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biologic therapy in children

Seong Heon Kim, Hye Young Kim, Su Young Kim
Hemolytic uremic syndrome (HUS) is often encountered in children with acute kidney injury. Besides the well-known shiga toxin-producing Escherichia coli -associated HUS, atypical HUS (aHUS) caused by genetic complement dysregulation has been studied recently. aHUS is a rare, chronic, and devastating disorder that progressively damages systemic organs, resulting in stroke, end-stage renal disease, and death. The traditional treatment for aHUS is mainly plasmapheresis or plasma infusion; however, many children with aHUS will progress to chronic kidney disease despite plasma therapy...
February 2018: Korean Journal of Pediatrics
Y J Xue, J Wu, Y X Zuo, Y P Jia, A D Lu, L P Zhang
Objective: To explore the clinical features and prognostic factors of Ph-positive and/or BCR-ABL positive acute lymphoblastic leukemia (Ph+ ALL) in children. Methods: The clinical data of 68 Ph+ ALL children who were treated at Peking University People's Hospital from December 2006 to December 2016 was retrospectively reviewed. Survival analysis were estimated by Kaplan-Meier method. Univariate analysis was estimated by Log-rank test and Chi-square, and multivariate analysis was estimated by Cox proportional hazards regression model...
March 14, 2018: Zhonghua Xue Ye Xue za Zhi, Zhonghua Xueyexue Zazhi
Paul J Choi, Joe Iwanaga, R Shane Tubbs, Emre Yilmaz
Owing to its rarity, rhabdomyosarcoma of the head and neck (HNRMS) has seldom been discussed in the literature. As most of the data is based only on the retrospective experiences of tertiary healthcare centers, there are difficulties in formulating a standard treatment protocol. Moreover, the disease is poorly understood at its pathological, genetic, and molecular levels. For instance, 20% of all histological assessment is inaccurate; even an experienced pathologist can confuse rhabdomyosarcoma (RMS) with neuroblastoma, Ewing's sarcoma, and lymphoma...
January 9, 2018: Curēus
Alexandra Okihiro, Rachana Hasija, Lillia Fung, Bonnie Cameron, Brian M Feldman, Ronald Laxer, Rayfel Schneider, Earl Silverman, Lynn Spiegel, Rae S M Yeung, Shirley M L Tse
BACKGROUND: Anti-TNF (Tumor necrosis factor) therapy is effective in treating pediatric patients with refractory rheumatic disease. There is however a concern that anti-TNF usage may increase the risk of malignancy. Reports on specific types of malignancy in this patient population have been emerging over the past decade, but there is a need for additional malignancy reports, as these events are rare. Therefore, a retrospective chart review was performed on the biologic database of pediatric rheumatology patients at The Hospital for Sick Children (SickKids) from 1997 to 2013 for neoplasms, patient demographic information and rheumatologic treatment course...
March 14, 2018: Pediatric Rheumatology Online Journal
Sean A McGhee
PURPOSE OF REVIEW: Although biologic therapies can provide outstanding efficacy in the management of lung disease, especially asthma, most of these agents have been approved only for adults. Recent findings provide new strategies for using these agents in children. RECENT FINDINGS: Extensive evidence has consistently demonstrated the efficacy and safety of biologic therapy for asthma. In addition, some studies have documented potentially important secondary effects, such as improving response to respiratory virus infection in asthmatic patients...
March 13, 2018: Current Opinion in Pediatrics
Meng Liu, Jigisha P Thakkar, Catherine R Garcia, Therese A Dolecek, Lars M Wagner, Emily Van Meter Dressler, John L Villano
Glioblastoma in children is an aggressive disease with no defined standard therapy. We evaluated hospital-based demographic and survival patterns obtained through the National Cancer Database to better characterize children with glioblastoma. Our study identified 1173 patients from 0 to 19 years of age between 1998 and 2011. Comparisons were made among demographics, clinical characteristics, treatment, and survival variables. Fifty-four percent of patients were over 10 years of age. Approximately 80% of patients underwent either partial or complete resection...
March 13, 2018: Cancer Medicine
Christopher C Dvorak, Prakash Satwani, Elliot Stieglitz, Mitchell S Cairo, Ha Dang, Qinglin Pei, Yun Gao, Donna Wall, Tali Mazor, Adam B Olshen, Joel S Parker, Samir Kahwash, Betsy Hirsch, Susana Raimondi, Neil Patel, Micah Skeens, Todd Cooper, Parinda A Mehta, Stephan A Grupp, Mignon L Loh
BACKGROUND: Most patients with juvenile myelomonocytic leukemia (JMML) are curable only with allogeneic hematopoietic cell transplantation (HCT). However, the current standard conditioning regimen, busulfan-cyclophosphamide-melphalan (Bu-Cy-Mel), may be associated with higher risks of morbidity and mortality. ASCT1221 was designed to test whether the potentially less-toxic myeloablative conditioning regimen containing busulfan-fludarabine (Bu-Flu) would be associated with equivalent outcomes...
