Editorial Material
Rheumatology
Michael Hughes, Christopher P. Denton, Dinesh Khanna
Summary: This editorial discusses the study by Goswami et al., which suggests that rituximab may have therapeutic effects on systemic sclerosis-related interstitial lung disease.
Article
Gastroenterology & Hepatology
Sumona Bhattacharya, Gianna Stoleru, Preet Patel, Ameer Abutaleb, Kristen Stashek, Raymond K. Cross
Summary: Researchers conducted a chart review to identify cases of mycophenolate mofetil gastrointestinal toxicity at their institution. The study found that friability was associated with severe disease, while nausea and erythema were associated with poor prognosis.
INFLAMMATORY BOWEL DISEASES
(2022)
Article
Medicine, General & Internal
Anne Pankow, Sena Sinno, Thorsten Derlin, Marcus Hiss, Annette D. Wagner
Summary: This study retrospectively evaluated the efficacy of MMF in inducing remission in GCA. Results showed that MMF treatment effectively controlled disease activity in all patients and achieved disease remission.
FRONTIERS IN MEDICINE
(2023)
Review
Medicine, General & Internal
Anji Xiong, Chen Xiong, Guancui Yang, Yu Shuai, Deng Liu, Linqian He, Zepeng Guo, Liangwen Zhang, Yi Liu, Yuan Yang, Beibei Cui, Shiquan Shuai
Summary: The study evaluated the efficacy of MMF as remission induction therapy in AAV, suggesting that MMF may be an alternative to CYC for AAV patients with MPO-ANCA positivity, mild to moderate renal involvement, and non-life-threatening state. Further studies are needed to observe MMF's effects in AAV and compare the differences between MMF and CYC, with risk stratification and subgrouping of AAV patients as essential considerations.
FRONTIERS IN MEDICINE
(2021)
Article
Clinical Neurology
Raffaella Pizzolato Umeton, Michael Waltz, Gregory S. Aaen, Leslie Benson, Mark Gorman, Manu Goyal, Jennifer S. Graves, Yolanda Harris, Lauren Krupp, Timothy E. Lotze, Nikita M. Shukla, Soe Mar, Jayne Ness, Mary Rensel, Teri Schreiner, Jan-Mendelt Tillema, Shelly Roalstad, Moses Rodriguez, John Rose, Emmanuelle Waubant, Bianca Weinstock-Guttman, Charles Casper, Tanuja Chitnis
Summary: This retrospective cohort study evaluated cases of children with NMOSD and found that the use of disease-modifying treatments, particularly rituximab, is associated with a lowered annualized relapse rate in children with NMOSD AQP4+.
Review
Transplantation
Alvise Berti, Mouaz Alsawas, Tabinda Jawaid, Larry J. Prokop, Jiwon M. Lee, Gwang Hun Jeong, Luis F. Quintana, Sergey Moiseev, August Vaglio, Vladimir Tesar, Duvuru Geetha, Jae Il Shin, Andreas Kronbichler
Summary: There are uncertainties about the use of mycophenolate mofetil in anti-neutrophil cytoplasmatic antibody-associated vasculitis. This study found that mycophenolate mofetil was significantly associated with higher sustained remission rates in trials enrolling only patients with kidney involvement, which may influence clinical practice.
NEPHROLOGY DIALYSIS TRANSPLANTATION
(2022)
Review
Medicine, General & Internal
Athanasios Vassilopoulos, Stephanos Vassilopoulos, Markos Kalligeros, Fadi Shehadeh, Eleftherios Mylonakis
Summary: This study assessed the incidence of serious infections and infection-related deaths in patients with ANCA-associated vasculitis (AAV) treated with rituximab and azathioprine. The overall cumulative incidence of serious infections was 15.99%, with 14.61% in the rituximab group and 5.93% in the azathioprine group during maintenance. Fatal infections during maintenance treatment were uncommon. Further clinical practice and long-term follow up are needed to verify these findings.
FRONTIERS IN MEDICINE
(2023)
Article
Immunology
Toshihiro Kitajima, Matthew Henry, Tommy Ivanics, Sirisha Yeddula, Kelly Collins, Michael Rizzari, Atsushi Yoshida, Marwan S. Abouljoud, Shunji Nagai, Dilip Moonka
Summary: Recipient age minus donor age greater than 20 years remains a significant risk factor for fatal GVHD. Basiliximab induction significantly increases the risk of fatal GVHD, while maintenance therapy with mycophenolate mofetil decreases the risk of fatal GVHD. These associations were more pronounced in patients with recipient minus donor age greater than 20 years, highlighting the importance of donor age and individualized immunosuppression regimens in the risk of fatal GVHD.
