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Preterm birth remains one of the most urgent unresolved medical problems in obstetrics, yet only 2 therapeutics for preventing preterm birth have ever been approved by the United States Food and Drug Administration, and neither remains on the market. The recent withdrawal of 17-hydroxyprogesterone caproate (17-OHPC, Makena) marks a new but familiar era for obstetrics with no Food and Drug Administration-approved pharmaceuticals to address preterm birth. The lack of pharmaceuticals reflects a broad and ineffective pipeline hindered by extensive regulatory hurdles, soaring costs of performing drug research, and concerns regarding adverse effects among a particularly vulnerable population. The pharmaceutical industry has historically limited investments in research for diseases with similarly small markets, such as cystic fibrosis, given their rarity and diminished projected financial return. The Orphan Drug Act, however, incentivizes drug development for "orphan diseases", defined as affecting <200,000 people in the United States annually. Although the total number of preterm births in the United States exceeds this threshold annually, the early subset of preterm birth (<34 weeks' gestation) would qualify, which is predominantly caused by inflammation and infection. The scientific rationale for classifying preterm birth into early and late subsets is strong given that their etiologies differ, and therapeutics that may be efficacious for one subset may not work for the other. For example, antiinflammatory therapeutics would be expected to be highly effective for early but not late preterm birth. A robust therapeutic pipeline of antiinflammatory drugs already exists, which could be used to target spontaneous early preterm birth, in combination with antibiotics shown to sterilize the amniotic cavity. New applications for therapeutics targeting spontaneous early preterm birth could categorize as orphan disease drugs, which could revitalize the preterm birth therapeutic pipeline. Herein, we describe why drugs targeting early preterm birth should qualify for orphan status, which may increase pharmaceutical interest for this vitally important obstetrical condition.
Assuntos
Nascimento Prematuro , Gravidez , Feminino , Recém-Nascido , Humanos , Estados Unidos , Nascimento Prematuro/prevenção & controle , Nascimento Prematuro/tratamento farmacológico , Hidroxiprogesteronas/uso terapêutico , Preparações Farmacêuticas , Doenças Raras/tratamento farmacológico , Caproato de 17 alfa-Hidroxiprogesterona/uso terapêuticoRESUMO
The COVID-19 pandemic has disproportionately affected pregnant people by increasing health risks of maternal morbidity and mortality, stillbirth, and preterm birth. Although numerous studies have supported the safety and efficacy of COVID-19 vaccination in pregnancy in preventing or mitigating the risk for these adverse outcomes, many pregnant people remain hesitant. Approximately half of US adults regularly consume news from social media platforms, which are a fertile ground for the spread of vaccine disinformation. The lack of information regarding COVID-19 vaccine safety early in the pandemic fueled vaccine myths targeting the fears of pregnant people about vaccination risks. Saddened by the spike in maternal deaths of unvaccinated individuals during the COVID-19 Delta variant surge in the fall of 2021, we created a social media campaign to promote scientific communication regarding the risks of COVID-19 disease in pregnancy and the benefits of vaccination. We called the campaign "One Vax Two Lives," which refers to the ability of 1 maternal vaccine to benefit the health and lives of both the pregnant individual and their fetus. We present a blueprint of how we leveraged a large, interdisciplinary student workforce to create a social media campaign and research program studying vaccine hesitancy, which can be replicated by other groups. Community engagement and partnerships with key stakeholders, such as the Washington State Department of Health, were essential for amplifying the campaign and providing our team with feedback on content and approach. We present the analytics of our social media advertisements, web articles, and video content that helped inform the iterative design process of the multimedia content. Moving forward, we are launching collaborative research programs to study vaccine hesitancy and inform the development of new social media content designed for pregnant individuals who are: (1) Spanish-speaking Hispanic/Latina/x, (2) Black or Afro-Latinx, and (3) residents of rural communities in the State of Washington. Data from these mixed methods studies will inform new communication campaigns to reach vaccine-hesitant individuals. Finally, we discuss lessons learned and how the most impactful elements of the campaign can be translated to related areas of maternal public health.
