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.
BACKGROUND
The outcome for patients with recurrent or progressive Ewing's sarcoma family of tumours (ESFT) is poor. High dose therapy (HDT) has been used for a number of years in an attempt to improve survival; however, evidence for the efficacy of this treatment remains limited.


METHODS
Between 1992 and 2004, 33 patients with recurrent or progressive ESFT were treated with HDT with bone marrow (n=2), peripheral blood stem cell (n=30), or bone marrow and peripheral blood stem cell support (n=1), at a single institution. HDT was with busulphan and melphalan in 22 patients; melphalan and etoposide in seven patients, three with total body irradiation (TBI); melphalan in three patients (2 with TBI), and busulphan and cyclophosphamide in one patient.


RESULTS
The 2 and 5 year event free survival was 42.5% (95% CI, 26-59%) and 38.2% (95% CI, 21-55%) respectively. There was one treatment related death from colitis, and grade 4 infection was observed in two patients.


CONCLUSIONS
Long-term survival can be attained in patients with recurrent or refractory ESFT treated with HDT. However, this treatment is associated with severe toxicity. A need remains for prospective randomised clinical trials of HDT in this group of patients.
McTiernan
A
A
Department of Oncology, UCL Hospitals NHS Foundation Trust, London, UK.
Driver
D
D
Michelagnoli
M P
MP
Kilby
A M
AM
Whelan
J S
JS
D000971:Antineoplastic Combined Chemotherapy Protocols / Q000009:adverse effects; D016026:Bone Marrow Transplantation; D001859:Bone Neoplasms / Q000188:drug therapy* / Q000401:mortality / Q000628:therapy; D002066:Busulfan / Q000008:administration & dosage / Q000009:adverse effects; D003131:Combined Modality Therapy; D018450:Disease Progression; D005047:Etoposide / Q000008:administration & dosage / Q000009:adverse effects; D005260:Female; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D008297:Male; D008558:Melphalan / Q000008:administration & dosage / Q000009:adverse effects; D009364:Neoplasm Recurrence, Local / Q000188:drug therapy* / Q000401:mortality / Q000628:therapy; D012512:Sarcoma, Ewing / Q000188:drug therapy* / Q000401:mortality / Q000628:therapy; D016896:Treatment Outcome
D005047:Etoposide; D002066:Busulfan; D008558:Melphalan