Pulmonary artery size as an indication for thoracoscopic repair of congenital diaphragmatic hernia in neonates

Purpose

We reviewed 24 consecutive cases of prenatally or immediately postnatally diagnosed left-sided congenital diaphragmatic hernia (CDH) to evaluate pulmonary artery (PA) size as an indication for thoracoscopic repair (TR).

Methods

CDH repair is planned once echocardiography confirms improvement in pulmonary hypertension. TR is chosen if cardiopulmonary status is stable more than 10?min in the decubitus position in the neonatal intensive care unit (NICU) under conventional mechanical or high frequency oscillatory ventilation (HFOV) with/without nitric oxide (NO) and the patient appears likely to tolerate manual ventilation during transfer to the operating room. Otherwise open repair (OR) is performed in NICU. Proximal right PA (RPA) and left PA (LPA) diameters measured at birth were assessed with respect to the type of repair.

Results

10/24 had TR and 14/24 had OR. TR cases had significantly larger RPA/LPA diameters (3.52?±?0.23 vs. 3.10?±?0.56?mm, p?p?p?p?n?=?3) and cardiopulmonary instability (n?=?1).

Conclusions

TR can be considered when RPA/LPA diameters are larger than 3.0/2.5?mm, respectively, and cardiopulmonary status is stable without NO.     

Differentiating Kawasaki disease from urinary tract infection in febrile children with pyuria and C-reactive protein elevation

Background

Kawasaki disease (KD) is sometimes confused with urinary tract infection (UTI) because both can present with pyuria and C-reactive protein (CRP) elevation. The present study investigated the clinical and laboratory findings that can differentiate KD from UTI in febrile children with pyuria and CRP elevation.

Methods

Medical records were retrospectively reviewed for children with KD and those with UTI. The clinical and laboratory findings between the KD with pyuria group (n?=?48) and the UTI group (n?=?118) were compared.

Results

The KD with pyuria group had older age (P?<?0.001) and longer duration of fever (P?<?0.001) than the UTI group. In blood tests, both groups showed increased CRP level, but the value of CRP was higher in the KD with pyuria group than in the UTI group (P?<?0.001). The KD with pyuria group also showed higher values for liver enzymes than the UTI group (P?<?0.001); >?70.0% of children in the KD with pyuria group, but <?20.0% of children in the UTI group possessed elevated liver enzymes (P?<?0.001). On urinalysis, 40.7% of the UTI group had a positive nitrite test, but 0.0% of the KD with pyuria group had a positive nitrite test (P?<?0.001).

Conclusions

Elevated liver enzymes are more specific to KD than to UTI, whereas a positive nitrite test is more specific to UTI than to KD. Our findings can be used as diagnostic clues to differentiate KD from UTI in febrile children with pyuria and CRP elevation.

     

A prospective randomly controlled clinical trial on azithromycin therapy for induction treatment of children with nephrotic syndrome

A prospective study was designed to evaluate the efficacy of azithromycin (AZM) when combined with prednisone therapy compared with prednisone therapy alone in children with primary nephrotic syndrome (PNS) undergoing induction treatment. A prospective randomly controlled clinical trial was conducted. Randomization was performed to select the research subjects who were composed of children with PNS and treated with AZM combined with prednisone (the intervention group) and with prednisone alone (the control group). A total of 211 randomly selected patients with PNS received either AZM combined with prednisone (n?=?106) or prednisone alone (n?=?105) for 6 months. At three months in the follow‐up period, 12 patients were lost to follow up (intervention group, 7; control group, 5), and 6 patients had a transient hypocomplementemia (intervention group, 4 ; control group, 2). AZM was administered at a dose of 10 mg/kg q.d (1 dose per day) for three consecutive days. The median duration before remission was 6 days in the intervention group and 9 days in the control group (p?<?0.0001). Relapse rate differed among the groups at 3 months (11.6 vs. 21.4 %, p?=?0.049). No difference in relapse rate was observed between the two groups within 4 to 6 months and at 6 months (p?=?0.168, 0.052). After 4 weeks of treatment, steroid resistance occurred in 1 out of 95 (1.05 %) patients in the intervention group and in 10 out of 98 (10.2 %) patients in the control group (p?=?0.006). After 8 weeks of treatment, no difference was found in steroid resistance between two groups (1/95 vs. 3/98, p?=?0.327). During follow-up at 6 months, no difference was exhibited by the two groups on frequent relapse rates (p?=?0.134). Conclusion: If a course of AZM is added to the glucocorticoid-induced treatment among children with PNS, then the sensitivity of prednisone increases. This increase consequently reduces duration to remission and decreases relapse. However, further studies are necessary to confirm these results.     

