Use of G‐CSF in Matched Sibling Donor Pediatric Allogeneic Transplantation: A Consensus Statement from the Children's Oncology Group (COG) Transplant Discipline Committee and Pediatric Blood and Marrow Transplant Consortium (PBMTC) Executive Committee

Abstract

Preliminary studies indicate that G-CSF-primed marrow harvesting may result in a graft with increased mononuclear cells collected, increased CD34(+) stem and progenitor cell dose and a potential for more rapid engraftment. Increased cell dose plus other potential positive effects of G-CSF priming have resulted in improved survival in non-randomized preliminary studies. These benefits may be available without the increased risk of chronic graft versus host disease (GVHD) that is experienced with allogeneic peripheral blood stem cell (PBSC) transplant. A phase III Children's Oncology Group (COG)/Pediatric Blood and Marrow Transplant Consortium (PBMTC) trial comparing G-CSF-primed marrow to standard marrow has been proposed. This document reviews background studies of G-CSF-primed marrow and addresses benefits and risks of G-CSF administration to normal pediatric donors. We conclude that the approach is promising and warrants further study. Risks of G-CSF to the donor are minimal and benefits to both donor and recipient may occur.

Risk of complications during hematopoietic stem cell collection in pediatric sibling donors: a prospective European Group for Blood and Marrow Transplantation Pediatric Diseases Working Party study

“…25 Several reports on adult donors have shown an incidence of life-threatening or debilitating complications of BM donation in 0.3%-0.4% of the donors. 11,[25][26][27] Death has been reported as contributing to an overall risk of 0.003%-0.02% in adults after BM collection. 19,28 The risk of death and SAE after PBSC collection in adults was 4-and 2-fold higher, respectively, compared with BM harvest.…”

“…4 Based on experience gained over the past 30 years, the use of BM from an HLA-identical sibling donor is considered the standard of care worldwide for children undergoing HSC transplantation. 11 Nevertheless, an increasing use of allogeneic peripheral blood stem cells (PBSCs) among matched-sibling pediatric transplantations was reported to the Pediatric Blood and Marrow Transplant Consortium between 1994 and 2002, which accounted for more than 23% of all pediatric HSC collections. 12,13 In the same period, PBSCs were collected from 4% of pediatric matchedsibling donors in EBMT centers.…”

Transplant Outcomes in Beta-Thalassemia Major Patients Receiving Combined Granulocyte Colony-Stimulating Factor-Primed Bone Marrow and Cord Blood Graft Compared to Granulocyte Colony-Stimulating Factor-Primed Bone Marrow Alone

“…BM harvesting is a safe procedure that primarily has mild and transient side effects. As most of the donors in this study were smaller than their respective recipients, we used G-CSFstimulated BM because it is a suitable alternative to unstimulated BM if the donor is young or small [28] and G-CSF administration is safe in pediatric patients [29][30][31]. Adverse events were rare in our pediatric donors, and a small proportion of donors underwent red blood cell transfusion.…”

Children as hematopoietic cell donors in research: when is it approvable?

“…For approximately one-third, the HSC donor is also a child, 1 typically a healthy sibling who undergoes BM harvest or large volume apheresis for collection of peripheral blood stem cells. Recognizing that HSC donors face risks [2][3][4][5] (Table 1) without the potential for direct medical benefit, the American Academy of Pediatrics recently published guidelines specifying when minors may ethically serve as HSC donors for a standard (nonresearch) transplant. 6 This raises the need for guidance on when minors may donate HSCs in the research setting.…”