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Ann Intern Med. 2002 May 7;136(9):667-72.

Reversibility of cirrhosis in patients cured of thalassemia by bone marrow transplantation.

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Unità Operativa di Anatomia Patologica, Azienda Ospedaliera San Salvatore di Pesaro, 6110 Pesaro, Italy.



Cirrhosis is a well-known complication of thalassemia major. In this context, it is a consequence of iron overload and hepatitis C virus infection and generally seems to be irreversible.


To determine whether cirrhosis in thalassemia major can be reversible.


Retrospective study.


Bone Marrow Transplantation Unit and Pathology Service, Pesaro Hospital, Pesaro, Italy.


6 patients who developed liver cirrhosis before or after their thalassemia was cured by bone marrow transplantation (age at transplantation, 11 to 25 years). After diagnosis of cirrhosis, the patients received iron depletion and antiviral therapies.


Each liver biopsy specimen was coded. A liver pathologist and a member of the transplantation center with expertise in hepatopathology graded the specimens by using the Ishak staging and grading systems. Neither knew the patient's identity or the sequence of biopsy with regard to the time of treatment.


After the patients received iron depletion and antiviral therapies, liver biopsy specimens showed impressive reduction in liver iron stores. In 4 patients, iron removal was complete. Serum aminotransferase levels decreased in all patients and normalized in 5; histologic inflammatory activity decreased in all patients and disappeared in 2. Follow-up biopsies showed regression of incomplete or definite cirrhosis in all patients; 3 patients had presented with portal fibrosis without bridging, and the others had portal fibrosis and portal-to-portal bridging. Several biopsies and the presence of many portal spaces confirmed the diagnosis of cirrhosis; follow-up biopsies confirmed regression of cirrhosis.


In some patients in whom bone marrow transplantation has cured thalassemia, cirrhosis may be reversible after iron removal treatment.

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