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120 Diseases | | 9 Products |
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46 Trials |  |
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- | Ovastat (treosulfan) / Medac, Medexus
Journal: Valutazione economica di treosulfan in pazienti sottoposti a trapianto allogenico di cellule staminali ematopoietiche. (Pubmed Central) - Jan 12, 2023
No abstract available
- Ovastat (treosulfan) / Medac, Medexus
NURSES (Room 243) - Dec 27, 2022 - Abstract #EBMT2023EBMT_148;
No abstract available The conditioning regimen consisted of ATG, thiotepa, fludarabine and treosulfan...Pain medication included paracetamol, tramadol, morphine and gabapentin...
- | Natesto (testosterone) / Acerus, Medac, Aytu BioPharma
Enrollment change, Trial completion date, Trial withdrawal, Trial primary completion date: Natesto Testosterone Nasal Gel for Hypogonadal Men (clinicaltrials.gov) - Dec 21, 2022
P4, N=0, Withdrawn, Sponsor: University of Utah
The conditioning regimen consisted of ATG, thiotepa, fludarabine and treosulfan...Pain medication included paracetamol, tramadol, morphine and gabapentin... N=50 --> 0 | Trial completion date: Jun 2026 --> Apr 2025 | Not yet recruiting --> Withdrawn | Trial primary completion date: Jun 2025 --> Jan 2024
- Ovastat (treosulfan) / Medac, Medexus
Early Immune Reconstitution Following Treosulfan-Based Reduced Toxicity Conditioning in Patients Undergoing Allogeneic Transplantation () - Dec 16, 2022 - Abstract #TCTASTCTCIBMTR2023TCT_ASTCT_CIBMTR_1054;
Conclusion The present study provides insights into the eIR pattern and could offer cues to the immunologic complications following Treo-based conditioning. Further analysis is ongoing to delineate immune reconstitution signature in predicting immunologic complications such as GvHD post Treo-based conditioning.
- Ovastat (treosulfan) / Medac, Medexus
Glutathione S-Transferase Gene Promoter Polymorphism (GSTA1*B) Influences Haematopoietic Cell Transplantation (HCT) Outcome in Patients with β-Thalassemia Receiving Treosulfan/Fludarabine/Thiotepa Regimen () - Dec 16, 2022 - Abstract #TCTASTCTCIBMTR2023TCT_ASTCT_CIBMTR_841;
It is possible that GSTA1*B polymorphism affects Treo metabolism, increasing S, S-EBDM exposure causing early toxicities, Graft versus Host disease (GvHD), resulting in high TRM and inferior TFS. We continue to explore the functional relevance of this variant in Treo metabolism.
- Ovastat (treosulfan) / Medac, Medexus
Treosulfan-Based Conditioning Prior to Allogeneic Hematopoietic Stem Cell Transplantation (alloHCT) for Patients with Myelodysplastic Syndrome (MDS): Promising Survival Outcome Including Patients with High-Risk Disease () - Dec 16, 2022 - Abstract #TCTASTCTCIBMTR2023TCT_ASTCT_CIBMTR_737;
Our data verify the favorable outcome data for MDS patients treated with treosulfan-based conditioning prior to alloHCT. Moreover, survival after alloHCT seemed to depend on disease biology, defined by cytogenetic and molecular changes, rather than on disease burden.
- || Natesto (testosterone) / Acerus, Medac, Aytu BioPharma
Trial completion date, Trial primary completion date, Adverse events: A Comparison of Side Effects in Hypogonadal Men Treated With Natesto Versus Testosterone Injections (clinicaltrials.gov) - Dec 16, 2022
P4, N=200, Active, not recruiting, Sponsor: University of Miami
Moreover, survival after alloHCT seemed to depend on disease biology, defined by cytogenetic and molecular changes, rather than on disease burden. Trial completion date: Dec 2022 --> Dec 2023 | Trial primary completion date: Dec 2022 --> Jun 2023
- ||| Ovastat (treosulfan) / Medac, Medexus, busulfan / Generic mfg.
