Torres Pradilla Mauricio, Álvarez Erick, Novoa Mónica, Lozano Ivonne, Trujillo Maribel
Fundación Universitaria de Ciencias de la Salud and Hospital Infantil de San José, Bogotá, Colombia.
Universidad De Cartagena, Hospital Infantil Napoleón Franco Pareja, Cartagena, Colombia.
Adv Ther. 2024 Feb;41(2):867-877. doi: 10.1007/s12325-023-02749-x. Epub 2024 Jan 3.
Epidermolysis bullosa (EB) is a group of rare, difficult-to-treat, inherited multisystem diseases affecting epithelial integrity. Impaired wound healing is central and can lead to serious clinical complications, deformities, and symptoms with a devastating impact on quality of life (QoL). Dressing changes and wound care are central to the management of EB. Recently Oleogel-S10 (also known as birch bark extract or birch triterpenes) was approved in Europe and the UK for treating EB wounds. This approval was based on data from the EASE phase 3 study, which demonstrated Oleogel-S10 accelerated wound healing, reduced total wound burden, and decreased the frequency of dressing changes in patients with EB. A retrospective analysis of medical records was conducted for up to 24 months in 13 patients with EB treated with Oleogel-S10 through an early access programme in Colombia. Effectiveness was assessed by measuring body surface area percentage (BSAP) and total body wound burden (EBDASI). Tolerability and safety were monitored throughout. This is the first report to evaluate the effectiveness of Oleogel-S10 in clinical practice. The results showed a reduction in percentage of BSA affected, from a mean of 27.3% at baseline to 10.4% at 24-month follow-up, despite treatment interruptions. A reduction in EBDASI skin activity score of - 16.2 (24 months) together with a reduced skin damage index score of - 15.4 (18 months) was also observed. Physicians, patients, and caregivers perceived faster wound closure. Adherence with therapy by patients was good, and patients expressed satisfaction with treatment and reported improvements in self-esteem, productivity, and social interaction. Oleogel-S10 was well tolerated; however, two patients reported worsening wounds related to gauze adherence. Two deaths during treatment interruption were reported and was not considered related to Oleogel-S10. This study supports the effectiveness of Oleogel-S10 in a real-world scenario in a country with scarce resources for the treatment of EB.
大疱性表皮松解症(EB)是一组罕见的、难以治疗的遗传性多系统疾病,会影响上皮完整性。伤口愈合受损是其核心问题,可导致严重的临床并发症、畸形和症状,对生活质量(QoL)产生毁灭性影响。换药和伤口护理是EB治疗的关键。最近,Oleogel-S10(也称为桦树皮提取物或桦树三萜)在欧洲和英国被批准用于治疗EB伤口。该批准基于EASE 3期研究的数据,该研究表明Oleogel-S10可加速伤口愈合、减轻总伤口负担并减少EB患者的换药频率。通过哥伦比亚的一项早期准入计划,对13例接受Oleogel-S10治疗的EB患者的病历进行了长达24个月的回顾性分析。通过测量体表面积百分比(BSAP)和全身伤口负担(EBDASI)来评估有效性。全程监测耐受性和安全性。这是第一份评估Oleogel-S10在临床实践中有效性的报告。结果显示,尽管治疗有中断,但受影响的体表面积百分比从基线时的平均27.3%降至24个月随访时的10.4%。还观察到EBDASI皮肤活动评分降低了-16.2(24个月),皮肤损伤指数评分降低了-15.4(18个月)。医生、患者和护理人员都感觉到伤口愈合加快。患者对治疗的依从性良好,患者对治疗表示满意,并报告自尊、生产力和社交互动有所改善。Oleogel-S10耐受性良好;然而,有两名患者报告与纱布粘连相关的伤口恶化。报告了治疗中断期间的两例死亡病例,认为与Oleogel-S10无关。本研究支持Oleogel-S10在一个资源稀缺的国家治疗EB的现实场景中的有效性。