| Summary: | 碩士 === 高雄醫學大學 === 臨床藥學研究所 === 100 === Background: Since the launch of Imatinib, the survival and disease progression of chronic myeloid leukemia (CML) has significantly improved, but also resulted in enormous increase in long-term costs. However, neither clinical outcomes nor health-related utility of CML patients has been investigated in Taiwan, and there is a lack of cost-effectiveness evidence to inform the decision-making on long-term resource allocation.
Aim and objectives: This study aimed to explore the medical utilization and clinical outcomes of imatinib treatment for CML, patients'' perception of disease and treatments, factors contributing to CML patients'' quality of life (QoL), and applicableness of preference-based QoL measurement tools for CML patients treated with imatinib in Taiwan.
Methods: (1) Systematic review: A structured search strategy was used to search evidence-based medicine database for studies reported health status or health preference for CML patient who has ever been treated with imatinib or normal population. (2) Chart review: A cohort study was conducted from January 2000 to October 2011 at a medical center in southern Taiwan. Records were followed from first imatinib prescription to patient died or the last record of chart, or chart review date. CML patients who received imatinib were included to collect patients'' basic information, prescribing history, laboratory data, and physical examination to evaluate prescribing pattern and clinical outcome (3) Patient interview: This cross-sectional survey was conducted from October 2011 to March 2012 at oncology outpatient clinics in the study centre. Patients who were diagnosed as CML, treated with imatinib were invited to participate. A semi-structure interview was conducted and the EuroQol group 5-dimension index (EQ-5D index) questionnaire, 100-mm visual analogue scale (EQ-5D VAS), and time-trade off (TTO) was used to evaluate participants'' utility.
Results: (1) Systematic review: 6 papers of the health status investigated chronic phase CML patients. Although resistance or intolerance to imatinib during long-term treatment, patients'' QoL appeared to be similar to normal population. However, young or female patients had marked limitations in social roles due to physical and emotional problems. Regardless disease stage, utility significantly associated with treatment response. In addition, significant cross country different was found for paitents at the same disease stages.
(2) Chart review: Of all, 124 patients were included and the mean follow-up period is approximately 3.8 year. Mean prescription possession ratios (PPR) were 89.73% and 29.41% for all patients whose imatinib treatment interrupted during follow-up, and PPR was associated with grade II thrombocytopenia. After 18 month of imatinib treatment, 67.24% of the patients achieved complete cytogenetic response. PPR was significantly associated with short-term treatment responses.
(3) Patient interview: Of all, 42 participants (mean age: 50.0±16 years, male: 54.5%) completed the interview survey. Mean utility of EQ-5D index, EQ-5D VAS, and TTO were 0.80±0.08, 0.78±0.13, and 0.80±0.18, respectively. Most patients thought disease become chronic was acceptable, and thought daily living was no longer different from general population. The distress about medication was due to side effect and drug-adhererence. Family economic burden was also a consideration after long-tern care.
Conclusion: Most CML patients treated with imatinib achieved short-term treatment responses, but adherence and treatment interruption were the concerns for long-term outcomes. However, since long-term clinical and cost-effectiveness of imatinib have not yet confirmed, future research on gathering evidence for cost-effectiveness studies are recommanded.
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