sargramostim   Click here for help

GtoPdb Ligand ID: 7905

Synonyms: Leukine®
Approved drug
sargramostim is an approved drug (FDA (1991))
Comment: Sargramostim is recombinant human GM-CSF, produced in E.coli. Compared to the endogenous peptide sargramostim has a Arg>Leu substitution at residue 23.
No information available.
Summary of Clinical Use Click here for help
Sargramostim is approved to promote myeloid reconstitution after autologous or allogeneic bone marrow transplantation and after bone marrow transplantation failure or graft failure. It is also used to treat neutropenia in AML patients receiving chemptherapy. It is used off-label to increase neutrophil counts in patients with myelodysplastic syndromes and in AIDS patients receiving zidovudine, to decrease nadir of leukopenia secondary to myelosuppressive chemotherapy, to decrease myelosuppression in preleukemic patients, to correct neutropenia in aplastic anemia patients and to decrease transplantation-associated organ system damage.
Recent results suggest that co-therapy with sargramostim and ipilimumab can increase overall survival in patients with advanced melanoma, compared to ipilimumab alone, but does not increase progression-free survival [1] (see clinical trial NCT01134614). However, given the rapid advances being made and new therapies entering the market for melanoma treatment (such as nivolumab and pembrolizumab), this result may be too late to be of wide-ranging clinical importance.
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