| Objective:The purpose of this study is to guide clinical treatment forβ-thalassemia major（β-TM）by investigating the changes in iron overload（IO）before and after hematopoietic stem cell transplantation（HSCT）in children who had suffered fromβ-TM,and evaluation the safety and efficacy of iron-removal treatment with deferiprone（DFP）.Methods:40 cases ofβ-TM children with IO after HSCT were collected in our hospital’s pediatrics from January 2014 to December 2019,which were divided into 3 groups according to diverse DFP treatment manners:standard treatment group（14 cases）,non-standard treatment group（16 cases）and clinical observation group without any iron-removal treatment also termed as the control group（10 cases）.Use SF value to evaluate IO,all feedback data,such as changes in the IO index before and after HSCT treatment,the therapeutic efficacy,adverse reactions（ADRs）,and related monitoring indicators changes of DFP treatment were conducted with retrospective analysis.Result:1.A total of 40 children were collected in three groups,14 child patients in the standard treatment group,with an average age of 7.8±1.5 years,a male to female ratio of 3.7:1,blood transfusion time before HSCT of 86±20months,and an average DFP dosage of 53.3mg.kg-1.d-1.16 child patients in the non-standard treatment group had an average age of 8.0±1.7,a male to female ratio of 4.3:1,a blood transfusion time before HSCT of 89±18 months,and an average dose of DFP of 34.2±9.7mg.kg-1.d-1.There were 10 child patients in the control group,with an average age of 7.2±1.7 years and a male to female ratio of4:1.The blood transfusion time before HSCT was 78±18 months,and no iron-relieving treatment was received.There were no statistical differences in age,gender composition,and transfusion time before HSCT among the three groups.There were statistical differences in the doses of DFP between the standard treatment group and the non-standard treatment group（P<0.05）.2.In this study,92.5%child patients（37 cases）were original IO before HSCT,of which 60%were severe IO,and the average SF value of these severe cases was 2702±1087ng/ml;One-year after HSCT,100%children were IO,of which 87.5%were severe IO,and the average value of SF was 3866±1119ng/ml.There was a statistically significant increase in the SF value and the proportion of severe IO from before HSCT to 1 year after HSCT.3.The SF value differed from 3 groups.Compared to before treatment,after One-year DFP treatment,The SF value of standard DFP treatment group decreased from 4624±770ng/ml to 2322±553ng/ml with an average drop of2301±556ng/ml;the non-standard treatment group decreased from3727±1107ng/ml to 2494±980ng/ml with an average drop of 1232±421ng/ml;the control group decreased from 2671ng/ml to 2333±830ng/ml with an average drop of 694±203ng/ml.The DFP treatment group exhibited a significantly lower SF value than the control group,and DFP treatment could more effectively alleviate the SF value.At the same time,the proportion of severe IO in the standard treatment group decreased from 100%before treatment to 35.75%after treatment,the difference was statistically significant（P<0.05）;However,the proportion of severe IO in the non-standard treatment group and the control groups decreased from 81.25%before treatment to 50%after treatment and 80%to 30%,both which showed no sense of statistics（P>0.05）.4.14 cases with ADRs were recorded in 30 child patients during 1-year DFP treatment.The common ADRs were gastrointestinal reactions（nausea,vomiting,increased appetite,abdominal pain）,all of which were reversible events.There were no neutropenia and hematopoietic agranulocytosis,no ADRs other than instructions occurred.No statistical difference in ADRs between the standard treatment group and the non-standard treatment group was obtained.5.Aspartate aminotransferase（AST）value decreased from 48.4±18.8U/L before treatment to 36.8±17.4U/L post-treatment,and alanine aminotransferase（ALT）decreased from 49.9±13.8U/L before treatment to 32.2±9.7 U/L,the difference was statistically significant（P<0.05）;AST value decreased from45.0±18.3U/L before treatment to 29.0±5.3U/L,and ALT decreased from46.9±16.4 before treatment to 26.2±9.5U/L,and the difference was also statistically significant（P<0.05）;There was no significant difference in ALT and AST between children in the control group.Conclusion:1.By monitoring the changes of SF,we found that the IO degree aggravated obviously after HSCT treatment than before,and iron-removal therapy was also needed.2.The application of DFP treatment in child patients with IO after HSCT could effectively reduce the SF level,and standard DFP treatment could more effectively alleviate children’s severe IO condition.3.DFP is an innocuous iron chelator for child patients with IO after HSCT.Compared with non-standard DFP treatment,the application of standard DFP treatment for IO child patients after HSCT shows no apparent increment of ADRs.4.The use of DFP in child patients with IO after HSCT can reduce abnormally elevated AST and ALT values. |
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