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Finding a balance in reduced toxicity hematopoietic stem cell transplantation for thalassemia: role of infused CD3+ cell count and immunosuppression

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Finding a balance in reduced toxicity hematopoietic stem cell transplantation for thalassemia: role of infused CD3+ cell count and immunosuppression. / Meissner, Barbara; Lang, Peter; Bader, Peter; Hoenig, Manfred; Müller, Ingo; Meisel, Roland; Greil, Johann; Sauer, Martin G; Metzler, Markus; Corbacioglu, Selim; Burkhardt, Birgit; Wölfl, Matthias; Strahm, Brigitte; Kafa, Kinan; Basu, Oliver; Lode, Holger N; Gruhn, Bernd; Cario, Holger; Ozga, Ann-Kathrin; Zimmermann, Martin; Jarisch, Andrea; Beier, Rita.

in: BONE MARROW TRANSPL, Jahrgang 59, Nr. 5, 05.2024, S. 587-596.

Publikationen: SCORING: Beitrag in Fachzeitschrift/Zeitung › SCORING: Zeitschriftenaufsatz › Forschung › Begutachtung

Harvard

Meissner, B, Lang, P, Bader, P, Hoenig, M, Müller, I, Meisel, R, Greil, J, Sauer, MG, Metzler, M, Corbacioglu, S, Burkhardt, B, Wölfl, M, Strahm, B, Kafa, K, Basu, O, Lode, HN, Gruhn, B, Cario, H, Ozga, A-K, Zimmermann, M, Jarisch, A & Beier, R 2024, 'Finding a balance in reduced toxicity hematopoietic stem cell transplantation for thalassemia: role of infused CD3+ cell count and immunosuppression', BONE MARROW TRANSPL, Jg. 59, Nr. 5, S. 587-596. https://doi.org/10.1038/s41409-024-02219-0

APA

Meissner, B., Lang, P., Bader, P., Hoenig, M., Müller, I., Meisel, R., Greil, J., Sauer, M. G., Metzler, M., Corbacioglu, S., Burkhardt, B., Wölfl, M., Strahm, B., Kafa, K., Basu, O., Lode, H. N., Gruhn, B., Cario, H., Ozga, A-K., ... Beier, R. (2024). Finding a balance in reduced toxicity hematopoietic stem cell transplantation for thalassemia: role of infused CD3+ cell count and immunosuppression. BONE MARROW TRANSPL, 59(5), 587-596. https://doi.org/10.1038/s41409-024-02219-0

Vancouver

Meissner B, Lang P, Bader P, Hoenig M, Müller I, Meisel R et al. Finding a balance in reduced toxicity hematopoietic stem cell transplantation for thalassemia: role of infused CD3+ cell count and immunosuppression. BONE MARROW TRANSPL. 2024 Mai;59(5):587-596. https://doi.org/10.1038/s41409-024-02219-0

Bibtex

@article{91441f253859465b9066596452dedb1b,
title = "Finding a balance in reduced toxicity hematopoietic stem cell transplantation for thalassemia: role of infused CD3+ cell count and immunosuppression",
abstract = "We performed a retrospective analysis on 124 patients with transfusion-dependent thalassemia who were registered in the German pediatric registry for stem cell transplantation. All patients underwent first allogeneic hematopoietic stem cell transplantation (HSCT) between 2011 and 2020 and belonged mainly to Pesaro risk class 1-2. Four-year overall (OS) and thalassemia-free survival (TFS) were 94.5% ± 2.9% and 88.0% ± 3.4% after treosulfan-fludarabine-thiotepa- and 96.9% ± 3.1% (P = 0.763) and 96.9% ± 3.1% (P = 0.155) after busulfan-fludarabine-based conditioning. Mixed chimerism below 75% occurred predominantly in treosulfan-based regimens (27.5% versus 6.2%). OS and TFS did not differ significantly between matched sibling, other matched family and matched unrelated donor (UD) HSCTs (OS: 100.0%, 100.0%, 96.3% ± 3.6%; TFS: 96.5% ± 2.4%, 90.0% ± 9.5%, 88.9% ± 6.0%). However, mismatched UD-HSCTs performed less favorable (OS: 84.7% ± 7.3% (P = 0.029); TFS: 79.9% ± 7.4% (P = 0.082)). We generated a scoring system reflecting the risk to develop mixed chimerism in our cohort. The main risk-reducing factors were a high CD3+ cell count (≥6 × 107/kg) in the graft, busulfan-conditioning, pre-conditioning therapy and low-targeted ciclosporin A trough levels. Acute GvHD grade III-IV in treosulfan-based concepts predominantly occurred in patients with UD and reduced GvHD prophylaxis but not in the context of high CD3+ cell doses. Taken together, this information might be used to develop more risk-adapted HSCT regimens for thalassemia patients.",
author = "Barbara Meissner and Peter Lang and Peter Bader and Manfred Hoenig and Ingo M{\"u}ller and Roland Meisel and Johann Greil and Sauer, {Martin G} and Markus Metzler and Selim Corbacioglu and Birgit Burkhardt and Matthias W{\"o}lfl and Brigitte Strahm and Kinan Kafa and Oliver Basu and Lode, {Holger N} and Bernd Gruhn and Holger Cario and Ann-Kathrin Ozga and Martin Zimmermann and Andrea Jarisch and Rita Beier",
note = "{\textcopyright} 2024. The Author(s).",
year = "2024",
month = may,
doi = "10.1038/s41409-024-02219-0",
language = "English",
volume = "59",
pages = "587--596",
journal = "BONE MARROW TRANSPL",
issn = "0268-3369",
publisher = "NATURE PUBLISHING GROUP",
number = "5",

