scholarly journals Review of Treatment Options for Myelofibrosis: Focus on Ruxolitinib

2013 ◽  
Vol 5 ◽  
pp. CMT.S9566
Author(s):  
Donal P. McLornan ◽  
John Laurie ◽  
Claire N. Harrison
Keyword(s):  
Treatment Options ◽  
Clinical Trial Data ◽  
Primary Myelofibrosis ◽  
The United States ◽  
Janus Kinase ◽  
European Medicines Agency ◽  
Polycythaemia Vera ◽  
Jak2 Inhibitor ◽  
United States Food ◽  
States Food

In November 2011, the United States Food and Drug Administration (FDA) approved the use of a novel Janus Kinase (JAK) 1/JAK2 inhibitor, INCB 018424 (ruxolitinib), for use in both intermediate and high risk myelofibrosis. Approvals of this agent in both Canada and Europe have followed most recently. The European Medicines Agency (EMA) concluded that ruxolitinib was indicated for disease-related splenomegaly or symptoms in adult patients with primary myelofibrosis, post-polycythaemia vera (PV) myelofibrosis, and post-essential thrombocythaemia (ET) myelofibrosis. In this review we will consider the rationale for targeting of the JAK-pathway, discuss the pharmacological profile of ruxolitinib and review the currently available clinical trial data. We will also postulate on the current and potential future roles of ruxolitinib within the MPN field.

Potassium binders

ESC CardioMed ◽  
2018 ◽  
pp. 218-221
Author(s):  
Keld P. Kjeldsen ◽  
Juan Tamargo ◽  
Thomas A. Schmidt
Keyword(s):  
United States ◽  
Clinical Pharmacology ◽  
Gastrointestinal Tract ◽  
The United States ◽  
European Medicines Agency ◽  
Sodium Polystyrene Sulfonate ◽  
United States Food ◽  
States Food ◽  
Potassium Binders ◽  
Sodium Zirconium Cyclosilicate

Potassium binders are used for the treatment of and prophylaxis against hyperkalaemia. Already in 1958, the United States Food and Drug Administration (FDA) approved sodium polystyrene sulfonate, a potassium binder exchanging sodium for potassium in the gastrointestinal tract. In 2015, the FDA approved a new potassium binder, patiromer sorbitex calcium (Veltassa®), exchanging calcium for potassium, and in 2017, it was approved by the European Medicines Agency (EMA). Furthermore, in 2018, the FDA and the EMA approved another new potassium binder, sodium zirconium cyclosilicate (Lokelma®), exchanging sodium for potassium. The clinical pharmacology aspects of potassium binders are reviewed in this chapter.

Potassium binders

ESC CardioMed ◽  
2018 ◽  
pp. 218-221
Author(s):  
Keld P. Kjeldsen ◽  
Juan Tamargo ◽  
Thomas A. Schmidt
Keyword(s):  
United States ◽  
Clinical Pharmacology ◽  
Gastrointestinal Tract ◽  
The United States ◽  
European Medicines Agency ◽  
Sodium Polystyrene Sulfonate ◽  
United States Food ◽  
States Food ◽  
Potassium Binders ◽  
Sodium Zirconium Cyclosilicate

Potassium binders are used for the treatment of and prophylaxis against hyperkalaemia. Already in 1958, the United States Food and Drug Administration (FDA) approved sodium polystyrene sulfonate, a potassium binder exchanging sodium for potassium in the gastrointestinal tract. In 2015, the FDA approved a new potassium binder, patiromer sorbitex calcium (Veltassa®), exchanging calcium for potassium, and in 2017, it was approved by the European Medicines Agency (EMA). Furthermore, in January 2018 approval by the FDA of another new potassium binder, sodium zirconium cyclosilicate (ZS-9®), exchanging sodium for potassium, is pending. The clinical pharmacology aspects of potassium binders are reviewed in this chapter.

