scholarly journals Unmet need in rheumatology: reports from the Targeted Therapies meeting 2018

2019 ◽  
Vol 78 (7) ◽  
pp. 872-878 ◽  
Author(s):  
Kevin L Winthrop ◽  
Michael E Weinblatt ◽  
Mary K Crow ◽  
Gerd R Burmester ◽  
Philip J Mease ◽  
...  
Keyword(s):  
Targeted Therapies ◽  
Lupus Erythematosus ◽  
Unmet Needs ◽  
Axial Spondyloarthritis ◽  
Clinical Care ◽  
Unmet Need ◽  
Connective Tissue Diseases ◽  
Treatment And Prevention ◽  
Therapeutic Development ◽  
Comprehensive Listing

To develop a comprehensive listing of the greatest unmet scientific and clinical needs in rheumatology. The 20th annual international Targeted Therapies meeting brought more than 100 leading basic scientists and clinical researchers in rheumatology, immunology, epidemiology, molecular biology and other specialties. During the meeting, breakout sessions were convened, consisting of five disease-specific groups with 20–30 experts assigned to each group based on expertise. Specific groups included rheumatoid arthritis, psoriatic arthritis, axial spondyloarthritis, systemic lupus erythematosus, connective tissue diseases and a basic science immunology group spanning all of these clinical domains. In each group, experts were asked to consider recent accomplishments within their clinical domain in the last year and update the unmet needs in three categorical areas: basic/translational science, clinical science and therapeutic development, and clinical care. While progress was noted among some of previously identified needs, both new needs were identified and themes from prior meetings were re-iterated: the need for better understanding the heterogeneity within each disease, and for identifying preclinical states of disease allowing treatment and prevention of disease in those at risk, and the elusive ability to cure disease. Within the clinical care realm, improved comorbidity management and patient-centred care continue to be unmet needs, and the need for new and affordable therapeutics was highlighted. Unmet needs for new and accessible targeted therapies, disease prevention and ultimately cure remain a priority in rheumatology.

scholarly journals Unmet need in rheumatology: reports from the Targeted Therapies meeting 2019

2019 ◽  
Vol 79 (1) ◽  
pp. 88-93 ◽  
Author(s):  
Kevin L Winthrop ◽  
Michael E Weinblatt ◽  
Joan Bathon ◽  
Gerd R Burmester ◽  
Philip J Mease ◽  
...  
Keyword(s):  
Targeted Therapies ◽  
Lupus Erythematosus ◽  
Axial Spondyloarthritis ◽  
Inflammatory Diseases ◽  
Unmet Need ◽  
Clinical Trial Design ◽  
Refractory Disease ◽  
Developing Regions ◽  
Disease States ◽  
Clinical And Translational Research

ObjectivesTo detail the greatest areas of unmet scientific and clinical needs in rheumatology.MethodsThe 21st annual international Advances in Targeted Therapies meeting brought together more than 100 leading basic scientists and clinical researchers in rheumatology, immunology, epidemiology, molecular biology and other specialties. During the meeting, breakout sessions were convened, consisting of 5 disease-specific groups with 20–30 experts assigned to each group based on expertise. Specific groups included: rheumatoid arthritis, psoriatic arthritis, axial spondyloarthritis, systemic lupus erythematosus and other systemic autoimmune rheumatic diseases. In each group, experts were asked to identify unmet clinical and translational research needs in general and then to prioritise and detail the most important specific needs within each disease area.ResultsOverarching themes across all disease states included the need to innovate clinical trial design with emphasis on studying patients with refractory disease, the development of trials that take into account disease endotypes and patients with overlapping inflammatory diseases, the need to better understand the prevalence and incidence of inflammatory diseases in developing regions of the world and ultimately to develop therapies that can cure inflammatory autoimmune diseases.ConclusionsUnmet needs for new therapies and trial designs, particularly for those with treatment refractory disease, remain a top priority in rheumatology.

