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  • ||||||||||  azacitidine / Generic mfg.
    ALLOGENEIC HAEMATOPOIETIC STEM CELL TRANSPLANTATION IN CHILDREN AND ADOLESCENTS WITH RUNX1 GERMLINE MUTATIONS: A JOINT EBMT PDWP AND EWOG-MDS ANALYSIS (South Hall 2A+B / Virtual Hall 5) -  Jan 30, 2022 - Abstract #EBMT2022EBMT_996;    
    In 19/32 patients with available information the karyotype was normal; karyotypic abnormalities included deletion 5 q (2), monosomy 7 (2), deletion 7q (1), trisomy 8 (2), and others (6). Two patients (1 AML, 1MDS EBt) received azacytidine, 15 patients (10 AML, 3 MDS-EB, 1 MPAL, 1T-ALL) received intensive chemotherapy before HSCT...30/35 patients received myeloablative conditioning with busulfan (20), treosulfan (6) or total body irradiation based (4)... HSCT is well tolerated in children and adolescents with RUNX1 germline mutation and outcomes appear to be dependent on the stage of disease. An improved understanding of the natural history of the disease will hopefully identify patients who are more prone to transformation and for whom a pre-emptive approach might be appropriate.
  • ||||||||||  Ovastat (treosulfan) / Medac, Medexus
    SECOND HEMATOPOIETIC STEM CELL TRANSPLANT IN CHILDREN WITH RELAPSED JMML: A THERAPEUTIC OPTION WITH LIMITED TOXICITY. (South Hall 2A+B / Virtual Hall 5) -  Jan 30, 2022 - Abstract #EBMT2022EBMT_995;    
    Most patients (13/20, 65%) received a busulfan-based conditioning for the first HSCT, 7/20 had a matched sibling donor (MSD), 6/20 a matched unrelated donor (MUD), 7 had a mismatched unrelated donor (MMUD)...Conditioning regimen for the second HSCT was treosulfan-based in 11/20... Second HSCT in JMML achieved high rates of remission and survival, similar to those described after first HSCT, with very low TRM. Despite the limitation of a small sample size and retrospective analysis, our data indicate that second HSCT should be considered in children with JMML who relapse after first HSCT, due to the high curative potential, low toxicity and lack of alternative therapeutic approaches.
  • ||||||||||  Ovastat (treosulfan) / Medac, Medexus
    TREOSULFAN-BASED COMPARED TO THIOTEPA-BUSULFAN-FLUDARABINE CONDITIONING FOR HAPLOIDENTICAL TRANSPLANT IN PATIENTS WITH ACUTE MYELOID LEUKEMIA.A STUDY FROM THE ACUTE LEUKEMIA WORKING PARTY OF THE EBMT. (Forum Hall / Virtual Hall 2) -  Jan 30, 2022 - Abstract #EBMT2022EBMT_979;    
    Only patients who received post-transplant cyclophosphamide as graft-versus-host disease (GVHD) prophylaxis were included in the analysis.Myeloablative conditioning (MAC) was defined as a busulfan dose of 9.6mg/Kg for TBF (TBF-MAC) and a treosulfan dose of 42 g/m2 for Treo (Treo-MAC).Reduced intensity conditioning (RIC) was defined as a busulfan dose of 6.4 mg/Kg for TBF (TBF-RIC) and a treosulfan dose less than or equal to 36 mg/m2 for Treo (Treo-RIC).  These data confirm that TBF represents a valid preparatory regimen for haploidentical transplant in patients with AML in remission, providing acceptable NRM and good survival.Treosulfan-based conditioning seems to provide a valid alternative which is associated with similar outcomes to TBF and warrants further investigation in this setting.
  • ||||||||||  Ovastat (treosulfan) / Medac, Medexus
    HEMATOPOIETIC STEM CELL TRANSPLANTATION IN ARPC1B DEFICIENCY (North Hall / Virtual Hall 6) -  Jan 30, 2022 - Abstract #EBMT2022EBMT_845;    
     This is the first case series reporting outcome after HSCT in patients with diagnosis of ARPC1B deficiency. The severe clinical phenotype at diagnosis and the high survival rate with limited transplant-related morbidity reported strongly support the indication to allo-HSCT for patients with this diagnosis.
  • ||||||||||  Ovastat (treosulfan) / Medac, Medexus
    Journal:  Treosulfan in platinum-resistant ovarian cancer. (Pubmed Central) -  Jan 29, 2022   
    In general, the treatment was well tolerated. Taking the mild adverse events and the response rates into account, palliative treosulfan mainly seems beneficial for patients with performance status 0-1.
  • ||||||||||  Natesto (testosterone) / Acerus, Medac, Aytu BioPharma
    Clinical, Journal:  Trabecular bone score in women with differentiated thyroid cancer on long-term TSH-suppressive therapy. (Pubmed Central) -  Jan 19, 2022   
    Trabecular microarchitecture assessed by TBS was similar between women on long-term suppressive therapy in DTC and those on LT4 replacement therapy aiming at a TSH level within the low-normal reference range. Low TBS values were observed in postmenopausal women of both groups, suggesting that not only suppressed TSH levels but also a low-normal TSH is associated with deteriorated bone microarchitecture in postmenopausal women following total thyroidectomy.
  • ||||||||||  Ovastat (treosulfan) / Medac, Medexus
    Durable Remissions of Refractory Lymphoma in Patients with Underlying Immunodeficiencies Treated with Allogeneic HSCT (Salt Palace Convention Center Hall A) -  Jan 9, 2022 - Abstract #TCTASTCTCIBMTR2022TCT_ASTCT_CIBMTR_1289;    
    Correction of the immune defect, if present, can lead to long-term remission, even with disease refractory to chemotherapy. Discovery of PID in these patients can change the priorities of therapy – potentially leading to earlier HSCT instead of repeated courses of potentially futile chemotherapy.
