- |||||||||| Nplate (romiplostim) / Amgen, Promacta (eltrombopag) / Novartis, Rituxan (rituximab) / Roche
Real-World Retrospective Analysis of Immune Thrombocytopenia Patient Demographics and Treatment () - Dec 7, 2024 - Abstract #ASH2024ASH_7521;
The most common third-line treatment was Romiplostim (7 patients, 23%) and Rituximab (7 patients, 23%), whereas the fourth-line treatment was Eltrombopag (7 patients, 32%).Conclusion(s)This retrospective study provides the largest ITP analysis to our knowledge. Further research, including survival analysis, is needed to optimize the sequencing of subsequent ITP treatments and enhance patient outcomes in this challenging disease phenomenon.
- |||||||||| A Single-Center Study of Long-Term Efficacy and Discontinuation Patterns of Thrombopoietin Receptor Agonists in Chronic Immune Thrombocytopenia () - Dec 7, 2024 - Abstract #ASH2024ASH_7507;
The majority of the patients (65%) received eltrombopag as initial TPO-RA treatment, while 35% were treated with romiplostim...Ten patients (19%) who did not initially respond to eltrombopag, switched to another TPO-RA (90% avatrombopag), with 70% of them achieving CR and 20% yielding a durable response...Furthermore, these agents can lead to sustained responses, thus allowing for treatment discontinuation and ultimately contributing to life quality improvement. Finally, switching to another TPO-RA may be beneficial for patients who do not initially respond.
- |||||||||| Nplate (romiplostim) / Amgen
Durability of Treatment Free Platelet Response in Children with Chronic Immune Thrombocytopenic Purpura Previously Treated with Romiplostim () - Dec 7, 2024 - Abstract #ASH2024ASH_7502;
Patient characteristics or drug responses identified in clinical trials (Tarantino, Lancet 2016; Tarantino, Haematologica 2019) as being associated with TFR, younger age at first romiplostim dose and better platelet response in the first four weeks of romiplostim treatment were also suggested in this analysis, however the small number of patients in the non TFR group, perhaps thwarted the differences reaching statistical significance. Certainly, broader surveillance of long-term use of romiplostim in children with ITP will better characterize its safety, efficacy, and the occurrence of TFRs.
- |||||||||| Neupogen (filgrastim) / Kyowa Kirin, Amgen, Rituxan (rituximab) / Roche
A Rare Case of Warm Autoimmune Hemolytic Anemia and Massive Splenomegaly in Uncontrolled Grave's Disease () - Dec 7, 2024 - Abstract #ASH2024ASH_7309;
Clinical signs include anemia, jaundice, and mild to moderate splenomegaly.Case Presentation : A 36-year-old man with a history of Graves' disease (GD) (on methimazole) presented with jaundice...He was started on filgrastim 300 mcg 2 times a week for his leukopenia...The patient was discharged on daily prednisone and planned weekly rituximab infusions for AIHA...The response to treatment for AIHA suggests an autoimmune nature of the condition, but the underlying GD did not respond to medical therapy. Overall, this case adds to the literature, as a unique hematologic presentation of an uncontrolled autoimmune disorder.
- |||||||||| Clinical, Retrospective data, Review, Journal: protein kinase inhibitors as Targeted therapy for glioblastoma: A meta-analysis of randomized controlled clinical trials. (Pubmed Central) - Dec 6, 2024
Upon disease progression, the anti-vascular endothelial growth factor-A (VEGF-A) monoclonal antibody bevacizumab, can be considered...For instance, when comparing PKI treatment with other treatments, median OS and PFS showed no significant difference (-0.78 months, 95% CI, -2.12-0.55, p=0.25; -0.23 months, 95% CI, -0.79-0.34, p=0.43, respectively), and similar non-significant results were observed in the pooled analyses (OS: HR=0.89, 95% CI, 0.59-1.32, p=0.55; PFS: HR=0.83, 95% CI, 0.63-1.11, p=0.21). Despite these overall negative findings, some data indicate improved clinical outcomes in a subset of GBM patients treated with certain PKIs (i.e., regorafenib) and encourage further research to identify PKIs with better blood-brain barrier penetration and lower risk for resistance development.
