- |||||||||| Ovastat (treosulfan) / Medac, Medexus
Donor Bone Marrow Derived Macrophage Engraftment into the Central Nervous System of Allogeneic Transplant Patients (GWCC - B308-B309, Level 3) - Nov 5, 2021 - Abstract #ASH2021ASH_2107; Donor engraftment is highest following myeloablative conditioning or in patients receiving multiple transplants, and lowest in non-myeloablative cases. Our studies document the magnitude of donor-derived macrophages in the CNS following a bone marrow transplant and serve as a basis for future gene therapy studies targeting neurodegenerative disorders.
- |||||||||| Enhertu (fam-trastuzumab deruxtecan-nxki) / Daiichi Sankyo, AstraZeneca, trastuzumab duocarmazine (SYD985) / Byondis
Clinical, Retrospective data, Journal: HER2-low status and response to neoadjuvant chemotherapy in HER2 negative early breast cancer. (Pubmed Central) - Nov 4, 2021 Our studies document the magnitude of donor-derived macrophages in the CNS following a bone marrow transplant and serve as a basis for future gene therapy studies targeting neurodegenerative disorders. Our data does not support HER2-low as a biologically distinct BC subtype, with no prognostic value on survival outcomes and no predictive effect for pCR after conventional NACT.
- |||||||||| Ovastat (treosulfan) / Medac, Medexus
Clinical, Journal: Successful mismatched hematopoietic stem cell transplantation for pediatric hemoglobinopathy by using ATG and post-transplant cyclophosphamide. (Pubmed Central) - Oct 15, 2021 We investigated hematopoietic stem cell transplantation (HSCT) outcomes of pediatric TM and SCD patients treated with a mismatched donor using a treosulfan-based conditioning in combination with ATG and post-transplant cyclophosphamide (PT-CY) and compared these results to the clinical outcome of patients treated by matched donor HSCT without PT-CY...Moreover, despite delayed neutrophil engraftment (day +22 vs. +26, p = 0.002) and immune recovery in the mismatched PT-Cy group, this did not result in more infectious complications. Therefore, we conclude that in the absence of an HLA-identical or a matched unrelated donor, HSCT with a mismatched unrelated or haploidentical donor in combination with ATG plus PT-CY can be considered a safe and effective treatment option for pediatric hemoglobinopathy patients.
- |||||||||| Ovastat (treosulfan) / Medac, Medexus
Clinical, P2 data, Journal: Treosulfan-Based Conditioning Regimen Prior to Allogeneic Stem Cell Transplantation: Long-Term Results From a Phase 2 Clinical Trial. (Pubmed Central) - Sep 28, 2021 P2 The aim of "AlloTreo" prospective phase 2 clinical trial was to evaluate the efficacy and safety of a conditioning regimen based on Treosulfan (42 g/m) and fludarabine (https://clinicaltrials.gov/ct2/show/NCT00598624)...Graft-versus-host disease (GvHD) prophylaxis consisted of cyclosporine-A and methotrexate...In multivariate analysis, older age (≥60 years) at transplant [hazard ratio (HR), 2.157; p = 0.004] and a high/very high disease risk index (HR, 1.913; p = 0.026) were significantly associated with a lower OS. Overall, our data confirmed the myeloablative potential and safe toxicity profile of full dose Treo (42 g/m) especially for the younger population.
- |||||||||| Ovastat (treosulfan) / Medac, Medexus, Thymoglobulin (anti-thymocyte globulin (rabbit)) / Sanofi
Journal: Treosulfan-Based Conditioning Regimen in Haematopoietic Stem Cell Transplantation with TCRαβ/CD19 Depletion in Nijmegen Breakage Syndrome. (Pubmed Central) - Sep 15, 2021 Twelve patients additionally received low-dose busulfan 4 mg/kg (Bu group) and 10 patients (including 2 recipients of a second HSCT) treosulfan (Treo group) 30 g/m...Low-dose Bu-containing CR in NBS leads to increased graft failure and low donor myeloid chimerism. Treo-CR followed by TCRαβ/CD19-depleted HSCT demonstrates a low level of early transplant-associated toxicity and enhanced graft function with stable donor chimerism.
- |||||||||| Spectrila (asparaginase Escherichia coli) / Medac
Enrollment closed: PK, PD, Safety and Immunogenicity of Spectrila in Adults With Acute B-cell Lymphoblastic Leukaemia (clinicaltrials.gov) - Sep 9, 2021 P2, N=40, Active, not recruiting, However, further studies, including studies about long-term toxicities and complications related to this regimen, are still needed to clarify and verify our conclusions. Recruiting --> Active, not recruiting
- |||||||||| Ovastat (treosulfan) / Medac, Medexus
[VIRTUAL] Cyclophosphamide-Based GVHD Prophylaxis After 9/10 and 10/10 HLA Tissue-Matched Unrelated Allogeneic Stem Cell Transplantation: A MultiCenter Experience () - Sep 6, 2021 - Abstract #SOHO2021SOHO_419; Comparative studies on anti-thymocyte globulin (ATG), calcineurin inhibitors, methotrexate, mycophenolatmofetil (MMF), post-transplant cyclophosphamide (PTCy), and their combinations are very limited...As a myeloablative regimen, 35 patients had TBi(800–1200 cGy)+Flu (30 mg/m2 x5), 5 had Cy (60 mg/kgx2)+TBi(1200 cGy),1 had treosulfan (10 gr/ m2 x3)+Flu (40 mg)/m2 x4)... GVHD prophylaxis containing PTCy after allo-HSCT from an unrelated donor is thought to be more effective and safe than ATG.
- |||||||||| Melblez Kit (melphalan hepatic delivery system) / Delcath
Review, Journal: A New Option for the Treatment of Intrahepatic Cholangiocarcinoma: Percutaneous Hepatic Perfusion with CHEMOSAT Delivery System. (Pubmed Central) - Aug 26, 2021 We reviewed available loco-regional therapeutic options, with particular focus on the CHEMOSAT Melphalan/Hepatic Delivery System (CS-HDS), which is uniquely positioned to perform a percutaneous hepatic perfusion (PHP), in order to treat the entire liver as a standalone or as complementary therapy...More than 650 procedures have been performed in Europe to date, mostly to treat liver metastases from UM. In CCA, experience is still limited, but retrospective analyses have been reported, while phase II and III studies are closed, waiting for results or ongoing.
- |||||||||| Ovastat (treosulfan) / Medac, Medexus
[VIRTUAL] Treosulfan Based Conditioning for Nonmalignant Diseases (CHANNEL C) - Jul 31, 2021 - Abstract #ICBMT2021ICBMT_60; In this presentation, I will review the recent progress in HSCT using treosulfan-based conditioning regimen in pediatric patients with non-malig- nant diseases. I will also introduce the clinical experience with this approach at our center.
- |||||||||| Ovastat (treosulfan) / Medac, Medexus
[VIRTUAL] HSCT Incorporation with Non-Traditional Agents: Clofarabine, Treosulfan (CHANNEL B) - Jul 31, 2021 - Abstract #ICBMT2021ICBMT_39; Clofarabine was mainly used as cytoreductive agents in the treatment of relapsing and refractory leukemia, but it has recently investigated as conditioning agent as well. Data available are encouraging but more data are needed to understand in which situations one drug may be better than another to further improve the prognosis of patients undergoing to hematopoietic stem cell transplantation.
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