Treatment Individualization Using Pharmacokinetic Studies and Joint Ultrasound Imaging in Pediatric Patients With Hemophilia.

Hemophilia is characterized by bleeding diathesis, primarily affecting the joints. Prophylactic use of missing factor aims at limiting the number of bleeds and, in the long term, the risk of permanent joint damage. However, standard prophylactic regimens are usually applied empirically, not adjusting for variations in bleeding phenotype or drug metabolism. Aim of the present study was to evaluate the need for individualizing prophylaxis, with guidance of pharmacokinetic (PK) studies and joint ultrasound in a setting of everyday clinical practice. To evaluate adequacy of applied regimens, joint status was assessed using the Hemophilia Joint Health Score as well as ultrasound imaging, while PK studies were performed using the Web-Accessible Population Pharmacokinetic Service-Hemophilia. Imaging results were consistent with early joint damage in a large proportion of pediatric patients, whereas PK measures were indicative of inadequate prophylaxis in many cases-despite the limited number of bleeds reported by patients. The study revealed the need for prophylaxis adjustment in the majority of patients. Real world data confirm that traditional prophylaxis is often unable to achieve therapeutic goals, while an individualized approach, guided by the use of novel modalities, may be of great benefit to young hemophilia patients.

How Parents' Lack of Awareness Could Be Associated With Foreign Body Aspiration in Children.

Foreign body aspiration (FBA) in preschool children is a worldwide challenging clinical problem that can result in life-threatening complications. Three cases of FBA in preschool children are presented. All the children were admitted to the hospital with asthma-like symptoms, without any aspiration history. Although FBA was considered in differential diagnosis, there was no strong evidence to support this diagnosis. The persistence of symptoms despite the appropriate treatment for the asthma symptoms was the main reason for the reconsideration of FBA. All of the children underwent bronchoscopy, with the successful removal of small organic food items from the main bronchi. The main cause of FBA was the parents' lack of awareness concerning the risk of FBA when small organic food items are provided to preschool children. These cases demonstrate that some parents are unfamiliar with the risk of FBA when small organic food items are provided to their children. It is crucial to make it understood that consideration of FBA should be maintained throughout the clinical course of patients with an acute onset of respiratory symptoms, despite the initial lack of evidence to support this diagnosis, and that parental education about the causes and hazards of this condition may be the best preventive measure to decrease its incidence.

A comparison between transcutaneous and total serum bilirubin in healthy-term greek neonates with clinical jaundice.

The accuracy of transcutaneous bilirubin meters has been assessed in newborns from various ethnic backgrounds. However, there are limited data on Greek newborns. Our study examined the accuracy of transcutaneous bilirubin measurements in clinically jaundiced healthy-term Greek newborns, using total serum bilirubin as the reference standard, in order to re-evaluate our local guidelines about neonatal jaundice. Clinically jaundiced newborns requiring total serum bilirubin level estimation were recruited prospectively. 368 pairs of total serum bilirubin/transcutaneous bilirubin measurements were taken in 222 newborns, using a direct spectrophotometric device and the BiliCheck device, respectively. The level of agreement between the obtained transcutaneous bilirubin and total serum bilirubin values was assessed. Our data were analysed using the Stata/SE 12.0 (StataCorp LP, USA) statistical programme. The mean (± SD) TSB was 225.4 ± 25.4 µmol/l and the mean (± SD) TcB was 237.9 ± 21.0 µmol/l. The correlation between the values was poor (Pearson's correlation coefficient 0.439; Lin's concordance coefficient 0.377 [95% CI 0.301 to 0.453]; P<0.001). The Bland-Altman analysis demonstrated that transcutaneous bilirubin measurements tended to overestimate the total serum bilirubin value (mean difference 12.5 ± 24.9 µmol/l), with wide 95% limits of agreement (-36.2 µmol/l to 61.3 µmol/l). Transcutaneous bilirubin values did not correlate well with total serum bilirubin values, being often imprecise in predicting the actual total serum bilirubin levels. This permits us to continue estimating total serum bilirubin in clinically jaundiced newborns according to our local guidelines, in order to safely decide the appropriate care plan.

Use of Deferasirox Film-Coated Tablets in Pediatric Patients with Transfusion Dependent Thalassemia: A Single Center Experience.

Thalassemic syndromes are characterized by clinical heterogenicity. For severe disease forms, lifelong blood transfusions remain the mainstay of therapy, while iron overload monitoring and adequate chelation treatment are required in order to ensure effective disease management. Compared to previous chelators, the new deferasirox film-coated tablet (DFX FCT) is considered to offer a more convenient and well-tolerated treatment scheme, aiming at better treatment-related and patient-related outcomes. The present study's objective was to prospectively evaluate the safety and efficacy of DFX FCT in children and adolescents with transfusion-dependent thalassemia. Data collected included patient demographics, hematology and biochemistry laboratory work up, magnetic resonance imaging of heart and liver for iron load, as well as ophthalmological and audiological examination prior to and a year following drug administration. Study results confirmed DFX FCT safety in older children in a manner similar to adults, but demonstrated increased frequency of adverse events in younger patients, mainly, involving liver function. With regards to efficacy, study results confirmed the preventive role of DFX FCT in iron loading of liver and heart, however, higher doses than generally recommended were required in order to ensure adequate chelation.

Tissue necrosis following extravasation of acyclovir in an adolescent: A case report.

OBJECTIVE: Extravasation of intravenously infused vesicant solutions is a common problem in medical practice, which can lead to severe and progressive tissue dysfunction, ranging from persistent tissue oedema and fibrosis to delayed tissue necrosis. Acyclovir is a known vesicant medication administrated in paediatric patients, which appears to irritate venous and soft tissue if extravasated. CASE REPORT: We present the first case involving the extravasation of intravenously infused acyclovir in a female adolescent patient, which caused tissue necrosis and left behind a residual scar lesion. Nursing and medical staff should be aware of the potential dermatological side effects of intravenously infused acyclovir and other medications, even a long time after infusion, and the possible lack of initial local symptoms and signs. CONCLUSION: Early recognition of extravasation and prompt management are critical in preventing further morbidity, and optimizing outcomes.

Guillain-Barré Syndrome presenting as unilateral hip pain in a child.

OBJECTIVE: The aim of this report is to highlight the importance of close observation and follow-up in children who present with an acutely irritable hip. This is because hip pain is a symptom of not only benign but also severe conditions. Thus, at the time of the initial presentation, hip pain can be misdiagnosed. This report serves as an example for a wide range of doctors such as orthopaedic surgeons, paediatricians, emergency room physicians or primary care physicians, because these are the first-line doctors who treat patients with a painful hip. CASE REPORT: We herein present a three-year-old child who was admitted to our hospital with pain in the right leg and initially diagnosed with transient synovitis of the hip. An additional examination two days later, after severe deterioration of the clinical picture, revealed that our patient was actually suffering from Guillain-Barré syndrome. Failure to diagnose Guillain-Barré syndrome and initiating prompt treatment is potentially life-threatening. CONCLUSION: Clinicians should be aware that hip pain could be the presenting complaint of Guillain-Barré syndrome, a syndrome that has many clinical features. Even when all the clinical and laboratory findings indicate a benign condition, Guillain- Barré syndrome should still be considered. Therefore, close observation and follow-up in children who present with an acutely irritable hip is highly recommended. In this way, the potentially catastrophic consequences of more severe conditions can be avoided.