Background and objectives. The aim of this observational retrospective cohort study was to demonstrate
indications and response rates of the patients with pediatric rheumatic diseases that used canakinumab.
Method. The files of the patients that used canakinumab between December 2012 and July 2017 were reviewed.
Canakinumab was used in 29 patients. Diagnosis of the patients were; colchicine resistant familial Mediterranean
fever (crFMF) (19 cases), hyperimmunoglobulin D syndrome-mevalonate kinase deficiency (HIDS-MKD) (3
cases), cryopyrin-associated periodic syndrome (3 cases), systemic juvenile idiopathic arthritis (sJIA) (2 cases),
idiopathic recurrent pericarditis (1 case) and pyoderma gangrenosum (1 case).
Results. Canakinumab was used for 21.8 ± 15.8 months (6-54 months). crFMF patients had a female predominance;
16 girls and 3 boys. Mean age at the first symptoms of FMF was 2.8 ± 2.2 years. Mean number of attacks per year
before colchicine was 18.7 ± 6.9 (10-36), after colchicine was 8.2 ± 2.7 (6-12) and after biologic agent the number
dropped to 0.1 ± 0.3 (0-1). Canakinumab led to resolution of attacks in 3 HIDS-MKD cases. Two familial cold
autoinflammatory syndrome patients were using canakinumab for 13 months with total remission. Chronic
infantile neurological cutaneous articular syndrome patient did not show dramatic response to standard doses
of IL-1 blockers and remission was achieved with high doses of canakinumab. Canakinumab led to the resolution
of all systemic and articular manifestations in one sJIA case but the other sJIA case developed polyarticular joint
involvement under canakinumab treatment. A severe pyoderma gangrenosum patient that failed dapson and
anakinra, also failed canakinumab treatment that was used for 9 months. We have successfully treated a case
of idiopathic recurrent pericarditis with canakinumab. Canakinumab was discontinued due to inefficacy only
in two cases.
Conclusion. Overall efficacy of canakinumab was 93.1% in this study. No major adverse event was observed
under canakinumab treatment. Canakinumab seems to be effective and safe in children with rheumatic diseases.
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Background and objectives. The aim of this study was to compare serial scores of amplitude-integrated electroencephalography (aEEG) in preterm infants with favorable neurologic outcome compared with those with unfavorable neurologic outcome and to evaluate whether aEEG in the early days of life has predictive value for short-term neurologic outcome in preterm infants. Methods. This prospective observational study included infants born at ≤32 weeks of gestational age and ≤1,500 g of birth weight. On the basis of brain ultrasonography findings, the infants were divided into two groups (favorable and unfavorable outcome group) at 36 weeks of corrected age or at discharge. aEEG was performed at 12-14 h (day-1), 46-48 h (day-2), 70-72 h (day-3), and 1 week (day-7) of life. The aEEG recordings were analyzed using the criteria described by Burdjalov et al.23 and the serial scores of aEEG were compared between the two groups. Results. Thirty five infants were enrolled and 18 infants and 17 infants were identified into both groups, respectively. Infants in the favorable outcome group showed high scores in almost all parameters and the score of all parameters increased over time. However, the scores of all components decreased in day-2 compared with those of day 1 in the unfavorable outcome group. The total score less than 3 of day-2 has predictive value of 70.6% of sensitivity and 72.2% of specificity for unfavorable outcome. Conclusion. We found that aEEG is a useful predictor for short-term neurologic outcome in preterm infants.
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Background and objective. Adverse reactions related to intravenous immunoglobulin (IVIG) infusions vary from 1 to 81%, with an average of 20%. They may be classified as immediate; occurring during the infusion itself or delayed; occurring after the infusion has been ceased. In the present study, we aimed to evaluate the frequency of immediate adverse reactions due to IVIG infusions in primary immune deficiency (PID) patients. Methods. The study population was composed of 109 patients. A total of 763 infusions were recorded for demographic data and adverse reactions. Results. The participants included 32 girls (29%) and 77 boys (71%). The mean age was 11.8 ± 5.7 years (0.6-33.5 years). Early adverse events (AE) were recorded in 34 (4.5%) among 763 IVIG infusions including 30 mild (88.2%), 3 moderate (8.8%) and 1 severe (2.9%). The most common immediate adverse reactions were fever (29.4%) and headache (29.4%). The risk of AE was higher among primary antibody deficiency (PAD), compared to combined immunodeficiency (OR 2.61, 95%CI 1.061-6.475; p = 0.037). Conclusions. Use of various intravenous immunoglobulin treatments should be considered with regard to side effect profiles observed. In our cohort, PID patient experienced mostly mild AE; PAD was associated with an increased risk of AE.
