High-grade B-cell lymphoma with 11q-aberration (HGBCL-11q) is a rare pediatric non-Hodgkin lymphoma. This study assessed outcome in 90 children with HGBCL-11q. With survival rates ≥95%, patients with HGBCL-11q and no predisposition are candidates for deescalated therapy in future prospective trials.
Very-early-onset inflammatory bowel disease (VEO-IBD), representing cases diagnosed before age 6 years, is increasing in prevalence. Although VEO-IBD often presents as severe, treatment-resistant disease requiring biologic agents, studies showing the effectiveness of biologics, such as ustekinumab (UST) and vedolizumab (VDZ), remain limited. We retrospectively analyzed patients with VEO-IBD treated for at least a year from 13 institutions in Japan, evaluating clinical course including effectiveness of biologics, such as infliximab (IFX), adalimumab (ADL), UST, and VDZ. Patients with monogenic IBD were excluded. Steroid-free clinical remission (SFCR) and treatment persistence were assessed separately for first-line and for second-line or subsequent biologic therapies. We studied 101 VEO-IBD patients (56
Background/Objectives: Although Tumor-Treating Fields (TTFields) therapy is an established treatment modality for adult glioblastoma, clinical data on its efficacy in pediatric brain tumors are extremely scarce. The present study aimed to evaluate the safety of TTFields therapy for pediatric diffuse high-grade glioma (HGG) and to conduct an exploratory analysis of its efficacy. Methods: A prespecified, interim analysis was performed to determine whether the study should be continued on the basis of safety and feasibility data on the first three patients. The target population was children aged 5 to 17 years with newly diagnosed, supratentorial HGG or its first recurrence following frontline therapy. After completion of initial, local treatment for the tumor (surgical removal and/or radiotherapy), all patients received TTFields therapy using OptuneTM for 28 days per course for up to 26 courses until disease progression. Results: The interim analysis, which was completed in October 2022, included three female patients aged 14, 17, and 9 years. All had a histological grade 4 tumor, two of which were radiation-induced, secondary HGG. No serious, treatment-related toxicities or device-related issues were observed. All three patients were able to continue using the device for 75% or more of the time in accordance with the protocol, suggesting that the treatment was feasible. The MRI findings of two patients indicated that the treatment has a potential antitumor effect. Based on these results, the study was resumed and is currently being continued at multiple centers. Conclusions: The initial results of the prespecified, interim analysis demonstrated that TTFields therapy was safe and feasible for children with HGG. This study was funded by the Japan Agency for Medical Research and Development (AMED) and was registered with the Japan Registry of Clinical Trials (jRCTs032200423).
OBJECTIVE:Developmental arrest or regression that results in intellectual disability (ID) is a common neurological sequela of infantile epileptic spasms syndrome. Spindles are the hallmark of non-rapid eye movement sleep, and their density correlates with cognitive performance. Recent evidence suggests that ictal and interictal epileptic activities hijack the thalamocortical network. Therefore, we aimed to investigate whether thalamocortical dysfunction, as represented by reduced spindle density, is related to cognitive outcomes. METHODS:We retrospectively recruited patients diagnosed with infantile epileptic spasms syndrome who were treated at Saitama Children's Hospital. Specifically, we included patients with an unknown etiology to minimize the effects of preceding thalamocortical dysfunction induced by various etiologies before the onset of infantile epileptic spasms syndrome. These cohorts were separated into two groups based on the presence or absence of ID, defined as an intelligence quotient (IQ) or developmental quotient (DQ) of <70, during follow-up. Information concerning patients' background characteristics, treatment response (defined as seizure cessation for at least 3 months), and cognitive performance was extracted. Pre- and posttreatment spindle features were derived from quantitative assessment of spindle features performed using automated algorithms, which were validated against expert human annotations. Additionally, logistic regression was used to validate the independent predictors of cognitive outcomes. RESULTS:Overall, 45 patients were included (23 and 22 patients in the ID and non-ID groups, respectively). Posttreatment spindle density was significantly higher in the non-ID group than in the ID group. Moreover, spindle density was positively correlated with DQ/IQ values across the entire cohort. Finally, a logistic regression model revealed that spindle density and treatment response were independent predictors of cognitive outcomes. SIGNIFICANCE:Reduced posttreatment spindle density was correlated with cognitive outcome in infantile epileptic spasms syndrome. The thalamocortical network may be a potential treatment target to achieve favorable cognitive outcomes.
