As one of the most breakthrough cutting-edge technologies in the biomedical field in recent years, organoid culture technology can use cells derived from, either (pluripotent) stem cells or tissue-derived differentiated/progenitor cells (foetal, neonatal, or adult) to form 3D multicellular structure organoids with self-organizing and recapitulating at least some features of the organ including tissue architecture or function abilities. Recently, organoids have been widely used in disease model construction, anti-cancer drug screening, gene or cell therapy, etc., providing an ideal model for basic biomedical research, drug development and clinical precision medicine, and has shown an important role in regenerative medicine.
Precision medicine is a medical paradigm founded on individual genetic information amalgamated with extensive clinical data to offer patients precise diagnoses and treatments. Genetic testing forms the cornerstone of accurate diagnosis, and skilled professionals in fields like clinical medicine, molecular biology, and bioinformatics play a crucial role in realizing the potential of precision medicine. This paper presents reference suggestions for the continuing education approach for relevant technical personnel. The main emphasis is on conducting routine face-to-face and hands-on training to enhance theoretical knowledge and professional skills. Secondly, there is a need to modify the training approach by reinforcing molecular biology, bioinformatics, and other courses, enhancing assessment methods, gradually implementing specialized training in precision medicine subspecialties, and ensuring effective clinical practice and management of precision medicine.
Liddle syndrome and Gordon syndrome are two rare single-gene inherited hypertension diseases. In patients≤40 years, the prevalence of Liddle syndrome is about 1% and Gordon syndrome is uncertain all over the word, for which is often misdiagnosed and mistreated. The therapies of those diseases are targeted at gene mutation sites, as well as combined with modified lifestyle, and can achieve satisfactory diseases control. This paper reports a patient who is diagnosed with Liddle syndrome and Gordon syndrome at the same time. We aimed to consolidate and improve the diagnosis and accurate treatment of those two diseases by sharing, studying and discussing together with clinical doctors.
Basing on development of medical model, new national diagnostic standard is interpreted according to three aspects: classification, diagnostic standard, and diagnostic contents. Tracheobronchial tuberculosis and tuberculous pleurisy are added into the classification. The value of molecular and pathological techniques for diagnosis of the pulmonary tuberculosis is emphasized. The status of drug-resistance is included in the diagnostic content. Two opinions are suggested: some practical methods such as diagnostic chemotherapy are indicated in some grassroots areas, while new molecular techniques for detection of DNA/RNA of mycobacteria and resistant mutation are encouraged in some suitable institutions.
The umbrella trial has received increasing attention in the design of clinical trials for oncology drugs in recent years. This trial design categorizes a single disease into multiple sub-types based on predictive biomarkers or other predictive factors, and simultaneously evaluates the efficacy of multiple targeted therapies. When compared with the traditional drug development model of phase Ⅰ, phaseⅡ, and phase Ⅲ randomized controlled trials, umbrella trials are a more scientifically rigorous trial design that can speed up drug evaluation to address the conflict between numerous untested drugs and diseases with a lack of effective treatment options. This article will focus on the concept, main characteristics, eligibility criteria, design and statistical considerations, ethical considerations, and future directions of umbrella trials, with the aim of providing methodological guidance for the design of clinical trials for oncology drugs.
ObjectiveTo explore the value of multidisciplinary team (MDT) discussion in the comprehensive treatment of HER-2 positive breast cancer.MethodThe clinical data of 2 patients with HER-2 positive breast cancer admitted to the Affiliated Hospital of Southwest Medical University after MDT discussions were analyzed retrospectively.ResultsCase 1 was a 32-year-old woman diagnosed with left breast non-special type invasive carcinoma at admission, cT2N1M0, stage ⅡB, WHO grade 2, ER (–), PR (–), HER-2 (+++), Ki-67 (+, 20%). After MDT discussion, the patient was treated with neoadjuvant chemotherapy for 6 cycles, and the efficacy evaluation was partial response, received left breast conserving surgery and axillary lymph node dissection (ALND), postoperative staging ypT1aN1ycM0, stage ⅡA, Miller-Payne grade 4, the patient was satisfied with the shape of breast, received radiotherapy and anti-HER-2 therapy after surgery. At present, there was no recurrence and metastasis during anti-HER-2 therapy. Case 2 was diagnosed with right breast non-special type invasive carcinoma at admission, cT3N0M0, stage ⅡB, WHO grade 3, ER (–), PR (–), HER-2 (+++), Ki-67 (+, 40%), local advanced breast cancer. After MDT discussion, the patient was treated with neoadjuvant chemotherapy for 2 cycles, and the efficacy evaluation was progressive disease. After the replacement of two neoadjuvant chemotherapy regimen, the efficacy evaluation was still progressive disease. Finally after MDT discussion, the patient received right breast mastectomy and ALND, postoperative staging ypT4bN1ycM0, stage ⅢB, Miller-Payne grade 1, received radiotherapy, adjuvant treatment with pyrotinib and capecitabine after surgery. The patient was followed up for 3 months by telephone, the patient did not follow the doctor’ instructions, no recurrence and metastasis was found in the review.ConclusionUnder the precision medical system, comprehensive treatment of breast cancer based on the MDT model could target patients’ disease characteristics, physical conditions, previous diagnosis and treatment, family situation, and other individual factors, formulate the best personal treatment plan for patients, and bring greater benefits to patients.
ObjectivesTo initially construct a scientific, reasonable and precision medicine technology value judgment framework suitable for China’s national conditions based on expert consultation method, so as to provide scientific value judgment system support for China's medical insurance decision-making.MethodsThe preliminary evaluation indicator system for precision medicine technology value was established by using literature analysis and expert consultation method, and the direct weighting method was used to determine the indicators weight.ResultsAfter two rounds of expert consultation, an indicator system suitable for the value judgment of precision medicine technology in China was constructed, including 5 primary indicators (health needs, health effects, economics, innovation and suitability) and 14 secondary indicators. Each indicator was weighted according to importance.ConclusionsA set of precision medicine value judgment indicator system suitable for China has been initially established, which lays a certain foundation for further measurement research of the indicator system and provides a scientific basis for medical insurance decision-making.
Precision medicine is an individualized clinical research model established according to gene, environment, lifestyle and other information. As an innovative method of clinical trials, the main scheme design breaks the barriers of traditional randomized controlled trials to the evaluation of targeted therapies in precision medicine and improves the efficiency of clinical research. This paper will systematically introduce the types, concepts and principles of the main scheme design of the new method of precision medicine clinical trial design, and summarize the advantages and limitations of the main scheme design combined with classic cases, aiming at providing scientific and rigorous methodological guidance and clinical practice experience for precision medicine scientific research design.