Clinical effectiveness and safety of sirolimus in pediatric patients with complex vascular anomalies: necessitating personalized and comprehensive approaches

BackgroundManaging complex vascular anomalies in pediatric care requires comprehensive approaches. Sirolimus, an mTOR inhibitor with immunosuppressive and anti-angiogenic properties, offers promise. We evaluated sirolimus's effectiveness and safety in pediatric patients with complex vascular anomalies at a tertiary children's hospital.MethodsOur study included 20 patients, aged 1 month to 19 years, with diverse vascular anomalies resistant to conventional therapies or located in high-risk areas precluding surgery. The evaluation of response encompassed measuring the reduction in the size of the targeted vascular or lymphatic lesions as observed on radiologic imaging, along with considering improvements reported by the patients.ResultsPatients used sirolimus for a median of 2.1 years, ranging from 0.6–4.3 years. Results indicated that 60% of patients achieved complete or partial response (CR/PR), whereas 40% had stable disease (SD). Notably, no disease progression occurred. Lesion size assessment was complex, yet patients' self-reported improvements were considered. Three patients reinitiated sirolimus after discontinuation due to worsening lesions. Sirolimus treatment demonstrated good tolerability, with minor complications except for one case of Pneumocystis jiroveci pneumonia. Group comparisons based on response highlighted better outcomes in patients with vascular tumors (CR/PR group 58.0% vs. SD group 0.0%, P = 0.015) or localized measurable lesions (83.3% vs. 12.5%, P = 0.005).ConclusionOur study underscores sirolimus's potential for treating complex vascular anomalies in pediatric patients. Challenges associated with optimal treatment duration and concurrent interventions necessitate a comprehensive approach and genetic testing to optimize outcomes

Successful mobilization using a combination of plerixafor and G-CSF in pediatric patients who failed previous chemomobilization with G-CSF alone and possible complications of the treatment

Peripheral blood stem cell (PBSC) mobilization, which uses plerixafor (AMD 3100), a newly developed specific inhibitor of the CXCR4 receptor, in combination with granulocyte-colony stimulating factor(G-CSF), has been shown to enhance the stem cell mobilization in adult patients, but pediatric data are scarce. We documented our experience with this drug in 6 Korean pediatric patients who had failed in chemomobilization, using G-CSF, alone. All patients were mobilized CD34+ cells (median, 11.08 × 106/kg: range, 6.34-28.97 × 106/kg) successfully within 2 to 3 cycles of apheresis, without complications. A total of 7 autologous transplantations were performed, including 1 tandem transplantation. However, 2 patients with brain tumors showed severe pulmonary complications, including spontaneous pneumomediastinum. This is the first study of PBSC mobilization with plerixafor in Asian pediatric patients. Furthermore our study suggests that mobilization with plerixafor may be effective in Korean pediatric patients, who have previously been heavily treated and have failed PBSC mobilization with classical chemomobilization, using G-CSF. However, further studies are needed to examine the possible complications of autologous transplantation, using a mobilized plerixafor product in children

The effect of hidden female smoking on the relationship between smoking and cardiovascular disease

Background: Smoking is a known risk factor for cardiovascular morbidity and mortality, but several Korean studies have shown differing results on the association of current smoking status and the risk of cardiovascular disease (CVD). The aim of the present study was to investigate the association between smoking status and CVD (myocardial infarction and stroke) using national representative populationbased samples. The aim was also to investigate the effects of hidden smokers on the association between CVD and smoking.Methods: Data were acquired from 28,620 participants (12,875 men and 15,745 women), age 19 years or older, who participated in the Korea National Health and Nutrition Examination Survey (KNHANES) conducted from 2008 to 2016.Results: The multivariable logistic regression analysis showed that ex-smoking status was correlated with CVD when self-reported (odds ratio [OR]: 1.62; 95% confidence interval [CI]: 1.20–2.19) and for survey-cotinine verified-smoking status (OR: 1.57; 95% CI: 1.20–2.19). Interestingly, the present study showed current smoking was not significantly associated with CVD. For the effect of sex on smoking and CVD, self-reported and survey-cotinine-verified ex-smoking status were correlated with CVD in males (OR: 1.45; 95% CI: 1.04–2.04 and OR: 1.43; 95% CI: 1.02–2.02) and in females (OR: 2.74; 95% CI: 1.59–4.71 and OR: 2.92; 95% CI: 1.64–5.18). The ratios of cotinine-verified to self-reported smoking rates were 1.95 for women and 1.08 for men.Conclusions: In the current study, while ex-smoking status was significantly associated with CVD, current smoking status was not. Female ex-smoking status had a higher adjusted odds ratio for CVD than males compared to non-smoking status. An effect of hidden female smoking was also found on the association between smoking status and CVD in Korean adults

Characteristics of Body Fat, Body Fat Percentage and Other Body Composition for Koreans from KNHANES IV

Accurate measurement of fat mass has become increasingly important with the increasing incidence of obesity. We assessed fat and muscle mass of Koreans with the Korea National Health and Nutrition Examination Survey IV (KNHANES IV). We studied 10,456 subjects (aged 20 to 85 yr; 4,476 men, 5,980 women). Fat and muscle mass were measured by dual-energy x-ray absorptiometry. Reference values of body compositions were obtained using the LMS method. The fat mass index (FMI, body fat mass/height2; kg/m2) of Korean men did not correlate with age (P = 0.452), but those of Korean women (P < 0.001) did. The ratio of percentage of fat in the trunk and legs was positively related with age in both the genders. The appendicular lean mass/height2 (kg/m2) of Korean men was negatively related to age (P < 0.001). In women, this ratio increased with age (P < 0.001). When we defined obesity according to the FMI classification, the rates of obesity were 6.1% (FMI > 9 kg/m2) in men and 2.7% (FMI > 13 kg/m2) in women. It is concluded that the muscle mass decreases and obesity increases with aging in Korean men, whereas both fat mass and obesity increase with aging in Korean women

A Pulmonary Nodule due to Pulmonary Infarction Diagnosed by Video-Assisted Thoracoscopy

We report a pulmonary infarction in 68-year-old man who was referred for an asymptomatic pulmonary nodule in chest radiography. Computed tomography (CT), positron emission tomography (PET), and transthoracic needle aspiration suggested suspicion for malignancy. Video-assisted thoracoscopic surgery (VATS) was performed for histologic diagnosis. Our case is a pulmonary nodule due to pulmonary infarction diagnosed by VATS in Korea

Hemorrhage from a jejunal polypoid hemangioma: single incisional laparoscopic approach

Bleeding lesions in the small bowel are a much more significant challenge in terms of detection and treatment than those of the stomach or the large bowel, and require extensive gastrointestinal evaluation before a diagnosis can be made. The authors report the case of an 81-year-old female patient who underwent small bowel segmental resection by single incisional laparoscopic approach for distal jejunalhemangioma, which caused severe anemia. An abdominal computed tomography scan demonstrated a highly enhancing polypoid tumor in the distal ileum. During the single incisional laparoscopic exploration using a 2 cm sized skin incision, jejuno-jejunal intussusceptions and a jejunal tumor were noted. Single incisional laparoscopy was performed to assist the jejunal segmental resection. Pathologic reports confirmed the lesion to be a jejunalhemangioma. The authors report an unusual case of jejunalhemangioma caused by intussusception and gastrointestinal hemorrhage, which was treated by single incisional laparoscopic surgery