Clinical outcomes and graft characteristics in pediatric matched sibling donor transplants using granulocyte colony‐stimulating factor‐primed bone marrow and steady‐state bone marrow

Abstract:  Matched sibling donor (MSD) transplant is a life‐saving procedure for children with various hematological malignancies and non‐malignancies. Traditionally, steady‐state bone marrow (S‐BM) has been used as the source of stem cells. More recently, peripheral blood stem cell (PBSC) after granulocyte‐colony stimulating factor (G‐CSF) mobilization has gained popularity. Adult studies of G‐CSF‐primed BM (G‐BM) have shown that it produces rapid white blood cell engraftment like PBSC, but with less chronic graft‐vs.‐host disease. No such study has been published in pediatric patients. We conducted a pilot clinical trial of G‐BM for pediatric patients. Ten patients were enrolled and were compared to a contemporaneous group of 12 patients who received S‐BM. Patients in the G‐BM group received a higher dose of total nucleated cells/kg (7.01 vs. 3.76 × 108, p = 0.0009), higher granulocyte–macrophage colony‐forming units (CFU‐GM)/kg (7.19 vs. 3.53 × 105, p = 0.01) and had shorter inpatient length of stay (28 vs. 40 days, p = 0.04). The engraftment, transfusion requirement and disease‐free survival between the two groups were similar. We concluded that G‐BM should be considered as an alternative graft source to S‐BM, with the benefits of larger graft cell dose, higher CFU‐GM dose and shorter length of stay.