#lösemi nakil rejimi:Treosülfan mı Busulfan mı içermeli? Genç hastalarda nüks #treosulfan grubunda yüksek; 55 yaş üzeri hastalarda akut GVHD #busulfan grubunda yüksek #hematoloji #akutmyeloidlosemi #AML #transplantasyon #kokhucrenakli #profdrfevzialtuntas https://t.co/2OZ5MVP6FK (Twitter) - Dec 10, 2022
- | Ovastat (treosulfan) / Medac, Medexus
New P2 trial: Clinical Trial Investigating the Chemotherapeutic Compound Treosulfan (Trecondi (clinicaltrials.gov) - Dec 5, 2022
P2, N=120, Not yet recruiting, Sponsor: University Hospital Inselspital, Berne
- Prevymis (letermovir) / Merck (MSD), Ovastat (treosulfan) / Medac, Medexus, Rituxan (rituximab) / Biogen, Zenyaku Kogyo, Roche
Haploidentical Hematopoietic Stem Cell Transplant Plus Add-Back NK Cells in Pediatric High-Risk ALL () - Nov 29, 2022 - Abstract #ASH2022ASH_7917;
Patients 1 and 2 received chemo-based conditioning regimens consisting of thyotepa, and fludarabine; In addition, patient1received treosulfan, and patient 2 received busulfan...All patients received rituximab and letermovirfor EBV and CMV prophylaxis, respectively, for all patients...Thus, infusion of mature donor NK cells may help to maintain control of MRD and virus reactivation through immunologic surveillance. Although our preliminary observations seem to be promising, this hypothesis should be confirmed in clinical trial of a larger number of patients.
- Ovastat (treosulfan) / Medac, Medexus
Treosulfan Therapeutic Drug Monitoring: Optimizing Conditioning Therapy in Pediatric Hematopoietic Stem Cell Transplantation () - Nov 29, 2022 - Abstract #ASH2022ASH_7857;
Enrollment continues, and follow-up of patients should allow the definition of the relationship between personalized Treosulfan dosing, toxicity and efficacy. Therefore, the development of a drug monitoring system for Treosulfan may be an important tool to optimize outcomes in the pediatric population.
- Ovastat (treosulfan) / Medac, Medexus, Thymoglobulin (anti-thymocyte globulin (rabbit)) / Sanofi
Fludrabine Treosulfan Conditioning Regimen Appears Safe and Effective in Patients with Acute Myeloid Leukemia(AML) and Myelodysplastic Syndrome (MDS) Undergoing Allogenic Hematopoietic Stem Cell Transplantation (HSCT) () - Nov 29, 2022 - Abstract #ASH2022ASH_7839;
Rabbit Antithymocyte Globulin (ATG) (2.5-5 mg/kg)/ post-transplant cyclophosphamide (PTCy) were used for matched unrelated donor transplants...However the study is limited by its retrospective design, the conditioning regimens being tried in two different time periods and the shorter follow up of the FluTreo group. Considering the difficulties in the feasibility of conducting a prospective randomized control trial in this setting, we believe our study shows early insights into safety and improved efficacy of FluTreo conditioning regimen in AML and MDS .
- Obnitix (MSC-FFM) / Medac
Sars-Cov-2 Fails to Infect Mesenchymal Stromal Cells (MSCs) - Pre-Clinical Investigations in Support of Therapeutic Use of MSCs for Severe COVID-19 () - Nov 29, 2022 - Abstract #ASH2022ASH_7766;
We consistently observe an inflammatory cellular phenotype and transcriptome which is, however, entirely unaffected by the presence of SARS-CoV-2. These studies support the expectation of safety of Obnitix use in COVID-19 ARDS.
- | Venclexta (venetoclax) / Roche, AbbVie, Darzalex (daratumumab) / J&J
Journal: Targeted therapy with venetoclax and daratumumab as part of HSCT preparative regimen in children with chemorefractory acute myeloid leukemia.: venetoclax and daratumumab as part of HSCT preparative regimen for AD AML. (Pubmed Central) - Nov 28, 2022
At 2 years, nonrelapse mortality was 10%, relapse incidence was 46%, event-free survival was 44%, and overall survival was 65%. Our data show the possibility of safely adding targeted agents to conditioning regimens; however, no evidence of a significant improvement in long-term transplantation outcomes in this cohort of patients was observed.
