LONGITUDINAL STUDY ON LIVER FUNCTIONS IN PATIENTS WITH THALASSEMIA MAJOR BEFORE AND AFTER DEFERASIROX (DFX) THERAPY
With regular blood transfusion and iron chelation therapy, most patients with thalassemia major (BTM) now survive beyond the third decade of life . Liver disease is becoming an important cause of morbidity and mortality in these patients. Chronic hepatitis and/or severe iron overload are important c...
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| Format: | Article |
| Language: | English |
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PAGEPress Publications
2014-04-01
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| Series: | Mediterranean Journal of Hematology and Infectious Diseases |
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| Online Access: | http://www.mjhid.org/index.php/mjhid/article/view/1385 |
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| author | Ashraf Tawfik Soliman Mohamed Yassin Fawzia AlYafei Lolwa Al-Naimi Noora Almarri Aml Sabt Vincenzo De Sanctis |
| author_facet | Ashraf Tawfik Soliman Mohamed Yassin Fawzia AlYafei Lolwa Al-Naimi Noora Almarri Aml Sabt Vincenzo De Sanctis |
| author_sort | Ashraf Tawfik Soliman |
| collection | DOAJ |
| description | With regular blood transfusion and iron chelation therapy, most patients with thalassemia major (BTM) now survive beyond the third decade of life . Liver disease is becoming an important cause of morbidity and mortality in these patients. Chronic hepatitis and/or severe iron overload are important causes of liver pathology. Iron chelation with desferrioxamine (Desferal) reduces excessive body iron, but its efficacy is limited by poor compliance and dose related toxicity. The recent use of Deferasirox (Exjade- DFX ), an oral single dose therapy has improved the compliance to chelation therapy.
Aims: To study the long-term liver functions in BMT patients, seronegative for liver infections before versus after DFX therapy in relation to ferritin level and IGF-I level.
Methods: Liver function tests including: serum bilirubin, alanine transferase (ALT), aspartate transferase (AST) , albumin, insulin-like growth factor – I (IGF-I) and serum ferritin concentrations were followed every 6 months in 40 patients with BTM, with hepatitis negative screening (checked every year), for at least five years of DFO therapy and 4-5 years of DFX therapy .
Results: DFX therapy (20 mg/kg/day) significantly decreased serum ferritin level in patients with BTM, this was associated with significant decrease in serum ALT, AST, ALP and increase in IGF-I concentrations. Albumin concentrations did not change after DFX treatment. ALT and AST levels were correlated significantly with serum ferritin concentrations ( r = 0.45 and 0.33 respectively , p < 0.05) . IGF-I concentrations were correlated significantly with serum ALT (r= 0.26, p = 0.05) but not with AST, ALP, bilirubin or albumin levels.
The negative correlation between serum ferritin concentrations and ALT suggests that impairment of hepatic function negatively affects IGF-I synthesis in these patients due to iron toxicity, even in the absence of hepatitis.
Conclusions: Some impairment of liver function can occur in hepatitis negative BMT patients with iron overload. The use of DFX was associated with mild but significant reduction of ALT, AST and ALP and increase in IGF-I levels. The negative correlation between IGF-I and ALT concentrations suggest that preventing hepatic dysfunction may improve the growth potential in these patients. |
| format | Article |
| id | doaj-art-d7a8e6cecdb6400abe05f6b3f5a103b1 |
| institution | Kabale University |
| issn | 2035-3006 |
| language | English |
| publishDate | 2014-04-01 |
| publisher | PAGEPress Publications |
| record_format | Article |
| series | Mediterranean Journal of Hematology and Infectious Diseases |
