Face validity of the Kids' ITP Tools (KIT) in the era of thrombopoietin receptor agonists.

The Kids' ITP Tools (KIT) is a questionnaire to assess quality of life of children with immune thrombocytopenia (ITP). The aim of this study was to update this previously validated tool to align with changes in clinical practice, specifically, treatment with thrombopoietin receptor agonists (TPO-RAs). Children aged 1-18 with ITP and/or their families were recruited to participate in interviews to review the KIT. Twenty-six interviews were conducted. Based on interview data from children and families, current guidelines, and expert opinion, five changes were made to the KIT in order to improve its face validity.

Gorham Stout disease of the temporal bone with cerebrospinal fluid leak.

Gorham Stout disease (GSD) is a rare disease characterized by the proliferation of endothelial lined vessels and replacement of bone by fibrous tissue. The main imaging features are progressive osteolysis and cortical resorption. Temporal bone involvement is rare but presents as a destructive bone lesion that may be misinterpreted as more common lytic processes in the pediatric population, such as infection or Langerhans cell histiocytosis. GSD of the temporal bone is associated with cerebrospinal fluid (CSF) leaks, may present with otorrhea, and can mimic other causes of ear drainage. Here, we report the clinical course, imaging features, and outcomes of a 3-year-old girl with GSD of the temporal bone presenting with CSF leak initially attributed to infection.

Neural preservation underlies speech improvement from auditory deprivation in young cochlear implant recipients.

Although cochlear implantation enables some children to attain age-appropriate speech and language development, communicative delays persist in others, and outcomes are quite variable and difficult to predict, even for children implanted early in life. To understand the neurobiological basis of this variability, we used presurgical neural morphological data obtained from MRI of individual pediatric cochlear implant (CI) candidates implanted younger than 3.5 years to predict variability of their speech-perception improvement after surgery. We first compared neuroanatomical density and spatial pattern similarity of CI candidates to that of age-matched children with normal hearing, which allowed us to detail neuroanatomical networks that were either affected or unaffected by auditory deprivation. This information enables us to build machine-learning models to predict the individual children's speech development following CI. We found that regions of the brain that were unaffected by auditory deprivation, in particular the auditory association and cognitive brain regions, produced the highest accuracy, specificity, and sensitivity in patient classification and the most precise prediction results. These findings suggest that brain areas unaffected by auditory deprivation are critical to developing closer to typical speech outcomes. Moreover, the findings suggest that determination of the type of neural reorganization caused by auditory deprivation before implantation is valuable for predicting post-CI language outcomes for young children.

An academic community hospital experience using commercially available molecular testing in the management of indeterminate thyroid nodules.

INTRODUCTION: Molecular thyroid testing is increasingly being used to further stratify risk of malignancy in cytologically indeterminate thyroid nodules. We report our experience using three commercially available tests in a community hospital setting. MATERIALS AND METHODS: All molecular test reports (Afirma, ThyroSeqV2, and ThyGENX/ThyraMIR) on thyroid nodules from Einstein Medical Center, Philadelphia, between April 2014 to March 2017 were compared with follow-up surgical results as part of a quality assurance exercise. Slides and records of disparities were reviewed. RESULTS: Ninety-five thyroid nodules with molecular testing were identified with surgical follow up available on 19. No benign Afirma results had surgical follow-up. All 7 suspicious Afirma results had surgery, with 3 being benign on follow-up. Ten ThyroseqV2 tested nodules had follow-up surgery and included 2 papillary carcinomas following a completely negative result and another papillary carcinoma following over expression of the NIS gene reported as likely benign. One case with a TP53 mutation was benign on follow-up total thyroidectomy. Follow-up on 1 NRAS point mutation by ThyGenX/ThyraMIR was confirmed malignant although the microRNA portion of the test was negative. CONCLUSIONS: Quality assurance review refined our utilization practices as we better appreciated the limitations of molecular testing and use relative to other factors in managing indeterminate thyroid nodules.

Management of acute complicated mastoiditis at an urban, tertiary care pediatric hospital.

