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Emerging DNA & RNA editing strategies for the treatment of epidermolysis bullosa. 治疗大疱性表皮松解症的新兴 DNA 和 RNA 编辑策略。
Pub Date : 2024-12-01 Epub Date: 2024-08-18 DOI: 10.1080/09546634.2024.2391452
Ulrich Koller, Johann W Bauer

Background: Epidermolysis bullosa (EB) is a clinically-heterogeneous genodermatosis with severe manifestations in the skin and other organs. The significant burden this condition places on patients justifies the development of gene therapeutic strategies targeting the genetic cause of the disease.

Methods: Emerging RNA and DNA editing tools have shown remarkable advances in efficiency and safety. Applicable both in ex vivo- and in vivo settings, these gene therapeutics based on gene replacement or editing are either at the pre-clinical or clinical stage.

Results: The recent landmark FDA approvals for gene editing based on CRISPR/Cas9, along with the first FDA-approved redosable in vivo gene replacement therapy for EB, will invigorate ongoing research efforts, increasing the likelihood of achieving local cure via CRISPR-based technologies in the near future.

Conclusions: This review discusses the status quo of current gene therapeutics that act at the level of RNA or DNA, all with the common aim of improving the quality of life for EB patients.

背景:大疱性表皮松解症(EB)是一种临床异质性遗传性皮肤病,在皮肤和其他器官有严重的表现。这种疾病给患者带来的沉重负担证明了针对该病遗传病因的基因治疗策略的发展是合理的:方法:新兴的 RNA 和 DNA 编辑工具在效率和安全性方面取得了显著进步。这些基于基因置换或编辑的基因疗法适用于体内外环境,目前处于临床前或临床阶段:最近,美国食品与药物管理局批准了基于CRISPR/Cas9的基因编辑技术,以及美国食品与药物管理局批准的首个用于EB的可重复使用的体内基因置换疗法,这两项具有里程碑意义的举措将为当前的研究工作注入新的活力,在不久的将来,通过基于CRISPR的技术实现局部治愈的可能性将大大增加:本综述讨论了目前在 RNA 或 DNA 水平上发挥作用的基因疗法的现状,所有这些疗法的共同目标都是改善 EB 患者的生活质量。
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引用次数: 0
The adjuvant treatment role of ω-3 fatty acids by regulating gut microbiota positively in the acne vulgaris. ω-3脂肪酸通过积极调节肠道微生物群对痤疮的辅助治疗作用。
Pub Date : 2024-12-01 Epub Date: 2024-01-02 DOI: 10.1080/09546634.2023.2299107
Yaxin Huang, Fuming Liu, Jindong Lai, Shiyu Jiang, Xiaoqi Tan, Lingna Chen, Yong Xu, Xia Xiong, Yongqiong Deng

Objectives:We aimed to explore the potential role of omega-3 (ω-3) fatty acids on acne vulgaris by modulating gut microbiota.Materials and Methods:We randomly divided the untreated acne patients into two groups with or without ω-3 fatty acids intervention for 12 weeks. The Sprague Dawley (SD) rats with acne model were given isotretinoin, ω-3 fatty acids or their combination respectively. Then the colonic contents samples of the drug intervention SD rats were transferred to the pseudo sterile rats with acne model. The severity of the disease was assessed by the Global Acne Grading System (GAGS) score of the patients, and the swelling rate of auricle and the pathological section of the rat with acne model. The 16S rDNA gene sequencing was performed to detect the alteration of the gut microbiota.Results:ω-3 fatty acids could increase the diversity of the gut microbiota and regulate the flora structure positively both in the patients and rats, increase the abundance of butyric acid producing bacteria and GAGS score in the patients, and alleviate the inflammation and comedones of rats.Conclusion:Supplementation of ω-3 fatty acids could alleviate the inflammation of acne vulgaris by increasing the abundance of butyric acid producing bacteria.

