A Systematic Literature Review on Symptom Burden and Impact of Disease on Quality of Life in Patients With Essential Thrombocythemia
Author(s)
Mavis Obeng-Kusi, PhD1, Xiaoqin Yang, PhD1, Rishabh Pandey, PhD2, Saikrishna Kandalam, PhD2, Shruthi Bethi, MPharm2;
1Merck & Co Inc, Rahway, NJ, USA, 2PAREXEL INTERNATIONAL, Hyderabad, India
1Merck & Co Inc, Rahway, NJ, USA, 2PAREXEL INTERNATIONAL, Hyderabad, India
Presentation Documents
OBJECTIVES: To better understand the symptom burden and quality of life (QoL) associated with essential thrombocythemia (ET), this systematic literature review aims to identify and summarize peer-reviewed literature to provide a comprehensive summary of these factors in patients with ET.
METHODS: A systematic literature search was conducted in the MEDLINE®, Embase®, and Cochrane databases from inception through October 6, 2023. The studies were screened based on the PICOTS criteria
RESULTS: Overall, 51 studies reported data on symptom burden and QoL associated with ET. The reported mean total symptom scores ranged from 13.3-28.6 out of 100 (higher scores indicate higher symptom severity) on MPN-SAF questionnaires. Fatigue was reported as the most prevalent symptom (82.0%-90.3%), with mean severity scores ranging from 2.5-5.0 out of 10 (higher scores indicate worse symptoms). Other prevalent symptoms reported were insomnia (58.0%-72.0%), night sweats (46.2%-63.0%), dizziness (54.2%-62.0%), concentration problems (49.0%-55.8%), early satiety (49.0%-56.0%), and inactivity (50.1%-54.0%). Patients reported (PR) a higher rate of symptoms compared to physician observations (PO), such as fatigue (PR:71.2%, PO:48.8%), inactivity (PR:46.3%, PO:27.1%), and concentration problems (PR:44.7%, PO:24.1%). ET patients had lower QoL compared to the general population. This difference was reported in both the EORTC-QLQ-C30 questionnaire (68.5vs.73.0) and the FACT-An fatigue subscale (51.6vs.71.1), where higher scores indicate better QoL. ET patients also reported lower physical (EORTC QLQ-C30:77.2vs.86.0 and Godin LAS:27.5vs.45.8), emotional (EORTC QLQ-C30:79.8vs.84.0), and social functioning (EORTC QLQ-C30:83.9vs.90.0) compared to the general population (higher score indicate higher levels of functioning). For ET patients, the reported mean percentage scores for work productivity loss ranged from 11.5%-35.7% (absenteeism:2.9%-7.4%; presenteeism:9.5%-30.7%), and activity impairment ranged from 18.3%-36.3%, higher scores indicate greater impairment and less productivity.
CONCLUSIONS: Patients with ET experience significant symptom burden, with fatigue being the most prevalent symptom, which can impair QoL, daily functioning, and work productivity. Results highlight a need for improved disease management strategies.
METHODS: A systematic literature search was conducted in the MEDLINE®, Embase®, and Cochrane databases from inception through October 6, 2023. The studies were screened based on the PICOTS criteria
RESULTS: Overall, 51 studies reported data on symptom burden and QoL associated with ET. The reported mean total symptom scores ranged from 13.3-28.6 out of 100 (higher scores indicate higher symptom severity) on MPN-SAF questionnaires. Fatigue was reported as the most prevalent symptom (82.0%-90.3%), with mean severity scores ranging from 2.5-5.0 out of 10 (higher scores indicate worse symptoms). Other prevalent symptoms reported were insomnia (58.0%-72.0%), night sweats (46.2%-63.0%), dizziness (54.2%-62.0%), concentration problems (49.0%-55.8%), early satiety (49.0%-56.0%), and inactivity (50.1%-54.0%). Patients reported (PR) a higher rate of symptoms compared to physician observations (PO), such as fatigue (PR:71.2%, PO:48.8%), inactivity (PR:46.3%, PO:27.1%), and concentration problems (PR:44.7%, PO:24.1%). ET patients had lower QoL compared to the general population. This difference was reported in both the EORTC-QLQ-C30 questionnaire (68.5vs.73.0) and the FACT-An fatigue subscale (51.6vs.71.1), where higher scores indicate better QoL. ET patients also reported lower physical (EORTC QLQ-C30:77.2vs.86.0 and Godin LAS:27.5vs.45.8), emotional (EORTC QLQ-C30:79.8vs.84.0), and social functioning (EORTC QLQ-C30:83.9vs.90.0) compared to the general population (higher score indicate higher levels of functioning). For ET patients, the reported mean percentage scores for work productivity loss ranged from 11.5%-35.7% (absenteeism:2.9%-7.4%; presenteeism:9.5%-30.7%), and activity impairment ranged from 18.3%-36.3%, higher scores indicate greater impairment and less productivity.
CONCLUSIONS: Patients with ET experience significant symptom burden, with fatigue being the most prevalent symptom, which can impair QoL, daily functioning, and work productivity. Results highlight a need for improved disease management strategies.
Conference/Value in Health Info
2025-05, ISPOR 2025, Montréal, Quebec, CA
Value in Health, Volume 28, Issue S1
Code
PCR240
Topic
Patient-Centered Research
Topic Subcategory
Patient-reported Outcomes & Quality of Life Outcomes
Disease
No Additional Disease & Conditions/Specialized Treatment Areas, SDC: Oncology