The effect of the broad-spectrum antibiotics for prevention of postoperative intra-abdominal abscess in pediatric acute appendicitis

Background

We investigated the efficacy of broad-spectrum antibiotics for prevention of postoperative intra-abdominal abscess in pediatric acute appendicitis with our 3 risk factors:—WBC?>?16.5 (×?10³/µl), CRP?>?3.1 (mg/dl) and appendix maximum short diameter on diagnostic imaging?>?11.4 mm.

Methods

Four hundred twenty-two patients were reviewed. Patients with 0–1 risk factors were assessed as low-risk and those with 2–3 were high-risk. In the low-risk group, Group A (n?=?66) patients received broad-spectrum antibiotics and Group B patients (n?=?265) received narrow-spectrum monotherapy. In the high-risk group, Group C patients (n?=?63) received broad-spectrum antibiotics and Group D patients (n?=?28), narrow-spectrum antibiotics. The outcomes were the incidence of postoperative abscess and the total duration of intravenous (IV) antibiotics.

Results

The incidence of intra-abdominal abscess was 6.06% in Group A versus 1.89% in Group B (p?=?0.08), and 19.05% in Group C versus 3.57% in Group D (p?=?0.06). Total IV antibiotic duration (days) were 6.12?±?2.87 in Group A versus 3.83?±?0.69 in Group B (p?<?0.01), and 7.84?±?4.57 in Group C versus 4.00?±?0.82 in Group D (p?<?0.01).

Conclusion

Broad-spectrum antibiotics did not prevent postoperative intra-abdominal abscess in either low or high-risk groups.

     

Adolescent Kawasaki disease: usefulness of 64-slice CT coronary angiography for follow-up investigation

Background

Kawasaki disease (KD) is a systemic vasculitis that mainly affects coronary arteries in children, and requires regular follow-up from the time of diagnosis.

Objective

To evaluate the feasibility of 64-slice CT angiography (CTA) for follow-up of patients with KD using previously performed invasive catheter coronary angiography (CCA) as reference standard.

Materials and methods

The study group comprised 12 patients (age 17.6?±?2.9?years, mean±SD) with a diagnosis of KD and a previously performed CCA (interval, 32.6?±?13.5?months) who underwent 64-slice cardiac CTA. The quality of the images for establishing the presence of coronary abnormalities was determined by two observers. The CTA findings were compared with those from the prior CCA.

Results

Adequate image quality was obtained in all patients. Mean effective dose for CTA was 6.56?±?0.95?mSv. CTA allowed accurate identification, characterization and measurement of all coronary aneurysms (n?=?32), stenoses (n?=?3) and occlusions (n?=?9) previously demonstrated by CCA. One patient with disease progression went on to have percutaneous coronary intervention.

Conclusion

Coronary lesions were reliably evaluated by 64-slice CTA in the follow-up of compliant patients with KD, reducing the need for repeated diagnostic invasive CCA. Hence, in an adequately selected patient population, the role of CCA could be limited almost only to therapeutic procedures.     