材料与方法:我们将未经治疗的痤疮患者随机分为两组,分别给予或不给予ω-3脂肪酸干预,为期12周。分别给予痤疮模型大鼠异维A酸、ω-3脂肪酸或它们的组合。然后将药物干预 SD 大鼠的结肠内容物样本转移到假性无菌痤疮模型大鼠体内。根据痤疮患者的全球痤疮分级系统(GAGS)评分、耳廓肿胀率和痤疮模型大鼠的病理切片评估疾病的严重程度。结果:ω-3 脂肪酸能增加患者和大鼠肠道微生物群的多样性并积极调节菌群结构,提高患者丁酸生成菌的丰度和 GAGS 评分,缓解大鼠的炎症和粉刺。
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引用次数: 0
Complete response of extensive alopecia areata refractory to baricitinib after five months of treatment with upadacitinib. 巴利昔尼难治的广泛性斑秃患者在接受奥达帕替尼治疗五个月后完全康复。
Pub Date : 2024-12-01 Epub Date: 2024-01-19 DOI: 10.1080/09546634.2024.2304630
Francisco Javier De la Torre-Gomar, Juan Pablo Velasco-Amador, Álvaro Prados-Carmona, Ricardo Ruiz-Villaverde
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引用次数: 0
Injection site reactions after dupilumab or tralokinumab for atopic dermatitis. 杜匹单抗或曲妥珠单抗治疗特应性皮炎后的注射部位反应。
Pub Date : 2024-12-01 Epub Date: 2024-01-19 DOI: 10.1080/09546634.2024.2304027
Fabrizio Martora, Cataldo Patruno, Silvia D'Ascenzo, Maddalena Napolitano

Background: Injection site reaction (ISR) is a local phenomenon defined as a constellation of symptoms, including swelling, erythema, pruritus, and pain around the site of injection.Objective: ISR is reported as a frequent adverse event after subcutaneous injection (SCI) of several biologics.Methods: We performed an observational real-life study to compare dupilumab and tralokinumab as regards ISR, analysing frequency, duration and intensity of symptoms related to SCI. From January 2023 to June 2023, we enrolled adult patients affected by moderate to severe AD and being on dupilumab or tralokinumab treatment. A 12 items questionnaire was administered to all enrolled patients.Results and conclusions: Three hundred and ninety-two patients were included. ISR was a frequent occurrence in both the treatment groups, with tralokinumab causing ISR more frequently than dupilumab. However, the reactions were generally mild and no patient stopped therapy.

背景:注射部位反应(ISR)是一种局部现象,定义为注射部位周围出现肿胀、红斑、瘙痒和疼痛等一系列症状:据报道,注射部位反应是几种生物制剂皮下注射(SCI)后经常发生的不良反应:我们进行了一项观察性真实生活研究,比较了杜匹单抗和曲妥珠单抗的 ISR,分析了与 SCI 相关的症状的频率、持续时间和强度。从2023年1月至2023年6月,我们招募了受中度至重度AD影响、正在接受dupilumab或曲妥珠单抗治疗的成年患者。我们对所有入组患者进行了12项问卷调查:共纳入 392 名患者。两个治疗组都经常出现ISR,其中曲妥珠单抗引起ISR的频率高于杜匹单抗。不过,这些反应一般都很轻微,没有患者停止治疗。
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引用次数: 0
Oral abrocitinib in the treatment of granuloma annulare: a case report. 口服阿罗西替尼治疗环状肉芽肿:病例报告。
Pub Date : 2024-12-01 Epub Date: 2024-02-05 DOI: 10.1080/09546634.2024.2313090
Wenyan Liu, Weifeng Chen, Xin Tian, Yihui Yu, Junhui Zhu, Jingyao Liang, Xibao Zhang

Aim: To evaluate the therapeutic efficacy and safety of JAK inhibitor abrocitinib in patients with localized granuloma annulare (GA) and to review the available cases documented in English.Methods: We presented a patient who had a persistent, localized granuloma anulare (GA) for one year and did not respond to traditional therapies. This patient was treated with oral abrocitinib at a dosage of 150 mg daily.Results: After 6 weeks of treatment with abrocitinib, the patient exhibited notable symptom improvement with no new lesions. No adverse events or recurrences were reported during the 5-month follow-up period.Conclusions: Abrocitinib may be a promising and safe treatment option for patients with localized GA who do not respond to traditional therapies.

