INTRODUCTION: Patients with myelodysplastic syndromes (MDS) are at continuous risk of complications and must face the reality of a relatively low survival while still mainly depending on supportive care. The chronic nature of MDS, the risk of progressive evolution, advanced age, anemia and the rapid changes in physical status make the MDS patient unique within the cancer population. Quality of Life (QoL) in MDS is undoubtedly compromised by functional, psychological, and disease-specific components. Treatment options are still scarce in this elderly population and hematologists must select candidates for experimental therapy. Patients’ preferences are requisites for therapeutic choice so that it is essential that clinicians understand patients’ perceptions.

STUDY DESIGN: We designed a multicenter Italian 18-month prospective observational study in 150 MDS patients from diagnosis to evaluate the correlation between QoL changes and changes in Hb and other MDS-related variables. Comparison between the patients’ and the physicians’ perception of QoL, QoL changes related to adaptation, progression and treatment response, and feasibility, consistency and validity of the QoL instruments were evaluated. Selection criteria included adult age, MDS at diagnosis with IPSS score < 2, at least 1 cytopenia, ability to complete the QoL questionnaires and ECOG PS < 3.

METHODS: The 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. Clinical and laboratory data were collected throughout the study.

RESULTS: Median age was 74 (IQ range 67–79); 85 were males (57%). Patients were classified according to WHO: 48 RA (32%), 30 RCMD (20%), 21 RAEB-1 (14%), 7 RAEB-2 (5%), 6 5q-syndrome (4%), 4 RARS (3%), 3 CMML (2%), 1 RCMD/RS (1%), 30 MDS-U (20%). Fifty-six patients (37%) had IPSS low risk, 62 (41%) Int-1 and 14 (9%) Int-2. In 18 (12%) patients IPSS was not evaluable due to karyotype. Thirty-nine patients (26%) were transfusion-dependent (TD) and 28 (72%) had at least 1 additional cytopenia. In the transfusion-free (TF) patients, median Hb was 10.5 g/dL (IQ range 9.2–12.6); 77 patients (69%) were anemic (Hb < 12.0 g/dL) and 48 (43%) had at least 1 additional cytopenia. ECOG PS was significantly better in TF patients (p=0.0061). Charlson’s comorbidity index was 0 in 78 patients (52%), 1 in 38 patients (25%), 2 in 20 patients (13%) and ≥ 3 in 14 patients (9%). Median serum epo was 50.4 U/L (N=94). Of the 29 QOL-E items, 25 items had a response rate > 90%. Though 26 of the 29 QOL-E items showed significant correlations between patients’ and physicians’ scores, there were significant score differences in 9 (31%) of the items with a general trend of physicians to overestimate patients’ problems. Several QoL scores correlated with Hb levels and were significantly lower in TD patients than in TF patients (Table).

Table. Patient QoL scores in transfusion-dependent (TD) and transfusion-free (TF) patients and correlations with Hb values.

QOL domainWhole groupCorrelation with Hb r (p)TD patients (n = 39)TF patients (n = 111)p
QOL-E, scores      
Q1 33 (33–67) 0.186 (0.023) 33 (0–33) 33 (33–67) 0.036 
Q2 67 (67–100) −0.032 (0.70) 67 (33–100) 67 (67–67) 0.11 
Physical 63 (38–75) 0.286 (0.0004) 50 (38–63) 63 (50–75) 0.0072 
Social 50 (25–63) 0.197 (0.017) 44 (17–63) 50 (38–63) 0.064 
Sexual 100 (50–100) 0.250 (0.011) 50 (0–100) 100 (75–100) 0.0011 
Fatigue 81 (71–86) 0.236 (0.0038) 78 (67–86) 81 (71–90) 0.14 
MDS-specific 81 (71–90) 0.287 (0.0005) 74 (63–83) 83 (74–93) 0.0005 
LASA, scores      
Energy 48 ± 25 0.285 (0.0004) 42 ± 25 50 ± 25 0.081 
Activity 54 ± 27 0.226 (0.0055) 50 ± 29 56 ± 26 0.24 
General 53 ± 27 0.304 (0.0002) 44 ± 25 57 ± 27 0.0093 
EQ-5D, scores      
Mobility 50 (50–100) 0.119 (0.15) 50 (50–100) 75 (50–100) 0.45 
Self-Care 100 (100–100) 0.148 (0.074) 100 (100–100) 100 (100–100) 0.14 
Usual Activity 100 (50–100) 0.129 (0.12) 50 (50–100) 100 (50–100) 0.0050 
Pain/Discomfort 50 (50–100) −0.027 (0.75) 100 (50–100) 50 (50–100) 0.48 
Anxiety/Depression 50 (50–100) 0.010 (0.90) 50 (50–100) 50 (50–100) 0.88 
VAS Health State 60 ± 20 0.212 (0.010) 52 ± 19 63 ± 19 0.0020 
QOL domainWhole groupCorrelation with Hb r (p)TD patients (n = 39)TF patients (n = 111)p
QOL-E, scores      
Q1 33 (33–67) 0.186 (0.023) 33 (0–33) 33 (33–67) 0.036 
Q2 67 (67–100) −0.032 (0.70) 67 (33–100) 67 (67–67) 0.11 
Physical 63 (38–75) 0.286 (0.0004) 50 (38–63) 63 (50–75) 0.0072 
Social 50 (25–63) 0.197 (0.017) 44 (17–63) 50 (38–63) 0.064 
Sexual 100 (50–100) 0.250 (0.011) 50 (0–100) 100 (75–100) 0.0011 
Fatigue 81 (71–86) 0.236 (0.0038) 78 (67–86) 81 (71–90) 0.14 
MDS-specific 81 (71–90) 0.287 (0.0005) 74 (63–83) 83 (74–93) 0.0005 
LASA, scores      
Energy 48 ± 25 0.285 (0.0004) 42 ± 25 50 ± 25 0.081 
Activity 54 ± 27 0.226 (0.0055) 50 ± 29 56 ± 26 0.24 
General 53 ± 27 0.304 (0.0002) 44 ± 25 57 ± 27 0.0093 
EQ-5D, scores      
Mobility 50 (50–100) 0.119 (0.15) 50 (50–100) 75 (50–100) 0.45 
Self-Care 100 (100–100) 0.148 (0.074) 100 (100–100) 100 (100–100) 0.14 
Usual Activity 100 (50–100) 0.129 (0.12) 50 (50–100) 100 (50–100) 0.0050 
Pain/Discomfort 50 (50–100) −0.027 (0.75) 100 (50–100) 50 (50–100) 0.48 
Anxiety/Depression 50 (50–100) 0.010 (0.90) 50 (50–100) 50 (50–100) 0.88 
VAS Health State 60 ± 20 0.212 (0.010) 52 ± 19 63 ± 19 0.0020 
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Data are reported as mean ± standard deviation or median and interquartile range as appropriate.

CONCLUSIONS: QoL is compromised in MDS and is associated with Hb values and transfusion-dependence. Patient preferences must be evaluated when considering treatment options since hematologists seem to overestimate patients’ perception of healthrelated problems. The disease-specific questionnaire, QOL-E, and the LASA scale are valid tools for the evaluation of QoL in MDS patients.

Topics:
myelodysplastic syndrome, quality of life assessment, transfusion, cytopenia, lipid-associated sialic acid, prostatic hypertrophy risk score, anemia, cytopenia, refractory, with multilineage dysplasia, electrocorticogram, refractory anemia with excess blasts

Disclosures: No relevant conflicts of interest to declare.

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Corresponding author

2008, The American Society of Hematology
2008
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