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Bibliographies thématiques / Treosulfan analog and chimerism

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Littérature scientifique sur le sujet « Treosulfan analog and chimerism »

Auteur : Grafiati

Publié le 7 juin 2025

Mis à jour le 2 août 2025

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Articles de revues sur le sujet "Treosulfan analog and chimerism"

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Srinivas, G., D.V. Ramanjaneyulu, E. Muralinath, et al. "An Important Parameters of Hematopoietic Stem Cell Include Anatomy as Well as Physiology, Indications, Treatment, Complications and Clinical Significance." Research and Reviews: Journal of Holistic Nursing 2, no. 2 (2025): 1–11. https://doi.org/10.5281/zenodo.15356016.

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<em>Since the Sickle Cell Anemia Act, there has been a steady improvement in sickle cell disease screening and treatment. Although new medical treatments have been developed, sickle cell disease is still incurable for the majority of those affected, and the only cure for those who have sickle cell disease is hematopoietic stem cell transplantation. Autologous hematopoietic stem cell transplantation involves genetically modifying the patient's stem cells to correct the genetic mutation that causes sickle cell disease. Allogeneic hematopoietic stem cell transplantation replaces the damaged stem

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Slatter, Mary A., Kanchan Rao, Persis Amrolia, et al. "Treosulfan-based conditioning regimens for hematopoietic stem cell transplantation in children with primary immunodeficiency: United Kingdom experience." Blood 117, no. 16 (2011): 4367–75. http://dx.doi.org/10.1182/blood-2010-10-312082.

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Abstract Children with primary immunodeficiency diseases, particularly those less than 1 year of age, experience significant toxicity after hematopoietic stem cell transplantation, with busulfan- or melphalan-based conditioning. Treosulfan causes less veno-occlusive disease than busulfan and does not require pharmacokinetic monitoring. We report its use in 70 children. Children received 42 g/m2 or 36 g/m2 with cyclophosphamide 200 mg/kg (n = 30) or fludarabine 150 mg/m2 (n = 40), with alemtuzumab in most. Median age at transplantation was 8.5 months (range, 1.2-175 months); 46 (66%) patients w

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Kalwak, Krzysztof, Monika Mielcarek, Katharine Patrick, et al. "Treosulfan–fludarabine–thiotepa-based conditioning treatment before allogeneic hematopoietic stem cell transplantation for pediatric patients with hematological malignancies." Bone Marrow Transplantation 55, no. 10 (2020): 1996–2007. http://dx.doi.org/10.1038/s41409-020-0869-6.

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Abstract Treosulfan-based conditioning prior to allogeneic transplantation has been shown to have myeloablative, immunosuppressive, and antineoplastic effects associated with reduced non-relapse mortality (NRM) in adults. Therefore, we prospectively evaluated the safety and efficacy of treosulfan-based conditioning in children with hematological malignancies in this phase II trial. Overall, 65 children with acute lymphoblastic leukemia (35.4%), acute myeloid leukemia (44.6%), myelodysplastic syndrome (15.4%), or juvenile myelomonocytic leukemia (4.6%) received treosulfan intravenously at a dos

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Nogai, Axel, Marc Thiele, Markus M. Heimesaat, Eckhard Thiel, Ulf B. Goebel, and Lutz Uharek. "Conditioning with Treosulfan and Cyclophosphamide without Application of Antibodies in MHC Mismatch Transplantations in Mice." Blood 108, no. 11 (2006): 5159. http://dx.doi.org/10.1182/blood.v108.11.5159.5159.

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Abstract BACKGROUND: Treosulfan is increasingly used in clinical conditioning regimens because of its myeloablative and immunosuppressive effects and the low hepatotoxicity compared to busulfane. The myeloablative and immunosuppressive characteristics of treosulfane was investigated in a murine MHC mismatch transplantation model. METHODS: C57BL/10 (H-2Db) female mice were treated with treosulfan (2000 mg/kg) on day -3 to -1 and increasing doses of cyclophosphamide (without, 100 or 200mg/kg) at day -1, or 3000 mg/kg treosulfan with and without 200 mg/kg cyclophosphamide at day -1. Some mice wer

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Slatter, Mary A., and Andrew R. Gennery. "Treosulfan-based conditioning for inborn errors of immunity." Therapeutic Advances in Hematology 12 (January 2021): 204062072110139. http://dx.doi.org/10.1177/20406207211013985.