目的:评估JAK抑制剂阿洛西替尼对局部性环状肉芽肿(GA)患者的疗效和安全性,并回顾现有的英文病例:我们介绍了一名持续局部肉芽肿(GA)一年且对传统疗法无反应的患者。该患者接受了每日150毫克剂量的阿罗西替尼口服治疗:结果:阿昔替尼治疗6周后,患者症状明显改善,且没有出现新的病灶。在5个月的随访期间,没有出现不良反应或复发:对于传统疗法无效的局部GA患者来说,阿罗西替尼可能是一种有前景且安全的治疗选择。
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引用次数: 0
Impairment in work and activities of daily life in patients with psoriasis: results of the prospective BioCAPTURE registry. 银屑病患者的工作和日常生活障碍:前瞻性 BioCAPTURE 登记结果。
Pub Date : 2024-12-01 Epub Date: 2024-01-23 DOI: 10.1080/09546634.2024.2304025
Tamara W van Hal, Juul M P A van den Reek, Mark H Wenink, Marisol E Otero, Paul M Ossenkoppele, Marcellus D Njoo, Annet Oostveen, Bas Peters, Milan Tjioe, Else N Kop, John E M Körver, Sharon R P Dodemont, Marloes M Kleinpenning, Maartje A M Berends, Wendelien R Veldkamp, Martijn B A van Doorn, Johannes M Mommers, Robert-Jan Lindhout, Astrid L A Kuijpers, Paula P van Lümig, C Els J de Jonge, Ron A Tupker, Judith Hendricksen, Romy R Keijsers, Frank H J van den Hoogen, Johanna E Vriezekolk, Elke M G J de Jong

Background: Little is known about the extent of impairments in work and activities of daily life (ADL) in patients with psoriasis, and the influence of contextual factors such as disease-related characteristics and treatment. Therefore, this study aimed to assess these impairments in patients with psoriasis who started using biologicals/small molecule inhibitors.Methods: Using data from the prospective BioCAPTURE registry, we collected patient, disease, and treatment parameters, as well as work/ADL impairments at baseline, 6 and 12 months. Changes in impairment parameters and correlations between impairment and patient/disease characteristics were assessed using generalized estimating equations.Results: We included 194 patients in our analysis. After biological initiation, disease activity decreased significantly (PASI 11.2 at baseline versus 3.9 at 12 months, p < 0.001). Work-for-pay in this cohort was lower than in the Dutch general population (53% versus 67%, p = 0.01). In patients who had work-for-pay, presenteeism improved over time (5% at baseline versus 0% at 12 months, p = 0.04). Up to half of the patients reported impairments in ADL, which did not change over time. Associations between impairments and contextual factors varied, but all impairments were associated with worse mental/physical general functioning.Conclusion: Patients with psoriasis using biologicals are less likely to have work-for-pay. Treatment improves the work productivity of employed patients, but we were unable to detect changes in ADL performance.

