Prospective single-arm study of pegfilgrastim activity and safety in children with poor-risk malignant tumours receiving chemotherapy

Abstract

The objective of this study was to assess the efficacy of an injection of 100 lg/kg of pegfilgrastim in haematopoietic recovery and mobilization in children following 32 courses of chemotherapy. End points were duration of neutropaenia, myeloid recovery and PBMC collection. Neutropaenia lasted a mean of 4.7 days ( ± 2.13 days). Myeloid recovery occurred at a median of 10 days (inter quartile range (IQR) 8-11). Febrile neutropaenia complicated 13 courses (40.6%). Mobilization was observed in 20 out of 26 assessable courses (76.9%). The rise in CD34 þ cells occurred at a median of 6 days (IQR 4-7) after PEG and remained 420 per ll for 6 days (IQR 4-8), with a median value of 80 per ll (IQR 48-170.5). The median CD34 þ cell peak was 165 per ll (IQR 82.5-331), 9 days (range 6-14) after PEG. PBMC were collected on average at day þ 5 ( þ 4 to þ 9) after PEG. In 93.3% of collections, at least 3 Â 10 6 per kg CD34 þ cells were collected through a single apheresis. Myeloid recovery occurred in all cases within 15 days, without concomitant thrombocytopaenia. The incidence of primary febrile episodes is in line with data in the literature and with our own historical experience. A long-lasting period of circulating CD34 þ cells allowed for more accurate scheduling of apheresis.

A Phase II study on the safety and efficacy of a single dose of pegfilgrastim for mobilization and transplantation of autologous hematopoietic stem cells in pediatric oncohematology patients

“…In this study, the use of pegfilgrastim resulted in 83% of patients being classified as good mobilizers, while the required target dose of CD34+ from a single leukapheresis was collected from 62% of patients. A previous single‐center, uncontrolled study by Dallorso and colleagues 19 showed that the use of pegfilgrastim in 28 children and adolescents resulted in 76.9% being classified as good mobilizers, but that group was not completely comparable with our study group because it included three patients who underwent repeated courses of mobilization; moreover, the underlying diseases were represented mostly by neuroblastoma, in half of the population, while no patients affected by acute leukemia or Hodgkin's disease were included. To assess the efficacy of pegfilgrastim we compared the results with those obtained in a historical control group, stimulated with filgrastim and matched for at least two of three characteristics such as type of underlying disease, sex, and age.…”

“…Pegfilgrastim was administered subcutaneously as a single injection of 100 µg/kg, up to a maximum of 6 mg, on Day +3 after the end of chemotherapy. The dose of 100 µg/kg of pegfilgrastim was chosen by reference to published pediatric experience 12‐19 . Blood count was checked every 2 to 3 days until the nadir of white blood cells (WBCs) was reached and then every 1 to 2 days until PBSC collection by leukapheresis.…”

“…Pegfilgrastim is not licensed for pediatric use, but several retrospective and prospective Phase II to III, single‐center studies have recently shown that its efficacy and safety in reducing severe neutropenia after chemotherapy is comparable to filgrastim 12‐18 . Limited data are available on the use of pegfilgrastim in pediatric patients as a mobilizing agent in association with chemotherapy, the studies being mainly single‐center experience and differing in the dose and mobilization schemes used 19‐21 . We report the results of a prospective, multicenter, Phase II study that evaluated the safety and efficacy of a single‐dose of 100 µg/kg pegfilgrastim in mobilizing PBSCs in pediatric patients who were candidates for autologous PBSC transplantation.…”

Peripheral Blood Stem Cell Mobilisation with Pegylated Granulocyte Colony Stimulating Factor in Children

“…Most of the experience with Peg-GCSF comes from its use for prophylaxis and treatment of chemotherapy associated neutropenia in children and adults as well as for PBSC mobilisation in adults (9)(10)(11)(12)(13)(14). There is paucity of literature of successful use of Peg-GCSF and its appropriate dosing for PBSC mobilisation in children (1,6,(15)(16)(17). We report our experience of PBSC mobilisation with Peg-GCSF in 9 children.…”

Hematopoietic progenitor cell mobilization and harvesting in children with malignancies: do the advantages of pegfilgrastim really translate into clinical benefit?

“…[5][6][7] In children, the data on pegfilgrastim are very limited and refer to the prevention and treatment of chemotherapy-induced neutropenia and chemo-induced stem cell mobilization. [8][9][10][11] The objective of this phase IIA study was to evaluate the percentage of children achieving steady-state mobilization of HPCs using pegfilgrastim, that is, an optimal harvest of 5 Â 10 6 CD34 þ cells/kg in one standard apheresis (p3 blood volumes processed (BVP)) after a single injection of 300 mg/kg pegfilgrastim.…”