Article
Gastroenterology & Hepatology
Pierluigi Toniutto, Edmondo Falleti, Sara Cmet, Annarosa Cussigh, Laura Veneto, Davide Bitetto, Ezio Fornasiere, Elisa Fumolo, Carlo Fabris, Assunta Sartor, Roberto Peressutti, Francesco Curcio, Laura Regattin, Lucrezia Grillone
Summary: This study explores the long-term antibody response and failure mechanism of COVID-19 vaccines in liver transplant recipients, revealing significantly lower antibody titers after vaccination compared to controls and identifying MMF as a major predictor of vaccination failure.
JOURNAL OF HEPATOLOGY
(2022)
Article
Medicine, General & Internal
Saki Tanaka, Takeshi Kawaguchi, Risa Kudo, Masatoshi Kimura, Yuki Rikitake, Chihiro Iwao, Mao Rikitake, Kosho Iwao, Ayako Aizawa, Yumi Kariya, Motohiro Matsuda, Shunichi Miyauchi, Ichiro Takajo, Kunihiko Umekita
Summary: Neuropsychiatric systemic lupus erythematosus (NPSLE) with cerebral vasculitis is a rare and unfavorable condition. High-dose glucocorticoids and cyclophosphamide are commonly used treatments, but the latter carries risks of reproductive toxicity. This case study suggests that mycophenolate mofetil (MMF) may be a successful treatment option without concerns for reproductive toxicity. Further research is needed to evaluate the efficacy of MMF in NPSLE.
Article
Ophthalmology
Amjad Ameen Saifaldein, Abdulrahman F. AlBloushi, Sulaiman M. Altariqi, Salman Aljarallah, Ahmed M. Abu El-Asrar
Summary: This study investigated the occurrence, clinical findings, and outcomes of occlusive retinal vasculitis in patients with multiple sclerosis (MS) and uveitis. The results showed that early recognition and prompt treatment can prevent complications and improve outcomes in patients with occlusive retinal vasculitis.
OCULAR IMMUNOLOGY AND INFLAMMATION
(2023)
Article
Clinical Neurology
Fredrik Piehl, Ann Eriksson-Dufva, Anna Budzianowska, Amalia Feresiadou, William Hansson, Max Albert Hietala, Irene Hakansson, Rune Johansson, Daniel Jons, Ivan Kmezic, Christopher Lindberg, Jonas Lindh, Fredrik Lundin, Ingela Nygren, Anna Rostedt Punga, Rayomand Press, Kristin Samuelsson, Peter Sundstrom, Oskar Wickberg, Susanna Brauner, Thomas Frisell
Summary: This study investigated the efficacy and safety of rituximab compared to placebo in the treatment of MG. The results showed that a single dose of 500 mg of rituximab was associated with a greater reduction in symptoms and decreased use of rescue medications compared to placebo.
Review
Medicine, General & Internal
Stefano Turolo, Alberto Edefonti, Marie Luise Syren, Giovanni Montini
Summary: Kidney transplantation is the preferred treatment for end-stage kidney disease. However, allograft rejection remains a challenge. Personalized medicine and precision medicine offer great potential in improving drug efficacy and minimizing toxicity for each patient. This review focuses on the pharmacogenomics of immunosuppressive drugs used in kidney transplantation, specifically the genes involved in their metabolism.
JOURNAL OF CLINICAL MEDICINE
(2023)
Review
Dermatology
Matthew L. Hrin, Arjun M. Bashyam, William W. Huang, Steven R. Feldman
Summary: Pyoderma gangrenosum, a rare skin disease commonly treated with systemic corticosteroids, may benefit from adjunctive therapy with mycophenolate mofetil due to its efficacy and tolerability in improving or maintaining disease clearance.