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Background and objectives. Most cases of severe scorpion envenomation occur in children and are associated with significant morbidity and mortality. Excessive systemic inflammatory response, which leads to multiple organ involvement, is an emerging challenge during severe envenomation. The aim of this study was to investigate if there was any relationship between initial hematological parameters and severe envenomation in pediatric patients presenting with scorpion envenomation. Method. This study was performed retrospectively, at the pediatric emergency unit and pediatric intensive care unit of the Çukurova University Medical School in Turkey. Two hundred and fifty-seven cases with scorpion envenomation, and a control group consisting of one hundred and fifteen healthy children were included in the study. Results. White blood cell, neutrophil, lymphocyte, platelet, neutrophil/lymphocyte ratio (NLR), platelet/lymphocyte ratio (PLR) and PDW values of patients were higher than the controls (p<0.05). Mean NLR was 3.8 ± 4.7 in patients. Patients were analyzed with the help of the decision tree model, and it was seen that in patients who had applied to hospital in less than an hour after the scorpion sting, 87.5% of the patients whose NLR value was between the 0.519-1.969 interval (below 2.1 which we found as the cut-off value) did not need to be hospitalized in the intensive care unit, 54.1% of the patients whose NLR value was higher than 1.969 needed to be hospitalized at the intensive care unit. Conclusions. Severe envenomation is associated with mortality and morbidity in children. Our findings showed that NLR seems to be a useful tool in predicting severe envenomation.
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Background and objectives. Kallistatin, a serine proteinase inhibitor, exerts its effect by vascular repair, angiogenesis inhibition, strong vasodilation, inhibition of vascular endothelial growth factor (VEGF), anti-inflammation, and anti-apoptosis. We hypothesized as to whether it has a protective role in pulmonary arterial hypertension (PAH). Methods. The study included 5 subgroups (78 patients; 44 male): Eisenmenger syndrome (n=16), PAH with left to right shunt (n=20), idiopathic PAH (n=7), patients with left to right shunt without PAH (n=19), and patients with innocent heart murmur (n=16). Physical examination, chest radiography, electrocardiography, and transthoracic echocardiography (TTE) were performed for each patient. PAH diagnosis was confirmed by catheterization. Serum kallistatin, tumor necrosis factor alpha (TNF-α), Interleukin-10 (IL-10) and N-terminal pro b-type natriuretic peptide (NT-proBNP) levels were studied for each patient.Results. The lowest median kallistatin value was found in Eisenmenger syndrome: 1.19 (0.87-3.30) μg/ml. The highest value belonged to control group with innocent murmur: 2.89 (1.19-5.66) μg/ml. Serum levels of kallistatin were significantly lower in patients with PAH (p<0.05). TNF-α values were increased and IL-10 values were decreased in pulmonary hypertension. However; no correlation was found between kallistatin levels and cytokines.Conclusions. Kallistatin may have a protective effect in pulmonary arterial hypertension by repairing vascular damage, inhibition of angiogenesis, strong vasodilator effect, inhibiting VEGF, and anti-inflammatory mechanism of action. To our knowledge, our study is the first one that shows the role of kallistatin in pulmonary hypertension. Kallistatin may represent a promising novel therapeutic approach for pulmonary hypertension in the near future.
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Background. Icterus tends to be one of the most prevalent causes of neonatal hospitalization. The present study aimed to evaluate the effects of the different doses of ursodeoxycholic acid (UDCA) on neonatal jaundice. Method. In this study, 120 newborns who were hospitalized for phototherapy were randomly assigned. Group A received phototherapy and UDCA 5 mg/kg/dose every 12 hours orally, group B patients were treated with phototherapy and UDCA 7.5 mg/kg/dose every 12 hours orally. Group C received phototherapy with a placebo. All patients were evaluated for bilirubin levels, the duration of phototherapy, and the length of hospital stay.Results. The mean bilirubin level at hospital admission was 19.88 ± 2.33 mg/dl in group A, 19.33 ± 2.51 mg/dl in group B, and 19.76 ± 2.64 mg/dl in group C (p= 0.58). The groups receiving phototherapy with UDCA showed a significant decrease in the bilirubin level. Bilirubin level decreased to 10.04 ± 1.11mg/dl in group A, 8.82 ± 1.11 mg/dl in group B, and 12.04 ± 2.05 mg/dl in group C (p= 0.000). Furthermore, the mean duration of phototherapy, as well as the average length of hospital stay, were significantly lower in group B as compared to the other groups (p= 0.000).Conclusion. The findings of this study indicated that the administration of UDCA in addition to phototherapy could effectively decrease the length of hospital stay and bilirubin levels in neonatal hyperbilirubinemia. However, further studies with a larger sample size are required before one can recommend the routine use of UDCA for the treatment of neonatal jaundice.
