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":null,"pages":null},"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":null,"pages":null},"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":null,"pages":null},"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":null,"pages":null},"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":null,"pages":null},"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":null,"pages":null},"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":null,"pages":null},"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":null,"pages":null},"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: 2024-09-04DOI: 10.1080/09546634.2024.2398170
Axel Svedbom, Christina Wennerström, Fredrik Hjelm, Anna Tjärnlund, Mona Ståhle
Background: The advent of biosimilars may increase the frequency of dose escalation with biologics in psoriasis.
Objective: To explore the frequency and outcomes of dose escalation with adalimumab etanercept, and ustekinumab.
Methods: Data were extracted from DermaReg-Pso, a psoriasis register in Stockholm, Sweden. The main exposure was treatment, and the main outcome was dose escalation. We describe outcomes with dose escalation by estimating drug survival and changes in the Psoriasis Area and Severity Index (PASI).
Results: 554 patients had 946 treatment episodes with adalimumab, etanercept, or ustekinumab. The cumulative incidence of dose escalation was 4.1 per 100 treatment years. The Hazard Ratios (HRs) for dose escalation with ustekinumab vs adalimumab and ustekinumab vs etanercept were 1.93 (95% CI: 1.25-2.98), and 2.20 (95% CI: 1.42-3.41), respectively. After dose escalation, the HRs for treatment discontinuation with adalimumab and etanercept compared with ustekinumab were 3.10 (95% CI: 1.56-6.18) and 7.15 (95% CI: 3.96-12.94), respectively. PASI was higher after compared to before dose escalation for etanercept (p = 0.036), but not for adalimumab (p = 0.832) or ustekinumab (p = 0.300).
Conclusions: Dose escalation was comparatively more frequent with ustekinumab than with adalimumab or etanercept; however, treatment discontinuation after dose escalation was more common with adalimumab and etanercept than ustekinumab.
{"title":"Frequency and outcomes of treatment dose escalation with biologics in moderate-to-severe psoriasis: a Swedish register study.","authors":"Axel Svedbom, Christina Wennerström, Fredrik Hjelm, Anna Tjärnlund, Mona Ståhle","doi":"10.1080/09546634.2024.2398170","DOIUrl":"https://doi.org/10.1080/09546634.2024.2398170","url":null,"abstract":"<p><strong>Background: </strong>The advent of biosimilars may increase the frequency of dose escalation with biologics in psoriasis.</p><p><strong>Objective: </strong>To explore the frequency and outcomes of dose escalation with adalimumab etanercept, and ustekinumab.</p><p><strong>Methods: </strong>Data were extracted from DermaReg-Pso, a psoriasis register in Stockholm, Sweden. The main exposure was treatment, and the main outcome was dose escalation. We describe outcomes with dose escalation by estimating drug survival and changes in the Psoriasis Area and Severity Index (PASI).</p><p><strong>Results: </strong>554 patients had 946 treatment episodes with adalimumab, etanercept, or ustekinumab. The cumulative incidence of dose escalation was 4.1 per 100 treatment years. The Hazard Ratios (HRs) for dose escalation with ustekinumab vs adalimumab and ustekinumab vs etanercept were 1.93 (95% CI: 1.25-2.98), and 2.20 (95% CI: 1.42-3.41), respectively. After dose escalation, the HRs for treatment discontinuation with adalimumab and etanercept compared with ustekinumab were 3.10 (95% CI: 1.56-6.18) and 7.15 (95% CI: 3.96-12.94), respectively. PASI was higher after compared to before dose escalation for etanercept (<i>p</i> = 0.036), but not for adalimumab (<i>p</i> = 0.832) or ustekinumab (<i>p</i> = 0.300).</p><p><strong>Conclusions: </strong>Dose escalation was comparatively more frequent with ustekinumab than with adalimumab or etanercept; however, treatment discontinuation after dose escalation was more common with adalimumab and etanercept than ustekinumab.</p>","PeriodicalId":94235,"journal":{"name":"The Journal of dermatological treatment","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142127793","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: 2024-03-17DOI: 10.1080/09546634.2024.2328180
Kevin M Burningham, Ritu N Swali, Peter L Rady, Anisha B Patel, Stephen K Tyring
Purpose: Trichodysplasia spinulosa (TS) is a rare, disfiguring skin condition which presents with widespread asymptomatic or pruritic, skin-colored papules with white protruding keratin spiculations in immunocompromised individuals. Due to its rarity, there is little data to guide treatment decisions. The purpose of this article is to report a case of TS that completely resolved after treatment with topical cidofovir.Materials and methods: A 19-year-old immunosuppressed female presented with widespread painful, itchy bumps on the nose and face. Upon examination, there were erythematous papules with hyperkeratinized spicules affecting the central face. Biopsy of the lesions was consistent with TS which was confirmed via PCR analysis. The tenderness of this patient's eruption was highly atypical for TS. Once daily topical application of compounded 1% cidofovir cream was prescribed.Results: The patient's symptoms resolved completely after 4 weeks of therapy with topical cidofovir 1% cream, without reduction of immunosuppression.Conclusions: Topical cidofovir 1% cream may be a valuable treatment for this rare disease.
{"title":"Resolution of painful trichodysplasia spinulosa with topical cidofovir: case report.","authors":"Kevin M Burningham, Ritu N Swali, Peter L Rady, Anisha B Patel, Stephen K Tyring","doi":"10.1080/09546634.2024.2328180","DOIUrl":"10.1080/09546634.2024.2328180","url":null,"abstract":"<p><p><b>Purpose:</b> Trichodysplasia spinulosa (TS) is a rare, disfiguring skin condition which presents with widespread asymptomatic or pruritic, skin-colored papules with white protruding keratin spiculations in immunocompromised individuals. Due to its rarity, there is little data to guide treatment decisions. The purpose of this article is to report a case of TS that completely resolved after treatment with topical cidofovir.<b>Materials and methods:</b> A 19-year-old immunosuppressed female presented with widespread painful, itchy bumps on the nose and face. Upon examination, there were erythematous papules with hyperkeratinized spicules affecting the central face. Biopsy of the lesions was consistent with TS which was confirmed via PCR analysis. The tenderness of this patient's eruption was highly atypical for TS. Once daily topical application of compounded 1% cidofovir cream was prescribed.<b>Results:</b> The patient's symptoms resolved completely after 4 weeks of therapy with topical cidofovir 1% cream, without reduction of immunosuppression.<b>Conclusions:</b> Topical cidofovir 1% cream may be a valuable treatment for this rare disease.</p>","PeriodicalId":94235,"journal":{"name":"The Journal of dermatological treatment","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140144976","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}