- |||||||||| Iclusig (ponatinib) / Takeda, Otsuka, Blincyto (blinatumomab) / Astellas, Amgen, Besponsa (inotuzumab ozogamicin) / Pfizer, UCB
Clinical Implication and Long-term Survival Outcome of Second Allogeneic HCT in Adult Patients with Relapsed Acute Lymphoblastic Leukemia before and after the Era of Novel Immunotherapeutic Agents (Room A (2F)) - Sep 24, 2024 - Abstract #ICBMT2024ICBMT_2;
Nowadays, emerging novel immunotherapeutic agents like blinatumomab and inotuzumab ozogamicin (INO) have improved safe remission and accessibility to second allo-HCT...Among them, 85 achieved complete remission after salvage therapy (conventional chemotherapy until 2016 and the era of blinatumomab, INO, and ponatinib since late 2016) and followed by second allo-HCT...Conclusions : Our data suggest the long-term outcomes of second allo-HCT is comparable to recent CAR-T outcomes in both Ph-positive and Ph-negative ALL, but the improvements are more evident in Ph-negative ALL with novel immunotherapeutic agents. The influence of the quality of salvage response on second allo-HCT outcome remains uncertain.Figure1.
- |||||||||| Avastin (bevacizumab) / Roche, Stivarga (regorafenib) / Bayer, Muphoran (fotemustine) / Servier
A novel machine learning (ML) model integrating clinical and molecular data to predict response to second line treatment in recurrent IDHwt-glioblastoma (rGBM) (Hall 5) - Sep 24, 2024 - Abstract #EANO2024EANO_644;
Background: Nitrosoureas (lomustine/fotemustine) and antiangiogenic drugs (bevacizumab or regorafenib) are second-line treatment options for patients with rGBM, but to date predictors of efficacy are lacking. The multi-classification ML model developed in this study was able to identify clinical and molecular signatures of rGBM responding to second-line with bevacizumab or regorafenib or nitrosoureas .This model could be useful to choose the more effective second-line therapy in rGBM patients.
- |||||||||| Stivarga (regorafenib) / Bayer
Drug sensitivity of Recurrent GBM patient-derived organoids correlates with patients (Hall 5) - Sep 24, 2024 - Abstract #EANO2024EANO_556;
Our findings suggest a potential role for NADH-FLIM-based drug response assessment in rGBM-EXP for predicting the efficacy of second-line therapies and improving the overall clinical outcome. Further prospective studies are warranted to validate these findings and potentially guide treatment decisions in clinical practice.
- |||||||||| navtemadlin (KRT-232) / Kartos Therap
Functional genomic approaches to understand mechanisms of drug response and resistance (Alsh) - Sep 24, 2024 - Abstract #EANO2024EANO_52;
We have characterized the effect of navtemadlin, one such MDM2 inhibitor, through a window of opportunity clinical trial in patients diagnosed with HGG...In the second part of the talk, I will exemplify how genome-wide and targeted CRISPR activation screens in patient-derived HGG cell line models were able to identify putative cell programs modulating drug response in the absence of p53 inactivating mutations. Our ongoing efforts are focused on validating putative drivers of resistance to MDM2 and PPM1D inhibition in additional cell line and animal models, as well as identifying combination therapies for the upfront treatment of these tumors.
- |||||||||| Tepezza (teprotumumab-trbw) / Roche, Amgen
Interleukin-11 Receptor Antibody, LASN01, May Be a Novel Treatment for TED, Regardless of Prior Treatment (DEMAND) - Sep 24, 2024 - Abstract #AAO2024AAO_2298;
P2
Conclusion The ability of LASN01 to inhibit HA was equivalent to teprotumumab in OF and was not impacted by any prior treatment or health characteristics. LASN01 is currently being evaluated in a Phase 2 study (NCT06226545) in patients with active TED, including a cohort of patients with prior teprotumumab treatment, and this may offer a differentiated treatment option based on the data generated in OF.
- |||||||||| Tepezza (teprotumumab-trbw) / Roche, Amgen
Interleukin-11 Receptor Antibody, LASN01, Blocks Fibroinflammatory Drivers in TED (DEMAND) - Sep 24, 2024 - Abstract #AAO2024AAO_2297;
P2
Conclusion IL-11 may be a novel mediator in TED, and IL-11R inhibition with LASN01 has the potential to target multiple pathogenic mechanisms. A Phase 2 study with LASN01 is currently open for patients with TED (NCT06226545), including those previously treated with teprotumumab.
