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treosulfan

L01AB02

Forms & Strengths

Intravenous powder for solution: 1 g, 5 g vials

Adult Dosing

Conditioning treatment prior to allogeneic hematopoietic stem cell transplantation (allo-HSCT): 10 g/m² to 14 g/m² body surface area intravenously daily on 3 consecutive days (Days -4, -3, and -2)

Pediatric Dosing

Safety and efficacy in pediatric patients under the age of 1 month have not been established
Pediatric dosing for allo-HSCT conditioning is based on body surface area or age-adjusted regimens in clinical trials, typically ranging from 10 g/m² to 14 g/m² daily for 3 days

Indications

In combination with fludarabine as part of conditioning treatment prior to allogeneic hematopoietic stem cell transplantation in patients with malignant and non-malignant diseases

Mechanism of Action

Bifunctional alkylating agent that spontaneously converts in vivo to epoxides, which form cross-links in DNA, leading to inhibition of DNA synthesis and function, and subsequent cell death

Contraindications

Hypersensitivity to treosulfan
Severe hepatic impairment
Severe renal impairment
Active, uncontrolled infections
Pregnancy and lactation

Adverse Reactions

Myelosuppression (prolonged pancytopenia)
Stomatitis and mucositis
Gastrointestinal disorders (nausea, vomiting, diarrhea)
Febrile neutropenia and serious infections
Veno-occlusive disease (sinusoidal obstruction syndrome) of the liver
Alopecia

Drug Interactions

Live vaccines: Avoid use due to risk of systemic and potentially fatal vaccine-induced disease in immunosuppressed patients
Nephrotoxic and hepatotoxic drugs: Concomitant use may increase the risk of renal or hepatic toxicity
CYP450 enzyme interactions: Minimal direct cytochrome P450 inhibition or induction, but additive myelosuppressive and immunosuppressive effects occur with other cytotoxic drugs

Curated Content: Needs Vetting Before Put to Clinical Use