塞浦路斯接受药物治疗的地中海贫血患者的生存率。1980年至2004年期间的趋势和风险因素。

Survival of medically treated thalassemia patients in Cyprus. Trends and risk factors over the period 1980-2004.

作者信息

Telfer Paul, Coen Pietro G, Christou Soteroula, Hadjigavriel Michael, Kolnakou Anita, Pangalou Evangelia, Pavlides Nicos, Psiloines Michael, Simamonian Krikor, Skordos Georghios, Sitarou Maria, Angastiniotis Michael

机构信息

Department of Paediatric Haematology and Oncology, St. Bartholomew's and The Royal London Hospitals NHS Trust, UK.

出版信息

Haematologica. 2006 Sep;91(9):1187-92.

PMID:16956817

Abstract

BACKGROUND AND OBJECTIVES

A large number of patients with thalassemia major have been born and treated exclusively in Cyprus. They have been managed according to standard international practice, but few have been transplanted. In 1999, a combination chelation regime with desferrioxamine and deferiprone was introduced. We analyzed survival trends in Cypriots and tried to identify factors associated with prolonged survival.

DESIGN AND METHODS

We had incomplete information on births pre-1974 and complete information from 1974 onwards. Clinical data were incomplete pre-1980 and complete thereafter. We analyzed data on 539 patients born after 1960 and followed over the period 1980 to the end of 2004.

RESULTS

There were 58 deaths, 31 (53.4%) of which where due to cardiac causes. In the complete birth cohort of 284 patients born after 1974, survival (95% CI) at 10, 20 and 30 years was 100% (0); 98.5% (96.1-99.4) and 92.7% (86.7-96.1) respectively. There was a significant trend of increasing cardiac deaths between 1980 and 2000 (p<0.001) and a decline after 2000 (p=0.06). In multivariate survival analysis, protective effects were found for female sex (hazard ratio, 0.37, 95% CI 0.21-0.66; p<0.001), and post-2000 follow-up (hazard ratio, 0.44, 95% CI 0.20-0.99; p<0.05), but not for genotype, treatment center or birth cohort.

INTERPRETATION AND CONCLUSIONS

Most patients born after 1974 survive to at least the age of 30. There has been a marked improvement in survival for patients of all ages since 2000, which may be due to the introduction of combination chelation therapy.

摘要

背景与目的

大量重型地中海贫血患者在塞浦路斯出生并接受治疗。他们一直按照国际标准治疗方案进行管理，但接受移植的患者较少。1999年，引入了去铁胺和地拉罗司联合螯合疗法。我们分析了塞浦路斯患者的生存趋势，并试图确定与长期生存相关的因素。

设计与方法

我们掌握1974年以前出生信息不完整，1974年以后信息完整。1980年以前临床数据不完整，之后完整。我们分析了1960年以后出生且在1980年至2004年底接受随访的539例患者的数据。

结果

共58例死亡，其中31例（53.4%）死于心脏相关原因。在1974年以后出生的284例完整出生队列中，10年、20年和30年生存率（95%可信区间）分别为100%（0）；98.5%（96.1 - 99.4）和92.7%（86.7 - 96.1）。1980年至2000年心脏死亡呈显著上升趋势（p<0.001），2000年后呈下降趋势（p = 0.06）。在多变量生存分析中，发现女性（风险比，0.37，95%可信区间0.21 - 0.66；p<0.001）和2000年后随访（风险比，0.44，95%可信区间0.20 - 0.99；p<0.05）具有保护作用，但基因型、治疗中心或出生队列无此作用。