Sargramostim
Recombinant GM-CSF that stimulates myeloid cell proliferation and function for neutropenia and stem cell mobilization.
Chemical Identity
| Property | Value |
|---|---|
| Molecular Weight | ~14-30 kDa (glycosylated) |
| Amino Acids | 127 (mature protein) |
| CAS Number | 123774-72-1 |
| Gene | CSF2 (GM-CSF) |
| Route | IV, SC |
Structure
Sargramostim (Leukine) is recombinant human granulocyte-macrophage colony-stimulating factor (GM-CSF) produced in yeast (Saccharomyces cerevisiae). It has a glycosylation pattern different from native human GM-CSF, which affects pharmacokinetics but not biological activity.
Mechanism of Action
GM-CSF binds GM-CSF receptors on myeloid progenitors, activating JAK2/STAT5 signaling. This stimulates proliferation and differentiation of neutrophils, monocytes, and macrophages. GM-CSF also enhances phagocytic function, antigen presentation, and cytokine production by mature myeloid cells.
Clinical Applications
- Autologous bone marrow transplant: Myeloid reconstitution
- Allogeneic bone marrow transplant: Engraftment and GVHD reduction
- Chemotherapy-induced neutropenia: Alternative to G-CSF
- AML induction: Following chemotherapy
- Radiation injury: Hematopoietic recovery (Nplate)
Pharmacokinetics
- Half-life: 1-2 hours (IV), 2-3 hours (SC)
- Tmax: 2-4 hours (SC)
- Bioavailability: 60-80% (SC)
- Dosing: 250 mcg/m2/day SC or IV
- Route: IV, SC
Safety and Side Effects
Bone pain, fever, fatigue, myalgia, fluid retention, and capillary leak syndrome (high doses). More inflammatory side effects than filgrastim due to broader myeloid activation.
References
- Nemunaitis, J., et al. (1993). GM-CSF for bone marrow transplant. Journal of Clinical Oncology, 11, 1643-1651.
- Armitage, J.O. (1998). Emerging applications of recombinant human GM-CSF. Blood, 92, 4491-4508.
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