International Journal of Hematology

DOI: 10.1007/s12185-017-2320-3 Pages: 471-475

Toxicological effects of fludarabine and treosulfan conditioning before allogeneic stem-cell transplantation

1. Karolinska University Hospital, Center for Allogeneic Stem Cell Transplantation Unit

2. Karolinska Institutet, Department of Oncology and Pathology

3. Karolinska University Hospital, Department of Hematology

4. Karolinska Institutet, Division of Hematology, Department of Medicine Huddinge

5. Astrid Lindgren Children’s Hospital, Karolinska University Hospital, Blood Disorders, Immunodeficiency and Stem Cell Transplantation

6. Karolinska Institutet, Division of Pediatrics, Department of Clinical Science, Intervention and Technology

7. Karolinska Institute, Division of Oral and Maxillofacial Surgery, Department of Dental Medicine

8. Karolinska Institutet, Experimental Cancer Medicine, Department of Laboratory Medicine

Correspondence to:
Mats Remberger



We studied early potential treosulfan-related toxicity in 118 patients treated with treosulfan-based conditioning before allogeneic hematopoietic stem-cell transplantation. Most patients (n = 93) had a hematological malignancy. In 80 cases, a HLA-A, -B and -DR matched unrelated donor was used, while 33 patients had a HLA-identical sibling donor, and five received an HLA-A, -B or -DR allele mismatched, unrelated donor. Levels of AST, ALT, and bilirubin were significantly increased 1 week after HSCT compared to before HSCT. However, only a few patients had transaminase levels >2 to 3 × the upper normal level. All patients became neutropenic; 61% were already so at the time of graft infusion. Nearly all patients engrafted, except for three who died very early. Non-relapse mortality was 7.5% at 100 days and 11.9% at 1 year after HSCT. Veno-occlusive disease of the liver occurred in one patient and hemorrhagic cystitis in two patients. This study shows that early regimen-related toxicity after HSCT was low despite similar marrow toxicities compared to myeloablative regimens.

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