NVivo software (QSR International, Melbourne, Australia) and a grounded ...
Health-Related Quality of Life in Patients With AL Amyloidosis: Qualitative Interviews With Physicians and Patients Michelle K. White,1 Martha Bayliss,1 Muriel Finkel,2 Isabelle Lousada,3 Spencer Guthrie4
Optum, Lincoln, Rhode Island; 2Amyloidosis Support Groups, Itasca, Illinois; 3Amyloidosis Research Consortium Inc., Boston, Massachusetts; 4Prothena Biosciences Inc, South San Francisco, California
–– Physician interviews were coded and analyzed by 1 researcher –– Patient interviews were dual coded and analyzed by 4 researchers; review meetings were held to ensure agreement between coders
–– Transcripts were divided into 4 groups and were coded 1 group
at a time to demonstrate saturation as the point at which no new relevant information emerges and no additional interviews are needed
RESULTS Physician Sample •• 3
hematologists and 1 nephrologist were interviewed
–– All 4 physicians routinely treated AL amyloidosis patients
agreed it would be good practice to use a standardized HRQoL assessment with every patient at every visit but worried about the time this would require
–– They need a fast and easy-to-use HRQoL assessment •• Physicians
recommended that patients complete the HRQoL assessment every 3 months, using a recall period of “past month”
–– Once complete hematologic response is achieved, assessment can be reduced to every 6 months
•• Physicians
agreed that the most important HRQoL concepts are physical functioning, vitality/fatigue, pain, ability to work and perform other roles, general health, and sleep
–– They also noted negative impacts on mental health, such as depression and anxiety
(≥25 patients treated in the past 12 months)
•• 3
practiced in the United States (2 East Coast; 1 Midwest), and 1 practiced in Germany
•• All
4 had >5 years of postresidency practice; 3 of the 4 had >10 years of postresidency practice
9
50
Journey to Diagnosis •• The average time to diagnosis was 2 years (range, 3 months to 4 years) •• Patients
reported having consulted an average of 3 different types of specialists before receiving the correct diagnosis average, patients had been diagnosed 2.3 years before the interview (range, 4 months to 8 years) of specialists consulted before diagnosis depended on organ involvement and included primary care physicians, cardiologists, hematologists, nephrologists, gastroenterologists, pulmonologists, and others
•• Before
diagnosis, patients reported multiple symptoms: 32 different symptoms were reported by at least 1 patient; the most commonly reported symptoms were fatigue, dizziness on standing, feelings of fullness in stomach when not full, shortness of breath, edema, numbness, cognition problems, pain, tingling or burning in limbs, and sleep problems
•• Patients
underwent many tests and frequently received incorrect diagnoses before the conclusive AL amyloidosis diagnosis
–– They expressed relief at receiving the ultimate diagnosis
because of the anxiety and uncertainty they felt beforehand (Figure 2)
Number of Patients
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- Patient 4
4
- Patient 5
3 I felt like I was on death’s doorstep, literally. Day 10 and day 11 after my stem cell transplant I would rather have been dead. I couldn’t move; I was debilitated. Nauseous, diarrhea, I couldn’t think, my hair had started to fall out. I was just a basket case. I just existed. I just sat in a chair and existed. I couldn’t drink anything. I couldn’t eat anything. It was significant. - Patient 5
2 1
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0
Overall, I think maybe you don't remember it. It's kind of like giving birth to a baby, you kind of forget the worst. It's bothersome, but well worth going through it because it's short-term. - Patient 3
Type of HRQoL Impairment
Figure 4. Quotes from patients in support of findings: impact on QoL.
•• On
•• Types
If the scale is 1-10, where 1 is really tolerable and 10 is I can’t bear this [after initial treatment with stem cell transplant and melphalan], it was at a 9 [now on bortezomib, dexamethasone, and doxycycline] it is a 3 or a 4. I can tolerate this. If fatigue is the greatest [side effect of treatment, although it may be] masked by COPD and sinus problem...I can deal with that.
or
of concept elicitation interview responses determined saturation and confirmed adequate sample size
Treatment Side Effect
W
•• Analysis
0
5
re ss ed
Patient Interview Results
1
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50% of patients reported multiple organ involvement.
