Putting UK DCTN studies into practice: a nurse and patient perspective of the investigator day for the BLISTER and STOP GAP trials

The BLISTER and STOP GAP studies are two important trials in the field of dermatology and also for the UK Dermatology Clinical Trials Network (UK DCTN) as a collaborative network. They make a significant contribution to our limited evidence base for the management of bullous pemphigoid and pyoderma gangrenosum respectively. In keeping with the priorities of the UK DCTN, both trials investigated treatments routinely available in dermatology and focused on rarer skin diseases which benefited from involvement of the network to recruit a sufficient number of patients. The ‘Putting UK DCTN Studies into Practice Event’ was an opportunity for clinicians, patients and researchers involved to share their experiences of the studies and the related skin conditions. Investigators from both trials commented on their experiences and how being involved in the BLISTER and STOP GAP trials had affected their attitude to research and changed their clinical practice. In this article we hope to continue the ethos of a shared experience by providing an insight of the investigator day from a nursing and patient perspective

Putting UK DCTN studies into practice: a nurse and patient perspective of the investigator day for the BLISTER and STOP GAP trials

The BLISTER and STOP GAP studies are two important trials in the field of dermatology and also for the UK Dermatology Clinical Trials Network (UK DCTN) as a collaborative network. They make a significant contribution to our limited evidence base for the management of bullous pemphigoid and pyoderma gangrenosum respectively. In keeping with the priorities of the UK DCTN, both trials investigated treatments routinely available in dermatology and focused on rarer skin diseases which benefited from involvement of the network to recruit a sufficient number of patients. The ‘Putting UK DCTN Studies into Practice Event’ was an opportunity for clinicians, patients and researchers involved to share their experiences of the studies and the related skin conditions. Investigators from both trials commented on their experiences and how being involved in the BLISTER and STOP GAP trials had affected their attitude to research and changed their clinical practice. In this article we hope to continue the ethos of a shared experience by providing an insight of the investigator day from a nursing and patient perspective

Putting UK DCTN studies into practice: a nurse and patient perspective of the investigator day for the BLISTER and STOP GAP trials

The BLISTER and STOP GAP studies are two important trials in the field of dermatology and also for the UK Dermatology Clinical Trials Network (UK DCTN) as a collaborative network. They make a significant contribution to our limited evidence base for the management of bullous pemphigoid and pyoderma gangrenosum respectively. In keeping with the priorities of the UK DCTN, both trials investigated treatments routinely available in dermatology and focused on rarer skin diseases which benefited from involvement of the network to recruit a sufficient number of patients. The ‘Putting UK DCTN Studies into Practice Event’ was an opportunity for clinicians, patients and researchers involved to share their experiences of the studies and the related skin conditions. Investigators from both trials commented on their experiences and how being involved in the BLISTER and STOP GAP trials had affected their attitude to research and changed their clinical practice. In this article we hope to continue the ethos of a shared experience by providing an insight of the investigator day from a nursing and patient perspective

(2006-2007) Assembly Resolution 04: In Support of Fiscal Accountability and Equal Access to Campus Recreational Facilities for Students who are Parents

Governmen

The top 10 research priorities for the treatment of bullous pemphigoid, mucous membrane pemphigoid and pemphigus vulgaris in the UK: results of a James Lind Alliance Priority Setting Partnership

To identify priorities for future research into the treatment of bullous pemphigoid, mucous membrane pemphigoid and pemphigus vulgaris, we conducted a Priority Setting Partnership (PSP) using James Lind Alliance methodology. The top 10 priorities identified in this PSP represent the key questions that patients, carers and healthcare professionals have about the treatment of these diseases and which future research will hopefully address

'North Sea' progressive myoclonus epilepsy: phenotype of subjects with GOSR2 mutation

We previously identified a homozygous mutation in the Golgi SNAP receptor complex 2 gene (GOSR2) in six patients with progressive myoclonus epilepsy. To define the syndrome better we analysed the clinical and electrophysiological phenotype in 12 patients with GOSR2 mutations, including six new unrelated subjects. Clinical presentation was remarkably similar with early onset ataxia (average 2 years of age), followed by myoclonic seizures at the average age of 6.5 years. Patients developed multiple seizure types, including generalized tonic clonic seizures, absence seizures and drop attacks. All patients developed scoliosis by adolescence, making this an important diagnostic clue. Additional skeletal deformities were present, including pes cavus in four patients and syndactyly in two patients. All patients had elevated serum creatine kinase levels (median 734 IU) in the context of normal muscle biopsies. Electroencephalography revealed pronounced generalized spike and wave discharges with a posterior predominance and photosensitivity in all patients, with focal EEG features seen in seven patients. The disease course showed a relentless decline; patients uniformly became wheelchair bound (mean age 13 years) and four had died during their third or early fourth decade. All 12 cases had the same variant (c.430G>T, G144W) and haplotype analyses confirmed a founder effect. The cases all came from countries bounding the North Sea, extending to the coastal region of Northern Norway. 'North Sea' progressive myoclonus epilepsy has a homogeneous clinical presentation and relentless disease course allowing ready identification from the other progressive myoclonus epilepsies.Lysa Boissé Lomax...Jozef Gecz... et al

Selective Requirement of PI3K/PDK1 Signaling for Kras Oncogene-Driven Pancreatic Cell Plasticity and Cancer

SummaryOncogenic Kras activates a plethora of signaling pathways, but our understanding of critical Ras effectors is still very limited. We show that cell-autonomous phosphoinositide 3-kinase (PI3K) and 3-phosphoinositide-dependent protein kinase 1 (PDK1), but not Craf, are key effectors of oncogenic Kras in the pancreas, mediating cell plasticity, acinar-to-ductal metaplasia (ADM), and pancreatic ductal adenocarcinoma (PDAC) formation. This contrasts with Kras-driven non-small cell lung cancer, where signaling via Craf, but not PDK1, is an essential tumor-initiating event. These in vivo genetic studies together with pharmacologic treatment studies in models of human ADM and PDAC demonstrate tissue-specific differences of oncogenic Kras signaling and define PI3K/PDK1 as a suitable target for therapeutic intervention specifically in PDAC

Of Mice and Monkeys: Can Animal Models Be Utilized to Study Neurological Consequences of Pediatric HIV-1 Infection?

Pediatric human immunodeficiency virus (HIV-1) infection remains a global health crisis. Children are much more susceptible to HIV-1 neurological impairments than adults, which can be exacerbated by coinfections. Neurological characteristics of pediatric HIV-1 infection suggest dysfunction in the frontal cortex as well as the hippocampus; limited MRI data indicate global cerebral atrophy, and pathological data suggest accelerated neuronal apoptosis in the cortex. An obstacle to pediatric HIV-1 research is a human representative model system. Host-species specificity of HIV-1 limits the ability to model neurological consequences of pediatric HIV-1 infection in animals. Several models have been proposed including neonatal intracranial injections of HIV-1 viral proteins in rats and perinatal simian immunodeficiency virus (SIV) infection of infant macaques. Nonhuman primate models recapitulate the complexity of pediatric HIV-1, neuropathogenesis while rodent models are able to elucidate the role specific viral proteins exert on neurodevelopment. Nonhuman primate models show similar behavioral and neuropathological characteristics to pediatric HIV-1 infection and offer a stage to investigate early viral mechanisms, latency reservoirs, and therapeutic interventions. Here we review the relative strengths and limitations of pediatric HIV-1 model systems. [Image: see text