背景:人们对银屑病患者在工作和日常生活活动(ADL)中的障碍程度以及疾病相关特征和治疗等环境因素的影响知之甚少。因此,本研究旨在评估开始使用生物制剂/小分子抑制剂的银屑病患者的这些障碍:利用前瞻性 BioCAPTURE 登记处的数据,我们收集了患者、疾病和治疗参数,以及基线、6 个月和 12 个月的工作/ADL 损伤情况。我们使用广义估计方程评估了损伤参数的变化以及损伤与患者/疾病特征之间的相关性:我们分析了 194 名患者。开始使用生物制剂后,疾病活动明显减少(基线时 PASI 为 11.2,12 个月时为 3.9,P = 0.01)。在有偿工作的患者中,旷工现象随着时间的推移有所改善(基线时为 5%,12 个月时为 0%,P = 0.04)。多达半数的患者报告了日常活动能力受损的情况,但这些情况并没有随着时间的推移而发生变化。损伤与环境因素之间的关系各不相同,但所有损伤都与精神/身体总体功能的恶化有关:结论:使用生物制剂的银屑病患者不太可能从事有报酬的工作。治疗可提高就业患者的工作效率,但我们无法检测到日常活动能力的变化。
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引用次数: 0
Effectiveness and safety of interferon α-2a combined with phototherapy for patients with early-stage mycosis fungoides - a single-arm prospective study in 13 patients. 干扰素α-2a联合光疗对早期真菌病患者的有效性和安全性--对13名患者进行的单臂前瞻性研究。
Pub Date : 2024-12-01 Epub Date: 2024-05-16 DOI: 10.1080/09546634.2024.2350231
Hongbin Song, Zhonghui Hu, Shiyu Zhang, Lu Yang, Jindi Feng, Lu Lu, Yuehua Liu, Tao Wang

Background: Mycosis fungoides (MF) is the most common type of cutaneous T-cell lymphoma.

Objectives: This study was conducted to evaluate efficacy and safety of interferon (IFN) α-2a combined with phototherapy for early-stage MF.

Methods: Thirteen patients with early-stage MF received subcutaneous injections of IFN α-2a at 3 million IU combined with phototherapy three times per week for 6 months. Treatment efficacy was measured by changes in body surface area (BSA) score and modified severity-weighted assessment tool (mSWAT) score at 1, 3, and 6 months after treatment. Histopathologic examinations of skin lesions were performed before and after treatment.

Results: After 3 months of treatment, all 13 patients achieved a partial response, and BSA and mSWAT scores were significantly lower than those at baseline (p < 0.001). After 6 months, BSA and mSWAT scores were significantly lower than those at baseline (p < 0.001) and after 3 months (p < 0.05). Eleven patients achieved complete remission and two patients achieved a partial response (overall response rate, 100%). Histopathologic examination showed a significant decrease in the number of atypical lymphocytes in both epidermis and dermis. No severe adverse effects occurred.

Conclusion: IFN α-2a in combination with phototherapy may be an effective and safe alternative modality for early-stage MF.

