Sonay Incesoy Ozdemir

Objective: Anthracycline-based chemotherapy is used in 50%-60% of childhood cancers despite well-known cardiotoxicity. The authors evaluated dexrazoxane (DEX) as a cardioprotective agent in children receiving anthracyclines. Methods: The authors retrospectively analyzed 142 children aged 0-18 years treated for childhood cancer. The control group comprised 87 patients who received anthracycline alone (2009-2019) and the case group 55 patients who received DEX + anthracycline (2019-2022, after DEX was licensed in Türkiye). Cardiac dysfunction was defined as a left ventricular ejection fraction (LVEF) below 50%. Results: Baseline LVEF was comparable. Mean LVEF fell by 0.71 Å} 6.55 points with DEX vs. 6.44 Å} 9.76 in controls (Time Å~ Group interaction F(1,140) = 14.77, P < .001), and the difference persisted after adjustment for follow-up duration and treatment covariates (β = +4.60, 95% CI = 1.10-8.10, P = .010). Cardiac dysfunction developed in 11 of 87 controls (12.6%) and in none of the 55 DEX patients (P = .007; log-rank P = .040); this held in a 36-month landmark analysis (0 of 52 vs. 4 of 25, P = .009). Mortality was higher in controls (21.8% vs. 5.5%, P = .009) and in patients with cardiac dysfunction (54.5% vs. 17.1%, P = .012). Conclusion: Dexrazoxane attenuated the early decline in LVEF and reduced the incidence of anthracycline-induced cardiotoxicity, and this persisted after adjustment for the difference in follow-up duration. With a median DEX follow-up of only 22.8 months, these data support early cardioprotection; longer follow-up is needed to assess protection against late-onset cardiotoxicity.

Çocukluk çağı kanserlerinin tedavi süreci, yalnızca tıbbi değil aynı zamanda psikososyal ve çevresel faktörlerin etkin yönetimini gerektiren karmaşık bir süreçtir. Aile katılımının sınırlı olduğu veya bulunmadığı devlet korumasındaki çocuklarda bu süreç daha fazla biyopsikososyal ve yönetsel zorluk içermektedir. Bu hasta grubunda kanser tedavisine ilişkin klinik deneyim ve literatür bilgisi sınırlıdır. Bu nedenle bildirimizde, devlet korumasındaki bir çocuk hastanın tedavi süreci üzerinden Türkiye’deki yasal düzenlemeler ve hekime düşen sorumluluklar tartışılmıştır.Devlet koruması altında yaşayan on yaşında erkek çocuk; solunum sistemi semptomları ile başvurmuş ve yapılan değerlendirmeler sonucunda T-lenfoblastik lenfoma tanısı almıştır. Kemik iliği ve santral sinir sistemi tutulumu saptanmayan hastaya COG kemoterapi protokolü başlanmış ve indüksiyon tedavisi sonunda tam yanıt elde edilmiştir. Tedavi sürecinde, refakatçi sürekliliğinin sağlanamaması, aydınlatılmış onam süreçlerinin kurumsal yapı üzerinden yürütülmesi, psikososyal destek gereksinimi ve kurum-hastane koordinasyonu gibi rutin hasta izleminde sık karşılaşılmayan güçlükler izlenmiştir.Devlet korumasındaki çocuklarda kanser tedavisi; tıbbi yaklaşımın yanı sıra yasal mevzuata hâkimiyet, sosyal hizmet birimleriyle yakın iş birliği ve multidisipliner ekip çalışmasını zorunlu kılmaktadır. Bu olgu, korunma ihtiyacı olan çocuklarda kanser tedavisinin bütüncül ve hasta merkezli bir yaklaşımla ele alınması gerektiğini vurgulamakta; pediatrik onkoloji pratiğinde bu özel hasta grubuna yönelik farkındalığın artırılmasının önemine dikkat çekmektedir.