Nephrogenic systemic fibrosis

2013 ◽  
pp. 1001-1006
Author(s):  
Cate H. Orteu
Keyword(s):  
United States ◽  
Renal Failure ◽  
Contrast Agents ◽  
Drug Administration ◽  
Nephrogenic Systemic Fibrosis ◽  
Food And Drug Administration ◽  
The United States ◽  
European Medicines Agency ◽  
United States Food ◽  
States Food

Nephrogenic systemic fibrosis (NSF) is a severe and disabling fibrosing condition of the skin and systemic organs. It occurs in patients with renal failure who have been exposed to gadolinum-based contrast agents (GBCAs). The development of NSF should be preventable if adequate precautions are taken in relation to GBCA exposure in susceptible patients. New cases should be reported to the European Medicines Agency and/or the United States Food and Drug Administration.

scholarly journals Severe haematuria of lower urinary tract origin with low dose dabigatran use in three Indian elderly patients: unresolved issues in the safety of novel oral anticoagulants

2017 ◽  
Vol 9 (1) ◽  
pp. 89-91 ◽  
Author(s):  
Upinder Kaur ◽  
Sankha Shubhra Chakrabarti ◽  
Sukdev Manna ◽  
Indrajeet Singh Gambhir
Keyword(s):  
Oral Anticoagulants ◽  
The Elderly ◽  
The United States ◽  
Novel Oral Anticoagulants ◽  
Thrombin Inhibitor ◽  
European Medicines Agency ◽  
Oral Direct Thrombin Inhibitor ◽  
United States Food ◽  
States Food ◽  
Lower Urinary

Dabigatran is a newer oral direct thrombin inhibitor approved by the United States Food and Drug Administration and the European Medicines Agency (EMA). The proper dosage of the drug, the potential for adverse drug reactions and the nature of bleeds with use of this drug as with other novel oral anticoagulants (NOACs), in the elderly population are still areas of uncertainty. Despite the existence of a specific antibody, idarucizumab which is an antidote to dabigatran toxicity, management of dabigatran-induced bleeds is an undefined area especially in resource constrained settings. We report severe haematuria with dabigatran in three elderly Indian patients at the lowest recommended therapeutic dose and explore these grey zones in dabigatran therapy.

scholarly journals Evolving AAV-delivered therapeutics towards ultimate cures

2021 ◽  
Author(s):  
Xiangjun He ◽  
Brian Anugerah Urip ◽  
Zhenjie Zhang ◽  
Chun Christopher Ngan ◽  
Bo Feng
Keyword(s):  
Gene Therapy ◽  
Gene Editing ◽  
Treatment Options ◽  
Genetic Diseases ◽  
The United States ◽  
European Medicines Agency ◽  
Gene Therapies ◽  
Therapeutic Modalities ◽  
United States Food

AbstractGene therapy has entered a new era after decades-long efforts, where the recombinant adeno-associated virus (AAV) has stood out as the most potent vector for in vivo gene transfer and demonstrated excellent efficacy and safety profiles in numerous preclinical and clinical studies. Since the first AAV-derived therapeutics Glybera was approved by the European Medicines Agency (EMA) in 2012, there is an increasing number of AAV-based gene augmentation therapies that have been developed and tested for treating incurable genetic diseases. In the subsequent years, the United States Food and Drug Administration (FDA) approved two additional AAV gene therapy products, Luxturna and Zolgensma, to be launched into the market. Recent breakthroughs in genome editing tools and the combined use with AAV vectors have introduced new therapeutic modalities using somatic gene editing strategies. The promising outcomes from preclinical studies have prompted the continuous evolution of AAV-delivered therapeutics and broadened the scope of treatment options for untreatable diseases. Here, we describe the clinical updates of AAV gene therapies and the latest development using AAV to deliver the CRISPR components as gene editing therapeutics. We also discuss the major challenges and safety concerns associated with AAV delivery and CRISPR therapeutics, and highlight the recent achievement and toxicity issues reported from clinical applications.

scholarly journals Immunotherapies in Genitourinary Oncology: Where Are We Now? Where Are We Going?