- || Kidrolase (L-asparaginase) / Jazz, Merck (MSD), Spectrila (asparaginase Escherichia coli) / Medac
ALL-T: how do you use Spectrila, same scheme as Kidrolase or less?#spectrila # ALL-T @AaronGoodman33 @smbenlazar @nihardesai7 @melmehdi (Twitter) - Nov 19, 2022
- | Journal: Two new oral testosterone products for hypogonadism. (Pubmed Central) - Nov 18, 2022
Our data show the possibility of safely adding targeted agents to conditioning regimens; however, no evidence of a significant improvement in long-term transplantation outcomes in this cohort of patients was observed. No abstract available
- ||| Kadcyla (ado-trastuzumab emtansine) / Roche, Enhertu (fam-trastuzumab deruxtecan-nxki) / Daiichi Sankyo, AstraZeneca, trastuzumab duocarmazine (SYD985) / Byondis, Medac
Review, Journal, Adverse events: Interstitial lung disease associated with anti-HER2 anti-body drug conjugates: results from clinical trials and the WHO's pharmacovigilance database. (Pubmed Central) - Nov 15, 2022
The median time to the onset of event was 86 days and 54.95% of events occurred within 3 months. Trastuzumab emtansine, trastuzumab deruxtecan and trastuzumab duocarmazine increased the risk of ILD, which can lead to serious outcomes and tends to occur early.
- Natesto (testosterone) / Acerus, Medac, Aytu BioPharma
Trial initiation date: Natesto Testosterone Nasal Gel for Hypogonadal Men (clinicaltrials.gov) - Nov 8, 2022
P4, N=50, Not yet recruiting, Sponsor: University of Utah
Trastuzumab emtansine, trastuzumab deruxtecan and trastuzumab duocarmazine increased the risk of ILD, which can lead to serious outcomes and tends to occur early. Initiation date: Sep 2022 --> Jan 2023
- ||||| Ovastat (treosulfan) / Medac, Medexus, fludarabine IV / Generic mfg., thiotepa / Generic mfg.
Clinical: Toxicity ⚠️ - High SOS (6/39; cumulative incidence 15%); 4 received thiotepa-based conditioning, and 2 received fludarabine and treosulfan - 15.4% developed grade 3-4 aGVHD; 4 patients had steroid-refractory acute GvHD - 2-year NRM 26% 3/6 (Twitter) - Nov 7, 2022
- || Ovastat (treosulfan) / Medac, Medexus
P2 data, Retrospective data, Clinical Trial,Phase II, Journal: Thiotepa-fludarabine-treosulfan conditioning for 2nd allogeneic HCT from an alternative unrelated donor for patients with AML: a prospective multicenter phase II trial. (Pubmed Central) - Nov 5, 2022
Still, relapse rates and NRM after 2nd allogeneic HCT remain a challenge. The trial is registered in the German Clinical Trials Registry (number DRKS00005126).
- Thymoglobulin (anti-thymocyte globulin (rabbit)) / Sanofi
Tcraß+/CD19+-Depletion in Hematopoietic Stem Cells Transplantation from Matched Unrelated and Haploidentical Donors in Pediatric Acute Lymphoblastic Leukemia Patients in Complete Remission (ENMCC - 393-396) - Nov 4, 2022 - Abstract #ASH2022ASH_5263;
We confirm that the depletion of TCR-alpha/beta and CD19 lymphocytes from the graft ensures high engraftment rate and acceptable transplant-related mortality in pediatric ALL patients. Use of TBI and MRD negative status before HSCT had a significant correlation with good outcome.
- Ovastat (treosulfan) / Medac, Medexus
Pre-Transplant Multiparameter Flow Cytometric Measurable Residual Disease in Acute Myeloid Leukemia As an Independent Prognostic Factor for Survival after Myeloablative Allogeneic Hematopoietic Cell Transplantation (ENMCC - Hall D) - Nov 4, 2022 - Abstract #ASH2022ASH_4836;
P3
GvHD prophylaxis consisted of calcineurin inhibitor (CNI) combined with MTX (with or without ATG) or was based on post-transplant cyclophosphamide in transplants from haploidentical donor or mMUD...Conclusions Our findings confirm that pre-transplant residual disease at a =0.1% level assessed by MFC is an independent poor prognostic factor for both LFS and OS in AML patients treated with MAC alloHCT. As predicted, outcomes after MAC alloHCT were also independently influenced by ELN cytogenetic/genetic risk and age.