Background: Fungemia caused by non-Candida yeasts is rare but represents an emerging clinical problem that remains less well recognized and studied. These organisms often exhibit intrinsic resistance or reduced susceptibility to commonly used empirical antifungal agents, such as fluconazole and echinocandins. This poses significant challenges for empirical antifungal therapy. Objectives: To describe the clinical characteristics, antifungal treatments, and outcomes of pediatric patients with bloodstream infections due to non-Candida yeasts and to summarize the antifungal susceptibility profiles of available isolates. Methods: This retrospective study reviewed all episodes of fungemia caused by non-Candida yeasts at a tertiary pediatric center between 1 January 2020 and 1 September 2025. Results: Of the 139 yeast-related fungemia episodes identified during the study period, five (3.6%) were caused by non-Candida yeasts: three by Trichosporon spp., one by Rhodotorula mucilaginosa, and one by Magnusiomyces clavatus (formerly Saprochaete clavatus). Two cases occurred as breakthrough infections under ongoing antifungal treatment. Empirical antifungal treatments most often included amphotericin B, fluconazole, or echinocandins. The median time to species-level identification after the first positive culture result was six days (range 4–7), highlighting a considerable delay that may critically affect clinical management. Overall mortality was 40%, while attributable mortality due to non-Candida fungemia was 20%. Conclusions: Non-Candida yeasts, although infrequent, represent clinically important pathogens in pediatric fungemia due to their potential resistance to standard empirical antifungal agents. Early species-level identification and awareness of expected susceptibility patterns are essential to guide appropriate initial therapy and improve outcomes.

Awareness, Knowledge, Attitudes, and Behaviors of Pediatric Hematology and Oncology Specialists on Preserving Reproductive Health in Children with Cancer: Barriers and Suggestions - Gazi Medical Journal

Background: Keratin-positive giant cell-rich tumor (KPGCT) is a rare bone and soft tissue neoplasm, with pediatric and metastatic cases being exceedingly uncommon. Observation: We report a 1.5-month-old male infant presenting with multifocal metastatic disease involving the skull, adrenal glands, vertebrae, mandible, soft tissue, and long bones. Histopathologic evaluation confirmed KPGCT, molecular testing was negative for HMGA2 rearrangement. Treatment with imatinib resulted in marked regression of lesions and clinical improvement without toxicity. Conclusions: This case expands the clinical spectrum of pediatric KPGCT and suggests that imatinib may be an effective treatment option in infants with advanced disease, even canonical HMGA2::NCOR2 fusion absent.

Inborn errors of immunity (IEI) are caused by deficiencies or functional abnormalities in the immune system, leading to increased susceptibility to infections, autoimmunity, autoinflammatory diseases, allergies, and/or malignancies. Primary hemophagocytic lymphohistiocytosis (HLH) arises from genetic mutations affecting the function of cytotoxic T lymphocytes and natural killer (NK) cells, while secondary HLH is triggered by infections, malignancies, rheumatologic disorders, or immune deficiencies. Treatment consists of remission induction, control of triggers, maintenance of remission, rescue treatment and HSCT as approach curative steps. The aim of this study is to evaluate the clinical and laboratory features, as well as the outcomes, of primary HLH patients who were diagnosed and treated in a multidisciplinary manner over the past 25 years. From 2000 to 2025, 30 HLH cases with primary HLH/IEI who were diagnosed and treated at the departments of pediatric hematology, immunology and oncology of Ankara University School of Medicine Children Hospital and Bone Marrow Transplantation Unit were included in this study. Of the 30 patients, 18 were boys and 12 were girls. The mean age at the onset of the first symptom was 10 months (range: 0.5-204 months), while the mean age at the time of admission to our center was 12.5 months (range: 1-204 months). Pedigree analysis showed that 21 patients were born to consanguineous parents. All patients had a fever lasting longer than five days, with a mean duration of 13.30±14.05 days (range: 5-60 days). Splenomegaly was detected in 29 patients (96.6%), and hepatomegaly in 25 patients (83%). Anemia was observed in 27 patients (90%), neutropenia in 23 patients (76.6%), and thrombocytopenia in 30 patients (100%). Genetic evaluation was performed in all patients, and a causative gene was identified in 19 out of 30 cases (63%). The most common genetic diagnosis was perforin deficiency (FHLH2), detected in 8 patients (26,6%), followed by UNC13D gene defect (FHLH3) in 4 patients (13,3%). HSCT was performed in 17 patients (56,6%), with 6 receiving transplants from matched related donors (MRD), 4 from matched sibling donors (MSD), 5 from matched unrelated donors (MURD), and 2 from mismatched related donors (MMRD). 13 patients remain alive, with a mean survival of 119.89 months. 17 patients (56.6%) died, primarily from mortality included multi-organ dysfunction syndrome (MODS), acute respiratory distress syndrome (ARDS), HLH reactivation, septic shock, and heart failure. HSCT patients had a significantly longer survival (mean 165.6 months) compared to non-HSCT patients (45.36 months, p <0.01). PICU admission, organ failure, and neurological involvement were identified as adverse prognostic factors, all significantly associated with higher mortality (p < 0.05). Given the increasing recognition of HLH as a possible manifestation of IEIs, comprehensive immunological and genetic evaluations should be pursued without delay in suspected cases. Our findings, in line with national and international cohorts, confirm that HSCT remains the only curative option for familial HLH, and should be performed as early as possible after achieving disease remission. Improving access to early diagnostics and HSCT could significantly enhance outcomes, particularly in genetically predisposed populations.

