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Treating Ewing Sarcoma When Underlying Liver Disease Is Present

June 19, 2023

UPMC Children’s Hospital of Pittsburgh Division of Pediatric Hematology/Oncology faculty members, along with the Departments of Pathology and Pharmacy and Therapeutics collaborators published details from a recent case in which a patient with progressive familial intrahepatic cholestasis type 1 (PFIC1) was diagnosed with localized, early-stage Ewing sarcoma and successfully treated with a modified plan of chemotherapeutic agents.

Their case report was published online first in the Journal of Pediatric Hematology/Oncology.

Because several of the first-line chemotherapeutic agents used to combat Ewing sarcoma are primarily metabolized and cleared from the body by the liver, patients with liver diseases or compromised hepatic function would be subjected to potentially extreme toxicities and further liver degradation if used.

To work around the difficulties presented by the liver dysfunction, the team utilized a therapeutic regimen that eliminated the use of several agents, for example, doxorubicin and vincristine, and modified dosing for other agents to account for the patient’s liver disease. To compensate for the elimination of two of the preferred agents, the team relied on cycles of topotecan and cyclophosphamide.

The team also notes in their case report the need to be vigilant for myelosuppression, lymphopenia, and infectious complications, such as sepsis, and in the case of this patient, a rare development of Cryptococcal meningitis, arising as secondary effects of the combined treatment agents and the underlying liver dysfunction.

Read the entire case report (open access), and the team’s approach to treatment in this complex case, using the reference link below.


Daley J, Halligan K, Howrie D, Salgado C, Superdock A, Friehling E, Bailey KM. Successful Treatment of Patient With Ewing Sarcoma in the Setting of Inherited Cholestatic Liver Disease. J Pediatr Hematol Oncol. 2023; Jan 10. Online ahead of print.