Evaluation of echogenicity of the heart in Kawasaki disease

Pathologic studies of the heart in patients with Kawasaki disease (KD) revealed vasculitis, valvulitis, myocarditis, and pericarditis. However, there have been no studies on the quantitative determination of multi-site echogenicity of the heart in KD patients. It is also undetermined whether the degree of echogenicity of each site of the heart in patients with KD might be related to the response to intravenous immunoglobulin (IVIG) treatment. In 81 KD patients and 30 control subjects, we prospectively analyzed echogenicity of the heart. Echogenicity was measured in four sites: coronary artery wall (CAW), mitral valve (MV), papillary muscle (PM), and ascending aortic wall (AAo wall) by the calibrated integrated backscatters (cIBs). The cIB values of all measurement sites at acute phase in KD patients were significantly higher than those in control subjects (KD patients vs control subjects; CAW, 19.8?±?6.2 dB vs 14.5?±?2.0 dB, p?<?0.05; MV, 23.3?±?5.3 dB vs 16.0?±?3.3 dB, p?<?0.05; PM, 22.4?±?5.1 dB vs 12.7?±?1.9 dB, p?<?0.05; AAo wall, 25.3?±?5.6 dB vs 18.3?±?3.4 dB, p?<?0.05). The cIB values of CAW at the acute phase in IVIG nonresponders were significantly higher than those in responders. Conclusion: Echogenicity of the heart in KD patients at the acute phase increased not only in the coronary artery wall but also in other parts of the heart. Echogenicity of CAW might be helpful in determining the unresponsiveness of IVIG treatment.     

Association of high ventilator pressures with the development of chronic pulmonary hypertension in congenital diaphragmatic hernia patients requiring ECMO

Purpose

Congenital diaphragmatic hernia (CDH) patients requiring extracorporeal membrane oxygenation (ECMO) were examined to determine, if aspects of their complex ventilatory management were associated with the development of chronic pulmonary hypertension (cPH).

Methods

CDH patients requiring ECMO from 1992 to 2007 were retrospectively reviewed. cPH was defined as pulmonary hypertension at 3?months of age. Demographic and clinical variables including peak ventilatory pressures (PVP) and mean airway pressures (MAP) were tabulated.

Results

10/31 (32?%) patients developed cPH. Gestational age, birth weight, inborn status, CDH side and liver position were not different between cPH and non-cPH patients. Pre-ECMO, both groups required statistically similar ventilatory support, though there was a trend toward higher oxygenation index and higher PVP for cPH patients. While ECMO duration was similar between groups, cPH patients required significantly higher PVP (30.0 vs. 25.0?cmH₂O, p?=?0.01) and MAP (11.5 vs. 9.0?cmH₂O, p?=?0.02) for ECMO decannulation. Post-ECMO, maximum PVP (50.0 vs. 26.0?cmH₂O, p?p?=?0.001), HFV requirement (90 vs. 10?%, p?p?Conclusion Not until after ECMO decannulation do we see clinical differences separating patients who ultimately develop cPH. Although the degree of pulmonary hypoplasia may ultimately dictate ECMO decannulation criteria, perhaps greater physiologic optimization before decannulation could decrease the incidence of cPH.     

Incomplete clinical manifestation as a risk factor for coronary artery abnormalities in Kawasaki disease: a meta-analysis

Incomplete Kawasaki disease (KD) comprises a large proportion of the total number of cases. Although it has the potential of delaying diagnosis, it is not conclusive whether an incomplete presentation is a risk factor for coronary artery abnormalities (CAAs). We performed a meta-analysis to establish the risk of CAA in 20 studies including 4,504 cases and 32,519 controls, and the risk of giant aneurysm in two studies including 5,390 cases and 37,648 controls. The pooled results indicated that incomplete KD was associated with an increased risk of CAA [odds ratio (OR)?=?1.447, 95 % confidence interval (CI)?=?1.158–1.808, p?=?0.001]. Subgroup analyses demonstrated higher associations in patients younger than 12 months (OR?=?2.023, 95 % CI?=?1.252–3.271, p?=?0.004), Asians and Indians (OR?=?1.57, 95 % CI?=?1.234–1.999, p?<?0.001 and OR?=?7.088, 95 % CI?=?1.640–30.631, p?=?0.009, respectively). Subgroup analysis according to the period of patient enrollment before and after 2004 showed increased association of incomplete KD with CAA only among studies with patients enrolled after 2004 (OR?=?1.969, 95 % CI?=?1.240–3.127, p?=?0.004). In conclusion, incomplete KD seems to be associated with an increased risk of CAA, and this is more prominent in patients younger than 12 months, Asians and Indians.     