- Ovastat (treosulfan) / Medac, Medexus
Treosulfan-Based Conditioning Prior to Allogeneic Hematopoietic Cell Transplantation (alloHCT) for Patients with Myelodysplastic Syndrome (MDS): Promising Survival Outcome Including Patients with High-Risk Disease (ENMCC - Hall D) - Nov 4, 2022 - Abstract #ASH2022ASH_4761;
P3
Moreover, survival after alloHCT seemed to depend on disease biology, defined by cytogenetic and molecular changes, rather than on disease burden defined by bone marrow blast count. In context with recent published study data, future treatment strategies should focus on timely realization of alloHCT, when indicated, post-transplant maintenance and not on pre-transplant treatment intensification.
- Ovastat (treosulfan) / Medac, Medexus
Outcomes of Allogeneic Hematopoietic Stem Cell Transplantation for Congenital Amegakaryocytic Thrombocytopenia, a PDWP/EBMT Study (ENMCC - Hall D) - Nov 4, 2022 - Abstract #ASH2022ASH_3330;
This study demonstrates that HSCT, the only available potentially curative option, has an acceptable 6-year overall survival and an acceptable rate of chronic GvHD. While the number of patients in such a rare disease does not allow to perform multivariable analyses, we can confirm that OS is better with the use of BM as the main stem cell source compared to the CB.
- Ovastat (treosulfan) / Medac, Medexus
Long-Term Data Confirm the Superiority of Total Body Irradiation-Containing Conditioning Regimen in Comparison to a Chemotherapy-Based Preparation in Children with Acute Lymphoblastic Leukemia Above the Age of 4 Years Given an Unmanipulated Allograft. Results of the Forum Randomized Clinical Trial (ENMCC - Hall D) - Nov 4, 2022 - Abstract #ASH2022ASH_2998;
Children transplanted from a matched sibling donor (MSD) received cyclosporine A only as graft-versus-host disease (GvHD) prophylaxis, whereas recipients of UD HSCT also received short-term methotrexate and anti-thymocyte globulin (ATG)...Conclusions With a significantly longer follow-up, we confirm that the use of TBI during the conditioning regimen is associated with better outcomes in children and adolescents with ALL above the age of 4 years transplanted in CR, mainly due to a lower risk of leukemia recurrence. The number of cases of secondary malignancies, particularly in patients offered radiotherapy, deserves particular attention and underlines the importance of continuous safety monitoring over time to comprehensively evaluate the long-term sequels observed in this population.
- Ovastat (treosulfan) / Medac, Medexus
Superior Graft-Versus-Leukemia Effect in Matched Unrelated Donor Versus HLA-Identical Sibling Pediatric Recipients Transplanted for Acute Lymphoblastic Leukemia within the Forum Study (ENMCC - 343-345) - Nov 4, 2022 - Abstract #ASH2022ASH_1793;
Our data indicate that the use of MUD is no longer a risk factor in HSCT for childhood ALL. The greater immune-genetic disparity in the matched unrelated donor/recipient pairs protects from the risk of disease recurrence, while the use of serotherapy prevents the occurrence of chronic GvHD, with both factors contributing to the observed improved GRFS in patients transplanted from an unrelated donor.
- Ovastat (treosulfan) / Medac, Medexus
Relapse Is the Most Common Treatment Failure Post HSCT in Children with ALL below 4 Years of Age Given a Chemo-Based Conditioning Regimen. Results from the Prospective Multinational Forum-Trial (ENMCC - 343-345) - Nov 4, 2022 - Abstract #ASH2022ASH_1791;
Because of decreased NRM, these results represent an improvement as compared to previously reported series; however, KMT2A-rearrangement continues to be an obstacle to successful HSCT. Pre- and post-transplant immunotherapeutic strategies aimed at reducing relapse-incidence without increasing chemotherapy-associated complications are needed.
- ||| Jivadco (trastuzumab duocarmazine) / Byondis, Medac, sodium thiosulfate (BYON5667) / Byondis
Enrollment closed, Enrollment change: Evaluation of Safety and Efficacy of Sodium Thiosulfate (BYON5667) Eye Drops to Reduce Ocular Toxicity in Cancer Patients Treated With SYD985 (clinicaltrials.gov) - Nov 3, 2022
P1/2, N=48, Active, not recruiting, Sponsor: Byondis B.V.