RHEUMATIC DISEASES AND PREGNANCY (LITERATURE REVIEW)

2021 ◽  
Vol 6 (4) ◽  
pp. 11-17
Author(s):  
T. Bondareva
Keyword(s):  
Rheumatoid Arthritis ◽  
Genetic Engineering ◽  
Rheumatic Diseases ◽  
Lupus Erythematosus ◽  
Pregnancy Outcomes ◽  
Axial Spondyloarthritis ◽  
Connective Tissue Diseases ◽  
Systemic Lupus ◽  
Tnf Α ◽  
Inflammatory Drugs

. The problem of pregnancy in rheumatic diseases does not lose its relevance. The most studied is the course of pregnancy in patients with rheumatoid arthritis, systemic lupus erythematosus and axial spondyloarthritis. Observations in other systemic connective tissue diseases are episodic and do not allow us to draw reliable conclusions. The course of the disease itself, as well as the therapy received by patients, can affect pregnancy, starting with the possibility of conception and ending with unfavorable pregnancy outcomes for the mother and fetus. The onset of pregnancy should be planned at the time of remission or minimal activity of the disease. Of particular interest is drug therapy in patients with rheumatic diseases during gestation. This issue has been and remains complex and has not been fully studied due to the fact that any conclusions about the effect of drugs on the fetus are made retrospectively and cannot be the result of a deliberately initiated study. Currently allowed, though with some reservations, during pregnancy drugs are glucocorticoids, non-steroidal anti-inflammatory drugs, hydroxychloroquine and sulfasalazine. The development of genetic engineering has raised the question of the possibility of using genetic engineering basic therapy during gestation. Currently, the largest number of observations is available for TNF-α inhibitors, the use of which is recognized as possible during pregnancy. The presence of rheumatic disease in itself is not an absolute indication for a caesarean section. The question of the method of delivery is decided individually. Pregnant patients with rheumatic diseases should be under regular joint supervision of an obstetrician-gynecologist, rheumatologist and neonatologist

scholarly journals The Main Challenges in Systemic Lupus Erythematosus: Where Do We Stand?

2021 ◽  
Vol 10 (2) ◽  
pp. 243
Author(s):  
Matteo Piga ◽  
Laurent Arnaud
Keyword(s):  
Systemic Lupus Erythematosus ◽  
Disease Activity ◽  
Lupus Erythematosus ◽  
Systemic Disease ◽  
Unmet Need ◽  
Clinical Manifestations ◽  
New Drugs ◽  
Systemic Lupus ◽  
Major Step ◽  
Damage Accrual

Systemic lupus erythematosus (SLE) is an immune-mediated multi-systemic disease characterized by a wide variability of clinical manifestations and a course frequently subject to unpredictable flares. Despite significant advances in the understanding of the pathophysiology and optimization of medical care, patients with SLE still have significant mortality and carry a risk of progressive organ damage accrual and reduced health-related quality of life. New tools allow earlier classification of SLE, whereas tailored early intervention and treatment strategies targeted to clinical remission or low disease activity could offer the opportunity to reduce damage, thus improving long-term outcomes. Nevertheless, the early diagnosis of SLE is still an unmet need for many patients. Further disentangling the SLE susceptibility and complex pathogenesis will allow to identify more accurate biomarkers and implement new ways to measure disease activity. This could represent a major step forward to find new trials modalities for developing new drugs, optimizing the use of currently available therapeutics and minimizing glucocorticoids. Preventing and treating comorbidities in SLE, improving the management of hard-to-treat manifestations including management of SLE during pregnancy are among the remaining major unmet needs. This review provides insights and a research agenda for the main challenges in SLE.

scholarly journals P051 Symptoms of COVID-19 and anxiety levels in adult patients receiving b-and ts-DMARDS using an online reporting system

