- |||||||||| Journal: The new biologic drugs: Which children with asthma should get what? (Pubmed Central) - Nov 27, 2024
We must be especially aware of the dangers of extrapolating data from adults to children, because the pathophysiology and role of biomarkers may differ significantly from adult asthma. Given the current level of knowledge, even after treatment has been initiated, children should be regularly reviewed to determine the efficacy of treatment, side-effect profile and consideration of when treatment with the biologic should be discontinued.
- |||||||||| Blincyto (blinatumomab) / Astellas, Amgen, Darzalex (daratumumab) / J&J, Rituxan (rituximab) / Roche
Journal, IO biomarker: Delineating MYC-Mediated Escape Mechanisms from Conventional and T Cell-Redirecting Therapeutic Antibodies. (Pubmed Central) - Nov 27, 2024
In conclusion, MYC overexpressing tumor cells mitigated the efficacy of therapeutic antibodies through several non-overlapping mechanisms. Given the challenges associated with direct MYC inhibition due to toxicity, successful modulation of MYC-mediated immune evasion mechanisms may improve the outcome of immunotherapeutic approaches in B-cell malignancies.
- |||||||||| Ibrance (palbociclib) / Pfizer, Kisqali (ribociclib) / Novartis
Journal, Metastases: Evaluation of CDK4/6 inhibitors in first-line in symptomatic and asymptomatic patients with metastatic breast cancer. (Pubmed Central) - Nov 26, 2024
Multivariate analysis identified the symptomatic disease and liver metastasis as independent predictors of shorter mPFS (HR; 1.835, 95% CI; 1.146-2.939 and HR; 2.433, 95% CI; 1.329-4.454, respectively). Our analysis revealed that although symptomatic individuals who underwent CDK4/6 inhibitor plus ET experienced a significant reduction in mPFS durations compared to asymptomatic patients, the 22-month mPFS indicated that CDK4/6 inhibitor plus ET is an effective treatment option.
- |||||||||| Humira (adalimumab) / AbbVie
Journal: Presumed Fungal Retinitis Following Adalimumab Therapy for Juvenile Idiopathic Arthritis: A Rare Case Report. (Pubmed Central) - Nov 26, 2024
A vitreous biopsy was performed, and intravitreal voriconazole was administered, but microbiological tests were negative...By the 6-week follow-up, her retinitis had fully resolved, maintaining excellent visual acuity. This case underscores the need for a high index of suspicion for infection in patients with long-term immunosuppression, highlighting the importance of early therapeutic intervention.
- |||||||||| Ravicti (glycerol phenylbutyrate) / Immedica, Amgen
Phenylbutyrate for syngap1-related Developmental and Epileptic Encephalopathy (South Hall H - Level 1 - LACC) - Nov 26, 2024 - Abstract #AES2024AES_2354;
Glycerol phenylbutyrate is a promising novel therapy for children with SYNGAP1. In ongoing work, we are (1) reviewing developmental assessment data and (2) enrolling additional children with SYNGAP1 and other monogenetic developmental and epileptic encephalopathies.
- |||||||||| Zolinza (vorinostat) / Merck (MSD), Ravicti (glycerol phenylbutyrate) / Immedica, Amgen
Mechanistic Insights into Pharmacological Interventions for SLC6A1 Variant-related Disorders (South Hall H - Level 1 - LACC) - Nov 26, 2024 - Abstract #AES2024AES_2289;
These findings suggest that the pharmacological agents tested may offer viable therapeutic options. Ravicti (glycerol-phenylbutyrate) is currently in clinical trials for SLC6A1-related disorders, but this study aims to tease out mechanisms to identify alternative therapeutic treatments.
- |||||||||| Tezspire (tezepelumab-ekko) / AstraZeneca, Amgen
Journal: Efficacy of tezepelumab in patients with severe asthma and persistent airflow obstruction. (Pubmed Central) - Nov 26, 2024
For patients with PAO at baseline, the proportion without PAO at week 52 was higher with tezepelumab (12.1%) than placebo (6.6%) (odds ratio 1.96, 95% CI: 1.30-2.94). Tezepelumab improved lung function and reduced exacerbations versus placebo in patients with severe, uncontrolled asthma with and without PAO.
- |||||||||| Blincyto (blinatumomab) / Astellas, Amgen
Retrospective data, Journal, IO biomarker: Childhood acute lymphoblastic leukemia with CREBBP gene mutation: a clinical analysis of 14 cases (Pubmed Central) - Nov 26, 2024
ALL with CREBBP gene mutation is more common in girls and has a low induction remission rate and a high recurrence rate, and it is often accompanied by other types of gene mutations and abnormal karyotypes. Most children with recurrence can achieve long-term survival after immunotherapy or hematopoietic stem cell transplantation.