背景:真菌病(MF)是最常见的皮肤T细胞淋巴瘤:真菌病(MF)是最常见的皮肤T细胞淋巴瘤:本研究旨在评估干扰素(IFN)α-2a联合光疗治疗早期MF的有效性和安全性:13名早期中耳炎患者接受了每周三次、每次300万IU的IFN α-2a皮下注射和光疗,为期6个月。治疗效果通过治疗后 1、3 和 6 个月体表面积 (BSA) 评分和改良的严重程度加权评估工具 (mSWAT) 评分的变化来衡量。治疗前后对皮损进行了组织病理学检查:IFN α-2a联合光疗可能是治疗早期MF的一种有效而安全的替代方法。
{"title":"Effectiveness and safety of interferon α-2a combined with phototherapy for patients with early-stage mycosis fungoides - a single-arm prospective study in 13 patients.","authors":"Hongbin Song, Zhonghui Hu, Shiyu Zhang, Lu Yang, Jindi Feng, Lu Lu, Yuehua Liu, Tao Wang","doi":"10.1080/09546634.2024.2350231","DOIUrl":"https://doi.org/10.1080/09546634.2024.2350231","url":null,"abstract":"<p><p><b>Background:</b> Mycosis fungoides (MF) is the most common type of cutaneous T-cell lymphoma.</p><p><p><b>Objectives:</b> This study was conducted to evaluate efficacy and safety of interferon (IFN) α-2a combined with phototherapy for early-stage MF.</p><p><p><b>Methods:</b> Thirteen patients with early-stage MF received subcutaneous injections of IFN α-2a at 3 million IU combined with phototherapy three times per week for 6 months. Treatment efficacy was measured by changes in body surface area (BSA) score and modified severity-weighted assessment tool (mSWAT) score at 1, 3, and 6 months after treatment. Histopathologic examinations of skin lesions were performed before and after treatment.</p><p><p><b>Results:</b> After 3 months of treatment, all 13 patients achieved a partial response, and BSA and mSWAT scores were significantly lower than those at baseline (<i>p</i> < 0.001). After 6 months, BSA and mSWAT scores were significantly lower than those at baseline (<i>p</i> < 0.001) and after 3 months (<i>p</i> < 0.05). Eleven patients achieved complete remission and two patients achieved a partial response (overall response rate, 100%). Histopathologic examination showed a significant decrease in the number of atypical lymphocytes in both epidermis and dermis. No severe adverse effects occurred.</p><p><p><b>Conclusion:</b> IFN α-2a in combination with phototherapy may be an effective and safe alternative modality for early-stage MF.</p>","PeriodicalId":94235,"journal":{"name":"The Journal of dermatological treatment","volume":"35 1","pages":"2350231"},"PeriodicalIF":0.0,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140961016","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
A multicenter, double-blind, randomized, parallel-group, active-controlled, phase 3 clinical trial to compare the effectiveness and safety of two botulinum toxin type A formulations for improving moderate to severe glabellar wrinkles in Asians. 一项多中心、双盲、随机、平行组、主动控制的 3 期临床试验,比较两种 A 型肉毒毒素制剂对改善亚洲人中度至重度睑部皱纹的有效性和安全性。
Pub Date : 2024-12-01 Epub Date: 2024-06-16 DOI: 10.1080/09546634.2024.2359511
Sun Young Choi, Young Gue Koh, Yang Won Lee, Hyung Seok Son, Yi Na Yoon, Gyeonghoon Kim, Chonghyun Won, Hyesoo Cho, Joo-Sun Son, Eun-Kyoung Kim, Beom Joon Kim

Botulinum toxin type A (BoNT-A) was first isolated in 1946, and since then, several formulations have been developed and widely used to treat wrinkles by inducing muscle paralysis. This multicenter, double-blind, randomized, parallel-group, active-controlled phase 3 clinical trial was designed to evaluate the efficacy and safety of a newly developed BoNT-A formulation, BMI2006, in improving moderate to severe glabellar wrinkles and to compare with existing onabotulinumtoxin A (OBoNT) injections. A total of 276 subjects were enrolled and received 20 units of the randomized material, which was intramuscularly injected into five different locations on the forehead. The primary endpoint, assessed at 4 weeks, showed no statistically significant difference in the improvement rate of glabellar wrinkles between the two groups, with BMI2006 demonstrating non-inferiority to comparator BoNT-A. Secondary endpoints, evaluated by both treating investigators and independent investigators, also exhibited similar improvement rates throughout the study period. Both groups reported high levels of satisfaction with no statistical difference between the two groups. Safety evaluations indicated mild and transient adverse events, with no serious reactions observed. In conclusion, BMI2006 is an effective and safe BoNT-A for treating glabellar wrinkles with an expected duration of action between 8 and 12 weeks.