OBJECTIVES/HYPOTHESIS: To describe the presentation and management of acute complicated mastoiditis in children. STUDY DESIGN: Retrospective case series. METHODS: An analysis of pediatric patients with acute complicated mastoiditis treated at an urban, tertiary care children's hospital from 2007 to 2014 was performed. RESULTS: Forty-eight patients presented with a total of 67 complications of acute mastoiditis. Mean age at presentation was 4.8 years (range = 0.1-15.3 years). The most common complications were subperiosteal abscess (n = 22, 45.8%), epidural abscess (n = 16, 33.3%), and sigmoid sinus thrombosis (n = 14, 29.2%). The most common pathogens isolated included Streptococcus pneumoniae (n = 14, 29.2%) and group A streptococcus (n = 10, 20.8%). Multidrug resistance was not associated with complication type. Surgical management included myringotomy ± tympanostomy tube placement in 46 (95.8%) patients (the only surgery in 10), drainage of subperiosteal abscess without mastoidectomy in 18 (37.5%) patients, and mastoidectomy in 21 (43.8%) total patients. Patients presenting with intracranial complications were the most likely to undergo a mastoidectomy. Anticoagulation was used in the management of nine of 14 (64.3%) patients presenting with sigmoid sinus thrombosis. Neurosurgical interventions (n = 7, 14.6% patients) were primarily performed to manage increased intracranial pressure. CONCLUSIONS: Subperiosteal abscess was the most common complication of acute mastoiditis, and when occurring as the sole complication was successfully managed with antibiotics and surgical intervention that did not include mastoidectomy. Epidural abscess and sigmoid sinus thrombosis were more prevalent than reported in prior series and were managed more aggressively. These patients were more likely to need neurosurgical interventions. LEVEL OF EVIDENCE: 4. Laryngoscope, 127:2321-2327, 2017.

A screening mechanism to recognize and support at-risk Aboriginal children.

OBJECTIVES: The Aboriginal Children's Health and Well-Being Measure© (ACHWM) was developed to assess health from the perspectives of Aboriginal children. The purpose of this paper is to document the screening process, embedded within the ACHWM, and assess its effectiveness. METHODS: The ACHWM was implemented in 2014/2015 with children 8 to 18 years of age living on the Wiikwemkoong Unceded Territory. Survey responses were screened to identify potential risk, using an automated algorithm run on computer tablets. Local mental health workers conducted brief mental health assessments to identify and support children at-risk. Data were analyzed to estimate effectiveness of this screening process. RESULTS: A total of 293 children completed the ACHWM. The screening tool identified 35% with potential risk. Mental health workers confirmed 18% of all participants as being at-risk, and all were referred for support. The sensitivity of the tool was 75% while specificity was 79%. Improvements to the screening algorithm resulted in a specificity of 97% and negative predictive value of 95%, with no loss of sensitivity. CONCLUSION: Responsible population health surveys require a process to recognize and respond to answers indicative of health risks. This paper provides an example of a screening and triage process that enabled our survey team to screen responses in real time, respond to potential risk immediately, and connect participants to local support services. This process proved essential to conducting an ethical survey. The high specificity and negative predictive value make it an effective triage tool that is particularly valuable in Aboriginal communities and with higher-risk populations.

International Pediatric Otolaryngology Group (IPOG) consensus recommendations: Hearing loss in the pediatric patient.

OBJECTIVE: To provide recommendations for the workup of hearing loss in the pediatric patient. METHODS: Expert opinion by the members of the International Pediatric Otolaryngology Group. RESULTS: Consensus recommendations include initial screening and diagnosis as well as the workup of sensorineural, conductive and mixed hearing loss in children. The consensus statement discusses the role of genetic testing and imaging and provides algorithms to guide the workup of children with hearing loss. CONCLUSION: The workup of children with hearing loss can be guided by the recommendations provided herein.

Magnetic Resonance Imaging of Cochlear Implant Recipients.

OBJECTIVE: Determine the diagnostic usefulness of postimplantation 1.5 T magnetic resonance imaging (MRI) and review magnet-related MRI complications. STUDY DESIGN: Retrospective chart review with additional review of MRIs. SETTING: Tertiary care children's hospital. PATIENTS: Twelve patients who underwent MRI after receiving a cochlear implant (CI). INTERVENTION: One or more episodes of 1.5 T MRI with CI in place. MAIN OUTCOME MEASURES: Occurrence of magnet-related complications; whether imaging was clinically useful. RESULTS: The 12 patients underwent 23 episodes of MRI, including 13 episodes in 11 patients (18 ears) during which a magnet was present and 17 studies were obtained. Complications related to the magnet occurred during 4 of the 13 imaging episodes (30.8%), all during body or spine studies. Magnet torsion with reversal of polarity occurred in three devices; reduced magnet strength in one; and displacement of the magnet from its housing in one. One patient required surgical magnet replacement, whereas other headpiece retention problems were resolved without surgery. All studies but one brain with bilateral magnets were clinically useful. CONCLUSIONS: CI patients who undergo MRI with a magnet in situ may experience complications, especially when imaged below the head. Most complications may be resolved without surgery. Diagnostic usefulness of non-cranial MRI is not likely to be limited by presence of the magnet, while a magnet may prevent clinically useful brain imaging. Obtaining MRI with the magnet in situ avoids the cost and risks associated with multiple surgeries to remove and replace the magnet or the entire implant.

