Review
Hematology
Keiji Nogami, Midori Shima
Summary: Non-factor products have been developed to treat patients with hemophilia by facilitating the coagulation pathway or blocking the anticoagulant pathway. Emicizumab, one of these products, has been globally approved and can alter the phenotype of severe hemophilia patients. However, there is a risk of thrombotic events in patients receiving these non-factor products, and monitoring of their hemostatic function is necessary.
LANCET HAEMATOLOGY
(2022)
Review
Endocrinology & Metabolism
Bernt Johan von Scholten, Frederik F. Kreiner, Stephen C. L. Gough, Matthias von Herrath
Summary: Insulin remains the key treatment for type 1 diabetes, but efforts are now shifting towards protecting beta cells to prevent dysfunction and destruction, with research focusing on antigen vaccination and agents that can reduce stress on the beta cells.
Review
Medicine, General & Internal
Jakub Wlodarczyk, Anna Wasniewska, Jakub Fichna, Adam Dziki, Lukasz Dziki, Marcin Wlodarczyk
Summary: Constipation is a major gastrointestinal disorder in Western countries, affecting almost 20% of the population. The pathophysiology is complex and multifactorial, making diagnosis and treatment challenging. Both nonpharmacological and pharmacological approaches are used, with surgery being a potential solution for refractory cases.
JOURNAL OF CLINICAL MEDICINE
(2021)
Review
Oncology
Aine Sally, Ryan McGowan, Karen Finn, Brian Michael Moran
Summary: Pancreatic cancer is a major cause of cancer-related death worldwide, primarily due to delayed diagnosis and resistance to traditional chemotherapy. Delayed diagnosis is often caused by the wide range of non-specific symptoms associated with the disease. Resistance to current chemotherapies, such as gemcitabine, develops due to genetic mutations that are either intrinsic or acquired. This has resulted in poor patient prognosis and justifies the requirement for new targeted therapies. Synthetic lethality approaches targeting specific loss-of-function mutations have shown great potential in pancreatic cancer treatment. Immunotherapies have also yielded promising results and are currently undergoing clinical trials. Monoclonal antibodies, immune checkpoint inhibitors, adoptive cell transfer, and vaccines have shown success in other cancers and could hold the same potential in pancreatic cancer treatment. This review focuses on currently approved therapies, challenges, and future directions in pancreatic cancer therapy, including synthetic lethality approaches, immunotherapy, and clinical trials.
Review
Oncology
Liang Rong, Ni Li, Zhenzhen Zhang
Summary: Glioblastoma (GBM) is a common brain tumor with poor prognosis. Immunotherapy, including immune checkpoint blockade, CAR T cell therapy, oncolytic virotherapy, and vaccine therapy, has shown promising results in improving GBM outcomes. Techniques to overcome the blood-brain barrier for targeted delivery are also being tested. This article reviews the rationales for these therapies, potential novel agents, current status of trials, and discusses challenges and future perspectives in glioblastoma immuno-oncology.
JOURNAL OF EXPERIMENTAL & CLINICAL CANCER RESEARCH
(2022)
Review
Hematology
Afaf E. G. Osman, Michael W. Deininger
Summary: CML is a myeloproliferative neoplasm caused by a specific gene fusion, with the majority of patients benefiting from BCR/ABL1 tyrosine kinase inhibitors, leading to long-term remission. However, a small percentage of patients develop resistance and may require hematopoietic stem cell transplantation for cure. Research focuses on overcoming TKI resistance, improving outcomes in BP-CML, and enhancing rates of TFR in CML.
Review
Pharmacology & Pharmacy
Martin K. R. Svacina, Helmar C. Lehmann
Summary: This review summarizes established therapies for chronic inflammatory demyelinating polyneuropathy (CIDP) and provides an outlook on future therapeutic approaches. Despite the availability of various treatment options, CIDP remains challenging to treat.
CURRENT PHARMACEUTICAL DESIGN
(2022)
Review
Medicine, General & Internal
Ki Wan Park, Peter Kullar, Charvi Malhotra, Konstantina M. Stankovic
Summary: Chronic subjective tinnitus is a condition where individuals perceive sound without an external source for longer than six months. Current treatments are not very effective, and there is a high demand for new therapies for tinnitus.
