Granulocyte Colony-Stimulating Factor Is Safe and Well Tolerated following Allogeneic Transplantation in Patients with Sickle Cell Disease

Niketa C. Shah, Yale University
Sweta Bhoopatiraju, St. Louis University
Allistair Abraham, Children's National Hospital
Eric Anderson, Rady Children's Hospital
Martin Andreansky, Baylor College of Medicine
Monica Bhatia, University of Columbia
Sonali Chaudhury, Northwestern University
Geoff D E Cuvelier, Cancer Care Manitoba
Kamar Godder, Nicklaus Children's Hospital
Michael Grimley, Cincinnati Children's Hospital
Gregory Hale, All Children's, St. Petersburg
Naynesh Kamani, Children's National Hospital
David Jacobsohn, Children's National Hospital
Alexander Ngwube, Phoenix Children's Hospital
Andrew L. Gilman, ICON, Raleigh
Jodi Skiles, Riley Children's Hospital
Lolie C. Yu, LSUHSC New Orleans
Shalini Shenoy, Washington University


Granulocyte colony-stimulating factor (G-CSF) used after hematopoietic stem cell transplantation (HSCT) can enhance neutrophil recovery in patients rendered neutropenic by the preparative regimen. G-CSF is contraindicated in patients with sickle cell disease (SCD), because life-threatening complications can ensue in the presence of sickle vasculopathy. The safety profile of G-CSF after HSCT for SCD has not been described, however. We report clinical outcomes in the first 100 days post-HSCT in 62 patients supported with G-CSF until neutrophil recovery on a clinical trial of reduced- intensity conditioning HSCT for SCD. The patients received G-CSF for a median of 9 days (range, 5 to 33 days) post-transplantation from the best available stem cell source. Preparation for transplantation included a target hemoglobin S level of ≤45%. Neutrophil engraftment (absolute neutrophil count >0.5 × 103/mL) was achieved at a median of 13 days (range, 10 to 34 days), and platelet engraftment (>50 × 103/mL) was achieved at a median of 19 days (range, 12 to 71 days). The median duration of inpatient hospitalization following stem cell infusion (day 0) was 21.5 days (range, 11 to 33 days). No patient developed SCD-related complications following G-CSF use. The most common organ toxicities encountered between G-CSF initiation (on day +7) and day +100 were anorexia (n = 14), hypertension (n = 11), and electrolyte imbalance requiring correction (n = 9). Central nervous system-related events were noted in 5 patients, all of whom had preexisting cerebral vasculopathy/moyamoya disease, attributed to reversible posterior leukoencephalopathy syndrome in the presence of calcineurin inhibitor therapy and hypertension. We conclude that G-CSF does not adversely impact SCD HSCT recipients and can be safely used post-transplantation to enhance neutrophil recovery.