Feeding intolerance in preterm infants fed with powdered or liquid formula: a randomized controlled, double-blind, pilot study

Feeding intolerance (FI) is usually defined as “gastric residual volume of more than 50 % of the previous feeding volume, emesis, abdominal distension or both of these symptoms and a decrease, delay or discontinuation of enteral feedings.” We aimed to compare the incidence of FI in preterm infants fed with powdered or liquid infant formula, and in a prospective, double-blind, pilot study, 78 preterm infants were randomized to receive powdered or liquid form of the same preterm infant formula. The primary outcomes were the incidence of FI in both groups. The pH of gastric fluids was measured in the fasting and postprandial periods on the seventh day of life, and gastrointestinal complications were recorded during the hospitalization period. The incidence of FI was significantly higher in infants fed with liquid formula (n?=?34) when compared with infants fed with powdered formula (n?=?44) [9 (26.5 %) vs 2 (4.5 %), p?<?0.01, respectively]. The median fasting gastric fluid pH was significantly lower and postprandial gastric fluid pH was significantly higher than in infants fed with powdered formula (2.9 vs 3.4, p?<?0.01 and 6.0 vs 5.9, p?<?0.05 respectively). Infants fed with liquid formula regained birth weight significantly later than infants fed with powdered formula (9.5 vs 8.0 days, p?<?0.01). Conclusion: Although the exact mechanisms are not clear, increased incidence of FI and delayed growth in the first weeks of life in preterm infants fed with liquid formula might be caused by altered gastric acidity or possible disrupted protein bioavailability due to different production and sterilization processes.     

Relation between biomarkers and clinical severity in patients with Smith–Lemli–Opitz syndrome

Smith–Lemli–Opitz syndrome (SLOS), a multiple congenital anomaly with severe mental retardation, is caused by decreased activity of 7-dehydrocholesterol reductase. Fifteen Hungarian patients were diagnosed with SLOS on the basis of clinical symptoms, serum cholesterol, 7-dehydrocholesterol, and molecular genetic testing. Their age at the time of diagnosis in mild SLOS (n?=?4, clinical score <20) was 0.5–18 years, cholesterol was 2.37?±?0.8 mmol/L, and 7DHC was 0.38?±?0.14 mmol/L. In the group of typical SLOS (n?=?7, score 20–50), the diagnosis was set up earlier (age of 0.1–7 years); t-cholesterol was 1.47?±?0.7 mmol/L, and 7DHC was 0.53?±?0.20 mmol/L. Patients with severe SLOS (n?=?4, clinical score?>?50) died as newborns and had the lowest t-cholesterol (0.66?±?0.27 mmol/L), and 7DHC was 0.47?±?0.14 mmol/L. Correlation coefficient with clinical severity was 0.74 for initial t-cholesterol and 0.669 for Cho/7DHC. Statistically significant difference was between the initial t-cholesterol of mild and severe SLOS (p?=?0.01), and between the Cho/7DHC ratios of groups (p?=?0.004). In severe SLOS, the percentage of α-lipoprotein was significantly lower than in typical (p?=?0.003) and mild SLOS (p?=?0.004). Although serum albumin, total bilirubin, and hemostasis parameters remained in the reference range during cholesterol supplementation (n?=?10) combined with statin therapy (n?=?9), increase of aspartate aminotransferase and alanine aminotransferase in 50 % of the patients probably refers to a reversible alteration of liver function; therefore, statin therapy was suspended. Conclusion: life expectancy is fundamentally determined by the initial t-cholesterol, but dehydrocholesterol and α-lipoprotein have prognostic value. Accumulation of hepatotoxic DHC may inhibit the synthesis of α-lipoproteins, decreasing the reverse cholesterol transport. During statin therapy, we suggest monitoring of lipid parameters and liver function.     

