FROM THE BRITISH JOURNAL OF HAEMATOLOGY
Patients with beta thalassemia major who were chelated only with oral deferasirox experienced significant improvements in liver stiffness, both from baseline and compared with patients who interrupted deferasirox therapy because of pregnancy, according to a small prospective 5-year study.
Transient elastography showed that continuous therapy with deferasirox (median dose, 35 mg per kg) yielded an 0.85 kPa average improvement in liver stiffness compared with baseline (P = .02), reported Nikolaos Sousos of Aristotle University of Thessaloniki, Greece, and his associates (Br J Haematol. 2017 Jan 20. doi: 10.1111/bjh.14509 ).
In contrast, interrupting therapy for a median of 16 months because of successful pregnancy led to an average increase in liver stiffness of 1.84 kPa – a significant difference between groups (P = .005) even after the researchers controlled for gender, age, ferritin levels, and T2-weighted magnetic resonance imaging (MRI) measurements of iron deposition in the liver and heart.
Patients with beta thalassemia major often develop liver fibrosis because of excessive intestinal iron absorption, iron overload from transfusions, or hepatitis C virus infection, the investigators noted. The median age of the patients in this study was 32 years (range, 20-47 years), they were HCV negative, and they received regular transfusions to maintain hemoglobin levels above 95 g/L. The seven female participants who temporarily stopped deferasirox because of pregnancy all restarted therapy at least 8 months before their follow-up transient elastography liver stiffness measurement, the investigators reported.
T2-weighted MRI measurements of liver iron concentration also had improved at follow-up, reflecting “better control of iron overload,” although the difference from baseline was not statistically significant, the investigators noted. This result reinforces previous findings ( Gastroenterology. 2011;141[4]:1202-11 ) that long-term deferasirox therapy can significantly improve liver fibrosis in patients with beta thalassemia, regardless of liver iron concentration, the researchers added. Together, those findings suggest that “improvement in liver fibrosis, rather than liver iron concentration should be the primary effect of chelation therapy,” they suggested.
The Research Committee of Aristotle University of Thessaloniki, Novartis Hellas (Novartis AG, Basel, Switzerland), and the Greek Thalassaemia Association funded the study. The authors declared having no competing interests.