Cancers ◽  
2021 ◽  
Vol 13 (20) ◽  
pp. 5065
Author(s):  
Albert Jang ◽  
David M. Adler ◽  
Grant P. Rauterkus ◽  
Mehmet A. Bilen ◽  
Pedro C. Barata
Keyword(s):  
Clinical Trials ◽  
Immune Checkpoint ◽  
Checkpoint Inhibitors ◽  
Treatment Options ◽  
The United States ◽  
The Past ◽  
United States Food ◽  
States Food ◽  
Genitourinary Cancers ◽  
Tumor Types

For decades, limited options existed to treat metastatic genitourinary cancers, including treatment options that could be classified as immunotherapy. Historically, immunotherapy centered on systemic cytokines for the treatment of metastatic kidney cancer, which had several adverse effects, as well as the Bacillus Calmette–Guérin vaccine for non-metastatic bladder cancer. Within the past decade, advances in immunotherapy have led to several approvals from the United States Food and Drug Administration, particularly in the field of immune checkpoint inhibition. Immune checkpoint inhibitors (ICIs) are now being used extensively to treat multiple solid tumors, including kidney and bladder cancers, and they are also being tested in many other cancers. Despite encouraging data from phase 2/3 clinical trials, less is known about biomarkers that may predict better response to ICIs. The effect of ICIs in genitourinary cancers is heterogeneous, with some tumor types having little clinical data available, or ICIs having limited activity in other tumors. In this review, we briefly discuss approved immunotherapy agents prior to the time of ICIs. Then, given the emergence of this class of agents, we summarize the several important ICIs and the clinical trials that led to their approval. Finally, we mention ongoing and future clinical trials.

scholarly journals Management Options in Advanced Prostate Cancer: What is the Role for Sipuleucel-T?

2011 ◽  
Vol 5 ◽  
pp. CMO.S5977 ◽  
Author(s):  
Rhonda L. Bitting ◽  
Andrew J. Armstrong ◽  
Daniel J. George
Keyword(s):  
Prostate Cancer ◽  
Clinical Trial Data ◽  
The United States ◽  
Abiraterone Acetate ◽  
Castration Resistant Prostate Cancer ◽  
Management Options ◽  
Castration Resistant ◽  
United States Food ◽  
States Food ◽  
Us Fda

Most prostate cancer-related deaths occur in patients with castration-resistant prostate cancer (CRPC). Until recently, only therapy with docetaxel and prednisone has been shown to prolong survival in men with metastatic CRPC. With the United States Food and Drug Administration (US FDA) approvals of sipuleucel-T, cabazitaxel, and abiraterone acetate, all based on improvement in overall survival, the landscape for management of men with metastatic CRPC has dramatically changed. In this review we will discuss the pivotal clinical trial data leading to these approvals, with particular focus on the unique indication for sipuleucel-T and the implications for optimal management and sequencing of treatment in this patient population.

scholarly journals Emerging treatment options for patients with high-risk myelodysplastic syndrome

2020 ◽  
Vol 11 ◽  
pp. 204062072095500
Author(s):  
Jan Philipp Bewersdorf ◽  
Hetty Carraway ◽  
Thomas Prebet
Keyword(s):  
Checkpoint Inhibitors ◽  
Treatment Options ◽  
The United States ◽  
Driver Mutations ◽  
Hematopoietic Stem ◽  
Oral Agents ◽  
United States Food ◽  
Risk Of Progression ◽  
Variable Success ◽  
States Food

