Análisis descriptivo de 4776 pacientes internados en servicios de clínica médica POR COVID-19. Resultados del Registro Multicéntrico Argentino - REMA-COVID-19 / Descriptive analysis of 4776 patients admitted to medical clinic services for COVID-19. Results of the Argentine Multi-Center Registry - REMA-COVID-19

Resumen Se realizó un registro multicéntrico que incluyó personas adultas internadas por COVID-19 en varias provincias de la Argentina, desde marzo a octubre de 2020. Los objetivos fueron describir las características epidemiológicas, manifestaciones clínicas, tratamientos, complicaciones y factores de riesgo, necesidad de admisión a unidades de cuidados críticos y mortalidad. El registro incluyó información de 4776 pacientes, en 37 centros de salud de Argentina. El 70.2% provenían de la ciudad de Buenos Aires y la provincia de Buenos Aires. El 52.3% eran hombres. La media de edad fue de 56 años (DE 20.3). Un 13.1% de pacientes manifestó ser personal de salud. La mediana de tiempo de síntomas al momento de la internación fue de 3 días (IC 1-6). Las comorbilidades más frecuentes fueron hipertensión arterial en 32.4% y diabetes mellitus en 15.8%. Los síntomas más frecuentes fueron: tos 58%, odinofagia 23.3%, mialgias 20.5% y fiebre/febrícula 19.9%. La estadía hospitalaria tuvo una mediana de internación de 8 días (IC 4-15). El 14.8% de los pacientes requirió cuidados críticos, en tanto que el 3.2%, que también lo requería, no pasó a unidad cerrada por adecuación del esfuerzo terapéutico. Las complicaciones más frecuentes en cuidados críticos fueron: eventos cardiovasculares (54.1%), shock séptico (33.3%), insuficiencia renal (9.7%) y neumonía asociada a la ventilación mecánica (12.5%). La mortalidad global fue del 12.3%. La edad avanzada, demencia y EPOC se comportaron como predictores independientes de mortalidad (p < 0.001, 0.007 y 0.002 respectivamente) en el análisis multivariado.

Abstract A multicenter registry that in cluded adults hospitalized for COVID-19 was carried out in various provinces of Argentina, from March to October 2020. The objectives were to describe the epidemiological characteristics, clinical manifestations, treatments, complications and risk factors, need for admission to critical care units and mortality. The registry included information on 4776 patients in 37 health centers in Argentina. Of them, 70.2% came from the city of Buenos Aires and from Buenos Aires Province; 52.3% were men. The mean age was 56 years (SD 20.3). Of them, 13.1% stated that they were health personnel. The median time of symptoms at the time of hospitalization was 3 days (CI 1-6). The most frequent comorbidities were hypertension in 32.4% and diabetes mellitus in 15.8%. The most frequent symptoms were: cough 58%, odynophagia 23.3%, myalgia 20.5% and fever / low-grade fever 19.9%. The hospital stay had a median of 8 days (CI 4-15). A 14.8% of the patients required critical care, while 3.2% who also required it, were not transferred to a closed unit due to adequacy of the thera peutic effort. The most frequent complications in critical care were: cardiovascular events (54.1%), septic shock (33.3%), renal failure (9.7%) and pneumonia associated with mechanical ventilation (12.5%). Overall mortality was 12.3%. Old age, dementia and COPD behaved as independent predictors of mortality (p < 0.001, 0.007 and 0.002 respectively) in the multivariate analysis.

1. The Advance of Utilizing Real World Data Under Pharmaceutical Regulatory Schemes Regarding the Approval Review and Post-marketing Safety Assessment / 薬剤疫学

Pharmaceutical regulatory schemes concerned with real world data have been changing remarkably in terms of both drug development and post-marketing.As for drug development, ICH proposed “GCP Renovation” , which includes modernization of E8 Guideline (General Consideration for Clinical Trials) and subsequent renovation of E6 Guideline (Good Clinical Practice). It covers pragmatic clinical trials, randomized controlled trials using patient registry data, and even observational studies using real world data. In the US, “The 21st Century Cures Act” refers to the proposal concerning speeding up the approval review by making clinical trials more efficient. In fact, there are some cases where real world data was used in the approval review of expanding the application. Also, in Japan, Clinical Innovation Network (CIN) plan was announced, and utilizing the natural history data like patient registry as the control group of clinical trials for the approval review is now under consideration.As regards post-marketing surveillance, “Conditional & Accelerated Approval” (October 20, 2017, Yakuseiyakushinhatsu 1020 No. 1) stated that post-marketing confirmation of validity of efficacy and safety using real world data, such as Medical Information Database Network (MID-NET) project and patients registries in CIN plan, is required to get the early approval. Moreover, “The Ordinance on Good Post-Marketing Practice” (October 26, 2017, MHLW Ordinance No. 116) newly provided “Post-marketing Database Study” , which is conducted by use of medical information database like MID-NET, and utilizing real world data for post-marketing safety monitoring began.

