CLIN-PEDIATRICS CLINICAL RESEARCH
Ontology highlight
ABSTRACT:
SUBMITTER: Packer R
PROVIDER: S-EPMC3488788 | biostudies-literature | 2012 Oct
REPOSITORIES: biostudies-literature
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Project description:Supratentorial primitive neuroectodermal tumors while histologically similar to medulloblastoma is less curable. Gross total resection and therefore early recognition is crucial to a favorable outcome. We report our experience with a 4 year old girl whose clinical course and radiology were atypical. The patient was referred to our center for management of a seizure disorder manifesting as episodic choking, spitting, drooling and vomiting. MRI showed cortical thickening (right perisylvian fissure; right frontal operculum; insula) patchy/gyriform enhancement of the right sylvian fissure resembling angiomatous dysplasia and clusters of dysplastic meningeal vessels. There was no mass effect, midline shift, ventriculomegaly and MRV/MRA were normal. The radiological differential diagnoses offered were polymicrogyria, focal cortical dysplasia, neuronal migrational disorders, neurofibromatosis and Sturge-Webber syndrome. She had no neurological deficits and seizures were controlled with Topiramate/Levetiracetam. Brain CT 3 months later showed focal dystrophic calcification in the right perisylvian region superimposed on the cortical dysplasia again suggestive of a phakomatosis. ASA was commenced to reduce risk of stroke. MRI performed two months later showed progression of leptomeningeal enhancement involving the suprasellar cistern/perimesencephalic cistern, left sylvian fissure, ventral brainstem and cerebellar vermis. There was tumefactive material in the right pre-pontine cistern and right ventricular effacement. Spinal cord MRI done at this time showed diffuse subarachnoid spread. Throughout this period apart from episodic facial pain the patient had no neurological, speech, ocular, cerebellar or major cognitive deficits. A biopsy was performed and histopathology was consistent with supratentorial PNET. CSF was negative for malignant cells. Due to the extent /inoperability of the tumor, minimal possibility of cure/high likelihood of severe treatment related side-effects parents opted for supportive care rather than curative treatment. The patient passed away a few months later. A high index of suspicion and low threshold to biopsy is recommended for early diagnosis of these tumors.
| S-EPMC3823900 | biostudies-literature
Project description: Not available
| S-EPMC3488786 | biostudies-literature
Project description:Although most children treated for acute lymphoblastic leukemia (ALL) survive, the noncognitive neurologic sequelae of ALL treatment are inadequately described, and most reported studies utilize self-administered questionnaires. We undertook a prospective, IRB-approved, cross-sectional study to define neurologic morbidity in ALL survivors who were more than 5 years from diagnosis. An investigator administered the questionnaire and disability scales, and a board-certified neurologist performed a quantitative neurologic examination. The PedMIDAS tool was completed to determine headache-related disability. The International Headache Society criteria were applied to classify headache type. Of 375 long-term survivors available, 162 consented and participated in the study. There were no significant differences in clinical or biological features between participants and non-participants. Headache was reported by 76 (47%) of the 162 patients: 34 of 70 (49%) girls and 42 of the 92 boys (46%). Median time from onset of the first headache episode was 5.7 years (0.1-18 years), and median age at headache onset was 9.5 years (3.1-27 years). Migraine was diagnosed in 46 survivors (61%), and 25 experienced one or more aura. Episodic tension-type headache was present in 39 (51%) patients, and 17 patients (22%) experienced both migraine and tension-type headaches. Chronic daily headaches were present in 12 survivors (8 with migraine and 4 with tension-type headache). Among survivors with headache, 13 (17%) reported a frequent need to interrupt activities, 36 (47%) occasionally restricted activity, and 27 (36%) did not restrict activity during a headache episode. More than two-thirds (67%) had never seen a physician for headache, and only 8% had visited the hospital emergency room over the last year. PedMIDAS scores revealed no headache-related disability in 42 (55%) patients, mild disability in 22 (29%) patients, and moderate disability in 12 (16%) patients. In conclusion, migraine and tension-type headaches are common in ALL survivors, affect both sexes equally, and in general cause relatively little morbidity.
