Rapid development of recently emerging precision medicine techniques represented by gene therapy has brought hope for the treatment of rare blinding eye diseases such as inherited retinal diseases (IRDs) for which there was no effective treatment previously. Although the globally growth of clinical trials for IRDs has increased rapidly over the past decade, due to the highly genetic and clinical phenotypic heterogeneity, as well as limited data on epidemiology and natural history of the disease, along with severe loss of vision function of majority patients for which the established measurements may not be appropriate, such studies lack standard outcome measurements and endpoints to asses clinical meaningful effectiveness, posing great challenges in terms of study design and evaluation of treatment efficacy, as well as clinical practice application. At present, there is no systematic nor standardized guidance on safety measures, clinical outcomes and endpoints of visual function for clinical trial design in IRDs. Therefore, in order to standardize the validated evaluation of IRDs clinical efficacy outcome measurements and endpoints, the Fundus Disease Group of Chinese Medical Association Ophthalmology Branch and Fundus Disease Committee of Chinese Medical Doctor Association Ophthalmology Branch organized domestic experts to put forward consensus and recommendations on standardizing outcome measurements and endpoints for clinical study design in IRDs, aiming to advance the study design of IRDs natural history research and clinical trials and to effectively evaluate disease progression and intervention efficacy. Along with the development of medical science and clinical trials, relevant content will be improved and updated accordingly.
Inherited retinal diseases (IRDs) are a group of severe retinal degenerative diseases leading to permanent visual impairment. IRDs are the major cause of irreversible blindness in children and working age groups. Gene therapy is a new clinical treatment method and currently the only clear and effective treatment for IRDs, while, there are still risks in clinical research and application. How to standardize perioperative management and reduce the potential risks of treatment is one of the keys to ensure the safety and effectiveness of treatment. However, there is no systematic and standardized guidance on the perioperative management for IRDs gene therapy. Therefore, in order to standardize the perioperative management, the Fundus Disease Group of Ophthalmology Society of Chinese Medical Association and Chinese Medical Doctor Association organized domestic experts to put forward standardized opinions on the perioperative management of IRDs gene therapy in China after repeated discussion and combined with domestic and foreign research experience, so as to provide clinicians with reference and application in clinical research and practice.
Membrane guided tissue regeneration is new biological concept. The basic theory of this concept includes the belief that during the healing process of wound, the different cells will show different speed of cell migration and regeneration in the wound. If an appropriate membrane being placed to form a mechanical barrier, so that only the needed cells can grow into that area and prevent others from going in, thus resulting in the creation of a guided area where the needed cells can undergo proliferation and differentiation under protection in completing an ideal tissue regeneration and repair. In this article, the experimental researches on the application of membrane guided tissue regeneration in the repair of tubular bone defects, skull defects and faciomaxillary defects were reviewed from literatures, and the degradable and non-degradable materials were introduced, particularly. The pros and cons of this method and the materials were evaluated. It is believed that this technique will push forward the progress in bone biology and reconstructive surgery.
An 89 years old male patient was admitted to the First Medical Center of Chinese PLA General Hospital due to chest tightness and shortness of breath for half a month. Severe aortic valve stenosis was found in post admission assessment. We proposed to perform transcatheter aortic valve replacement surgery. Preoperative evaluation showed severe distortion of the descending aorta. We used the double guide wire technique and a 14F long sheath to assist the stepwise balloon expansion, and successfully completed the valve implantation. For patients with severe tortuous aorta, how to successfully complete transcatheter aortic valve replacement, this case may provide some reference.
Adherence to reporting guidelines contributes to report methodology and outcomes of research distinctly and transparently. There are some checklists with specific study types related to surgery on the EQUATOR Network’s website. However, the IDEAL framework focuses on stepwise evaluation of surgical innovation through all stages with some key elements, which those existing guidelines may not mention. This likely results in the inaccuracy in reporting in studies attempting to follow the IDEAL recommendations and suggests a pressing need for IDEAL reporting guidelines. Considering these limitations, the IDEAL developed the IDEAL reporting guidelines between October 2018 and May 2019. The paper aimed to provide interpretation of IDEAL reporting guideline, and promote its understanding and use among Chinese researchers.
