Afamelanotide for Treatment of the Protoporphyrias: Impact on Quality of Life and Laboratory Parameters in a US Cohort.

BACKGROUND: Erythropoietic protoporphyria (EPP) and X-linked protoporphyria (XLP) are rare disorders of heme biosynthesis characterized by severe cutaneous phototoxicity. Afamelanotide, an α-melanocyte-stimulating hormone analogue, is the only approved treatment for protoporphyria and leads to increased light tolerance and improved quality of life (QoL). However, published experience with afamelanotide in the US is limited. METHODS: Here, we report on all adults who received at least one dose of afamelanotide at the Massachusetts General Hospital Porphyria Center from 2021 to 2022. Changes in the time to phototoxic symptom onset, QoL, and laboratory parameters were assessed before and during treatment with afamelanotide. RESULTS: A total of 29 patients with protoporphyria were included, 26 of whom (72.2%) received ≥2 afamelanotide implants. Among the patients who received ≥2 implants, the median time to symptom onset following sunlight exposure was 12.5 min (IQR, 5-20) prior to the initiation of afamelanotide and 120 min (IQR, 60-240) after treatment (p < 0.001). Improvements in QoL during afamelanotide treatment were measured using two QoL tools, with good correlation observed between these two instruments. Finally, we found no improvements in the median levels of metal-free erythrocyte protoporphyrin, plasma protoporphyrin, or liver biochemistries during versus prior to the initiation of afamelanotide treatment. CONCLUSIONS: This study highlights a dramatic clinical benefit of afamelanotide in relation to light tolerance and QoL in protoporphyria, albeit without improvement in protoporphyrin levels or measures of liver function.

Dose Optimization of the Administered Activity in Pediatric Bone Scintigraphy: Validation of the North American Consensus Guidelines.

UNLABELLED: The 2010 North American Consensus Guidelines (NACG) for pediatric administered doses and the European Association of Nuclear Medicine (EANM) Dosage Card guidelines recommend lower activities than those administered at our institution. We compared the quality of the lower-activity images with the higher-activity images to determine whether the reduction in counts affects overall image quality. METHODS: Twenty patients presenting to our pediatric radiology department for bone scintigraphy were evaluated. Their mean weight was 20 kg. The patients were referred for oncologic (n = 10), infectious/inflammatory (n = 5), and pain (n = 5) evaluation. Dynamic anterior and posterior images were acquired for 5 min for each patient. Data were subsampled to represent different administered activities corresponding to the activities recommended by the NACG and the EANM Dosage Card. Images were evaluated twice, first for diagnostic quality and then for acceptability for daily clinical use. RESULTS: There was no statistically significant difference in the diagnostic quality of the images from any of the 3 protocols. Pathologic uptake was correctly identified independent of the administered activity, although there was a single false-positive result for an EANM image. When images were subjectively evaluated as acceptable for daily clinical use, there was a slight preference for the higher-activity images over the NACG (P = 0.04). CONCLUSION: The recommended administered activities of the NACG produce images of diagnostic quality while reducing patient radiation exposure.

The essence of life purpose.

Life purpose is an important thread of critical care nursing. However, no consensus exists for a definition of life purpose. In addition, ambiguity prevails regarding the manner in which life purpose is incorporated into nursing practice and research. Therefore, through a conceptual synthesis process, this article aims to clarify the essence of life purpose with relevance to health and critical care nursing today. The outcome of the conceptual synthesis is an operational definition to be used in future nursing research. Information was obtained from a literature search of scholarly articles using (1) searches of electronic databases of literature about life purpose and (2) research studies addressing conceptual, substantive, and methodological domains. Topics consisted of the philosophical underpinnings of life purpose, its attributes, definitions, and theoretical frameworks, along with differences in theories and empirical support. Finally, emerging from this process, the article culminates with a proposed conceptual definition of life purpose, which may be applied broadly to older adults in various critical care settings.

Heart failure: epidemiologic update.

Heart failure is a disease of epidemic proportions in the United States affecting almost 6 million people. This heart failure overview includes a brief description of the etiology of this extremely prevalent coronary artery disease. Myocardial (ventricular) remodeling is described as being either physiological or pathological. Patients must initially be taught that heart failure is a chronic and ongoing disease to comprehend the need for lifestyle changes and management of life problems. Because adherence is a key determinate of clinical outcomes, promoting patient adherence with medications is a major focus for clinicians working with patients with heart failure. Various diagnostic tests are explained as well as the treatment of heart failure. Treatment includes pharmacologic, nonpharmacologic, and surgical interventions. Although there have been substantial advances in care and management, this information is important for healthcare service professionals to know because the number of people diagnosed with heart failure continues to increase each year.

Factors impacting readmissions of older patients with heart failure.

The overall purpose of this study was to explore individual perceptions of life purpose, health-related quality of life, and hospital readmissions among older adults with heart failure. The problem addressed the heart failure patient's lack of understanding about the diagnosis, treatment, management, and predictors of heart failure within a framework of the older adult's life purpose and health-related quality of life. The goal was to provide a foundation for development of safe and effective holistic intervention strategies to decrease costly hospital readmissions for patients with heart failure. The research design was descriptive, correlational mixed method using a qualitative and quantitative concurrent triangulation. The setting for the study was San Antonio, Texas, the seventh largest city in the United States. The purposive sample consisted of 41 male and female participants aged 60 years and older. The research findings suggest a moderate, significant relationship between life purpose and health-related quality of life. Higher numbers of hospital readmissions were significantly related to more difficulty with management of heart failure and a poor sense of life purpose. This study may contribute to science by providing useful information that may be used in the management of heart failure among older adults.

Collaboration with city agencies: a winning approach to community assessment.

Five graduate nursing students made a difference in the health of San Antonio citizens by conducting a community assessment as part of a standard clinical activity in their community health nursing course. The students and their professor were able to effect city-wide change for health protection and promotion through collaboration with the City of San Antonio Planning Department (CSAPD). By compiling information, linking organizations, and speaking before community groups about the importance of a fluoridated water supply, the students generated public interest and momentum, which resulted in a successful vote to add fluoride to the water supply. In addition, they were able to add to the assessment compiled by the Planning Department employees and increase the CSAPD's awareness of health concerns as an important area to assess within a community. The resulting assessment was more comprehensive than it would have been without the students' input. This use of collaboration provides a model other faculty may adopt for community assessment.