  • ||||||||||  Ovastat (treosulfan) / Medac, Medexus
    T-Replete Cord Blood Transplantation for High-Risk Acute Myeloid Leukemia in Children (Salt Palace Convention Center Hall A) -  Jan 9, 2022 - Abstract #TCTASTCTCIBMTR2022TCT_ASTCT_CIBMTR_1069;    
    Many studies have described reduced relapse rate and hence improved survival after CB transplant compared to other cell sources. The rate of chronic GVHD is also low in UCB recipients despite having HLA-mismatch compared with the well-matched MUD recipients.
  • ||||||||||  Ovastat (treosulfan) / Medac, Medexus, Kineret (anakinra) / SOBI, Ilaris (canakinumab) / Novartis
    Journal:  HAPLOIDENTICAL α/β T-CELL AND B-CELL STEM CELL TRANSPLANTATION IN SEVERE MEVALONATE KINASE DEFICIENCY. (Pubmed Central) -  Jan 1, 2022   
    Prophylaxis with anakinra in the acute phase after transplant could represent a safe and effective approach. Further biological studies are required to clarify the pathophysiology of inflammatory attacks in MKD in order to better define the therapeutic role of SCT.
  • ||||||||||  oNKord (GCT NK cells) / Glycostem, Radboud University
    Journal:  Boosting Natural Killer Cell Therapies in Glioblastoma Multiforme Using Supramolecular Cationic Inhibitors of Heat Shock Protein 90. (Pubmed Central) -  Dec 21, 2021   
    Here, we describe the use of a blood-brain barrier (BBB) permissive supramolecular cationic drug vehicle comprising an inhibitor of the chaperone heat shock protein 90 (Hsp90), which sustains a cytotoxic effect on GBM cells, boosts the expression of MICA/B and ULBPs on the residual population, and augments the activity of clinical-grade aNK cells (GTA002)...Using a longitudinal in vitro model, we demonstrate >350% relative cell killing is achieved in SCI-101-treated cell lines compared to vehicle controls. In summary, these data provide a first-of-its-kind BBB-penetrating, long-acting inhibitor of Hsp90 with monotherapy efficacy, which improves response to aNK cells and thus may rapidly alter the treatment paradigm for patients with GBM.
  • ||||||||||  Ovastat (treosulfan) / Medac, Medexus
    Journal:  Docking analysis of aryl derivatives of diepoxide alkylating agents. (Pubmed Central) -  Nov 11, 2021   
    Both linearly bonded and fused aryl rings in higher number, between the epoxide rings, gave the strongest binding with the binding energy up to -8.1 and -8.7 Kcal/mol, respectively. These relationships can immensely help in designing and synthesis of derivatives of treosulfan like diepoxide based alkylating agents.
  • ||||||||||  Campath (alemtuzumab) / Sanofi, Thymoglobulin (anti-thymocyte globulin (rabbit)) / Sanofi
    Complex Clonal Evolution Can Occur Following Transplantation for Transfusion Dependent Thalassaemia in the Context of Mixed Myeloid Chimerism and Reduced Conditioning Regimens (GWCC - Hall B5, Level 1) -  Nov 5, 2021 - Abstract #ASH2021ASH_4555;    
    All patients had pre-transplantation endogenous haemopoiesis was suppressed pre-transplantation with hypertransfusions for a minimum of 8 weeks, and/or the use of hydroxycarbamide and azathioprine...A second BMT with busulfan based conditioning resulted in long-term cure...In conclusion, complex clonal evolution also occurs post HSCT in TDT, at least in the context of reduction of conditioning intensity and development of mixed myeloid chimerism. This finding warrants further investigation and may have significant implications for the design of both conventional HSCT and gene therapy strategies.
  • ||||||||||  cyclosporine / Generic mfg.
    Immediate Allogeneic Hematopietic Stem Cell Transplantation for Patients with NPM1-Mutated AML in Molecular Relapse (GWCC - Hall B5, Level 1) -  Nov 5, 2021 - Abstract #ASH2021ASH_3909;    
    The conditioning regimen consisted of Treosulfan (Treo, 30g/m 2 ) and Fludarabin (Flu, 120mg/m 2 )...Immunosuppression consisted of ATG and Ciclosporin in combination with MTX or MMF and posttransplant Cyclophosphamide for haploidentical HSCT (2 pts)...Although hematological regeneration took longer for these pts compared to pts without melphalan, there were not more infectious or bleeding complications...2 pt relapsed 6 and 13 mos after HSCT, but reached CR again after DLI (1 pt.) and venetoclax(1pt)...Conclusion This analysis shows the efficacy and feasibility of early allogeneic HSCT without reinduction chemotherapy in molecular or early hematological relapse for NPM1- mutated AML pts. For this strategy pts have to be monitored strictly at regular intervals after end of chemotherapy consolidation, because most relapses can be detected at molecular level.
  • ||||||||||  cisplatin / Generic mfg.
    Mutational Consequences of Chemotherapy in Hematopoietic Stem and Progenitor Cells of Pediatric Cancer Patients (GWCC - Hall B5, Level 1) -  Nov 5, 2021 - Abstract #ASH2021ASH_3766;    
    Conclusions Enhanced mutation accumulation after pediatric cancer treatment is caused by both direct and indirect mechanisms. The variance in mutagenic effects of (chemo)therapies on healthy HSPCs may influence the risk an individual patient has to develop t-AML and could, in future, play a role in t-AML risk assessment of pediatric cancer survivors and the development of new treatment regimens.