Clinical manifestations associated with Kawasaki disease shock syndrome in Mexican children

Recently, there have been increasing reports of severe forms of Kawasaki disease (KD) associated with shock that have been managed in pediatric intensive care units. It has been suggested that KD is more severe in the Hispanic population. We conducted a study to determine the frequency of Kawasaki disease shock syndrome (KDSS) in our population and compared characteristics between patients with KD without shock and patients with KDSS. Data from 214 patients with KD treated in a tertiary pediatric hospital were collected during a 12-year period. We compared clinical and laboratory features of KD patients without shock and KDSS patients. Of 214 consecutive patients with KD, 11 (5 %) met the definition for KDSS. All of these patients received fluid resuscitation, seven (64 %) required inotropic treatment, and six (54 %), ventilatory support. On admission, seven of these patients (64 %) had an incomplete presentation of the disease, whereas in the group of patients without shock, the relative frequency of an incomplete presentation was 29 %. Twenty percent (3/11) of patients with KDSS presented giant coronary aneurysms versus none of 203 KD patients without shock (p?=?0.001); myocardial infarction, 27 % (3/11), versus 1 % (2/203) (p?=?0.001); and intravenous immunoglobulin (IVIG) resistance, 60 % (6/11), versus 12 % (24/203). Gastrointestinal manifestations in the acute phase occurred in 91 % of KDSS patients versus 30 % patients without shock (p?=?0.001). Conclusion. Patients with KD presenting in shock seem to have an increase in gastrointestinal manifestations, incomplete presentation, IVIG resistance, and worse cardiac outcomes. Larger, prospective, multicentre studies should be carried out to corroborate these findings.     

The influence of gestational age,mode of delivery and abdominal wall closure method on the surgical outcome of neonates with uncomplicated gastroschisis

Aim of the study

To evaluate if gestational age (GA), mode of delivery and abdominal wall closure method influence outcomes in uncomplicated gastroschisis (GTC).

Methods

Retrospective review of NICU admissions for gastroschisis, August 2008–July 2016. Primary outcomes were: time to start enteral feeds (on-EF), time to discontinue parenteral nutrition (off-PN), and length of stay (LOS).

Main results

A total of 200 patients with GTC were admitted to our NICU. Patients initially operated elsewhere (n?=?13) were excluded. Patients with medical/surgical complications (n?=?62) were analyzed separately. The study included 125 cases of uncomplicated GTC. There were no statistically significant differences in the outcomes of patients born late preterm (34 0/7–36 6/7; n?=?70) and term (n?=?40): on-EF 19 (5–54) versus 17 (7–34) days (p?=?0.29), off-PN 32 (12–101) versus 30 (16–52) days (p?=?0.46) and LOS 40 (18–137) versus 37 (21–67) days (p?=?0.29), respectively. Patients born before 34 weeks GA (n?=?15) had significantly longer on-EF, off-PN and LOS times compared to late preterm patients: 26 (12–50) days (p?=?0.01), 41 (20–105) days (p?=?0.04) and 62 (34–150) days (p?<?0.01), respectively. There were no significant differences in outcomes between patients delivered by C-section (n?=?62) and patients delivered vaginally (n?=?63): on-EF 20 (5–50) versus 19 (7–54) days (p?=?0.72), off-PN 32 (12–78) versus 33 (15–105) days (p?=?0.83), LOS 42 (18–150) versus 41 (18–139) days (p?=?0.68), respectively. There were significant differences in outcomes between patients who underwent primary reduction (n?=?37) and patients who had a silo (88): on-EF 15 (5–37) versus 22 (6–54) days (p?<?0.01), off-PN 28 (12–52) versus 34 (15–105) days (p?=?0.04), LOS 36 (18–72) versus 44 (21–150) days (p?=?0.04), respectively.

