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Deferasirox plus deferoxamine is more effective than deferasirox alone for treating iron overload in patients with thalassemia major, according to a single-center study.
Over the course of 1 year of treatment, deferasirox plus deferoxamine significantly increased myocardial T2* and significantly reduced serum ferritin, whereas deferasirox alone had no significant effect on either endpoint. Neither treatment had a significant effect on hepatic iron.
Deferasirox plus deferoxamine caused a significantly greater increase in alanine aminotransferase, aspartate aminotransferase, and bilirubin. Other adverse events were similar between the treatment groups.
Aziz Eghbali, MD, of Arak (Iran) University of Medical Sciences and colleagues described this study in Transfusion and Apheresis Science.
The team conducted a randomized, double-blind trial of patients with thalassemia major. Of the 62 patients enrolled, 55 were randomized and evaluable. At baseline, the mean patient age was 24.5 years, and 67.3% were female.
The patients were randomized to receive oral deferasirox at 30 mg/kg daily either alone (n = 27) or with subcutaneous deferoxamine at 50 mg/kg for 5 days a week (n = 28). In both groups, patients received treatment for 12 months.
There were no significant differences between the groups in baseline characteristics such as myocardial or hepatic iron, transfusion volume, or white blood cell and platelet counts.
Results
The study’s primary endpoints were changes in myocardial T2* and hepatic T2* from baseline to 12 months. Changes in serum ferritin and adverse events were secondary endpoints.
Myocardial T2* decreased slightly in the monotherapy group, from 23.3 plus or minus 7.4 ms at baseline to 22.1 plus or minus 6.9 ms at 12 months (P = .3) but increased significantly in the combination group, from 23.1 plus or minus 7.5 ms to 27.1 plus or minus 7.0 ms (P less than .05). The difference between the groups was significant (P = .01).
There was no significant change in hepatic iron in either group. Hepatic T2* was 7.0 plus or minus 5.6 ms at baseline and 7.0 plus or minus 5.3 ms at 12 months in the monotherapy group (P = .7). In the combination group, hepatic T2* increased from 9.8 plus or minus 8.8 ms to 10.2 plus or minus 8.2 ms (P = .5). The between-group difference was not significant (P = .094).
Serum ferritin decreased from 1,390 plus or minus 816 mcg/ml to 1,085 plus or minus 919 mcg/mL in the monotherapy group (P = .06) and from 1,446 plus or minus 987 mcg/mL to 737 plus or minus 459 mcg/mL in the combination group (P less than .01). The between-group difference was significant (P = .001).
Increases in alanine aminotransferase, aspartate aminotransferase, and bilirubin were significantly greater in the combination group than in the monotherapy group (P less than .05 for all). Combination therapy also prompted an increase in alkaline phosphatase, but this was not significantly greater than in the monotherapy group (P = .3).
Blood urea nitrogen levels increased in both groups, but levels remained within the normal range. There were no increases in serum creatinine in either group.
Rates of mild gastrointestinal adverse events were similar in the monotherapy and combination groups (40% and 39%, respectively), as were rates of transient skin rashes (18% and 14%, respectively).
There were no deaths, and none of the patients stopped treatment because of severe adverse events.
This study was supported by Arak University of Medical Sciences. The researchers reported having no conflicts of interest.
SOURCE: Eghbali A et al. Transfus Apher Sci. 2019 Aug;58(4):429-33.
Deferasirox plus deferoxamine is more effective than deferasirox alone for treating iron overload in patients with thalassemia major, according to a single-center study.
Over the course of 1 year of treatment, deferasirox plus deferoxamine significantly increased myocardial T2* and significantly reduced serum ferritin, whereas deferasirox alone had no significant effect on either endpoint. Neither treatment had a significant effect on hepatic iron.
Deferasirox plus deferoxamine caused a significantly greater increase in alanine aminotransferase, aspartate aminotransferase, and bilirubin. Other adverse events were similar between the treatment groups.
Aziz Eghbali, MD, of Arak (Iran) University of Medical Sciences and colleagues described this study in Transfusion and Apheresis Science.
The team conducted a randomized, double-blind trial of patients with thalassemia major. Of the 62 patients enrolled, 55 were randomized and evaluable. At baseline, the mean patient age was 24.5 years, and 67.3% were female.
The patients were randomized to receive oral deferasirox at 30 mg/kg daily either alone (n = 27) or with subcutaneous deferoxamine at 50 mg/kg for 5 days a week (n = 28). In both groups, patients received treatment for 12 months.
There were no significant differences between the groups in baseline characteristics such as myocardial or hepatic iron, transfusion volume, or white blood cell and platelet counts.
Results
The study’s primary endpoints were changes in myocardial T2* and hepatic T2* from baseline to 12 months. Changes in serum ferritin and adverse events were secondary endpoints.
Myocardial T2* decreased slightly in the monotherapy group, from 23.3 plus or minus 7.4 ms at baseline to 22.1 plus or minus 6.9 ms at 12 months (P = .3) but increased significantly in the combination group, from 23.1 plus or minus 7.5 ms to 27.1 plus or minus 7.0 ms (P less than .05). The difference between the groups was significant (P = .01).
