1. ¹Department of Pediatric Oncology, New York University, New York, NY, USA
2. ²Statistical Center, Center for International Blood and Marrow Transplant Research, Medical College of Wisconsin, Milwaukee, WI, USA
3. ³Department of Statistics & Actuarial Science, University of Waterloo, Waterloo, Ontario, Canada
4. ⁴Department of Pediatrics, Medical College of Wisconsin, Milwaukee, WI, USA
5. ⁵BMT Internal Medicine, Mayo Clinic Arizona, Phoenix, AZ, USA
6. ⁶Department of Pediatric Oncology, John Hopkins Hospital, Baltimore, MD, USA
7. ⁷Department of Bone Marrow Transplantation, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA
8. ⁸Department of Bone Marrow Transplantation, Florida Hospital Cancer Institute, Orlando, FL, USA
9. ⁹Department of Pediatrics, University of Nebraska Medical Center, Omaha, NE, USA
10. ¹⁰Department of Bone Marrow Transplantation, St Jude Children's Research Hospital, Memphis, TN, USA
11. ¹¹Department of Hematology/Oncology, University Hospitals of Cleveland, Cleveland, OH, USA
12. ¹²Department of Hematology/Oncology, University of Minnesota, Minneapolis, MN, USA
13. ¹³Department of Bone Marrow Transplantation, Loyola University Medical Center, Maywood, IL, USA

Correspondence: Dr M Eapen, Statistical Center, Center for International Blood and Marrow Transplant Research, Medical College of Wisconsin, 8701 Watertown Plank Road, Milwaukee, WI 53226, USA. E-mail: meapen@mcw.edu

Received 20 August 2007; Revised 19 December 2007; Accepted 20 December 2007; Published online 4 February 2008.

Abstract

The aim of this study was to identify risk factors associated with PFS in patients with Ewing sarcoma undergoing ASCT; 116 patients underwent ASCT in 1989–2000 and reported to the Center for International Blood and Marrow Transplant Research. Eighty patients (69% ) received ASCT as first-line therapy and 36 (31% ), for recurrent disease. Risk factors affecting ASCT were analyzed with use of the Cox regression method. Metastatic disease at diagnosis, recurrence prior to ASCT and performance score <90 were associated with higher rates of disease recurrence/progression. Five-year probabilities of PFS in patients with localized and metastatic disease at diagnosis who received ASCT as first-line therapy were 49% (95% CI 30–69) and 34% (95% CI 22–47) respectively. The 5-year probability of PFS in patients with localized disease at diagnosis, and received ASCT after recurrence was 14% (95% CI 3–30). PFS rates after ASCT are comparable to published rates in patients with similar disease characteristics treated with conventional chemotherapy, surgery and irradiation suggesting a limited role for ASCT in these patients. Therefore, ASCT if considered should be for high-risk patients in the setting of carefully controlled clinical trials.