| S-EPMC3488775 | biostudies-literature
Project description:Skull base tumors are challenging lesions for neurosurgeons, but these cannot always be removed via surgical resection. Helical tomotherapy (HT) delivers small beamlets of photons in continuous rotational treatment arcs and has an advantage of soft-tissue verification using computed tomography. We present our experience with HT for skull base tumors when the tumors were not accessible via surgery or recurred after surgery. From December 2005 to October 2010, 60 patients underwent HT for brain tumors. Among them, 13 patients had skull base tumors; four patients had petroclival meningiomas; two patients had cerebellopontine angle (CPA) meningiomas; two patients had acoustic schwannomas; one patient had foramen magnum meningioma; one patient had sphenoid meningioma; one patient had cavernous meningioma; one patient had jugular foramen meningioma; and one patient had clival meninigioma. Eight patients (62%) underwent surgery before HT. The target size ranged from 0.55 to 141.86 cm3 (median 3.85 cm3), and the target dose ranged from 9 to 57 Gy in 1-31 fractions (median 25 Gy in 5 fractions). The median follow-up period was 11 months. The results of HT for skull base tumors are described in Table 1. Twelve patients (92%) had favorable radiologic response. Six patients (46%) had partial response, and another six patients (46%) had stable disease after HT. One patient with CPA meningioma (case 11) had disease progression and underwent a second surgery. One patient with acoustic schwannoma (case 8) had transient radiation-induced changes on the cerebellum, but other patients did not have radiation-induced complications. HT is a useful treatment modality for skull base tumors when surgical access is not possible, and HT is well tolerated by patients with skull base tumors. PURPOSE: A breast cancer-specific graded prognostic assessment (GPA) was developed to better estimate prognosis for breast cancer patients with brain metastases. This GPA was further refined by adding genetic subtype on the basis of a retrospective, multi-institutional analysis. We sought to externally validate this breast-cancer GPA via an independent, large, multi-institutional data set. MATERIALS AND METHODS: Between 1993 and 2012, 275 women from two institutions who had breast cancer metastases to the brain and complete information available to assign breast-GPA (age, Karnofsky Performance Score, and genetic subtype of luminal A or B, HER2, or basal) were evaluated. Median overall survival (OS) with 95% confidence interval (95% CI) was calculated for each breast-GPA group. Differences between groups were tested using the log-rank test. RESULTS: Of the 275 women, 208 (76%) are deceased. The median OS was 11.5 months (95% CI 8.4, 12.9). The median OS by GPA scores of 0-1.0, 1.5-2.0, 2.5-3.0, 3.5-4.0 were 3.9 (n = 46), 8.0 (n = 88), 13.8 (n = 95), and 17.6 (n = 46) months, respectively (p < 0.0001). These results compared favorably with those of Sperduto et al.: median OS of 3.4 (n = 23), 7.7 (n = 104), 15.1 (n = 140), and 25.3 (n = 133) months, respectively. Genetic subtype was associated with OS, with luminal A and B, HER2, and basal subtypes having a median OS of 12.5 (n = 81), 16.2 (n = 66), 14.9 (n = 34), and 5.8 months (n = 94), respectively (p < 0.0001). CONCLUSIONS: In this cohort of women, the breast-GPA incorporating genetic subtype demonstrated wide variation in prognosis according to GPA score and tumor subtype, in a manner similar to the original report. This analysis validates the prognostic value of including genetic subtype in the breast-GPA, which has been shown to have superior predictive power compared with tumor subtype alone.
| S-EPMC3488792 | biostudies-literature
Project description:As germline genetic testing capacities have improved over the last two decades, increasingly more people are newly diagnosed with germline cancer susceptibility mutations. In the wake of this growth, there remain limitations in both testing strategies and translation of these results into morbidity- and mortality-reducing practices, with pediatric populations remaining especially vulnerable. To face the challenges evoked by an expanding diversity of germline cancer mutations, we can draw upon a model cancer-associated genetic condition for which we have developed a breadth of expertise in managing, Trisomy 21. We can additionally apply advances in other disciplines, such as oncofertility and pharmacogenomics, to enhance care delivery. Herein, we describe the history of germline mutation testing, epidemiology of known germline cancer mutations and their associations with childhood cancer, testing limitations, and future directions for research and clinical care.
| S-EPMC9548803 | biostudies-literature
Project description:The use of genetic information to guide medication decisions holds great promise to improve therapeutic outcomes through increased efficacy and reduced adverse events. As in many areas of medicine, pediatric research and clinical implementation in pharmacogenetics lag behind corresponding adult discovery and clinical applications. In adults, genotype-guided clinical decision support for medications such as clopidogrel, warfarin and simvastatin are in use in some medical centers. However, research conducted in pediatric populations demonstrates that the models and practices developed in adults may be inaccurate in children, and some applications lack any pediatric research to guide clinical decisions. To account for additional factors introduced by developmental considerations in pediatric populations and provide pediatric patients with maximal benefit from genotype-guided therapy, the field will need to develop and employ creative solutions. In this article, we detail some concerns about research and clinical implementation of pharmacogenetics in pediatrics, and present potential mechanisms for addressing them.
| S-EPMC3866691 | biostudies-literature
Project description:Artificial intelligence (AI) broadly describes a branch of computer science focused on developing machines capable of performing tasks typically associated with human intelligence. Those who connect AI with the world of science fiction may meet its growing rise with hesitancy or outright skepticism. However, AI is becoming increasingly pervasive in our society, from algorithms helping to sift through airline fares to substituting words in emails and SMS text messages based on user choices. Data collection is ongoing and is being leveraged by software platforms to analyze patterns and make predictions across multiple industries. Health care is gradually becoming part of this technological transformation, as advancements in computational power and storage converge with the rapid expansion of digitized medical information. Given the growing and inevitable integration of AI into health care systems, it is our viewpoint that pediatricians urgently require training and orientation to the uses, promises, and pitfalls of AI in medicine. AI is unlikely to solve the full array of complex challenges confronting pediatricians today; however, if used responsibly, it holds great potential to improve many aspects of care for providers, children, and families. Our aim in this viewpoint is to provide clinicians with a targeted introduction to the field of AI in pediatrics, including key promises, pitfalls, and clinical applications, so they can play a more active role in shaping the future impact of AI in medicine.
