reviewed april 2026|next review october 2026|88 physicians psi has verified|391691 published studies
Plerixafor
Plerixafor (Mozobil) is an FDA-approved chemokine receptor antagonist (targeting CXCR4) used to mobilize hematopoietic stem cells from bone marrow into peripheral blood for collection and autologous transplantation in patients with non-Hodgkin lymphoma and multiple myeloma.
Evidence landscape: 391691 published studies
1,247 published items indexed. 89 human studies and 203 animal studies. A well-characterized compound with a deep evidence base for the approved indication.
- 15 Human
- 142 Animal
- 43 Reviews
- 391491 Other research
FDA-approved as Mozobil for stem cell mobilization in combination with granulocyte colony-stimulating factor (G-CSF) for autologous transplantation in non-Hodgkin lymphoma and multiple myeloma.
Standard rescue agent for patients who do not mobilize enough stem cells with G-CSF alone. Administered as a single subcutaneous injection 11 hours before apheresis, a procedure that filters specific cells from the blood.
The CXCR4-SDF-1 axis is exploited by many cancers for metastasis. CXCR4 antagonism is under investigation as an anti-cancer strategy beyond the transplant setting.
PSI Assessment
Stem cell transplantation depends on collecting enough stem cells from the patient's blood before the procedure. Some patients do not mobilize enough cells with standard treatment alone. Plerixafor, sold as Mozobil, solved that problem. It is FDA-approved as a stem cell mobilization agent for patients with non-Hodgkin lymphoma and multiple myeloma, working by breaking the molecular anchor (the CXCR4 receptor) that holds stem cells in bone marrow. Beyond transplant, the same CXCR4 pathway is under investigation as a cancer target.
FDA-approved for stem cell mobilization. Solved the poor-mobilizer problem in transplant medicine. The same CXCR4 pathway is now being studied in oncology.
The mechanism is CXCR4 antagonism. CXCR4 is a chemokine receptor on the surface of hematopoietic stem cells that binds to SDF-1 (CXCL12) in the bone marrow niche, anchoring stem cells in place. Plerixafor blocks this interaction, releasing CD34-positive stem cells into peripheral blood where they can be collected by apheresis, a procedure that filters specific cells from the blood. The same CXCR4-SDF-1 axis is exploited by many cancers for metastasis, which is why CXCR4 antagonism is being studied as an anti-cancer strategy.
What the evidence supports
FDA-approved for stem cell mobilization in combination with G-CSF for non-Hodgkin lymphoma and multiple myeloma. Phase III data demonstrates significantly increased CD34-positive cell yields.
What is not yet established
Whether CXCR4 antagonism has therapeutic value as an anti-cancer strategy beyond the transplant setting. Optimal patient selection for poor mobilizers. Single-agent use without G-CSF.
Research Evidence
The findings below cover the established transplant indication and the investigational oncology applications.
Evidence by condition
Evidence dimensions across plerixafor's approved and investigational uses. Stem cell mobilization has the deepest evidence with Phase III replication. Oncology applications are at an early investigational stage.
| Condition | Mechanism | Animal evidence | Human evidence | Replication |
|---|---|---|---|---|
| Stem Cell Mobilization | ||||
| Cancer Biology / CXCR4 Research |
Phase III trials in both non-Hodgkin lymphoma (NHL) and multiple myeloma demonstrated that plerixafor plus G-CSF significantly increased the proportion of patients achieving target CD34-positive cell yields compared to G-CSF alone. In the NHL trial, 59% of plerixafor patients reached target versus 20% with G-CSF alone.
These pivotal trials established plerixafor as the standard rescue for poor mobilizers and formed the basis for FDA approval in 2008.
Clinical experience studies confirmed plerixafor as a reliable salvage agent for patients who fail initial G-CSF mobilization. It became standard practice in transplant centers for poor mobilizers.
The real-world transplant experience has validated the Phase III findings. Plerixafor has become an essential tool in the transplant physician's approach.
A Phase I/II trial explored plerixafor as a chemosensitizer in acute myeloid leukemia, based on the concept that disrupting the CXCR4/SDF-1 bone marrow niche could expose leukemic cells to chemotherapy.
The oncology application is mechanistically rational but at an early clinical stage. Whether CXCR4 antagonism has therapeutic value beyond stem cell mobilization remains an open question.
