Efficacy and safety of deferasirox in pediatric patients of thalassemia at a Tertiary Care Teaching Hospital
Objective: To evaluate efficacy, safety and utilization pattern of deferasirox in paediatric patients of transfusion dependant β Thalassemia Major at a tertiary care teaching hospital in Gujarat. Materials and Methods: This observational, prospective-retrospective, single centre, continuous study wa...
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Thieme Medical and Scientific Publishers Pvt. Ltd.
2017-01-01
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| Series: | Indian Journal of Medical and Paediatric Oncology |
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| Online Access: | http://www.ijmpo.org/article.asp?issn=0971-5851;year=2017;volume=38;issue=2;spage=103;epage=110;aulast=Thakor |
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| author | Dhaval R Thakor Chetna K Desai Jigar D Kapadia Ram K Dikshit K M Mehariya |
| author_facet | Dhaval R Thakor Chetna K Desai Jigar D Kapadia Ram K Dikshit K M Mehariya |
| author_sort | Dhaval R Thakor |
| collection | DOAJ |
| description | Objective: To evaluate efficacy, safety and utilization pattern of deferasirox in paediatric patients of transfusion dependant β Thalassemia Major at a tertiary care teaching hospital in Gujarat. Materials and Methods: This observational, prospective-retrospective, single centre, continuous study was conducted in a tertiary care teaching hospital among paediatric patients of transfusion dependent β Thalassemia Major. Patients treated with deferasirox for not more than 12 weeks were enrolled. Details of blood transfusions, relevant investigations performed every 3 weeks and 3 months and drugs used were recorded in a pretested case record form. Parents were provided with a diary to record the details of ADRs. Data were analyzed for demographic characteristics, number and mean volume of blood transfusions, changes in serum ferritin and iron levels, number and types of ADRs and progression, causality, severity and preventability of ADRs. Results: Of the 60 patients enrolled, one patient was lost to follow up and four withdrew their consent. Of the remaining 55 patients, 36 were boys and 19 were girls (mean age: 6 ± 3.14 years), including patients of 1-3 years (11), 4-6 years (24), 7-10 years (12) and 11-12 years (8). Thirty six patients were born of consanguineous marriages. Adherence to blood transfusion guidelines and deferasirox prescribing and administration guidelines was observed. A serial and significant decrease in mean serum ferritin and serum iron at 3 weeks and 3 months with deferasirox treatment was observed in all age groups except that of 11-12 years. A total of 117 ADRs were observed in 52 patients from 19498 doses, most common being diarrhea (24), raised serum creatinine (15), raised hepatic enzymes (14), abdominal pain (14) and rashes (14). A reduction in dose was required in 32 cases, while a temporary stoppage was indicated in 41 cases. Deferasirox was the possible and probable cause of 65 and 51 ADRs respectively as assessed by WHO-UMC scale. Majority of ADRs were definitely preventable and mild in nature. Conclusion: β Thalassemia Major is more common in males. A rational prescribing of deferasirox was observed. Deferasirox effectively reduced serum ferritin and serum iron levels in these patients. |
| format | Article |
| id | doaj-art-89b284481381420ab1903cf2d844ce54 |
| institution | OA Journals |
| issn | 0971-5851 |
| language | English |
| publishDate | 2017-01-01 |
| publisher | Thieme Medical and Scientific Publishers Pvt. Ltd. |
| record_format | Article |
| series | Indian Journal of Medical and Paediatric Oncology |
| spelling | doaj-art-89b284481381420ab1903cf2d844ce542025-08-20T02:28:08ZengThieme Medical and Scientific Publishers Pvt. Ltd.Indian Journal of Medical and Paediatric Oncology0971-58512017-01-0138210311010.4103/ijmpo.ijmpo_103_16Efficacy and safety of deferasirox in pediatric patients of thalassemia at a Tertiary Care Teaching HospitalDhaval R ThakorChetna K DesaiJigar D KapadiaRam K DikshitK M MehariyaObjective: To evaluate efficacy, safety and utilization pattern of deferasirox in paediatric patients of transfusion dependant β Thalassemia Major at a tertiary care teaching hospital in Gujarat. Materials and Methods: This observational, prospective-retrospective, single centre, continuous study was conducted in a tertiary care teaching hospital among paediatric patients of transfusion dependent β Thalassemia Major. Patients treated with deferasirox for not more than 12 weeks were enrolled. Details of blood transfusions, relevant investigations performed every 3 weeks and 3 months and drugs used were recorded in a pretested case record form. Parents were provided with a diary to record the details of ADRs. Data were analyzed for demographic characteristics, number and mean volume of blood transfusions, changes in serum ferritin and iron levels, number and types of ADRs and progression, causality, severity and preventability of ADRs. Results: Of the 60 patients enrolled, one patient was lost to follow up and four withdrew their consent. Of the remaining 55 patients, 36 were boys and 19 were girls (mean age: 6 ± 3.14 years), including patients of 1-3 years (11), 4-6 years (24), 7-10 years (12) and 11-12 years (8). Thirty six patients were born of consanguineous marriages. Adherence to blood transfusion guidelines and deferasirox prescribing and administration guidelines was observed. A serial and significant decrease in mean serum ferritin and serum iron at 3 weeks and 3 months with deferasirox treatment was observed in all age groups except that of 11-12 years. A total of 117 ADRs were observed in 52 patients from 19498 doses, most common being diarrhea (24), raised serum creatinine (15), raised hepatic enzymes (14), abdominal pain (14) and rashes (14). A reduction in dose was required in 32 cases, while a temporary stoppage was indicated in 41 cases. Deferasirox was the possible and probable cause of 65 and 51 ADRs respectively as assessed by WHO-UMC scale. Majority of ADRs were definitely preventable and mild in nature. Conclusion: β Thalassemia Major is more common in males. A rational prescribing of deferasirox was observed. Deferasirox effectively reduced serum ferritin and serum iron levels in these patients.http://www.ijmpo.org/article.asp?issn=0971-5851;year=2017;volume=38;issue=2;spage=103;epage=110;aulast=ThakorAdverse drug reactionsblood transfusiondeferasiroxiron chelatorsthalassemia |
| spellingShingle | Dhaval R Thakor Chetna K Desai Jigar D Kapadia Ram K Dikshit K M Mehariya Efficacy and safety of deferasirox in pediatric patients of thalassemia at a Tertiary Care Teaching Hospital Indian Journal of Medical and Paediatric Oncology Adverse drug reactions blood transfusion deferasirox iron chelators thalassemia |
| title | Efficacy and safety of deferasirox in pediatric patients of thalassemia at a Tertiary Care Teaching Hospital |
| title_full | Efficacy and safety of deferasirox in pediatric patients of thalassemia at a Tertiary Care Teaching Hospital |
| title_fullStr | Efficacy and safety of deferasirox in pediatric patients of thalassemia at a Tertiary Care Teaching Hospital |
| title_full_unstemmed | Efficacy and safety of deferasirox in pediatric patients of thalassemia at a Tertiary Care Teaching Hospital |
| title_short | Efficacy and safety of deferasirox in pediatric patients of thalassemia at a Tertiary Care Teaching Hospital |
| title_sort | efficacy and safety of deferasirox in pediatric patients of thalassemia at a tertiary care teaching hospital |
| topic | Adverse drug reactions blood transfusion deferasirox iron chelators thalassemia |
| url | http://www.ijmpo.org/article.asp?issn=0971-5851;year=2017;volume=38;issue=2;spage=103;epage=110;aulast=Thakor |
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