- |||||||||| Tezspire (tezepelumab-ekko) / AstraZeneca, Amgen
Review, Journal, Stroma: Thymic Stromal Lymphopoietin (TSLP): Evidence in Respiratory Epithelial-driven Diseases Including Chronic Rhinosinusitis with Nasal Polyps. (Pubmed Central) - Dec 5, 2024
The role of TSLP in asthma is well established and supported by the efficacy of tezepelumab, the first anti-TSLP monoclonal antibody approved for both type 2 (T2)-high and T2-low severe asthma...While further research is required to clarify the extent of TSLP contribution in CRSwNP, this review highlights the potential of anti-TSLP therapies as a novel approach for managing severe, uncontrolled CRSwNP. If these preliminary findings are confirmed, targeting TSLP could become a promising strategy to treat CRSwNP with or without comorbid asthma.
- |||||||||| Enbrel (etanercept) / Pfizer, Amgen
Review, Journal: Guillain-Barr (Pubmed Central) - Dec 5, 2024
GBS was suspected based on her symptoms, and nerve conduction studies confirmed the diagnosis. She was successfully treated with plasmapheresis.
- |||||||||| CCX832 / GSK, Amgen
Journal: Vascular Chemerin from PVAT contributes to Norepinephrine and Serotonin-Induced Vasoconstriction and Vascular Stiffness in a Sex-Dependent Manner. (Pubmed Central) - Dec 5, 2024
Chemerin1 receptor antagonism via selective inhibitor CCX832 reduced maximal contraction to norepinephrine (NE) and serotonin (5-HT), but not angiotensin II, in isolated thoracic aorta (PVAT intact) from male Dahl SS rat...Aortic PVAT from females expressed less chemerin protein than males, suggesting PVAT as the primary source of active chemerin. We show that chemerin made by the PVAT amplifies NE and 5-HT-induced contraction and potentially induces aortic stiffening in a sex-dependent manner, highlighting the potential for chemerin to be a key factor in blood pressure control and aortic stiffening.
- |||||||||| umibecestat (CNP520) / Novartis, Amgen, Banner Alzheimer's Institute
Review, Journal: Small molecule inhibits BACE1 activity by a dual mechanism confirmed by (Pubmed Central) - Dec 5, 2024
We have used Umibecestat (CNP-520) as a positive control...The two shortlisted molecules were then subjected to atomistic molecular dynamics simulations study. Overall, our study proposes a much better inhibitor and a rational molecule for lead development against AD.
- |||||||||| Prolia (denosumab) / Amgen
Journal: Giant cell tumor of the cervical spine: A case report. (Pubmed Central) - Dec 5, 2024
Surgeons must have a high level of awareness and perform an adequate preoperative workup, before definitive treatment. The patient should undergo regular follow-ups to detect any recurrence or metastasis at an early stage.
- |||||||||| Enbrel (etanercept) / Pfizer, Amgen
Observational data, Retrospective data, Journal: Toxic epidermal necrolysis: management strategies in burns units. (Pubmed Central) - Dec 4, 2024
Current treatments may offer benefits, though their efficacy remains unclear. Clinicians in burns units should develop standardised protocols and collaborate on long-term studies to enhance patient care.
- |||||||||| Tezspire (tezepelumab-ekko) / AstraZeneca, Amgen
Journal: Exploratory Analysis of the Economically Justifiable Price of Tezepelumab for asthma severe in Colombia. (Pubmed Central) - Dec 4, 2024
The economically justifiable cost for tezepelumab in Colombia is between U$795 to U$3384 per dose, depending on the WTP used to decide its implementation. This result should encourage more studies in the region that optimize decision-making processes when incorporating this drug into the health plans of each country.
- |||||||||| Epogen (epoetin alfa) / Amgen
Trial completion date: Pharmacological Countermeasures for High Altitude (clinicaltrials.gov) - Dec 4, 2024
P4, N=18, Active, not recruiting,
Further research in larger cohorts is required to identify which marker should be considered gold standard. Trial completion date: Dec 2024 --> Mar 2025
- |||||||||| Prolia (denosumab) / Amgen, Evenity (romosozumab-aqqg) / Astellas, Amgen, UCB
Trial completion date, Trial primary completion date: Anabolic Therapy in Postmenopausal Osteoporosis (clinicaltrials.gov) - Dec 4, 2024
P4, N=50, Active, not recruiting,
Phase classification: P1b --> P1 Trial completion date: May 2024 --> May 2025 | Trial primary completion date: May 2024 --> May 2025
- |||||||||| Nplate (romiplostim) / Amgen
P2/3 data, Journal: Mitani K, Lee JW, Jang JH, et (Pubmed Central) - Dec 3, 2024
Trial completion date: Oct 2024 --> Oct 2025 No abstract available
- |||||||||| Prolia (denosumab) / Amgen
Review, Journal: Pregnancy- and Lactation-Associated Osteoporosis: A Literature Review Based on a Clinical Case. (Pubmed Central) - Dec 3, 2024
Therapies such as bisphosphonates, teriparatide, calcitonin, denosumab, and strontium ranelate have been evaluated, considering their effectiveness and safety...In conclusion, PLO poses a diagnostic challenge due to its low incidence and lack of comprehension of underlying mechanisms. Early diagnosis, personalized treatment, and strict monitoring are crucial for improving the prognosis of these patients.
