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Introduction: Hereditary angioedema (HAE) is a rare genetic disease that impairs quality of life and could be life-threatening. The aim of this study was to apply a multicriteria decision analysis to assess the value of three long-term prophylactic (LTP) therapies for HAE in Spain., Methods: A multidisciplinary committee of 10 experts assessed the value of lanadelumab (subcutaneous use), C1-inhibitor (C1-INH; intravenous), and danazol (orally), using placebo as comparator. We followed the EVIDEM methodology that considers a set of 13 quantitative criteria. The overall estimated value of each intervention was obtained combining the weighting of each criterion with the scoring of each intervention in each criterion. We used two alternative weighting methods: hierarchical point allocation (HPA) and direct rating scale (DRS). A reevaluation of weightings and scores was performed., Results: Lanadelumab obtained higher mean scores than C1-INH and danazol in all criteria, except for the cost of the intervention and clinical practice guidelines. Under the HPA method, the estimated values were 0.51 (95% confidence interval [CI]: 0.44-0.58) for lanadelumab, 0.47 (95%CI: 0.41-0.53) for C1-INH, and 0.31 (95%CI: 0.24-0.39) for danazol. Similar results were obtained with the DRS method: 0.51 (95%CI: 0.42-0.60), 0.47 (95%CI: 0.40-0.54), and 0.27 (95%CI: 0.18-0.37), respectively. The comparative cost of the intervention was the only criterion that contributed negatively to the values of lanadelumab and C1-INH. For danazol, four criteria contributed negatively, mainly comparative safety., Conclusion: Lanadelumab was assessed as a high-value intervention, better than C1-INH and substantially better than danazol for LTP treatment of HAE.

Outcomes of out-of-hospital cardiac arrest are poor irrespective of the patient age group and circumstances. Survival to discharge after out-of-hospital arrest in children is less than 10%. Use of extracorporeal cardiopulmonary resuscitation is increasing and has been shown to improve outcomes in some situations. However, the candidacy for such augmentation is based on patient selection, institutional practices, and availability of an extracorporeal membrane oxygenation center. Often, duration of resuscitation, low flow state, presenting pH, and circumstances of arrest dictate candidacy for extracorporeal membrane oxygenation. We present a case of extremely prolonged resuscitation for out-of-hospital arrest in a pediatric patient, and we describe the use of mechanical compression device and transition to extracorporeal membrane oxygenation. We present the case outcome as well as brief discussion about controversies in extracorporeal cardiopulmonary resuscitation. We hope the case provides an opportunity for further discussion regarding opportunities to improve selection, use of extracorporeal cardiopulmonary resuscitation, and impact outcomes.

Idiopathic infantile arterial calcification is a rare cause of infantile ischemic cardiac failure with extremely poor prognosis. We present the first case report of successful extracorporeal membrane oxygenation support and outcome in a child with idiopathic infantile arterial calcification (IIAC). This 6-week-old infant presented with cardiogenic shock and circulatory collapse. The patient underwent extracorporeal cardiopulmonary resuscitation, allowing stabilization, diagnosis, and treatment with etidronate, followed by successful discharge to home.

Based on data drawn from the 2007 Madrid Regional Health Survey (MRHS), we sought to: estimate influenza vaccination coverage among people ages 16-59 years old suffering from chronic conditions (cardiovascular diseases, diabetes, respiratory diseases, kidney diseases, malignant neoplasm and fibromyalgia) living in the region of Madrid and to determine which socio-demographic and health-related variables were associated with the likelihood of being vaccinated. We analyzed data from 8,337 subjects. The reply to the question "Were you vaccinated against influenza in the last vaccination campaign?" was taken as the dependent variable. Coverage was calculated for different specific diseases that constitute an indication for vaccination. Independent variables included socio-demographics, health-related and use of health care services variables. The proportion of vaccinated adults suffering from any chronic condition in 2007 was 23.5%. Very low coverages were found among kidney diseases (16.1%), malignant neoplasm (10.9%) and fibromyalgia (14.2%) sufferers. The variables which increased the likelihood of being vaccinated among adults suffering any chronic condition were: higher age, being Spanish, absence of a smoking habit and having primary studies. We conclude that influenza vaccination coverage among people living in Madrid and suffering from a chronic condition is unacceptably low, thereby making it necessary for strategies to be urgently implemented aimed at improving the use of influenza vaccine.