There was no significant change in hepatic iron in either group. Hepatic T2* was 7.0 plus or minus 5.6 ms at baseline and 7.0 plus or minus 5.3 ms at 12 months in the monotherapy group (P = .7). In the combination group, hepatic T2* increased from 9.8 plus or minus 8.8 ms to 10.2 plus or minus 8.2 ms (P = .5). The between-group difference was not significant (P = .094).
Serum ferritin decreased from 1,390 plus or minus 816 mcg/ml to 1,085 plus or minus 919 mcg/mL in the monotherapy group (P = .06) and from 1,446 plus or minus 987 mcg/mL to 737 plus or minus 459 mcg/mL in the combination group (P less than .01). The between-group difference was significant (P = .001).
Increases in alanine aminotransferase, aspartate aminotransferase, and bilirubin were significantly greater in the combination group than in the monotherapy group (P less than .05 for all). Combination therapy also prompted an increase in alkaline phosphatase, but this was not significantly greater than in the monotherapy group (P = .3).
Blood urea nitrogen levels increased in both groups, but levels remained within the normal range. There were no increases in serum creatinine in either group.
Rates of mild gastrointestinal adverse events were similar in the monotherapy and combination groups (40% and 39%, respectively), as were rates of transient skin rashes (18% and 14%, respectively).
There were no deaths, and none of the patients stopped treatment because of severe adverse events.
This study was supported by Arak University of Medical Sciences. The researchers reported having no conflicts of interest.
SOURCE: Eghbali A et al. Transfus Apher Sci. 2019 Aug;58(4):429-33.
Deferasirox plus deferoxamine is more effective than deferasirox alone for treating iron overload in patients with thalassemia major, according to a single-center study.
Over the course of 1 year of treatment, deferasirox plus deferoxamine significantly increased myocardial T2* and significantly reduced serum ferritin, whereas deferasirox alone had no significant effect on either endpoint. Neither treatment had a significant effect on hepatic iron.
Deferasirox plus deferoxamine caused a significantly greater increase in alanine aminotransferase, aspartate aminotransferase, and bilirubin. Other adverse events were similar between the treatment groups.
Aziz Eghbali, MD, of Arak (Iran) University of Medical Sciences and colleagues described this study in Transfusion and Apheresis Science.
The team conducted a randomized, double-blind trial of patients with thalassemia major. Of the 62 patients enrolled, 55 were randomized and evaluable. At baseline, the mean patient age was 24.5 years, and 67.3% were female.
The patients were randomized to receive oral deferasirox at 30 mg/kg daily either alone (n = 27) or with subcutaneous deferoxamine at 50 mg/kg for 5 days a week (n = 28). In both groups, patients received treatment for 12 months.
There were no significant differences between the groups in baseline characteristics such as myocardial or hepatic iron, transfusion volume, or white blood cell and platelet counts.
Results
The study’s primary endpoints were changes in myocardial T2* and hepatic T2* from baseline to 12 months. Changes in serum ferritin and adverse events were secondary endpoints.
Myocardial T2* decreased slightly in the monotherapy group, from 23.3 plus or minus 7.4 ms at baseline to 22.1 plus or minus 6.9 ms at 12 months (P = .3) but increased significantly in the combination group, from 23.1 plus or minus 7.5 ms to 27.1 plus or minus 7.0 ms (P less than .05). The difference between the groups was significant (P = .01).
There was no significant change in hepatic iron in either group. Hepatic T2* was 7.0 plus or minus 5.6 ms at baseline and 7.0 plus or minus 5.3 ms at 12 months in the monotherapy group (P = .7). In the combination group, hepatic T2* increased from 9.8 plus or minus 8.8 ms to 10.2 plus or minus 8.2 ms (P = .5). The between-group difference was not significant (P = .094).
Serum ferritin decreased from 1,390 plus or minus 816 mcg/ml to 1,085 plus or minus 919 mcg/mL in the monotherapy group (P = .06) and from 1,446 plus or minus 987 mcg/mL to 737 plus or minus 459 mcg/mL in the combination group (P less than .01). The between-group difference was significant (P = .001).
Increases in alanine aminotransferase, aspartate aminotransferase, and bilirubin were significantly greater in the combination group than in the monotherapy group (P less than .05 for all). Combination therapy also prompted an increase in alkaline phosphatase, but this was not significantly greater than in the monotherapy group (P = .3).
Blood urea nitrogen levels increased in both groups, but levels remained within the normal range. There were no increases in serum creatinine in either group.
Rates of mild gastrointestinal adverse events were similar in the monotherapy and combination groups (40% and 39%, respectively), as were rates of transient skin rashes (18% and 14%, respectively).
There were no deaths, and none of the patients stopped treatment because of severe adverse events.
This study was supported by Arak University of Medical Sciences. The researchers reported having no conflicts of interest.
SOURCE: Eghbali A et al. Transfus Apher Sci. 2019 Aug;58(4):429-33.
FROM TRANSFUSION AND APHERESIS SCIENCE