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Maria Domenica Cappellini John B. Porter Vip Viprakasit Ali T. Taher 《Blood reviews》2018,32(4):300-311
Beta-thalassaemia causes defective haemoglobin synthesis leading to ineffective erythropoiesis, chronic haemolytic anaemia, and subsequent clinical complications. Blood transfusion and iron chelation allow long-term disease control, and haematopoietic stem cell transplantation offers a potential cure for some patients. Nonetheless, there are still many challenges in the management of beta-thalassaemia. The main treatment option for most patients is supportive care; furthermore, the long-term efficacy and safety of current therapeutic strategies are limited and adherence is suboptimal. An increasing understanding of the underlying molecular and cellular disease mechanisms plus an awareness of limitations of current management strategies are driving research into novel therapeutic options. Here we provide an overview of the current pathophysiology, clinical manifestations, and global burden of beta-thalassaemia. We reflect on what has been achieved to date, describe the challenges associated with currently available therapy, and discuss how these issues might be addressed by novel therapeutic approaches in development. 相似文献
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Muhammad Jamil Omar Ahmad Kian Keong Poh Choon Hwai Yap 《Ultrasound in medicine & biology》2017,43(7):1314-1330
Current Doppler echocardiography quantification of mitral regurgitation (MR) severity has shortcomings. Proximal isovelocity surface area (PISA)-based methods, for example, are unable to account for the fact that ultrasound Doppler can measure only one velocity component: toward or away from the transducer. In the present study, we used ultrasound-based computational fluid dynamics (Ub-CFD) to quantify mitral regurgitation and study its advantages and disadvantages compared with 2-D and 3-D PISA methods. For Ub-CFD, patient-specific mitral valve geometry and velocity data were obtained from clinical ultrasound followed by 3-D CFD simulations at an assumed flow rate. We then obtained the average ratio of the ultrasound Doppler velocities to CFD velocities in the flow convergence region, and scaled CFD flow rate with this ratio as the final measured flow rate. We evaluated Ub-CFD, 2-D PISA and 3-D PISA with an in vitro flow loop, which featured regurgitation flow through (i) a simplified flat plate with round orifice and (ii) a 3-D printed realistic mitral valve and regurgitation orifice. The Ub-CFD and 3-D PISA methods had higher precision than the 2-D PISA method. Ub-CFD had consistent accuracy under all conditions tested, whereas 2-D PISA had the lowest overall accuracy. In vitro investigations indicated that the accuracy of 2-D and 3-D PISA depended significantly on the choice of aliasing velocity. Evaluation of these techniques was also performed for two clinical cases, and the dependency of PISA on aliasing velocity was similarly observed. Ub-CFD was robustly accurate and precise and has promise for future translation to clinical practice. 相似文献
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Luis Fernando Calimano-Ramirez Taher Daoud Dheeraj Reddy Gopireddy Ajaykumar C Morani Rebecca Waters Kazim Gumus Albert Russell Klekers Priya R Bhosale Mayur K Virarkar 《World journal of gastroenterology : WJG》2022,28(40):5827-5844
Acinar cell carcinoma (ACC) is a rare pancreatic malignancy with distinctive clinical, molecular, and morphological features. The long-term survival of ACC patients is substantially superior to that of pancreatic adenocarcinoma patients. As there are no significant patient series about ACCs, our understanding of this illness is mainly based on case reports and limited patient series. Surgical resection is the treatment of choice for patients with the disease restricted to one organ; however, with recent breakthroughs in precision medicine, medicines targeting the one-of-a-kind molecular profile of ACC are on the horizon. There are no standard treatment protocols available for people in which a total surgical resection to cure the condition is not possible. As a result of shared genetic alterations, ACCs are chemosensitive to agents with activity against pancreatic adenocarcinomas and colorectal carcinomas. The role of neoadjuvant or adjuvant chemoradiotherapy has not been established. This article aims to do a comprehensive literature study and present the most recent information on acinar cell cancer. 相似文献
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Farrukh Aslam Khalid Muhammad Younas Mehrose Muhammad Saleem Muhammad Amin Yousaf Abdul Malik Mujahid Saif Ur Rehman Sania Ahmad Moazzam Nazeer Tarar 《Burns : journal of the International Society for Burn Injuries》2019,45(1):69-75
The treatment of keloid and hypertrophic scar is challenging with no universally accepted mode for permanent ablation. Conventional therapies yield unpredictable results, significant complications and require elaborate hardware.
Objective
The objective was to establish the safety and efficacy of intralesional 5-fluorouracil (5-FU) for the treatment of keloids and hypertrophic scars.Study design
Randomized controlled trial (RCT).Place and duration
It was conducted at the Jinnah Burn and Reconstructive Surgery Center/Allama Iqbal Medical College, Lahore, Pakistan from May 2012 to March 2013.Subjects and methods
We included 120 patients divided in two groups. The group A patients received intralesional triamcinolone acetonide (TAC) and the group B patients received both 5-FU and TAC. 8 injections at a week interval were given and patients were evaluated at the start of treatment and then at 4th and at 8th week during the treatment and then 4 weeks after the end of treatment. Patents were assessed for mean reduction in scar height, efficacy and complications.Results
Total of 108 patients completed the study. The mean reduction in the scar height in group B (5-FU + TAC) 1.144 + .4717 was markedly better than that of group A (TAC alone) 1.894 + 1.0751 (t = 4.781, p = .000). The efficacy (defined previously as >50% reduction in initial scar height) was superior in group B 44 (77.2%) than that of group A 25 (49.0% (X2 = 9.260, p = .002). Recurrence was seen in 39.2% (20) of patients of the group A while in only 17.5% (10) of the cases of group B (P = 0.012). Mean follow up was of 22 months.Conclusion
5-FU + TAC is safe, easy to administer and effective treatment for problematic scars and has the lower rate of recurrence on larger follow up. 相似文献99.
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