Management of neonatal and infant aortic thrombosis: A single-center case series.

Neonatal and infant aortic thrombosis is a rare albeit life-threatening thrombotic event, particularly seen in premature infants with an arterial catheter in place. We describe our institutional experience and approach to the management of 11 infants with occlusive or nearly occlusive aortic thrombosis. We observed at least partial thrombus resolution in all patients. Complications related to our management included minor bleeding in two children receiving thrombolytic therapy, and two major bleeding events in children receiving anticoagulation alone. Our experience adds to the growing body of evidence that thrombolysis and thrombectomy should be considered in managing neonatal/infant aortic thrombosis.

Concomitant sickle cell disease and systemic lupus erythematosus: A single-center case series.

Sickle cell disease (SCD) and systemic lupus erythematosus (SLE) are two uncommon disorders each characterized by multisystemic manifestations. Individuals with SCD exhibit abnormalities in the complement pathway, which may predispose patients to develop autoimmune disorders such as SLE. As many manifestations of SLE mimic those of SCD, diagnosis and therapeutic management of SLE in a patient with known SCD may be delayed. In this study, we describe our institutional experience of diagnosing and managing concomitant SCD and SLE. We offer insights into the complex interplay between these conditions to enhance early recognition and effective management of concurrent SCD and SLE.

Eculizumab for management of hyperhemolysis syndrome in pediatric patients with sickle cell disease: A single-center case series.

Chronic hemolytic anemia and vascular occlusion are hallmarks of sickle cell disease (SCD). Blood transfusions are critical for supportive and preventive management of SCD complications. Patients with SCD are at risk for hyperhemolysis syndrome (HHS), a subtype of delayed hemolytic transfusion reactions. HHS management includes intravenous immunoglobulin, corticosteroids, and avoidance of further transfusions. Not all patients respond to first-line agents. Eculizumab, which blocks terminal complement activation, has been proposed as second-line management of HHS. We describe two patients who received eculizumab for refractory HHS. In our experience, eculizumab is a safe and effective option for refractory pediatric HHS.

New Horizons in Emergency Department Management of Pediatric Sickle Cell Disease.

ABSTRACT: Sickle cell disease (SCD) is an important topic for emergency medicine audiences because complications of the disease account for a large proportion of hematologic emergencies that are seen in the emergency department each year. Early recognition and aggressive management of emergency complications of SCD can help to reduce the morbidity and mortality associated with this disease. Although the treatment recommendations for some complications of SCD are based on expert opinion, there has been advancement in the understanding of the pathogenesis of the disease and evidence regarding the treatment options available for managing acute complications. This continuing medical education article will provide a summary of the clinical manifestation and management of the most common acute complications of SCD: infection, vaso-occlusive episode, acute chest syndrome, splenic sequestration, stroke, and priapism.

A Qualitative Systematic Review of Pediatric Patient and Caregiver Perspectives on Pain Management for Vaso-Occlusive Episodes in the Emergency Department.

OBJECTIVES: The primary objective of this study is to describe the experiences of pediatric patients with sickle cell disease (SCD) and their caregivers who have presented to the emergency department (ED) for management of vaso-occlusive pain events. METHODS: We conducted a qualitative systematic review. The search protocol was developed to identify both published and unpublished literature that met inclusion/exclusion criteria. Included articles were primary hospital-based research with study populations that included (but were not limited to) pediatric patients aged 21 years or younger and qualitative or mixed-method analysis. RESULTS: Four themes were identified: (1) patients and caregivers perceive the ED as the last resort; (2) health care professionals in the ED lacked knowledge about SCD but rejected patients' and caregiver's attempts to share experience or advocate for their needs; (3) patients' accounts of pain are doubted because they do not always have "typical" signs of pain; and (4) caregivers identify racism as a reason for suboptimal care in the ED. CONCLUSIONS: There are multiple opportunities to improve management for vaso-occlusive pain events in the ED, including education of health care providers about SCD and complications, partnership between patients/caregivers and providers, and efforts to reduce the impact of systemic racism on health care delivery.

Long-Term Kidney and Cardiovascular Complications in Pediatric Cancer Survivors.