Pre- and post-transplant immunotherapeutic strategies aimed at reducing relapse-incidence without increasing chemotherapy-associated complications are needed. Recruiting --> Active, not recruiting | N=196 --> 48
- Ovastat (treosulfan) / Medac, Medexus
MONOCENTRIC EXPERIENCE ON THE USE OF THE THIOTEPA TREOSULFANO-FLUDARABINE (TTF) SCHEME AS CONDITIONING REGIME FOR ALLOGENIC TRANSPLANT IN MYELOPHIBORISIS AND MYELODYSPLASIA (PLENARY ROOM) - Oct 29, 2022 - Abstract #SIE2022SIE_356;
- | oNKord (inaleucel) / Glycostem, Medac, Korea Kolmar
Enrollment open, Trial completion date, Trial primary completion date: INTRO: Intraperitoneal Infusion of ex Vivo-cultured Allogeneic NK Cells in Recurrent Ovarian Carcinoma Patients (clinicaltrials.gov) - Oct 28, 2022
P1, N=12, Recruiting, Sponsor: Radboud University Medical Center
Recruiting --> Active, not recruiting | N=196 --> 48 Unknown status --> Recruiting | Trial completion date: May 2020 --> Oct 2023 | Trial primary completion date: May 2020 --> May 2023
- || Natesto (testosterone) / Acerus, Medac, Aytu BioPharma
Enrollment closed, Adverse events: A Comparison of Side Effects in Hypogonadal Men Treated With Natesto Versus Testosterone Injections (clinicaltrials.gov) - Oct 18, 2022
P4, N=200, Active, not recruiting, Sponsor: University of Miami
Unknown status --> Recruiting | Trial completion date: May 2020 --> Oct 2023 | Trial primary completion date: May 2020 --> May 2023 Recruiting --> Active, not recruiting
- ||| Melblez Kit (melphalan hepatic delivery system) / Delcath
Trial termination: Melphalan for Use With the Hepatic Delivery System Treatment in Patients With Unresectable Hepatocellular Carcinoma or Intra Hepatic Cholangiocarcinoma (clinicaltrials.gov) - Oct 17, 2022
P2, N=17, Terminated, Sponsor: Delcath Systems Inc.
Recruiting --> Active, not recruiting Suspended --> Terminated; Limited enrollment
- Ovastat (treosulfan) / Medac, Medexus, Campath (alemtuzumab) / Sanofi, Amsidine (amsacrine) / Kyowa Kirin
REDUCED INTENSITY CONDITIONING FOR ALLOGENEIC STEM CELL TRANSPLANTATION (HSCT) IN ACUTE MYELOID LEUKEMIA () - Oct 16, 2022 - Abstract #EHOC2022EHOC_35;
Efficient safe pre transplant conditioning protocols are continuing to be developed. Future protocols will most probably incorporate specific anti leukemic targeted novel compounds as well as monoclonal and radiolabeled antibodies.
- | cyclophosphamide / Generic mfg., busulfan / Generic mfg.
Journal: Busulfan (Bu) and Cyclophosphamide (Cy) based conditioning regimen still holds the promise of being a safe and efficacious regimen for Allogeneic Transplantation in patients with Transfusion Dependent Thalassemia (TDT), even in high risk. (Pubmed Central) - Oct 13, 2022
In our experience Flu/Bu/Cy/ATG regimen is safe and effective even in high risk TDT. However, one needs to be vigilant for mixed chimerism.
- | Ovastat (treosulfan) / Medac, Medexus
Retrospective data, Journal: A retrospective study of treosulfan versus busulfan-based conditioning in pediatric patients. (Pubmed Central) - Oct 13, 2022
Busulfan-based conditioning regimens are used more frequently for children undergoing allogenic HSCT, but treosulfan-based conditioning is gaining acceptance. Treosulfan-based conditioning is associated with lower rates of acute GvHD, and no significant differences on overall survival were observed compared with busulfan.
- | Kadcyla (ado-trastuzumab emtansine) / Roche, trastuzumab duocarmazine (SYD985) / Byondis, Medac
Journal: Therapy-induced senescence enhances the efficacy of HER2-targeted antibody-drug conjugates in breast cancer. (Pubmed Central) - Oct 13, 2022
T-DM1, a trastuzumab-based ADC that relies on lysosomal processing to release the payload, is approved for HER2-positive breast cancer...Treatment with the DNA-damaging agent doxorubicin and CDK4/6 inhibitor induced lysosomal enlargement and senescence in several breast cancer cell lines...In breast cancer patient-derived xenografts, a combination treatment of CDK4/6 inhibitor and SYD985 showed improved anti-tumor effects over either treatment alone. These data support the strategy of combining next-generation ADCs targeting HER2 with senescence-inducing therapies for tumors with heterogenous and low HER2 expression.
