• Title/Summary/Keyword: clinical benefit

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Gene Therapy of Inherited Muscle Diseases (유전성 근육질환의 유전자 치료)

  • Shin, Jin-Hong
    • Annals of Clinical Neurophysiology
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    • v.14 no.2
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    • pp.53-58
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    • 2012
  • For the last decades, molecular genetics has achieved great advances that the genes on the list of inherited muscle diseases are piling up. Those diseases of overlapping clinico-pathologic findings are now understood with discrete molecular pathogeneses. We are facing an exciting era that the long-waited gene therapy may eventually come true. Skipping of dystrophin exon 51 is on successful clinical trials, which will benefit about 13% of the children suffering from Duchenne muscular dystrophy. Exon skipping is under active investigation to expand the candidates. Hopefully it may cover majority of Duchenne muscular dystrophy mutations and some of other diseases. Adeno-associated virus is one of the most versatile tools for gene transfer. It may overcome the limitation of exon skipping. Here we review exon skipping technique of Duchenne muscular dystrophy and briefly discuss the other strategies being studied to cure inherited muscle diseases.

Chemotherapy for Lung Cancer in the Era of Personalized Medicine

  • Lee, Seung Hyeun
    • Tuberculosis and Respiratory Diseases
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    • v.82 no.3
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    • pp.179-189
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    • 2019
  • Although recent advances in molecular targeted therapy and immuno-oncology have revolutionized the landscape of lung cancer therapeutics, cytotoxic chemotherapy remains an essential component of lung cancer treatment. Extensive evidence has demonstrated the clinical benefit of chemotherapy, either alone or in combination with other treatment modalities, on survival and quality of life of patients with early and advanced lung cancer. Combinational approaches with other classes of anti-neoplastic agents and new drug-delivery systems have revealed promising data and are areas of active investigation. Chemotherapy is recommended as a standard of care in patients that have progressed after tyrosine kinase inhibitors or immune checkpoint inhibitors. Chemotherapy remains the fundamental means of lung cancer management and keeps expanding its clinical implication. This review will discuss the current position and future role of chemotherapy, and specific consideration for its clinical application in the era of precision medicine.

Genetic variations affecting response of radiotherapy

  • Choi, Eun Kyung
    • Journal of Genetic Medicine
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    • v.19 no.1
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    • pp.1-6
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    • 2022
  • Radiation therapy (RT) is a very important treatment for cancer that irradiates a large amount of radiation to lead cancer cells and tissues to death. The progression of RT in the aspect of personalized medicine has greatly advanced over the past few decades in the field of technical precision responding anatomical characteristics of each patient. However, the consideration of biological heterogeneity that makes different effect in individual patients has not actually applied to clinical practice. There have been numerous discovery and validation of biomarkers that can be applied to improve the efficiency of radiotherapy, among which those related to genomic information are very promising developments. These genome-based biomarkers can be applied to identify patients who can benefit most from altering their therapeutic dose and to select the best chemotherapy improving sensitivity to radiotherapy. The genomics-based biomarkers in radiation oncology focus on mutational changes, particularly oncogenes and DNA damage response pathways. Although few have translated into clinically viable tools, there are many promising candidates in this field. In this review the prominent mutation-based biomarkers and their potential for clinical translation will be discussed.

Establishing Rationale for the Clinical Development of Cell Therapy Products: Consensus between Risk and Benefit

