Survival and complication rates in patients with thalassemia major in Taiwan

Abstract

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.

The effect of iron chelation therapy on overall survival in sickle cell disease and β‐thalassemia: A systematic review

“…A recent study from Taiwan measured the improvement in survival after introduction of deferasirox in 2007 . Although the study notes that the true impact of specific ICT on outcomes could not be evaluated because of the switching of agents due to side effects and the patient condition, the incidence of death decreased from 2.9% in 2007 to 0.7% in 2011 …”

“…A total of 18 individual papers describing 15 different studies were published between 2000 and 2017, and are summarized in Table . These studies included 7189 patients; however, some patients likely were counted more than once since the multicenter studies from Borgna‐Pignatti et al, although distinct, drew on the same patient populations in Italy .…”

“…Only 1 of the retrospective/longitudinal studies compared survival outcomes in β‐thalassemia patients according to the ICT received; a second study compared monotherapy to combination therapy . The remaining studies investigated survival outcomes according to various baseline and treatment characteristics: 1 investigated factors associated with death in a case‐control study; 4 studied outcomes according to compliance with or duration of ICT; 4 compared survival outcomes according to period of treatment (i.e., before or after availability of different iron chelation treatments); 1 measured survival in patients with thalassemia major and thalassemia intermedia before and after introduction of deferoxamine; and 1 measured rates of death in hepatitis C virus (HCV)‐infected and noninfected patients receiving ICT with deferoxamine …”

“…Of note, while there was no difference in the risk of new cardiac events for those with mild or moderate hemosiderosis, there was a significantly increased risk of cardiac events for deferoxamine‐treated patients with severe hemosiderosis versus deferiprone ( P = .005). A recent study from Taiwan measured the improvement in survival after introduction of deferasirox in 2007 . Although the study notes that the true impact of specific ICT on outcomes could not be evaluated because of the switching of agents due to side effects and the patient condition, the incidence of death decreased from 2.9% in 2007 to 0.7% in 2011 …”

“…However, uncontrolled studies suggest that deferiprone may be better tolerated and may be associated with better outcomes than deferoxamine. The only study to date suggesting a survival benefit for deferasirox versus deferiprone was a retrospective analysis, which showed improved survival in Taiwan after the introduction of deferasirox …”