JOURNAL OF CLINICAL MEDICINE
(2023)
Article
Pharmacology & Pharmacy
Arathi Kulkarni, Shuchi Patel, Deepesh Khanna, Mayur S. Parmar
Summary: Celiac Disease (CeD) is an autoimmune disorder caused by the consumption of gluten, leading to inflammation in the small intestine. The current treatment recommendation is to avoid gluten-based products, as there is no FDA-approved medication for CeD. However, several clinical trials are underway to develop pharmacological approaches to treat CeD.
EUROPEAN JOURNAL OF PHARMACOLOGY
(2021)
Review
Rheumatology
Raphaele Seror, Gaetane Nocturne, Xavier Mariette
Summary: Primary Sjogren syndrome is a systemic autoimmune disease characterized by dryness, pain, and fatigue. Despite the lack of effective treatments, progress has been made in epidemiology, classification criteria, and understanding the pathogenic mechanisms. Therapeutic decisions are mostly based on experience, but the 2019 management recommendations from EULAR are now influencing clinical management.
NATURE REVIEWS RHEUMATOLOGY
(2021)
Review
Medicine, General & Internal
Richa Rathore, Brian A. Van Tine
Summary: Osteosarcoma is the most common primary malignant bone tumor in children and young adults, with a standard treatment that has remained unchanged for over 40 years, but personalized therapies are needed based on unique genetics and biology. Understanding the role of normal bone biology in osteosarcomagenesis, describing the pathology and current standard of care, and exploring novel therapeutics for osteosarcoma due to its complex heterogeneity.
JOURNAL OF CLINICAL MEDICINE
(2021)
Review
Oncology
Alessandra Maleddu, Jessica Zhu, Michael Roy Clay, Breelyn Ann Wilky
Summary: Locally aggressive mesenchymal tumors are a diverse group of tumors that affect soft tissue and bone. While they have limited ability to spread and a good prognosis, these tumors can still cause symptoms and require extensive treatments such as surgery and chemotherapy. The management of these tumors has evolved over time, with a focus on minimizing aggressive treatments and considering individual patient factors. This review aims to discuss three representative locally aggressive mesenchymal tumors and their biology, clinical management, and future treatment options.
FRONTIERS IN ONCOLOGY
(2023)
Review
Clinical Neurology
Monica Margoni, Paolo Preziosa, Massimo Filippi, Maria A. Rocca
Summary: Multiple sclerosis is a chronic disease affecting the central nervous system, and recent advancements in immune pathophysiology have led to the identification of selective B-cell-depleting therapies like anti-CD20 monoclonal antibodies, which have shown strong efficacy and safety in treating MS. This has expanded the therapeutic options for both relapsing and progressive MS patients, highlighting the important role of B cells in the disease process.
JOURNAL OF NEUROLOGY
(2022)
Review
Oncology
Sujit S. Nair, Dimple Chakravarty, Vaibhav Patel, Nina Bhardwaj, Ashutosh K. Tewari
Summary: Neoadjuvant therapies are important for reducing tumor volume, facilitating surgery, and assessing treatment response. They have the potential to improve survival in patients with localized or locally advanced genitourinary cancer. The use of biomarkers and comprehensive studies will advance precision neoadjuvant therapies.
Review
Clinical Neurology
Debopam Samanta
Summary: Seizure emergencies require rapid evaluation and treatment, with intravenous benzodiazepines being the first-line therapy. New options like intranasal midazolam and diazepam nose spray have been approved by the FDA, while ongoing research is exploring alternative non-intravenous treatment options. Challenges remain in utilizing rescue medicines in community settings, prompting the need for cost-effective analysis and further development of non-benzodiazepine drugs for seizure emergencies.
