- |||||||||| Ovastat (treosulfan) / Medac, busulfan / Generic mfg.
[VIRTUAL] HEMATOPOETIC STEM CELL TRANSPLANTATION IN CD40 LIGAND DEFICIENCY (ePoster Area) - Feb 8, 2020 - Abstract #EBMT2020EBMT_1421; The conditioning regimens were predominantly based on myeloablative protocols that consisted of treosulfan or busulfan with weight-based dosages, except for two twin cases wherein a non-myeloablative regimen was used in their first transplantation. Our data show that HSCT for patients with CD40 ligand deficiency is a potentially effective treatment for long-term disease control.
- |||||||||| Rituxan (rituximab) / Biogen, Zenyaku Kogyo, Roche
[VIRTUAL] SINGLE-CENTER EXPERIENCE OF HAPLOIDENTICAL HEMATOPOIETIC STEM CELL TRANSPLANTATION WITH TCR αβ DEPLETION IN CHILDREN WITH PRIMARY IMMUNODEFICIENCY SYNDROMES (ePoster Area) - Feb 8, 2020 - Abstract #EBMT2020EBMT_1414; Most patients (74.1%) received reduced-toxicity myeloablative conditioning consisting of treosulfan (n=13) or busulfan (n=6), fludarabine and thiotepa...Fourteen patients received rituximab to reduce the risk of EBV-related posttransplantation lymphoproliferative disease...Graft versus host disease (GVHD) prophylaxis consisted of cyclosporine in 10 patients, CSA+mycophenolate mofetil in 16 patients... We conclude that use of TCR αβ depleted haploidentical transplantation ensures a high engraftment rate; good immune reconstitution; low incidence of significant aGvHD, and acceptable posttransplantation morbidity in children with a range of PIDs and should be considered in children with PIDs lacking an HLA-matched donor, when urgent HSCT is indicated.
- |||||||||| Campath (alemtuzumab) / Sanofi, tacrolimus / Generic mfg., cyclophosphamide intravenous / Generic mfg.
[VIRTUAL] BONE MARROW TRANSPLANT IN PATIENTS WITH SICKLE CELL ANAEMIA. EXPERIENCE IN ONE CENTRE (ePoster Area) - Feb 8, 2020 - Abstract #EBMT2020EBMT_1364; The outcome of our center, the largest HCST series in Spain is similar to the international cohort and confirms the role of HLA-identical sibling transplantation for children with SCD. Since 2015 we improved our results, with less toxicity and without mortality.
- |||||||||| Ovastat (treosulfan) / Medac
Preclinical, Journal: In Vitro Study of the Enzymatic and Nonenzymatic Conjugation of Treosulfan with Glutathione. (Pubmed Central) - Feb 7, 2020 Treosulfan does not undergo either spontaneous or enzymatic conjugation with GSH at a noticeable rate. The result indicates that the clearance of treosulfan is independent of glutathione S-transferase activity, GSH stores, and co-administration of drugs utilizing the GSH metabolic pathway.
- |||||||||| Natesto (testosterone) / Acerus, Aytu BioScience, Medac
The Effect of Natesto on Spermatogenesis, Reproductive Hormones, and Hypogonadal Symptoms. A Phase IV Study (ENDOExpo) - Feb 7, 2020 - Abstract #ENDO2020ENDO_1693; This phase IV clinical trial demonstrated that Natesto increased serum T, improved hypogonadal symptoms, maintained gonadotropins, testis volume, intratesticular testosterone and semen parameters. Natesto, and other short acting forms of testosterone therapy may help hypogonadal men maintain fertility.
- |||||||||| Ovastat (treosulfan) / Medac, spanlecortemlocel (MGTA-456) / Magenta Therap, busulfan / Generic mfg.
High Dose Hematopoietic Stem Cell Transplantation Leads to Rapid Neural and Peripheral Disease Cross-Correction Via Robust Hematopoietic and Microglia Recovery (World Center Marriott - Palms & Cypress Foyers) - Jan 14, 2020 - Abstract #TCTASTCTCIBMTR2020TCT_1083; At 1-16 weeks post-HSCT, transplant of increasing doses of total bone marrow cells following myeloablative busulfan conditioning led to a dose-dependent increase microglial engraftment (26-fold for 10x106 cells vs 0.3x106 cells, p<0.01), with 91-99% peripheral donor chimerism...In contrast, transplant of 10x106 cells into mice conditioned with a myeloablative dose of treosulfan, an agent that does not permit brain microglial engraftment (Figure A), did not correct CNS defects (Figure B-C), suggesting that donor engraftment in the CNS is required for disease correction...We show that high dose HSCT leads to improved disease correction, including normalization of behavioral outcomes, via robust engraftment. High dose cell therapies, like MGTA-456, may rapidly and durably resolve peripheral and neurologic disease in patients with IMDs and other neurodegenerative diseases caused by defective microglia.
- |||||||||| Ovastat (treosulfan) / Medac, fludarabine / Generic mfg., busulfan / Generic mfg.
Clinical, P3 data, Clinical Trial,Phase III, Journal, Head-to-Head: Treosulfan or busulfan plus fludarabine as conditioning treatment before allogeneic haemopoietic stem cell transplantation for older patients with acute myeloid leukaemia or myelodysplastic syndrome (MC-FludT.14/L): a randomised, non-inferiority, phase 3 trial. (Pubmed Central) - Jan 5, 2020 P3 Treosulfan was non-inferior to busulfan when used in combination with fludarabine as a conditioning regimen for allogeneic HSCT for older or comorbid patients with acute myeloid leukaemia or myelodysplastic syndrome. The improved outcomes in patients treated with the treosulfan-fludarabine regimen suggest its potential to become a standard preparative regimen in this population.
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