  • Seunghoon Han;Hyeon Woo Yim;Hyunsuk Jeong;Suein Choi;Sungpil Han
    • International Journal of Stem Cells
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    • v.16 no.1
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    • pp.16-26
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    • 2023
  • Despite long-term research achievements, the development of cell therapy (CT) products remains challenging. This is because the risks experienced by the subject and therapeutic effects in the clinical trial stage are unclear due to the various uncertainties of CT when administered to humans. Nevertheless, as autologous cell products for systemic administration have recently been approved for marketing, CT product development is accelerating, particularly in the field of unmet medical needs. The human experience of CT remains insufficient compared with other classes of pharmaceuticals, while there are countless products for clinical development. Therefore, for many sponsors, understanding the rationale of human application of an investigational product based on the consensus and improving the ability to apply it appropriately for CT are necessary. Thus, defining the level of evidence for safety and efficacy fundamentally required for initiating the clinical development and preparing it using a reliable method for CT. Furthermore, the expertise should be strengthened in the design of the first-in-human trial, such as the starting dose and dose-escalation plan, based on a sufficiently acceptable rationale. Cultivating development professionals with these skills will increase the opportunity for more candidates to enter the clinical development phase.

A Survey of the Recognition on the Practice Pattern, Diagnosis, and Treatment of Korean Medicine of Dementia and Mild Cognitive Impairment - Focusing on the Differences between Neuropsychiatrists of Korean Medicine and General Physicians - (치매, 경도인지장애의 한의진료 현황, 진단 및 치료에 대한 한의사의 인식도 조사 연구 - 한방신경정신과 전문의와 일반의의 차이를 중심으로 -)

  • Seo, Young Kyung;You, Dong Keun;Kim, Hwan;Kim, Siyeon;Lee, Go eun;Kim, Sang-Ho;Kang, Hyung-Won;Jung, In Chul
    • Journal of Oriental Neuropsychiatry
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    • v.28 no.3
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    • pp.263-274
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    • 2017
  • Objectives: To identify the current status of Korean medical practice pattern, diagnosis and treatment of dementia through recognition survey, and to use it as a preliminary data for various dementia research. Methods: Questionnaires were developed through expert meetings. The disease was defined as dementia and mild cognitive impairment, and areas were designated to practice pattern, diagnosis and treatment. From December 18, 2016-January 18, 2017, 221 respondents, including 36 neuropsychiatrists of Korean Medicine and 185 general physicians (including other medical specialists) were included. Results: 1. In both groups, the most commonly used KCD (Korean standard classification of disease and cause of death) were in the order of Unspecified Alzheimer's Dementia (F00.9), Mild Cognitive Impairment (F06.7), and Unspecified Dementia (F03). 2. The most commonly used pattern identification were zang-fu and qi-blood-yin-yang in both groups. 3. Diagnostic evaluation tools were mainly conducted by MMSE, radiologic examination, K-DRS, GDS and CDR in both groups. 4. Both groups reported using acupuncture and herbal medicine mainly. 5. In both groups, the acupuncture method was used extensively in the order of Body, Scalp, and Sa-Am. 6. Neuropsychiatrists used a variety of herbal medicines such as Wonjiseokchangpo-san (Yuanzhushichangpu-san), Yukmijihwang-tang (Liuweidihuang-tang), Palmijihwang-won (Baweidihuang-won), Sunghyangjungki-san (Xingxiang Zhengqi-san) and Ondam-tanggami (Wendan-tangjiawei). General physicians used a variety of herbal medicines such as Ondam-tanggami (Wendan-tangjiawei), Bojungikgi-tang (Buzhongyiqi-tang), Yukmijihwang-tang (Liuweidihuang-tang). 7. Neuropsychiatrists used a variety of Korean herbal preparation products (benefit and non-benefit) such as Ekgan-sangajinpibanha (Yigan-sanjiachenpibanxia), Yukmijihwang-tang (Liuweidihuang-tang), Jodeung-san (houteng-san), Palmijihwang-won (Baweidihuang-won). General physicians used a variety of Korean herbal preparation products such as Bojungikgi-tang (Buzhongyiqi-tang), Banhabaegchulcheonma-tang (banxiabaizhutianma-tang), Yukmijihwang-tang (Liuweidihuang-tang), Ekgan-sangajinpibanha (Yigan-sanjiachenpibanxia), Palmijihwang-won (Baweidihuang-won). Conclusions: By confirming awareness of Korean medical doctors treating dementia in clinical fields and understanding differences between neuropsychiatrists of Korean medicine and general physicians, it can be used to understand guideline users' needs and confirm clinical questions during development of future clinical practice guidelines for dementia.

