Objective: Glucagonoma is a rare neuroendocrine tumor of the pancreas. We present the case of a young female patient who presented with the major clinical manifestations of glucagonoma syndrome. Methods: The major clinical manifestations of glucagonoma syndrome are described in a 44-year-old, female patient. Beyond glucagonoma, the patient also displayed deep venous thrombosis, depression, diabetes, and necrolytic migratory erythema. We discuss the difficulty of treatment of patients with glucagonoma due to the low prevalence of the disorder, scarcity of medical evidence, lateness of diagnosis with liver metastases in most cases, and poor response to chemotherapy with high rates of relapse after surgery. In this case, pancreatectomy and hepatic lobectomy followed by somatostatin analogue therapy was the chosen treatment strategy. Results: The clinical findings were pancreatic and hepatic masses, proximal deep venous thrombosis, depression, diabetes, and necrolytic migratory erythema. The patient also had elevated levels of glucagon. Pancreatectomy and right hepatic lobectomy were performed and confirmed the glucagonoma. Conclusion: Our case adds new knowledge about glucagonoma which is important due to the low incidence of the disease and the particular characteristics of the syndrome.
7016 Background: Many patients (pts) with myelofibrosis (MF) experience anemia resulting from disease or JAK inhibitor therapy. The open-label, phase 2 ACE-536-MF-001 study (NCT03194542) evaluated the safety and efficacy of luspatercept (LUSPA) for the treatment of MF-related anemia. Methods: Eligible pts comprised 4 cohorts based on transfusion dependence (TD, defined as receiving 4–12 red blood cell [RBC] units/84 days [d] immediately up to cycle 1 d 1) and stable ruxolitinib (RUX) treatment; cohort 1: no TD, no RUX; cohort 2: TD, no RUX; cohort 3A: no TD, RUX; cohort 3B: TD, RUX. Pts received subcutaneous LUSPA 1.0 mg/kg (1.33 mg/kg for pts in cohort 3B expansion) with titration up to 1.75 mg/kg in 21-d cycles. Disease response was assessed at d 169 and treatment extended for pts showing clinical benefit; pts were followed for ≥ 3 years (y) post last dose. The primary endpoint was anemia response (defined as 84 consecutive d: ≥ 1.5 g/dL hemoglobin [Hgb] from baseline without transfusion [cohorts 1 and 3A] or RBC transfusion-free [cohorts 2 and 3B]); secondary endpoints included duration of response (DOR), transfusion frequency, change in Hgb level, and symptom response. Results: Of 95 pts in the ITT group, the most common reason for treatment discontinuation was lack of clinical benefit at d 169 (n = 28). Median (range) age was 71.0 (50–89) y. Most (54.7%) pts had primary MF, intermediate-risk 2 disease (75.8%), and > 2 y since initial diagnosis (74.7%). Median prior RUX therapy was 18.1 months (cohorts 3A/3B) and 84.7% of pts had baseline transfusion burden 6–12 units/84 d (cohorts 2/3B). Mean treatment duration was 42.8 weeks. Cohort 3B had the highest anemia response rate with 26.3% (95% CI, 13.4–43.1) of pts transfusion independent during the primary treatment period and 31.6% (95% CI, 17.5–48.7) transfusion independent during the entire treatment period; median (range) DOR was 448 d (85–1582). In cohort 3B: 19 (50.0%) pts had reduced transfusion burden by ≥ 50% during the primary treatment period; 8 (21.1%) pts achieved mean Hgb increase ≥ 1.5 g/dL from baseline throughout the entire treatment period and 6 (15.8%) achieved a mean ≥ 50% reduction in symptom score from baseline. Overall, 47.4% of pts had ≥ 1 treatment-related adverse event (TRAE), most frequently hypertension (17.9%, 5.3% grade ≥ 3); 3.2% of pts had a serious TRAE. One TRAE led to LUSPA discontinuation. Two (2.1%) pts had transformation to acute myeloid leukemia. Nine pts died on treatment from: post-procedural hemorrhage, pneumonia, intracranial hemorrhage, septic shock, ischemic stroke, multiple organ dysfunction syndrome, renal failure, pneumonitis, and myelofibrosis (n = 1 each); no deaths were considered related to the study drug. Conclusions: In pts with MF, the safety profile of LUSPA was consistent with previous studies and efficacy results showed promising improvements in anemia and transfusion burden in all cohorts. Clinical trial information: NCT03194542 .
Many patients (pts) with myelofibrosis (MF) experience anemia due to disease or JAK inhibitor (JAKi) therapy.
El artículo presenta una propuesta de definición para el concepto de vulnerabilidad basada en limitantes de acceso a la información preventiva que se publica desde los sistemas de prevención del riesgo de desastres. Estas limitantes se han propuesto desde cuatro puntos de vista que abarcan diversos aspectos políticos, académicos, económicos y físicos que en conjunto pueden ocasionar que la sociedad expuesta a uno o varios fenómenos naturales vea incrementada su vulnerabilidad por no tener garantizado el acceso a las alertas difundidas por diferentes medios de comunicación. La nueva definición se construyó revisando la literatura existente asociada y realizando un análisis cualitativo apoyado en el software Nvivo utilizando herramientas como matrices de codificación y análisis de conglomerados. Se pudo establecer cuáles son las relaciones más fuertes que existen entre las restricciones planteadas y cómo corresponden a las propuestas de algunos autores a la vez que se establece si el enfoque del presente estudio ya ha sido utilizado previamente.
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