Osteosarcoma (OS) bone tumor of childhood although the etiology of OS has not been fully elucidated, there is evidence linking it with factors related to pubertal development. In recent years, plasma CNP concentration and height velocity in children and CNP have been implicated as an emerging new growth marker during childhood. We aimed to determine the serum NT-proCNP levels of patients with pediatric osteosarcoma and its relation with clinical-laboratory growth parameters and look for any correlation of serum NT-proCNP levels with different prognostic factors in childhood osteosarcoma. This study enrolled 15 newly diagnosed OS patients and 31 healthy controls. All subjects were physically examined. Plasma NT-proCNP concentration was measured by enzyme-linked immunosorbent assay. Our results demonstrated that serum NT-proCNP concentration was significantly different between OS patients and control groups. At diagnosis, the OS patient’s mean blood NT-proCNP concentration was 49.7 ± 3.3 pmol/l, which was substantially lower than the control group’s concentration of 61.4 ± 3.10 pmol/l (p < 0.005). No significant correlation was found between serum NT-proCNP concentration and growth parameters. Conclusion: In conclusion, we found that the serum NT-proCNP concentration was significantly different between OS patients and control groups. It is predicted that our results will contribute to osteosarcoma biology. What is Known: • Osteosarcoma (OS) is the most common malignant bone tumor of childhood, affecting rapidly growing bones with factors associated with rapid bone growth, although its etiology is not fully understood. • The current literature suggests that the main role of the CNP/NPR-B signaling pathway is associated with endochondral bone development and related growth in long bones. What is New: • We demonstrated that serum NT-proCNP concentration was significantly different between OS patients and control groups and could be used as a potential biomarker. • To our knowledge, this is the first clinical study evaluating the relation of serum serum NT-proCNP levels with anthropometric parameters in osteosarcoma patient with a particular interest to identify the possible role of CNP in pediatric osteosarcoma and its relationship with prognostic factors.

Objective: In our study, we aimed to share the clinical experiences of our center regarding osteosarcoma cases, the most common primary malignant bone tumor in children and adolescents. Materials and Methods: With approval from the Clinical Research Ethics Committee of our center, the data of 59 pediatric patients who were followed up in our center with the diagnosis of osteosarcoma between 2007 and 2021 were evaluated retrospectively. Results: The mean time between the onset of symptoms and diagnosis was 3 months. Although not statistically significant, patients with a diagnostic delay of 3 months or less had a higher rate of recurrence and mortality. 59.3% of patients had metastatic disease, and the presence of metastases was associated with higher rates of recurrence and mortality. Significant number of patients had multiple surgical operations. Amputation as the first operation and the need for multiple surgeries were associated with higher mortality. Pathologically poor response to chemotherapy is associated with mortality. 42.4% of patients died, and the 5-year overall and disease-free survival rates were 47.5% and 30.5%, respectively. Survival rates were highest in non-metastatic and non-relapsed patients, and lowest in metastatic patients and patients with poor response to chemotherapy. Renal problems and cardiotoxicity were most frequently treatment-related complications. Conclusion: Significant improvements have been achieved in the survival and quality of life in osteosarcoma cases compared to previous years; however, there is still a long way to go, and more multicenter and multidisciplinary studies are needed on osteosarcoma. Cite this article as: Altıntaş M, Cabi Ünal E, Taçyıldız N, İncesoy Özdemir S, Dinçaslan HU. Clinical and prognostic characteristics in childhood osteosarcoma: A single-center experience in Türkiye. Turk Arch Pediatr. 2024;59(5):461-468.