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Phase II study of recombinant human granulocyte colony-stimulating factor in children undergoing bone marrow

G Dini1, R Floris, A Pession

  • 1Bone Marrow Transplant Unit, G. Gaslini Institute, Genova, Italy.

Insights

Filgrastim reduced fever days in children after autologous bone marrow transplants but delayed platelet and red blood cell recovery. No significant benefits were seen in allogeneic transplants.

Area of Science:

  • Pediatric Hematology/Oncology
  • Hematopoietic Stem Cell Transplantation
  • Growth Factor Therapy

Background:

  • Bone marrow transplantation (BMT) is a critical treatment for pediatric hematological disorders and solid tumors.
  • Granulocyte colony-stimulating factor (filgrastim) is used to accelerate neutrophil recovery post-transplant.
  • The efficacy and safety of filgrastim in pediatric BMT require further investigation.

Purpose of the Study:

  • To evaluate the impact of filgrastim on myeloid reconstitution and clinical outcomes in children undergoing allogeneic or autologous BMT.
  • To compare outcomes between children receiving filgrastim and a matched control group.

Main Methods:

  • Prospective evaluation of 23 children receiving filgrastim (Group I) post-BMT.
  • Comparison with a retrospective, matched cohort of 31 children not receiving filgrastim (Group II).
  • Filgrastim administered at 5 mcg/Kg daily for 21 days or until neutrophil recovery.

Main Results:

  • Similar myeloid reconstitution times between groups (13 vs 14 days).
  • Delayed platelet and red blood cell recovery in the filgrastim group (p < .05 and p < .005, respectively).
  • Reduced febrile days in children undergoing autologous BMT with filgrastim (6 vs 10 days, p < .05).

Conclusions:

  • Filgrastim significantly reduced febrile days in pediatric autologous BMT but did not benefit allogeneic BMT.
  • Filgrastim use was associated with delayed erythrocyte and platelet recovery.
  • Further randomized studies are needed to determine the cost-benefit of filgrastim in pediatric BMT.

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