The ganglioside GD2 is an important target in childhood cancer. Nevertheless, the only therapy targeting GD2 that is approved to date is the monoclonal antibody dinutuximab, which is used in the therapy of neuroblastoma. The relevance of GD2 as a target in other tumor entities remains to be elucidated. Here, we analyzed the expression of GD2 in different pediatric tumor entities by flow cytometry and tested two approaches for targeting GD2. H3K27M-mutant diffuse midline glioma (H3K27M-mutant DMG) samples showed the highest expression of GD2 with all cells strongly positive for the antigen. Ewing’s sarcoma (ES) samples also showed high expression, but displayed intra- and intertumor heterogeneity. Osteosarcoma had low to intermediate expression with a high percentage of GD2-negative cells. Dinutuximab beta in combination with irinotecan and temozolomide was used to treat a five-year-old girl with refractory ES. Disease control lasted over 12 months until a single partially GD2-negative intracranial metastasis was detected. In order to target GD2 in H3K27M-mutant DMG, we blocked ganglioside synthesis via eliglustat, since dinutuximab cannot cross the blood–brain barrier. Eliglustat is an inhibitor of glucosylceramide synthase, and it is used for treating children with Gaucher’s disease. Eliglustat completely inhibited the proliferation of primary H3K27M-mutant DMG cells in vitro. In summary, our data provide evidence that dinutuximab might be effective in tumors with high GD2 expression. Moreover, disrupting the ganglioside metabolism in H3K27M-mutant DMG could open up a new therapeutic option for this highly fatal cancer.

Nasopharyngeal carcinoma (NPC) in children and young adults has been treated within two consecutive prospective trials in Germany, the NPC-91 and the NPC-2003 study of the German Society of Pediatric Oncology and Hematology (GPOH). In these studies, multimodal treatment with induction chemotherapy, followed by radio (chemo)therapy and interferon-beta maintenance, yielded promising survival rates even after adapting total radiation doses to tumor response. The outcome of 45 patients in the NPC-2003 study was reassessed after a median follow-up of 85 months. In addition, we analyzed 21 further patients after closure of the NPC-2003 study, recruited between 2011 and 2017, and treated as per the NPC-2003 study protocol. The EFS and OS of 66 patients with locoregionally advanced NPC were 93.6% and 96.7%, respectively, after a median follow-up of 73 months. Seven patients with CR after induction therapy received a reduced radiation dose of 54 Gy; none relapsed. In young patients with advanced locoregional NPC, excellent long-term survival rates can be achieved by multimodal treatment, including interferon-beta. Radiation doses may be reduced in patients with complete remission after induction chemotherapy and may limit radiogenic late effects.

Zusammenfassung: Das akute Skrotum gehört zu den häufigsten urologischen Notfällen bei Kindern und Jugendlichen und bezeichnet das plötzliche Auftreten von Schmerzen im Skrotum, meist einhergehend mit begleitender Schwellung und Rötung des betroffenen Hodens. Am wichtigsten ist es zunächst festzustellen, ob ein chirurgisches Vorgehen mit Hodenfreilegung notwendig ist, oder ein konservatives Vorgehen gerechtfertigt ist. Da Anamnese und körperliche Untersuchung nicht immer eindeutig sind, kommt ergänzend oft die Sonografie mit farbkodierter Dopplersonografie als Bildgebung der 1. Wahl zum Einsatz. Hiermit gelingt es meist, eine OP-Pflichtige Hodentorsion oder auch eine Hodenruptur nach Trauma von ihren Differenzialdiagnosen, wie z. B. die Hydatidentorsion oder die Epididymorchitis zu unterscheiden. Weiterführende bildgebende Verfahren wie MRT, Szintigrafie oder kontrastmittelverstärkte Sonografie sind nur bei unklaren Fällen oder Komplikationen im Verlauf indiziert.

