scholarly journals Global consensus building and prioritisation of fundamental lupus challenges: the ALPHA project

2019 ◽  
Vol 6 (1) ◽  
pp. e000342 ◽  
Author(s):  
Susan Manzi ◽  
Sandra Raymond ◽  
Karin Tse ◽  
Yaritza Peña ◽  
Annick Anderson ◽  
...  
Keyword(s):  
Drug Development ◽  
Clinical Care ◽  
Treatment Options ◽  
Clinical Trial Design ◽  
Expert Interviews ◽  
Long Term Outcomes ◽  
Global Consensus ◽  
Autoimmune Conditions ◽  
The Continuum

ObjectiveLupus is a complex, heterogeneous autoimmune disease that has yet to see significant progress towards more timely diagnosis, improved treatment options for short-term and long-term outcomes, and appropriate access to care. The Addressing Lupus Pillars for Health Advancement (ALPHA) project is the first step in establishing global consensus and developing concrete strategies to address the challenges limiting progress.MethodsA Global Advisory Committee of 13 individuals guided the project and began barrier identification. Seventeen expert interviews were conducted to further characterise key barriers. Transcripts were analysed using Nvivo and a codebook was created containing a list of thematic ‘nodes’ (topics) and their descriptions. Findings were used to develop a final survey instrument that was fielded to a diverse, international stakeholder audience to achieve broad consensus.ResultsExpert interviews identified lupus heterogeneity as the primary barrier hindering advancement. Subsequent barriers were categorised into three areas: (1) Drug development. (2) Clinical care. (3) Access and value. The global survey received 127 completed responses from experts across 20 countries. Respondents identified barriers as high priority including the lack of biomarkers for clinical and drug development use, flawed clinical trial design, lack of access to clinicians familiar with lupus, and obstacles to effective management of lupus due to social determinants of care. Respondents also identified 30 autoimmune conditions that may be lupus-related based on overlapping features, shared autoantibodies and pathophysiology.ConclusionsALPHA is a comprehensive initiative to identify and prioritise the continuum of challenges facing people with lupus by engaging a global audience of lupus experts. It also explored views on lupus as a spectrum of related diseases. Conclusions from this effort provide a framework to generate actionable approaches to the identified high-priority barriers.

Treatment options and long-term outcomes in pediatric spinal cord vascular malformations: a case report and review of the literature

2020 ◽  
Vol 36 (12) ◽  
pp. 3147-3152
Author(s):  
Helen J. Zhang ◽  
Nicole Silva ◽  
Elena Solli ◽  
Amanda C. Ayala ◽  
Luke Tomycz ◽  
...  
Keyword(s):  
Spinal Cord ◽  
Case Report ◽  
Vascular Malformations ◽  
Treatment Options ◽  
Review Of The Literature ◽  
Long Term Outcomes

Endovascular management of acute ischemic stroke

2009 ◽  
Vol 26 (3) ◽  
pp. E2 ◽  
Author(s):  
Chirag D. Gandhi ◽  
Lana D. Christiano ◽  
Charles J. Prestigiacomo
Keyword(s):  
Acute Stroke ◽  
Plasminogen Activator ◽  
Tissue Plasminogen Activator ◽  
Treatment Options ◽  
Antiplatelet Agents ◽  
Interventional Treatment ◽  
Long Term Outcomes ◽  
Treatment Protocols ◽  
Tissue Plasminogen

The management of stroke has progressed significantly over the past 2 decades due to successful treatment protocols including intravenous and intraarterial options. The intravenous administration of tissue plasminogen activator within an established treatment window has been proven in large, well-designed studies. The evolution of endovascular strategies for acute stroke has been prompted by the limits of the intravenous treatment, as well as by the desire to demonstrate improved recanalization rates and improved long-term outcomes. The interventional treatment options available today are the intraarterial administration of tissue plasminogen activator and newer antiplatelet agents, mechanical thrombectomy with the MERCI device and the Penumbra system, and intracranial angioplasty and stent placement. In this review the authors outline the major studies that have defined the current field of acute stroke management and discuss the basic treatment paradigms that are commonly used today.

