Treosulfan is primarily used in the field of medicine, particularly in the treatment of ovarian cancer. It functions as an alkylating agent, which means it works by damaging the DNA of cancer cells, thereby inhibiting their growth and proliferation. This makes it a valuable component in chemotherapy regimens aimed at controlling and reducing tumor size.

In addition to its use in ovarian cancer, treosulfan is also employed in conditioning regimens prior to hematopoietic stem cell transplantation (HSCT). It is used to prepare patients by suppressing their immune system and destroying bone marrow cells, making room for the transplanted stem cells to engraft and proliferate. This application is particularly important in treating various hematological malignancies and genetic disorders.

Treosulfan is appreciated for its relatively favorable safety profile compared to some other alkylating agents, which makes it a suitable option for patients who may not tolerate more aggressive treatments. Its role in both cancer therapy and transplantation underscores its versatility and importance in modern medical practice.