Drug intelligence / Profile preview

cyclophosphamide + eltrombopag + methylprednisolone

Development stage
Preclinical
Lead developer
Novartis
Modality
Orthosteric Ligands → Classical Binding Small Molecules → Small Molecules, DNA Intercalators/Alkylators → Nucleic Acid-Directed Small Molecules → Small Molecules
Administration
Oral, Intravenous, Intramuscular
01

Overview

This is a combination therapy consisting of three drugs: - **Cyclophosphamide** is an alkylating agent (nitrogen mustard) used primarily as an antineoplastic and immunosuppressive agent. It works by cross-linking DNA, leading to cell death, particularly in rapidly dividing cells such as cancer cells and immune cells[2]. - **Eltrombopag** is a small molecule thrombopoietin receptor agonist that stimulates the proliferation and differentiation of bone marrow progenitor cells into megakaryocytes, thereby increasing platelet production. It is mainly used for thrombocytopenia in chronic immune thrombocytopenic purpura (ITP), aplastic anemia, and hepatitis C-associated thrombocytopenia[3][5]. - **Methylprednisolone** is a synthetic glucocorticoid with potent anti-inflammatory and immunosuppressive properties. It modulates gene expression to suppress inflammation and immune responses. Methylprednisolone is widely used for autoimmune diseases, severe allergies, certain cancers, and other inflammatory conditions[7]. The combination of these agents may be considered in severe or refractory autoimmune hematologic disorders (such as lupus nephritis or refractory ITP), leveraging their complementary mechanisms—immunosuppression from cyclophosphamide/methylprednisolone plus stimulation of platelet production from eltrombopag—to control disease activity while managing cytopenias[1][6][8].

02

Targets

DNAGR (Glucocorticoid receptor)TPOR (Thrombopoietin receptor)

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