- |||||||||| Tepezza (teprotumumab-trbw) / Roche, Amgen
SD-OCT Imaging Findings in Patients Receiving Teprotumumab for TED (DEMAND) - Sep 24, 2024 - Abstract #AAO2024AAO_2296;
Conclusion There were significant decreases in choroidal, RNFL and GCC thickness in TED patients treated with teprotumumab. Further research is needed to understand the full effect of retinal IGF-1R blockade.
- |||||||||| Tepezza (teprotumumab-trbw) / Roche, Amgen
Untangling Teprotumumab's Adverse Events Through a National Analysis (POSTER THEATER) - Sep 24, 2024 - Abstract #AAO2024AAO_1829;
Conclusion Besides teprotumumab's previously described AEs, we found positive signals for lesser-described events, like increased blood pressure, permanent deafness and diverticulitis. Further investigation is needed.
- |||||||||| Tepezza (teprotumumab-trbw) / Roche, Amgen
Identified Risks and Stratification for Teprotumumab-Associated Chronic Ototoxicty (S405) - Sep 24, 2024 - Abstract #AAO2024AAO_1674;
Conclusion Teprotumamab-induced chronic ototoxicty occurs when patients have baseline hearing loss or demonstrate decline by the midpoint of therapy or are elderly, current smokers or super proptosis responders. Determining patients at risk will be beneficial in determining who may be likely to experience chronic ototoxicty and obviating comorbidity.
- |||||||||| Ibrance (palbociclib) / Pfizer, Tafinlar (dabrafenib) / Novartis
Journal: Mechanistic Regulation of Epidermal Growth Factor and Hormonal Receptors by Kinase Inhibitors and Organofluorines in Breast Cancer Therapy. (Pubmed Central) - Sep 24, 2024
Molecular simulation studies revealed Dabrafenib's thermodynamically stable interactions (?G), tighter binding, and less structural deviation in the order EGFR?>?HER-2?>?ER?>?PR as compared to Palbociclib (HER-2?>?ER?>?PR?=?EGFR). These results indicate that Dabrafenib, compared to Palbociclib, more effectively regulates breast cancer cell proliferation through specific interactions with hormonal and growth factor receptors towards a repurposing approach.
- |||||||||| Review, Journal, IO biomarker: Targeted and combination immunotherapies using biologics for gastric cancer: the state-of-the-art. (Pubmed Central) - Sep 24, 2024
Additionally, bemarituzumab, ananti-FGFR2b monoclonal antibody, has shown improvements in combination withchemotherapy in those with HER2 negative GAC with FGFR2 overexpression...Lastly, TROP-2 has emergedas an exciting solid tumor target and study is expected in GAC. All three ofthese therapeutic targets have seen an abundance of drug development in recentyears, and we anticipate newer targeted agents driving therapeutic decisions inGAC in the coming years.
- |||||||||| Prolia (denosumab) / Amgen
Journal: Inhibition of choroidal osteoma progression using bisphosphonate and RANKL-inhibitory treatment. (Pubmed Central) - Sep 24, 2024
A patient with bilateral posterior choroidal osteomas was treated with bisphosphonates and RANK ligand inhibitors to prevent osteoma resorption in the better-seeing left eye. This has led to stability over six years of outer retinal layers thickness, choroidal osteoma thickness, and choroidal osteoma calcification.
- |||||||||| Review, Journal: Evolving Horizons in Pediatric Leukemia: Novel Insights, Challenges, and the Journey Ahead. (Pubmed Central) - Sep 23, 2024
This review also discusses the long-term outcomes among pediatric leukemia survivors, focusing on late-onset side effects associated with treatments such as chemotherapy and bone marrow transplantation, encompassing secondary malignancies, organ dysfunction, and neurocognitive impacts. Ongoing research and clinical trials are crucial to refine these therapies, enhance their efficacy, and reduce adverse effects, ultimately improving young patients' survival and quality of life.
- |||||||||| Ibrance (palbociclib) / Pfizer
PK/PD data, Journal, Metastases: Exploring pharmacokinetic variability of palbociclib in HR+/HER2- metastatic breast cancer: a focus on age, renal function, and drug-gene interactions. (Pubmed Central) - Sep 23, 2024
Furthermore, exploring the drug-drug-gene interactions between inhibitors of CYP3A and P-glycoprotein with their respective genetic variants revealed two patient groups with statistically different percentage deviations (p = 0.0075, p = 0.0012, and p = 0.0191, respectively). These results could help address cases where pharmacokinetic covariates or subclinical conditions impair palbociclib adherence or response, aiming to offer tailored dosing strategies or monitoring for individual patients.