Post-brushing and fine-needle aspiration biopsy follow-up and treatment options for patients with pancreatobiliary lesions: The Papanicolaou Society of Cytopathology Guidelines.

The Papanicolaou Society of Cytopathology (PSC) has developed a set of guidelines for pancreatobiliary cytology including indications for endoscopic ultrasound (EUS) guided fine-needle aspiration (FNA) biopsy, techniques of EUS-FNA, terminology and nomenclature for pancreatobiliary cytology, ancillary testing and post-procedure management. All documents are based on the expertise of the authors, a review of the literature and discussions of the draft document at several national and international meetings over an 18 month period and synthesis of online comments of the draft document on the PSC web site (www.papsociety.org). This document selectively presents the results of these discussions and focuses on the follow-up and treatment options for patients after procedures performed for obtaining cytology samples for the evaluation of biliary strictures and solid and cystic masses in the pancreas. These recommendations follow the six-tiered terminology and nomenclature scheme proposed by committee III.

Postbrushing and fine-needle aspiration biopsy follow-up and treatment options for patients with pancreatobiliary lesions: the Papanicolaou Society of Cytopathology guidelines.

The papanicolaou society of cytopathology (PSC) has developed a set of guidelines for pancreatobiliary cytology including indications for endoscopic ultrasound (EUS) guided fine-needle aspiration (FNA) biopsy, techniques of EUS-FNA, terminology and nomenclature for pancreatobiliary cytology, ancillary testing, and postprocedure management. All documents are based on the expertise of the authors, a review of the literature, discussions of the draft document at several national and international meetings over an 18 month period and synthesis of online comments of the draft document on the PSC web site [www.papsociety.org]. This document selectively presents the results of these discussions and focuses on the follow-up and treatment options for patients after procedures performed for obtaining cytology samples for the evaluation of biliary strictures and solid and cystic masses in the pancreas. These recommendations follow the six-tiered terminology and nomenclature scheme proposed by Committee III.

An examination of adolescent bone tumor patient responses on the Activities Scale for Kids (ASK).

This study provides an examination of responses on the Activities Scale for Kids, performance version (ASKp), for evaluating physical function in adolescents with malignant lower extremity bone tumors. Twenty-one participants (ages 10.4 to 17.9 years), who had tumor resection surgery, completed the ASKp on two occasions. ASKp data were examined for ceiling and/or floor effects, item distributions, and Not Applicable (NA) responses, as well as textual comments. Ceiling effects were 12.5% and 19%, and 0% demonstrated floor effects. The extreme response options were chosen most frequently, and approximately one third of respondents used NA more than 10% of the time. Overall, this population demonstrated moderately high NA rates and higher than anticipated ceiling effects on the ASKp. These data suggest that caution should be taken when interpreting item-level data in this population and further studies guiding the scoring and interpretation of NA responses are recommended.

Approach to intraoperative consultation for donor liver biopsies.

CONTEXT: As demand for organs to treat end-stage liver disease increases, donor livers once deemed only marginally suitable for donation are being considered for transplantation. Pathologists are increasingly being asked to evaluate these livers for acceptability. This article provides guidelines for frozen section evaluation of livers for transplantation. OBJECTIVE: This article concentrates on the histopathologic features of transplant suitability with appropriate clinicopathologic correlation for the practicing pathologist. Recommendations for proper handling and sampling of tissue are discussed. Relative and absolute contraindications as well as artifacts and benign conditions are emphasized. DATA SOURCES: Sources include a compilation of the authors' experiences in academic and community liver transplantation centers. In addition, relevant medical literature was reviewed, as well as Web sites specializing in organ transplantation, such as Transplant Pathology Internet Services and the Organ Procurement and Transplantation Network. CONCLUSIONS: Malignancy and extensive necrosis in the liver are absolute contraindications to transplantation. Evaluation of macrosteatosis, fibrosis, hepatitis, and necrosis depends on the severity of disease and correlation with the clinical situation. Donor age of greater than 60 years does not preclude transplantation. Artifacts and benign conditions need to be understood to prevent wastage of precious organs and to ensure that an appropriate organ is provided for the recipient.