March 12, 2018: Pediatric Blood & Cancer
Joshua L Pierce, A Lindsay Frazier, James F Amatruda
Germ cell tumors (GCTs) arising in infants, children, and adolescents present a set of special challenges. GCTs make up about 3% of malignancies in children aged 0-18 and nearly 15% of cancers in adolescents. Epidemiologic and molecular evidence suggests that GCTs in young children likely represent a distinct biologic group as compared to GCTs of older adolescents and adults. Despite this difference, pediatric GCTs are typically treated with cisplatin-based multiagent regimens similar to those used in adults...
2018: Advances in Urology
Fabian Speth, Claas H Hinze, Susanne Andel, Thomas Mertens, Johannes-Peter Haas
BACKGROUND: The goal of this study was to apply the varicella zoster virus (VZV) vaccine to patients with pediatric rheumatic diseases (PRD) at risk for severe chickenpox, without interrupting their current immunosuppression, including biological agents, using an immunological-based pre-vaccination checklist to assure safety. A pre-vaccination checklist was implemented to ensure adequate immune competence prior to immunization. METHODS: This prospective study included seronegative patients (VZV-IgG ≤200 mIU/ml) and patients who had previously received only a single dose of VZV vaccine...
March 2, 2018: Pediatric Rheumatology Online Journal
Nataliya P Buxbaum, Cemre Robinson, Ninet Sinaii, Alexander Ling, Lauren M Curtis, Steven Z Pavletic, Kristin Baird, Maya B Lodish
Pediatric allogeneic hematopoietic stem cell transplant (AHSCT) recipients with chronic graft-versus-host disease (cGVHD) are at high risk of endocrinopathies, particularly impaired bone mineral density (BMD). However, rates of BMD impairment in pediatric AHSCT recipients with cGVHD have not been well documented. We describe 33 patients with cGVHD that were referred to the NIH for the Natural History of Clinical and Biological Factors Determining Outcomes in Chronic Graft-Versus-Host Disease Study (NCT 0092235) and underwent formal BMD assessments via dual-energy X-ray absorptiometry (DEXA)...
February 26, 2018: Biology of Blood and Marrow Transplantation
Ryan Ivancic, Hassan Iqbal, Brad deSilva, Quintin Pan, Laura Matrka
Objectives: Recurrent respiratory papillomatosis (RRP) is a chronic disease of the respiratory tract that occurs in both children and adults. It is caused by the human papillomavirus (HPV), in particular low-risk HPV6 and HPV11, and aggressiveness varies among patients. RRP remains a chronic disease that is difficult to manage. This review provides perspectives on current and future management of RRP. Results: The current standard of care is surgical excision, with adjuvant therapies as needed...
February 2018: Laryngoscope Investigative Otolaryngology
Beat Bojaxhiu, Frank Ahlhelm, Marc Walser, Lorenzo Placidi, Ulrike Kliebsch, Lorentzos Mikroutsikos, Petra Morach, Alessandra Bolsi, Tony Lomax, Alessia Pica, Damien C Weber
PURPOSE: To assess the rate of radiation necrosis (RN) and white matter lesions (WMLs) in pediatric patients with primary brain tumors treated with pencil beam scanning (PBS) proton therapy (PT) with or without concomitant chemotherapy at the PSI. METHODS AND MATERIALS: Between 1999 and 2015, 171 pediatric patients (age <18 years) were treated with PT. Median age at diagnosis was 3.3 years (range, 0.3-17.0 years), and the median delivered dose was 54 Gy (relative biological effectiveness) (range, 40...
March 15, 2018: International Journal of Radiation Oncology, Biology, Physics
Laetitia Padovani, Frédérique Chapon, Nicolas André, Mohamed Boucekine, Anne Geoffray, Franck Bourdeau, Julien Masliah-Planchon, Line Claude, Aymeri Huchet, Anne Laprie, Stephane Supiot, Bernard Coche-Dequéant, Christine Kerr, Claire Alapetite, Julie Leseur, Tandat Nguyen, Sophie Chapet, Valerie Bernier, Pierre-Yves Bondiau, Georges Noel, Jean Louis Habrand, Stephanie Bolle, François Doz, Christelle Dufour, Xavier Muracciole, Christian Carrie
PURPOSE: To identify the incidence of patients with perihippocampal metastases to assess the risk of brain relapse when sparing the hippocampal area. Medulloblastoma (MB) represents 20% of pediatric brain tumors. For high-risk MB patients, the 3- to 5-year event-free survival rate has recently improved from 50% to >76%. Many survivors, however, experience neurocognitive side effects. Several retrospective studies of patients receiving whole brain irradiation (WBI) have suggested a relationship between the radiation dose to the hippocampus and neurocognitive decline...