INTERNATIONAL JOURNAL OF DERMATOLOGY
(2021)
Review
Biotechnology & Applied Microbiology
Wenwen Feng, Yifang Hu, Chengzhou Zhang, He Shi, Peng Zhang, Yanli Yang, Shiying Chen, Weijiang Cui, Dai Cui
Summary: This study evaluated the benefit and safety of mycophenolate mofetil (MMF) in the treatment of Graves' orbitopathy (GO) through a meta-analysis of clinical control trials. The results showed that MMF, either alone or in combination with glucocorticoid (GC), was superior to GC alone in treating GO. MMF monotherapy was more effective than GC, and a combination of MMF and methylprednisolone (MP) was more effective than MP monotherapy. MMF treatment in GO showed promising results and was safer compared to GC therapy.
Review
Clinical Neurology
Martin Kirkpatrick, Finbar O'Callaghan
Summary: Despite changes in UK law allowing for the legal prescription of cannabis-based medicinal products (CBMPs), almost no children have received unlicensed CBMPs under the UK NHS. This review explores the science behind CBMPs in pediatric epilepsies and highlights areas for future research.
DEVELOPMENTAL MEDICINE AND CHILD NEUROLOGY
(2022)
Editorial Material
Clinical Neurology
Rob Forsyth
DEVELOPMENTAL MEDICINE AND CHILD NEUROLOGY
(2022)
Article
Clinical Neurology
Rob Forsyth, Colin Hamilton, Matthew Ingram, Gemma Kelly, Tim Grove, Lorna Wales, Mark S. Gilthorpe
Summary: The study found a complex relationship between rehabilitation dose and outcomes after pediatric acquired brain injury. Content-aware measures of rehabilitation dose showed a significant positive correlation with rehabilitation treatment outcomes. These findings contribute to advancing comparative effectiveness research in pediatric neurorehabilitation.
DEVELOPMENTAL NEUROREHABILITATION
(2022)
Article
Rehabilitation
Adrian M. Svingos, Stacy J. Suskauer, Beth S. Slomine, Hsuan Wei Chen, Michael E. Ellis-Stockley, Rob J. Forsyth
Summary: This study aimed to determine the distribution of items on the Cognitive and Linguistic Scale (CALS) and explore the relationship between Rasch-derived Cognitive Ability Estimates and outcome trajectory parameters. The study concluded that the Cognitive Ability Estimates derived from the CALS may serve as an ideal outcome measure for evaluating interventions in pediatric rehabilitation settings for acquired brain injury.
ARCHIVES OF PHYSICAL MEDICINE AND REHABILITATION
(2022)
Article
Clinical Neurology
Christopher Hatton, Simona S. Ghanem, David J. Koss, Ilham Y. Abdi, Elizabeth Gibbons, Rita Guerreiro, Jose Bras, Lauren Walker, Ellen Gelpi, Wendy Heywood, Tiago F. Outeiro, Johannes Attems, Robert McFarland, Rob Forsyth, Omar M. El-Agnaf, Daniel Erskine
Summary: This study identified prion-like alpha-synuclein in the brain tissue of infants with Krabbe disease, challenging the assumption that alpha-synuclein pathology is solely age-associated and suggesting a link to biological pathways such as sphingolipid metabolism.
Article
Rheumatology
Eve M. D. Smith, Kukatharmini Tharmaratnam, Eslam Al-Abadi, Kate Armon, Kathryn Bailey, Mary Brennan, Coziana Ciurtin, Janet Gardner-Medwin, Kirsty E. Haslam, Daniel Hawley, Alice Leahy, Valentina Leone, Gulshan Malik, Zoe McLaren, Clarissa Pilkington, Athimalaipet Ramanan, Satyapal Rangaraj, Annie Ratcliffe, Philip Riley, Ethan Sen, Arani Sridhar, Nick Wilkinson, Christian M. Hedrich, Andrea Jorgensen, Michael W. Beresford
Summary: This study demonstrates that adult-SLE-derived definitions of low disease activity (LDA) or remission are achievable in childhood-onset SLE (cSLE), significantly reducing the risk of severe flare or new damage. Among the LDA definitions, LLDAS performs best, leading to a statistically comparable reduction in the risk of severe flare as attainment of clinical remission.