| S-EPMC10940991 | biostudies-literature
Project description:BackgroundRate of nonadherence to antiepileptic drugs (AEDs) in children is about 33%. Engaging clinical pharmacists in the management of patients has proved to increase adherence to medications which will improve the outcomes of treatment.ObjectivesTo investigate the effect of a clinical pharmacist-led education on the adherence to AEDs in pediatric patients with epilepsy. Secondary outcomes include effectiveness and safety of AEDs, satisfaction with information about AEDs provided to the caregivers, and patients quality of life (QoL).MethodsThis was an interventional study where pediatric patients were randomly assigned to the intervention (n = 41) or the control (n = 40) group. A 30-minute clinical pharmacist-led educational interview to the parent/caregiver was provided to the first group as add-on to standard medical care received by latter. Outcomes were measured at baseline and after 8-week follow-up.ResultsThe intervention group had an increase in mean adherence score from 6 ± 1.09 at baseline to 7.6 ± 0.9 at follow-up (P value < 0.001), while the control group had no significant change (P value > 0.05), the difference between the 2 groups at follow-up was significant (P value < 0.0001). No significant difference was observed between groups at follow-up with regard to effectiveness (P value > 0.05), and safety (P value = 0.08). While higher satisfaction with information (P value < 0.0001), and higher QoL (P value < 0.05) was observed in the intervention group.Conclusion and relevanceClinical pharmacist-led education had a positive outcome on pediatric patients with epilepsy with regard to adherence, effectiveness, safety, satisfaction with information about AEDs, and QoL.
| S-EPMC9420919 | biostudies-literature
Project description:ObjectivesHaving shown previously that an electronic prescription writer and decision support system improved pediatric prescribing behavior for otitis media in an academic clinic setting, we assessed whether point-of-care delivery of evidence could demonstrate similar effects for a wide range of other common pediatric conditions.DesignCluster randomized controlled trial.SettingA teaching clinic/clinical practice site and a primary care pediatric clinic serving a rural and semi-urban patient mix.ParticipantsA total of 36 providers at the teaching clinic/practice site and eight providers at the private primary pediatric clinic.InterventionAn evidence-based message system that presented real-time evidence to providers based on prescribing practices for acute otitis media, allergic rhinitis, sinusitis, constipation, pharyngitis, croup, urticaria, and bronchiolitis.Outcome measuresThe proportion of prescriptions dispensed in accordance with evidence.ResultsThe proportion of prescriptions dispensed in accordance with evidence improved four percentage points, from 38% at baseline to 42% following the intervention. The control group improved by one percentage point, from 39% at baseline to 40% at trial's conclusion. The adjusted difference between the intervention and control groups was 8% (95% confidence interval 1%, 15%). Intervention effectiveness did not decrease with time.ConclusionFor common pediatric outpatient conditions, a point-of-care evidence-based prescription writer and decision support system was associated with significant improvements in prescribing practices.
| S-EPMC1876598 | biostudies-literature
Project description:BackgroundCOVID-19 pandemic has significantly impacted the field of medical training, necessitating innovative approaches to education and practice. During this period, the use of novel technologies like virtual reality (VR), augmented reality (AR), and mixed reality (MR) has become increasingly vital. These technologies offer the advantage of transcending the limitations of time and space, thus enabling medical professionals to access various personalized programs for both education and service delivery. This shift is particularly relevant in the realm of pediatric medicine, where traditional training and clinical methods face unique challenges.PurposeThe primary aim of this study is to explore the application of VR, AR, and MR technologies in pediatric medical settings, with a focus on both clinical applications and the training of pediatric medical professionals. We aim to comprehensively search and review studies that have utilized these technologies in the treatment of pediatric patients and the education of healthcare providers in this field.MethodsPeer-reviewed articles published in PubMed, the Cochrane Library, ScienceDirect, Google Scholar, and Scopus from January 1, 2018, to March 1, 2023, were comprehensively searched. The review was conducted according to the PRISMA (Preferred Reporting Items for Systematic review and Meta-Analyses) guidelines. Among the 89 studies, 63 investigated the clinical applications of VR (n=60) or AR (n=3) in pediatric patients, and 25 investigated the applications of VR (n=19), AR (n=5), or MR (n=1) for training medical professionals.ResultsA total of 36 randomized controlled trials (RCTs) for clinical application (n=31) and medical training (n=5) were retrieved. Among the RCTs, 21 reported significant improvements in clinical applications (n=17) and medical training (n=4).ConclusionDespite a few limitations in conducting research on innovative technology, such research has rapidly expanded, indicating that an increasing number of researchers are involved in pediatric research using these technologies.
| S-EPMC10839193 | biostudies-literature