Health-Related Quality of Life and Vulnerability among People with Myelodysplastic Syndromes: A US National Study

Health-related quality of life (HRQoL) and vulnerability are variably affected in patients with myelodysplastic syndromes myelodysplastic syndromes: (my-eh-lo-diss-PLASS-tik SIN-dromez) A group of disorders where the bone marrow does not work well, and the bone marrow cells fail to make enough healthy blood cells. Myelo refers to the bone marrow. Dysplastic means abnormal growth or development. People with MDS have low blood cell count for at… (MDS) and other cytopenic states; however, the heterogeneous composition of these diseases has limited our understanding of these domains. The NHLBI-sponsored MDS Natural History Study (NCT02775383) is a prospective cohort enrolling patients undergoing diagnostic work up for suspected MDS or MDS/myeloproliferative neoplasms (MPNs) in the setting of cytopenias. Untreated patients undergo bone marrow bone marrow: The soft, spongy tissue inside most bones. Blood cells are formed in the bone marrow. assessment with central histopathology review for assignment as MDS, MDS/MPN, idiopathic idiopathic: Usually refers to any condition with no known cause. cytopenia cytopenia: (sie-tuh-PEE-nee-uh) A shortage of one or more blood cell types. Also called a low blood count. of undetermined significance (ICUS), acute myeloid leukemia acute myeloid leukemia: (uh-KYOOT my-uh-LOYD loo-KEE-mee-uh) A cancer of the blood cells. It happens when very young white blood cells (blasts) in the bone marrow fail to mature. The blast cells stay in the bone marrow and become to numerous. This slows production of red blood cells and platelets. Some cases of MDS become… (AML) with <30% blasts blasts: See Blast Cells. , and "At-Risk." HRQoL data are collected at enrollment including MDS-specific (QUALMS) and general (e.g., PROMIS Fatigue) instruments. Dichotomized vulnerability is assessed with the VES-13. Baseline HRQoL scores from 449 patients with MDS (n=248), MDS/MPN (n=40), AML<30% (n=15), ICUS (n=48), or At-Risk (n=98) were similar among diagnoses. In MDS, HRQoL was worse for vulnerable participants (e.g., mean PROMIS Fatigue of 56.0 vs. 49.5; p<0.001) and those with worse prognosis (e.g., mean EQ-5D-5L of 73.4, 72.7, and 64.1 for low, intermediate, and high-risk disease; p=0.005). In vulnerable MDS participants (n=84), most had difficulty with prolonged physical activity (88%) such as walking a quarter mile (74%). These data suggest that cytopenias leading to MDS evaluation are associated with similar HRQoL-regardless of eventual diagnosis-with worse HRQoL among the vulnerable. Among those with MDS, lower-risk disease was associated with better HRQoL, but the relationship was lost among the vulnerable, showing for the first time that vulnerability trumps disease risk in affecting HRQoL.