Publications by authors named "M Tarkan Aksozen"
Article Synopsis
- - NICE uses quality-adjusted life years (QALYs) to evaluate the cost-effectiveness of treatments, combining clinical benefits with the duration of patient benefits.
- - A study on NICE's evaluations for rare diseases found that the median time horizon for QALY calculations was 97.5 years, often exceeding expected treatment durations or life expectancies.
- - The only treatment with a negative funding decision had a shorter time horizon than the expected treatment duration, raising concerns about the fairness and consistency of NICE's evaluation process.
Article Synopsis
- Erythropoietic protoporphyria (EPP) is a rare metabolic disorder that causes severe pain from light exposure, making it hard to gauge patients' quality of life (QoL).
- A feasibility study involved five EPP patients on long-term treatment with afamelanotide, assessing QoL using the EQ-5D tool, especially after they experienced treatment interruptions.
- Findings showed that patients' QoL while treated was similar to the general population, and their experiences during treatment interruptions compared to patients with chronic pain and burn injuries, supporting afamelanotide's cost-effectiveness evaluation in England.
Conditioning regimens used during stem cell transplant provide prolonged control or cure of the disease in patients with acute lymphoblastic leukemia (ALL). In this study, we present a comparison of treatment results for 95 patients with ALL who underwent allogeneic hematopoietic stem cell transplant (AHSCT) with total body irradiation plus cyclophosphamide (TBI + Cy) or busulfan plus cyclophosphamide (Bu + Cy) as conditioning regimen. Median age was 25 (range: 9-54) years.
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