NEUROLOGICAL SCIENCES
(2021)
Letter
Gastroenterology & Hepatology
Lukas Michaja Balsiger, Karen Van den Houte, Tenghao Zheng, Joran Toth, Linde Besard, Andre Franke, Mauro D'Amato, Jan Tack, Florencia Carbone
Article
Gastroenterology & Hepatology
I-Hsuan Huang, Jolien Schol, Florencia Carbone, Yaozhu J. Chen, Karen Van den Houte, Lukas Michaja Balsiger, Bert Broeders, Tim Vanuytsel, Jan Tack
Summary: This study investigated the symptom patterns in distinguishing gastroparesis from functional dyspepsia. The results showed that patients with gastroparesis-like symptoms had a higher prevalence of delayed gastric emptying and higher symptom severity compared to patients with FD symptoms.
ALIMENTARY PHARMACOLOGY & THERAPEUTICS
(2023)
Article
Gastroenterology & Hepatology
Chris Varghese, Gabriel Schamberg, Stefan Calder, Stephen Waite, Daniel Carson, Daphne Foong, William Jiaen Wang, Vincent Ho, Jonathan Woodhead, Charlotte Daker, William Xu, Peng Du, Thomas L. Abell, Henry P. Parkman, Jan Tack, Christopher N. Andrews, Gregory O'Grady, Armen A. Gharibans
Summary: This study aimed to define normative reference intervals for BSGM spectral metrics in healthy controls. The results showed the median and reference intervals for BSGM metrics, including the Principal Gastric Frequency, Gastric Alimetry Rhythm Index, BMI-adjusted amplitude, and fed:fasted amplitude ratio. This study provides reference values for diagnostic interpretations of abnormal gastric function using BSGM spectral data.
AMERICAN JOURNAL OF GASTROENTEROLOGY
(2023)
Article
Gastroenterology & Hepatology
Lin Chang, Brooks D. D. Cash, Anthony Lembo, David C. C. Kunkel, Brett A. A. English, Beatriz Lindstrom, Guibao Gu, Sharon Skare, Kye Gilder, Stewart Turner, Fabio Cataldi, Donald Lipkis, Tack Jan
Summary: This study assessed the efficacy and safety of olorinab to treat abdominal pain in patients with irritable bowel syndrome. Although no significant difference was found in pain relief, olorinab significantly improved symptoms in patients with moderate-to-severe pain.
NEUROGASTROENTEROLOGY AND MOTILITY
(2023)
Article
Biochemistry & Molecular Biology
Hideki Mori, Wout Verbeure, Rina Tanemoto, Emily Ruilova Sosoranga, Jan Tack
Summary: Motilin, secreted by the duodenum, regulates the migrating motor complex during fasting and affects various gastrointestinal functions. It stimulates gallbladder contraction, pepsin secretion, and pancreatic juice and insulin secretion. Motilin is also associated with appetite and potential treatments for obesity. Gastric acid and bile are the main regulators of motilin release. Different caloric foods have diverse effects on motilin levels, and non-caloric bitter substances can suppress appetite. Motilin receptor agonists and antagonists have been developed but are not yet clinically utilized.
Article
Gastroenterology & Hepatology
Nick Goelen, Mike Jones, I-Hsuan Huang, Florenca Carbone, Pieter Janssen, Jan Tack
Summary: Gastroparesis and functional dyspepsia are disorders characterized by upper gastrointestinal symptoms and multifaceted etiologies. The association between symptom improvement and acceleration of gastric emptying (GE) by prokinetic agents is not clear. This meta-analysis aimed to provide a transparent update on the association between symptom improvement and GE acceleration in patients with gastroparesis.
UNITED EUROPEAN GASTROENTEROLOGY JOURNAL
(2023)
Article
Gastroenterology & Hepatology
Esther Colomier, Joost P. Algera, Karen Van den Houte, Magnus Simren, Jan Tack
Summary: A subgroup of patients with a disorder of gut-brain interaction (DGBI) experience various symptoms after eating, such as abdominal pain, gas-related symptoms, dyspeptic symptoms, and loose stool or urgency. Previous studies have examined the effects of dietary therapies, such as fiber-rich or restrictive diets, on patients with irritable bowel syndrome and other related disorders, but there is limited research on the underlying mechanisms of food-related symptoms. This review focuses on these potential mechanisms, including nutrient sensing and tasting, physical considerations, malabsorption, allergy-like reactions, and their interaction with the gut microbiota.
