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Permanent URI for this collectionhttps://hdl.handle.net/20.500.14288/6
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Publication Open Access A systematic review of recent and ongoing clinical trials in patients with the neurofibromatoses(Elsevier, 2022) Bedolla, Edwin Nieblas; Armstrong, Amy E.; Hirbe, Angela C.; Acar, Simge; Undergraduate Student; School of MedicineIntroduction: the neurofibromatoses comprise three different genetic conditions causing considerable morbidity and mortality: neurofibromatosis type 1 (NF1), neurofibromatosis type 2 (NF2), and schwannomatosis (SWN). This review summarizes recent and ongoing clinical trials involving patients with neurofibromatoses to better understand the current state of clinical trial research centered around these conditions and inform areas of need. Methods: a search was conducted using the Cochrane Central Register of Controlled Trials and clinicaltrials.gov databases. Inclusion and exclusion criteria were designed to identify clinical trials focused on patients with NF1, NF2, or SWN completed in or after 2010 and in process as of December 31, 2021. Information was collected using standardized guidelines. Results: a total of 134 clinical trials were included, with 75 (56%) completed and 59 (44%) in process. For completed trials, 74% (n = 56) involved patients with NF1, and of those based on specific tumors (n = 26, 46%), the majority focused on plexiform neurofibromas (PNs) (n = 12, 46%). For ongoing trials, 79% (n = 47) involve patients with NF1, and of those based on specific tumors (n = 29, 61%), the majority are focused on PNs (n = 13, 45%). Conclusion: both recent and ongoing clinical trials have primarily focused on patients with NF1 and the treatment of PNs. This research has led to the first FDA-approved drug for NF1-PN and has changed management of these tumors, allowing for systemic therapy rather than reliance on only a surgical modality. Trials evaluating comorbid psychiatric conditions and quality of life among patients with any of the neurofibromatoses appear less common. These areas may warrant focus in future studies to improve clinical management.Publication Open Access Apple peel atresia with isolated fetal ascites and digit anomalies(Springer, 2019) Özlü, Can; Özen, Mehmet Ali; Gürsoy, Tuğba; Oğuzkurt, Nigar Pelin; Faculty Member; Faculty Member; Koç University Hospital; N/A; N/A; 214691; N/APublication Open Access Hemodynamically significant ductus arteriosus and bronchopulmonary dysplasia(Elsevier, 2015) Ovali, Fahri; Gürsoy, Tuğba; Faculty Member; School of Medicine; Koç University Hospital; 214691Publication Open Access COVID-19 associated multisystemic inflammatory syndrome in 614 children with and without overlap with Kawasaki disease-Turk MIS-C study group(Springer, 2022) Çiftdoğan, Dilek Yılmaz; Keleş, Yıldız Ekemen; Çetin, Benhur Şirvan; Karabulut, Nazan Dalgıç; Emiroğlu, Melike; Bağcı, Zafer; Büyükçam, Ayşe; Erdeniz, Emine Hafize; Arga, Gül; Yeşil, Edanur; Çakıcı, Özlem; Karbuz, Adem; Bal, Zümrüt Şahbudak; Kara, Soner Sertan; Özer, Arife; Akcan, Özge Metin; Bozdemir, Şefika Elmas; Anıl, Ayşe Berna; Uygun, Hatice; Kılıç, Ömer; Torun, Selda Hancerli; Ümit, Zuhal; Sütcü, Murat; Özmen, Berfin ÖzgÖkce; Asrak, Hatice Karaoğlu; Alkan, Gülsüm; Aksay, Ahu Kara; Uğur, Cüneyt; Birbilen, Ahmet Ziya; Duramaz, Burcu Bursal; Özkan, Esra Akyüz; Burakay, Özgür; Arslan, Sema Yıldırım; Öncel, Eda Karadağ; Celik, Serkan Fazlı; Kılıç, Ahmet Osman; Özen, Seval; Sarıkaya, Remzi; Demirkol, Demet; Arslan, Gazi; Türel, Özden; Sert, Ahmet; Sarı, Ergül; Orbak, Zerrin; Şahin, İrfan