Conclusion

In our experience, late preterm delivery did not affect outcomes compared to term delivery in uncomplicated GTC. Outcomes were also not influenced by the mode of delivery. Patients who underwent primary reduction had better outcomes than patients who underwent silo placement.

     

Fn14 hepatic progenitor cells are associated with liver fibrosis in biliary atresia

Purpose

The liver in biliary atresia (BA) is characterized by progressing fibrosis which is promoted by unclear reasons. We aimed to understand the factors influencing liver fibrosis. This study hypothesized that HPCs (hepatic progenitor cells) are activated and associated with liver fibrosis in biliary atresia.

Methods

Liver samples from biliary atresia patients are as BA group, and the normal liver derived from hepatoblastoma infants during operation are control group. The extent of fibrosis in liver samples was blindly evaluated by two experienced pathologists depending on Ishak system. The BA liver samples were divided into mild liver fibrosis group (grade I–IV, BA_a) and severe liver fibrosis group (grade V–VI, BA_b) to detect Fn14 protein expression.

Results

In mRNA level, Fn14 expression was 21.23?±?8.3 vs. 1.00?±?0.17, p?=?0.023?<?0.05 and CD133 expression was 6.02?±?2.16 vs. 1.14?±?0.75, p?=?0.008?<?0.01 between BA group and control group. Fn14 cells co-expressed the progenitor marker CD133 in liver, and activated in BA. Fn14 andα-SMA were co-location in fibrous area in liver. Compared to the control group, Fn14, CD133, and α-SMA protein expression were 2.10?±?0.53 vs. 0.97?±?0.2, p?=?0.001, 2.23?±?0.57 vs. 1.00?±?0.03, p?=?0.000, 4.96?±?2.4 vs. 1.00?±?0.22, p?=?0.001. The Fn14 protein expression was 2.60?±?0.35 vs. 1.86?±?0.42, p?=?0.012, between BA_b and BA_a group.

Conclusion

Fn14 cells, which co-express the progenitor marker CD133 in liver, are HPCs and activated in BA. Fn14?+?HPCs are associated with liver fibrosis in BA.

     

Iron Stores in Low and Normal Birth Weight Infants at Birth and in Early Infancy

Serum ferritin levels of low birth weight (LBW; BW?<?2,500 g) and normal birth weight (NBW; BW?≥?2,500 g) infants were evaluated at birth and at 3 mo using electrochemiluminescence immunoassay. At birth, levels were 318.6 (31.0–829.5) ng/mL in LBW (n?=?217) and 366.2 (122.4–858.5) ng/mL in NBW infants (n?=?116; p?<?0.01), with 1.4 % of LBW and none of the NBW infants having levels <12 ng/mL (p?=?0.20). At follow up, levels were 66.9 (4.5–567.7) ng/mL in LBW (n?=?126) and 126.2 (6.8–553.7) ng/mL in NBW infants (n?=?76; p?=?0.27), with 11.9 % of LBW and 11.8 % of NBW infants having levels <12 ng/mL (p?=?0.80).     

Effectiveness of gabapentin as a postoperative analgesic in children undergoing appendectomy

Purpose

Though gabapentin is increasingly used as a perioperative analgesic, data regarding effectiveness in children are limited. The purpose of this study was to evaluate gabapentin as a postoperative analgesic in children undergoing appendectomy.

Methods

A 12-month retrospective review of children undergoing appendectomy was performed at a two-hospital children’s institution. Patients receiving gabapentin (GP) were matched (1:2) with patients who did not receive gabapentin (NG) based on age, sex and appendicitis severity. Outcome measures included postoperative opioid use, pain scores, and revisits/readmissions.

