpful to the decision-making process.
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Special Thanks
Morie A Gertz, M.D., M.A.C.P.
Consultant | Division of Hematology | Roland Seidler Jr. Professor Department of Medicine | College of Medicine | Mayo Distinguished Clinician
Mayo Clinic
Vaishali Sanchorawala, M.D.
Professor of Medicine | Director, Autologous Stem Cell Transplant Program | Director, Amyloidosis Center
Boston Medical Center and Boston University School of Medicine
Have you recently been diagnosed with Amyloidosis? Wondering if joining a support group, whether online or in person, would be right for you?
When I was diagnosed with AL Amyloidosis, I thought that it wouldn’t be for me. It took me over a year to join. I had a picture in my mind of what it would be like. I envisioned a group of people gathered together to complain about how sick they were. I was afraid to get a glimpse of my future. I pictured a giant pity party.
What I experienced was extremely different. These groups are full of survivors! They are handling this disease with the most positive attitude possible and are making the best of the hand they have been dealt. I have learned so much from these people.
In these groups, you will find people who have been where you are, recently diagnosed, scared, and overwhelmed. You will find people willing to share the tips and tricks that helped them face some of their darkest moments and most difficult times. You will find people to help support and encourage you through your journey.
To find out more about the role of the support group, I reached out to Muriel Finkel from the Amyloidosis Support Groups (ASG) and Marsha McWhinnie from the Canadian Amyloidosis Support Network (CASN).
WHO IS THE SUPPORT GROUP FOR?
Support groups are for patients with all types of Amyloidosis and their caregivers. Their websites are full of information and resources. Online support groups such as the Amyloidosis Support Groups on Facebook, CASN, Smart Patients, and One Amyloidosis Voice are private and require access permission by the administrators. Such security for access offers comfort for the participants to reveal more personal information. In person support groups provided by Amyloidosis Support Groups, CASN, and the Amyloidosis Foundation, offer additional services and support for patients, as well as access to expert medical professionals.
WHAT IS THE GOAL OF THE SUPPORT GROUP?
Support groups, in general, are concerned with awareness and education. Their goal is to educate and empower patients, promote awareness, support medical research, and to improve the quality of life for those with Amyloidosis.
WHAT SERVICES DO SUPPORT GROUPS PROVIDE?
WHAT A TYPICAL SUPPORT GROUP MEETING LOOKS LIKE
Although each meeting varies and ends up with a flow of its own, administrators do follow a basic outline which includes the following:
The atmosphere is laid back and comfortable, and patient confidentiality is paramount. It is absolutely your choice as to whether you share your story and ask questions.
BENEFITS OF THE SUPPORT GROUP
I am so glad that I decided to join these support groups. Not only do I get to hear the inspiring stories of those who are making life happen despite the challenges they face, but I have learned SO MUCH. Amyloidosis patients who have been living with this disease for years have a wealth of information. The administrators of the groups are also up to date with current treatments, clinical trials, and medical breakthroughs happening in the field of Amyloidosis.
I have met people whose advice has helped me through challenging times. There is something special about talking with someone who has been where you are now. Someone who has received the same diagnosis and faced the same overwhelming feelings of fear and uncertainty. To know that others have been where you are and have come out the other side is comforting and inspiring.
Thanks to these support groups I know I am never alone.
How to contact a support group near you
Amyloidosis Support Group
http://www.amyloidosissupport.org/
1-866-404-7539
Canadian Amyloidosis Support Network
1-877-303-4999
Amyloidosis Foundation
http://amyloidosis.org/resources/#websites-and-support-groups
One Amyloidosis Voice
https://www.oneamyloidosisvoice.com/
Smart Patients
https://www.smartpatients.com/
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. Enjoy!
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
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:
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:
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.
Caregivers play an important role during the treatment of amyloidosis. In fact, so important that certain treatment will not proceed without confirmation caregiver support is in place. Educated by the medical team, caregivers are involved before, during, and after a transplant and help with basic day-to-day medical and practical issues, as well as provide emotional support. During the transplant the care needs are most intensive, requiring availability 24/7.
Hear first-hand from Mackenzie and Dr. Betsy Mencher who discuss their thoughts about the role of a caregiver. Mackenzie speaks from the perspective of a patient, and Dr. Mencher shares her experience as the caregiver for her husband.