Health-related quality of life in lower-risk MDS patients compared with age- and sex-matched reference populations: a European LeukemiaNet study

Abstract

In myelodysplastic syndromes (MDS), health-related quality of life (HRQoL) represents a relevant patient-reported outcome, which is essential in individualized therapy planning. Prospective data on HRQoL in lower-risk MDS remain rare. We assessed HRQOL by EQ-5D questionnaire at initial diagnosis in 1690 consecutive IPSS-Low/Int-1 MDS patients from the European LeukemiaNet Registry. Impairments were compared with age- and sex-matched EuroQol Group norms. A significant proportion of MDS patients reported moderate/severe problems in the dimensions pain/discomfort (49.5%), mobility (41.0%), anxiety/depression (37.9%), and usual activities (36.1%). Limitations in mobility, self-care, usual activities, pain/discomfort, and EQ-VAS were significantly more frequent in the old, in females, and in those with high co-morbidity burden, low haemoglobin levels, or red blood cells transfusion need (p < 0.001). In comparison to age- and sex-matched peers, the proportion of problems in usual activities and anxiety/depression was significantly higher in MDS patients (p < 0.001). MDS-related restrictions in the dimension mobility were most prominent in males, and in older people (p < 0.001); in anxiety/depression in females and in younger people (p < 0.001); and in EQ-VAS in women and in persons older than 75 years (p < 0.05). Patients newly diagnosed with IPSS lower-risk MDS experience a pronounced reduction in HRQoL and a clustering of restrictions in distinct dimensions of HRQoL as compared with reference populations.

Red cell transfusion in outpatients with myelodysplastic syndromes: a feasibility and exploratory randomised trial

“…Red cell transfusion dependency is associated with decreased overall and leukaemia‐free survival in MDS, independent of cytogenetic risk groups and with a reduced QoL (Jadersten et al , ; Malcovati et al , ; de Swart et al , ). The European MDS (EUMDS) Registry programme has also demonstrated inferior QoL and decreased health utility in transfusion‐dependent compared with transfusion‐independent patients (Stauder et al , ). Although haemoglobin concentration is reported to have the greatest impact on QoL in MDS patient (Stauder et al , ), optimal transfusion support in MDS has not been established and it remains unclear whether different red cell transfusion policies would modify clinical outcomes, QoL and functional measures.…”

Outpatient transfusions: time to study what matters to patients

“…Our center initiated an MDS registry, now with national participation, that has been conducting prospective assessments of MDS patients every 4 to 6 months using these quality‐of‐life instruments. This registry and others have shown that transfusion dependence and lower hemoglobin level are consistent independent predictors of inferior quality of life as measured by global health status, fatigue, and self‐rated utility . In one analysis, a hemoglobin level of 10 g/dL or greater was the key threshold at which many function and symptom scores plateaued .…”

Patient‐reported outcome measures in studies of myelodysplastic syndromes and acute myeloid leukemia: Literature review and landscape analysis

“…22 Both MDS and AML patients experience a significant symptom burden and restrictions in their HRQOL, which can negatively influence their clinical outcomes. 5,15 Furthermore, their self-reported experience could even provide a reliable prediction for overall survival. 23 Therefore, it is crucial to find the most suitable PROMs that have been validated for use in these patient populations.…”

“…3 Patients with MDS often present with symptoms related to low blood counts such as fatigue, paler skin, frequent infections, bleeding, and bruising. 4,5 However, fatigue and impaired health-related quality of life (HRQOL) can only be partially explained by anemia and may also be caused by molecular aberrations, hyperinflammation, and dyspnea. [6][7][8][9] AML is a heterogeneous blood cancer, most commonly associated with bone marrow dysfunction and the production of high numbers of immature myeloid cells.…”

“…15 Therapeutic options in MDS and AML include watchful waiting, supportive therapy (including red blood cell transfusions), application of growth factors, hypomethylating agents, immunomodulatory drugs, low-dose and high-dose chemotherapy, and stem cell transplantation. 5,11 Patient-reported outcome (PRO) is a collective name for any information about a patient's health condition that comes directly from the patient without any interference or interpretation from clinical experts. 16 Patient-reported outcome measures (PROMs) are designed to measure the patient experience that cannot be obtained from an observer, but from the patient only, for example, fatigue, symptom severity, impact on daily activities, 17 and HRQOL.…”