A prospective study of G-CSF–primed bone marrow as a stem-cell source for allogeneic bone marrow transplantation in children: a Pediatric Blood and Marrow Transplant Consortium (PBMTC) study

Author:

Frangoul Haydar1,Nemecek Eneida R.2,Billheimer Dean3,Pulsipher Michael A.4,Khan Shakila5,Woolfrey Ann6,Manes Becky1,Cole Catherine7,Walters Mark C.8,Ayas Mouhab9,Ravindranath Yaddanapudi10,Levine John E.11,Grupp Stephan A.12

Affiliation:

1. Department of Pediatrics, Vanderbilt University, Nashville, TN;

2. Department of Pediatrics, Oregon Health & Science University, Portland, OR;

3. Department of Biostatistics, Vanderbilt University, Nashville, TN;

4. Department of Pediatrics, Primary Children's Medical Center, University of Utah, Salt Lake City, UT;

5. Department of Pediatrics, Mayo Clinic, Rochester, MN;

6. Department of Pediatrics, Fred Hutchinson Cancer Center, Seattle, WA;

7. Department of Pediatrics, Princess Margaret Hospital for Children, Perth, Australia;

8. Department of Pediatrics, Children's Hospital Oakland & Research Center, Oakland, CA;

9. Department of Pediatrics, King Faisal Medical Center, Riyadh, Saudi Arabia;

10. Department of Pediatrics, Wayne State University, Detroit, MI;

11. Department of Pediatrics, University of Michigan, Ann Arbor, MI; and

12. Department of Pediatrics, Children's Hospital of Philadelphia, Philadelphia, PA

Abstract

A prospective multicenter trial was conducted to evaluate the safety and feasibility of granulocyte colony-stimulating factor (G-CSF)–primed bone marrow (G-BM) in children receiving allogeneic bone marrow transplantation (BMT). A total of 42 children with a median age of 9.8 years (range, 0.8-17 years) were enrolled. Donors with median age of 9.2 years (range, 1.1-22 years) received 5 μg/kg per day of subcutaneous G-CSF for 5 consecutive days. BM was harvested on the fifth day. No donor experienced complications related to G-CSF administration or marrow har-vest. Median nucleated (NC) and CD34 cells infused was 6.7 × 108/kg (range, 2.4-18.5 × 108/kg) and 7.4 × 106/kg (range, 2-27.6 × 106/kg), respectively. Neutrophil and platelet engraftment was at a median of 19 days (range, 13-28 days) and 20 days (range, 9-44 days), respectively. A total of 13 (32%) patients developed grade 2 graft-versus-host disease (GVHD), and 5 (13%) of 40 evaluable patients developed chronic GVHD (3 limited and 2 extensive). Higher cell dose was not associated with increased risk of acute or chronic GVHD. Overall survival and event-free survival at 2 years were 81% and 69%, respectively. Collection of G-BM from pediatric donors is safe, and can result in high NC and CD34 cell doses that facilitate engraftment after myeloablative BMT without a discernable increase in the risk of GVHD.

Publisher

American Society of Hematology

Subject

Cell Biology,Hematology,Immunology,Biochemistry

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