Results

We matched 29 (33.3%) GP patients with 58 (66.6%) NG patients (n?=?87). The GP group required significantly less postoperative opioids than the NG group (0.034 mg morphine equivalents/kg (ME/kg) vs. 0.106 ME/kg, p?<?0.01). Groups had similar lengths of time from operation to pain scores?≤?3 (GP 12.21 vs. NG 17.01 h, p?=?0.23). GP and NG had similar rates of revisit to the emergency department (13.8 vs. 10.3%, p?=?0.73), readmission (6.9 vs. 1.7%, p?=?0.26), and revisits secondary to surgical pain (3.4 vs. 3.4%, p?=?1.00).

Conclusion

In this single-center, retrospective cohort study, gabapentin is associated with a reduction in total postoperative opioid use in children with appendicitis. While promising, further prospective validation of clinical effectiveness is needed.

     

Lack of CD4+CD25+FOXP3+ regulatory T cells is associated with resistance to intravenous immunoglobulin therapy in patients with Kawasaki disease

The aim of this study was to investigate changes in CD4⁺CD25⁺FOXP3⁺ regulatory T cells (Tregs) throughout the clinical course of Kawasaki disease (KD) and correlations with response to intravenous immunoglobulin (IVIg) therapy. Participants comprised 18 patients who fulfilled the diagnostic criteria for KD and 20 healthy subjects. Expressions of CD25 and FOXP3 among all CD4⁺ T cells in peripheral blood mononuclear cells were analyzed by flow cytometry before and 7 and 30 days after IVIg therapy. Before treatment, percentages of CD4⁺CD25⁺FOXP3⁺ Tregs among total CD4⁺ Tregs were significantly lower among KD patients (4.19 %; range, 0.16–8.11 %) than among healthy subjects (7.32 %; 4.18–13.42 %; P?=?0.0001). Both percentages and absolute numbers of CD4⁺CD25⁺FOXP3⁺ Tregs on day?7 after IVIg therapy were significantly increased compared with values before treatment (8.02 % (range, 0.51–12.6 %) vs. 4.19 % (range, 0.16–8.11 %), P?=?0.0005; 93.25/?μL (range, 6.67–258.05) vs. 41.85/?μL (range, 0.44–160.62), P?<?0.0001, respectively). Moreover, percentages and absolute numbers of CD4⁺CD25⁺FOXP3⁺ Tregs before treatment were significantly lower in the IVIg-resistant group than in the IVIg-sensitive group (0.18 % (range, 0.16–3.34 %) vs. 4.52 % (range, 2.8–8.11 %), P?=?0.0022; 0.68/μL (range, 0.44–53.81) vs. 51.66/μL (range, 2.88–160.62), P?=?0.0098, respectively). The frequency of CD4⁺CD25⁺FOXP3⁺ Tregs in four of the five IVIg-resistant patients at diagnosis was more than 3 standard deviations below that in healthy subjects. Two of these four patients displayed coronary abnormalities, and one of these two patients developed coronary aneurysm. Conclusion: Lack of CD4⁺CD25⁺FOXP3⁺ Tregs before treatment may predict resistance to IVIg therapy in patients with KD.     

Primary ciliary dyskinesia presentation in 60 children according to ciliary ultrastructure

Primary ciliary dyskinesia (PCD) is an inherited disease related to ciliary dysfunction, with heterogeneity in clinical presentation and in ciliary ultrastructural defect. Our study intended to determine if there are phenotypic differences in patients with PCD based on ciliary ultrastructural abnormality. In this retrospective study carried out among 60 children with a definitive diagnosis of PCD, we analyzed clinical, radiological, and functional features at diagnosis and at last recorded visit, according to cilia defect (absence of dynein arms: DAD group, n?=?36; abnormalities of the central complex: CCA group, n?=?24). Onset of respiratory symptoms occurred later in the CCA than in the DAD group (9.5 versus 0.5 months, p?=?0.03). Situs inversus was only observed in the DAD group, while respiratory disease in siblings were more frequent in the CCA group (p?=?0.003). At diagnosis, clinical presentation was more severe in the CCA group: frequency of respiratory tract infections (p?=?0.008), rhinosinusitis (p?=?0.02), otitis complications (p?=?0.0001), bilateral bronchiectasis (p?=?0.04), and number of hypoxemic patients (p?=?0.03). Pulmonary function remained stable in both groups, but outcome was better in the CCA than in the DAD group: less antibiotic therapy and hypoxemic patients (p?=?0.004). In conclusion, our results underlined the relationship between the severity of clinical presentation and the ultrastructural ciliary defect.     

