Despite the use of therapies to address the clinical manifestations of myelofibrosis (MF) and essential thrombocythemia (ET), the symptom burdens of these conditions continue to negatively affect patient quality of life (QOL).
Patients with myelofibrosis (MF) or essential thrombocythemia (ET) experience a range of symptoms that can affect quality of life (QOL). An analysis of the ongoing, noninterventional Myelofibrosis and Essential Thrombocythemia Observational STudy (MOST) published in Leukemia & Lymphoma found that individuals with lower-risk MF and low- or high-risk ET may experience a symptom burden that affects their QOL and ability to work.
MF and ET are both acquired Philadelphia chromosome–negative myeloproliferative neoplasms (MPNs)—malignant blood disorders caused by abnormal clonal proliferation of at least 1 myeloid cell line. Splenomegaly, cytopenias, constitutional symptoms, and progressive bone marrow fibrosis are the main clinical characteristics of MF, whereas ET is characterized by increased platelet and megakaryocyte production and a greater risk of vascular events.
The patient-reported symptoms of MF and ET vary but can include abdominal discomfort, bone pain, fatigue, itching, night sweats, unexplained weight loss, and potentially fever. Symptoms of spleen enlargement such as abdominal pain, left subcostal pain, and early satiety are also commonly seen in patients with MF, while additional symptoms of ET include headaches, dizziness, erythromelalgia, and concentration problems. These symptoms were found to negatively impact QOL in a majority of patients with MF and ET, including patients in the lowest symptom severity quartile, in the MPN Landmark study.
“A practice of observation and monitoring of signs/symptoms for disease progression is recommended for lower-risk asymptomatic MF or low-risk ET; however, patients with lower-risk symptomatic MF may receive cytoreductive therapy, ruxolitinib, or interferon at the physician’s discretion,” the authors of the current study wrote. “Therefore, characterizing symptom burden in these patients may help guide more effective disease management and treatment strategies.”
Patient-reported outcome (PRO) data on symptom burden for patients with lower risk MF or ET are lacking compared with available data for high-risk disease. MOST is an ongoing study of clinical characteristics, PROs, and treatment patterns of patients with low- or intermediate-1-risk (due to age alone) MF, high-risk ET, or low-risk ET currently receiving ET-directed therapy at community and academic centers across the United States.
Of 233 patients with MF and 1235 patients with ET, 125 (53.6%) and 801 (64.9%) were eligible for the current analysis. PROs assessed via validated questionnaires included symptom burden and QOL, work productivity, and activity.
In responses to both the Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF) and European Organization for Research and Treatment of Cancer Core Quality of Life Questionnaire (EORTC QLQ-C30), fatigue was the most common and most severe symptom reported in both MF and ET. “Reflecting this, fatigue is often the symptom that patients most commonly want to resolve, and treatment strategies that address disease-related fatigue may contribute to improved QOL,” the authors noted.
Other MF symptoms commonly reported in the MPN-SAF were inactivity, early satiety, and concentration problems. In the ET cohort, early satiety, inactivity, and numbness/tingling followed fatigue as the most commonly reported symptoms. In both cohorts, women had higher mean individual symptom scores across most subcategories and had higher mean total symptom scores (TSS) than men. Mean TSS scores were similar between MF risk groups, but patients with low-risk ET reported higher mean TSS and symptom scores than patients with high-risk ET.
Patients with MF who completed the EORTC QLQ-30 reported the highest mean individual symptom scores for fatigue and insomnia, respectively. Women with MF reported a greater symptom burden for both fatigue and insomnia and had worse mean scores for global health status/QOL and physical, cognitive, and emotional functioning compared with men. However, women had better social and role functioning scores than men with MF. In the ET cohort, the highest mean individual symptom scores were also for fatigue and insomnia, and women again reported higher mean symptom scores than men. Women also reported higher symptom burden in all EORTC QLQ-30 functional scales.
In general, the findings demonstrate that general symptoms such as fatigue seem to affect QOL more than symptoms specific to MPNs, such as bone pain or itching. Prior research has drawn similar conclusions, particularly regarding fatigue, the authors noted.
Another notable finding was that women reported higher mean TSS, mean symptom scores, and reduced QOL compared with men. This is also in line with evidence that women with MPNs experience more significant QOL impacts and worse overall health than men with MPNs. However, research on the cause of these differences is lacking.
In conclusion, the findings suggest that despite the use of therapies to mitigate the clinical manifestations of MF and ET, the symptom burdens of these conditions continue to negatively impact QOL for patients—including those considered lower risk. Further analyses based on MOST data will provide more insight into the QOL impacts of these conditions.
Ritchie E, Al-Janadi A, Kessler C, et al. Patient-reported outcomes of patients with myelofibrosis or essential thrombocythemia enrolled in the MOST study. Leuk Lymphoma. Published online October 7, 2022. doi:10.1080/10428194.2022.2113531