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OBJECTIVES: Resistance to thyroid hormone (RTH) is a genetic condition, caused by mutations in the thyroid hormone receptor gene and characterized by impaired end organ responsiveness to thyroid hormone. Here we describe a novel case of THR associated with large goiter mimicking infiltrative c. CASE PRESENTATION: A 13-year-old male with a hyperthyroid phenotype of RTH diagnosed as a toddler, on methimazole and nadolol therapies presented with an increase in goiter size and possible nodule. Thyroid ultrasound was concerning for a diffuse infiltrative process or malignancy. Methimazole was discontinued and he underwent further imaging, fine needle aspiration and core biopsies. Biopsy results were reassuring and imaging findings were subsequently attributed to RTH rather than malignancy. He started every other day liothyronine therapy, which led to a decrease in goiter size, thyroglobulin level, and improvement of hyperthyroid symptoms. CONCLUSIONS: This is the first case to our knowledge describing the above thyroid imaging findings in association with RTH. It also adds important information to the pediatric literature regarding management of the hyperthyroid phenotype of RTH, including the role of liothyronine therapy.
Assuntos
Carcinoma , Bócio , Hipertireoidismo , Síndrome da Resistência aos Hormônios Tireóideos , Masculino , Humanos , Criança , Adolescente , Síndrome da Resistência aos Hormônios Tireóideos/complicações , Síndrome da Resistência aos Hormônios Tireóideos/diagnóstico , Síndrome da Resistência aos Hormônios Tireóideos/genética , Tri-Iodotironina , Metimazol , Hormônios Tireóideos , Bócio/diagnóstico , Hipertireoidismo/complicações , Carcinoma/complicaçõesRESUMO
(1) Background: Severe primary insulin-like growth factor-I deficiency (SPIGFD) is a rare disorder causing short stature in children due to low insulin-like growth factor 1 (IGF-1) levels. Given the sparsity of reported cases of SPIGFD worldwide, the condition may be underdiagnosed, potentially preventing affected children from receiving therapy with recombinant human IGF-1 (rhIGF-1). Our objective was to determine the prevalence of SPIGFD among children with short stature at a large pediatric tertiary care center through the use of a novel electronic medical record (EMR) algorithm. (2) Methods: We queried our EMR using an algorithm that detected all children seen at our center between 1 November 2013 and 31 August 2021 with short stature and low IGF-1. We then conducted chart reviews, applying established diagnostic criteria for those identified with potential SPIGFD. (3) Results: From a cohort of 4863 children with short stature, our algorithm identified 30 (0.6%) patients with potential SPIGFD. Using chart reviews, we determined that none of these patients had SPIGFD. (4) Conclusions: Our algorithm can be used in other EMRs to identify which patients are likely to have SPIGFD and thus benefit from treatment with rhIGF-1. This model can be replicated for other rare diseases.
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OBJECTIVES: Severe and very severe hypertriglyceridemia although rare within the pediatric population occur more often among oncology patients, secondary to chemotherapeutic agents. Currently there exists minimal literature to guide management of severe hypertriglyceridemia among pediatric patients. Very-low-fat dietary restriction should be considered over nil per os (NPO) for initial management of severe hypertriglyceridemia in stable pediatric patients. Pediatricians caring for oncology patients must consider chylomicronemia as a potential etiology for presenting symptoms. Pediatric severe hypertriglyceridemia management guidelines are needed as pediatricians must currently rely on anecdotal experiences for management decisions. CASE PRESENTATION: Three children receiving treatment for acute lymphoblastic leukemia required hospitalization for very severe hypertriglyceridemia. Management varied among the cases but included: NPO or very-low-fat diet, insulin, intravenous fluids, fibrates, and omega-3 fatty acids. CONCLUSIONS: These cases suggest that pediatric severe hypertriglyceridemia management, in the absence of pancreatitis should allow a very-low-fat diet initially rather than NPO followed by pharmacologic therapies.