Introduction: This uncontrolled study examined the outcome of a program of progressive cigarette reduction, using nicotine gum, as a prelude to complete cessation among 116 smokers (70 men, 46 women; mean age = 45.7 years, SD = 12.65; mean baseline smoking = 28.3 cigarettes/day) who sought treatment in a smokers' clinic but did not want to quit abruptly., Methods: Subjects participated in a two-stage program consisting of a 4-month reduction phase followed by a 6-month abstinence phase. The aim was to reduce the number of cigarettes smoked daily by at least 50% by Week 8 and to quit at the end of Week 16. During the reduction phase, subjects used nicotine gum (2 or 4 mg) to progressively decrease smoking. During the abstinence phase, subjects used any type of nicotine replacement therapy (NRT) to remain smoke free. Psychological treatment and NRT were provided at no cost to participants., Results: At Month 2, 76 subjects (68%) achieved the target of 50% reduction. At the target quit date in Week 16, 66 subjects (57%) achieved carbon monoxide-validated abstinence and 45 subjects (39%) maintained continuous abstinence at the 6-month follow-up. No symptoms of nicotine overdose were detected., Discussion: A program of progressive cigarette reduction using nicotine gum is feasible in practice and may be a useful strategy for smokers who are unable or unwilling to quit by abrupt cessation.

Background: Indications for extracorporeal membrane oxygenation therapy have expanded to include cardiopulmonary arrest and support after congenital heart surgery. Data from a national registry have reported that cardiac patients have the poorest survival of all extracorporeal membrane oxygenation recipients. Concerns have been raised about the appropriateness of such an aggressive strategy, especially in light of the high costs and potential for long-term neurologic disability. We reviewed our experience with salvage cardiac extracorporeal membrane oxygenation to determine the cost-utility, which accounts for both costs and quality of life., Methods: Medical records of patients with congenital heart disease receiving salvage cardiac extracorporeal membrane oxygenation between January 2000 and May 2004 were reviewed. Charges for all medical care after the institution of extracorporeal membrane oxygenation were determined and converted to costs by published standards. The quality-of-life status of survivors was determined with the Health Utilities Index Mark II., Results: Salvage cardiac extracorporeal membrane oxygenation was instituted in 32 patients (18 for cardiopulmonary arrest and 14 for cardiac failure after heart surgery) at a median age of 2.0 months (range, 4 days to 5.1 years). Congenital heart disease was present in 27 (84%). The mean duration of extracorporeal membrane oxygenation support was 5.1 +/- 4.1 days. Survival to hospital discharge was 50%, including 1 patient bridged to heart transplantation. Survival to 1 year was 47%. The mean score of the Health Utilities Index for the survivors was 0.75 +/- 0.19 (range, 0.41-1.0). The median cost for hospital stay after the institution of extracorporeal membrane oxygenation was USD 156,324 per patient. The calculated cost-utility for salvage extracorporeal membrane oxygenation in this population was USD 24,386 per quality-adjusted life-year saved, which would be considered within the range of accepted cost-efficacy (< USD 50,000 per quality-adjusted life-year saved)., Conclusions: Salvage cardiac extracorporeal membrane oxygenation results in reasonable survival and is justified on a cost-utility basis.

Background: A recent modification to the Norwood procedure involves the use of a right-ventricle (RV) to pulmonary artery (PA) conduit to provide pulmonary blood flow for patients with hypoplastic left heart syndrome (HLHS). This modification is thought to provide more stable hemodynamics by avoiding the diastolic "run-off" that occurs with a Blalock-Taussig shunt., Methods: We reviewed our experience with the first 11 patients undergoing the RV-PA conduit modification of the Norwood operation and compared their outcomes with those of the preceding 22 patients who underwent a conventional Norwood procedure., Results: Between July 1999 and March 2002, 33 patients with HLHS underwent the Norwood procedure at a median age of 5 days (range 1 to 31 days). Aortic atresia was present in 28 (85%). No significant difference was noted between the RV-PA (n = 11) and conventional Norwood (n = 22) groups with respect to measures of morbidity such as duration of mechanical ventilation or hospital stay. Patients who underwent the conventional Norwood procedure did have significantly lower diastolic blood pressure in the early postoperative period (38.4 +/- 4.4 mm Hg versus 49.5 +/- 4.3 mm Hg, p = 0.001). The operative and 1-year survival rates were 81% and 81%, respectively, for patients with the RV-PA modification, which was not significantly different from those of patients who underwent the conventional procedure, 81% and 73% (p = 1.00 and p = 0.36). Two patients developed a pseudoaneurysm of the RV infundibulum after placement of RV-PA conduit. Four sudden deaths occurred after hospital discharge, all occurring in the conventional Norwood group., Conclusions: The RV-PA conduit modification of the Norwood procedure results in excellent early survival. By avoiding low diastolic blood pressure this modification may provide superior perfusion to the coronary vascular bed and potentially reduce the risk of sudden unexpected death.

