Is phototherapy exposure associated with better or worse outcomes in 501‐ to 1000‐g‐birth‐weight infants?

Aim: To compare risk‐adjusted outcomes at 18‐ to 22‐month‐corrected age for extremely low birth weight (ELBW) infants who never received phototherapy (NoPTx) to those who received any phototherapy (PTx) in the NICHD Neonatal Research Network randomized trial of Aggressive vs. Conservative Phototherapy. Methods: Outcomes at 18 to 22‐month‐corrected age included death, neurodevelopmental impairment (NDI) and Bayley Scales Mental Developmental Index (MDI). Regression models evaluated the independent association of PTx with adverse outcomes controlling for centre and other potentially confounding variables. Results: Of 1972 infants, 216 were NoPTx and 1756 were PTx. For the entire 501‐ to 1000‐g‐BW cohort, PTx was not independently associated with death or NDI (OR 0.85, 95% CI: 0.60–1.20), death or adverse neurodevelopmental endpoints. However, among infants 501–750 g BW, the rate of significant developmental impairment with MDI < 50 was significantly higher for NoPTx (29%) than PTx (12%) (p = 0.004). Conclusions: Phototherapy did not appear to be independently associated with death or NDI for the overall ELBW group. Whether PTx increases mortality could not be excluded because of bias from deaths before reaching conservative treatment threshold. The higher rate of MDI < 50 in the 501‐ to 750‐g‐BW NoPTx group is concerning and consistent with NRN Trial results.     

Use of subcutaneous treprostinil in pediatric pulmonary arterial hypertension—Bridge‐to‐transplant or long‐term treatment?

PAH is a progressive life‐threatening disease in children. While parenteral prostacyclin therapy improves survival in patients with severe PAH, central line‐related complications are common. Our aim was to assess the efficacy, safety, and tolerability of subcutaneous treprostinil treatment in pediatric PAH patients. Eight patients were treated with subcutaneous treprostinil at the Pediatric Heart Center Budapest. Indications for subcutaneous treprostinil therapy were clinical worsening and/or echocardiographic progression or switch from intravenous to subcutaneous therapy. Following treprostinil initiation, clinical status improved or did not change in four of eight patients. Two patients were lost early during treprostinil therapy, parenteral treprostinil as a rescue therapy being insufficient in these cases. The final dose in long‐term treated patients was between 60 and 100 ng/kg/min. Aside from thrombocytopenia, other severe side effects were not observed. Potts shunt was performed as palliative treatment in two cases. Three patients had successful lung transplantation, and one died while on the waiting list. Long‐term subcutaneous treprostinil could be a safe and well‐tolerated therapy in children with severe PAH even at higher doses. It may serve as an alternative to intravenous prostacyclin treatment allowing to avoid the potential complications of permanent central line placement.     

Diagnosis of gastrointestinal graft‐versus‐host disease—Is rectal biopsy enough?

BACKGROUND: Graft-versus-host disease (GVHD) is an important cause of morbidity and mortality after allogeneic bone marrow transplantation (BMT). The clinical diagnosis of gastrointestinal GVHD can be difficult to establish and endoscopic diagnosis entails a procedural risk. The aim of this study was to determine whether rectal biopsy alone might be sufficient to establish or exclude the diagnosis of intestinal GVHD. METHODS: A retrospective chart review of children with histological evidence of gastrointestinal GVHD after allogeneic BMT at the Royal Children's Hospital in Melbourne, Australia, between January 1981 and July 2004. RESULTS: During the study period, 264 patients received allogeneic BMT. Thirty-three had either an upper or lower gastrointestinal endoscopy, or both. Of these, 14 (8 M: 6 F, mean age 9 years 5 months at the time of BMT) had histological features suggestive of GVHD in at least one gastrointestinal biopsy. Overall, 9 of 14 could have been diagnosed with GVHD on the basis of rectal biopsies alone (negative predictive value: 64%). Gastroscopy was needed to establish the diagnosis in a further five patients. Multiple biopsies obtained from each site in the lower gastrointestinal tract showed similar histological findings, but there was frequent non-agreement between biopsies obtained at differing sites within the upper gastrointestinal tract. CONCLUSIONS: Based on these results, we suggest that regardless of symptoms, rectal biopsy should initially be performed to identify gastrointestinal GVHD. Gastroscopy should be added only if the rectal biopsy is unhelpful and there is still good reason to suspect GVHD.     