Rheumatology ◽  
2021 ◽  
Vol 60 (Supplement_1) ◽  
Author(s):  
May Nwe Lwin ◽  
Christopher Holroyd ◽  
Dinny Wallis ◽  
Saul Faust ◽  
Hans De Graaf ◽  
...  
Keyword(s):  
Lupus Erythematosus ◽  
Joint Pain ◽  
Connective Tissue Diseases ◽  
Anxiety Score ◽  
Online Portal ◽  
Inflammatory Conditions ◽  
Advanced Therapy ◽  
Number Of Patients ◽  
Patient Reported ◽  
Anxiety Levels

Abstract Background/Aims  The coronavirus disease-2019 (COVID-19) caused by the severe acute respiratory syndrome coronavirus-2 (SARS-CoV-2) is responsible for over 120,000 UK deaths. Those with chronic inflammatory conditions or receiving immunosuppressive medications are at higher risk of COVID-19 than the general population. As a result, rheumatology patients taking b- or ts-DMARDs were advised to shield. We planned to observe COVID-19 related symptoms and anxiety levels reported by rheumatology patients during the pandemic. Methods  From April 2020, 1,004 rheumatology patients from an advanced therapy database were invited to participate in the adult ImmunoCOVID study to collect daily symptoms (fever, cough, shortness of breath (SOB), sore throat, blocked nose, red-eye, headache, fatigue, joint pain, muscle pain, chills, nausea, diarrhoea and vomiting, loss of senses) and anxiety level using an online portal. Loss of senses were not recorded until week 7 as these were not officially recognized at the pandemic onset. Results  153 patients (rheumatoid arthritis, n = 75, psoriatic arthritis, n = 28, Axial spondyloarthropathy, n = 24, systemic lupus erythematosus, n = 2 and other connective tissue diseases, n = 24) consented and participated. By week 25, 142 patients remained. Among those, 36.57% (±6.09%) (average (±SD)) reported no symptoms over the 25 week period. The main symptoms reported were joint pain (mean=47.94%) followed by fatigue (27.17%). Few patients reported fever (0.94%), cough (8.34%), SOB (4.53%), or loss of senses (1.11%) with more symptoms reported during the first 8 weeks (April/May 2020) and another increase in September/October 2020. The anxiety score (pragmatic 10-point scale) mean (±SD) was 5.60 (±0.34) and remained elevated throughout the study though higher when lockdown began. Conclusion  During the first peak of SARS-CoV-2, the number of patients reporting COVID-19 symptoms appeared high and was associated with high levels of anxiety. As only a small number have been swab-tested, this may suggest that larger numbers of untested individuals have had COVID-19 with mild symptoms. Features of inflammatory rheumatic illnesses may mimic COVID-19 symptoms and create diagnostic difficulty (joint pain and fatigue) whilst anxiety may lead to over-reporting of symptoms in the absence of infection. The key symptoms of fever, cough and SOB were less common and may be most reliable. Disclosure  M. Lwin: None. C. Holroyd: None. D. Wallis: None. S. Faust: None. H. De Graaf: None. C.J. Edwards: Honoraria; Abbvie, Biogen, BMS, Celgene, Fresenius, GSK, Janssen, Lilly, Mundipharma, Pfizer, Roche, Sanofi, UCB. Member of speakers’ bureau; Abbvie, Biogen, BMS, Celgene, Janssen, Lilly, Sanofi, Pfizer, Roche. Grants/research support; Abbvie, Biogen, Pfizer. P051 Table 1:patient reported symptoms and anxiety score from immunoCOVID studyWeek & (number of participants)Fever (%)Cough (%)SOB (%)Joint pain (%)Fatigue (%)Loss of senses (%)No symptoms (%)Tested (n)Test positive (n)Anxiety score1 (26)3.857.6911.5446.1530.77NA30.77006.312 (42)2.3311.639.3052.3834.88NA28.57005.833 (69)1.4514.494.3552.1737.68NA23.19415.884 (92)1.0911.966.5254.3531.52NA27.17206.225 (110)0.0011.716.3145.9533.33NA30.00006.156 (108)0.0010.193.7050.0026.85NA34.26205.747 (119)0.8410.084.2049.5828.57NA34.45205.938 (120)0.007.505.0051.6734.170.8329.17305.629 (124)0.817.263.2352.4229.840.8136.29405.6410 (118)0.008.473.3948.3129.660.8534.75205.2811 (116)0.858.476.7849.1529.661.6933.62305.6512 (131)0.006.114.5856.4926.720.7635.11205.4513 (110)0.916.362.7350.0029.091.8242.73105.4414 (121)0.837.442.4847.1125.620.8339.67805.2815 (100)1.007.003.0046.0023.001.0041.00405.4816 (114)0.887.893.5139.4725.441.7542.98905.2717 (105)0.008.573.8144.7622.860.9543.81425.1018 (107)0.006.543.7443.9319.630.9343.93405.3019 (99)0.005.052.0240.4019.191.0145.45505.0820 (110)0.914.552.7350.9124.550.9139.09NA0Missing data21 (106)0.946.602.8350.0020.750.9439.62405.2822 (104)2.889.626.7349.0430.770.9635.58305.5023 (106)1.897.553.7742.4526.420.9436.79805.8924 (108)0.938.332.7844.4422.220.9341.67605.6125 (94)1.067.454.2641.4915.962.1344.68605.49Average0.948.344.5347.9427.171.1136.575.60SD0.972.312.254.395.350.426.090.34Weekly data are the average of daily reported symptoms and anxiety levels.