- |||||||||| Tavneos (avacopan) / Amgen
From Lice to the Heart and Kidneys (Exhibit Hall, Convention Center) - Sep 23, 2024 - Abstract #KIDNEYWEEK2024KIDNEY_WEEK_4261;
Here we present a case of Bartonella-associated dual-positive myeloperoxidase (MPO) and PR3 renal-limited ANCA vasculitisCase Description: A 55-year-old male with a history of stage 3 chronic kidney disease, heart failure with preserved ejection fraction (HFpEF), severe mitral regurgitation (MR), Hepatitis B on entecavir, and Hepatitis C presented with shortness of breath, lower extremity edema, and abdominal distention...Tissue confirmed Bartonella endocarditis and he was started on doxycycline and rifampin...Treatment with immunosuppression began within days of starting antimicrobial therapy and resulted in early signs of renal recovery nearing discharge. Early initiation of Avacopan therapy (post completion of rifampin to avoid its inducing effects) can be utilized to expedite a prednisone taper in patients with underlying infectious complications or need for adequate wound-healing 10x magnification of PAS stain showing diffuse cellular crescents
- |||||||||| Tavneos (avacopan) / Amgen
Case Series of Avacopan in Dual-Positive Anti-glomerular Basement Membrane (GBM) and ANCA Disease (Exhibit Hall, Convention Center) - Sep 23, 2024 - Abstract #KIDNEYWEEK2024KIDNEY_WEEK_4257;
Treatment include steroids, plasma exchange(PLEX) & cyclophosphamide(CYC)...She was treated with CYC, rituximab & PLEX...One patient was managed without steroids, a rare approach in the management of anti-GBM disease & all maintained independent renal function. We suggest Avacopan should be considered in the management of patients with dual positive antibodies
- |||||||||| Tavneos (avacopan) / Amgen
Use of Avacopan outside the ADVOCATE Inclusion Criteria (Exhibit Hall, Convention Center) - Sep 23, 2024 - Abstract #KIDNEYWEEK2024KIDNEY_WEEK_4227;
This small case series demonstrate successful use of Avacopan outside ADVOCATE inclusion criteria. Further real-life data is likely to support use of Avacopan for a wider and more severe presentations of AAV.
- |||||||||| Tavneos (avacopan) / Amgen
Characteristics and Treatment Patterns before Initiation of Avacopan in the United States (Exhibit Hall, Convention Center) - Sep 23, 2024 - Abstract #KIDNEYWEEK2024KIDNEY_WEEK_4225;
Among initial users of avacopan in the US, most had kidney (including ESKD and dialysis) and pulmonary involvement, and used GCs in the year before starting avacopan. Future studies will assess outcomes following treatment with avacopan in a real-world setting.
- |||||||||| Tavneos (avacopan) / Amgen
Antineutrophil Cytoplasmic Autoantibody Levels in Patients in the Avacopan Phase 3 Trial (Exhibit Hall, Convention Center) - Sep 23, 2024 - Abstract #KIDNEYWEEK2024KIDNEY_WEEK_4223;
The addition of avacopan to RTX or CYC for treatment of GPA or MPA does not appear to impact ANCA levels. The lack of correlation between ANCA levels and outcome of treatment in the ADVOCATE trial suggests that ANCA levels are an unreliable marker of treatment response.
- |||||||||| Tavneos (avacopan) / Amgen
Baseline Characteristics of the First Patients in AvacoStar, a Real-World Study of Avacopan in ANCA-Associated Vasculitis (Exhibit Hall, Convention Center) - Sep 23, 2024 - Abstract #KIDNEYWEEK2024KIDNEY_WEEK_4222;
P
Funding: Commercial Support - Vifor Fresenius Medical Care Renal Pharma AG Background: Avacopan, an oral, selective C5a receptor antagonist, was approved by the EMA in January 2022 for the treatment of adults with severe, active granulomatosis with polyangiitis (GPA) or microscopic polyangiitis (MPA), in combination with rituximab or cyclophosphamide. AvacoStar baseline characteristics are currently similar between groups and consistent with clinical practice, suggesting study outcomes may yield generalizable insights on safety and use patterns of avacopan.
- |||||||||| Tavneos (avacopan) / Amgen
General, Nervous System, Eye, and Skin Involvement in the Phase 3 Trial of Avacopan for the Treatment of ANCA-Associated Vasculitis (Exhibit Hall, Convention Center) - Sep 23, 2024 - Abstract #KIDNEYWEEK2024KIDNEY_WEEK_4221;
AvacoStar baseline characteristics are currently similar between groups and consistent with clinical practice, suggesting study outcomes may yield generalizable insights on safety and use patterns of avacopan. In the ADVOCATE trial, treatment with either avacopan or a prednisone taper was associated with the reversal of nearly all active general, nervous system, mucous membranes/eyes, and skin manifestations of GPA or MPA.
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