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Survival and complication rates in patients with thalassemia major in Taiwan
Author(s) -
Wu HanPing,
Lin ChengLi,
Chang YinChen,
Wu KangHsi,
Lei RouhLih,
Peng ChingTien,
Weng Tefu,
Tai YuMan,
Chao YuHua
Publication year - 2017
Publication title -
pediatric blood and cancer
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.116
H-Index - 105
eISSN - 1545-5017
pISSN - 1545-5009
DOI - 10.1002/pbc.26181
Subject(s) - medicine , thalassemia , chelation therapy , blood transfusion , hazard ratio , cohort , mortality rate , diabetes mellitus , population , quality of life (healthcare) , cirrhosis , deferasirox , complication , heart failure , pediatrics , confidence interval , nursing , environmental health , endocrinology
Background While transfusion and iron chelation therapy for thalassemia major (TM) has improved dramatically in recent years, the consequences of this improvement (current rates of survival and TM‐related complications) remain unknown. Methods This nationwide population‐based cohort study analyzed 2007–2011 data obtained from the Taiwanese National Health Insurance Research Database. Results After excluding those patients receiving hematopoietic stem cell transplantation, we enrolled 454 patients with TM who received transfusion and chelation therapy (median age, 17.2 years). Among these patients, the mortality rate was 2.9% in 2007, 2.3% in 2008, 2.9% in 2009, 2.6% in 2010, and 0.7% in 2011. Heart was the most common target organ of TM‐related complications. There were 80 patients (17.6%) with arrhythmia and 86 patients (18.9%) with congestive heart failure. Dysfunction of endocrine organs was common, and the most common endocrinopathy was hypogonadism (23.1%), followed by diabetes (21.2%). There were 75 patients (16.5%) with liver cirrhosis and 79 patients (17.4%) with osteoporosis. Conclusions Adequate red blood cell transfusion and iron chelation is available to all patients with TM in Taiwan under the universal health insurance system, and has resulted in reduction of TM‐related mortality to very low levels. As these patients get older, early detection of complications and adequate intervention are important to quality‐of‐life improvement.