Quality of Life in Myelodysplastic Syndromes and Physicians' Perception.

The heterogeneous nature of myelodysplastic syndromes (MDS), age- and disease-related factors (complications and progression to acute leukemia) are associated with the complexity of quality of Life (QoL). Patients are offered mainly non-curative or experimental drugs and/or supportive care. The exploration of QoL in MDS is a prerequisite for adequate therapeutic choice.

We designed an observational study in MDS patients with IPSS risk score ≤2 to evaluate determinants of QoL and its correlates. Clinical and laboratory data were collected up to 18 months and QoL instruments (QOL-E v.2, LASA scale, and EQ-5D) were completed by patients and physicians (both blind to each other's responses) at baseline, months 1, 3, 6, 12, and 18. After diagnosis, treatment was assigned based on investigators' judgment.

Of 148 patients enrolled (mean age 72 years, 56% males), 115 (78%) patients were anemic at diagnosis and 38 (26%) had already received transfusions. Mean (± SD) Hb was 10.3 ± 2.1, ANC 2.3 ± 1.9×10³/μL and PLT count 155 ± 118×10³/μL. Charlson's Comorbidity Score was > 1 in 33 (22%). Physical and functional QoL, health, energy, activity and general states were generally poor (<65). In 94 cases ECOG PS was assigned a 0 (best) value, though 36 patients had a physical QoL-E score < 60. Surprisingly, there were significant correlations between patients' and physicians' QoL-E scores in all but those regarding the disturbances due to dependency on hospital and staff and to the inability to travel. However, MDS-specific well-being was systematically overestimated by physicians in the MDS-specific domain (p<0.0001). After 12 months, 94 patients have completed the QoL instruments. There was a transient increase in Hb with parallel changes in QoL and no significant change at 12 months (only MDS-specific QoL-E scores worsened from 79 ± 17 to 73 ± 22, p=0.005). At univariate analysis, Hb correlated with all QoL-E and LASA activity scores at baseline. After 12 months, Hb correlated with social, fatigue, MDS-specific, health status and LASA scales, while transfusion dependence with MDS-specific QOL-E scores (p<0.01). At multivariate analysis (Table), major determinants of QoL at baseline were comorbidities, IPSS, and Hb; at 12 months QoL was independently predicted by age, comorbidities, transfusion-dependence and mainly by Hb, while thrombocytopenia (PLT < 50000 microliters) and neutropenia did not seem to have a relevant impact.

QoL, together with prognostic scores, should guide therapeutic choice in MDS. Avid research in MDS tends to focus on younger and fit patients to offer more aggressive or novel therapies with the aim of increasing survival. However, patients of advanced age with comorbidities complicated with severe anemia and transfusion-dependence represent a fragile category with particularly poor QoL that actually require special therapeutic attention to favor transfusion-independence and possibly an hemoglobin response.

No relevant conflicts of interest to declare.