Determinants of quality of life in children with chronic somatic disease: pilot data from the GapS Questionnaire.

PURPOSE: Quality of life (QoL) is a ubiquitous yet poorly defined concept; the precise determinants of QoL are rarely identified. We used pilot data from the GapS Questionnaire to investigate the most important determinants of QoL in children with chronic somatic illness. METHODS: We enrolled 92 participants including 60 parents and 32 of their children. The sample comprised rheumatology, diabetes, epilepsy, gastroenterology, cystic fibrosis, and day unit patients. Trained interviewers administered the GapS Questionnaire to parents, and to children if ≥ 10 years. We determined the relative importance of different items for QoL. RESULTS: Child participants had a mean age of 14.7 years. Children identified "having good friendships", "being happy most days", and "getting along with parents" as most important. Parents ranked most highly "being allowed to do all the things you like doing", "getting told you have done a good job at something", and "being physically able to do everything you enjoy doing". CONCLUSIONS: Physical health items were not as important as social and psychological determinants of QoL in our pilot sample.

Quality of life in childhood immune thrombocytopenia: international validation of the kids' ITP tools.

BACKGROUND: The Kids ITP Tools (KIT) is a disease-specific measure of health-related quality of life for children with immune thrombocytopenia (ITP). To facilitate use in international trials it has been cross-culturally adapted for France, Germany, the United Kingdom and Uruguay. This study assessed the validity and reliability of the translated KIT in comparison to generic quality of life measures. METHODS: Children 2-18 years of age with ITP and their parents were recruited in France, Germany, the United Kingdom and Uruguay. Participants completed the KIT, PedsQL and KINDL. We examined the Pearson's correlation between these measures for our pooled sample and estimated the reliability over a 2-week time period. Findings were further explored by country. RESULTS: A total of 127 families (81 children self-reported) participated. Mean child-reported scores were: KIT 74.3 (SD = 15.3), PedsQL 81.3 (SD = 13.0), and KINDL 70.5 (SD = 14.3). Corresponding mean parent proxy-reported scores were: 70.6 (SD = 18.1), 75.7 (SD = 16.8) and 72.3 (SD = 12.7), respectively. Correlation between KIT and the generic measures was consistent with our a priori hypothesis (PedsQL r = 0.54, KINDL r = 0.48, both P < 0.0001). Child KIT scores for newly diagnosed ITP patients were significantly lower than for chronic ITP patients (67.3 vs. 77.3; P = 0.005). There was a significant correlation (P < 0.001) between the child and parent proxy KIT scores (ICC = 0.52). Child KIT test-retest reliability was acceptable at 0.71. CONCLUSIONS: The cross-culturally translated KIT is valid and reliable with acceptable correlation to the PedsQL and KINDL. There is a significant difference in child self-reported KIT scores between newly diagnosed and chronic ITP.

Pilot study of the effect of romiplostim on child health-related quality of life (HRQoL) and parental burden in immune thrombocytopenia (ITP).

BACKGROUND: Childhood ITP can have a negative impact on the child and his/her family even though it is typically a benign disorder with low risk of serious bleeding. In adults and now children, romiplostim increases the platelet count without significant adverse effects. In this study, the impact of romiplostim treatment on the HRQoL of children with chronic ITP was assessed using the Kid's ITP Tools (KIT). PROCEDURE: Subjects 1-18 years old, with chronic ITP (>6 months), were enrolled in a multi-center, randomized, double-blind, placebo-controlled phase 1/2 treatment study with romiplostim (reported elsewhere). Subjects and/or proxies completed the KIT at baseline, week 5, and week 13. Scores were computed for child self-report (children >7 years), proxy-report, and parent impact. Changes in mean scores from baseline to week 13 were computed. RESULTS: Twenty-two children (17 receiving romiplostim, 5 placebo) and/or their parents provided data. Change in mean scores demonstrated significant improvement in HRQoL for romiplostim versus placebo for parent impact (24 ± 17 vs. -6 ± 8; P = 0.008). Change scores for child self-report trended toward improvement with romiplostim and decreased with placebo (5 ± 10 vs. -7 ± 17; P = 0.29). Romiplostim proxy-report mean change scores were 6 points higher than placebo (8 ± 16 vs. 2 ± 12; P = 0.50). CONCLUSIONS: Romiplostim significantly reduced parental burden in this study. Whether the same and/or additional improvements in HRQoL would be demonstrated by a larger, longer study of romiplostim-treated children with ITP remains to be determined. Pediatr Blood Cancer 2012; 58: 395-398. © 2011 Wiley Periodicals, Inc.

