Health-related quality of life and symptom assessment in patients with myelodysplastic syndromes

AbstractMyelodysplastic syndromes (MDS) are in the majority of cases characterized by anemia. Both anemia and MDS per se may directly contribute to impairments in health-related quality of life (HRQoL). In this study, we aimed to investigate the anemia-independent impact of MDS on HRQoL. We evaluated participants (≥ 50 years) from the large population-based Lifelines cohort (N = 44,694, mean age 59.0 ± 7.4 years, 43.6% male) and the European MDS Registry (EUMDS) (N = 1538, mean age 73.4 ± 9.0 years, 63.0% male), which comprises a cohort of lower-risk MDS patients. To enable comparison concerning HRQoL, SF-36 scores measured in Lifelines were converted to EQ-5D-3L index (range 0–1) and dimension scores. Lower-risk MDS patients had significantly lower HRQoL than those from the Lifelines cohort, as illustrated in both the index score and in the five different dimensions. Multivariable linear regression analysis demonstrated that MDS had an adjusted total impact on the EQ-5D index score (B = − 0.12, p < 0.001) and an anemia-independent “direct” impact (B = − 0.10, p < 0.001). Multivariable logistic regression analysis revealed an anemia-independent impact of MDS in the dimension mobility, self-care, usual activities, and anxiety/depression (all except pain/discomfort). This study demonstrates that the major part of the negative impact of lower-risk MDS on HRQoL is not mediated via anemia. Thus, the therapeutic focus should include treatment strategies directed at underlying pathogenic mechanisms to improve HRQoL, rather than aiming predominantly at increasing hemoglobin levels.

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Health-Related Quality of Life Profile and Request of Prognostic Information on Survival At the Time of Diagnosis in Patients with High-Risk Myelodysplastic Syndromes

Blood ◽

10.1182/blood.v118.21.2078.2078 ◽

2011 ◽

Vol 118 (21) ◽

pp. 2078-2078

Author(s):

Fabio Efficace ◽

Gianluca Gaidano ◽

Reinhard Stauder ◽

Giovanni Caocci ◽

Maria Teresa Voso ◽

...

Keyword(s):

Quality Of Life ◽

High Risk ◽

Myelodysplastic Syndromes ◽

Health Related Quality ◽

Prognostic Information ◽

Related Quality ◽

Eortc Qlq C30 ◽

Health Related ◽

Eortc Qlq

Abstract Abstract 2078 Background: Health-related quality of life profile (HRQOL) of patients diagnosed with high-risk myelodysplastic syndromes (MDS) can be compromised already at the time of diagnosis before receiving any kind of treatment. Clinical decision-making is challenging due to the poor prognosis and no data exist on the possible relationships between patient's HRQOL and the request of prognostic information on survival during consultation. Aim: The main objectives were to assess preferences for prognostic information of patients with high-risk MDS and the relationship between such preferences and patient characteristics including HRQOL. To date no such evidence exists in this population. Patients and Methods: Data were gathered through an ongoing international prospective observational study that recruits newly diagnosed patients with MDS. These patients typically have a limited life expectancy. At the time of diagnosis, and during one of the first clinical consultations in which treatment options were discussed, patients completed the European Organization for Research and treatment of Cancer, Quality of life Questionnaire (EORTC QLQ-C30). The EORTC QLQ-C30 is a psychometrically robust generic HRQOL cancer measure assessing both symptoms and functional aspects. Physicians also completed an extensive survey about their patient's preference for involvement in treatment decisions and whether the patient explicitly requested prognostic information for survival. Associations between request for prognostic information, HRQOL socio-demographic characteristics (i.e., living arrangements, age, gender, education) and clinical data including: performance status, comorbidity and disease severity (i.e. IPSS risk category intermediate 2 vs. high risk) were investigated using Fisher's exact test and Wilcoxon-Mann-Whitney test as appropriate. Results: Overall, 184 patients (36% female and 64% male) were analyzed. Mean age of patients was 70 years (range: 31–88). 65% explicitly requested information about expected survival at the time of diagnosis. The symptom profile of patients requesting prognostic information was better than those who did not in 7 out of the 8 symptoms evaluated. The largest clinically meaningful difference was found for fatigue with a mean score of 39 (SD:26) and 52 (SD:28) respectively for those requesting prognostic information versus those who did not. Request for prognostic information was significantly associated with younger age (P=.01) and fewer comorbidities (P=.04). In addition, better physical functioning (P=.009), better role functioning (P=.002) and a lower level of fatigue (P=.002) were also associated with a request for prognostic information during consultation. Additional supportive analysis revealed that patients with a higher overall mean symptom score did not request information about survival (P=.02). Conclusion: These data suggest that the majority of patients with high-risk MDS request prognostic information on survival from their physicians at the time of diagnosis. There is also an indication that patients who are more likely to request such information are those who are in better health condition reporting higher functional abilities and lower symptoms. Disclosures: No relevant conflicts of interest to declare.

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Health-related quality of life and symptom assessment in clinical research of patients with hematologic malignancies: where are we now and where do we go from here?

Haematologica ◽

10.3324/haematol.11710 ◽

2007 ◽

Vol 92 (12) ◽

pp. 1596-1598 ◽

Cited By ~ 27

Author(s):

F. Efficace ◽

A. Novik ◽

M. Vignetti ◽

F. Mandelli ◽

C. S. Cleeland

Keyword(s):

Quality Of Life ◽

Clinical Research ◽

Symptom Assessment ◽

Hematologic Malignancies ◽

Health Related Quality ◽

Related Quality ◽

Health Related

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Health-Related Quality of Life Outcomes in Patients with Myelodysplastic Syndromes with Ring Sideroblasts Treated with Luspatercept in the MEDALIST Phase 3 Trial

Journal of Clinical Medicine ◽

10.3390/jcm11010027 ◽

2021 ◽

Vol 11 (1) ◽

pp. 27

Author(s):

Esther Natalie Oliva ◽

Uwe Platzbecker ◽

Guillermo Garcia-Manero ◽

Ghulam J. Mufti ◽

Valeria Santini ◽

...

Keyword(s):

Quality Of Life ◽

Myelodysplastic Syndromes ◽

Red Blood Cell Transfusion ◽

Health Related Quality ◽

Ring Sideroblasts ◽

Related Quality ◽

Eortc Qlq C30 ◽

Health Related ◽

Eortc Qlq

Patients with myelodysplastic syndromes (MDS) often experience chronic anemia and long-term red blood cell transfusion dependence associated with significant burden on clinical and health-related quality of life (HRQoL) outcomes. In the MEDALIST trial (NCT02631070), luspatercept significantly reduced transfusion burden in patients with lower-risk MDS who had ring sideroblasts and were refractory to, intolerant to, or ineligible for prior treatment with erythropoiesis-stimulating agents. We evaluated the effect of luspatercept on HRQoL in patients enrolled in MEDALIST using the EORTC QLQ-C30 and the QOL-E questionnaire. Change in HRQoL was assessed every 6 weeks in patients receiving luspatercept with best supportive care (+ BSC) and placebo + BSC from baseline through week 25. No clinically meaningful within-group changes and between-group differences across all domains of the EORTC QLQ-C30 and QOL-E were observed. On one item of the QOL-E MDS-specific disturbances domain, patients treated with luspatercept reported marked improvements in their daily life owing to the reduced transfusion burden, relative to placebo. Taken together with previous reports of luspatercept + BSC reducing transfusion burden in patients from baseline through week 25 in MEDALIST, these results suggest luspatercept may offer a treatment option for patients that reduces transfusion burden while providing stability in HRQoL.

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