Researchers from the United Kingdom have reported that high-dose chemotherapy with autologous peripheral blood stem cell support results in a five-year event-free survival (EFS) of 38% in patients with relapsed or refractory Ewing’s sarcoma. The details of this report appeared in the August 2006 issue of Annals of Oncology .

The Ewing’s sarcoma family of tumors are sensitive to chemotherapy, but once relapse occurs cure is difficult to achieve. The role of high-dose chemotherapy for Ewing’s sarcoma has been controversial but utilized by most groups treating children and young adults. A recent report from the Fred Hutchinson Cancer Research Center  showed that 46% of chemotherapy responsive patients became long term survivors compared to 0% of those who were resistant to re-induction chemotherapy. They also reported that tandem transplants were more effective than single transplants with survivals of 75% and 20%, respectively (see related news).

A recent French study reviewed the outcomes of 46 adult patients with Ewing’s sarcoma treated with high-dose chemotherapy and autologous stem cell transplantation between 1987 and 2000. They reported a five-year overall survival of 63% and a progression-free survival of 47%. Tandem transplants also appeared to be more effective than single transplants. Neither study had any treatment-related deaths.

The current British study was a single institution experience involving 33 patients with recurrent or progressive disease treated between 1992 and 2004. High-dose regimens involved busulfan and melphalan or etoposide or cyclophosphamide in 30 patients and a total body irradiation regimen in 3. They reported that the five-year EFS was 38% with one treatment related death.

Conclusions: These data confirm that long-term EFS can be attained in patients with recurrent or refractory Ewing’s sarcoma treated with high-dose chemotherapy. However, the most effective regimen or regimens have yet to be determined. There is a need for prospective randomized clinical trials of high-dose therapy for early non-refractory disease. The low or absent treatment related deaths make this feasible. Of major interest is the apparent improved effectiveness of tandem transplants.

Reference: McTiernan A, Driver D, Michelagnoli M P, et al. High dose chemotherapy with bone marrow or peripheral stem cell rescue is an effective treatment option for patients with relapsed or progressive Ewing's sarcoma family of tumours. Annals of Oncology . 2006;17:1301-1305.

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