Pub Date : 2024-12-01Epub Date: 2024-08-18DOI: 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。
{"title":"Emerging DNA & RNA editing strategies for the treatment of epidermolysis bullosa.","authors":"Ulrich Koller, Johann W Bauer","doi":"10.1080/09546634.2024.2391452","DOIUrl":"https://doi.org/10.1080/09546634.2024.2391452","url":null,"abstract":"<p><p><b>Background:</b> 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.</p><p><p><b>Methods:</b> Emerging RNA and DNA editing tools have shown remarkable advances in efficiency and safety. Applicable both in <i>ex vivo</i>- and <i>in vivo</i> settings, these gene therapeutics based on gene replacement or editing are either at the pre-clinical or clinical stage.</p><p><p><b>Results:</b> The recent landmark FDA approvals for gene editing based on CRISPR/Cas9, along with the first FDA-approved redosable <i>in vivo</i> 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.</p><p><p><b>Conclusions:</b> 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.</p>","PeriodicalId":94235,"journal":{"name":"The Journal of dermatological treatment","volume":"35 1","pages":"2391452"},"PeriodicalIF":0.0,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142001633","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}
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.
{"title":"The adjuvant treatment role of ω-3 fatty acids by regulating gut microbiota positively in the acne vulgaris.","authors":"Yaxin Huang, Fuming Liu, Jindong Lai, Shiyu Jiang, Xiaoqi Tan, Lingna Chen, Yong Xu, Xia Xiong, Yongqiong Deng","doi":"10.1080/09546634.2023.2299107","DOIUrl":"10.1080/09546634.2023.2299107","url":null,"abstract":"<p><p><b>Objectives:</b>We aimed to explore the potential role of omega-3 (ω-3) fatty acids on acne vulgaris by modulating gut microbiota.<b>Materials and Methods:</b>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.<b>Results:</b>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.<b>Conclusion:</b>Supplementation of ω-3 fatty acids could alleviate the inflammation of acne vulgaris by increasing the abundance of butyric acid producing bacteria.</p>","PeriodicalId":94235,"journal":{"name":"The Journal of dermatological treatment","volume":"35 1","pages":"2299107"},"PeriodicalIF":0.0,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139072488","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}
Pub Date : 2024-12-01Epub Date: 2024-01-19DOI: 10.1080/09546634.2024.2304630
Francisco Javier De la Torre-Gomar, Juan Pablo Velasco-Amador, Álvaro Prados-Carmona, Ricardo Ruiz-Villaverde
{"title":"Complete response of extensive alopecia areata refractory to baricitinib after five months of treatment with upadacitinib.","authors":"Francisco Javier De la Torre-Gomar, Juan Pablo Velasco-Amador, Álvaro Prados-Carmona, Ricardo Ruiz-Villaverde","doi":"10.1080/09546634.2024.2304630","DOIUrl":"10.1080/09546634.2024.2304630","url":null,"abstract":"","PeriodicalId":94235,"journal":{"name":"The Journal of dermatological treatment","volume":"35 1","pages":"2304630"},"PeriodicalIF":0.0,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139492979","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}
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.
{"title":"Injection site reactions after dupilumab or tralokinumab for atopic dermatitis.","authors":"Fabrizio Martora, Cataldo Patruno, Silvia D'Ascenzo, Maddalena Napolitano","doi":"10.1080/09546634.2024.2304027","DOIUrl":"10.1080/09546634.2024.2304027","url":null,"abstract":"<p><p><b>Background:</b> 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.<b>Objective:</b> ISR is reported as a frequent adverse event after subcutaneous injection (SCI) of several biologics.<b>Methods:</b> 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.<b>Results and conclusions:</b> 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.</p>","PeriodicalId":94235,"journal":{"name":"The Journal of dermatological treatment","volume":"35 1","pages":"2304027"},"PeriodicalIF":0.0,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139492985","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}
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.
{"title":"Oral abrocitinib in the treatment of granuloma annulare: a case report.","authors":"Wenyan Liu, Weifeng Chen, Xin Tian, Yihui Yu, Junhui Zhu, Jingyao Liang, Xibao Zhang","doi":"10.1080/09546634.2024.2313090","DOIUrl":"10.1080/09546634.2024.2313090","url":null,"abstract":"<p><p><b>Aim:</b> 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.<b>Methods:</b> 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.<b>Results:</b> 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.<b>Conclusions:</b> Abrocitinib may be a promising and safe treatment option for patients with localized GA who do not respond to traditional therapies.</p>","PeriodicalId":94235,"journal":{"name":"The Journal of dermatological treatment","volume":"35 1","pages":"2313090"},"PeriodicalIF":0.0,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139693837","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}
Pub Date : 2024-12-01Epub Date: 2024-01-23DOI: 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.
{"title":"Impairment in work and activities of daily life in patients with psoriasis: results of the prospective BioCAPTURE registry.","authors":"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","doi":"10.1080/09546634.2024.2304025","DOIUrl":"10.1080/09546634.2024.2304025","url":null,"abstract":"<p><p><b>Background:</b> 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.<b>Methods:</b> 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.<b>Results:</b> 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, <i>p</i> < 0.001). Work-for-pay in this cohort was lower than in the Dutch general population (53% versus 67%, <i>p</i> = 0.01). In patients who had work-for-pay, presenteeism improved over time (5% at baseline versus 0% at 12 months, <i>p</i> = 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.<b>Conclusion:</b> 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.</p>","PeriodicalId":94235,"journal":{"name":"The Journal of dermatological treatment","volume":"35 1","pages":"2304025"},"PeriodicalIF":0.0,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139542597","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}
Pub Date : 2024-12-01Epub Date: 2024-05-16DOI: 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.
{"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}
Pub Date : 2024-12-01Epub Date: 2024-06-16DOI: 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.
{"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}
Pub Date : 2024-12-01Epub Date: 2024-11-17DOI: 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.
{"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}
Pub Date : 2024-12-01Epub Date: 2024-11-19DOI: 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。
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