Hematopoietic cell transplantation (HCT) uses cytotoxic chemotherapy and/or radiation followed by intravenous infusion of stem cells to cure malignancies, bone marrow failure and inborn errors of immunity, hemoglobin and metabolism. Lung injury is a known complication of the process, due in part to disruption in the pulmonary microenvironment by insults such as infection, alloreactive inflammation and cellular toxicity. How microorganisms, immunity and the respiratory epithelium interact to contribute to lung injury is uncertain, limiting the development of prevention and treatment strategies.
View Article and Find Full Text PDFObjective: Despite the identified pathophysiology of vaso-occlusive pain in sickle cell disease (SCD), predictors of pain in youth with SCD remain elusive. In this study, we measured changes in pain frequency, intensity, and interference over 1 year and examined biopsychosocial risk factors (SCD disease severity, age, female, depression, and sleep quality) as possible longitudinal predictors.
Methods: Medical history was obtained from retrospective chart review for 79 children with SCD (ages 2-18 years; 48.
Congenital amegakaryocytic thrombocytopenia is a rare disorder causing thrombocytopenia that progresses to pancytopenia and bone marrow failure if untreated. It is caused by variants in the MPL gene which encodes the thrombopoeitin receptor. In this report, we review 5 cases of congenital amegakaryocytic thrombocytopenia, all of whom belong to the Mississippi Band of Choctaw Indians.
View Article and Find Full Text PDFThis report describes an infant with vomiting and significant weight loss attributable to cricopharyngeal achalasia, a rare finding in children. The infant responded to balloon dilation of the upper esophageal sphincter, with resolution of symptoms and return to presymptomatic growth parameters. A brief description of the clinical features, diagnostic evaluation, and treatment options for cricopharyngeal achalasia is included.
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