ObjectiveTo explore the clinical efficacy and safety of ultrasound-guided intra-articular injection of platelet-rich plasma (PRP) in the treatment of avascular necrosis of the femoral head.MethodsWe retrospectively collected and analyzed the clinical characteristics, imaging data, and clinical outcomes of patients with femoral head necrosis who received ultrasound-guided intra-articular PRP injection in the Department of Rehabilitation Medicine of Sun Yat-sen Memorial Hospital, Sun Yat-sen University between June 2019 and June 2020. All the patients received 4 injections at one-week intervals. The Visual Analogue Scale (VAS), Western Ontario and McMaster University Osteoarthritis Index (WOMAC), and Harris Hip Joint Function Scale (HHS) were evaluated before treatment and 1 month, 3 months, and 6 months after the first injections. Adverse events were recorded. The normally distributed data were presented as mean±standard deviation, and analyzed by one-way repeated measures analysis of variance; the non-normally distributed data were presented as median (lower quartile, upper quartile), and analyzed by Friedman test.ResultsA total of 29 patients were included. According to the Association Research Circulation Osseous classification standard, 2 patients were classified as stageⅠ, 11 as stageⅡ, 11 as stage Ⅲ, and 5 as stage Ⅳ. Before treatment and 1 month, 3 months, and 6 months after treatment, the VAS scores were 7.0 (5.5, 8.0), 4.0 (3.0, 5.0), 3.0 (2.0, 3.0), and 3.0 (2.0, 5.0), respectively, the WOMAC scores were 39.27±11.70, 28.34±8.08, 22.82±6.09, and 24.13±7.55, respectively, and the HHS were 46.0 (40.0, 64.0), 71.0 (57.5, 75.0), 78.0 (68.0, 80.5), and 78.0 (64.0, 80.0), respectively. The time effects in VAS (χ2=65.423, P<0.001), WOMAC (F=46.710, P<0.001), and HHS (χ2=66.347, P<0.001) were all statistically significant. There were significant differences in each index between the values 1 month, 3 months, and 6 months after treatment and those before treatment respectively, and there was also a significant difference in each index between the value 1 month after treatment and that 3 months after treatment (P<0.05). There was no significant difference in any indicator between the value 6 months after treatment and that 3 months after treatment (P>0.05). Significant difference was shown between the value 6 months after treatment and that 1 month after treatment in WOMAC (P=0.016), but not in VAS or HHS (P>0.05). No obvious adverse event was reported during the follow-up period.ConclusionsUltrasound-guided intra-articular PRP injection can effectively alleviate the pain and improve the hip joint function of patients with femoral head necrosis for at least 6 months. However, randomized controlled studies with a larger sample size and longer-term follow-up are needed in the future to confirm the efficacy and safety of PRP injection in femoral head necrosis.
ObjectiveTo systematically review the quality of evidence-based guidelines (EBGs) on medication therapy for neonatal bacterial meningitis, and compare differences and similarities of the drugs recommended, in order to provide references for clinical application. MethodsDatabases such as the TRIP, PubMed, CNKI, VIP, WanFang, CBM, National Guideline Clearinghouse and Guidelines International Network were searched to collect evidence-based guidelines on medication therapy for neonatal bacterial meningitis. Methodological quality of included studies was assessed according to the AGREE Ⅱ instrument, and the differences and similarities among recommendations were compared. ResultsA total of 4 EBGs were included. Among them, one guideline was developed by the America and three guidelines were by the UK. Only one guideline was developed specially for neonates, while the rest were for neonates and children of different ages. According to the AGREE Ⅱ instrument, "scope and purpose", "stakeholder involvement", "rigor of development", "clarity and presentation", "applicability" and "editorial independence" were scored more than 60%. The recommendations of different guidelines were basically the same, only with conflicts in some areas. ConclusionAlthough most guidelines concerning neonatal bacterial meningitis are of high quality, grading levels of evidence and strength of recommendation should be unified.
