Oleogel-S10 in Dystrophic Epidermolysis Bullosa: A Case Series Evaluating the Impact on Wound Burden Over Two Years.

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.

Consensus guidelines for diagnosis and management of anemia in epidermolysis bullosa.

BACKGROUND: Anemia is a common complication of severe forms of epidermolysis bullosa (EB). To date, there are no guidelines outlining best clinical practices to manage anemia in the EB population. The objective of this manuscript is to present the first consensus guidelines for the diagnosis and management of anemia in EB. RESULTS: Due to the lack of high-quality evidence, a consensus methodology was followed. An initial survey exploring patient preferences, concerns and symptoms related to anemia was sent to EB patients and their family members. A second survey was distributed to EB experts and focused on screening, diagnosis, monitoring and management of anemia in the different types of EB. Information from these surveys was collated and used by the panel to generate 26 consensus statements. Consensus statements were sent to healthcare providers that care for EB patients through EB-Clinet. Statements that received more than 70% approval (completely agree/agree) were adopted. CONCLUSIONS: The end result was a series of 6 recommendations which include 20 statements that will help guide management of anemia in EB patients. In patients with moderate to severe forms of EB, the minimum desirable level of Hb is 100 g/L. Treatment should be individualized. Dietary measures should be offered as part of management of anemia in all EB patients, oral iron supplementation should be used for mild anemia; while iron infusion is reserved for moderate to severe anemia, if Hb levels of > 80-100 g/L (8-10 g/dL) and symptomatic; and transfusion should be administered if Hb is < 80 g/L (8 g/dL) in adults and < 60 g/L (6 g/dL) in children.

Outcomes and Predictors for Re-stenosis of Esophageal Stricture in Epidermolysis Bullosa: A Multicenter Cohort Study.

BACKGROUND: Esophageal strictures are the common gastrointestinal complications in patients with epidermolysis bullosa (EB) requiring dilation. There is limited information on the best type of intervention, outcomes, and predictors for re-stenosis. OBJECTIVES: We aimed to investigate the frequency, clinical presentation of esophageal strictures in EB patients, and to ascertain the predictors of re-stenosis. METHODS: We conducted a retrospective, multicenter cohort study involving 7 specialized, international EB centers on patients who were 0 to 50 years of age. Descriptive statistics and hazard risks for re-stenosis were calculated. RESULTS: We identified 125 patients with 497 esophageal stricture episodes over a mean period of observation of 17 (standard deviation [SD] = 11.91) years. Dilations were attempted in 90.74% of episodes, using guided fluoroscopy 45.23%, retrograde endoscopy 33.04%, and antegrade endoscopy 19.07%. Successful dilation was accomplished in 99.33% of attempts. Patients experienced a median of 2 (interquartile range [IQR]: 1-7) stricture episodes with a median interval between dilations of 7 (IQR: 4-12) months. Predictors for re-stenosis included: number of strictures (2 vs 1 stricture: χ = 4.293, P = 0.038, hazard ratio [HR] = 1.294 (95% confidence interval [CI]: 1.014--1.652 and 3 vs 1 stricture:χ = 7.986, P = 0.005, HR = 1.785 [95% CI: 1.194, 2.667]) and a long (≥1 cm) segment stricture (χ = 4.599, P = 0.032, HR = 1.347 (95% CI: 1.026--1.769). Complications were more common with the endoscopic approach (8/86, antegrade endoscopy; 2 /149, retrograde endoscopy vs 2/204, fluoroscopy; χ = 17.39, P-value <0.000). CONCLUSIONS: We found excellent dilation outcomes irrespective of the dilation procedure; however, with higher complications in the endoscopic approach. Long (>1 cm) segment involvement and multiple locations were predictive of stricture reoccurrence.

Intralesional Candida Antigen Immunotherapy for the Treatment of Recalcitrant and Multiple Warts in Children.

BACKGROUND: Intralesional injection of Candida antigen appears to be an effective alternative for the treatment of warts. AIM: To determine the efficacy and safety of this treatment. METHODS: We retrospectively reviewed records of all children who received intralesional injection of Candida antigen at our center from January 2008 to July 2013. RESULTS: From a total of 220 patients, 156 (70.9%) had a complete response, 37 (16.8%) had a partial response, and 27 (12.2%) had no improvement. An average of 2.73 treatments was needed. Forty-seven of the patients with more than one wart (21.3%) also noted at least partial resolution of untreated warts at distant sites. Twenty-seven of the 47 patients (57.4%) had complete resolution. All treated patients experienced some discomfort at the time of the injection, but no serious side effects were reported. DISCUSSION: We report our results using this approach in a large group of children. CONCLUSION: Intralesional injection of Candida antigen is an effective and safe therapy for children with multiple and recalcitrant cutaneous warts.

Failure of intralesional propranolol in infantile hemangiomas.

The purpose of this study was to evaluate the use of intralesional propranolol injection in the management of small, noncomplicated infantile hemangiomas (IHs) located in areas of cosmetic concern. A prospective study was performed in six female infants with small, non-complicated IHs in areas of cosmetic concern. The parents had refused oral propranolol or the patients had no response to topical timolol or had relapsed after oral propranolol and the parents refused further systemic treatment. All six patients were treated with 1 mg/mL propranolol solution at a dose of 0.2 mL/cm(2). The size, color, and growth of the hemangiomas were monitored and recorded every 4 weeks. Treatment response was evaluated using a 5-point scale: much better (+2), better (+1), same (0), worse (-1), and much worse (-2). Heart rate and blood pressure were measured before and 1 hour after each injection. Adverse effects after medication were evaluated and managed accordingly. All hemangiomas stopped growing during therapy, but no significant changes in size or color were observed, even after repeated injections, and all patients were evaluated as 0 (same). One patient whose hemangioma stopped growing during treatment presented rebound growth after therapy cessation. No changes in heart rate or blood pressure were observed after intralesional propranolol injection. Adverse effects observed were pain and redness after injection. Intralesional propranolol seems safe but is not effective for the treatment of IH.