Oğuz; Varan, Celal; Öz, Sadiye Kübra Tuter; Durak, Fatih; Oflaz, Mehmet Burhan; Kara, Manolya; Karpuz, Derya; Petmezci, Mey Talip; Hatipoğlu, Nevin; Öncel, Selim; Turgut, Mehmet; Elmalı, Ferhan; Somer, Ayper; Kuyucu, Necdet; Dinleyici, Ener Cağrı; Kurugöl, Zafer; Çiftci, Ergin; Kara, Ateş; Aktürk, Hacer; Faculty Member; Koç University HospitalMultisystemic inflammatory syndrome (MIS-C) diagnosis remains difficult because the clinical features overlap with Kawasaki disease (KD). The study aims to highlight the clinical and laboratory features and outcomes of patients with MISC whose clinical manifestations overlap with or without KD. This study is a retrospective analysis of a case series designed for patients aged 1 month to 18 years in 28 hospitals between November 1, 2020, and June 9, 2021. Patient demographics, complaints, laboratory results, echocardiographic results, system involvement, and outcomes were recorded. A total of 614 patients were enrolled; the median age was 7.4 years (interquartile range (IQR) 3.9-12 years). A total of 277 (45.1%) patients with MIS-C had manifestations that overlapped with KD, including 92 (33.3%) patients with complete KD and 185 (66.7%) with incomplete KD. Lymphocyte and platelet counts were significantly lower in patients with MISC, overlapped with KD (lymphocyte count 1080 vs. 1280 cells x mu L, p = 0.028; platelet count 166 vs. 216 cells x 10(3)/mu L, p < 0.001). The median serum procalcitonin levels were statistically higher in patients overlapped with KD (3.18 vs. 1.68 mu g/L, p = 0.001). Coronary artery dilatation was statistically significant in patients with overlap with KD (13.4% vs. 6.8%, p = 0.007), while myocarditis was significantly more common in patients without overlap with KD features (2.6% vs 7.4%, p = 0.009). The association between clinical and laboratory findings and overlap with KD was investigated. Age > 12 years reduced the risk of overlap with KD by 66% (p < 0.001, 95% CI 0.217-0.550), lethargy increased the risk of overlap with KD by 2.6-fold (p = 0.011, 95% CI 1.244-5.439), and each unit more albumin (g/dl) reduced the risk of overlap with KD by 60% (p < 0.001, 95% CI 0.298-0.559). Conclusion: Almost half of the patients with MISC had clinical features that overlapped with KD; in particular, incomplete KD was present. The median age was lower in patients with KD-like features. Lymphocyte and platelet counts were lower, and ferritin and procalcitonin levels were significantly higher in patients with overlap with KD.Publication Open Access Can serum periostin, YKL-40, and osteopontin levels in pre-school children with recurrent wheezing predict later development of asthma?(Wiley, 2021) Güvenir, Hakan; Çelik, İlknur Külhaş; Civelek, Ersoy; Süloğlu, Aysun Kılıç; Karaaslan, Çağatay; Genç, Selcan; Mısırlıoğlu, Emine Dibek; Toyran, Müge; Giniş, Tayfur; Kocabaş, Can N.; Büyüktiryaki, Ayşe Betül; Faculty Member; Koç University HospitalBackground: currently, there are no reliable clinical tools available for predicting asthma in pre-school-aged children with recurrent wheezing. The aim of this study was to evaluate the usefulness of serum periostin, YKL-40, and osteopontin biomarkers in wheezy pre-school-aged children for predicting the development of asthma in school ages. Methods: the study was prospectively conducted between 2011 and 2017. The clinical features of the pre-school-aged children with recurrent wheezing and the levels of serum periostin, YKL-40, and osteopontin were measured. The same participants were reevaluated in school-age period, and participants with asthma were identified. Relative risk (RR) for the development of asthma was