Treosulfan-based conditioning regimen for children and adolescents with hemophagocytic lymphohistiocytosis
Lehmberg K, Albert MH, Beier R, Beutel K, Gruhn B, Kröger N, Meisel R, Schulz A, Stachel D, Woessmann W, Janka G, Müller I (2014)
Publication Type: Journal article
Publication year: 2014
Journal
Publisher: Ferrata Storti Foundation
Book Volume: 99
Pages Range: 180-4
Journal Issue: 1
DOI: 10.3324/haematol.2013.094730
Abstract
In hematopoietic stem cell transplantation for hemophagocytic lymphohistiocytosis, high transplant-related mortality after busulfan-based myeloablative regimens has been observed. Conditioning regimens with reduced toxicity based on melphalan or treosulfan are promising alternatives. We retrospectively analyzed hematopoietic stem cell transplantations in 19 hemophagocytic lymphohistiocytosis patients after conditioning with fludarabine, treosulfan, alemtuzumab, with or without thiotepa. Overall and disease-free survivals were 100% (follow up 7-31 months). Two patients required second transplant (1 after haploidentical transplantation). In 6 patients, overall donor chimerism dropped below 75% and prompted donor lymphocyte infusions. Administration of donor lymphocytes or second transplantation were significantly more frequent after transplantation from a human leukocyte antigen mismatched (9/10) versus matched (10/10) donor (P=0.018). The toxicity profile was favorable, with one veno-occlusive disease, one grade 3 graft-versus-host disease after donor lymphocyte infusion, and 2 severe viral infections (1 influenza, 1 Epstein Barr virus). In conclusion, the treosulfan-based regimen in hemophagocytic lymphohistiocytosis is effective with low toxicity and gives excellent overall and disease-free survival rates. In the future, the incidence of mixed chimerism, particularly after human leukocyte antigen mismatched donor transplants, needs to be addressed.
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Additional Organisation(s)
Involved external institutions
Universitätsklinikum Hamburg-Eppendorf (UKE)
Germany (DE)
Klinikum der Universität München (Großhadern und Innenstadt)
Germany (DE)
Medizinische Hochschule Hannover (MHH) / Hannover Medical School
Germany (DE)
Universitätsklinikum Münster
Germany (DE)
Universitätsklinikum Jena
Germany (DE)
Heinrich-Heine-Universität Düsseldorf
Germany (DE)
Universitätsklinikum Ulm
Germany (DE)
Universitätsklinikum Gießen und Marburg (UKGM)
Germany (DE)
Germany (DE)
Klinikum der Universität München (Großhadern und Innenstadt)
Germany (DE)
Medizinische Hochschule Hannover (MHH) / Hannover Medical School
Germany (DE)
Universitätsklinikum Münster
Germany (DE)
Universitätsklinikum Jena
Germany (DE)
Heinrich-Heine-Universität Düsseldorf
Germany (DE)
Universitätsklinikum Ulm
Germany (DE)
Universitätsklinikum Gießen und Marburg (UKGM)
Germany (DE)
How to cite
APA:
Lehmberg, K., Albert, M.H., Beier, R., Beutel, K., Gruhn, B., Kröger, N.,... Müller, I. (2014). Treosulfan-based conditioning regimen for children and adolescents with hemophagocytic lymphohistiocytosis. Haematologica, 99(1), 180-4. https://dx.doi.org/10.3324/haematol.2013.094730
MLA:
Lehmberg, Kai, et al. "Treosulfan-based conditioning regimen for children and adolescents with hemophagocytic lymphohistiocytosis." Haematologica 99.1 (2014): 180-4.
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