International consensus on a proposed score system for muscle biopsy evaluation in patients with juvenile dermatomyositis: A tool for potential use in clinical trials

SummaryCancer is a major and independent risk factor of venous thromboembolism (VTE). In clinical practice, a high number of VTE events occurs in patients with cancer, and treatment of cancerassociated VTE differs in several aspects from treatment of VTE in the general population. However, treatment in cancer patients remains a major challenge, as the risk of recurrence of VTE as well as the risk of major bleeding during anticoagulation is substantially higher in patients with cancer than in those without cancer. In several clinical trials, different anticoagulants and regimens have been investigated for treatment of acute VTE and secondary prophylaxis in cancer patients to prevent recurrence. Based on the results of these trials, anticoagulant therapy with low-molecular-weight heparins (LMWH) has become the treatment of choice in cancer patients with acute VTE in the initial period and for extended and long-term anticoagulation for 3-6 months. New oral anticoagulants directly inhibiting thrombin or factor Xa, have been developed in the past decade and studied in large phase III clinical trials. Results from currently completed trials are promising and indicate their potential use for treatment of VTE. However, the role of the new oral thrombin and factor Xa inhibitors for VTE treatment in cancer patients still has to be clarified in further studies specifically focusing on cancer-associated VTE. This brief review will summarize the current strategies of initial and long-term VTE treatment in patients with cancer and discuss the potential use of the new oral anticoagulants.

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Progress in Gene Therapy to Prevent Retinal Ganglion Cell Loss in Glaucoma and Leber’s Hereditary Optic Neuropathy

Neural Plasticity ◽

10.1155/2018/7108948 ◽

2018 ◽

Vol 2018 ◽

pp. 1-11 ◽

Cited By ~ 12

Author(s):

Sara E. Ratican ◽

Andrew Osborne ◽

Keith R. Martin

Keyword(s):

Gene Therapy ◽

Clinical Trials ◽

Optic Nerve ◽

Genome Editing ◽

Optic Neuropathy ◽

Single Gene ◽

Leber’S Hereditary Optic Neuropathy ◽

Leber's Hereditary Optic Neuropathy ◽

Potential Use ◽

Hereditary Optic Neuropathy

The eye is at the forefront of the application of gene therapy techniques to medicine. In the United States, a gene therapy treatment for Leber’s congenital amaurosis, a rare inherited retinal disease, recently became the first gene therapy to be approved by the FDA for the treatment of disease caused by mutations in a specific gene. Phase III clinical trials of gene therapy for other single-gene defect diseases of the retina and optic nerve are also currently underway. However, for optic nerve diseases not caused by single-gene defects, gene therapy strategies are likely to focus on slowing or preventing neuronal death through the expression of neuroprotective agents. In addition to these strategies, there has also been recent interest in the potential use of precise genome editing techniques to treat ocular disease. This review focuses on recent developments in gene therapy techniques for the treatment of glaucoma and Leber’s hereditary optic neuropathy (LHON). We discuss recent successes in clinical trials for the treatment of LHON using gene supplementation therapy, promising neuroprotective strategies that have been employed in animal models of glaucoma and the potential use of genome editing techniques in treating optic nerve disease.

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Clinical features, muscle biopsy scores, myositis specific antibody profiles and outcome in juvenile dermatomyositis

Seminars in Arthritis and Rheumatism ◽

10.1016/j.semarthrit.2020.10.007 ◽

2021 ◽

Vol 51 (1) ◽

pp. 95-100

Author(s):

Erdal Sag ◽

Selcan Demir ◽

Yelda Bilginer ◽

Beril Talim ◽

Goknur Haliloglu ◽

...

Keyword(s):

Clinical Features ◽

Muscle Biopsy ◽

Specific Antibody ◽

Juvenile Dermatomyositis

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Validation of a score tool for measurement of histological severity in juvenile dermatomyositis and association with clinical severity of disease

Annals of the Rheumatic Diseases ◽

10.1136/annrheumdis-2013-203396 ◽

2013 ◽

Vol 74 (1) ◽

pp. 204-210 ◽

Cited By ~ 31

Author(s):

Hemlata Varsani ◽

Susan C Charman ◽

Charles K Li ◽

Suely K N Marie ◽

Anthony A Amato ◽

...