analyzed. Results: of the 197 pre-school-aged children with recurrent wheezing who were reevaluated in school-age years, 32% of them had asthma. Serum periostin, YKL-40, and osteopontin levels at admission could not predict participants who would have asthma symptoms in school-age years. The RR for continuing of asthma symptoms was higher in participants who had their first wheezing episode before 1 year of age, preterm birth, cesarean section delivery, prenatal smoking exposure, multi-trigger wheezing, parental asthma, modified asthma predictive index positivity, prophylactic vitamin D intake < 12 months, breastfeeding times 12 month, and aeroallergen sensitivity [RR (95% CI) and P value: 2.813 (1.299-6.091), 0,002; 1.972 (1.274-3.052), 0.009; 1.929 (1.195-3.114), 0.004; 2.232 (1.463-3.406), <0.001; 3.152 (1.949-5.097), <0.001; 1.730 (1.144-2.615), 0.016; 2.427 (1.559-3.777), <0.001; 2.955 (1.558-5.604), <0.001; 1.767 (1.084-2.881), 0.016; 0.765 (0.556-1.053), 0.016; respectively]. Conclusion: results have shown that clinical features were more valuable than biomarkers in predicting having asthma in school-age years in participants who had recurrent wheezing in pre-school-age period.Publication Open Access COVID-19 in pediatric patients undergoing chronic dialysis and kidney transplantation(Springer, 2021) Canpolat, Nur; Yıldırım, Zeynep Yürük; Yıldız, Nurdan; Göknar, Nilufer; Evrengül, Havva; Gülmez, Ruveyda; Aksu, Bağdagül; Dursun, Hasan; Özçelik, Gül; Yavaşcan, Önder; Çicek, Rumeysa Yasemin; Tulpar, Sebahat; HacIhamdioğlu, Duygu Övünç; Nayır, Ahmet; Alpay, Harika; Taşdemir, Mehmet; Doctor; Koç University HospitalThe study aims to present the incidence of COVID-19 in pediatric patients undergoing renal replacement therapy (RRT) and to compare the severity and outcomes of the disease between the dialysis and kidney transplant (KTx) groups. This multicenter observational study was conducted between 1 April and 31 December 2020 in Istanbul. Members of the Istanbul branch of the Turkish Pediatric Nephrology Association were asked to report all confirmed cases of COVID-19 who were on RRT, as well as the number of prevalent RRT patients under the age of 20. A total of 46 confirmed cases of COVID-19 were reported from 12 centers, of which 17 were dialysis patients, and 29 were KTx recipients. Thus, the incidence rate of COVID-19 was 9.3% among dialysis patients and 9.2% among KTx recipients over a 9-month period in Istanbul. Twelve KTx recipients and three dialysis patients were asymptomatic (p = 0.12). Most of the symptomatic patients in both the dialysis and KTx groups had a mild respiratory illness. Only two patients, one in each group, experienced a severe disease course, and only one hemodialysis patient had a critical illness that required mechanical ventilation. In the entire cohort, one hemodialysis patient with multiple comorbidities died. Conclusion: while most cases are asymptomatic or have a mild disease course, pediatric patients undergoing dialysis and a kidney transplant are at increased risk for COVID-19. What is known: in adult population, both dialysis patients and kidney transplant recipients are at increased risk for severe illness of COVID-19 and have higher mortality rate. Children with kidney transplantation are not at increased risk for COVID-19 and most have mild disease course. Data on children on dialysis are scarce. What is new: pediatric patients undergoing dialysis and kidney transplantation have an increased risk for COVID-19. Most patients undergoing renal replacement therapy either on dialysis or transplanted develop asymptomatic or mild COVID-19 disease with a favorable outcome.