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Cyclophosphamide and Topotecan as First-line Salvage Therapy in Patients With Relapsed Ewing Sarcoma at a Single Institution

Farhat, Rawad MD*; Raad, Roy MD; Khoury, Nabil J. MD; Feghaly, Julien BS*; Eid, Toufic MD; Muwakkit, Samar MD*; Abboud, Miguel MD*; El-Solh, Hassan MD*; Saab, Raya MD*

Journal of Pediatric Hematology/Oncology: July 2013 - Volume 35 - Issue 5 - p 356–360
doi: 10.1097/MPH.0b013e318270a343
Original Articles

The combination of cyclophosphamide and topotecan (cyclo/topo) has shown objective responses in relapsed Ewing sarcoma, but the response duration is not well documented. We reviewed characteristics and outcome of 14 patients with Ewing sarcoma, treated uniformly at a single institution and offered cyclo/topo at first relapse. Six patients (43%) had relapse at distant sites. All patients received first-line salvage therapy with cyclophosphamide 250 mg/m2 and topotecan 0.75 mg/m2, daily for 5 days repeated every 21 days. The median number of cycles was 4 (range 1 to 10). All toxicities were manageable, the most common being transient cytopenias. There were also 4 episodes of febrile neutropenia, and 3 episodes of gross hematuria. Response was assessable in 13 patients and showed progressive disease in 6 (46%), stable disease in 4 (31%), and partial response in 3 (23%). Nine patients had local control, consisting of radical surgery in 2, radiation in 3, and a combination in 4 patients. Response, when it occurred, was maintained for a median of 8 months (range, 4 to 28 mo). Four patients (29%) are alive at 3, 7, 9, and 110 months after relapse; 1 is receiving cyclo/topo, 1 is on third-line therapy, and 2 are in second and fourth remission. The low toxicity of this combination, and the lack of sustained responses, warrant its investigation in combination with targeted or novel therapeutic agents in relapsed disease.

Departments of *Pediatric and Adolescent Medicine

Diagnostic Radiology

Radiation Oncology, American University of Beirut, Beirut, Lebanon

The authors declare no conflict of interest.

Reprints: Raya Saab, MD, Department of Pediatrics, American University of Beirut, Riad El Solh Street, Beirut 1107 2020, Lebanon (e-mail: rs88@aub.edu.lb).

Received February 27, 2012

Accepted August 23, 2012

© 2013 by Lippincott Williams & Wilkins.