Currently, there is a lack of clarity and standardization regarding the implementation details of interventions in traditional Chinese medicine clinical practice guidelines (CPGs). This in methodological guidance for standardizing the implementation prescription adversely impacts the quality of implementation and hinders the clinical application rate of recommendations. Through in-depth analysis of implementation prescription of evidence-based CPGs in traditional Chinese medicine, we identified the challenges associated with standardization. In response, we propose enhancing the technical specifications of implementation prescriptions, advocating for improved formulation processes, diverse reporting approaches, and standardizedological guidelines. These recommendations aim to serve as a methodological reference and guidance for clinical practice guideline developers.
Objective To review the application of three-dimensional (3D) printing patient-specific cutting guides (PSCG) in open-wedge high tibial osteotomy (OWHTO). Methods The domestic and foreign literature about the use of 3D printing PSCG to assist the OWHTO in recent years was reviewed, and the effectiveness of different types of 3D printing PSCG to assist OWHTO was summarized. Results Many scholars design and use different 3D printing PSCGs to confirm the precise positioning of the osteotomy site (the bone surface around the cutting line, the “H” point of the proximal tibia, the internal and external malleolus fixators, etc.) and the correction angle (the pre-drilled holes, the wedge-shaped filling blocks, the angle-guided connecting rod, etc.) during operation, and all of them achieve good effectiveness. ConclusionCompared with conventional OWHTO, 3D printing PSCG assisted OWHTO has many obvious advantages, such as shortening the operation time, and the frequency of fluoroscopy, and being closer to the expected preoperative correction, etc. However, the effectiveness between different 3D printing PSCGs still need to be discussed in the follow-up studies.
Objective To explore the application of personalized guide plate combined with intraoperative real-time navigation in repairing of mandibular defect using fibula muscle flap, providing the basis for the precise repair and reconstruction of mandible. Methods The clinical data of 12 patients (9 males and 3 females) aged from 23 to 71 years (mean, 55.5 years) between July 2019 and December 2021 were recorded. These patients were diagnosed as benign or malignant mandibular tumors, including 2 cases of ameloblastoma, 6 cases of squamous cell carcinoma, 2 cases of osteosarcoma, 1 case of adenoid cystic carcinoma, and 1 case of squamous carcinoma. All patients were treated with mandibular amputation, and then repaired by double-stacked three-segment fibula muscle flap. Preoperative virtual design scheme and guide plate were performed. During the operation, personalized guide plate combined with real-time navigation was used for fibular osteotomy and shaping. Thin-slice CT examination was performed at 2-3 weeks after operation, and was fitted with the preoperative virtual design scheme. The difference between the distance of bilateral mandibular angles relative to the reference plane in three-dimensional directions (left-right, vertical, and anterior-posterior) and the difference of the medial angle of the lower edge of the mandible reconstructed by fibula were measured, and the mean error of chromatographic fitting degree was calculated. Results The guide plate and navigation were applied well, and the fibula shaping and positioning were accurate. The fibula muscle flap survived, the incision healed well, and the occlusal relationship was good. All 12 patients were followed up 1-29 months, with an average of 17 months. There was no significant difference on the distance of bilateral mandibular angles relative to the reference plane in the left-right [(?0.24±1.35) mm; t=?0.618, P=0.549], vertical [?0.85 (?1.35, 1.40) mm; Z=?0.079, P=0.937], and anterior-posterior [(?0.46±0.78) mm; t=?2.036, P=0.067] directions. The difference of the medial angle of the lower edge of the mandible reconstructed by fibula was also not significant [(?1.35±4.34)°; t=?1.081, P=0.303)]. Postoperative CT and preoperative virtual design fitting verified that there was no significant difference in the change of the mandibular angle on both sides, and the average error was (0.47±1.39) mm. ConclusionThe personalized guide combined with intraoperative real-time navigation improves the accuracy of peroneal muscle flap reconstruction of the mandible, reduces the complications, and provides a preliminary basis for the application of visual intraoperative navigation in fibula muscle flap reconstruction of the mandible.