New advances in the diagnosis and management of hepatocellular carcinoma

BMJ ◽  
2020 ◽  
pp. m3544 ◽  
Author(s):  
Ju Dong Yang ◽  
Julie K Heimbach
Keyword(s):  
Hepatocellular Carcinoma ◽  
Systemic Treatment ◽  
Early Stage ◽  
Treatment Options ◽  
Intermediate Stage ◽  
Phase Iii ◽  
Advanced Hepatocellular Carcinoma ◽  
Long Term Outcomes ◽  
Stage Disease

ABSTRACT Hepatocellular carcinoma is one of the leading causes of cancer related death in the world. Biannual surveillance for the disease in patients with cirrhosis and in high risk carriers of hepatitis B virus allows early stage cancer detection and treatment with good long term outcomes. Liver ultrasonography and serum α fetoprotein are the most commonly used surveillance tests. If suspicious results are found on the surveillance test, multiphasic computed tomography or magnetic resonance imaging should be undertaken to confirm the diagnosis of hepatocellular carcinoma. If radiologic tests show inconclusive results, liver biopsy or repeat imaging could be considered for confirmation of hepatocellular carcinoma. Management of the disease is complex. Patients should be evaluated by a multidisciplinary team, and the selection of treatment should consider factors such as tumor burden, severity of liver dysfunction, medical comorbidities, local expertise, and preference of patients. Early stage hepatocellular carcinoma is best managed by curative treatment, which includes resection, ablation, or transplantation. Patients with intermediate stage disease often receive locoregional treatment. Systemic treatment is reserved for patients with advanced disease. Several positive, phase III, randomized controlled trials have expanded the systemic treatment options for advanced hepatocellular carcinoma with promising long term outcomes, especially trials using combination treatments, which could also have eventual implications for the treatment of earlier stage disease.

scholarly journals Clinical Features and Long-Term Follow-Up of Patients with Retinoblastoma in Turkish Children Older Than 5 Years of Age

2020 ◽  
Vol 2020 ◽  
pp. 1-5
Author(s):  
Zafer Cebeci ◽  
Samuray Tuncer ◽  
Rejin Kebudi
Keyword(s):  
Clinical Features ◽  
Treatment Options ◽  
Older Children ◽  
Long Term Outcomes ◽  
Turkish Children ◽  
Turkish Patient ◽  
Long Term Follow Up ◽  
Salvage Rate

Purpose. The diagnosis and management of retinoblastoma can be difficult in older children. This study reports the clinical features and long-term outcomes of such patients in a Turkish patient population. Methods. Medical records of 18 patients, between January 1992 and December 2017, were reviewed retrospectively. Results. Of 298 consecutive patients with retinoblastoma, 18 (6%) were at the age of 5 years and older. The median age at diagnosis was 9 years (range, 5–18 years). Misdiagnosis was noted in 8 patients (44.4%). Treatment options included enucleation in 16 patients (88.8%), adjuvant systemic chemotherapy in 3 (16.6%), intra-arterial chemotherapy in one (5.5%), and additional orbital irradiation in one (5.5%). After a median follow-up of 97 months (range, 6–252 months), all patients survived without any recurrence. Conclusions. Our series showed that 6% of patients with retinoblastoma were 5 years of age and older. Over a 15-year period, ocular salvage rate of 11.2% and survival rate of 100% were attained.

scholarly journals Aortic valve surgery: management and outcomes in the paediatric population

2021 ◽  
Author(s):  
Mariam Zaidi ◽  
Ganeshkumar Premkumar ◽  
Rimel Naqvi ◽  
Arwa Khashkhusha ◽  
Zahra Aslam ◽  
...  
Keyword(s):  
Aortic Valve ◽  
Treatment Options ◽  
Surgical Techniques ◽  
Paediatric Population ◽  
Long Term Outcomes ◽  
Mechanical Prosthesis ◽  
Pulmonic Valve ◽  
Replacement Surgery ◽  
Surgical Options