- | Ovastat (treosulfan) / Medac, Medexus
Retrospective data, Journal: Late endocrine effects after hematopoietic stem cell transplantation in children with nonmalignant diseases. (Pubmed Central) - Oct 7, 2022
In females, treosulfan seems less gonadotoxic than busulfan. Careful long-term endocrine follow-up is indicated.
- oNKord (GCT NK cells) / Glycostem, Medac, Korea Kolmar
Cord blood CD34+ stem cells are efficiently transduced with anti-CD19-CAR and expanded and differentiated into viveNK™ Natural Killer cells which display selective cytotoxicity against B-cell leukemia (Hall C) - Oct 6, 2022 - Abstract #SITC2022SITC_582;
P1/2
Glycostem Therapeutics has developed a closed, automated, and feeder-free system for ex vivo expansion and differentiation of umbilical cord blood-derived CD34 + stem cells into highly functional NK cells, currently evaluated in a Phase I/II clinical study (ClinicalTrials.gov ID: NCT04632316 )...Additionally, inherent innate NK cell phenotype and responses and the mechanism of action driving CD19-CAR viveNK™ cytotoxicity were investigated via flow cytometry-based analysis and single-cell RNA-sequencing (scRNA-Seq) of CAR-transduced vs non-transduced donors. Conclusions Our data show how off-the-shelf, highly functional, and antigen-directed CAR-NK cells can be generated ex vivo, offering an option to target cancers which are often resistant or difficult to treat with standard immunotherapy.
- || Ovastat (treosulfan) / Medac, Medexus
Clinical, Journal: Comparative study of treosulfan plus Fludarabine (FT14) with busulfan plus Fludarabine (FB4) for acute myeloid leukemia in first or second complete remission: An analysis from the European Society for Blood and Marrow Transplantation (EBMT) Acute Leukemia Working Party (ALWP). (Pubmed Central) - Sep 25, 2022
In patients aged≥55 years, acute GVHD CI was higher in FB4, without significant differences in other outcomes. Although FT14 has been used for higher-risk HSCT patients, our large real-world multicenter study suggests that FB4 is associated with better outcomes compared to FT14 in younger patients.
- | Enhertu (fam-trastuzumab deruxtecan-nxki) / Daiichi Sankyo, AstraZeneca, trastuzumab duocarmazine (SYD985) / Byondis, Medac, zenocutuzumab (MCLA-128) / Merus
Journal: An Overview of Clinical Development of Agents for Metastatic or Advanced Breast Cancer Without ERBB2 Amplification (HER2-Low). (Pubmed Central) - Sep 20, 2022
This review suggests that ERBB2-low may be a distinct, clinically relevant breast cancer entity warranting reassessment of traditional diagnostic and therapeutic paradigms. Ongoing clinical trials and further investigations may provide optimized strategies for diagnosing and treating ERBB2-low breast cancer, including reproducible, consistent definitions to identify patients in this diagnostic category and demonstration of benefits of emerging therapies.
- Natesto (testosterone) / Acerus, Medac, Aytu BioPharma
Antenatal kidneys hypoplasia reveals a new variant of Townes-Brocks syndrome-1. (Poster Area - Exhibition Hall E) - Sep 20, 2022 - Abstract #IPNA2022IPNA_254;
Kidney structural abnormalities (hypoplasia, dysplasia, polycystic kidneys), sometimes leading to kidney failure, have already been reported but seldom as the main impairment especially during childhood. TBS-1 should be suspected in antenatally diagnosed kidneys hypoplasia.
- Ovastat (treosulfan) / Medac, Medexus
Steroid-resistant nephrotic syndrome (NS) after allogeneic hematopoietic stem cell transplantation (HSCT) in a girl with acute myeloid leukemia (AML) (Poster Area - Exhibition Hall E) - Sep 20, 2022 - Abstract #IPNA2022IPNA_88;
The coincidence of AML, HSCT and NS might be accidental. Nevertheless, the role of WT1 mutation, which is common phenomenon in AML, not previously related to NS in these patients, needs further evaluation.
