Shargian-Alon Liat, Pasvolsky Oren, Raanani Pia
Institute of Hematology, Rabin Medical Center (Beilinson Campus), Petah Tikva, associated with Sackler Faculty of Medicine, Tel Aviv University, Tel Aviv, Israel.
Isr Med Assoc J. 2017 Dec;19(12):767-771.
During the past decades, beta thalassemia major (TM) and beta thalassemia intermedia (TI) have transformed from a universally fatal disease at a young age into a chronic disease. This advancement is attributed to improved chelation therapy as well as enhanced management strategies, with focused attention on disease and treatment-related complications.
To describe characteristics of adults with thalassemia as well as treatment modalities, disease and treatment-related complications, and socioeconomic information of the patients.
We preformed a retrospective analysis of 14 adult patients > 35 years of age with TM and TI who were treated at our institute, a single center specializing in the care of adult thalassemia patients living in Israel, between the years 2006 and 2016.
The median age of patients was 37 years and most patients were transfusion-dependent. The median number of chelation therapeutic lines was three, and 85.7% of patients were treated at one point by combination chelation therapy. Most patients suffered from at least some form of endocrine dysfunction (n=12), and four patients developed overt heart failure. Of the patients, 85% had completed at least a high school education, 78% were employed, and 64.2% were married.
Prolonged survival of thalassemia patients in recent years has been accompanied by a new set of challenges for both the patients and the treating staff. Further research is warranted to improve both medical management and the socioeconomic well-being of this unique group of adult thalassemia patients.
在过去几十年中，重型β地中海贫血（TM）和中间型β地中海贫血（TI）已从一种普遍在年轻时致命的疾病转变为一种慢性病。这一进展归因于螯合疗法的改进以及管理策略的加强，同时重点关注了与疾病和治疗相关的并发症。
描述地中海贫血成年患者的特征、治疗方式、与疾病和治疗相关的并发症以及患者的社会经济信息。
我们对2006年至2016年间在我们研究所接受治疗的14例年龄大于35岁的TM和TI成年患者进行了回顾性分析。我们的研究所是一家专门照顾居住在以色列的成年地中海贫血患者的单一中心。
患者的中位年龄为37岁，大多数患者依赖输血。螯合治疗线的中位数为三条，85.7%的患者曾在某一时刻接受过联合螯合治疗。大多数患者至少患有某种形式的内分泌功能障碍（n = 12），4例患者出现明显的心衰。在这些患者中，85%至少完成了高中学业，78%受雇，64.2%已婚。
近年来地中海贫血患者的长期生存给患者和治疗人员都带来了一系列新的挑战。有必要进行进一步研究，以改善这一独特成年地中海贫血患者群体的医疗管理和社会经济福祉。
