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Six villages in Boko Health Zone, Bandundu Province, DRC, were studied with 4588 people, 144 konzo cases and konzo prevalences of 2.0–5.2%. Konzo incidence is increasing rapidly in this area. Food consumption scores were obtained from the households with konzo and the mean % malnutrition calculated for each village. Urine samples were obtained from 50 school children from each village and % high urinary thiocyanate content (>350 μmol/L) determined. The experimental data relating % konzo prevalence (%K) to % children with high urinary thiocyanate content (%T) and % malnutrition (%M) for the six villages were fitted to an equation %K = 0.06%T + 0.035%M. This confirms that konzo is due to a combination of high cyanide intake and malnutrition. The village women used the wetting method to remove cyanogens from cassava flour. During the 9-month intervention there were no new cases of konzo; cyanide in flour had reduced to WHO safe levels and mean urinary thiocyanate levels were greatly reduced. To prevent konzo at least 60–70% of women should use the wetting method regularly. The wetting method is now accepted by the World Bank, FAO and WHO as a sensitive intervention. Four successful konzo interventions have involved nearly 10,000 people in 13 villages, the cost is now $16 per person and the methodology is well established.

Objective: To design, develop, and field test the HPV Decide decision tool to facilitate shared clinical decision-making recommendation for mid-adult HPV vaccination., Methods: The 'HPV Decide' online tool was developed through a 6-step process, involving community and provider advisory boards, usability testing with 10 end users (unvaccinated adults aged 27-45), field testing interviews with another 10, and interviews with 18 healthcare providers. The process incorporated both inductive and deductive qualitative data analyses., Results: The logic model, based on the Ottawa Decision Support Framework, highlighted the importance of objective knowledge and managing decisional conflict. Iterative modifications were informed by the advisory boards, alpha testing, and usability testing. Field testing showed utility to end users, highlighting the straightforward design, and lack of medical jargon. Many expressed a desire to access the tool before doctor appointments, emphasizing its value in a healthcare context and in partnership with providers. Lastly, healthcare provider feedback highlighted information quality, structure, acceptability, and future implementation., Discussion: While users recognized the tool's information quality and user-centric design, full evaluation focused on changes in informed decision making and clinic-based implementation are needed., Practical Value: The HPV Decide decision tool demonstrates promise in supporting informed decisions about HPV vaccination among mid-adults., Competing Interests: Declaration of competing interest The authors declare the following financial interests/personal relationships which may be considered as potential competing interests: Dr. Thompson is a paid consultant for Merck for work on HPV vaccination. Dr. Zimet has received consulting fees from Merck for work on HPV vaccination, has served on external advisory committees for Moderna (COVID-19 vaccination) and Pfizer (meningococcal vaccination), and has received investigator-initiated grant funding from Merck administered through Indiana University., (Copyright © 2024 Elsevier Ltd. All rights reserved.)

Abstract: Chronically transfused patients with sickle cell disease typically do not exhibit iron-mediated extrahepatic toxicity. However, we demonstrate that the pituitary gland is vulnerable to iron deposition, and it occurs regardless of other extrahepatic involvement. Severe pituitary siderosis is associated with early organ dysfunction., (© 2024 American Society of Hematology. Published by Elsevier Inc. All rights are reserved, including those for text and data mining, AI training, and similar technologies.)

Gastrointestinal manifestations in patients with cystic fibrosis (CF) are extremely common and have recently become a research focus. Gastrointestinal (GI) dysfunction is poorly understood in the CF population, despite many speculations including the role of luminal pH, bacterial overgrowth, and abnormal microbiome. Nevertheless, dysmotility is emerging as a possible key player in CF intestinal symptoms. Our review article aims to explore the sequelae of defective cystic fibrosis transmembrane conductance regulator (CFTR) genes on the GI tract as studied in both animals and humans, describe various presentations of intestinal dysmotility in CF, review newer diagnostic motility techniques including intraluminal manometry, and review the current literature regarding the potential role of dysmotility in CF-related intestinal pathologies., Competing Interests: Declaration of Competing Interest The authors whose names are listed immediately below certify that they have NO affiliations with or involvement in any organization or entity with any financial interest (such as honoraria; educational grants; participation in speakers’ bureaus; membership, employment, consultancies, stock ownership, or other equity interest; and expert testimony or patent-licensing arrangements), or non-financial interest (such as personal or professional relationships, affiliations, knowledge or beliefs) in the subject matter or materials discussed in this manuscript., (Copyright © 2021 The Authors. Published by Elsevier B.V. All rights reserved.)