Development of Case Management System and Analysis of Economic Feasibility under the Fee-For-Service Reimbursement (행위별 수가 지불제도 하에서의 사례관리시스템 개발 및 경제성 분석)

  • Choi, Mi Young;Chae, Young Moon;Tark, Kwan Chul;Kim, In Suk;Chun, Ja Hae
    • Quality Improvement in Health Care
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    • v.11 no.1
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    • pp.46-60
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    • 2004
  • Background : Recently, we have experienced various changes in the healthcare environment. Healthcare organizations are facing a financial crisis due to more competitive relationships among themselves as well with low health insurance fees. The purposes of the current study were: (1) to develop a data warehouse-based system for evaluating and monitoring the case management activities, and (2) to measure and analyze its effects. Methods : In order to collect the data for the study, the database on discharged patients was utilized at a university hospital located in Seoul from June 1, 2002 through December 31, 2002. Initially, a data warehouse was built for the case management system. The case management activities were analyzed using structured methodology to establish the case management system. Results : The findings of this study were as follows: (1) A case management system was developed to make it possible to monitor of healthcare quality and resource utilization. The Case management System included monitoring functions regarding utilization reviews, critical pathways, and clinical indicators. (2) Utilizing the case management system, unplanned readmissions were documented among total discharged patients during two months from November 1, 2002 through December 31, 2002. The unplanned readmission rate was 2.3%(276 patients) in total of 11,960 discharged patients. Among them 81 patients(0.7% of total discharges, 29.3% of unplanned readmission) were readmitted to the same physician in charge under the same diagnosis. No significant differences were found in the demographic variables such as gender and age among the patients. (3) After implementing the case management system, 2.9% of average length of stay reduced. Applying cost-benefit analysis, the 2.9% reduction of length of stay represents net profit of ${\backslash}$ 279,592,000 in the year of 2004. In addition, applying value acceleration analysis, cumulative net benefit of ${\backslash}$ 1,481,000,000 was expected by the year of 2007. Also we were able to expect ${\backslash}$ 247,800,000 of cumulative benefit for the prospective 5 years in value linkage analysis. It represents average ${\backslash}$ 787,700,000 of pure net benefit a year. Conclusion : The value of present study would be not only implementing the knowledge management system into the existing case management activities, but also evaluating its effects and estimating its financial benefits. This study suggested that the case management system would be a supportive tool for monitoring and improving the quality of healthcare, and a cost-effective tool for increment of healthcare organization's financial benefit.

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MR Technology Beyond 4T

  • Vaughan, Thomas
    • Proceedings of the KSMRM Conference
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    • 2003.10a
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    • pp.109-111
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    • 2003
  • Compared to the present clinical field strengths, MR at 47 and above promises to improve anatomic imaging quality by factors, and to bring metabolic and functional imaging to the forefront of research and diagnostic modalities. While human bore sized magnets as high as 9.4T are now installed, realization of the potential benefit of these magnets will require more of the MR system than a simple field, frequency or power scaling from technologies used at lower fields. New constraints on the high field MR studies, both physical and physiological, will require new technical developments to be considered for the highest field systems.

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Role Performance and Related Factors of the Clinical Research Coordinator (임상연구코디네이터의 역할수행과 관련 요인)

