Bone marrow transplantation for non-malignant diseases using treosulfan-based conditioning

Yael Dinur-Schejter, Aviva C. Krauss, Odeya Erlich, Natan Gorelik, Anat Yahel, Iris Porat, Michael Weintraub, Jerry Stein, Irina Zaidman, Polina Stepensky*

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review


Treosulfan (treo) is an alkylating agent with a low acute toxicity profile that is increasingly used in hematopoietic stem cell transplantation, predominantly in non-malignant diseases. Treosulfan is usually combined with additional agents, but there is scant evidence to allow comparison between different conditioning protocols using treosulfan. We present the experience of three pediatric transplantation centers in Israel using different treosulfan-based conditioning regimens. Procedure: Data were collected retrospectively on 44 children who underwent 45 hematopoietic stem cell transplantations using treosulfan in combination with either fludarabine (flu) and thiotepa (tt) (n=20), cyclophosphamide (cy) (n=6) or fludarabine alone (n=19). Results: Overall survival (OS) was 70.5%. Disease free survival (DFS) was 54.6%. There was no statistically significant difference between treatment groups in either OS or DFS. Overall survival in patients younger than one year was higher (88.2%). There were significantly more patients with 100% donor chimerism transplanted with flu/treo/tt compared with flu/treo or treo/cy (94.7% compared to 66.7% and 16.7%, respectively). Further prospective studies are required to determine the optimal treosulfan-based preparative regimen for children with non-malignant diseases.

Original languageEnglish
Pages (from-to)299-304
Number of pages6
JournalPediatric Blood and Cancer
Issue number2
StatePublished - 1 Feb 2015
Externally publishedYes


  • Bone marrow transplantation
  • Conditioning
  • Treosulfan


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