Contributions
Abstract: PB2367
Type: Publication Only
Background
In Greece, the Quality of Life (QoL) in Transfusion-Dependent Thalassemia (TDT) has been studied in only small groups of patients and all assessments involved the administration of generic QoL questionnaires. TranQol, is a novel, disease-specific QoL questionnaire for TDT patients that has been recently validated and will be administered in this study
Aims
The aim of this study is to evaluate the QoL of TDT patients in Greece using a novel, disease-specific QoL questionnaire (TranQol) in combination with a generic QoL questionnaire (SF-36).
Methods
In this multicentre study, we recruited 288 consecutive adult TDT patients from four Thalassemia Centers in Greece, according to specific inclusion and exclusion criteria. The participants had a mean age of 32±7 years, 42% were male and 58% were female. Demographic and clinical characteristics were retrieved from the patients' medical records. All participants completed a set of two QoL questionnaires, the generic SF-36v2 and the disease-specific TranQol, during their scheduled transfusion visits. The data from SF-36v2 were used in order to compare the QoL of TDT patients with the healthy population in Greece. The more sensitive TranQol was used to describe possible differences in QoL between the following subgroups of TDT patients 1) type of iron chelation therapy 2) presence of co-morbidities 3) level of liver hemosiderosis. Parametric and non-parametric tests will be used to compare data with or without normal distribution, respectively. Statistical significance is defined as p < 0.05
Results
Compared to the available data from the healthy population in Greece, the SF-36 scores of TDT patients were lower in all Qol domains which involved Physical Functioning (81% vs 50%), Role Physical (79% vs 50%), Bodily Pain (73% vs 52%), General Health (67% vs 47%), Vitality (66% vs 55%), Social Functioning (82% vs 50%), Role Emotional (82% vs 47%) and Mental Health (68% vs 49%). The mean summary score of TranQol was 71%±15, whereas the scores of the domains physical health, emotional health, sexuality, family functioning and school/career where 71%±15, 67%±17, 77%±23, 74%±19 and 75%±21 respectively. The mean TranQol summary score was lower for patients treated with Deferiprone compared to patients treated with Deferoxamine (mean difference=11%, 95% C.I: 2-21), Deferasirox (mean difference=12%, 95% C.I: 15-21) or with the combination of Deferiprone-Deferoxamine (mean difference=12%, 95% C.I :15-21). Assessment of comorbidities revealed that patients with osteoporosis have significantly lower TranQol scores (70%±14 vs 74%±13). The TranQol mean scores did not differ between groups with different levels of liver iron but patients with mild liver hemosiderosis had lower physical health scores compared to patients with normal levels of liver iron (mean difference=7%, 95% C.I: 6-14).
Conclusion
This is the first QoL study in a representative sample of the TDT population in Greece. Compared to the healthy population, TDT patients have a lower QoL. The use of a disease-specific QoL questionnaire revealed distinct groups of TDT patients with worse QoL. Our findings could help the physicians to early recognize these groups of TDT patients and help them improve their QoL. In the era of novel treatments for TDT patients, each new drug should be evaluated in terms of both clinical efficacy and improvement in health-related QoL. Our results may provide a baseline index score of QoL in TDT patients to be compared with relevant outcomes from future clinical trials
Session topic: 36. Quality of life, palliative care, ethics and health economics
Keyword(s): Quality of Life, Thalassemia
Abstract: PB2367
Type: Publication Only
Background
In Greece, the Quality of Life (QoL) in Transfusion-Dependent Thalassemia (TDT) has been studied in only small groups of patients and all assessments involved the administration of generic QoL questionnaires. TranQol, is a novel, disease-specific QoL questionnaire for TDT patients that has been recently validated and will be administered in this study
Aims
The aim of this study is to evaluate the QoL of TDT patients in Greece using a novel, disease-specific QoL questionnaire (TranQol) in combination with a generic QoL questionnaire (SF-36).
Methods
In this multicentre study, we recruited 288 consecutive adult TDT patients from four Thalassemia Centers in Greece, according to specific inclusion and exclusion criteria. The participants had a mean age of 32±7 years, 42% were male and 58% were female. Demographic and clinical characteristics were retrieved from the patients' medical records. All participants completed a set of two QoL questionnaires, the generic SF-36v2 and the disease-specific TranQol, during their scheduled transfusion visits. The data from SF-36v2 were used in order to compare the QoL of TDT patients with the healthy population in Greece. The more sensitive TranQol was used to describe possible differences in QoL between the following subgroups of TDT patients 1) type of iron chelation therapy 2) presence of co-morbidities 3) level of liver hemosiderosis. Parametric and non-parametric tests will be used to compare data with or without normal distribution, respectively. Statistical significance is defined as p < 0.05
Results
Compared to the available data from the healthy population in Greece, the SF-36 scores of TDT patients were lower in all Qol domains which involved Physical Functioning (81% vs 50%), Role Physical (79% vs 50%), Bodily Pain (73% vs 52%), General Health (67% vs 47%), Vitality (66% vs 55%), Social Functioning (82% vs 50%), Role Emotional (82% vs 47%) and Mental Health (68% vs 49%). The mean summary score of TranQol was 71%±15, whereas the scores of the domains physical health, emotional health, sexuality, family functioning and school/career where 71%±15, 67%±17, 77%±23, 74%±19 and 75%±21 respectively. The mean TranQol summary score was lower for patients treated with Deferiprone compared to patients treated with Deferoxamine (mean difference=11%, 95% C.I: 2-21), Deferasirox (mean difference=12%, 95% C.I: 15-21) or with the combination of Deferiprone-Deferoxamine (mean difference=12%, 95% C.I :15-21). Assessment of comorbidities revealed that patients with osteoporosis have significantly lower TranQol scores (70%±14 vs 74%±13). The TranQol mean scores did not differ between groups with different levels of liver iron but patients with mild liver hemosiderosis had lower physical health scores compared to patients with normal levels of liver iron (mean difference=7%, 95% C.I: 6-14).
Conclusion
This is the first QoL study in a representative sample of the TDT population in Greece. Compared to the healthy population, TDT patients have a lower QoL. The use of a disease-specific QoL questionnaire revealed distinct groups of TDT patients with worse QoL. Our findings could help the physicians to early recognize these groups of TDT patients and help them improve their QoL. In the era of novel treatments for TDT patients, each new drug should be evaluated in terms of both clinical efficacy and improvement in health-related QoL. Our results may provide a baseline index score of QoL in TDT patients to be compared with relevant outcomes from future clinical trials
Session topic: 36. Quality of life, palliative care, ethics and health economics
Keyword(s): Quality of Life, Thalassemia