Patient-Reported Outcomes and Quality-of-Life Measures in Myeloid Disease Can Help Inform Treatment Decisions

CURE, Hematology Special Issue, Volume 1, Issue 1

Two posters presented at the annual meeting of the American Society of Hematology discussed patient-reported outcome measures and quality-of-life indicators as supportive tools for treatment in individuals being treated for acute myeloid leukemia and myelodysplastic syndrome.

Patients’ assements of their disease and treatment symptoms can have a significant impact on outcomes, possibly due to adherence issues. Currently, adequately validated measures for patient-reported disease- and treatment-related symptom burden in acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS) are scarce. Identifying this gap, scientists at the MD Anderson Cancer Center developed a short, validated, reliable patient-reported outcome measure of symptoms and symptom burden experienced by AML and MDS patients. The results from this study were consolidated in a poster that was presented on the first day of the annual meeting and exposition of the American Society of Hematology, held December 5-8, 2015, in Orlando, Florida.

A total of 152 patients with AML and 97 patients with MDS who were recruited to this study rated the 13 core symptom items (pain, fatigue, nausea, disturbed sleep, distress, shortness of breath, trouble remembering, lack of appetite, drowsiness, dry mouth, sadness, vomiting, and numbness and tingling), six proposed AML/MDS symptom items (muscle weakness, malaise, fever, headache, diarrhea, skin problems), and six interference items (general activities, mood, work, relations with others, walking and enjoyment of life) on a 0 to 10 scale (0 = not present or no interference; 10 = as bad as can be imagined or complete interference), twice, one to two days apart. Patient clinical and demographic information was collected from medical records and analyzed using descriptive statistics.

The study found that both groups of patients endorsed similar symptoms, and none of the means of the four final AML/MDS symptoms were significantly different between the groups.

This study led the authors to conclude that the lack of recognition of symptoms experienced by patients with AML and MDS can lead to inadequate management of symptoms, interfere with the ability of patients to function and enjoy life, and impact the tolerability of and adherence to treatment regimens. The authors believe that their questionnaire, a patient-reported outcomes measure (PROM), is sensitive because it could recognize significant differences in symptom severity between AML inpatients and MDS outpatients.

The study’s lead author, Loretta A. Williams, Ph.D., R.N., M.S.N., assistant professor in the Department of Symptom Research at MD Anderson Cancer Center, told The American Journal of Managed Care, “We were not sure that a single instrument would be appropriate for both AML and MDS. We were glad that, while the instrument is sensitive to differences in severity of symptom burden between the two diseases, the same set of symptoms was appropriate for both.” Williams sees potential for this PROM to be used by pharmaceutical companies in clinical trials of leukemia therapy because it was developed using the FDA guidance for patient-reported outcomes for labeling indications.

Some of the treatments used in patients with AML and MDS — many of whom are in their 60s and 70s — are quite harsh, and choosing these treatments for these older patients can be a difficult decision, considering their potential impact on quality of life (QOL). In the absence of cure, improving a patient’s QOL is important in the last year or so of life. With this objective, researchers from the Moffitt Cancer Center compared QOL between groups receiving intensive therapy, non-intensive therapy and supportive care, the results of which were presented in the same poster session Dec. 5.

Eighty-five patients, 60 years of age and older and diagnosed with high-risk MDS and AML, were recruited from Moffitt Cancer Center between December 2013 and April 2015. Forty-six patients received intensive therapy, 34 received non-intensive therapy and five received supportive therapy. The Functional Assessment of Cancer Therapy-Leukemia (FACT-Leu) questionnaire was used as the measure for QOL, with the following goals:

  • Comparing the difference in QOL scores measured by the FACT-Leu versions for intensive chemotherapy, non-intensive therapy and supportive care within seven days of new treatment, or of a decision to pursue supportive care at one month or later.
  • To determine QOL predictors of AML and high-risk MDS from age, comorbidity, fatigue and diagnosis.
  • To test the moderating effect of treatment with age, comorbidity and fatigue on QoL.

The authors observed that the intensive treatment group had a significant improvement in their QOL scores at one month post-treatment. Considering the predictors of QOL, a significantly negative correlation was recorded between fatigue and QOL, indicating that, as fatigue increased, QOL decreased. However, the QOL scores for age, comorbidity and fatigue were not moderated by treatment.

The authors conclude that the most intensive treatment improved QOL scores at one month and that fatigue is a significant predictor of QOL in this patient population. They suggest further studies with a larger, more diverse sample to explore the relationship between treatment approaches and QOL, in addition to intervention studies in AML and high-risk MDS focused on fatigue management.

Lead study author Sara M. Tinsley, Ph.D., A.R.N.P., A.O.C.N., from the Moffitt Cancer Center, said in an email that, “In our practice at Moffitt, we discuss various treatment options and their most common side effects, with very limited quality-of-life data to guide our discussions. We discuss risk-to-benefit ratio, with the risk of death from treatment and the benefit of prolonged survival. From our preliminary findings, we can also inform patients that treatment with intensive treatment can improve their quality of life at one month post-treatment.” She hopes that these findings will inform decision making and lead to more longitudinal evaluation of QoL in patients with high-risk MDS and AML.