- |||||||||| Opdivo (nivolumab) / BMS, Stivarga (regorafenib) / Bayer, Yervoy (ipilimumab) / BMS
Clinical, P1 data, Journal, Metastases: Updated survival outcome of regorafenib, ipilimumab, and nivolumab in refractory microsatellite stable non-liver metastatic colorectal cancer: A phase I nonrandomized clinical trial. (Pubmed Central) - Nov 25, 2024
P1
Most children with recurrence can achieve long-term survival after immunotherapy or hematopoietic stem cell transplantation. With extended follow-up, RIN combination therapy demonstrated durable clinical benefit in a subset of patients with NLM MSS metastatic colorectal cancer, including potential cure in 3 responders who remain disease-free >
- |||||||||| Ibrance (palbociclib) / Pfizer, Inlyta (axitinib) / Pfizer, Cabometyx (cabozantinib tablet) / Exelixis
Journal, Adverse drug reaction: Profile of adverse drug reactions reported via the Continuum+ platform: Results from three-year regional follow-up. (Pubmed Central) - Nov 25, 2024
The most used drugs were tyrosine kinase inhibitors: palbociclib (29.7%), axitinib (16.2%), and cabozantinib (13.2%)...The lack of medical elements and context in notifications is a massive problem for analyzing pharmacovigilance reports. Improved access to the platform's medical information for RCPVs and pharmacovigilance training for healthcare professionals would make Continuum+ a helpful tool in pharmacovigilance.
- |||||||||| Praluent (alirocumab) / Sanofi, Regeneron, Repatha (evolocumab) / Amgen, Astellas
Retrospective data, Journal: Association of PCSK9 inhibitors with risk of nonmelanoma skin cancer: a retrospective cohort study. (Pubmed Central) - Nov 25, 2024
Improved access to the platform's medical information for RCPVs and pharmacovigilance training for healthcare professionals would make Continuum+ a helpful tool in pharmacovigilance. Our study indicated ASCVD patients with PCSK9 inhibitors have a lower risk of incident NMSC than those without PCSK9 inhibitors.
- |||||||||| Folotyn (pralatrexate) / Aurobindo
Journal: Pralatrexate represses the resistance of HCC cells to molecular targeted agents via the miRNA-34a/Notch pathway. (Pubmed Central) - Nov 25, 2024
In HCC cells, knockdown of DHFR or treatment with pralatrexate enhanced the sensitivity of HCC cells to molecularly targeted agents, such as sorafenib, regorafenib, lenvatinib, cabozantinib, or anlotinib...Therefore, pralatrexate upregulates the sensitivity of HCC cells to molecularly targeted drugs. These results expand our understanding of folate metabolism and HCC and can help provide more options for HCC treatment.
- |||||||||| Avastin (bevacizumab) / Roche, Mvasi (bevacizumab-awwb) / Daiichi Sankyo, Amgen, AbbVie
Journal, HEOR: Comparison of Efficacy, Safety, and Economic Outcomes Between Biosimilar ABP 215 and Originator Bevacizumab in Japanese Patients With Colorectal Cancer. (Pubmed Central) - Nov 25, 2024
Economic outcomes indicated a potential savings of approximately 800,000 Japanese yen per patient with biosimilar ABP 215 use. Conclusions Although this is a single-center, retrospective, observational study with limitations in terms of the number of cases and background factors, the use of the bevacizumab biosimilar ABP 215 product is recommended in Japan from the perspective of reducing medical costs, given the findings of no differences in efficacy and safety.
- |||||||||| Nplate (romiplostim) / Amgen
Journal: Role of Serum Interleukin-10 and Interleukin-27 Levels in the Prognosis of Immune Thrombocytopenia in Iraqi Children. (Pubmed Central) - Nov 25, 2024
No abstract available The included immune thrombocytopenic children were sub-grouped according to their treatment into three groups: Romiplostim group (group 1), Prednisolone group (group 2), Prednisolone and intravenous immunoglobulin (IVIG) or Prednisolone and mycophenolate group (group 3)...The mean value of platelet count of patient children was significantly increased by all types of treatment in whole immune thrombocytopenic children (117.48
- |||||||||| dexamethasone / Generic mfg., floxuridine / Generic mfg.