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Background and objectives. Parents who have a premature child in neonatal intensive care units (NICUs) are in a stressful situation. The aim of this paper is to analyze the emotional support received by parents with premature children admitted to NICUs. Methods. A phenomenological qualitative study with an explanatory and interpretative approach was employed. Results. The findings are: 1) The experience and emotions of a premature delivery; showing sadness, guilt and despair, stress, anxiety, and uncertainty over the future of their child. 2) The emotional support received by the father/mother of the partner; discussion of how their partner is cared for, as well as the care given to the premature child and other children in the family; the stress that this causes them on not being able visit all at once. 3) The emotional support offered by the health professionals (doctors, nurses, etc.); parents indicate that they have received very strong support from the nurses, but also that they were not always asked about their feelings when in the NICU. 4) The informal emotional support of relatives and parents in the NICU. After talking with other support mothers, the mothers then felt less guilty. Conclusion. As regards premature birth, the mothers showed feelings of sadness and guilt, asked themselves where they had failed and what they had done wrong.
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Background and objectives. Right ventricular failure is an important cause of mortality and morbidity after orthotopic heart transplantation (OHT). The right ventricle of the donor may fail to accommodate to the high pulmonary vascular resistance (PVR) of the recipient. Pulmonary hypertension (PH) due to chronic heart failure with PVRi > 4 Wood units.m2, transpulmonary gradient > 15 mmHg adversely affect the outcome of OHT. In this study we aimed to evaluate management strategies in our pediatric cardiac transplantation candidates with PH and high PVR prior to OHT. Method. Twenty-six cardiac transplantation candidates (age: 10.2 ± 4.6, 1-17 years) underwent cardiac catheterization for the determination of PVR and pulmonary arterial pressure. They were admitted to the hospital and received 1-3 days of intravenous (IV) vasodilator therapy; 0.5-3 μg/kg/min nitroglyserin and/or 0.5-3 μg/kg/min nitroprusside, 5-15 μg/kg/min dobutamin and/or dopamin to keep systolic blood pressure above 80 mmHg. Results. Thirteen patients had dilated cardiomyopathy (CMP), 11 had restrictive CMP, one had hypertrophic CMP and one had congenital heart disease (CHD). Nineteen of the 26 patients underwent OHT. Mean pulmonary arterial pressure of the patients ranged between 11 and 82 mmHg (30.4 ± 16 mmHg) and PVRi between 0.41-21.4 Wood units.m2 (5.3 ± 5.7). Nine patients had PVRi above 4 Wood units.m2. Six of these patients had IV treatment for longer than three days and some received specific anti-PH treatment. Eventually they underwent a pulmonary vasoreactivity test with IV iloprost and six had PVRi <4 Wood units.m2. Five of them underwent OHT. Conclusion. Cardiac transplantation candidates with PH and high PVR should be evaluated after conditioning with vasodilator and inotropic treatment. Specific treatment for PH and vasoreactivity testing may help selected patients reenter the transplantation list.
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Background and objectives. To compare the corneal endothelial morphology and anterior segment parameters in type 1 diabetes mellitus children (T1DM) and healthy controls.Methods. This cross-sectional prospective study included 56 patients with T1DM and 50 eyes of 50 age-matched controls. Endothelial morphology was analyzed with EM-3000 specular microscopy, and anterior parameters were analyzed with Sirius Scheimpflug topography. Endothelial cell density (ECD), coefficient of variation (CV) of cell area, central corneal thickness (CCT), anterior chamber depth (ACD), iridocorneal angle (ICA), K1 and K2, pupillary diameter (PD), horizontal visible iris diameter (HVID), duration of T1DM, and HbA1c levels were noted. Results. The mean age of the T1DM group was 14.3 ± 3.2 years, compared to 13.2 ± 3.7 years in the healthy group (p = 0.140). The mean duration of diabetes mellitus was 4.5 ± 3.5 years. The mean HbA1c was 9.5 ± 1.9% (minimum 6%, maximum 14%). The mean values of CCT were 556 ± 30 μm and 536 ± 36 μm in T1DM and healthy groups, respectively (p = 0.003). The mean values of ACD were 3.69 ± 0.31 mm and 3.83 ± 0.27 mm in T1DM and healthy groups, respectively (p = 0.02). The mean values of PD were 4.29 ± 1.2 mm and 5.17 ± 1.36 mm in T1DM and healthy groups, respectively (p = 0.001). There was no statistically significant difference between groups in terms of ECD, CV, ICA, K1, K2, and HVID (p > 0.05).Conclusion. Type 1 diabetes mellitus children have thicker corneas, shallower anterior chamber depth, and smaller pupillary diameter than healthy subjects.
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Background. Vici syndrome is a rare autosomal recessive disease with phenotypically heterogeneous presentation. Characteristic features of the disease are oculocutaneous albinism, corpus callosum agenesis, cataract, cardiomyopathy, and immunodeficiency.Case. Here we report two Turkish patients with Vici syndrome. One of these patients had a novel mutation in EPG5 and presented with idiopathic thrombocytopenic purpura (ITP) and maculopapular rashes similar to Stevens–Johnson syndrome, which has been previously reported in only a few cases in the literature. Conclusion. Vici syndrome presents with a typical phenotype which may facilitate diagnosis for infants with multisystemic disorders. ITP and maculopapular rashes might be added to the spectrum of findings of patients with Vici syndrome.
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