- |||||||||| Imdelltra (tarlatamab-dlle) / Amgen
Journal, IO biomarker: Recent advances in immunotherapy for small cell lung cancer. (Pubmed Central) - Dec 3, 2024
Despite the improved outcomes associated with immunotherapy in SCLC, the overall clinical benefit remains modest. Further preclinical and clinical studies are essential to identify optimal treatment regimens and enhance therapeutic efficacy.
- |||||||||| Review, Journal: Update on treatment of ANCA-associated vasculitis (Pubmed Central) - Dec 3, 2024
Further preclinical and clinical studies are essential to identify optimal treatment regimens and enhance therapeutic efficacy. Rituximab (RTX) is used for remission induction and maintenance in organ-threatening and non-organ-threatening granulomatosis with polyangiitis (GPA) and microscopic polyangiitis (MPA)...The dosing of GC for remission induction in GPA and MPA is now lower than was previously used and additionally, avacopan is approved as a
- |||||||||| Aimovig (erenumab-aooe) / Amgen, Novartis
Retrospective data, Journal: Erenumab escalation in migraine - double dose without additional benefit - a retrospective experience. (Pubmed Central) - Dec 3, 2024
Rituximab (RTX) is used for remission induction and maintenance in organ-threatening and non-organ-threatening granulomatosis with polyangiitis (GPA) and microscopic polyangiitis (MPA)...The dosing of GC for remission induction in GPA and MPA is now lower than was previously used and additionally, avacopan is approved as a In this difficult-to-treat patient cohort we reconfirmed the effectiveness of erenumab, but could not detect any additional benefit for a dosage escalation from 70
- |||||||||| Ibrance (palbociclib) / Pfizer
Review, Journal: HDAC-driven mechanisms in anticancer resistance: epigenetics and beyond. (Pubmed Central) - Dec 3, 2024
Other targets of HDACs that are not histones can also contribute to resistance. This review describes the contribution of HDACs to the mechanisms that, in some cases, may determine resistance to chemotherapy or other cancer treatments.
- |||||||||| Prolia (denosumab) / Amgen, Pluvicto (lutetium Lu 177 vipivotide tetraxetan) / Novartis
Journal: Repurposing of PSMA-targeted diagnostic and therapeutic agents for the detection and treatment of giant cell tumors of bone. (Pubmed Central) - Dec 2, 2024
Giant cell tumor of bone (GCTB) is a rare bone tumor often necessitating surgical intervention, radiation therapy, or treatment with bisphosphonates or denosumab...PSMA-targeted imaging and radiotherapeutic agents Locametz and Pluvicto are a powerful theranostic pair for detecting and treating PSMA-positive metastatic tumors, including those in bone, and thus have considerable potential to be repurposed for GCTB...Incubation of fresh GCTB tissue samples with FAM-C6-1298 showed increased fluorescence intensity compared to controls, indicating successful targeting of PSMA in GCTB tissue. In conclusion, our data established that PSMA is not only present in the tumor vasculature of GCTB patient tissue but can be effectively targeted with repurposed PSMA-specific radiopharmaceuticals for diagnosis and therapy.
- |||||||||| Prolia (denosumab) / Amgen
Journal: McCune-Albright Syndrome: A Case From Mauritius. (Pubmed Central) - Dec 2, 2024
This case indicates that this novel therapy may be an efficacious option in other pulmonary neuroendocrine cancers. We present a 15-year-old male patient from Mauritius with MAS having multiple caf
- |||||||||| Clinical, Retrospective data, Review, Journal: Targeted agents in patients with progressive glioblastoma-A systematic meta-analysis of randomized clinical trials. (Pubmed Central) - Dec 2, 2024
The aim of this systematic meta-analysis was to establish evidence for the use of targeted therapies in progressive GB. While some studies demonstrated benefits for OS and/or PFS, those results have to be interpreted with caution as most studies had major methodological weaknesses, including potential differences in sample size, trial design, or the initial distribution of prognostic factors.
|