Enrollment closed, Enrollment change, Trial completion date, Trial primary completion date: Hepatic Artery Infusion Pump Chemotherapy With Floxuridine and Dexamethasone in Combination With Systemic Chemotherapy for Patients With Colorectal Cancer Metastatic to the Liver (clinicaltrials.gov) - Nov 25, 2024
P2, N=23, Active, not recruiting,
The included immune thrombocytopenic children were sub-grouped according to their treatment into three groups: Romiplostim group (group 1), Prednisolone group (group 2), Prednisolone and intravenous immunoglobulin (IVIG) or Prednisolone and mycophenolate group (group 3)...The mean value of platelet count of patient children was significantly increased by all types of treatment in whole immune thrombocytopenic children (117.48 Recruiting --> Active, not recruiting | N=40 --> 23 | Trial completion date: Dec 2030 --> Jan 2027 | Trial primary completion date: Dec 2029 --> Dec 2026
- |||||||||| Review, Journal: The evolution of BRAF-targeted therapies in melanoma: overcoming hurdles and unleashing novel strategies. (Pubmed Central) - Nov 25, 2024
The review also delves into the role of advanced therapeutic tools, such as mRNA vaccines and CRISPR-Cas9, in revolutionizing personalized oncology by targeting specific genetic mutations and enhancing immune responses against melanoma. The ongoing synergy between advancing research, targeted interventions, strategic treatment combinations, and cost-effectiveness evaluations offers a promising pathway to elevate patient outcomes in the persistent battle against melanoma significantly.
- |||||||||| AMG 193 / Amgen
Trial completion date, Trial primary completion date: A Phase 2 Study of AMG 193 in Participants With MTAP-deleted Advanced NSCLC (clinicaltrials.gov) - Nov 25, 2024
P2, N=200, Not yet recruiting,
The ongoing synergy between advancing research, targeted interventions, strategic treatment combinations, and cost-effectiveness evaluations offers a promising pathway to elevate patient outcomes in the persistent battle against melanoma significantly. Trial completion date: Sep 2029 --> Feb 2029 | Trial primary completion date: Sep 2027 --> Feb 2027
- |||||||||| Tepezza (teprotumumab-trbw) / Roche, Amgen
Review, Journal: Advances of IGF-1R inhibitors in Graves' ophthalmopathy. (Pubmed Central) - Nov 23, 2024
Clinical trials have shown that teprotumumab reduces proptosis better than placebo, and may be beneficial for patients with worsening disease after steroid cessation. In this review, we discuss the role and prospects of IGF-1R inhibitors in thyroid-associated ophthalmopathy.
- |||||||||| Clinical, Review, Journal: Lp(a): A Rapidly Evolving Therapeutic Landscape. (Pubmed Central) - Nov 22, 2024
While there are presently no Lp(a)-lowering drugs available for routine clinical use, several promising candidates are currently under investigation. If these prove to be effective in randomized clinical trials, they will expand the cardiovascular care armamentarium and will allow clinicians to treat a presently unmitigated cardiovascular risk factor.
- |||||||||| Prolia (denosumab) / Amgen
Journal: Cellular signatures in human blood track bone mineral density in postmenopausal women. (Pubmed Central) - Nov 22, 2024
Our study reveals the distinctive identity of human cOCPs and the potential link between the dynamic regulation of cOCPs and osteoporosis and its treatment. Taken together, our study enhances our understanding of human cOCPs and highlights a potential opportunity to measure cOCPs through a simple blood test, which could potentially identify high-risk individuals.
- |||||||||| Ibrance (palbociclib) / Pfizer, NMI-900 / Nemucore, barasertib (AZD1152) / AstraZeneca
Journal, PD(L)-1 Biomarker, IO biomarker: Cell cycle inhibitors activate hypoxia-induced DDX41-STING pathway to mediate anti-tumor immune response in liver cancer. (Pubmed Central) - Nov 22, 2024
We observed a trend that Paclitaxel suppressed STINGWT HCC more effectively than STINGKO HCC, suggesting that STING might contribute to the anti-tumor effects of Paclitaxel. Our study revealed the immune-mediated tumor-suppressing properties of cell cycle inhibitors and suggested combined treatment with immunotherapy as a potential therapeutic approach.
- |||||||||| alsevalimab (FPA150) / Amgen
Phase classification, Trial termination, Metastases: FPA150-001: FPA150 in Patients With Advanced Solid Tumors (clinicaltrials.gov) - Nov 22, 2024
P1, N=95, Terminated,
https://osf.io/x4m28/. Phase classification: P1a/1b --> P1 | Completed --> Terminated; The Sponsor terminated the study for reasons unrelated to safety.
- |||||||||| Tepezza (teprotumumab-trbw) / Roche, Amgen
Journal: Teprotumumab for the treatment of Thyroid eye disease. (Pubmed Central) - Nov 21, 2024
Since 2020, over 5, 800 patients have been treated with teprotumumab and it appears to be well tolerated. The American Thyroid Association and the European Thyroid Association have recommended it as first line therapy for patients with moderate to severe TED, who display features of proptosis and diplopia.
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