15 Human|142 Animal|43 Reviews
View all 391691 indexed studiesHow Plerixafor Works
Plerixafor is a bicyclam small molecule that acts as a selective CXCR4 chemokine receptor antagonist. It blocks the SDF-1/CXCR4 interaction that retains hematopoietic stem cells in bone marrow.
Stem cells normally live in bone marrow, held in place by a molecular anchor (the CXCR4-SDF1 interaction). Plerixafor breaks this anchor, releasing stem cells into the bloodstream where they can be collected for transplant.
For a more detailed view of the biology, here is what researchers have observed at the molecular level.
Plerixafor (AMD3100) competitively antagonizes CXCR4, blocking SDF-1 (CXCL12) binding. CXCR4 is expressed on hematopoietic stem cells and signals through Gi-coupled pathways to maintain stem cell retention in the bone marrow niche. Blocking this interaction causes rapid mobilization of CD34-positive cells into peripheral blood, typically peaking 6-11 hours after subcutaneous injection. The same CXCR4-SDF-1 axis mediates chemotaxis in many cancer types: tumor cells express CXCR4 and migrate toward SDF-1 gradients in metastatic niches (bone marrow, liver, lung). This is the mechanistic basis for investigating CXCR4 antagonism as an anti-metastatic or chemosensitization strategy.
What is Plerixafor being studied for?
Researchers are studying Plerixafor across several health conditions. Each condition below is labeled with the strength of evidence that exists for that specific use, not for Plerixafor overall. This means a compound can have human studies for one condition but only animal data for another.
Stem Cell Mobilization
·FDA ApprovedFDA-approved in combination with G-CSF for autologous stem cell transplant in patients with non-Hodgkin lymphoma and multiple myeloma. Significantly increases CD34-positive cell yields.
Limitations: Approved only for specific hematologic malignancies in the autologous transplant setting. Used as rescue for poor mobilizers rather than first-line in all patients.
Cancer Biology / CXCR4 Research
·Animal StudiesCXCR4 is overexpressed in many cancers and mediates metastasis. Blocking CXCR4 may disrupt tumor cell migration and sensitize cancer cells to chemotherapy. Early-phase clinical trials have been conducted.
Limitations: Anti-cancer applications are investigational. No approvals beyond stem cell mobilization. Phase I/II data only for chemosensitization.
Safety and Regulatory Status
FDA Status: FDA-approved as Mozobil for stem cell mobilization in combination with G-CSF. Administered under specialist supervision in the transplant setting.
Administration: Single subcutaneous injection 11 hours before apheresis, a procedure that filters specific cells from the blood. Generally a single-dose treatment in the transplant protocol.
Common side effects include diarrhea, nausea, injection site reactions, and fatigue. The drug is administered in the controlled transplant setting under specialist supervision.
Questions and Comparisons
Questions the evidence raises for a Plerixafor discussion.
Comparison and Related Research
Plerixafor is compared with other stem cell mobilization agents and CXCR4-targeted compounds.
Related compounds
Frequently Asked Questions
References
Each citation links to the original study on PubMed, the U.S. National Library of Medicine database.
- 1.Phase III pivotal trial in non-Hodgkin lymphoma. Plerixafor plus G-CSF significantly increased the proportion of patients achieving target CD34+ cell yields compared to G-CSF alone. Basis for the 2008 FDA approval.DiPersio JF et al., 2009 in Blood. View on PubMed
- 2.Phase III pivotal trial in multiple myeloma. Confirmed the stem cell mobilization benefit in the second approved cancer population.DiPersio JF et al., 2009 in J Clin Oncol. View on PubMed
- 3.Clinical experience study demonstrating plerixafor as a rescue agent for patients who fail initial G-CSF mobilization. Established its role as standard salvage in poor mobilizers.Pusic I et al., 2010 in Biol Blood Marrow Transplant. View on PubMed
- 4.Phase I/II trial of plerixafor as a chemosensitizer in AML. Explored the concept that disrupting the CXCR4/SDF-1 bone marrow niche could expose leukemic cells to chemotherapy.Uy GL et al., 2012 in Blood. View on PubMed
Medical Disclaimer
This content is for educational and informational purposes only and does not constitute medical advice. The information presented reflects published research as indexed by PSI and should not be used to make treatment decisions. Always consult a qualified healthcare provider before starting, stopping, or modifying any treatment.