Steroid Pulse Therapy for Children With Intravenous Immunoglobulin Therapy–Resistant Kawasaki Disease: A Prospective Study

Patients with Kawasaki disease (KD) who did not respond to the initial IVIG are known to have higher risk for developing coronary arterial lesions (CALs). Our aim is to clarify whether patients with initial IVIG resistant KD may benefit from methylprednisolone pulse therapy (MPT) in comparison with re- treatment of IVIG (2nd IVIG). A total of 237 patients (median age: 2 years 2 months; range 1 months–10 years) with KD were initially treated with IVIG (2 g/kg). Among them, 41 patients (22 %) were assessed as IVIG resistance: these patients were allocated to either group A receiving MPT (n = 14) or group B receiving the 2nd IVIG (n = 27). Patients with resistant to the additional therapy (MPT or 2nd IVIG) were received second IVIG (group A) or MPT (group B). Changes in leukocyte count, C-reactive protein and albumin before and after an additional therapy were significantly greater in group A than those in group B. However, the prevalence of CALs did not differ between the groups (36 % in group A and 26 % in group B, p > 0.05). There was no significant difference in the medical cost between the groups (median cost: 92,032 JPY in group A and 97,331 JPY in group B). MPT does not reduce the risk of development to CAL and does not seem to be beneficial as single agent therapy for IVIG resistant KD.     

Variations in the Number of CCL3L1 Gene Copies and Kawasaki Disease in Korean Children

High-dose intravenous immunoglobulin (IVIG) therapy is the highly effective and standard treatment for Kawasaki disease (KD). However, ~20?% of KD patients have persistent fever or recurrence of fever after the initial IVIG treatment, which increases the risk for coronary artery lesions (CALs). Furthermore, the mechanism of IVIG resistance in KD patients still is unknown. The number of CC chemokine ligand 3-like 1 (CCL3L1) gene copies is reported to be associated with KD and IVIG resistance in Japanese patients. In addition, the authors observed significant upregulation of the CCL3L1 gene expression after in vitro immunoglobulin treatment in B cell lines derived from KD patients. Therefore, this study of 459?KD patients and 496 healthy control subjects tested whether the number of CCL3L1 gene copies is associated with a risk of KD, CALs, and/or IVIG resistance in Korean KD patients. However, the number of CCL3L1 gene copies was not associated with KD (P?=?0.18), CAL formation (P?=?0.062), or the IVIG resistance (P?=?0.90). Therefore, the results indicate that the number of CCL3L1 gene copies does not have a role in susceptibility to KD or CALs nor with IVIG resistance in Korean KD patients.     

Can we predict the prognosis of resectable hepatoblastoma from serum alpha-fetoprotein response during preoperative chemotherapy?

Purpose

The objective of this study was to clarify whether the alpha-fetoprotein (AFP) reduction rate during preoperative chemotherapy represents a prognostic factor for hepatoblastoma.

Method

We divided 14 hepatoblastoma patients who underwent preoperative chemotherapy and curative resection into Group A (no recurrence; n?=?10) and Group B (recurrence; n?=?4). We then compared AFP levels before and after preoperative chemotherapy between groups.

Result

Mean AFP level after completing the first cycle of chemotherapy was reduced to 7.28?% (range 1.2–36.8?%) in Group A and 17.05?% (range 12.0–20.5?%) in Group B (p?p?1 log after the first cycle of preoperative chemotherapy survived without recurrence.

Conclusion

A large, early decrease in AFP level during preoperative chemotherapy may offer a strong indicator of survival. Patients in whom AFP levels do not decrease easily during preoperative chemotherapy may have increased risk of recurrence and should be followed very closely.