广义脓疱型牛皮癣(3GPP)是一种罕见的,慢性和潜在威胁生命的自身炎症性皮肤病,其特征是无菌脓疱的广泛爆发,有或没有全身性炎症。3GPP可以显著降低患者的生活质量(QoL)。文献中已经描述了几种治疗方法,但对最佳治疗尚无共识。在这次审查中,我们总结了已发表的关于疗效的文献,安全性和QoL结果与当前使用批准和未批准的产品治疗的GMP相关。检索了Embase和MEDLINE数据库(1980年至2023年9月)。根据系统审查和荟萃分析的首选报告项目(PRISMA)指南设计了搜索方案,并在PROSPERO数据库(CRD42021215437)上注册。出版物的详细信息,人口,干预,功效,安全性和QoL由独立审核员捕获并检查。总的来说,包括118种出版物,只有19%的出版物报告临床试验的结果。所报告的治疗方式包括非生物全身性治疗,如类维生素A,环孢菌素和甲氨蝶呤,局部用药,生物制品和小分子,在其他人中。结果高度异质性,方法学质量很低,只有白介素-36R抑制剂spesolimab报告安慰剂对照随机试验的结果;基于此,spesolimab现在被批准在包括美国在内的地区用于3GPP治疗,Japan,中国,欧盟和其他几个国家。其他一些生物制剂仅在日本和台湾获得批准,用于基于开放标签研究,患者数量少,而不是双盲研究。研究中临床结果的非标准化仍然是达成最佳治疗共识的主要障碍。然而,最近的试验已经进行了使用明确的,疾病特定的终点,以评估3GPP靶向治疗,这有望推进患者护理。总之,这篇综述强调了需要前瞻性随机研究,并结合3GPP特异性终点来确定最佳治疗策略.
泛发性脓疱型银屑病(3GPP)是一种罕见的,以疼痛为特征的慢性皮肤病,可以在全身发生的无菌脓疱。这些脓疱也可能伴有全身性炎症,这可能导致严重的健康并发症。3GPP显著影响患者的生活质量,甚至可能危及生命。因为这种疾病非常罕见,治疗指南通常基于斑块状银屑病。然而,这些指南并没有具体解决3GPP的独特需求。在这次审查中,我们分析了已发表的关于3GPP管理的文献,专注于治疗效果,安全性和生活质量结果。我们在文献数据库Embase和MEDLINE中搜索了1980年至2023年9月之间发表的文章。总的来说,我们确定了118种关于这个主题的出版物,涵盖广泛的疗法;只有其中一种疗法,spesolimab,报告来自安慰剂对照随机试验的结果。基于这些试验,spesolimab现在在美国被批准用于3GPP治疗,Japan,中国,欧盟和其他几个国家。其他一些疗法仅在日本和台湾获得批准,在缺乏高质量数据的情况下进行开放标签研究。迄今为止,比较治疗方法具有挑战性,因为用于衡量有效性的临床结局不同.然而,最近已经开发出了明确定义的、针对3GPP的终点,并在试验中使用.总之,我们的综述强调有必要进行针对3GPP特定终点的前瞻性随机研究,以确定最佳治疗策略.
Generalized pustular psoriasis (GPP) is a rare, chronic and potentially life-threatening autoinflammatory skin disease characterized by widespread eruption of sterile pustules, with or without systemic inflammation. GPP can significantly reduce patients\' quality of life (QoL). Several therapeutic approaches have been described in the literature, but there is no consensus on optimal treatment. In this review, we summarize published literature on efficacy, safety and QoL outcomes associated with current treatment of GPP with both approved and non-approved products. Embase and MEDLINE databases were searched (1980-September 2023). A search protocol was designed in accordance with the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) guidelines and registered on the PROSPERO database (CRD42021215437). Details on publication, population, intervention, efficacy, safety and QoL were captured and checked by independent reviewers. In total, 118 publications were included, with only 19% of publications reporting on the results of clinical trials. Treatment modalities reported for GPP included non-biologic systemic therapies such as retinoids, cyclosporine and methotrexate, topical agents, biologics and small molecules, among others. Results were highly heterogeneous and methodological quality was very low, with only the interleukin-36R inhibitor spesolimab reporting results from placebo-controlled randomized trials; based on this, spesolimab is now approved for GPP treatment in regions including the USA, Japan, China, the EU and several other countries. Some other biologics are approved exclusively in Japan and Taiwan for the treatment of GPP based on open-label studies with small patient numbers in lieu of double-blind studies. Non-standardization of clinical outcomes across studies remains a major hurdle in reaching a consensus on optimal treatment. However, recently trials have been conducted using well-defined, disease-specific endpoints to evaluate GPP-targeted treatments, which will hopefully advance patient care. In conclusion, this review highlights the need for prospective randomized studies with GPP-specific endpoints to determine the optimal treatment strategy.
Generalized pustular psoriasis (GPP) is a rare, chronic skin condition characterized by painful, sterile pustules that can occur all over the body. These pustules may also be accompanied by systemic inflammation, which can lead to serious health complications. GPP significantly impacts patients’ quality of life and can even be life-threatening. Because the disease is so rare, treatment guidelines have typically been based on those for plaque psoriasis. However, these guidelines do not specifically address the unique needs of GPP. In this review, we analysed the published literature on GPP management, focussing on treatment efficacy, safety and quality of life outcomes. We searched the literature databases Embase and MEDLINE for articles published between 1980 and September 2023. In total, we identified 118 publications on this topic, covering a wide range of therapies; only one of these therapies, spesolimab, reported results from placebo-controlled randomized trials. Based on these trials, spesolimab is now approved for GPP treatment in the USA, Japan, China, the EU and several other countries. Some other therapies are approved exclusively in Japan and Taiwan based on small, open-label studies in the absence of higher-quality data. To date, comparing treatments has been challenging because of different clinical outcomes used to measure effectiveness. However, well-defined endpoints specific to GPP have recently been developed and used in trials. In conclusion, our review highlights the need for prospective randomized studies with GPP-specific endpoints to determine the best treatment strategy.