scholarly journals Gender differences in patient journey to diagnosis and disease outcomes: results from the European Map of Axial Spondyloarthritis (EMAS)

2021 ◽  
Author(s):  
Marco Garrido-Cumbrera ◽  
◽  
Denis Poddubnyy ◽  
Laure Gossec ◽  
Raj Mahapatra ◽  
...  
Keyword(s):  
Gender Differences ◽  
Psychological Distress ◽  
Disease Activity ◽  
Healthcare Professionals ◽  
Unmet Needs ◽  
Axial Spondyloarthritis ◽  
Diagnostic Delay ◽  
Hla B27 ◽  
Patient Journey ◽  
Disease Characteristics

Abstract Introduction/objectives To evaluate the journey to diagnosis, disease characteristics and burden of disease in male and female patients with axial spondyloarthritis (axSpA) across Europe. Method Data from 2846 unselected patients participating in the European Map of Axial Spondyloarthritis (EMAS) study through an online survey (2017–2018) across 13 countries were analysed. Sociodemographic characteristics, lifestyle, diagnosis, disease characteristics and patient-reported outcomes (PROs) [disease activity –BASDAI (0–10), spinal stiffness (3–12), functional limitations (0–54) and psychological distress (GHQ-12)] were compared between males and females using chi-square (for categorical variables) and student t (for continuous variables) tests. Results In total, 1100 (38.7%) males and 1746 (61.3%) females participated in the EMAS. Compared with males, females reported considerable longer diagnostic delay (6.1 ± 7.4 vs 8.2 ± 8.9 years; p < 0.001), higher number of visits to physiotherapists (34.5% vs 49.5%; p < 0.001) and to osteopaths (13.3% vs 24.4%; p < 0.001) before being diagnosed and lower frequency of HLA-B27 carriership (80.2% vs 66.7%; p < 0.001). In addition, females reported higher degree of disease activity in all BASDAI aspects and greater psychological distress through GHQ-12 (4.4 ± 4.2 vs 5.3 ± 4.1; p < 0.001), as well as a greater use of alternative therapies. Conclusion The patient journey to diagnosis of axSpA is much longer and arduous in females, which may be related to physician bias and lower frequency of HLA-B27 carriership. Regarding PROs, females experience higher disease activity and poorer psychological health compared with males. These results reflect specific unmet needs in females with axSpA needing particular attention. Key Points• Healthcare professionals’ perception of axSpA as a predominantly male disease may introduce some bias during the diagnosis and management of the disease. However, evidence about male-female differences in axSpA is scarce.• EMAS results highlight how female axSpA patients report longer diagnostic delay and higher burden of the disease in a large sample of 2846 participants of 13 European countries.• Results reflect unmet needs of European female patients. Healthcare professionals should pay close attention in order to accurately diagnose and efficiently manage axSpA cases while further research should be developed on the cause of reported gender differences.