A 型肉毒杆菌毒素(BoNT-A)于 1946 年首次被分离出来,从那时起,通过诱导肌肉麻痹来治疗皱纹的几种配方已被开发并广泛使用。这项多中心、双盲、随机、平行组、主动对照的 3 期临床试验旨在评估新开发的 BoNT-A 制剂 BMI2006 在改善中度至重度眉间皱纹方面的疗效和安全性,并与现有的奥博毒素 A (OBoNT) 注射进行比较。共有 276 名受试者参加了这项研究,他们接受了 20 个单位的随机材料,并将其肌肉注射到前额的五个不同位置。主要终点在4周时进行评估,结果显示,两组受试者的眉间皱纹改善率在统计学上没有显著差异,BMI2006的效果不劣于对比药物BoNT-A。由治疗研究人员和独立研究人员评估的次要终点在整个研究期间也显示出相似的改善率。两组患者的满意度都很高,没有统计学差异。安全评估显示,该疗法有轻微和短暂的不良反应,没有发现严重的不良反应。总之,BMI2006 是一种有效、安全的治疗睑部皱纹的 BoNT-A,预计疗程为 8 至 12 周。
{"title":"A multicenter, double-blind, randomized, parallel-group, active-controlled, phase 3 clinical trial to compare the effectiveness and safety of two botulinum toxin type A formulations for improving moderate to severe glabellar wrinkles in Asians.","authors":"Sun Young Choi, Young Gue Koh, Yang Won Lee, Hyung Seok Son, Yi Na Yoon, Gyeonghoon Kim, Chonghyun Won, Hyesoo Cho, Joo-Sun Son, Eun-Kyoung Kim, Beom Joon Kim","doi":"10.1080/09546634.2024.2359511","DOIUrl":"10.1080/09546634.2024.2359511","url":null,"abstract":"<p><p>Botulinum toxin type A (BoNT-A) was first isolated in 1946, and since then, several formulations have been developed and widely used to treat wrinkles by inducing muscle paralysis. This multicenter, double-blind, randomized, parallel-group, active-controlled phase 3 clinical trial was designed to evaluate the efficacy and safety of a newly developed BoNT-A formulation, BMI2006, in improving moderate to severe glabellar wrinkles and to compare with existing onabotulinumtoxin A (OBoNT) injections. A total of 276 subjects were enrolled and received 20 units of the randomized material, which was intramuscularly injected into five different locations on the forehead. The primary endpoint, assessed at 4 weeks, showed no statistically significant difference in the improvement rate of glabellar wrinkles between the two groups, with BMI2006 demonstrating non-inferiority to comparator BoNT-A. Secondary endpoints, evaluated by both treating investigators and independent investigators, also exhibited similar improvement rates throughout the study period. Both groups reported high levels of satisfaction with no statistical difference between the two groups. Safety evaluations indicated mild and transient adverse events, with no serious reactions observed. In conclusion, BMI2006 is an effective and safe BoNT-A for treating glabellar wrinkles with an expected duration of action between 8 and 12 weeks.</p>","PeriodicalId":94235,"journal":{"name":"The Journal of dermatological treatment","volume":"35 1","pages":"2359511"},"PeriodicalIF":0.0,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141332799","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Systemic treatment of recessive dystrophic epidermolysis bullosa with mesenchymal stromal cells: a scoping review of the literature and conclusions for future clinical research. 间充质基质细胞治疗隐性萎缩性表皮松解症:文献综述及未来临床研究结论。
Pub Date : 2024-12-01 Epub Date: 2024-11-17 DOI: 10.1080/09546634.2024.2419931
Elke Niebergall-Roth, Kathrin Dieter, Markus H Frank, Mark A Kluth

Background: The ability of mesenchymal stromal cells (MSCs) to facilitate regenerative responses in inflamed and injured tissues, coupled with preclinical data suggesting potential to restore defective collagen VII at the dermo-epidermal junction, has raised the hope that MSCs may provide an effective disease-modifying therapy for patients suffering from recessive dystrophic epidermolysis bullosa (RDEB).

Methods: We present a descriptive analysis of the clinical research on systemic MSC administration to RDEB patients available in PubMed, including six early-phase studies and one case report, involving 59 patients who received 1-3 intravenous infusions of MSCs from various sources.

Results: Based on 133 MSC infusions, a total of 44 mostly mild adverse events were reported as definitely, possibly or likely related to the study treatment, only two of which led to treatment discontinuation. Improvements were seen in skin manifestations, disease activity, pain, pruritus and quality of life, with considerable heterogeneity in reported outcome variables and measurement tools between studies, and large inter-patient variability within studies.

Conclusions: Although the current evidence base is limited, reflecting the typical challenges of clinical research in rare diseases, the reported results suggest potential treatment benefits for patients and provide a rationale for continuing to pursue this therapeutic approach.

