Videos: Patient Experience

Dr. John [1]
John describes the physical health and professional life after ATTR-CM and how he adjust to the new normal
Paul [2]
Currently in a clinical trial, Paul shares the physical limitations and his message of hope for newly-diagnosed ATTR-CM patients
James
James was recovering from a knee replacement when he had a stroke and was subsequently diagnosed with ATTRwt.
. . . “I would tell other patients to be optimistic. Because the best of my understanding is this is a good time because there are emerging answers and I am encouraged, my family is encouraged, because we hear that there are new treatments around the corner that may be great options for this disease.”
  1. https://www.youtube.com/watch?v=2seyBOlLVTE
  2. https://vimeo.com/624305214
  3. Amyloidosis Research Consortium. Youtube Channel. https://youtu.be/KceywPAvV0A.

Impact on Physical Health

ATTR-CM patients are subject to a range of symptoms that impact daily life
Responsive Image
“The interesting thing that finally brought this all to a head was I love golfing and go away every year with a bunch of doctors on golf trips. Well, after three days of 36 holes a day I suddenly realized my legs were like stove pipes. My belly was out to here, I could hardly walk 20 feet.” [5]
“I had my first carpal tunnel surgery and had begun to have problems walking. I ruptured my biceps tendon while stretching with a resistance band. Right about that time, I also started having shortness of breath. I felt very tired, and my mornings were difficult.” [6]
Adapting to the physical limitations often means basic activities are a challenge and degrees of independence are lost
Responsive Image
“If there's any hills, you get very breathless. You can't dance like you used to. You have to cope with that. I mean, that's a limitation that won't go away.” [1]
“Now I have good days and bad days. By the time I was diagnosed with ATTR-CM it had been years. Even with a correct diagnosis and treatment, I still have to check my pulmonary artery pressure daily and have been hospitalized this year.” [3]
“Primarily, it's reduced my exercise tolerance. I find I really struggle to climb hills. I struggle if my blood pressure is higher, so I have to avoid salt. I have to check my blood pressure regularly. I continue to play golf, but I have to take a power cart.”
Patients also need to make life adjustments to fight the disease
Responsive Image
“I stopped ALCOHOL, CAFFEINE, DAIRY PRODUCTS, GLUTEN and SALT. I also started on a regiment of EGCG (Green Tea). What I did was all from research of what others had already done with positive results.” [2]
“Salt is a no-no, but we have found lots of good no salt foods even no salt potato chips. No salt in cooking, at the table plus NO processed foods allowed in our home. We now find food at restaurants or family gatherings too salty for us. Surprising how quickly we got used to no-salt, except that found naturally in foods.” [4]

Impact on Emotional Well-Being

Dealing with the symptoms of ATTR-CM can be a tremendous emotional burden
Responsive Image
“What a change of life for my wife and myself. I had lost my independence. I needed help with almost everything I did. The physical and mental tiredness we all experienced was huge, the extent of which cannot be underestimated.” [6]
Patients try to remain positive...
Responsive Image
“I have to stop and tell myself, ‘Look, there are people who are a lot worse off than you. And you have limitations, you have to live with it.’ And so every time I get myself in that position where I'm thinking negative thoughts, I try to pull myself out of it, take a deep breath and let that subside and then try to be positive about the next step.” [1]
“I do get bouts of feeling sorry for myself, but I do try and live my life to the full, as full as I can make it.” [2]
“It’s disappointing that we can’t do some of the things we used to do but we still do interesting things together.” [3]
“[The patient] is a grandfather to five beautiful children and they want Pops around for many more Christmases. So, we’re in it to win it.” [5]
. . . but negative emotions are sometimes even worse than physical symptoms
Responsive Image
“Not knowing. Not knowing where I am on the path. Not knowing how things will pan out.” [4]
“You spend a lot of time in that depression/mood/ mortality thing wondering what your future is going to be like.” [5]
For Family / Caregivers, as well
“I worry about him, that he has all these medications he takes. He’s very concerned that he does them properly, and I—I don’t know how I can help make that happen. He’s very organized . . . but I worry that his life-style has changed for him so much, he gets frustrated at it—and I hate to see that. I have anxiety. I want him to be well. I want to reach in and take that amyloidosis outside of his heart! … And sometimes I feel bad that I, I’m healthy. I like being healthy.” [5]
1.https://vimeo.com/310240454?embedded=true&source=video_title&owner=2008339
4.Pfizer - Heart Matters: ATTR-CM community
5.Rintell, D., Heath, D., Braga Mendendez, F. et al. Patient and family experience with transthyretin amyloid cardiomyopathy (ATTR-CM) and polyneuropathy (ATTR-PN) amyloidosis: results of two focus groups. Orphanet J Rare Dis 16, 70 (2021). https://doi.org/10.1186/s13023-021-01706-7

