ISSN 1866-8836
Клеточная терапия и трансплантация

NM-07. Treosulfan-based conditioning for hematopoietic stem cell transplantation in patients with Diamond-Blackfan anemia

Svetlana A. Radygina, Svetlana N. Kozlovskaya, Anna P. Vasilieva, Irina P. Shipitsina, Аnna А. Bogoyavlenskaya, Larisa N. Shelikhova, Yulia V. Skvortsova, Dmitry N. Balashov, Michael A. Maschan

Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology, Moscow, Russia

Contact: Svetlana A. Radygina, phone: +7 (495) 287-65-70, e-mail:

doi 10.18620/ctt-1866-8836-2023-12-3-1-176


Busulfan-based conditioning regimens prior to hematopoietic stem cell transplantation (HSCT) in patients with Diamond-Blackfan anemia (DBA) are the standard therapy for a long time. Unfortunately, the high incidence of toxic complications is the cause of transplant-related mortality (TRM) or low quality of life. Treosulfan-based conditioning is very attractive, however only limited data exists of its administration in DBA patients. In this article, we present the experience of treosulfan usage along with novel approaches to graft-versus-host disease (GvHD) prophylaxis in Dmitry Rogachev NMRC of Pediatric Hematology, Oncology, and Immunology.

Patients and methods

From 2012 to 2020, 9 patients with DBA underwent HSCT. Matched unrelated donors were used in 7 patients, mismatched related in 1, and HLA-identical sibling in 1 patient. All patients received treosulfan-based conditioning. The sources of HSC were bone marrow (n=3) and peripheral blood after TCRaß+/CD19+ graft depletion (n=6). Eight patients received various regimens of post-transplant prophylaxis, included calcineurin inhibitors alone or in combinations, 1 patient received mycophenolate mofetil.


All transplanted patients achieved successful engraftment. Median follow-up in survivors (n=8) was 4.7 years (3.78-6.66). ОS was 89% (95% CI:68.4-100). One patient with pre-transplant Karnofsky index of 40% died on day +58 due to multiorgan failure, caused by toxic and infectious complications. Moreover, three patients had clinically significant toxic complications: oral mucositis grade 3 in 1 patient, treosulfan skin toxicity in 2, and moderate veno-occlusive-disease in one case. Five patients had acute GvHD grade II with complete response to the 1st line therapy. There was no evidence of acute GvHD grade III-IV as well as chronic GvHD.


Our data demonstrate that treosulfan-based conditioning, alongside with novel cellular engineering approaches is effective options for HSCT outcomes in patients with DBA.


Diamond-Blackfan anemia, hematopoietic stem cell transplantation, treosulfan, conditioning, toxicity.

Supplement 12-3

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doi 10.18620/ctt-1866-8836-2023-12-3-1-176

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