AbstractCongenital anomalies of the aortic valve frequently necessitate intervention in childhood. The most common aortic valve pathologies present in childhood are aortic stenosis and insufficiency. Presentation of aortic valve disease depends on severity and presence of concomitant syndromes and valvular disorders. Treatment options are largely categorised as medical, percutaneous repair or surgical repair and replacement. Surgical techniques have been refined over the last few years making this the mainstay of treatment in paediatric cases. Whilst repair is considered in most instances before replacement, there are substantial limitations which are reflected in the frequency of reintervention and restenosis rate. Replacements are typically undertaken with tissue or mechanical prosthesis. The current gold-standard aortic valve replacement surgery is called the Ross procedure—where replacement is undertaken with a competent pulmonic valve and a simultaneous pulmonary homograft.Conclusion: In this review, we aim to outline the various surgical options and discuss efficacy and complications of various interventions. What is Known: • Congenital aortic valve defects repair options medically and surgically What is New: • Comparisons between surgical options for aortic valve repair including efficacy, risks and long-term outcomes.

scholarly journals Pregnancy and childbirth during the COVID-19 pandemic in Poland: qualitative evidence from expert interviews.

2020 ◽  
Author(s):  
Paula Pustułka ◽  
Marta Buler
Keyword(s):  
Maternal Health ◽  
Maternal Mental Health ◽  
Clinical Care ◽  
Expert Interviews ◽  
Maternal Support ◽  
Care Model ◽  
Pregnancy And Childbirth ◽  
Recorded Data ◽  
Health Experts

Abstract Background: The COVID-19 outbreak has significantly altered the provisions and shape of medical services for expecting mothers worldwide. In Poland, the country where pregnancy and childbirth are subject to comparably greater medicalization, those shifts are particularly tangible. This study aimed at capturing how maternal health professionals tackle care over pregnant and birthing women during a pandemic, highlighting possible long-term implications for the expecting and new mothers. Methods: This is a qualitative study which addresses the themes around the emerging implications of the viral outbreak. Semi-structured expert interviews were conducted with 46 individuals working in either medical or adjacent sectors connected with reproductive medicine. In this analysis, 7 digital experts were excluded as observers rather than field practitioners. Among 38 interviews, 5 have been conducted with OB-GYNs, 13 with midwives, 6 with doulas, 14 broadly-conceived category of maternal support experts. The recorded data were input into thematic grids and meticulously transcribed prior to being subject to an inductive, thematic analysis. Results: The findings revealed that the context of the heightened medicalization of pregnancy and hospital care during birth in Poland, resulted in strong restrictions and immediate fall back to a clinical care model. The majority claimed that the COVID-19 pandemic exacerbated the implications of the decades of Polish maternal health recommendations contradicting the WHO regulations. Professionals’ anxieties concern limited access to services assisting women during pregnancies. While those linked to hospitals pointed out physiological risks, other maternal support experts drew attention to the effects of stress and uncertainty on maternal mental health. Conclusions: Experts face various challenges when providing care to pregnant and birthing women in Poland. They underscore both the information chaos surrounding recommendations and procedures, and see the pandemic as a moment of reflection, mostly being vocal in their critiques of the over-medicalized Polish care model. The interviewed maternal health experts believe that the pandemic will have multifaceted consequences that are being exacerbated by a sudden cessation of extensive degree of medicalization during pregnancy, and a fallback to alienation during intervention-focused hospital deliveries.

Developing tomorrow's antipsychotics: the need for a more personalised approach

2014 ◽  
Vol 20 (1) ◽  
pp. 3-12 ◽  
Author(s):  
Robert Hunter
Keyword(s):  
Risk Factors ◽  
Differential Diagnosis ◽  
Drug Discovery ◽  
Molecular Biology ◽  
Drug Development ◽  
Environmental Risk ◽  
Response To Treatment ◽  
Long Term Outcomes ◽  
Complex Interplay

SummaryThere has been little pharmacological advance in the treatment of schizophrenia since the introduction of chlorpromazine in the 1950s. This may be set to change as recent advances in molecular biology offer the prospect of a better understanding of the pathophysiology of the disorder and allow investigation of the complex interplay of genetic and environmental risk factors. In this review I discuss future approaches to antipsychotic drug development, highlighting the need to better define symptom areas and develop drugs based on an understanding of neurobiological mechanisms. The development of biomarkers has the potential in future to improve differential diagnosis and help predict response to treatment. These developments herald the possibility of a more integrated drug discovery approach and the subsequent provision of more stratified healthcare, and hopefully significant improvements in patient care and improved long-term outcomes.

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