Limitations of universal newborn hearing screening in early identification of pediatric cochlear implant candidates.

OBJECTIVES: To determine whether implementation of universal newborn hearing screening (UNHS) in the state of Illinois has affected the ages at diagnosis of hearing loss and implantation in children receiving cochlear implants and to determine how often children undergoing implantation had UNHS results with no indication of hearing loss (pass). DESIGN: Retrospective case review of 417 randomly selected pediatric implant recipients born before and after UNHS was mandated by law in Illinois. Data analyzed included hearing screening status, ages at initial diagnosis of sensorineural hearing loss (SNHL) and severe to profound SNHL, and age at implantation. SETTING: Tertiary care medical center. PATIENTS: Children receiving implants from 1991 through 2008. MAIN OUTCOME MEASURES: Ages at diagnosis of SNHL and implantation. RESULTS: Children born after legally mandated UNHS had significantly younger ages at diagnosis and implantation. However, a younger age at diagnosis of SNHL was not achieved in children who had passed UNHS or who were not screened. Approximately 30% of pediatric implant recipients passed UNHS, regardless of the cause of hearing loss or the presence or absence of known risk factors. CONCLUSIONS: Almost one-third of our pediatric implant recipients pass UNHS and are older at the time of initial diagnosis and implantation than their peers who fail UNHS. Delayed onset of SNHL limits our ability to achieve early diagnosis and implantation of a significant number of deaf children. This problem will not be solved by the current design of universal hearing screening programs.

Current techniques in management of postmeningitic deafness in children.

OBJECTIVES: To determine pneumococcal vaccination status of children with recent postmeningitic deafness and to review our current approach for achieving early implantation in this special population that is at significant risk for cochlear ossification. DESIGN: Review of imaging studies and test results. SETTING: Tertiary care/referral children's hospital. PATIENTS: Five children ranging in age from 15 months to 10 years who experienced recent onset of profound bilateral sensorineural hearing loss due to pneumococcal meningitis. INTERVENTIONS: All children underwent preoperative magnetic resonance imaging with 3-dimensional heavily T2-weighted steady-state free precession sequences. Four children underwent auditory steady-state response testing. All underwent bilateral cochlear implantation. MAIN OUTCOME MEASURE: Degree of electrode insertion using standard surgical procedures. RESULTS: All children developed meningitis despite a history of pneumococcal vaccination. Complete electrode insertion in both ears was achieved. CONCLUSIONS: Pneumococcal vaccination has reduced but not eliminated childhood deafness secondary to pneumococcal disease. Auditory steady-state response testing and 3-dimensional steady-state free precession imaging are modalities that expedite candidacy evaluation of this population. Early bilateral simultaneous implantation increases the likelihood of binaural hearing and ensures implantation of the better ear in this population of children whose course is often complicated by formation of scar tissue and ossification within the cochlea.

Falsely elevated tacrolimus levels caused by immunoassay interference secondary to beta-galactosidase antibodies in an infected liver transplant recipient.

Careful interpretation of tacrolimus levels is essential to ensure optimal immunosuppressive therapy while avoiding toxicity. Interference with tacrolimus assays may be an underreported event that has the potential to result in negative patient outcomes through unnecessary modifications of therapy. We describe a 55-year-old liver transplant recipient who had falsely elevated tacrolimus levels that led to the eventual disruption of his immunosuppressive therapy and subsequent rejection of his allograft.Although his increased tacrolimus levels did not correlate with clinical signs and symptoms of tacrolimus toxicity, interruption of therapy in this patient was supported by an acute infection and a slight elevation in serum creatinine concentration. Tacrolimus levels were analyzed by using an antibody conjugated magnetic immunoassay method, and levels as high as 79.7 ng/ml were observed, despite discontinuation of tacrolimus. We conducted an evaluation for assay interference by using an alternative assay method(microparticle enzyme immunoassay), by testing plasma samples that were not hemolyzed, and by analyzing levels of an unrelated drug that uses the same technology as the initial tacrolimus assay. beta-galactosidase antibodies were ultimately confirmed as the cause of the immunoassay interference. Inpatients receiving tacrolimus, spuriously high tacrolimus levels should be carefully evaluated, and drastic adjustments to therapy should be made only within the context of clinical toxicity.