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Inborn errors of immunity (IEI) are inherited disorders that lead to defects in the development and/or function of the immune system. The number of disorders that can be treated by haematopoietic stem-cell transplantation (HSCT) has increased rapidly with the advent of next-generation sequencing. The methods used to transplant children with IEI have improved dramatically over the last 20 years. The introduction of reduced-toxicity conditioning is an important factor in the improved outcome of HSCT. Treosulfan has myeloablative and immunosuppressive properties, enabling engraftment with less to

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Casper, Jochen, Wolfgang Knauf, Thomas Kiefer, et al. "Treosulfan and fludarabine: a new toxicity-reduced conditioning regimen for allogeneic hematopoietic stem cell transplantation." Blood 103, no. 2 (2004): 725–31. http://dx.doi.org/10.1182/blood-2002-11-3615.

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Abstract New conditioning regimens are being explored to reduce toxicity and enable allogeneic bone marrow transplantation in patients not eligible for conventional transplantation. We have investigated treosulfan, an alkylating agent, with the aim of developing an efficient and reliable but less-toxic conditioning regimen. A series of 30 patients who were not eligible for standard conditioning therapy received transplants from HLA-matched related (n = 14) or unrelated (n = 16) donors after administration of treosulfan 10 g/m2 intravenously daily for 3 days and fludarabine 30 mg/m2 intravenous

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Wojnar, Jerzy, Sebastian Giebel, Miroslaw Markiewicz, et al. "Treosulfan-Based Reduced Toxicity Regimen Prior to Allogeneic Hematopoietic Cell Transplantation in Non-Malignant Disorders." Blood 108, no. 11 (2006): 5245. http://dx.doi.org/10.1182/blood.v108.11.5245.5245.

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Abstract Graft rejection is a major cause of failure after alloHCT in non-malignant hematopoietic disorders including severe aplastic anemia (SAA) and paroxysmal nocturnal hemaoglobinuria (PNH). For patients with high risk of this complication we introduced a novel conditioning regimens, based on treosulfan - an alkylating agent possesing both immuno- and myeloablative properties. Between 2003–2006, eleven patients (age: 23(14–35) years) with SAA (n=6) or PNH (n=5) were treated in a single institution with alloHSCT from either HLA-identical sibling (n=3) or an unrelated volunteer (n=8). For pa

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ten Brink, Marloes H., Robbert G. M. Bredius, Juliëtte Zwaveling, et al. "Treosulfan-Based Conditioning in Pediatric Hematopoietic Stem Cell Transplantation: A Prospective Study on Pharmacokinetics and Early Clinical Outcomes." Blood 124, no. 21 (2014): 3865. http://dx.doi.org/10.1182/blood.v124.21.3865.3865.

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Abstract Introduction Treosulfan is an alkylating agent which is increasing applied in regimens prior to allogeneic hematopoietic stem cell transplantation (HSCT) in children. It has strong myeloablative and immunosuppressive activity and, in comparison with busulfan and total body irradiation, a relatively mild toxicity profile. The optimal dose of treosulfan in pediatric patients, remains to be established. To optimize the balance between treosulfan efficacy and toxicity, pharmacokinetic (PK) monitoring may be a valuable tool. With this purpose, we recently developed a PK and limited samplin

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Marzollo, Antonio, Elisabetta Calore, Manuela Tumino, et al. "Treosulfan-based conditioning regimen in sibling and alternative donor hematopoietic stem cell transplantation for children with sickle cell disease." Mediterranean Journal of Hematology and Infectious Diseases 9, no. 1 (2017): e2017014. http://dx.doi.org/10.4084/mjhid.2017.014.

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Background and objectives Lack of suitable donors and regimen related toxicity are major barriers for hematopoietic stem cell transplantation (HSCT) in patients with sickle cell disease (SCD) when employing the most frequently used Busulfan-based conditioning regimen. The aim of the study is the assessment of efficacy and toxicity of Treosulfan-based conditioning regimen for SCD also when alternative donors such as mismatched unrelated donor and haploidentical donor are employed.Methods We report our single-center experience: 11 patients with sickle cell disease received HSCT with a Treosulfan

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Suh, Jin Kyung, Ho Joon Im, Sung Han Kang, Hyery Kim, Eun Seok Choi, and Kyung-Nam Koh. "Successful Treatment Outcomes of Hematopoietic Stem Cell Transplantation with Reduced-Toxicity Conditioning Regimen Incorporating Treosulfan in Pediatric Patients with XIAP Deficiency." Blood 142, Supplement 1 (2023): 3922. http://dx.doi.org/10.1182/blood-2023-190661.

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Background X-linked inhibitor of apoptosis (XIAP) defciency is an inherited primary immunodefciency characterized by chronic infammasome overactivity and associated with hemophagocytic lymphohistiocytosis (HLH) and infammatory bowel disease (IBD). Although hematopoietic stem celltransplantation (HSCT) is the only curative therapy, the outcomes of HSCT for XIAP deficiency remain unsatisfactory. Methods We have performed HSCT with treosulfan based RTC regimen for pediatric patients with non-malignant disorders since January 2016, and reviewed the medical records of patients with XIAP deficiency

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