Does a meso‐caval shunt have positive effects in a pig large‐for‐size liver transplantation model?

In pediatric liver transplantations with LFS grafts, higher incidences of graft dysfunction probably occur due to IRI. It was postulated that increasing the blood supply to the graft by means of a meso‐caval shunt could ameliorate the IRI. Eleven pigs underwent liver transplantation and were divided into two groups: LFS and LFS+SHUNT group. A series of flowmetric, metabolic, histologic, and molecular studies were performed. No significant metabolic differences were observed between the groups. One hour after reperfusion, portal flow was significantly lower in the recipients than in the donors, proving that the graft was maintained in low portal blood flow, although the shunt could promote a transient increase in the portal blood flow and a decrease in the arterial flow. Finally, it was verified that the shunt promoted a decrease in inflammation and steatosis scores and a decrease in the expression of the eNOS gene (responsible for the generation of nitric oxide in the vascular endothelium) and an increase in the expression of the proapoptotic gene BAX. The meso‐caval shunt was responsible for some positive effects, although other deleterious flowmetric and molecular alterations also occurred.     

How do parents of 4‐ to 5‐year‐old children perceive the weight of their children?

Introduction: A heavier weight in adults is becoming the norm rather than an abnormal weight. Whether the same trend is happening in children is unknown. Objective: To assess the perception of the weight of 4‐ to 5‐year‐old children and the recognition of overweight by both parents. Design: Population‐based survey. Participants: A questionnaire was sent to parents of 1155 4‐ to 5‐year‐old children. Results: In total, 439 questionnaires (35%) were returned. Of all, 90% of the children had a normal weight, 9.3% were overweight and 4.1% were obese. For all weight classes, the parents depicted the child as lighter on both the verbal and visual scale. Of all, 75% of mothers of overweight children stated that the child had a normal weight. In obese children, 50% of the mothers believed that the child had a normal weight. Conclusion: Children with a weight in the normal range were considered by their parents as a little too light or too light. Overweight was considered as normal weight, and obesity as normal or a little too heavy. The perception of a normal weight in children at 4–5 years is distorted.     

Non‐compliance in children post‐liver transplant. Who are the culprits?

Although non-compliance in pediatric liver transplants is known to be a major cause of late graft loss and patient mortality, follow-up seems inconsistent. As liver transplant becomes a luxury because of the shortage of organs, the need to maximize graft and patient survival by intense monitoring becomes a necessity. When evaluating children with elevated liver enzymes post-transplant, early or late non-compliance should always be suspected. The risk of non-compliance in children with chronic illness varies from 10 to 89%. In a study by Sudan et al. non-compliance was one of the leading causes of late mortality in children age 10-17 yr. Although it is well documented that teenagers have a high rate of non-compliance, the rate in the younger children has not been documented. In our series, we found that parental non-compliance comprises the majority of our problems with liver dysfunction, hospitalization, and graft loss. The purpose of this study was to evaluate the incidence of non-compliance in children post-liver transplant. A retrospective chart review of patient records from admissions and outpatient records was performed for documentation of elevated enzymes and low immunosuppressive levels. From July 1987 to December 2002, our program performed 266 liver transplants in 234 children, with 1-yr graft survival of 84% and 1-yr patient survival of 90%. Our overall patient survival was 85% with 77% graft survival. There were 40 children with documented non-compliance with mild to severe liver dysfunction in this study. Twenty-eight of these children were younger than 10 yr [28 of 40 (46%) <5 yr], and 12 (30%) were older than 10 yr at the time of rejection. In 10 of 40 children, there was one documented incident of non-compliance, while 26 of 40 had two to four incidents, and four had five or more documented events. Our children (50%) came from two-parent households. The remaining 50% were from single households. In 27 of 40 (68%) children, rejection was confirmed by liver biopsy. In children on cyclosporine (Neoral; Novartis, East Hanover, NJ, USA) with a known history of non-compliance and low immunosuppressive levels, C2 monitoring was performed to verify absorption. Admission for drug monitoring and verification of non-compliance was accomplished in 32 of 40 (80%). Four of the 40 children (10%) were retransplanted, and one child had died. In conclusion, non-adherence to medications remains a major source of graft loss and morbidity post-transplant. We found that non-compliance crosses all socio-economic and cultural groups and that flexibility of clinic hours, shortened time between visits, and decreased numbers and times of medication will increase adherence.