BEST PRACTICE & RESEARCH CLINICAL GASTROENTEROLOGY
(2023)
Review
Gastroenterology & Hepatology
Lukas Michaja Balsiger, Florencia Carbone, Karlien Raymenants, Emidio Scarpellini, Jan Tack
Summary: Disorders of gut-brain interaction (DGBI) are common in clinical practice, and there are established recommendations for diagnosis and management. Patients with multiple DGBI diagnoses have more severe symptoms and impact, and the management approach for overlapping diagnoses is not well established. This Review aims to guide clinicians in understanding and managing overlapping DGBI through identifying causes, pitfalls, and practical approaches to diagnosis, treatment, and follow-up.
LANCET GASTROENTEROLOGY & HEPATOLOGY
(2023)
Review
Gastroenterology & Hepatology
Jan Tack, Katayoun Derakhchan, Andre Gabriel, William Spalding, Brian Terreri, Ashraf Youssef, Bahij Kreidieh, Peter R. Kowey, Mena Boules
Summary: This review summarizes the cardiovascular safety data of prucalopride, a high-affinity 5-HT4 agonist, in the treatment of chronic idiopathic constipation (CIC), and concludes that prucalopride does not have significant effects on cardiovascular safety.
AMERICAN JOURNAL OF GASTROENTEROLOGY
(2023)
Review
Pharmacology & Pharmacy
Emidio Scarpellini, Bert Broeders, Jolien Schol, Pierangelo Santori, Mariachiara Addarii, Luigi Boccuto, Florencia Carbone, Ludovico Abenavoli, Jan Tack
Summary: Peppermint oil is a safe herbal medicine therapy with promising scientific perspectives and rapidly expanding use in clinical practice in the field of gastroenterology. It can improve endoscopic performance and treat functional dyspepsia and irritable bowel syndrome.
CURRENT PHARMACEUTICAL DESIGN
(2023)
Article
Gastroenterology & Hepatology
Johann P. Hreinsson, Hans Tornblom, Jan Tack, Douglas A. Drossman, William E. Whitehead, Shrikant I. Bangdiwala, Ami D. Sperber, Olafur S. Palsson, Magnus Simren
Summary: This study evaluated the validity of the Rome IV criteria for gut-brain interaction disorders through factor analysis in global, geographical, gender, and age-group dimensions. The results indicate that the Rome IV criteria are globally valid and represent universal diagnostic entities for these disorders.
Article
Gastroenterology & Hepatology
Caroline M. G. Saleh, Pietro Familiari, Barbara A. J. Bastiaansen, Paul Fockens, Jan Tack, Guy Boeckxstaens, Raf Bisschops, Aaltje Lei, Marlies P. Schijven, Jan Guido Costamagna, Albert J. Bredenoord
Summary: This study compares the efficacy of POEM and PD for patients with persistent or recurrent symptoms after LHM. The results show that POEM has a higher success rate than PD, with a higher incidence of reflux esophagitis in the POEM group.
Article
Gastroenterology & Hepatology
Navkiran Thind Tornkvist, Magnus Simren, Johann P. Hreinsson, Jan Tack, Shrikant I. Bangdiwala, Ami D. Sperber, Olafur S. Palsson, Axel Josefsson, Hans Tornblom
Summary: This study aimed to define the prevalence and impact of disorders of gut-brain interaction (DGBI) in Sweden. The results showed that the prevalence of DGBI in Sweden is similar to global data, and patients often experience anxiety and/or depression, reduced quality of life, and frequent healthcare utilization. Prescription medication can provide relief for GI symptoms.
NEUROGASTROENTEROLOGY AND MOTILITY
(2023)
Article
Pharmacology & Pharmacy
Tom de Waal, Joachim Brouwers, Philippe Berben, Talia Flanagan, Jan Tack, Wim Vandenberghe, Tim Vanuytsel, Patrick Augustijns
Summary: Parkinson's disease may not only impact the motor system but also the physiology of the gastrointestinal tract. The composition of intestinal fluids in PD patients is generally similar to healthy controls, with only minor differences. These differences may be attributed to slower gastric emptying and altered intestinal bacterial metabolism.
Editorial Material
Gastroenterology & Hepatology
Michael Camilleri, Jan Tack