Prognosis and Overall Survival of Thalassemia Patients using Semi-Parametric Mixture Cure-Rate Model: a 30-Year Retrospective Cohort Study

CLINICAL LABORATORY(2022)

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摘要
Background: Understanding the determinants of long-term overall survival (OS) of thalassemia patients (TPs) is the mainstay of care. Methods: As a retrospective survey, we assessed the data of 769 TPs who had regular follow-up and blood transfu-sion for at least 30 years from 1990 -2019. We utilized semi-parametric proportional hazards mixture cure-rate regression to discover the factors with a significant effect on short-and long-term OS separately. Results: The 25-and 30-year OS for the TPs were calculated to be 98.7% and 90.4%, respectively. Each five-year age escalation was associated with a 30% decrease in the probability of being short-term survivors (HR = 1.06, p = 0.047). Parental family relationship influenced both cured (OR = 3.00, p = 0.017) and uncured (HR = 0.50, p = 0.046) TPs. Moreover, the type of iron chelation drug, liver iron concentration, and normal EF results had a sig-nificant effect on long-term OS. Aging, parental consanguinity, liver and cardiac siderosis, higher ferritin levels, and low hemoglobin level were associated with poorer prognosis in TPs. Conclusions: However, deferoxamine followed by multiple drugs as iron chelation, severe liver siderosis, and ab -normal EF declined the probability of long-term OS among TPs. This can be considered by health policy decision-makers to enforce the screening program more strictly. (Clin. Lab. 2022;68:xx-xx. DOI: 10.7754/Clin.Lab.2021.211016)
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关键词
cure-rate analyses, iron chelating agent, ejection frac-tion, beta thalassemia
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