Fatigue, Quality of Life and Related Symptoms: Patient Reported Outcomes in Myelodysplastic Syndrome, Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria

Background: Fatigue is common and very distressing among patients with myelodysplastic syndrome (MDS), aplastic anemia (AA), and paroxysmal nocturnal hemoglobinuria (PNH), frequently affecting their quality of life. Often, this is combined with other symptoms such as pain, depression, anxiety, and stress. Limited data exists on the perceived level and impact of fatigue, quality of life and related symptoms in these patients.

The objectives are to describe fatigue, quality of life (QOL) and related symptoms in patients with MDS, AA, and PNH by prospectively assessing these using the Functional Assessment of Cancer Therapy-Anemia (FACT-An) for fatigue and QOL (subscales within FACT- An), pain using the Brief Pain Inventory (BPI), and depression, anxiety and stress using the DASS-21, and to define management strategies routinely used.

Methods: Surveys were administered via the AA and MDS International Foundation's patient database from 10/2014 through 1/2015 via a secure internet portal associated with the Foundation's website. Descriptive statistics were utilized.

Results: Of 313 pts, 145 (46%) had MDS, 84 (27%) had AA, 74 (24%) PNH, and 10 (3%) unreported [31 (10%), >1 diagnosis]. The mean age was 57 years with 210 (67%) female, 197 (92%) white among 214 with known race and 70 (25%) received a blood transfusion in the past 90 days. The mean fatigue score overall was 25 (range 1-52) and 28, 25, and 24 for AA, MDS, and PNH, respectively, p=0.12. (severe level). The overall quality of life score was 68 (range 10-104) and 67, 69, 67 for AA, MDS, PNH, respectively, p=0.82. Please note with the FACT-An, FACT-G and FACT-F -The higher the score, the better the QOL. The overall ranges for stress were normal; pain and depression, mild; and anxiety, moderate. Among the subgroups, stress was normal (all); pain and depression were mild (all); anxiety was mild in MDS, moderate in AA, PNH. None of the subgroups had statistical significance for these symptoms including anxiety.

Most common management strategies used for fatigue in the past month were preserving energy 252 (81%); physical activity 234 (75%); naps 228 (73%). The strategies that were helpful to extremely helpful were preserving energy 216/252 (86%), physical activity 162/234 (69%), and naps 154/228 (68%). Among subgroups, physical activity (p=0.03) and meditation (p=0.03) showed significant differences. Frequency of use 3 or more times/week were preserving energy 166/237 (70%), physical activity 128/226 (57%), and naps 131/213 (62%) among those who indicated the frequency of use. Among subgroups, the frequency of physical activity p= 0.03, eating healthy p=0.005, and counseling p=.005 showed significant differences.

Conclusions: There are few patient reported outcomes of fatigue, QOL and related symptoms in this population of rare disorders. Fatigue and QOL are significant challenges with similar findings of fatigue, QOL and related symptoms among the subgroups. However, there were differences among the types of management strategies and the frequency of use among the subgroups. Further focus on development of interventions tailored for AA, MDS, and PNH may assist in better management of fatigue with potential improvement in QOL.