scholarly journals Digital Progression Biomarkers as Novel Endpoints in Clinical Trials: A Multistakeholder Perspective

2021 ◽  
pp. 1-7
Author(s):  
Diane Stephenson ◽  
Reham Badawy ◽  
Soania Mathur ◽  
Maria Tome ◽  
Lynn Rochester
Keyword(s):  
Disease Progression ◽  
Digital Health ◽  
Low Cost ◽  
Unmet Need ◽  
Disease Status ◽  
Standard Test ◽  
Imaging Biomarkers ◽  
Health Technologies ◽  
Therapeutic Development ◽  
The Rich

The burden of Parkinson’s disease (PD) continues to grow at an unsustainable pace particularly given that it now represents the fastest growing brain disease. Despite seminal discoveries in genetics and pathogenesis, people living with PD oftentimes wait years to obtain an accurate diagnosis and have no way to know their own prognostic fate once they do learn they have the disease. Currently, there is no objective biomarker to measure the onset, progression, and severity of PD along the disease continuum. Without such tools, the effectiveness of any given treatment, experimental or conventional cannot be measured. Such tools are urgently needed now more than ever given the rich number of new candidate therapies in the pipeline. Over the last decade, millions of dollars have been directed to identify biomarkers to inform progression of PD typically using molecular, fluid or imaging modalities). These efforts have produced novel insights in our understanding of PD including mechanistic targets, disease subtypes and imaging biomarkers. While we have learned a lot along the way, implementation of robust disease progression biomarkers as tools for quantifying changes in disease status or severity remains elusive. Biomarkers have improved health outcomes and led to accelerated drug approvals in key areas of unmet need such as oncology. Quantitative biomarker measures such as HbA1c a standard test for the monitoring of diabetes has impacted patient care and management, both for the healthcare professionals and the patient community. Such advances accelerate opportunities for early intervention including prevention of disease in high-risk individuals. In PD, progression markers are needed at all stages of the disease in order to catalyze drug development—this allows interventions aimed to halt or slow disease progression, very early, but also facilitates symptomatic treatments at moderate stages of the disease. Recently, attention has turned to the role of digital health technologies to complement the traditional modalities as they are relatively low cost, objective and scalable. Success in this endeavor would be transformative for clinical research and therapeutic development. Consequently, significant investment has led to a number of collaborative efforts to identify and validate suitable digital biomarkers of disease progression.

Patients' Supportive Care Needs Beyond the End of Cancer Treatment: A Prospective, Longitudinal Survey

2009 ◽  
Vol 27 (36) ◽  
pp. 6172-6179 ◽  
Author(s):  
Jo Armes ◽  
Maggie Crowe ◽  
Lynne Colbourne ◽  
Helen Morgan ◽  
Trevor Murrells ◽  
...  
Keyword(s):  
Negative Affect ◽  
Supportive Care ◽  
Unmet Needs ◽  
Unmet Need ◽  
Hormone Treatment ◽  
Longitudinal Survey ◽  
Supportive Care Needs ◽  
Fear Of Recurrence ◽  
Care Needs ◽  
Prospective Longitudinal

