Poster #074, UCSF Benioff Children's Hospital Oakland
Liver injury in children with transfusion dependent thalassemia on chelation
Mentor: Madhav Vissa, MD
Pediatric patients with transfusion dependent thalassemia (TDT) often require chelation therapy to treat iron overload. Deferasirox is the primary oral iron chelator; however, transaminitis frequently interrupts chelation treatment. We aimed to characterize the longitudinal correlation between liver function; chelator pauses and evaluations for alternative causes of liver dysfunction. We hypothesize that many children with transfusion dependent beta thalassemia will have frequent transaminitis that will interrupt optimal chelation in pediatric patients with TDT.
We performed an observational, retrospective study of pediatric patients aged 1-25 with transfusion dependent thalassemia who have been on chelation treatment. We automatedly and manually chart reviewed to extract laboratory, clinical, and medication data. We evaluated how many patients had alanine aminotransferase (ALT) elevations and how often chelation doses were held or reduced. We calculated how often alternative causes for liver disease were evaluated. Descriptive statistics were used to summarize data.
Thirty-one patients met the inclusion criteria, including 27 with evaluable longitudinal liver enzyme data. Four had no ALT elevations, 12 had 1-5 elevations, 4 had 5-10 elevations, and 7 experienced more than 10 ALT elevations. Deferasirox was held in 17 patients, and doses were reduced in 23 patients. Reductions were not always directly related toxicities; some were made in response to decreasing iron burden. Respiratory viral panels were obtained 33 times in 20 patients and were positive in 31 of 33 evaluations. Among the 7 patients with more than 10 ALT elevations, 5 had liver ultrasounds, 4 had RVPs, 4 underwent autoimmune evaluation, and 4 were referred to gastroenterology. Aside from positive RVPs, there were no alternatives causes of ALT elevation identified.
Transaminitis regularly affected deferasirox treatment, but alternative causes of liver injury were rarely evaluated. Respiratory viral infections may contribute to ALT elevations and should be considered when evaluating transaminitis.