Discussion on Patient Registry about Acupuncture Therapy for Premature Ovarian Insufficiency / 世界科学技术-中医药现代化

Premature ovarian insufficiency (POI) is characterized by amenorrhea before age 40 with raised FSH level and decreased E2 level. POI, as a main reason for infertility, poses great threat on patients. physical and psychological health.By reviewing clinical trials of acupuncture in treating premature ovarian insufficiency, this study focuses on the significance and feasibility of patient registry about acupuncture therapy for premature ovarian insufficiency. Patient registry can be used in collecting real-world evidence and exploring effective intervention to improve pregnancy rate

Key Technologies of International Patient Registry Study for Acupuncture-moxibustion Based on Real World / 世界科学技术-中医药现代化

Acupuncture-moxibustion has been used in more than 180 countries,but high quality evidences for its efficacy and safety are still insufficient,which is a key issue limiting the development of acupuncture-moxibustion.Combined with characteristics of acupuncture-moxibustion discipline,this study was aimed to use international patient registry to collect real world clinical data and design meaningful clinical outcome in order to exploit the efficacy and safety of acupuncture-moxibustion and to provide important basis and supplement for confirmatory research.This paper summarized key technologies of international patient registry study for acupuncture-moxibustion based on real world to guide the design,implementation and evaluation.

The national rare diseases registry system of China and the related cohorts studies:vision and roadmap / 中华内分泌代谢杂志

[Summary] Rare diseases are one of the major challenges we face today in the era of precision medicine, because of the low incidence and prevalence, difficulty in diagnosis, lack of sufficient therapeutic methods, as well as their significant impacts on affected individuals, families and the society. Integration of clinical phenotypic and biological omics data and the further analysis are providing a way to illustrate the mechanisms of rare diseases, discovering novel diagnostic and prognostic biomarkers, developing orphan drugs and other therapeutics, and improving clinical outcomes and quality of life for the patients. A nation-wide registry system and the cohorts studies based on the registry are vital to the research of rare diseases. National Rare Diseases Registry System ( NRDRS) of China will provide this essential platform to promote the rare diseases research in China. With the collaboration of 20 leading medical institutes and innovation in medical informatics technologies, this system will, for the first time in China, collect the epidemiological, clinical, socio-economical, genomics and metabolomics data of more than 50 rare diseases and not less than 50 000 cases. As a national strategy for enhancing the development of medical sciences and the improvement of population health in China, NRDRS and its cohort studies will provide the pivotal support to policy making, clinical care, novel drug discovery, patient advocacy, and finally scientific progress in the field of rare diseases.

Clinical Outcome Research through Patient Registries / 임상당뇨병

As our population ages, so grows the amount of medical expenditures. In the future, elderly people will be better organized, more assertive and have higher expectations of their quality of life. More advanced health technologies have been developed with potential harm as well as beneficial effectiveness. We should provide the patients with health technologies with high scientific evidences. Randomized control trials (RCTs) provide the highest level of scientific evidence, but cannot always be performed because of ethical, practical or financial limitations, and do not always reflect what occurs in real clinical practice. However, patient registries can be used to evaluate patient outcomes through prospective evaluation of a structured collection of clinical information. Therefore, patient registries can complement RCTs.

Korean Gaucher Disease Patient Registry / 대한소아혈액종양학회지

BACKGROUND: This study is aimed to identify Gaucher disease(GD) patients(pts.) in Korea in order to better understand the incidence, clinical phenotype and its natural history and to establish pt. registry. The registry is to provide collective data, which will lead to early diagnosis, effective treatment and better management of pts. METHODS: (1)Questionnaires designed to identify GD pts. were sent to all members of Korean Pediatric Hemato-Oncology Society in 1997 and other inquiring physicians. (2) All published papers on GD were searched in Korean Medical Journals to Feb. 1988. RESULTS: Review of data obtained from questionnaire survey and literature search yielded a total of 44 pts. in 31 pedigrees. 33 of them were reported as type I(chronic, non- neuronopathic), 6 pts. as type II(acute, neuronopathic) and 5 pts. as type III(subacute, neuronopathic). As for the most common type I pts, all 33 but one were diagnosed in infancy and childhood(7mon~18y.o), 11 of them died during childhood(1yr~18y.o), 10 pts. were not known whether alive or dead and 12 pts. are alive and followed up. All 6 type II pts. were diagnosed during infancy and 4 of them died before age of two. All 5 type III pts. in 2 families developed myoclonic seizures in late teens and two died in early twenties and three of them being followed up by neurologists. CONCLUSION: A total of 44 GD pts. in 31 Korean families are identified, which is consistant with autosomal recessive mode of inheritance pattern in all three clinical phenotypes. As of Feb. 1988, 18 pts. were registered in Korean Gaucher Registry; 12 type I pts, 2 type II pts. and 4 type III pts. In case of type I pts., early onset in younger age and rapid progression of disease resulted in high mortality and morbidity compare to GD pts. among Caucasian esp. the most prevalent Ashkenazi Jewish population. Therefore, early diagnosis and effective treatment with Enzyme Replacement Therapy will be desirable for better management of Korean GD pts.