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. PATIENTS AND 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.
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. PATIENTS AND 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.
Authors: Lianne M Haveman; Roelof van Ewijk; Elvira C van Dalen; Willemijn B Breunis; Leontien Cm Kremer; Henk van den Berg; Uta Dirksen; Johannes Hm Merks Journal: Cochrane Database Syst Rev Date: 2021-09-02
Authors: Robert Grimer; Nick Athanasou; Craig Gerrand; Ian Judson; Ian Lewis; Bruce Morland; David Peake; Beatrice Seddon; Jeremy Whelan Journal: Sarcoma Date: 2010-12-29
Authors: Uwe Thiel; Angela Wawer; Irene von Luettichau; Hans-Ulrich Bender; Franziska Blaeschke; Thomas G P Grunewald; Marc Steinborn; Barbara Röper; Halvard Bonig; Thomas Klingebiel; Peter Bader; Ewa Koscielniak; Michael Paulussen; Uta Dirksen; Heribert Juergens; Hans-Jochem Kolb; Stefan E G Burdach Journal: Oncotarget Date: 2016-10-25
Authors: Juhee Seo; Dong Ho Kim; Jung Sub Lim; Jae-Soo Koh; Ji Young Yoo; Chang-Bae Kong; Won Seok Song; Wan Hyeong Cho; Dae-Geun Jeon; Soo-Yong Lee; Jun Ah Lee Journal: Korean J Pediatr Date: 2013-09-30
Authors: Craig Gerrand; Nick Athanasou; Bernadette Brennan; Robert Grimer; Ian Judson; Bruce Morland; David Peake; Beatrice Seddon; Jeremy Whelan Journal: Clin Sarcoma Res Date: 2016-05-04