OBJECTIVE: The objective of this study was to describe the burden of adverse kidney and hypertension outcomes in patients evaluated by pediatric nephrology in a multidisciplinary survivorship clinic. STUDY DESIGN: Retrospective chart review of all patients followed up by nephrology in our multidisciplinary survivorship clinic from August 2013 to June 2021. Data included clinic blood pressure, longitudinal ambulatory blood pressure monitoring (ABPM), echocardiography, serum creatinine, and first-morning urine protein/creatinine ratios. For patients with multiple ABPMs, results of initial and most recent ABPMs were compared. RESULTS: Of 422 patients followed in the multidisciplinary cancer survivorship clinic, 130 were seen by nephrology. The median time after therapy completion to first nephrology visit was 8 years. The most common diagnoses were leukemia/myelodysplastic syndrome (27%), neuroblastoma (24%), and Wilms tumor (15%). At the last follow-up, 68% had impaired kidney function, 38% had a clinical diagnosis of hypertension, and 12% had proteinuria. There were 91 ABPMs performed in 55 (42%) patients. Patients with multiple ABPMs (n = 21) had statistically significant reductions in overall median blood pressure loads: systolic initial load 37% vs most recent 10% (P = .005) and diastolic load 36% vs 14% (P = .017). Patients with impaired kidney function were more likely to have received ifosfamide. Patients with hypertension were more likely to have received total body irradiation or allogeneic stem cell transplant. CONCLUSIONS: History of leukemia/myelodysplastic syndrome, neuroblastoma, and Wilms tumor was frequent among survivors seen by nephrology. There was significant improvement in cardiovascular measures with increased recognition of hypertension and subsequent treatment.

A Qualitative Study of Caregiver Perspectives on Health Policy Discussions Initiated by Pediatricians.

BACKGROUND AND OBJECTIVE: Prior studies suggest that pediatricians believe discussing health policy issues with families is important. Caregiver preferences on these discussions, however, have not been examined. We explored circumstances in which caregivers may be receptive to discussing health policy issues with pediatricians. METHODS: We conducted 26 semistructured interviews with mostly Black female caregivers at 3 urban academic pediatric primary care practices. Using both structured and open response questions, we explored 4 primary content areas: 1) caregivers' perspectives on discussing health policy issues in pediatricians' offices; 2) which health policy topics caregivers may prefer to discuss; 3) factors that render policy discussions in the clinic inappropriate to caregivers; and 4) which communication modalities caregivers prefer. Interview transcripts were coded and analyzed using content analysis. RESULTS: Themes that emerged from interviews included: 1) pediatricians are perceived as trusted information sources on health policy; 2) caregivers want to talk with pediatricians about children's health insurance policy changes; 3) time constraints are a barrier to health policy discussions; 4) caregivers prefer to discuss health policy topics during well-child visits; 5) caregivers want the option to opt-out of these conversations; 6) preferred modalities for communicating about health policy issues, including printed materials and health fairs or educational events. CONCLUSIONS: Caregivers expressed a satisfactory view of pediatricians discussing directly relevant health policy issues, including congressional debates on health insurance, in the primary care setting. Our findings highlight other caregiver preferences for engaging families in health policy discussions, including the timing of these conversations.

Heterotrimeric G-protein, Gα16, is a critical downstream effector of non-canonical Wnt signaling and a potent inhibitor of transformed cell growth in non small cell lung cancer.

G-protein-coupled receptors (GPCR) are the largest family of cell surface molecules that play important role/s in a number of biological and pathological processes including cancers. Earlier studies have highlighted the importance of Wnt7a signaling via its cognate receptor Frizzled9, a GPCR, in inhibition of cell proliferation, anchorage-independent growth, and reversal of transformed phenotype in non small cell lung cancer primarily through activation of the tumor suppressor, PPARγ. However, the G-protein effectors that couple to this important tumor suppressor pathway have not been identified, and are of potential therapeutic interest. In this study, by using two independent Wnt7a/Frizzled9-specific read-outs, we identify Gα16 as a novel downstream effector of Wnt7a/Frizzled9 signaling. Interestingly, Gα16 expression is severely down-regulated, both at the messenger RNA levels and protein levels, in many non small cell lung cancer cell lines. Additionally, through gene-specific knock-downs and expression of GTPase-deficient forms (Q212L) of Gα16, we also establish Gα16 as a novel regulator of non small cell lung cancer cell proliferation and anchorage-independent cell growth. Taken together, our data not only establish the importance of Gα16 as a critical downstream effector of the non-canonical Wnt signaling pathway but also as a potential therapeutic target for the treatment of non small cell lung cancer.