Myelodysplastic syndromes (MDS) are clonal hematopoietic stem cell disorders characterized by ineffective hematopoiesis with peripheral blood cytopenias, dysplastic cell morphology, and a variable risk of progression to acute myeloid leukemia (AML). The hypomethylating agents (HMA) azacitidine and decitabine have been used for over a decade in MDS treatment and lead to a modest survival benefit. However, response rates are only around 40% and responses are mostly transient. For HMA-refractory patients the prognosis is poor and there are no therapies approved by the United States Food and Drug Administration. Combinations of HMAs, especially along with immune checkpoint inhibitors, have shown promising signals in both the frontline and HMA-refractory setting. Several other novel agents including orally available and longer acting HMAs, the BCL-2 inhibitor venetoclax, oral agents targeting driver mutations ( IDH1/2, FLT3), immunotherapies, and new options for intensive chemotherapy have been studied with variable success and will be reviewed herein. Except for the minority of patients with targetable driver mutations, HMAs – likely as part of combination therapies – will remain the backbone of frontline MDS treatment. However, the wider use of genetic testing may enable a more targeted and individualized therapy of MDS patients.

Delirium Secondary to Lamotrigine Toxicity

2020 ◽  
Vol 15 (2) ◽  
pp. 156-159 ◽  
Author(s):  
Deborah L. Sanchez ◽  
Adam J. Fusick ◽  
Steven R. Gunther ◽  
Michael J. Hernandez ◽  
Gregory A. Sullivan ◽  
...  
Keyword(s):  
Bipolar Disorder ◽  
Valproic Acid ◽  
Mental Status ◽  
The United States ◽  
Clinical Correlation ◽  
Medication Regimen ◽  
Medication Effects ◽  
United States Food ◽  
States Food ◽  
Rule Out

Background: Lamotrigine is a phenyltriazine medication that has been approved by the United States Food and Drug Administration as monotherapy and as an adjunctive agent for the treatment of seizure disorder. It was later approved by the FDA for the treatment of bipolar disorder. Lamotrigine is generally well tolerated by patients, but some serious symptoms can occur during treatment. These severe side effects include rashes and multi-organ failure. Lamotrigine has also been associated with the development of mental status changes, frequently when used concurrently with other medications that may impact the metabolism of lamotrigine. Objective: To present the case of a 65-year-old man being treated with lamotrigine and valproic acid who developed mental status changes after the addition of sertraline to his medication regimen, and to compare this case to existing cases reported in the literature. Discussion: Our case adds to the existing literature by demonstrating that patients may experience adverse medication effects despite lamotrigine levels that are normally considered to be in the therapeutic range, highlighting the importance of clinical correlation when obtaining medication levels. Conclusion: Clinicians should use caution interpreting lamotrigine levels when working up delirium, as normal levels may not rule out the development of lamotrigine toxicity.

scholarly journals D-Penicillamine: The State of the Art in Humans and in Dogs from a Pharmacological and Regulatory Perspective

Antibiotics ◽  
2021 ◽  
Vol 10 (6) ◽  
pp. 648
Author(s):  
Michela Pugliese ◽  
Vito Biondi ◽  
Enrico Gugliandolo ◽  
Patrizia Licata ◽  
Alessio Filippo Peritore ◽  
...  
Keyword(s):  
Veterinary Medicine ◽  
State Of The Art ◽  
European Medicines Agency ◽  
Therapeutic Goals ◽  
First Choice ◽  
Regulatory Perspective ◽  
The World ◽  
United States Food ◽  
States Food ◽  
Food And Drugs Administration

Chelant agents are the mainstay of treatment in copper-associated hepatitis in humans, where D-penicillamine is the chelant agent of first choice. In veterinary medicine, the use of D-penicillamine has increased with the recent recognition of copper-associated hepatopathies that occur in several breeds of dogs. Although the different regulatory authorities in the world (United States Food and Drugs Administration—U.S. FDA, European Medicines Agency—EMEA, etc.) do not approve D-penicillamine for use in dogs, it has been used to treat copper-associated hepatitis in dogs since the 1970s, and is prescribed legally by veterinarians as an extra-label drug to treat this disease and alleviate suffering. The present study aims to: (a) address the pharmacological features; (b) outline the clinical scenario underlying the increased interest in D-penicillamine by overviewing the evolution of its main therapeutic goals in humans and dogs; and finally, (c) provide a discussion on its use and prescription in veterinary medicine from a regulatory perspective.

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