}

RIS

TY - JOUR

T1 - Finding a balance in reduced toxicity hematopoietic stem cell transplantation for thalassemia: role of infused CD3+ cell count and immunosuppression

AU - Meissner, Barbara

AU - Lang, Peter

AU - Bader, Peter

AU - Hoenig, Manfred

AU - Müller, Ingo

AU - Meisel, Roland

AU - Greil, Johann

AU - Sauer, Martin G

AU - Metzler, Markus

AU - Corbacioglu, Selim

AU - Burkhardt, Birgit

AU - Wölfl, Matthias

AU - Strahm, Brigitte

AU - Kafa, Kinan

AU - Basu, Oliver

AU - Lode, Holger N

AU - Gruhn, Bernd

AU - Cario, Holger

AU - Ozga, Ann-Kathrin

AU - Zimmermann, Martin

AU - Jarisch, Andrea

AU - Beier, Rita

N1 - © 2024. The Author(s).

PY - 2024/5

Y1 - 2024/5

N2 - We performed a retrospective analysis on 124 patients with transfusion-dependent thalassemia who were registered in the German pediatric registry for stem cell transplantation. All patients underwent first allogeneic hematopoietic stem cell transplantation (HSCT) between 2011 and 2020 and belonged mainly to Pesaro risk class 1-2. Four-year overall (OS) and thalassemia-free survival (TFS) were 94.5% ± 2.9% and 88.0% ± 3.4% after treosulfan-fludarabine-thiotepa- and 96.9% ± 3.1% (P = 0.763) and 96.9% ± 3.1% (P = 0.155) after busulfan-fludarabine-based conditioning. Mixed chimerism below 75% occurred predominantly in treosulfan-based regimens (27.5% versus 6.2%). OS and TFS did not differ significantly between matched sibling, other matched family and matched unrelated donor (UD) HSCTs (OS: 100.0%, 100.0%, 96.3% ± 3.6%; TFS: 96.5% ± 2.4%, 90.0% ± 9.5%, 88.9% ± 6.0%). However, mismatched UD-HSCTs performed less favorable (OS: 84.7% ± 7.3% (P = 0.029); TFS: 79.9% ± 7.4% (P = 0.082)). We generated a scoring system reflecting the risk to develop mixed chimerism in our cohort. The main risk-reducing factors were a high CD3+ cell count (≥6 × 107/kg) in the graft, busulfan-conditioning, pre-conditioning therapy and low-targeted ciclosporin A trough levels. Acute GvHD grade III-IV in treosulfan-based concepts predominantly occurred in patients with UD and reduced GvHD prophylaxis but not in the context of high CD3+ cell doses. Taken together, this information might be used to develop more risk-adapted HSCT regimens for thalassemia patients.

AB - We performed a retrospective analysis on 124 patients with transfusion-dependent thalassemia who were registered in the German pediatric registry for stem cell transplantation. All patients underwent first allogeneic hematopoietic stem cell transplantation (HSCT) between 2011 and 2020 and belonged mainly to Pesaro risk class 1-2. Four-year overall (OS) and thalassemia-free survival (TFS) were 94.5% ± 2.9% and 88.0% ± 3.4% after treosulfan-fludarabine-thiotepa- and 96.9% ± 3.1% (P = 0.763) and 96.9% ± 3.1% (P = 0.155) after busulfan-fludarabine-based conditioning. Mixed chimerism below 75% occurred predominantly in treosulfan-based regimens (27.5% versus 6.2%). OS and TFS did not differ significantly between matched sibling, other matched family and matched unrelated donor (UD) HSCTs (OS: 100.0%, 100.0%, 96.3% ± 3.6%; TFS: 96.5% ± 2.4%, 90.0% ± 9.5%, 88.9% ± 6.0%). However, mismatched UD-HSCTs performed less favorable (OS: 84.7% ± 7.3% (P = 0.029); TFS: 79.9% ± 7.4% (P = 0.082)). We generated a scoring system reflecting the risk to develop mixed chimerism in our cohort. The main risk-reducing factors were a high CD3+ cell count (≥6 × 107/kg) in the graft, busulfan-conditioning, pre-conditioning therapy and low-targeted ciclosporin A trough levels. Acute GvHD grade III-IV in treosulfan-based concepts predominantly occurred in patients with UD and reduced GvHD prophylaxis but not in the context of high CD3+ cell doses. Taken together, this information might be used to develop more risk-adapted HSCT regimens for thalassemia patients.

U2 - 10.1038/s41409-024-02219-0

DO - 10.1038/s41409-024-02219-0

M3 - SCORING: Journal article

C2 - 38326567

VL - 59

SP - 587

EP - 596

JO - BONE MARROW TRANSPL

JF - BONE MARROW TRANSPL

SN - 0268-3369

IS - 5

ER -