Addition of plerixafor to G-CSF in poor mobilizing healthy related donors overcame mobilization failure: An observational case series on behalf of the Grupo Español de Trasplante Hematopoyético (GETH)

Plerixafor (Mozobil, Sanofi) is approved for using in patients with lymphoma and multiple myeloma when steady-state mobilization strategies fail. Although off-label use of plerixafor in healthy related donors (HRD) is known, limited data are available and no recommendations exist to guide its use in this setting. With the aim of collecting data from HRDs who received plerixafor in our country, we designed an observational case series study within the Spanish Group of Hematopoietic Transplant and Cell Therapy (GETH). Plerixafor was administered subcutaneously to 30 HRDs at a median dose of 0.24 mg/Kg (interquartile range (IQR): 0.23?0.25) because mobilization failure after using mobilization with G-CSF (mobilization failure was defined as collection of <4.0 × 10⁶ CD34+ cells/Kg recipient). All HRDs received G-CSF at a median dose of 11 μg/Kg/day (IQR: 10–12) for 4–5 days. Leukocytapheresis after G-CSF mobilization was performed in 23 (77 %) HRDs collecting a median of 1.6 × 10⁶ CD34+ cells/Kg recipient weight (IQR: 0.9–2.5). Addition of plerixafor allowed the collection of a higher median number of CD34 cells (4.98 × 10⁶ CD34+ cells/Kg recipient weight (IQR: 3.5–5.8)) when compared with the collection of CD34+ cells with G-CSF alone (p < 0.01). The final median total number of CD34+ cells collected was 6.1 × 10⁶/Kg recipient weight (IQR: 4.8–7.3). Mild adverse events related with plerixafor administration were reported in 8 (27 %) donors. In conclusion, addition of plerixafor after G-CSF mobilization failure in HRDs allowed collecting higher number of CD34+ cells in comparison with steady-state mobilization.     

A multi-center,randomized, controlled trial of parenteral nutrition titrated to resting energy expenditure in children undergoing hematopoietic stem cell transplantation (“PNTREE”): Rationale and design

BackgroundChildren undergoing hematopoietic stem cell transplantation (HSCT) frequently require prolonged courses of parenteral nutrition (PN) as a consequence of gastrointestinal dysfunction related to preparative chemotherapy and radiation. PN has been associated with shorter engraftment time and decreased mortality during HSCT, however, it is also linked with complications, including infections, liver disease, and metabolic disturbances. Some of these complications may be a result of providing PN in excess of nutrient requirements. We previously described significant reductions in resting energy expenditure (REE), as measured by indirect calorimetry, over the course of HSCT. We also documented a decline in mid-arm muscle area, suggesting depletion of muscle mass, while triceps skinfold, a marker of fat stores, was unchanged. These results suggested the need for further study of energy expenditure, body composition and nutritional intake in this group of high risk patients.Design and hypothesisWe hypothesize that changes in body composition affect REE during HSCT, and that standard nutritional support may lead to overfeeding. We are performing a randomized controlled trial of parenteral nutrition among children undergoing allogeneic HSCT. Subjects are randomized to receive PN designed to provide 100% of measured REE, or standard PN, i.e., 140% of estimated energy expenditure. The primary outcome variable is change in percent body fat. Secondary outcomes include glycemic control and frequency of infections, changes in REE and body composition.ConclusionThis study will provide unique and comprehensive nutritional data and its results will guide nutritional therapy for children undergoing HSCT and possibly other catabolic patients.