A prospective study of autologous bone marrow or peripheral blood stem cell transplantation after intensive chemotherapy in myelodysplastic syndromes

Abstract

We prospectively assessed autologous stem cell transplantation for consolidation treatment in a trial of intensive chemotherapy in high risk myelodysplastic syndromes (MDS). In this trial, patients aged 55 years or less with no HLA-identical sibling and achieving CR were scheduled to receive unmanipulated autologous bone marrow transplantation (ABMT) preceded by a consolidation chemotherapy course. Forty-two of the 83 patients aged 55 years or less included in the trial (51%) achieved CR. Three were allografted in CR. Twenty-four of the remaining 39 patients who achieved CR (62%) received ABMT (16 patients) or autologous peripheral blood stem cell transplantation (APSCT) (eight patients). Indeed, as bone marrow harvest was often insufficient, APSCT was subsequently proposed after mobilization by consolidation chemotherapy followed by G-CSF. The conditioning regimen combined cyclophosphamide and busulfan. ABMT and APSCT were performed 1-7 months (median 3) after CR achievement. Hematological reconstitution occurred in all patients and tended to be faster after APSCT than ABMT although not significantly. Three patients died from the procedure, nine relapsed after 2-26 months and 12 (50%) were still in CR after 8-55 months. In autografted patients, median Kaplan-Meier disease-free survival and survival were 29 and 33 months from the autograft, respectively. Thus, ABMT or APSCT can be performed in almost two-thirds of MDS patients who achieve CR with intensive chemotherapy. PBSC collection may yield higher numbers of stem cells than marrow collection in some cases, and could improve the percentage of MDS patients autografted in CR. Longer follow-up is required to determine if autograft will prolong CR duration in at least some patients.

Maintenance treatment with azacytidine for patients with high‐risk myelodysplastic syndromes (MDS) or acute myeloid leukaemia following MDS in complete remission after induction chemotherapy

“…Most patients relapse within the first year, and conventional consolidation chemotherapy does not seem to prolong CR duration (Wattel et al, 1997;Ganser et al, 2000;Hast et al, 2003;Kantarjian et al, 2006b;Hofmann et al, 2007). If there is a potential benefit from autologous stem cell transplantation, this is restricted to younger patients (De Witte et al, 1997;Wattel et al, 1999). This trial is the first to evaluate safety and feasibility of maintenance treatment with azacytidine for patients with highrisk MDS and MDS-AML with CR after induction chemotherapy.…”

Value of allogeneic versus autologous stem cell transplantation and chemotherapy in patients with myelodysplastic syndromes and secondary acute myeloid leukemia. Final results of a prospective randomized European Intergroup Trial

“…[20][21][22] In a previous study by our group, we analyzed 100 patients in first complete remission who were candidates for SCT. 9,23 The 4-year disease-free survival rates in the donor versus no-donor groups were similar: 31% and 27%, respectively.…”

Autologous stem cell transplantation for therapy-related acute myeloid leukemia and myelodysplastic syndrome

“…A prospective French study investigated the feasibility of autologous stem cell transplantation after conditioning with busulfan and cyclophosphamide in those patients who achieved a CR after induction chemotherapy, and the study reported about 50% of the patients who were alive 8-55 months after transplantation. 20 More recently, the EORTC presented results from 159 patients with MDS who received a transplant from a HLA-identical sibling or (if not available) an autologous transplantation after induction chemotherapy. The 4-year RFS was 23% for patients with a donor and 22% for patients without a donor (P ¼ 0.7), and the authors concluded that allogeneic and autologous stem cell transplantation result in similar survival rates.…”