| spelling | doaj-art-d7a8e6cecdb6400abe05f6b3f5a103b12024-12-02T09:47:06ZengPAGEPress PublicationsMediterranean Journal of Hematology and Infectious Diseases2035-30062014-04-0161e2014025e201402510.4084/mjhid.2014.0251145LONGITUDINAL STUDY ON LIVER FUNCTIONS IN PATIENTS WITH THALASSEMIA MAJOR BEFORE AND AFTER DEFERASIROX (DFX) THERAPYAshraf Tawfik Soliman0Mohamed YassinFawzia AlYafeiLolwa Al-NaimiNoora AlmarriAml SabtVincenzo De SanctisDivision of Pediatric Endocrinology Hamad Medical Center Doha QatarWith regular blood transfusion and iron chelation therapy, most patients with thalassemia major (BTM) now survive beyond the third decade of life . Liver disease is becoming an important cause of morbidity and mortality in these patients. Chronic hepatitis and/or severe iron overload are important causes of liver pathology. Iron chelation with desferrioxamine (Desferal) reduces excessive body iron, but its efficacy is limited by poor compliance and dose related toxicity. The recent use of Deferasirox (Exjade- DFX ), an oral single dose therapy has improved the compliance to chelation therapy. Aims: To study the long-term liver functions in BMT patients, seronegative for liver infections before versus after DFX therapy in relation to ferritin level and IGF-I level. Methods: Liver function tests including: serum bilirubin, alanine transferase (ALT), aspartate transferase (AST) , albumin, insulin-like growth factor – I (IGF-I) and serum ferritin concentrations were followed every 6 months in 40 patients with BTM, with hepatitis negative screening (checked every year), for at least five years of DFO therapy and 4-5 years of DFX therapy . Results: DFX therapy (20 mg/kg/day) significantly decreased serum ferritin level in patients with BTM, this was associated with significant decrease in serum ALT, AST, ALP and increase in IGF-I concentrations. Albumin concentrations did not change after DFX treatment. ALT and AST levels were correlated significantly with serum ferritin concentrations ( r = 0.45 and 0.33 respectively , p < 0.05) . IGF-I concentrations were correlated significantly with serum ALT (r= 0.26, p = 0.05) but not with AST, ALP, bilirubin or albumin levels. The negative correlation between serum ferritin concentrations and ALT suggests that impairment of hepatic function negatively affects IGF-I synthesis in these patients due to iron toxicity, even in the absence of hepatitis. Conclusions: Some impairment of liver function can occur in hepatitis negative BMT patients with iron overload. The use of DFX was associated with mild but significant reduction of ALT, AST and ALP and increase in IGF-I levels. The negative correlation between IGF-I and ALT concentrations suggest that preventing hepatic dysfunction may improve the growth potential in these patients.http://www.mjhid.org/index.php/mjhid/article/view/1385Thalassemia, liver enzymes, Insulin-like growth factor-I (IGF-I), chelation therapy |
| spellingShingle | Ashraf Tawfik Soliman Mohamed Yassin Fawzia AlYafei Lolwa Al-Naimi Noora Almarri Aml Sabt Vincenzo De Sanctis LONGITUDINAL STUDY ON LIVER FUNCTIONS IN PATIENTS WITH THALASSEMIA MAJOR BEFORE AND AFTER DEFERASIROX (DFX) THERAPY Mediterranean Journal of Hematology and Infectious Diseases Thalassemia, liver enzymes, Insulin-like growth factor-I (IGF-I), chelation therapy |
| title | LONGITUDINAL STUDY ON LIVER FUNCTIONS IN PATIENTS WITH THALASSEMIA MAJOR BEFORE AND AFTER DEFERASIROX (DFX) THERAPY |
| title_full | LONGITUDINAL STUDY ON LIVER FUNCTIONS IN PATIENTS WITH THALASSEMIA MAJOR BEFORE AND AFTER DEFERASIROX (DFX) THERAPY |
| title_fullStr | LONGITUDINAL STUDY ON LIVER FUNCTIONS IN PATIENTS WITH THALASSEMIA MAJOR BEFORE AND AFTER DEFERASIROX (DFX) THERAPY |
| title_full_unstemmed | LONGITUDINAL STUDY ON LIVER FUNCTIONS IN PATIENTS WITH THALASSEMIA MAJOR BEFORE AND AFTER DEFERASIROX (DFX) THERAPY |
| title_short | LONGITUDINAL STUDY ON LIVER FUNCTIONS IN PATIENTS WITH THALASSEMIA MAJOR BEFORE AND AFTER DEFERASIROX (DFX) THERAPY |
| title_sort | longitudinal study on liver functions in patients with thalassemia major before and after deferasirox dfx therapy |
| topic | Thalassemia, liver enzymes, Insulin-like growth factor-I (IGF-I), chelation therapy |
| url | http://www.mjhid.org/index.php/mjhid/article/view/1385 |
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