Drug intelligence / Profile preview

anti-thymocyte globulin + busulfan + cyclophosphamide

Development stage
Phase 4
Lead developer
Sanofi
Modality
Classical Binding Small Molecules → Small Molecules, Covalent Small Molecules → Small Molecules, Nucleic Acid-Directed Small Molecules → Small Molecules
Administration
Intravenous, Oral
01

Overview

Anti-thymocyte globulin (ATG) + busulfan + cyclophosphamide is a conditioning regimen used in allogeneic hematopoietic stem cell transplantation, particularly for haploidentical peripheral blood stem cell transplantation (haplo-PBSCT). This combination serves multiple purposes: 1. **Myeloablative Conditioning**: Busulfan and cyclophosphamide work together to eliminate the patient's existing bone marrow cells, creating space for donor cells. 2. **Immunosuppression**: ATG specifically targets T-cells to prevent graft rejection and reduce the risk of graft-versus-host disease (GVHD). 3. **GVHD Prophylaxis**: The combination has shown effectiveness in preventing both acute and chronic GVHD compared to standard protocols. The regimen has been studied in various modifications, including: - Standard ATG-based regimens (often called the "Beijing Protocol" with ATG at 10 mg/kg) - Modified combinations with reduced-dose post-transplant cyclophosphamide (PTCy) plus low-dose ATG - Different timing sequences (BuCy vs. CyBu) that may affect toxicity profiles Clinical trials have demonstrated that this combination can significantly reduce the incidence of acute GVHD and improve overall survival, disease-free survival, and GVHD-free/relapse-free survival in patients with hematological malignancies[1][3][4]. The canonical name for this drug combination is "anti-thymocyte globulin + busulfan + cyclophosphamide," and it is classified as a pharmaceutical combination drug used primarily in the context of hematopoietic stem cell transplantation. The regimen is often modified with the addition of other agents like fludarabine or cytarabine, or with adjustments to dosing schedules to optimize outcomes and minimize toxicity[1][4]. This combination has been studied extensively in clinical trials, with research showing that the order of administration (busulfan before cyclophosphamide or vice versa) can impact liver toxicity and overall outcomes[5]. Some studies have also explored replacing cyclophosphamide with fludarabine to reduce side effects while maintaining immunosuppressive properties[7]. The canonical name for this drug combination is "anti-thymocyte globulin + busulfan + cyclophosphamide," and it is classified as a pharmaceutical combination drug used primarily in the context of hematopoietic stem cell transplantation.

Other names
Beijing Protocol
02

Targets

CD2 (T-cell Surface Antigen CD2)IL2RA (Interleukin-2 receptor alpha subunit)CD3 (T-cell surface glycoprotein CD3)CD4 (T lymphocyte surface antigen)CD44 (CD44 antigen)PTPRC (Receptor-type tyrosine-protein phosphatase C)CD8A (CD8 alpha/beta Coreceptor)HLA-DR (Human leukocyte antigen DR)ITGAL (Integrin alpha L (CD11a))ITGB2 (Complement receptor 4)MHC I (MHC class I})ITGB1 (Integrin beta-1)MR1 (Thioesterase PNKD)CD86 (T-lymphocyte activation antigen CD86)DNAN7-G (DNA guanine-N7 adduct)

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