  • Hwang, Youn-Sun;Ko, Il-Sun
    • Journal of Korean Academy of Nursing Administration
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    • v.17 no.4
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    • pp.524-537
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    • 2011
  • Purpose: The purpose of this study was to analyze the role performance and related factors of the Clinical Research Coordinator (CRC). Methods: A quantitative, cross-sectional design was used. Data were collected using a structured questionnaire from 134 Clinical Research Coordinators in 4 hospitals and analyzed using descriptive statistics, t-test, ANOVA, and Pearson correlation coefficient. Results: The scores for role performance of CRC were high (average 336.51 points of a possible 420 points, mean average $5.74{\pm}0.67$ of a possible 7) and the degree of role performance in the management task sub dimension (mean average $6.12{\pm}0.77$ of a possible 7) was higher than any other sub dimension. Role performance of the CRC was also related to job satisfaction (r=.198, p=.023). Conclusion: The study results indicate that most CRC are nurses and perceive their role as very important. Further the CRC performance of activities of management task and the Coordination/Advocacy task sub dimension were high and higher levels of job satisfaction were related to higher levels of role performance of CRC was. Therefore, strategies related to improvement in the fringe benefit system and work environment should be developed to increase job satisfaction and thus increase job performance.

Reviews on the Hepatotoxicity of Tyrosine Kinase Inhibitors (티로신 키나아제 저해제의 간독성에 대한 고찰)

  • Han, Ji Min;Gwak, Hye Sun
    • Korean Journal of Clinical Pharmacy
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    • v.29 no.4
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    • pp.223-230
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    • 2019
  • Background: Small-molecule tyrosine kinase inhibitors (TKIs) have had major impacts on anticancer therapy by targeting the catalytic activities of dysregulated tyrosine kinases. TKIs have not presented traditional toxicities; however, some serious adverse effects, including hepatotoxicity, have been documented in clinical trials and post-marketing surveillance. Although TKI-induced hepatotoxicity can cause severe clinical complications in patients, the underlying mechanism is still unclear. Methods: Studies on TKI-induced hepatotoxicity were identified by Pubmed search, and relevant articles were reviewed. Results: Immunoallergic reaction, cytochrome P (CYP) 450 polymorphisms, and formation of reactive metabolites are under consideration as mechanisms of TKI-induced hepatotoxicity. Host protein-drug metabolite conjugates are recognized as antigens by class II major histocompatibility complexes and are believed to cause liver injuries. Polymorphisms in CYP, which influences TKI metabolism, can slow TKI metabolism and may induce development of hepatotoxicity. The formation of reactive metabolites during drug metabolism can induce hepatotoxicity by directly causing cytotoxicity, leading to cell dysfunction, and indirect toxicity by mediating secondary immune reactions. Concurrent use of various medications with TKI can also cause hepatotoxicity by affecting drug transporter or enzyme activities. Conclusion: Periodic monitoring of patients taking TKIs and risk/benefit reassessments though post marketing surveillance are necessary to prevent hepatotoxicity.

Cell-based Immunotherapy for Colorectal Cancer with Cytokine-induced Killer Cells

  • Ji Sung Kim;Yong Guk Kim;Eun Jae Park;Boyeong Kim;Hong Kyung Lee;Jin Tae Hong;Youngsoo Kim;Sang-Bae Han
    • IMMUNE NETWORK
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    • v.16 no.2
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    • pp.99-108
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    • 2016
  • Colorectal cancer is the third leading cancer worldwide. Although incidence and mortality of colorectal cancer are gradually decreasing in the US, patients with metastatic colorectal cancer have poor prognosis with an estimated 5-year survival rate of less than 10%. Over the past decade, advances in combination chemotherapy regimens for colorectal cancer have led to significant improvement in progression-free and overall survival. However, patients with metastatic disease gain little clinical benefit from conventional therapy, which is associated with grade 3~4 toxicity with negative effects on quality of life. In previous clinical studies, cell-based immunotherapy using dendritic cell vaccines and sentinel lymph node T cell therapy showed promising therapeutic results for metastatic colorectal cancer. In our preclinical and previous clinical studies, cytokine-induced killer (CIK) cells treatment for colorectal cancer showed favorable responses without toxicities. Here, we review current treatment options for colorectal cancer and summarize available clinical studies utilizing cell-based immunotherapy. Based on these studies, we recommend the use CIK cell therapy as a promising therapeutic strategy for patients with metastatic colorectal cancer.