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Published on: May 19, 2023
Plerixafor stem cell mobilization in Japanese children: A post-marketing study
Hiroaki Goto1, Rie Kanamori2, Satoshi Nishina3
1Division of Hematology/Oncology, Kanagawa Children's Medical Center, Yokohama, Japan.
Insights
Plerixafor (0.24 mg/kg) shows promise for hematopoietic stem cell mobilization in Japanese children under 15. Most patients achieved target CD34+ cell counts, with manageable adverse events observed.
Area of Science:
- Hematology
- Pediatric Oncology
- Pharmacology
Background:
- Plerixafor is approved in Japan for stem cell mobilization but data in children is limited.
- This study addresses the safety and efficacy of plerixafor in pediatric patients (<15 years).
Purpose of the Study:
- To evaluate the safety and effectiveness of plerixafor in Japanese children (<15 years) undergoing autologous stem cell transplant.
- To assess plerixafor's role in routine clinical practice for pediatric hematopoietic stem cell mobilization.
Main Methods:
- A multicenter, post-marketing surveillance study in Japan.
- Subgroup analysis of pediatric patients (<15 years) receiving once-daily subcutaneous plerixafor.
- Primary endpoint: proportion of patients achieving ≥2 × 10^6 CD34+ cells/kg within 4 days via apheresis.
Main Results:
- Eighteen pediatric patients (median age 6 years) with solid tumors were analyzed.
- Twelve patients (66.7%) achieved the primary efficacy endpoint.
- Adverse drug reactions (ADRs) occurred in 38.9% of patients (≥6 years, ≥16 kg), most commonly pyrexia and vomiting.
Conclusions:
- Plerixafor at 0.24 mg/kg appears safe and effective for pediatric stem cell mobilization in Japanese clinical practice.
- Further research with larger studies is warranted to confirm these findings.
Background:
Plerixafor is approved in Japan for hematopoietic stem cell mobilization prior to autologous transplant, but limited data are available on the use in children. This study evaluates the safety and effectiveness of plerixafor in Japanese children aged <15 years.
Methods:
A multicenter, post-marketing surveillance study was conducted in Japan to evaluate the safety and effectiveness of plerixafor in routine clinical practice. This subgroup analysis examined the safety and effectiveness of plerixafor administered as a once-daily, subcutaneous injection in children aged <15 years. The primary effectiveness outcome was the proportion of patients with 2 × 106 cells CD34+ cells/kg collected via apheresis within 4 days.
Results:
Eighteen patients with solid tumors were included in this analysis; (median age 6.0 years, range, 1-13 years). In addition to granulocyte colony-stimulating factor, all patients had received chemotherapy immediately prior to plerixafor administration. The mean (SD) daily dose of plerixafor was 0.24 (0.01) mg/kg. Seven of the 18 patients (38.9%) developed adverse drug reactions (ADRs), all occurring in patients aged ≥6 years and weighing ≥16 kg. The most common ADRs were pyrexia (n = 4), vomiting (n = 3), nausea (n = 2), and abdominal pain (n = 2). Twelve patients (66.7%) achieved a CD34+ cell count ≥2 × 106 cells/kg within 4 days after the start of plerixafor administration.
Conclusions:
The results provide an encouraging sign that plerixafor 0.24 mg/kg may be safe and effective in pediatric patients in routine clinical practice in Japan, but further research in larger studies is needed.
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