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Haemopoietic Stem Cell Transplantation for Rheumatoid Arthritis—World Experience and Future Trials
Published in Richard K. Burt, Alberto M. Marmont, Stem Cell Therapy for Autoimmune Disease, 2019
John A. Snowden, John J. Moore, Sarah J. Bingham, Steve Z. Pavletic, Richard K. Burt
The study continued as an Australian multicentre trial in which patients were randomised to either unmanipulated or CD34+ selected grafts.38 The trial recruited 33 patients who had failed therapy with methotrexate and at least one other disease modifying agent. Patients received high dose immunosuppression with cyclophosphamide 200 mg/kg followed by randomised rescue with unmanipulated or CD34 selected cells. Thirty-one patients proceeded to transplant. There were no deaths and no major unexpected toxicities. On an intention to treat basis (including 2 patients who failed to mobilise stem cells), ACR 20, 50, and 70 responses were achieved in 70%, 45% and 39% of patients. There were no significant differences between the unmanipulated and the CD34 selected groups in terms of response and time to relapse and re-introduction of disease modifying therapy. Although not part of the trial, a minority of patients were observed closely after re-introduction of disease modifying therapy. Responses, some as profound as ACR 70, were observed in two thirds of the patients followed up, supporting the hypothesis that the transplant procedure provides disease debulking such that subsequent control is possible with low dose agents.
Do personality traits impact upon midwives’ decision-making and practice?
Published in Elaine Jefford, Julie Jomeen, Empowering Decision-Making in Midwifery, 2019
Steve Provost, Anna Smyth, Thejal Rupnarain, Shahna Mailey, Harvey Ward, Elaine Jefford
Risk sensitivity has been shown to be relevant to ‘patient’ decision-making within disciplines such as neurology and pharmacology. For example, when multiple sclerosis patients were asked whether or not they would take a new disease-modifying therapy depending upon the likely probability of benefit and risk of side effects, those who were most risk-averse also exhibited poorer treatment adherence (Bruce et al., 2016). In another study, Tompkins et al. (2018) found chronic pain sufferers who were most at risk of opioid abuse discounted the addiction risk of a hypothetical new pain relief drug less steeply than those at lower risk. In Chapter 5, risk within midwifery has been discussed, yet whether risk sensitivity of practitioners might impact on their decision-making and practice is yet to be determined in any discipline, including midwifery.
Answers
Published in Calver Pang, Ibraz Hussain, John Mayberry, Pre-Clinical Medicine, 2017
Calver Pang, Ibraz Hussain, John Mayberry
This question focuses on immunosuppression and disease modifying therapy in rheumatoid arthritis. Examples of disease modifying anti-rheumatic drugs include methotrexate, sulfasalazine, anti-TNF agents and rituximab. Immunosuppressants include corticosteroids, azathioprine, ciclosporin, tacrolimus and mycophenolate mofetil. Ciclosporin and tacrolimus are known as calcineurin inhibitors and are active against helper T cells preventing the production of IL-2 via calcineurin inhibition. Some adverse effects include nephrotoxicity, hypertension, hyperlipidaemia, gingival hyperplasia and hyperuricemia. Methotrexate is the gold-standard treatment for rheumatoid arthritis. This drug works by competitively and reversibly inhibiting dihydrofolate reductase therefore inhibiting DNA, RNA and protein synthesis.
Impact of high-intensity concurrent training on cardiovascular risk factors in persons with multiple sclerosis – pilot study
Published in Disability and Rehabilitation, 2019
Charly Keytsman, Dominique Hansen, Inez Wens, Bert O. Eijnde
Following local advertisement and written informed consent, 16 persons with MS (mean Expanded Disability Status Scale; EDSS 2.6 ± 0.2) were included. Subjects were excluded if they were pregnant, aged <18 years, participated in another study, experienced an acute exacerbation 6 months prior to the start of the study, had contraindications to perform physical exercise or had an EDSS score >6. Use of disease-modifying therapy and other medication intake was inventoried. Subjects were asked to maintain their usual medication intake constant throughout the study course. All data were collected at the Rehabilitation Research Centre of Hasselt University. The study was approved by the local Ethical Committee of the Jessa hospital and Hasselt University and was performed in accordance with the Declaration of Helsinki of 1975. This study was registered at ClinicalTrials.gov (NCT02466165).
Investigational new drugs for the treatment of Dravet syndrome: an update
Published in Expert Opinion on Investigational Drugs, 2023
Slobodan M. Janković, Snežana V. Janković, Radiša Vojinović, Snežana Lukić
Considering the dynamics of research into both disease-modifying and symptomatic therapy, great progress can be expected in both areas. In the next 5–10 years, treatment of Dravet syndrome with antisense oligonucleotides will most likely become a standard procedure that can be performed on every patient with confirmed haploinsufficiency of the SCN1A gene, while increasingly effective anticonvulsant therapy will continue to be needed to treat residual symptomatology. Other forms of disease-modifying therapy will take longer until their effectiveness and safety are established. Also, our knowledge about possible side effects of all current investigational drugs will be much more complete, and will enable us to respond in a timely manner to any threat.
Prediction in treatment outcomes in multiple sclerosis: challenges and recent advances
Published in Expert Review of Clinical Immunology, 2021
Deja R. Rose, Moein Amin, Daniel Ontaneda
Multiple Sclerosis (MS) is a chronic demyelinating disease of the central nervous system (CNS) characterized by inflammation and neurodegeneration. It is one of the most common causes of neurologic disability in young adults, with significant burden on individuals and the healthcare system [1]. People with MS (pwMS) have a higher prevalence of chronic medical conditions (psychiatric, vascular and chronic lung diseases) and up to 50% lose employment within 5 years of diagnosis [2,3]. Lifetime direct medical costs may reach up to 4.8 billion United States dollars (USD) and include a high price for disease modifying therapy (DMT), often exceeding 90,000 USD per year [4]table 1.