Plerixafor-augmented peripheral blood stem cell mobilization in AL amyloidosis with cardiac involvement: a case
Steve Y Lee1, Vaishali Sanchorawala, David C Seldin
1Department of Medicine, Boston University Medical Center , Boston, MA , USA.
Insights
Plerixafor combined with G-CSF effectively mobilizes peripheral blood stem cells in AL amyloidosis patients with cardiac involvement. This approach showed good tolerability and successful engraftment post-transplant.
Area of Science:
- Hematology
- Oncology
- Cardiology
Background:
- Cardiac involvement is common in AL amyloidosis, predicting a poor prognosis.
- High-dose melphalan and autologous stem-cell transplantation (HDM/SCT) improves outcomes but requires stem cell mobilization.
- G-CSF mobilization can cause complications like fluid overload and arrhythmias in these patients.
Observation:
- This study evaluated plerixafor combined with G-CSF for stem cell mobilization in five AL amyloidosis patients with cardiac involvement.
- Two patients had recent heart transplants; three received plerixafor after G-CSF alone failed.
- No significant toxicities were observed during mobilization and collection.
Findings:
- Plerixafor and G-CSF effectively mobilized peripheral blood stem cells (PBSCs), with a median yield of 5.9 × 10^6 CD34+ cells/kg over 2 leukapheresis days.
- Neutrophil and platelet engraftment occurred by day 9 and day 13 post-HDM/SCT, respectively.
- Plerixafor was well-tolerated, whether used initially or as a rescue therapy.
Implications:
- Plerixafor offers a safe and effective alternative for PBSC mobilization in AL amyloidosis patients with cardiac compromise.
- This strategy may expand eligibility for potentially curative HDM/SCT in a high-risk patient population.
- Further research can confirm these findings in larger cohorts.
Abstract:
Nearly half of AL amyloidosis patients have cardiac involvement, an independent predictor of poor prognosis. High-dose melphalan and autologous stem-cell transplantation (HDM/SCT) can induce complete hematologic responses and prolong survival in AL amyloidosis. Granulocyte colony-stimulating factor (G-CSF)-induced mobilization of peripheral blood stem cell (PBSC) in AL amyloidosis patients is associated with volume overload, arrhythmias and capillary leak syndrome. Plerixafor has a different mechanism of action and has non-overlapping toxicities with G-CSF. We describe our experience in five patients with AL amyloidosis and cardiac involvement who received plerixafor with G-CSF for PBSC mobilization. Median age was 56 years; two patients had undergone heart transplantation within the year prior to HDM/SCT. Three patients received plerixafor after an initial trial of mobilization with G-CSF alone. No patient had any significant toxicities during mobilization and PBSC collection. The median total yield of PBSCs collected was 5.9 × 10(6) CD34+ cells/kg; the median number of leukapheresis days was 2. Neutrophil engraftment after HDM/SCT occurred at a median of nine days, platelet engraftment at a median of 13 days. Plerixafor was effective and well tolerated when used upfront or as rescue for PBSC mobilization in AL amyloidosis patients with cardiac involvement.
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