Objective

Symptom burden is the combined impact of symptoms from disease and treatment on daily functioning. Lack of symptom recognition may result in failure to address symptoms and maximize patient functioning. Patient-reported outcomes (PROs) can be endpoints in clinical trials to establish treatment benefits. The purpose of this study was to define the content domain for a PRO measure of acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS) symptom burden.

Materials and Methods

This is Part 1 of a study to develop a PRO AML/MDS symptom-burden measure. Patients with AML and MDS described symptoms over their disease courses in single qualitative interviews. Content analysis was used to identify symptoms and define the content domain for a PRO measure. After the interview, patients rated the worst severity of 13 common cancer-related symptoms in the last 24 hours on the M. D. Anderson Symptom Inventory (MDASI) to provide context for analysis.

Results

Thirty patients participated in an interview (see Table 1). Content analysis found a total of 39 symptoms, 14 reported by 20% or more patients, occurring over the disease course. Fatigue and distress (followed by pain) was the symptom reported most often. The mean number of symptoms reported by patients with AML (5.46; sd = 2.83) and by patients with MDS (7.33; sd=4.18) was not significantly different. Proportionately patients with MDS reported more fatigue, disturbed sleep, drowsiness, shortness of breath, headache, and sadness but less pain and numbness/tingling than AML patients. Both groups of patients described symptom interference with day-to-day activities. On the MDASI, fatigue (followed by distress and disturbed sleep) was the symptom reported most often in the last 24 hours. Fatigue had the highest mean severity rating (3.37, sd = 3.36, 0-10 scale) on the MDASI.

Table 1

Patient Characteristics

N=30MeanSD*
Age (years) 55.5 19.1 
Time from diagnosis to interview (months) 21.3 39.3 
 N % 
Gender - Male 16 53.3 
Race - White, Non-Hispanic 19 63.3 
AML - newly diagnosed 2 6.7 
AML - remission or minimal residual disease 11 36.7 
AML - relapse 3 10 
AML - refractory 8 26.7 
MDS 6 20 
ECOG# performance status < 2 24 80 
Active treatment at time of interview 25 83.3 
Hospitalized at time of interview 17 56.7 
Alive 6 months after interview 18 60 
N=30MeanSD*
Age (years) 55.5 19.1 
Time from diagnosis to interview (months) 21.3 39.3 
 N % 
Gender - Male 16 53.3 
Race - White, Non-Hispanic 19 63.3 
AML - newly diagnosed 2 6.7 
AML - remission or minimal residual disease 11 36.7 
AML - relapse 3 10 
AML - refractory 8 26.7 
MDS 6 20 
ECOG# performance status < 2 24 80 
Active treatment at time of interview 25 83.3 
Hospitalized at time of interview 17 56.7 
Alive 6 months after interview 18 60 
*

SD = standard deviation

#

ECOG = Eastern Cooperative Oncology Group

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Conclusions

Patients with AML and MDS experience numerous symptoms related to disease and treatment, with fatigue and distress being the most prominent throughout the disease course. Qualitatively, the symptom burden experienced by the two groups of patients is similar. These symptoms can cause significant interference with daily activities. Accurate assessment and management of symptoms is critical to ensure optimal treatment with the best quality of life. The content domain for a PRO symptom-burden measure of AML and MDS encompasses the severity and activity interference of common symptoms of AML and MDS and its treatment.

Disclosures:

No relevant conflicts of interest to declare.

Topics:
leukemia, myelocytic, acute, myelodysplastic syndrome, fatigue, disease progression, pain, cancer, disease remission, drowsiness, dyspnea, headache

Author notes

*

Asterisk with author names denotes non-ASH members.

© 2013 by The American Society of Hematology
2013
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