RHGM-CSF VS PLACEBO FOLLOWING RHGM-CSF-MOBILIZED PBPC TRANSPLANTATION- A PHASE-III DOUBLE-BLIND RANDOMIZED TRIAL

In this placebo-controlled randomized trial we evaluated the hematolog ical and clinical effects of r-Hu GM-CSF after high-dose chemotherapy (HDC) followed by GM-CSF-mobilized PBPC transplantation. Fifty patient s with poor prognosis malignancies were randomized in a double-blind s tudy to receive either GMCSF or placebo after HDC followed by PBPC res cue. For all patients, PBPCs were recruited using a combination of VP- 16 (300 mg/m(2) on days 1 and 2), cytoxan (3 g/m(2) on days 3 and 4) a nd GM-CSF (5 mu g/kg from day 5), No differences were demonstrated bet ween the two groups in median time to neutrophil or platelet recoverie s. There was no significant difference between the GM-CSF group and th e placebo group in the median duration of post-transplant hospitalizat ion, in the number of days of antibiotic treatment, in the number of i nfections and in red blood cell or platelet transfusion requirements. There was a significant difference with an advantage for the placebo g roup in the mean duration of febrile days (P = 0.01). We conclude that the administration of GM-CSF in patients transplanted with GM-CSF-mob ilized PBPC is not associated with a clinical benefit in term of tempo of engraftment, numbers of documented infections, transfusion require ments and mucositis grading.