Clinical benefits of granulocyte colony-stimulating factor therapy after hematopoietic stem cell transplant in children: results of a prospective randomized trial.
OBJECTIVE
Hematopoietic stem cell transplantation (HSCT) is associated with profound neutropenia and significant morbidity and mortality. To evaluate the safety and efficacy of non-glycosylated recombinant human granulocyte colony-stimulating factor (rHuG-CSF) in accelerating myeloid recovery and its influence on infections, supportive therapy, and transplant-related mortality we carried out a randomized study in pediatric patients receiving HSCT.


METHODS
Two hundred and twenty-one consecutive children, recipients of an allogeneic or autologous bone marrow (BM) or peripheral blood progenitor cell (PBPC) transplant, were randomized to either receive rHuG-CSF 10 mg/kg (n=110) or not (n=111).


RESULTS
Myeloid engraftment was faster in the treated arm (14 vs 20 days, p=0.0001). Neutrophil recovery was accelerated both in the BM subgroups (allogeneic and autologous, p=0.002) and in the PBPC group (p=0.0005). All the other evaluated variables showed an advantage in favor of rHuG-CSF treated patients that was significant for platelet transfusion independence and time to discharge (p=0.02 and p=0.04, respectively) only in the BM subgroup.


CONCLUSIONS
We conclude that faster neutrophil recovery in BM recipients receiving rHuG-CSF led to clinical benefits, while, in the PBPC subgroup, it did not translate into clinical advantages.
Dallorso
Sandro
S
U.O. Ematologia ed Oncologia Pediatrica, Istituto G. Gaslini, Genoa, Italy.
Rondelli
Roberto
R
Messina
Chiara
C
Pession
Andrea
A
Giorgiani
Giovanna
G
Fagioli
Franca
F
Locatelli
Franco
F
Manzitti
Carla
C
Balduzzi
Adriana
A
Prete
Arcangelo
A
Cesaro
Simone
S
Lanino
Edoardo
E
Dini
Giorgio
G
D000293:Adolescent; D016026:Bone Marrow Transplantation; D002648:Child; D002675:Child, Preschool; D005260:Female; D006085:Graft Survival / Q000187:drug effects; D016179:Granulocyte Colony-Stimulating Factor / Q000008:administration & dosage*; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods*; D006801:Humans; D007223:Infant; D008297:Male; D009503:Neutropenia / Q000517:prevention & control; D009504:Neutrophils / Q000166:cytology; D036102:Peripheral Blood Stem Cell Transplantation; D011446:Prospective Studies
D016179:Granulocyte Colony-Stimulating Factor