- || Ovastat (treosulfan) / Medac, Medexus
New P1 trial: Effect of Moderate Renal Impairment and Race/Ethnicity on Treosulfan Pharmacokinetics (clinicaltrials.gov) - Sep 9, 2022
P1, N=36, Not yet recruiting, Sponsor: medac GmbH
- || Trodelvy (sacituzumab govitecan-hziy) / Everest Medicines, Gilead, Enhertu (fam-trastuzumab deruxtecan-nxki) / Daiichi Sankyo, AstraZeneca, trastuzumab duocarmazine (SYD985) / Byondis, Medac
Review, Journal: New antibody-drug conjugates (ADCs) in breast cancer-an overview of ADCs recently approved and in later stages of development. (Pubmed Central) - Sep 3, 2022
This overview focuses on the newer ADCs, including T-DXd and SG, their pharmacology, mechanisms of action, and relevant studies. In addition, the latest results from trials investigating some newer ADCs, in further stages of development are presented.
- | Ovastat (treosulfan) / Medac, Medexus
Journal: Treosulfan-based Reduced-intensity Allogeneic Hematopoietic Cell Transplantation in Adults With Primary Immunodeficiency: A Case Series. (Pubmed Central) - Aug 31, 2022
In addition, the latest results from trials investigating some newer ADCs, in further stages of development are presented. This series highlights issues of transplantation for PID in adults and treosulfan-based conditioning, which is feasible for PID patients; infectious complications are the major issue of concern.
- | Natesto (testosterone) / Acerus, Medac, Aytu BioPharma
Journal: An 86 amino acids motif in CAPN3 is essential for formation of the nucleolus-localized Def-CAPN3 complex. (Pubmed Central) - Aug 24, 2022
This complex mediates the degradation of the tumor suppressor p53 and ribosome biogenesis factor mitotic phosphorylated protein 10 (Mpp10) in nucleolus, demonstrating the importance of this complex in regulating cell cycle and ribosome biogenesis...We further identify the 2/3 C-terminus of hDef is responsible for mediating the hDef-hCAPN3 interaction, and the corresponding region is conserved for the zDef and zCapn3b interaction. Our results lay the ground to resolve the structure of the Def-CAPN3 complex in the future.
- | Ovastat (treosulfan) / Medac, Medexus
Retrospective data, Journal: Comparison of fludarabine-melphalan and fludarabine-treosulfan as conditioning prior to allogeneic hematopoietic cell transplantation-a registry study on behalf of the EBMT Acute Leukemia Working Party. (Pubmed Central) - Aug 10, 2022
However, patients treated with FluMel showed a reduced 3-year relapse incidence (32.4% vs. 40.4%, p value < 0.001) and slightly increased non-relapse mortality (NRM) (25.7% vs. 20.2%, p value = 0.06) compared to patients treated with FluTreo. Our data may serve as a basis for further studies examining the role of additional agents/ intensifications in conditioning prior to allo-HCT.
- | Ovastat (treosulfan) / Medac, Medexus
Retrospective data, Journal: The efficacy and safety of allogeneic stem cell transplantation in Mevalonate Kinase Deficiency. (Pubmed Central) - Aug 7, 2022
However, treatment-related morbidity and mortality are significant. Transplantation may be justified in patients with a severe disease course on conservative therapy.
- | Natesto (testosterone) / Acerus, Medac, Aytu BioPharma
Journal: Direct conversion from long-acting testosterone replacement therapy to Natesto allows for spermatogenesis resumption: Proof of concept. (Pubmed Central) - Aug 5, 2022
One couple achieved a pregnancy 4 months after converting to Natesto. Hypogonadal men on long-acting TRT interested in resumption of spermatogenesis may convert directly to Natesto for an opportunity to do so while remaining on a form of TRT and achieving lower E2 levels.
- | Ovastat (treosulfan) / Medac, Medexus
Observational data, Retrospective data, Journal: Thyroid disorders following hematopoietic stem cell transplantation in childhood: the impact of the conditioning regimen on thyroid dysfunction, volume changes and occurrence of nodules. (Pubmed Central) - Aug 5, 2022
Accordingly, BU can be regarded as the most thyrotoxic agent among those administered as a part of a chemo-only conditioning regimen. Finally, patients conditioned with TBI, but not with other regimens, show a progressive decrease in thyroid volume over time, as assessed by sequential ultrasounds.