Reacciones medicamentosas severas Síndrome Stevens Johnson y síndrome DRESS / Severe drug reactions Stevens Johnson syndrome and DRESS syndrome

Propósito: revisar y actualizar el síndrome de Steven-Johnson y el síndrome DRESS, que a pesar de no ser las reacciones medicamentosas más frecuentes, sí constituyen las más severas. Fuente de datos: Realizamos una revisión sistemática de la literatura médica, expuesta en las bases de datos: Pubmed, Medline y Cochrane. Selección de estudios: limitamos la búsqueda a artículos de revisión de literatura, guías de manejo y metaanálisis, publicados en inglés y español, sin límite en edad o sexo, utilizando términos MESH: Stevens-Johnson syndrome, DRESS syndrome. Los acoplamos a términos como assessment, associations, update y review. Extracción de datos y resultados: realizamos una lectura inicial de 338 trabajos, encontrando que varios de las referencias eran citadas en otros trabajos, por lo que seleccionamos 10 artículos y con base en estos realizamos la lectura crítica, clasificación y ordenamiento, para luego proceder a la redacción del texto. Conclusiones: hemos revisado dos importantes reacciones medicamentosas, entidades con un espectro clínico y etiopatogénico muy distinto, y numerosas asociaciones medicamentosas, sobre las que no existen realmente concensos globales de tratamiento, pero en la que todos los autores revisados coinciden en la suspensión de los medicamentos, como primera línea de tratamiento.

Purpose: to review and update the Stevens-Johnson syndrome and DRESS syndrome, which despite not being the most frequent drug reactions, are the most severe ones. Data sources: we conducted a systematic review of the medical literature, as described in the databases: PubMed, Medline and Cochrane. Study selection: we limited the search to articles of literature review, management guidelines and meta-analysis published in English and Spanish, with no limit on age or sex, using MESH terms: Stevens-Johnson syndrome, DRESS syndrome. We linked them to terms such as assessment, associations, update and review. Data extraction and results: we performed an initial reading of 338 studies, finding that several of the references were cited in other works, so we selected 10 articles and based on this we performed the critical reading, sorting and ordering, and then proceeded to the text drafting. Conclusions: we reviewed two important drug reactions, clinical entities with very different clinical and etiopathogenic spectrum and numerous drug combinations on which there is not a global treatment concensus, but in which all authors agree in the drug suspension as the frst line of treatment.

Acuerdo entre el examen dermatológico directo y teledermatología asincrónica prueba piloto, Hospital de San José, Bogotá DC, Colombia / Diagnostic agreement between face-toface dermatologic exam and asynchronious teledermatology evaluations - a pilot study - Hospital de San José, Bogotá DC

la patología dermatológica es frecuente y el acceso a especialistas está limitado tanto en lugares remotos como en las grandes ciudades. El empleo de teledermatología permite el diagnóstico y el tratamiento oportuno. Objetivo: describir la frecuencia de acuerdo intra e interobservador entre la valoración dermatológica de rutina (examen físico directo) y la de teledermatología asincrónica. Métodos: se realizó una prueba piloto previa implementación de la plataforma de teledermatología. Dos dermatólogos expertos valoraron 39 pacientes que consultaron por primera vez al servicio de dermatología del Hospital de San José, Bogotá DC. Emitieron su diagnóstico independiente para evaluar el acuerdo interobservador. La plataforma de telemedicina asincrónica se utilizó para presentar las fotografías de las lesiones dos meses después de la valoración clínica directa. En esta fase se describe el acuerdo intra e interobservador y el grado de satisfacción de los dermatólogos con la plataforma. Resultados: el acuerdo intraobservador del examen físico directo y el teledermatológico fue 51 y 59% (evaluadores 1 y 2), siendo mayor ante patología tumoral (68 y 74%). En el examen físico directo el acuerdo interobservador fue 64% y se reduce a 46% con telemedicina. Conclusiones: la frecuencia de acuerdo entre el examen dermatológico directo y el teledermatológico fue aceptable y mejora ante patología tumoral. Estos resultados coinciden con lo descrito en la literatura en series mayores.

Skin diseases are common and access to specialists is limited in remote locations as well as in large cities. The use of teledermatology allows a prompt diagnosis and treatment. Objective: to determine the intra and interobserver diagnostic agreement between traditional dermatologic examination (face-to-face physical exam) and asynchronous teledermatology evaluations. Methods: a pilot study was conducted prior to the teledermatology platform implementation. Two expert dermatologists examined 39 first-time patients attended at Hospital de San José, Bogotá DC dermatology service. They provided their own independent diagnosis in order to conduct the interobserver diagnostic correlation evaluation. The asynchronous teledermatology platform was used to show photographs of the lesions two months later. This phase describes the intra and interobserver agreement and satisfaction among dermatologists regarding the platform. Results: the rate of intraobserver agreement between face-to-face physical exam and teledermatology evaluations was 51 and 59% (examiners 1 and 2) and greater for tumoral lesions (68% and 74%). The rate of interobserver agreement was 64% for direct physical exam and declined to 46% when using telemedicine. Conclusions: the correlation rate between direct dermatologic exam and teledermatology evaluations was acceptable and improves in tumoral pathology. These results match those reported in the literature regarding greater series.