The management of the infant with congenital heart disease is a multidisciplinary collaborative effort that is individualized to each patient. Low cardiac output is frequently seen in the postoperative infant with arrhythmia, preload, afterload, and contractility alterations; it can be a significant complication after open heart surgery. The management of the younger patient, the higher acuity, and the high-technology environment of the cardiac intensive care unit require astute assessment and manipulation of therapies to minimize deleterious effects in caring for these patients.

Background: Excessive pulmonary blood flow increases ventricular volume work in the face of inadequate systemic cardiac output, low diastolic blood pressure, and inadequate coronary perfusion. Using the smallest available 3-mm polytetrafluoroethylene shunts have been successful, although catastrophic shunt thrombosis has occasionally been observed. To avoid thrombosis with a smaller conduit, saphenous vein homografts (SVG) were used to construct the modified Blalock-Taussig (BT) shunts., Methods: From January 1998 to April 1999, 25 patients weighing 3.1 kg (3.0 kg or less, n = 9), at a mean age of 8.9 days, underwent stage I Norwood using an SVG BT shunt. Common heart defects were aortic atresia (n = 8), mitral atresia and double-outlet right ventricle (n = 5), and unbalanced AVC (n = 5). Mean BT shunt size was 3.2 mm, with 12 patients having shunts that were 3 mm or smaller., Results: Thirty-day hospital mortality was 8% (2 of 25). No shunt thrombosis was seen, despite banding the BT shunt in 3 patients. One patient had BT revision because of an anatomic issue not directly related to the shunt material., Conclusions: Excellent results may be achieved using SVG BT shunts in the Norwood operation. This conduit seems less likely to thrombose, both acutely and chronically, allowing the use of appropriately smaller-sized shunts in small neonates.

Background: Cardiac transplantation is an accepted treatment for children with end-stage heart failure or complex or inoperable congenital defects., Methods: Since 1988, 95 transplants have been performed in 89 children aged 4 days to 18 years (median 6.9 years, 42 patients 0-5 years). Fifty-eight (61%) had congenital or acquired heart disease, 31 (33%) had idiopathic cardiomyopathy, and 6 (6%) were retransplants. Fifty-seven of the patients had prior cardiac surgery with a range of one to eight procedures (mean 3.4 procedures/patient). At the time of transplantation, 53 (56%) were United Network for Organ Sharing (UNOS) status I, including 23 children on mechanical ventilation and 4 with mechanical circulatory support., Results: Thirty-day survival in this group was 96%. Posttransplant results showed a median time of ventilation of 1 day (mean 3.0+/-5.7 days), median duration of inotropic support of 2 days (mean 2.7+/-2.3 days), median intensive care unit (ICU) stay of 4 days (mean 6.9+/-9.6 days), and median hospitalization of 9 days (mean 14.3+/-13.9 days). Follow-up from 1 month to 10.3 years (mean 3.1 years) has demonstrated a 1-year actuarial survival of 79% and a 5-year actuarial survival of 69%. Rejection, both acute and chronic, accounted for the vast majority of deaths., Conclusions: Pediatric heart transplantation can be accomplished with excellent early survival despite multiple prior cardiac operations and relative severity of illness. Parameters such as postoperative ventilation, inotropic support, ICU stay, and hospitalization can be kept at reasonable levels with acceptable long-term results, although rejection remains a serious problem.

To determine the effects of diabetic ketoacidosis on the brain, we studied nine consecutive patients between the ages of 6 and 17 years by performing cranial CT on three occasions: on admission, before treatment; 6-8 hr after beginning treatment; and 7 days after admission. Both the pretreatment scans and those at 6-8 hr showed significantly reduced size of the lateral and third ventricles in comparison with the 7-day scans. A comparison of the pretreatment scans with those done at 6-8 hr showed no statistically significant differences. We conclude that the cerebral swelling of diabetic ketoacidosis is usually present before treatment.

The use of a transthoracic bioimpedance monitor to determine cardiac output was evaluated in critically ill children. The children ranged in age from 10 months to 8 yr and their height and weight ranged from the third to the 97th percentile. Each child had a thermodilution catheter in place to monitor cardiac output. The bioimpedance monitor used in this study, the NCCOM-3, required the input of a constant (L), which was obtained for each individual patient by adjusting the L setting until cardiac output measured by bioimpedance (COBI) was within 10% of cardiac output measured by thermodilution (COTD). This method of determining L was superior to using either measured thoracic length or the manufacturer's guidelines to obtain L and resulted in an excellent correlation between COTD and COBI (r = .94; p less than .05; n = 59). In children less than 125 cm in height, measured thoracic length alone was inadequate to use for L but provided a good approximation of L when multiplied by 1.25. This study suggests that the use of transthoracic bioimpedance to determine cardiac output compares favorably with thermodilution techniques and it is noninvasive.