背景:间充质基质细胞(MSCs)能够促进炎症和损伤组织的再生反应,而且临床前数据表明间充质基质细胞具有恢复真皮表皮交界处缺陷胶原蛋白VII的潜力,这使人们希望间充质基质细胞能够为隐性萎缩性表皮松解症(RDEB)患者提供一种有效的疾病调节疗法:我们对PubMed上有关RDEB患者全身性间充质干细胞应用的临床研究进行了描述性分析,其中包括6项早期研究和1项病例报告,共有59名患者接受了1-3次不同来源间充质干细胞的静脉输注:在133例间叶干细胞输注中,共有44例轻微不良事件被报告为肯定、可能或很可能与研究治疗有关,其中只有2例导致治疗中止。皮肤表现、疾病活动、疼痛、瘙痒和生活质量均有所改善,但不同研究间报告的结果变量和测量工具存在很大的异质性,研究内部患者间的差异也很大:尽管目前的证据基础有限,反映了罕见病临床研究的典型挑战,但所报告的结果表明治疗可能会给患者带来益处,并为继续采用这种治疗方法提供了依据。
{"title":"Systemic treatment of recessive dystrophic epidermolysis bullosa with mesenchymal stromal cells: a scoping review of the literature and conclusions for future clinical research.","authors":"Elke Niebergall-Roth, Kathrin Dieter, Markus H Frank, Mark A Kluth","doi":"10.1080/09546634.2024.2419931","DOIUrl":"10.1080/09546634.2024.2419931","url":null,"abstract":"<p><p><b>Background:</b> The ability of mesenchymal stromal cells (MSCs) to facilitate regenerative responses in inflamed and injured tissues, coupled with preclinical data suggesting potential to restore defective collagen VII at the dermo-epidermal junction, has raised the hope that MSCs may provide an effective disease-modifying therapy for patients suffering from recessive dystrophic epidermolysis bullosa (RDEB).</p><p><p><b>Methods:</b> We present a descriptive analysis of the clinical research on systemic MSC administration to RDEB patients available in PubMed, including six early-phase studies and one case report, involving 59 patients who received 1-3 intravenous infusions of MSCs from various sources.</p><p><p><b>Results:</b> Based on 133 MSC infusions, a total of 44 mostly mild adverse events were reported as definitely, possibly or likely related to the study treatment, only two of which led to treatment discontinuation. Improvements were seen in skin manifestations, disease activity, pain, pruritus and quality of life, with considerable heterogeneity in reported outcome variables and measurement tools between studies, and large inter-patient variability within studies.</p><p><p><b>Conclusions:</b> Although the current evidence base is limited, reflecting the typical challenges of clinical research in rare diseases, the reported results suggest potential treatment benefits for patients and provide a rationale for continuing to pursue this therapeutic approach.</p>","PeriodicalId":94235,"journal":{"name":"The Journal of dermatological treatment","volume":"35 1","pages":"2419931"},"PeriodicalIF":0.0,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142650078","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Polynucleotide-based treatments for various facial scars including combat injuries. 基于多核苷酸的各种面部疤痕(包括战伤)治疗方法。
Pub Date : 2024-12-01 Epub Date: 2024-11-19 DOI: 10.1080/09546634.2024.2426626
Michael James Kim, Jovian Wan, Lopatkina Oksana, Lavrik Yuliia, Olena Chugay, Olena Platonova, Olena Sydorchuk, Kyu-Ho Yi

Introduction: This study examines the therapeutic efficacy of polynucleotide-based therapies, specifically REJURAN®, for managing scars and burns. Scars and burns present significant challenges in dermatology, often affecting patients' quality of life. Traditional treatments can be limited in effectiveness, prompting the exploration of innovative approaches like polynucleotide therapy, which utilizes salmon DNA to stimulate tissue repair, modulate inflammation, and enhance collagen production.