Keyword(s):

Disease Activity ◽

Muscle Biopsy ◽

Juvenile Dermatomyositis ◽

Intraclass Correlation ◽

Latin Square ◽

Quadriceps Muscle ◽

Clinical Severity ◽

Intraobserver Agreement ◽

Biopsy Tissue ◽

Clinical Measures

ObjectivesTo study muscle biopsy tissue from patients with juvenile dermatomyositis (JDM) in order to test the reliability of a score tool designed to quantify the severity of histological abnormalities when applied to biceps humeri in addition to quadriceps femoris. Additionally, to evaluate whether elements of the tool correlate with clinical measures of disease severity.Methods55 patients with JDM with muscle biopsy tissue and clinical data available were included. Biopsy samples (33 quadriceps, 22 biceps) were prepared and stained using standardised protocols. A Latin square design was used by the International Juvenile Dermatomyositis Biopsy Consensus Group to score cases using our previously published score tool. Reliability was assessed by intraclass correlation coefficient (ICC) and scorer agreement (α) by assessing variation in scorers’ ratings. Scores from the most reliable tool items correlated with clinical measures of disease activity at the time of biopsy.ResultsInter- and intraobserver agreement was good or high for many tool items, including overall assessment of severity using a Visual Analogue Scale. The tool functioned equally well on biceps and quadriceps samples. A modified tool using the most reliable score items showed good correlation with measures of disease activity.ConclusionsThe JDM biopsy score tool has high inter- and intraobserver agreement and can be used on both biceps and quadriceps muscle tissue. Importantly, the modified tool correlates well with clinical measures of disease activity. We propose that standardised assessment of muscle biopsy tissue should be considered in diagnostic investigation and clinical trials in JDM.

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Treatment and secondary prevention of venous thrombo -embolism in cancer patients

Hämostaseologie ◽

10.5482/ha-1168 ◽

2012 ◽

Vol 32 (02) ◽

pp. 139-144 ◽

Cited By ~ 3

Author(s):

I. Pabinger ◽

C. Ay

Keyword(s):

Clinical Trials ◽

Cancer Patients ◽

Oral Anticoagulants ◽

Initial Period ◽

Factor Xa ◽

Secondary Prophylaxis ◽

Phase Iii ◽

Patients With Cancer ◽

Potential Use

SummaryCancer is a major and independent risk factor of venous thromboembolism (VTE). In clinical practice, a high number of VTE events occurs in patients with cancer, and treatment of cancer-associated VTE differs in several aspects from treatment of VTE in the general population. However, treatment in cancer patients remains a major challenge, as the risk of recurrence of VTE as well as the risk of major bleeding during anticoagulation is substantially higher in patients with cancer than in those without cancer. In several clinical trials, different anticoagulants and regimens have been investigated for treatment of acute VTE and secondary prophylaxis in cancer patients to prevent recurrence. Based on the results of these trials, anticoagulant therapy with low-molecular-weight heparins (LMWH) has become the treatment of choice in cancer patients with acute VTE in the initial period and for extended and long-term anticoagulation for 3–6 months. New oral anti-coagulants directly inhibiting thrombin or factor Xa, have been developed in the past decade and studied in large phase III clinical trials. Results from currently completed trials are promising and indicate their potential use for treatment of VTE also in cancer patients. However, the role of the new oral thrombin and factor Xa inhibitors for VTE treatment in cancer patients still has to be clarified in further studies specifically focusing on cancer-associated VTE. This brief review will summarize the current strategies of initial and long-term VTE treatment in patients with cancer and discuss the potential use of the new oral anticoagulants.