Die Kinderradiologie steht heute vor weitreichenden Veränderungen. Basismodalitäten wie Röntgen und Ultraschall werden zunehmend durch neuere, moderne Techniken ergänzt. Dieser Übersichtsartikel stellt Fortschritte in der Kinderradiologie sowie technische Innovationen vor, welche in Zukunft noch größere Bedeutung erlangen könnten. Hierzu werden CT-Dosisreduktionstechniken inklusive der Anwendung künstlicher Intelligenz sowie Fortschritte in den Gebieten der Magnetresonanztomografie und molekularen Bildgebung dargestellt. Kernaussagen Significant changes can be expected in modern pediatric radiology. New imaging techniques are progressively added to basic modalities like Xrays and ultrasound. This essay summarizes recent advances and technical innovations in pediatric radiology, which are supposed to gain further importance in the future. Thus, CT dose reduction techniques including artificial intelligence as well as advances in the fields of magnetic resonance and molecular imaging are presented. Key Points Citation Format

Background/Objectives: There is an increasing need for cross-sectional imaging in pediatric patients with congenital heart disease. This patient group is highly sensitive to ionizing radiation. The purpose of this article was to evaluate differences in radiation dose and image quality between a first-generation photon-counting (PC)-CT system and a 256-slice single-source energy-integrating detector (EID)-CT in these patients. Methods: We retrospectively assessed effective dose, CT dose index-volume (CTDI_vol), dose length product (DLP), and image quality of all prospectively electrocardiography-gated CTs of the thorax in all patients < 18 years of age examined between February 2021 and August 2024 (n = 43). Two independent observers subjectively scored image quality, vascular contrast, and noise on a 5-point Likert scale. In addition, we assessed the signal-to-noise-ratio (SNR) and contrast-to-noise-ratio (CNR) quantitatively. Results: All dose parameters were significantly lower in the PC-CT (n = 27) versus the EID-CT (n = 16) group (mean effective dose: 0.8 ± 0.64 versus 2.2 ± 0.88 mSv, p < 0.001; mean CTDI_vol: 1.22 ± 0.96 versus 4.8 ± 1.08 mGy, p < 0.001; mean DLP: 30.7 ± 31.9 versus 73.7 ± 50.7 mGy*cm, p < 0.001). Overall subjective image quality and contrast were rated higher in the PC-CT group (p = 0.046 and < 0.001, respectively). Quantitative CNR was significantly higher in the PC-CT group (mean 39.1 ± 12.9 versus 26.2 ± 10.8, p = 0.002). Conclusions: PC-CT enables high-quality examinations for the evaluation of congenital heart disease with a highly significant dose reduction compared with a 256-slice single-source EID-CT.

Objectives: To evaluate correlations between cardiac magnetic resonance imaging (cMRI) at rest including strain imaging and variables derived from quantitative cardiopulmonary exercise testing using a treadmill in patients with pectus excavatum. Methods: We retrospectively correlated the results of cMRI and cardiopulmonary exercise testing in 17 patients with pectus excavatum, in whom both examinations were performed during their pre-operative clinical evaluation. In addition to cardiac volumetry, we assessed the strain rates of both ventricles using a feature-tracking algorithm of a piece of commercially available post-processing software. Results: Right ventricular (RV) ejection fraction correlated negatively with heart rate at anaerobic threshold (rho = −0.543, p = 0.024). A positive correlation between radial strain rate at the RV base and percentage of predicted maximum heart rate (rho = 0.72, p = 0.001) was shown, with equivalent results for circumferential strain rate (rho = −0.64, p = 0.005). Radial strain rate at the RV base correlated in a strongly negative way with maximum oxygen uptake (rho = −0.8, p < 0.001), with a correspondingly positive correlation for circumferential strain rate (rho = 0.73, p = 0.001). Conclusions: Quantitative parameters derived from cMRI at rest, especially those acquired at the most severely compressed RV base, correlated with cardiopulmonary exercise testing variables. The compression of the RV base by the sternum might be partially compensated by an increased strain rate to induce higher heart frequencies during exercise. However, high strain rates were associated with a higher disease severity and a lower maximum oxygen uptake, indicating a limitation of this compensation mechanism.