title: Bone marrow transplantation for non-malignant diseases using treosulfan-based conditioning.
abstract: 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.
  
  
  
  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).
  
  
  
  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. Pediatr Blood Cancer 2015;62:299-304. © 2014 Wiley Periodicals, Inc.
authors.affiliation: Department of Pediatric Hematology-Oncology and Bone Marrow Transplantation, Hadassah Hebrew University Medical Center, Jerusalem, Israel.
authors.forename: Yael
authors.initials: Y
authors.lastname: Dinur-Schejter
authors.affiliation: Department of Pediatric Hematology Oncology and BMT Unit, Schneider Children's Medical Center of Israel, Petah-Tikva, Israel.
authors.forename: Aviva C
authors.initials: AC
authors.lastname: Krauss
authors.affiliation: Department of Pediatric Hematology-Oncology and Bone Marrow Transplantation, Hadassah Hebrew University Medical Center, Jerusalem, Israel.
authors.forename: Odeya
authors.initials: O
authors.lastname: Erlich
authors.affiliation: Department of Pediatric Hematology-Oncology and Bone Marrow Transplantation, Hadassah Hebrew University Medical Center, Jerusalem, Israel.
authors.forename: Natan
authors.initials: N
authors.lastname: Gorelik
authors.affiliation: Department of Pediatric Hematology Oncology and BMT Unit, Schneider Children's Medical Center of Israel, Petah-Tikva, Israel.
authors.forename: Anat
authors.initials: A
authors.lastname: Yahel
authors.affiliation: Department of Pediatric Hematology Oncology, Meyer Children's Hospital, Rambam Health Care Campus, Haifa, Israel.
authors.forename: Iris
authors.initials: I
authors.lastname: Porat
authors.affiliation: Department of Pediatric Hematology-Oncology and Bone Marrow Transplantation, Hadassah Hebrew University Medical Center, Jerusalem, Israel.
authors.forename: Michael
authors.initials: M
authors.lastname: Weintraub
authors.affiliation: Department of Pediatric Hematology Oncology and BMT Unit, Schneider Children's Medical Center of Israel, Petah-Tikva, Israel.
authors.forename: Jerry
authors.initials: J
authors.lastname: Stein
authors.affiliation: Department of Pediatric Hematology Oncology, Meyer Children's Hospital, Rambam Health Care Campus, Haifa, Israel.
authors.forename: Irina
authors.initials: I
authors.lastname: Zaidman
authors.affiliation: Department of Pediatric Hematology-Oncology and Bone Marrow Transplantation, Hadassah Hebrew University Medical Center, Jerusalem, Israel.
authors.forename: Polina
authors.initials: P
authors.lastname: Stepensky
mesh_terms: D000293:Adolescent; D000477:Alkylating Agents / Q000009:adverse effects / Q000627:therapeutic use*; D016026:Bone Marrow Transplantation / Q000379:methods*; D002066:Busulfan / Q000009:adverse effects / Q000031:analogs & derivatives* / Q000627:therapeutic use; D002648:Child; D002675:Child, Preschool; D003520:Cyclophosphamide / Q000627:therapeutic use; D018572:Disease-Free Survival; D005260:Female; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods*; D006801:Humans; D007223:Infant; D008297:Male; D019653:Myeloablative Agonists / Q000627:therapeutic use*; D012189:Retrospective Studies; D013852:Thiotepa / Q000627:therapeutic use; D014740:Vidarabine / Q000031:analogs & derivatives / Q000627:therapeutic use
chemical_list: D000477:Alkylating Agents; D019653:Myeloablative Agonists; D003520:Cyclophosphamide; D013852:Thiotepa; C018404:treosulfan; D014740:Vidarabine; D002066:Busulfan; C024352:fludarabine
keywords: bone marrow transplantation; conditioning; treosulfan