Video: Patient Experience

Paul
Paul was put on standard heart failure drugs because there weren’t any treatments available. He tried to stay positive but knowing there is no cure placed an emotional burden on the whole family
  1.  ATTR amyloidosis patient's association. https://vimeo.com/624305214. 

Patient Physical Experience

Receiving a diagnosis of ATTR-CM does not guarantee that the patient will receive appropriate treatment
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“They (HCPs) prescribed Metoprolol and Lisinopril, and those are not appropriate drugs for amyloidosis, and I went to various docs kind of that I knew and worked around. Finally, I realize I’m not getting anywhere with this.” [1]
Patients frequently report difficulty finding knowledgeable HCPs who can guide them through their disease . . .
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“He is being treated at the Heart Success Center in Morristown. His cardiologist specializes in Amyloidosis. But they can’t tell us how much it has progressed/how much protein is in his heart. They diagnosed him through bloodwork. Is it normal for a doctor to say we don’t know how far along it is?” – Caregiver [2]
. . . as a result, patients may take matters into their own hands for Tx decisions and their search for answers
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“[He] became an amyloidosis expert by looking everything up online, and he was telling the cardiologist. His cardiologist admitted he didn’t know anything about it, either.” – Spouse of ATTR patient [1]
“I researched online and chose the Cleveland Clinic in Ohio due to the distance from our home, insurance, and their reputation in the research and care of patients with this disease.” [3]
Some patients may experience rapid decline or even mortality within weeks after the diagnosis 
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“I went through fast deterioration with my hubby. He passed 5 weeks after we first learned the disease existed.” – Caregiver [4]
  1. 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.
  2. Amyloidosis Awareness, https://www.facebook.com/groups/150410138391756
  3. https://amyloidosis.org/where-will-this-attrwt-amyloidosis-journey-take-me/1
  4. Facebook – amyloidosis awareness (posted Dec. 10/12/2021, accessed 15/12/2021)
Patients may receive a combination of treatments depending on the manifestations
PACEMAKER
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“To prevent any potentially fatal consequences, Phil’s medical team recommended a surgically implanted pacemaker.” [1]
TREATMENT COMBINATION
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“I'm taking Tafamidis as well as Sotalol for the heart, Lyrica for nerve spasms in knee and Eliquis to thin the blood.” [2]
Heart transplant may be appropriate in limited cases but is uncommon due to the often-advanced age of patients, donor shortages, recurrence concerns, etc.
“Len’s name was added to the heart transplant list. And just about three months later, his cardiologist called him with the good news: a healthy heart was waiting for him.” [6]
“Doctors told him that the only true cure for the disease would be a double transplant: a heart and a kidney. He chose not to pursue that route and instead chose to make the most of every day and find inspiration for the future.” [3]
Outcomes of Treatments are Highly Variable
Some patients experience a positive response...
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“Since I've been doing reasonably well, Dr. Fine has encouraged me to try and get back into shape because it's safe to do. I'm now encouraged that I can actually start getting back in shape.” [4]
. . . while others suffer from side effects and continuous declining
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“Coughing is extremely painful. He (my husband) had a cardiac tamponade resulting in two sternotomies. His prognosis is poor and is in Stage 3 CHF. His pain is not being managed. . . . He needs pain relief and quality of life and dignity in death.” – Caregiver [5]
 
  1. https://www.eidostx.com/phils-story/ 
  2. Facebook, Amyloidosis- Wild Type- ATTRwt –wtATTR (Private) (Posted 02/12/2021, accessed 15/12/2021)
  3. https://www.eidostx.com/arts-story/
  4. https://www.youtube.com/watch?v=2seyBOlLVTE
  5. Amyloidosis- Wild Type- ATTRwt –wtATTR, https://www.facebook.com/groups/1487530094602375
  6. https://www.eidostx.com/lens-story/

Patient Emotional Experience

Trying to survive
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“Until they come up with a way to pull the amyloid crap out of my heart, my plan is staying active, staying lean and tafamidis. It’s working so far.” [4]
“She (Cardiologist) said, ‘You're far too young to live a life that is controlled by this disease. And so, We Are Going To Fight Back!’” [5]
Uncertain
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“My quality of life has deteriorated dramatically within the last year, particularly within the last six months. My question is, is there any hope that some endurance or strength can return with treatment?” [3]
“It took a long process to get there (ATTR diagnosis). I think that the struggle that most of us encounter, is the process of going from wondering what the hell's wrong with us to, ‘Oh, now I know the answer, now what am I supposed to do?’” [2]
hopeless
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“My husband is suffering. Much difficulty breathing and can’t walk. Coughing is extremely painful. I need help and feel hopeless. How do I change this endless situation of hospital admissions for everything?” [1]
  1. Facebook, Amyloidosis- Wild Type- ATTRwt –wtATTR (Private) (Posted 07/12/2021, accessed 15/12/2021)
  2. https://www.youtube.com/watch?v=UMIxcaamdg4
  3. Facebook, Amyloidosis- Wild Type- ATTRwt –wtATTR (Private) (Posted 01/10/2021, accessed 15/12/2021)
  4. https://amyloidosis.org/cardiac-amyloidosis-needs-to-be-on-the-decision-tree/
  5. https://www.youtube.com/watch?v=KceywPAvV0A