Impact on Family & Social Life

  • Family members and caregivers are deeply affected, financially, emotionally, and physically
  • Patients may be unable to participate in activities they used to enjoy, and, unable to participate fully in the outside world, they may turn to social media for medical advice and information
IMPACT ON CAREGIVER & FAMILY
Responsive Image
“My wife had a doubling of her workload as well as dealing with the huge concern of whether I would survive. Our son had to step in and assist with driving and some shopping.” [1]
“As a caregiver we tend to modify things. We make it so that it works for what you’re going to do. You’re going to go on a hike, maybe you’re not going to hike ten miles. You’re only going to hike one. So, you modify everything. You do that with food, as well. You don’t make a big deal. You make, half. So, you’re only walking the one mile. You modify it so that it’s not a big bone of contention. I think we modify so that we don’t have that… I can’t do the Christmas lights all at one time. Maybe I’ll take three days to do it. Which is fine. It’s going to change the quality of your life, but it’s not going to end having quality to your life.” [4]
“Right of out of the blue somebody said to us ‘you’re going to have to have a heart transplant,’ It’s terrifying, and I was just totally knocked of balance. Crying, not knowing  what are we going to do. He’s too young to die. I just, it’s just, so my anxiety and fear is very strong.” [2] – ATTR caregiver
RESTRICTIONS OF SOCIAL / FAMILY LIFE
Responsive Image
“Although he is no longer able to scuba dive or walk miles while traveling with his wife, Ivan says he still finds intrigue and adventure in different, more accessible ways. Together, he and Nora enjoy reading, spending time outside in their backyard, and keeping up with their two-year old granddaughter.” [3]
“The disease means that Art becomes easily short of breath, even from a brief stroll or a walk up the stairs and can’t participate in many family activities he once loved, such as camping.” [4]
Patient groups, e.g., Facebook groups, offer support and connection to others who understand what the patient / caregiver is going through
“Has anyone experienced nosebleeds? My husband has had them a few times a week recently. Perhaps the house or his nose is dry. It could possibly be the blood thinner (Eliquis) he takes. No other unexpected issues since being diagnosed and prescribed Vyndaqel three years ago.” [5]
  1. https://amyloidosis.net.au/multiple-myeloma-and-attrwt-balancing-two-difficult-diagnoses/
  2. Rintell, D., Heath, D., Braga Mendendez, F. et al. Patient and family experience with transthyretin amyloid cardiomyopathy (ATTR-CM) and polyneuropathy (ATTR-PN) amyloidosis: results of two focus groups. Orphanet J Rare Dis 16, 70 (2021). https://doi.org/10.1186/s13023-021-01706-7.
  3. https://www.eidostx.com/ivans-story/
  4. https://www.eidostx.com/arts-story/
  5. Amyloidosis- Wild Type- ATTRwt –wtATTR, https://www.facebook.com/groups/1487530094602375

Impact on Professional Life & Financial Impact

Symptoms of ATTR-CM can make work nearly impossible and patients may be forced to quit and/or retire from a career they were passionate about
Discontinuing Work
Responsive Image
“Before I got sick, I was enjoying a rich, full life. I had a various jobs in the tech sector and even founded a kombucha company! But during the latter part of my career, I started having symptoms of an illness that became so severe, I couldn’t work anymore. At the time, I didn’t know what was making me so sick.” [1]
“During a routine check-up with my cardiologist in 2018, I mentioned that I could no longer work because of carpal tunnel syndrome in each wrist limiting dexterity.” [2]
Seeking financial aid could be challenging
usa Responsive Image
Financial Impact
Responsive Image
“We are living on our ever dwindling Super, if  [patient] qualified for disability support it would help us cope financially. Centrelink (social security program) told us to come back when he had a year to live. That night we both said it’d be OK to go to sleep and not wake up.” Carer ATTR [3]
“My husband Joe was diagnosed with Wild Type Amyloidosis in Sept/Oct 2020. Less than stage 1, we was given the OK from Mayo Clinic to start Vyndamax 61 mg in Jan 2021 to avoid paying a 5K co-pay in Oct and again in Jan. We have tried everything for financial assistance without any luck.” [5]
“I was diagnosed last fall, had many, many tests but still unable to get any medication yet. Everyday dealing with different mental and physical things. Hopefully after a two week heart halter test and a heart biopsy my heart specialists will be able to prove to the government that I actually suffer from this crazy disease and I will be eligible for financial assistance.” [4]
  1. https://www.pfizer.com/news/hot-topics/my_journey_to_diagnosis_an_attr_cm_patient_s_story
  2. https://amyloidosis.net.au/multiple-myeloma-and-attrwt-balancing-two-difficult-diagnoses/
  3. Pfizer - Heart Matters: ATTR-CM community
  4. Facebook, Amyloidosis- Wild Type- ATTRwt –wtATTR (Private) (Posted 10/07/2021, accessed 19/12/2021)
  5. Facebook, Amyloidosis- Wild Type- ATTRwt –wtATTR (Private) (Posted 30/10/2020, accessed 19/12/2021)