Methods: A series of case studies was conducted, including diverse patient profiles with various types of scars and burns. The study evaluated the impact of REJURAN® on scar appearance, texture, and overall skin quality. Some cases also explored the combination of polynucleotide therapy with other treatments such as Platelet-Rich Plasma (PRP) and botulinum toxin.

Results: Notable improvements were observed across cases, including reduced scar visibility and enhanced skin texture. The therapy proved effective in treating post-traumatic, post-operative, and burn-related scars, demonstrating its versatility and potential benefits. Combination therapies showed promising results, though further research is needed.

Conclusion: Polynucleotide-based therapy with REJURAN® shows promise in scar and burn management, offering a novel approach that leverages the body's regenerative processes. Further studies are recommended to confirm these findings and refine treatment protocols.

简介:本研究探讨了基于多核苷酸的疗法(特别是 REJURAN®)在治疗疤痕和烧伤方面的疗效。疤痕和烧伤是皮肤科面临的重大挑战,通常会影响患者的生活质量。传统治疗方法的效果可能有限,这促使人们探索创新方法,如利用鲑鱼 DNA 刺激组织修复、调节炎症和促进胶原蛋白生成的多核苷酸疗法:方法:进行了一系列病例研究,包括不同类型的疤痕和烧伤患者。研究评估了 REJURAN® 对疤痕外观、质地和整体皮肤质量的影响。一些病例还探讨了多核苷酸疗法与其他疗法(如富血小板血浆(PRP)和肉毒杆菌毒素)的结合:各病例均有明显改善,包括疤痕减淡和皮肤质地改善。该疗法在治疗创伤后、术后和烧伤相关疤痕方面效果显著,证明了它的多功能性和潜在优势。尽管还需要进一步研究,但联合疗法显示出了良好的效果:基于 REJURAN® 的多核苷酸疗法在疤痕和烧伤治疗方面大有可为,提供了一种利用人体再生过程的新方法。建议开展进一步研究,以证实这些发现并完善治疗方案。
{"title":"Polynucleotide-based treatments for various facial scars including combat injuries.","authors":"Michael James Kim, Jovian Wan, Lopatkina Oksana, Lavrik Yuliia, Olena Chugay, Olena Platonova, Olena Sydorchuk, Kyu-Ho Yi","doi":"10.1080/09546634.2024.2426626","DOIUrl":"10.1080/09546634.2024.2426626","url":null,"abstract":"<p><strong>Introduction: </strong>This study examines the therapeutic efficacy of polynucleotide-based therapies, specifically REJURAN<sup>®</sup>, for managing scars and burns. Scars and burns present significant challenges in dermatology, often affecting patients' quality of life. Traditional treatments can be limited in effectiveness, prompting the exploration of innovative approaches like polynucleotide therapy, which utilizes salmon DNA to stimulate tissue repair, modulate inflammation, and enhance collagen production.</p><p><strong>Methods: </strong>A series of case studies was conducted, including diverse patient profiles with various types of scars and burns. The study evaluated the impact of REJURAN<sup>®</sup> on scar appearance, texture, and overall skin quality. Some cases also explored the combination of polynucleotide therapy with other treatments such as Platelet-Rich Plasma (PRP) and botulinum toxin.</p><p><strong>Results: </strong>Notable improvements were observed across cases, including reduced scar visibility and enhanced skin texture. The therapy proved effective in treating post-traumatic, post-operative, and burn-related scars, demonstrating its versatility and potential benefits. Combination therapies showed promising results, though further research is needed.</p><p><strong>Conclusion: </strong>Polynucleotide-based therapy with REJURAN<sup>®</sup> shows promise in scar and burn management, offering a novel approach that leverages the body's regenerative processes. Further studies are recommended to confirm these findings and refine treatment protocols.</p>","PeriodicalId":94235,"journal":{"name":"The Journal of dermatological treatment","volume":"35 1","pages":"2426626"},"PeriodicalIF":0.0,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142678149","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
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The Journal of dermatological treatment
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