Purpose To estimate prevalence and severity of patients' self-perceived supportive care needs in the immediate post-treatment phase and identify predictors of unmet need. Patients and Methods A multicenter, prospective, longitudinal survey was conducted. Sixty-six centers recruited patients for 12 weeks. Patients receiving treatment for the following cancers were recruited: breast, prostate, colorectal, and gynecologic cancer and non-Hodgkin's lymphoma. Measures of supportive care needs, anxiety and depression, fear of recurrence, and positive and negative affect were completed at the end of treatment (T0) and 6 months later (T1). Results Of 1,850 patients given questionnaire packs, 1,425 (79%) returned questionnaires at T0, and 1,152 (62%) returned questionnaires at T1. Mean age was 61 years; and most respondents were female (69%) and had breast cancer (57%). Most patients had no or few moderate or severe unmet supportive care needs. However, 30% reported more than five unmet needs at baseline, and for 60% of these patients, the situation did not improve. At both assessments, the most frequently endorsed unmet needs were psychological needs and fear of recurrence. Logistic regression revealed several statistically significant predictors of unmet need, including receipt of hormone treatment, negative affect, and experiencing an unrelated significant event between assessments. Conclusion Most patients do not express unmet needs for supportive care after treatment. Thirty percent reported more than five moderate or severe unmet needs at both assessments. Unmet needs were predicted by hormone treatment, negative mood, and experiencing a significant event. Our results suggest that there is a proportion of survivors with unmet needs who might benefit from the targeted application of psychosocial resources.

scholarly journals Social listening – revealing Parkinson’s disease over day and night

BMC Neurology ◽  
2021 ◽  
Vol 21 (1) ◽  
Author(s):  
Hui Zhang ◽  
Fanwen Meng ◽  
Xingyu Li ◽  
Yali Ning ◽  
Meng Cai
Keyword(s):  
Parkinson’S Disease ◽  
Parkinson's Disease ◽  
Unmet Needs ◽  
Unmet Need ◽  
Motor Symptoms ◽  
Motor Complications ◽  
Sentiment Score ◽  
Negative Sentiment ◽  
Non Motor Symptoms ◽  
Social Listening

Abstract Background Nocturnal symptoms in Parkinson’s disease are often treated after management of daytime manifestations. In order to better understand the unmet needs of nocturnal symptoms management, we analyzed the characteristics and burden of nocturnal symptoms from patients’ perspectives and explored their changes over time. Overall symptoms (occurring at day or night) were collected to compare whether the unmet needs related to nocturnal symptoms and to overall symptoms are different. Methods We used a Social Listening big-data technique to analyze large amounts of Parkinson’s disease symptoms in dialogues available from social media platforms in 2016 to 2018. These symptoms were classified as either overall symptoms or nocturnal symptoms. We used share of voice (SOV) of symptoms as a proportion of total dialogues per year to reflect the characteristics of symptoms. Negative sentiment score of symptoms was analyzed to find out their related burden. Results We found the SOV for overall motor symptoms was 79% and had not increased between 2016 and 2018 (79%, p = 0.5). The SOV for non-motor symptoms was 69% and had grown by 7% in 2018 (p <  0.01). The SOV for motor complications was 9% and had increased by 6% in 2018 (p <  0.01). The SOV of motor symptoms was larger than non-motor symptoms and motor complications (p <  0.01). The SOV of non-motor symptoms was larger than motor complications (p <  0.01). For nocturnal symptoms, 45% of the analyzed PD population reported nocturnal symptoms in 2018, growing by 6% (p <  0.01). The SOV for nocturnal-occurring motor symptoms was higher than most non-motor symptoms. However, non-motor symptoms had the higher increases and evoked higher negative sentiment regardless of whether they occurred during the day or night. For symptoms that can occur at either day or night, each nocturnal symptom was rated with a higher negative sentiment score than the same symptom during the day. Conclusions The growing SOV and the greater negative sentiment of nocturnal symptoms suggest management of nocturnal symptoms is an unmet need of patients. A greater emphasis on detecting and treating nocturnal symptoms with 24-h care is encouraged.

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