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Achieving international consensus on the assessment of psoriatic arthritis in psoriasis clinical trials: an International Dermatology Outcome Measures (IDEOM) initiative

Archives of Dermatological Research ◽

10.1007/s00403-018-1855-3 ◽

2018 ◽

Vol 310 (9) ◽

pp. 701-710 ◽

Cited By ~ 5

Author(s):

Lourdes Maria Perez-Chada ◽

Jeffrey M. Cohen ◽

Alice Bendix Gottlieb ◽

Kristina Callis Duffin ◽

Amit Garg ◽

...

Keyword(s):

Clinical Trials ◽

Psoriatic Arthritis ◽

Outcome Measures ◽

International Consensus

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Diagnostic evaluation and monitoring of patients with posterior cortical atrophy

Neurodegenerative Disease Management ◽

10.2217/nmt-2018-0052 ◽

2019 ◽

Vol 9 (4) ◽

pp. 217-239

Author(s):

Bonnie Wong ◽

Diane E Lucente ◽

Julie MacLean ◽

Jaya Padmanabhan ◽

Megan Quimby ◽

...

Keyword(s):

Clinical Trials ◽

Clinical Research ◽

Clinical Status ◽

Cortical Atrophy ◽

Posterior Cortical Atrophy ◽

International Consensus ◽

Visuospatial Functions ◽

Consensus Classification ◽

Evaluation And Monitoring ◽

Illness Progression

Posterior cortical atrophy (PCA) is a progressive neurocognitive syndrome, most commonly associated with the loss of complex visuospatial functions. Diagnosis is challenging, and international consensus classification and nomenclature for PCA subtypes have only recently been reached. Presently, no established treatments exist. Efforts to develop treatments are hampered by the lack of standardized methods to monitor illness progression. Although measures developed from work with Alzheimer’s disease and other dementias provide a foundation for diagnosing and monitoring progression, PCA presents unique challenges for clinicians counseling patients and families on clinical status and prognosis, and experts designing clinical trials of interventions. Here, we review issues facing PCA clinical research and care, summarize our approach to diagnosis and monitoring of disease progression, and outline ideas for developing tools for these purposes.

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Hip protectors: recommendations for conducting clinical trials—an international consensus statement (part II)

Osteoporosis International ◽

10.1007/s00198-009-1055-2 ◽

2009 ◽

Vol 21 (1) ◽

pp. 1-10 ◽

Cited By ~ 25

Author(s):

I. D. Cameron ◽

S. Robinovitch ◽

S. Birge ◽

P. Kannus ◽

K. Khan ◽

...

Keyword(s):

Clinical Trials ◽

Consensus Statement ◽

International Consensus ◽

Hip Protectors ◽

International Consensus Statement

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Clinical Trials in Recurrent Ovarian Cancer

International Journal of Gynecological Cancer ◽

10.1097/igc.0b013e31821bb8aa ◽

2011 ◽

Vol 21 (4) ◽

pp. 771-775 ◽

Cited By ~ 112

Author(s):

Michael Friedlander ◽

Edward Trimble ◽

Anna Tinker ◽

David Alberts ◽

Elisabeth Avall-Lundqvist ◽

...

Keyword(s):

Ovarian Cancer ◽

Clinical Trials ◽

Gynecologic Cancer ◽

Recurrent Ovarian Cancer ◽

Consensus Conference ◽

Ca 125 ◽

Therapeutic Approaches ◽

Cooperative Research ◽

International Consensus

The 4th Ovarian Cancer Consensus Conference of the Gynecologic Cancer InterGroup was held in Vancouver, Canada, in June 2010. Representatives of 23 cooperative research groups studying gynecologic cancers gathered to establish international consensus on issues critical to the conduct of large randomized trials. Group C, 1 of the 3 discussion groups, examined recurrent ovarian cancer, and we report the consensus reached regarding 4 questions. These included the following: (1) What is the role of cytoreductive surgery for recurrent ovarian cancer? (2) How do we define distinct patient populations in need of specific therapeutic approaches? (3) Should end points for trials with recurrent disease vary from those of first-line trials? (4) Is CA-125 progression alone sufficient for entry/eligibility into clinical trials?

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