Randomized, controlled, proof-of-concept trial of gefapixant for endometriosis-related pain.

OBJECTIVE: To evaluate the P2X3 receptor antagonist, gefapixant, for treating moderate-to-severe endometriosis-related pain. DESIGN: Randomized, double-blind, Phase 2, proof-of-concept trial. PARTICIPANTS: Premenopausal women age 18-49y with moderate-to-severe endometriosis-related pain who were not using hormonal treatment. INTERVENTIONS: Gefapixant 45mg twice-daily or placebo over two menstrual cycles. MAIN OUTCOME MEASURES: Participants rated peak pelvic pain severity daily on a 0 (no pain) - 10 (extremely severe pain) scale. The primary endpoint was change-from-baseline in average daily peak pelvic pain severity during Treatment Cycle 2. RESULTS: All 187 participants randomized (gefapixant N=94, placebo N=93) took ≥1 dose of investigational treatment and all but 6 in each treatment group completed the trial. The model-based least squares mean reduction-from-baseline in average daily peak pelvic pain severity during Treatment Cycle 2 was -2.2 for gefapixant and -1.7 for placebo (difference = -0.5 [95% confidence interval: -1.01, 0.03], P=0.066). In secondary analyses, the difference between gefapixant and placebo in peak pelvic pain severity reduction-from-baseline on menstrual days was -0.6 [95% confidence interval: -1.18, -0.06], and -0.5 [95% confidence interval: -1.04, 0.03] on non-menstrual days. Taste-related adverse events were reported in 31.9% of participants for gefapixant versus 4.3% for placebo. Pharmacokinetic assessments at Month 1 and Month 2 clinic visits indicated that of the 94 participants in the gefapixant group, 39 had detectable levels of gefapixant in the blood for both assessments while 38 had no detectable levels for ≥1 assessment. CONCLUSION: Gefapixant 45mg twice-daily was not shown to be superior to placebo in reducing endometriosis-related pain, although the results directionally favored gefapixant. This trial result should be considered inconclusive given possible issues with treatment compliance. (Clinical trial registration: NCT03654326).

The endometriosis daily diary: qualitative research to explore the patient experience of endometriosis and inform the development of a patient-reported outcome (PRO) for endometriosis-related pain.

PURPOSE: Endometriosis is a chronic disorder of the female reproductive system characterized by debilitating symptoms, particularly endometriosis-related pain (ERP). Patient-reported outcome (PRO) measures of symptoms and impacts are required to assess disease severity in ERP clinical studies and clinical practice. A content-valid instrument was developed by modifying the Dysmenorrhea Daily Diary (DysDD) to form the Endometriosis Daily Diary (EDD), an electronic PRO administered via handheld device. METHODS: Qualitative research with US females with ERP was conducted in three stages: (1) Development of an endometriosis conceptual model based on qualitative literature and conduct of concept elicitation (CE) interviews (N = 30). (2) Cognitive debriefing (CD) interviews (N = 30) conducted across two rounds to assess relevance and understanding of the EDD, with modifications between interview rounds. (3) Pilot testing to assess usability/feasibility of administrating the EDD daily on an electronic handheld device (N = 15). Clinical experts provided guidance throughout the study. RESULTS: The conceptual model provided a comprehensive summary of endometriosis to inform modifications to the DysDD, forming the EDD. CD results demonstrated that EDD items were relevant for most participants. Instructions, items, response scales, and recall period were well-understood. The resulting daily diary assesses severity of cyclic and non-cyclic pelvic pain, dyspareunia, impact of ERP on functioning and daily life, symptoms associated with ERP, and bowel symptoms. Participants were able to complete the diary daily and found the device easy to use. CONCLUSION: The EDD demonstrated good content validity in females experiencing ERP. The next step is to perform psychometric validation in an ERP sample.

Comorbidities in a sample of adults with HIV in Puerto Rico: an exploratory study.

BACKGROUND: Puerto Rico is among the areas with the highest estimated rates of people living with HIV in the United States. Despite the epidemiologic data available, there is limited real-world information that can help understand the comorbidities of people with HIV. In this study, we describe common comorbidities among adults with HIV attending treatment clinics in Puerto Rico. METHODS: An exploratory, retrospective, cross-sectional study was conducted at five HIV clinics in Puerto Rico. A random sample of medical records was reviewed. Descriptive statistics were used to summarize patient demographics, morbidity, and clinical characteristics. Multivariate analyses were conducted to explore comorbidities by age and sex. RESULTS: A total of 250 (179 men; 71 women) medical records were reviewed. Participants' mean age was 47.9 years and on average they had been living with HIV for 9 years. Most (97.6%) had at least one comorbidity. The most common comorbidities were dyslipidemia and hypertension. Men were more likely to have been diagnosed with alcohol misuse while women were more likely to have been diagnosed with obesity, human papillomavirus (HPV), hypothyroidism, and osteoporosis. Participants younger than 50 years of age were more likely to have history of alcohol misuse while older individuals (50 years and old) were more likely to have been diagnosed with dyslipidemia, hypertension, and diabetes. Adjusting by sex and age, women were more likely to have been diagnosed with obesity and depression and those older than 50 years were more likely to have had a diagnosis of dyslipidemia, hypertension, HPV, and diabetes. CONCLUSIONS: This is one of the few studies assessing comorbidities among adults with HIV in Puerto Rico, among Latino/Hispanics within the United States, and Latin America. Consistent with other studies, cardiovascular diseases are common among adults with HIV in Puerto Rico. Findings support the need for awareness and real-world evidence about comorbidities among people with HIV when implementing screenings and prescribing drugs.

Manejo laparascópico de un teratoma quístico maduro bilateral. Reporte de un caso

El manejo laparoscópico de las masas anexiales benignas se ha difundido recientemente. La mayor experienciade los cirujanos y el diagnóstico imagenológico certero, han hecho que cada vez más las pacientes pueden beneficiarse del manejo laparoscópico, con las conocidas ventajas de costo, tiempo de hospitalización, tipo de abordaje y tiempo de incapacidad. Se presenta aquí el resumen de una paciente con teratomas quísticos maduros bilaterales manejada por vía laparoscópica en la Unidad de Endoscopia Gineco-Obstétrica (UEGO)del Hospital Infantil Universitario Lorencita Villegas de Santos de Santafé de Bogotá

Falla ovárica prematura: causas genéticas

Presentamos una paciente de 33 años de edad que consulta por amenorrea secundaria causada por una Falla Ovárica Prematura(FOP) secundaria a un Síndrome Turner en mosaico. La FOP definida como amenorrea hipergonadotrópica en pacientes menores de 40 años tiene una incidencia del 2 al 10 por ciento. Es causada por diferentes entidades entre las que encontramos desórdenes autoinmunes, defectos metabólicos y enzimáticos, enfermedades infecciosas, causas iatrogénicas, defectos en las gonadotropinas y causas genéticas. Estas últimas son responsables de FOP especialmente en pacientes menores de 30 años, en las que se justifica la solicitud de cariotipo como parte del estudio. Describimos las características de la disgenesia gonadal, del Síndrome Turner y los elementos funcionales del cromosoma X cuya ausencia altera la función gonadal. También mencionamos los diferentes mosaicos descritos en relación al Síndrome Turner y sus consecuencias en el desarrollo somático y gonadal