3
I certainly considered stopping maintenance for now just because I wonder how long I can keep this up and keep doing it. It seems to get eventually worse.
tio n
40
4
2
6
ni
No response/disease progression
5
7
ci al
10
6
8
So
Partial hematologic response
7
Figure 6. Quotes from patients in support of findings: problems tolerating treatment and impact of treatment on HRQoL.
10
Ea tin g
20
m e
Nervous system
8
Figure 3. Most reported types of impairment on HRQoL.
Ti
30
e
Gastrointestinal
9
described the level of impairment as severe in many areas (Figure 4)
s
50
10
•• Patients
ur
Kidney
Figure 5. Common side effects impacting HRQoL across all treatments.
reported a broad range of serious HRQoL impairment spanning physical disability to emotional distress (Figure 3)
is
60
Complete hematologic response
a
•• Physicians
Heart
patients (78%) reported treatment tolerability problems, including having to stop a treatment or to reduce the dosage (Figure 6)
•• Patients
Hematologic response to treatment (remission status), %
Poor tolerability [means that] the patient will get very sick. Maybe they’ll end up getting very swollen or perhaps they have other infectious complications. - Hematologist, East Coast United States
did report asking patients generally about fatigue, sleep, pain, and impact on work
10
re
I know standardization is very hard for this disease because each patient is different – has a different protein that is harming him. That’s the problem. - Hematologist, Germany
•• Physicians
West Coast
Le
were audiotaped, transcribed, coded, and analyzed using NVivo software (QSR International, Melbourne, Australia) and a grounded theory approach; this approach allows themes to emerge from the data rather than imposing a priori hypotheses
40
•• Most
Impact of AL Amyloidosis on HRQoL •• HRQoL is impacted by the symptoms of AL amyloidosis and the side effects of treatment
ho
•• Interviews
South
4 most reported organs/areas affected by AL amyloidosis,a %
Putting aside the efficacy of our treatments, I think one of the biggest challenges we have in taking care of these patients is coming up with a smooth system for their multidisciplinary care. Coordinating their care is very difficult. - Hematologist, East Coast United States
Assessing HRQoL in Patients With AL Amyloidosis •• None of the physicians used a standardized assessment of HRQoL with their AL amyloidosis patients
30
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• Their journey to diagnosis • The impact of AL amyloidosis and its treatment on their HRQoL
Midwest
ld
–– Patients provided input on
20
es
Figure 1. Quotes from physicians in support of findings: challenges in treating patients with AL amyloidosis.
East Coast
side effects were reported regardless of the treatment patients received (Figure 5). Patients reported experiencing, on average, 13 different side effects across all treatments received before the interview
- Patient 10, on shock of diagnosis
ho
• Challenges in diagnosing and treating patients with AL amyloidosis • Whether and how HRQoL is evaluated in everyday practice • Ways in which AL amyloidosis affects how patients feel and function
30
se
–– Physicians provided input on
Advanced degree (MA, PhD, MD)
bi
AL amyloidosis
I think that often patients are referred when they have advanced kidney disease, and I think it’s very hard because a lot of patients go on to develop endstage kidney disease and need dialysis once your kidneys are scarred there’s no way to reverse that process. I think that’s the biggest challenge I face, and it can be devastating for patients. - Hematologist, East Coast United States
30
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individual interviews (1 hour each) were conducted by telephone using a concept elicitation method and semistructured interview guides
College degree (BA/BS)
The longer it went the more and more frightened I got because they couldn’t identify it When they finally said it’s amyloidosis, it’s almost a relief because I finally knew what it was. - Patient 1, on relief of anxiety upon diagnosis
US region, %
•• Rigorous •• In-depth
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