Assuntos
Hipertrigliceridemia , Pancreatite , Leucemia-Linfoma Linfoblástico de Células Precursoras , Humanos , Criança , Hipertrigliceridemia/complicações , Hipertrigliceridemia/terapia , Leucemia-Linfoma Linfoblástico de Células Precursoras/terapia , Leucemia-Linfoma Linfoblástico de Células Precursoras/tratamento farmacológico , Pancreatite/terapia , Pancreatite/complicações , Insulina/uso terapêutico , Ácidos Fíbricos/uso terapêutico , TriglicerídeosRESUMO
The coronavirus disease 2019 (COVID-19) pandemic exposed the vulnerability of pregnant women to excess morbidity and mortality, as well as the disproportionate disease burden in certain racial, ethnic, and sociodemographic groups. Vaccine hesitancy represents a major threat to public health, and crafting messages that reach vulnerable groups and address their intersectionality remains a weakness for pandemic preparedness. We sought to investigate factors that influenced vaccine acceptance and social media ad response in a mixed-methods study of Spanish-speaking women living in the rural Western United States who were pregnant or recently pregnant between November 2022 and June 2023. Direct interviews were translated, transcribed, and coded, while the ad ratings were analyzed using linear mixed models. Participants most favorably rated ads that featured doctors and text-heavy content describing benefits of vaccination. Qualitative data illustrated how information from trusted medical providers along with generational and cultural history of vaccine acceptance positively impacted perspectives on vaccination. Immigration status had varying influences on vaccination perspectives. Future vaccination campaigns targeting Spanish-speaking pregnant individuals in rural communities should use medical providers as ad messengers and dispel fears that vaccine acceptance may lead to problems with immigration status.
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Pregnant women are a highly vaccine-resistant population and face unique circumstances that complicate vaccine decision-making. Pregnant women are also at increased risk of adverse maternal and neonatal outcomes to many vaccine-preventable diseases. Several models have been proposed to describe factors informing vaccine hesitancy and acceptance. However, none of these existing models are applicable to the complex decision-making involved with vaccine acceptance during pregnancy. We propose a model for vaccine decision-making in pregnancy that incorporates the following key factors: (1) perceived information sufficiency regarding vaccination risks during pregnancy, (2) harm avoidance to protect the fetus, (3) relationship with a healthcare provider, (4) perceived benefits of vaccination, and (5) perceived disease susceptibility and severity during pregnancy. In addition to these factors, the availability of research on vaccine safety during pregnancy, social determinants of health, structural barriers to vaccine access, prior vaccine acceptance, and trust in the healthcare system play roles in decision-making. As a final step, the pregnant individual must balance the risks and benefits of vaccination for themselves and their fetus, which adds greater complexity to the decision. Our model represents a first step in synthesizing factors informing vaccine decision-making by pregnant women, who represent a highly vaccine-resistant population and who are also at high risk for adverse outcomes for many infectious diseases.
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This mixed-method study investigated vaccine hesitancy among pregnant women living in rural western United States and their response to social media ads promoting COVID-19 vaccine uptake. Thirty pregnant or recently pregnant participants who live in rural zip codes in Washington, Oregon, California, and Idaho were interviewed between November 2022 and March 2023. Interviews were transcribed and coded, while the ad ratings were analyzed using linear mixed models. The study identified five main themes related to vaccine uptake, including perceived risk of COVID, sources of health information, vaccine hesitancy, and relationships with care providers. Participants rated ads most highly that used peer-based messengers and negative outcome-based content. Ads with faith-based and elder messengers were rated significantly lower than peer messengers (p = 0.04 and 0.001, respectively). An activation message was also rated significantly less favorably than negative outcome-based content (p = 0.001). Participants preferred evidence-based information and the ability to conduct their own research on vaccine safety and efficacy rather than being told to get vaccinated. Primary concerns of vaccine-hesitant respondents included the short amount of time the vaccine had been available and perceived lack of research on its safety during pregnancy. Our findings suggests that tailored messaging using peer-based messengers and negative outcome-based content can positively impact vaccine uptake among pregnant women living in rural areas of the Western United States.