Yael Dinur-Schejter1, Aviva C Krauss2, Odeya Erlich1, Natan Gorelik1, Anat Yahel2, Iris Porat3, Michael Weintraub1, Jerry Stein2, Irina Zaidman3, Polina Stepensky1. 1. Department of Pediatric Hematology-Oncology and Bone Marrow Transplantation, Hadassah Hebrew University Medical Center, Jerusalem, Israel. 2. Department of Pediatric Hematology Oncology and BMT Unit, Schneider Children's Medical Center of Israel, Petah-Tikva, Israel. 3. Department of Pediatric Hematology Oncology, Meyer Children's Hospital, Rambam Health Care Campus, Haifa, Israel.
Abstract
BACKGROUND: 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. Pediatr Blood Cancer 2015;62:299-304.
BACKGROUND: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. Pediatr Blood Cancer 2015;62:299-304.
Authors: Antonio Marzollo; Elisabetta Calore; Manuela Tumino; Marta Pillon; Maria Vittoria Gazzola; Roberta Destro; Raffaella Colombatti; Piero Marson; Tiziana Tison; Anna Colpo; Chiara Mainardi; Maria Gabelli; Maria Paola Boaro; Sara Rossin; Aurora Strano; Nadia Quaglia; Federica Menzato; Giuseppe Basso; Laura Sainati; Chiara Messina Journal: Mediterr J Hematol Infect Dis Date: 2017-02-15 Impact factor: 2.576
Authors: Krzysztof Kalwak; Monika Mielcarek; Katharine Patrick; Jan Styczynski; Peter Bader; Selim Corbacioglu; Birgit Burkhardt; Karl Walter Sykora; Katarzyna Drabko; Jolanta Gozdzik; Franca Fagioli; Johann Greil; Bernd Gruhn; Rita Beier; Franco Locatelli; Ingo Müller; Paul Gerhardt Schlegel; Petr Sedlacek; Klaus Daniel Stachel; Claudia Hemmelmann; Ann-Kristin Möller; Joachim Baumgart; Ajay Vora Journal: Bone Marrow Transplant Date: 2020-03-20 Impact factor: 5.483