Pain and vaso-occlusive crises (VOC) are hallmark complications of sickle cell disease (SCD) and result in significant physical and psychosocial impairment. The variability in SCD pain frequency and triggers for the transition from steady state to VOC are not well understood. This paper summarizes the harmful physiological effects of pain and emotional stressors on autonomically-mediated vascular function in individuals with SCD and the effects of a cognitive, neuromodulatory intervention (i.e. hypnosis) on microvascular blood flow. We reviewed recent studies from the authors' vascular physiology laboratory that assessed microvascular responses to laboratory stressors in individuals with SCD. Results indicate that participants with SCD exhibit marked neurally mediated vascular reactivity in response to pain, pain-related fear, and mental stress. Further, pilot study results show that engagement in hypnosis may attenuate harmful microvascular responses to pain. The collective results demonstrate that autonomically-mediated vascular responses to pain and mental stress represent an important SCD intervention target. This ongoing work provides physiological justification for the inclusion of cognitive, neuromodulatory and complementary treatments in SCD disease management and may inform the development of targeted, integrative interventions that prevent the enhancement of autonomic vascular dysfunction in SCD., (Copyright © 2020 Elsevier Ltd. All rights reserved.)

The 2016 World Health Organization (2016 WHO) classification of hematopoietic malignancies classifies neoplasms with a fusion between the FIP1L1 and PDGFRA genes in 4q12 into a group called "myeloid and lymphoid neoplasms with eosinophilia and abnormalities of PDGFRA, PDGFRB or FGFR1 or with PCM1-JAK2". Neoplasms characterized by this fusion are pluripotent stem cell disorders that can show both myeloid and lymphoid differentiation. They typically occur in adult patients and most are characterized by eosinophilia. We describe identification of a FIP1L1-PDGFRA fusion in a 13-year-old boy who presented with T-lymphoblastic leukemia/lymphoma without eosinophilia. Detection of FIP1L1-PDGFRA driven neoplasms at diagnosis is usually critical for proper treatment, since almost all reported cases responded to tyrosine kinase inhibitors. However, our patient's leukemia was refractory to standard chemotherapy, and did not show a meaningful response to tyrosine kinase inhibitor therapy. Testing for a FIP1L1-PDGFRA rearrangement is at present limited to patients with idiopathic hypereosinophilia, and we hypothesize that this abnormality may be under-diagnosed in children with acute leukemias., (Copyright © 2017 Elsevier Inc. All rights reserved.)

RISE-SSc is a randomized, double-blind, placebo-controlled phase 2 study investigating the efficacy and safety of riociguat in patients with diffuse cutaneous systemic sclerosis (dcSSc). Based on positive results from riociguat trials in patients with pulmonary hypertension and chronic thromboembolic pulmonary hypertension in combination with the known antiproliferative and antifibrotic effects seen in animal models, patients with SSc may benefit from treatment with riociguat. Patients with SSc meeting the ACR/EULAR systemic sclerosis classification criteria with diffuse cutaneous SSc (dcSSc) subset per LeRoy criteria, and a disease duration of less than or equal to 18 months will be randomized to placebo or riociguat 0.5 mg (up-titrated to a maximum dose of 2.5 mg TID over 10 weeks) and maintained on therapy for a total of 52 weeks. During the first 10 weeks of the long-term extension phase, placebo subjects will be up-titrated on riociguat, and all patients will be followed for up to 6 years. The primary endpoint of change in modified Rodnan skin score (mRSS) from baseline will be assessed at 52 weeks, as will be secondary endpoints such as mRSS progression and regression rates, patient quality of life, digital ulcer burden, and change in forced vital capacity and carbon monoxide diffusing capacity. This review will further define the clinical rationale for the use of riociguat in the treatment of SSc and provide details on study protocol, design, and outcome reporting., Trial Registration: Clinicaltrials.gov identifier: NCT02283762., (Copyright © 2016. Published by Elsevier Ltd.)