Haemopoietic growth factors in paediatric oncology: a review of the literature

Paediatr Drugs. 2001;3(3):195-217. doi: 10.2165/00128072-200103030-00003.


Recombinant haemopoietic growth factors (HGFs) are an attractive adjunct to reduce morbidity from chemotherapy regimens and their use has become widespread in paediatric oncology. Although patients receiving HGFs often have faster haematological recovery after intensive chemotherapy, this does not always translate into meaningful clinical benefits. This article reviews the clinical effectiveness of HGFs in a variety of different contexts. Most published studies have used granulocyte colony-stimulating factor (G-CSF) or granulocyte-macrophage colony-stimulating factor (GM-CSF) as prophylaxis to ameliorate the subsequent neutropenia following intensive chemotherapy. These 2 agents have also been used to mobilise peripheral blood stem cells for autologous transplantation. HGFs specific for anaemia and thrombocytopenia are currently in paediatric clinical trials and it is hoped that the proper context and administration strategy can be found to make their use clinically effective. This article also reviews data on toxicity, specifically focusing on differences between various formulations of growth factors. HGFs are expensive, and cost-benefit analyses reviewed in this article give an important perspective on the financial aspects of paediatric cancer care. Because HGFs do not benefit every child receiving chemotherapy and overuse increases costs and may result in unnecessary adverse effects, evidence-based guidelines for their rational use in paediatric oncology are proposed.

Publication types

  • Research Support, U.S. Gov't, P.H.S.
  • Review

MeSH terms

  • Anemia / chemically induced
  • Anemia / drug therapy
  • Antineoplastic Agents / adverse effects
  • Bone Marrow Transplantation
  • Chemotherapy, Adjuvant / adverse effects
  • Chemotherapy, Adjuvant / economics
  • Child
  • Colony-Stimulating Factors / adverse effects
  • Colony-Stimulating Factors / economics
  • Colony-Stimulating Factors / therapeutic use*
  • Cost-Benefit Analysis
  • Hematopoietic Stem Cell Transplantation
  • Humans
  • Interleukin-11 / therapeutic use
  • Neoplasms / complications
  • Neoplasms / therapy
  • Neutropenia / chemically induced
  • Neutropenia / drug therapy*
  • Neutropenia / prevention & control
  • Thrombocytopenia / chemically induced
  • Thrombocytopenia / drug therapy
  • Thrombopoietin / therapeutic use


  • Antineoplastic Agents
  • Colony-Stimulating Factors
  • Interleukin-11
  • Thrombopoietin