March 15, 2018: International Journal of Radiation Oncology, Biology, Physics
Kiran Tawana, Michael W Drazer, Jane E Churpek
Comprehensive genomic profiling of myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML) cases have enabled the detection and differentiation of driver and subclonal mutations, informed risk prognostication, and defined targeted therapies. These insights into disease biology, and management have made multigene-acquired mutation testing a critical part of the diagnostic assessment of patients with sporadic MDS and AML. More recently, our understanding of the role of an increasing number of inherited genetic factors on MDS/AML risk and management has rapidly progressed...
February 27, 2018: Leukemia: Official Journal of the Leukemia Society of America, Leukemia Research Fund, U.K
Jendrik Hardes, Georg Gosheger, Timo Budny
Bone sarcomas are extremely rare representing approximately 0.2% among all cancer types. Due to the rarity of these tumors both the patients and the physicians can overlook or misinterpret the first often unspecific symptoms of these tumors (pain, limping and swelling). Therefore, radiographic examination (X-ray and/or MRI) of the involved region in case of inexplicable symptoms is strongly recommended. The most common primary malignant bone tumor entity is osteosarcoma, followed by chondrosarcoma and Ewing sarcoma...
February 2018: Zeitschrift Für Orthopädie und Unfallchirurgie
Virginia Mirra, Silvia Montella, Francesca Santamaria
BACKGROUND: The primary goal of asthma management is to achieve disease control for reducing the risk of future exacerbations and progressive loss of lung function. Asthma not responding to treatment may result in significant morbidity. In many children with uncontrolled symptoms, the diagnosis of asthma may be wrong or adherence to treatment may be poor. It is then crucial to distinguish these cases from the truly "severe therapy-resistant" asthmatics by a proper filtering process...
February 21, 2018: BMC Pediatrics
Pratap Kumar Patra, Manish Kumar
Background: Data on rheumatological disorders in children from developing countries like India are scarce. Hence, this study aimed to understand the clinical and epidemiological profile of rheumatological disorders in children as this can help organize comprehensive evidence-based health care services. Methodology: A retrospective hospital-based study was designed in pediatric rheumatology clinic of the All India Institute of Medical Sciences, Patna, India, from January 2015 to December 2016...
January 2018: Journal of Natural Science, Biology, and Medicine
Sandy R Durrani, Vincent A Mukkada, Theresa W Guilbert
Eosinophilic esophagitis and asthma are frequently found as comorbid conditions in children and adults along with other manifestations of atopic diathesis. These two conditions have similar T helper 2 responses-driven pathophysiology and share common management strategies such as using systemic corticosteroids and targeted anti-cytokine biologic therapies. Review of the literature finds that asthma is often a comorbid condition in eosinophilic esophagitis in both children and adults; however, the EoE-asthma relationship remains poorly characterized mechanistically and clinically...
February 18, 2018: Clinical Reviews in Allergy & Immunology
Iris Deitch, Radgonde Amer, Oren Tomkins-Netzer, Zohar Habot-Wilner, Ronit Friling, Ron Neumann, Michal Kramer
PURPOSE: This study aimed to report the clinical outcome of children with uveitis treated with anti-tumor necrosis factor alpha (TNF-α) agents. METHODS: This included a retrospective cohort study. Children with uveitis treated with infliximab or adalimumab in 2008-2014 at five dedicated uveitis clinics were identified by database search. Their medical records were reviewed for demographic data, clinical presentation, ocular complications, and visual outcome. Systemic side effects and the steroid-sparing effect of treatment were documented...
February 18, 2018: Graefe's Archive for Clinical and Experimental Ophthalmology
Shavali Shaik, Bridget Kennis, Shinji Maegawa, Keri Schadler, Yang Yanwen, Keri Callegari, Rishi R Lulla, Stewart Goldman, Javad Nazarian, Veena Rajaram, Jason Fangusaro, Vidya Gopalakrishnan
Diffuse intrinsic pontine glioma (DIPG) is a highly aggressive glial tumor that occurs in children. The extremely poor median and 5-year survival in children afflicted with DIPG highlights the need for novel biology-driven therapeutics. Here, we have implicated the chromatin remodeler and regulator of brain development called RE1 Silencing Transcription Factor (REST), in DIPG pathology. We show that REST protein is aberrantly elevated in at least 21% of DIPG tumors compared to normal controls. Its knockdown in DIPG cell lines diminished cell growth and decreased their tumorigenicity in mouse intracranial models...
January 12, 2018: Oncotarget
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