Allogeneic and autologous stem cell transplantation in advanced small round cell sarcomas

Small round cell sarcomas (SRSs) comprise a heterogeneous group of malignant neoplasms with similar cytomorphology characterized by small, round, relatively undifferentiated cells; the group includes Ewing family sarcomas and rhabdomyosarcoma. These malignancies are chemotherapy-sensitive and potentially curable, and are treated by multimodality, dose-intensive, neoadjuvant protocols. For Ewing family sarcomas and rhabdomyosarcoma, actinomycin D-based chemotherapy combined with vincristine and cyclophosphamide is widely accepted as a standard therapy regimen. Since the beginning of the 1970s, such regimens have signifi cantly improved the patients’ prognosis. However, even after induction of these regimens, few patients with overt metastasis at diagnosis or with localized but extensive unresectable primary lesions of the trunk are cured. Most high-risk patients initially respond to treatment, but most of them eventually suffer a recurrence of the tumor and die of disseminated disease. To try to improve systemic control, hematopoietic stem cell transplantation (HSCT) has been considered as a possible approach. Autologous blood stem cell transplantation, such as peripheral blood cell transplantation (PBSCT), rescues patients who are myelosuppressed following high-dose chemotherapy. Allogeneic blood stem cell transplantation, such as umbilical cord blood transplantation (UCBT), may have the advantage of having graft-versus-tumor (GVT) effects. Here, we report two cases of patients with SRS who suffered a refractory recurrence and systemic metastasis of their tumor. Without any consolidation therapy, we could not prevent complete paralysis (case 1) or general dissemination (case 2). We performed high-dose chemotherapy rescued by autologous PBSCT (auto-PBSCT) and UCBT. Our treatment produced a marked response and induced a partial remission. We describe the courses of the patients and the residual problems associated with our treatment. All the treatments were performed after informed consent was obtained from the patients and their families, and approval of the chief of our institution was obtained according to the institutional code of ethics. The patients and their families were informed that data from the case would be submitted for publication and gave their consent.