Article
Immunology
Eve M. D. Smith, Naomi Egbivwie, Andrea L. Jorgensen, Coziana Ciurtin, Eslam Al-Abadi, Kate Armon, Kathryn Bailey, Mary Brennan, Janet Gardner-Medwin, Kirsty Haslam, Daniel P. Hawley, Alice Leahy, Valentina Leone, Gulshan Malik, Zoe McLaren, Clarissa Pilkington, Athimalaipet V. Ramanan, Satyapal Rangaraj, Annie Ratcliffe, Phil Riley, Ethan Sen, Arani Sridhar, Nick Wilkinson, Fiona Wood, Michael W. Beresford, Christian M. Hedrich
Summary: Through analyzing longitudinal data from the UK JSLE Cohort Study, the study found that most patients require the use of two or more immunomodulating drugs for treatment, with mycophenolate mofetil (MMF) being the most commonly used medication.
CLINICAL IMMUNOLOGY
(2022)
Article
Clinical Neurology
Rob J. Forsyth, Liz Roberts, Rob Henderson, Lorna Wales
Summary: This study aims to describe the variation in neurorehabilitation content provided to young people after severe paediatric acquired brain injury (pABI) and relate it to observed functional recovery. The study found significant correlations between aspects of delivered therapy and recovery, suggesting that rehabilitation plays a role in the recovery process. However, further research is needed to understand the causal relationship between rehabilitation and recovery after pABI.
DEVELOPMENTAL MEDICINE AND CHILD NEUROLOGY
(2022)
Editorial Material
Rheumatology
Ethan S. Sen, Dalila Julandani, Athimalaipet Ramanan
Article
Clinical Neurology
John P. Osborne, Stuart W. Edwards, Fabienne Dietrich Alber, Eleanor Hancock, Anthony L. Johnson, Colin R. Kennedy, Marcus Likeman, Andrew L. Lux, Mark Mackay, Andrew Mallick, Richard W. Newton, Melinda Nolan, Ronit Pressler, Dietz Rating, Bernhard Schmitt, Christopher M. Verity, Finbar J. K. O'Callaghan
Summary: This study analyzed data from two randomized controlled trials to compare the efficacy of prednisolone and tetracosactide depot in treating infantile epileptic spasms syndrome (IESS). The results showed no significant difference in the effectiveness of the two treatments, and both were effective in relieving spasms in patients.
EUROPEAN JOURNAL OF PAEDIATRIC NEUROLOGY
(2023)
Review
Pharmacology & Pharmacy
Victoria M. Defelippe, Ghislaine J. M. W. van Thiel, Willem M. Otte, Roger E. G. Schutgens, Bas Stunnenberg, Helen J. Cross, Finbar O'Callaghan, Valentina De Giorgis, Floor E. Jansen, Emilio Perucca, Eva H. Brilstra, Kees P. J. Braun
Summary: N-of-1 strategies provide high-quality evidence for individual treatment efficacy and optimize treatment selection for rare diseases. The debate over whether N-of-1 strategies should be governed by research or care regulations continues.
DRUG DISCOVERY TODAY
(2023)
Article
Clinical Neurology
Thomas B. Atkinson, Rob J. Forsyth
Summary: The effect of age at injury on outcomes after brain injury is debated. Some argue that the immature brain has greater plasticity for recovery, while others argue that early injury impairs future learning abilities. This study found that it is easier to maintain or recover previously established functions than to learn them for the first time in an injured brain, supporting the latter viewpoint.
EUROPEAN JOURNAL OF PAEDIATRIC NEUROLOGY
(2023)
Meeting Abstract
Clinical Neurology
Lorna Wales, Rob Forsyth, Gemma Kelly
JOURNAL OF HEAD TRAUMA REHABILITATION
(2022)
Article
Pediatrics
Jacqueline Y. Thompson, Julie C. Menzies, Joseph C. Manning, Jennifer McAnuff, Emily Clare Brush, Francesca Ryde, Tim Rapley, Nazima Pathan, Stephen Brett, David J. Moore, Michelle Geary, Gillian A. Colville, Kevin P. Morris, Roger Charles Parslow, Richard G. Feltbower, Sophie Lockley, Fenella J. Kirkham, Rob J. Forsyth, Barnaby R. Scholefield
Summary: The survey found that key components of ERM as defined by participants included tailored, multidisciplinary rehabilitation packages focused on promoting recovery. Multidisciplinary involvement in initiating ERM was commonly reported. Over half of respondents favored delivering ERM after achieving physiological stability.
BMJ PAEDIATRICS OPEN
(2022)
Meeting Abstract
Neurosciences
Lorna Wales, Rob Forsyth, Gemma Kelly
