Neuroblastoma 4S is a rare subtype of metastatic neuroblastoma found in children younger than 12 months, characterized by liver, skin, or bone marrow metastases. While the prognosis for patients is generally favorable, rapid progression of liver metastases can lead to life-threatening organ insufficiency. In such cases, immediate treatment with chemotherapy or radiotherapy is necessary. Given the recent decline in radiotherapy utilization, this study aims to reassess its role, evaluating its effectiveness and toxicity. We conducted a systematic review and an institutional retrospective analysis to assess the use of radiotherapy for hepatomegaly in patients with neuroblastoma 4S. The study included data from 164 patients from the literature and 16 patients from our institutional cohort. We extracted and analyzed data on short- and long-term outcomes, as well as reports of radiotherapy-induced toxicity. Our institutional data showed that 81 % of patients responded to low-dose radiotherapy administered at a median dose of 450 cGy in three fractions, resulting in liver shrinkage and symptom resolution. Based on the systematic review, 1-year survival rate was 80 %, while 5-year survival rate was 75 %. No serious toxicity was observed with the current low-dose radiotherapy; however, one case of induced secondary malignancy was reported. Radiation therapy is an effective treatment modality for hepatomegaly in patients with neuroblastoma 4S, with a success rate of about 80 %. Despite being administered to infants, a low dose of 450–600 cGy does not result in toxicity related to the kidneys, liver, or posture defects.

Frontline blinatumomab combined with chemotherapy has resulted in improved outcomes in both pediatric and adult patients with acute lymphoblastic leukemia and is considered the new cornerstone of acute lymphoblastic leukemia treatment. The role of asparaginase in this new landscape of acute lymphoblastic leukemia treatment was extensively discussed by an expert panel of hemato‐oncologists to reach a consensus on the safety and efficacy of asparaginase in diverse settings with and without blinatumomab. Herein, the following consensus topics are presented: (1) optimal dosing of asparaginase and therapeutic drug monitoring; (2) prevention of asparaginase hypersensitivity reactions and inactivation; (3) integration of frontline blinatumomab, asparaginase, and antimetabolites; and (4) special issues of asparaginase formulations and adaptations for resource‐limited settings.

Background and Clinical Significance: Acute megakaryoblastic leukemia (AMKL) is a rare and aggressive hematologic malignancy. The presence of genetic abnormalities often increases the complexity of AMKL. Among these, patients with monosomy 7 constitute a high-risk group associated with a poorer prognosis and greater chemoresistance. We report the case of a 10-year-old boy who had AMKL along with monosomy 7 and familial GATA2 deficiency. The case highlights the diagnostic and therapeutic challenges faced, as well as the critical importance of early genetic screening and timely hematopoietic stem cell transplantation (HSCT). Case Presentation: A 10-year-old boy presented with easy bruising and pancytopenia. AMKL was diagnosed with the help of a bone marrow biopsy and immunophenotyping. Genetic testing showed a GATA2 mutation and monosomy 7. Two induction cycles with daunorubicin and cytarabine were administered but failed to eliminate residual disease. The patient also developed pneumonia of a fungal origin. HSCT was delayed due to liver toxicity and elevated minimal residual disease (MRD). Azacitidine and venetoclax stabilized the disease, thereby allowing for successful haploidentical HSCT. The patient achieved complete remission with full donor chimerism. Conclusions: This case emphasizes the importance of early molecular diagnostics in pediatric AMKL. Identifying GATA2 mutations and monosomy 7 early can help guide risk stratification and the timing of HSCT. Multimodal therapy, which includes the use of infection control and targeted agents, is important for improving the outcomes in high-risk patients.

Pediatric B-cell precursor lymphoblastic lymphoma (BCP-LBL) is a rare subtype of non-Hodgkin lymphoma with a predominantly extramedullary presentation. Cutaneous and subcutaneous involvement, particularly in the head region, is a recognized manifestation; however, presentation as a solitary scalp mass mimicking a benign surgical lesion remains diagnostically challenging. A 10-year-old female patient presented to the Neurosurgery Department for a growing mass in the right frontal region of the scalp for the past 4 months. After excision, the mass was diagnosed as BCP-LBL and the patient was referred to oncologic treatment. This report aims to increase awareness among surgeons and physicians that persistent or progressive pediatric scalp lesions, even when clinically benign-appearing, may represent an underlying malignant hematologic disease requiring prompt histopathological verification and specialist referral.

Background: Lymphatic malformations (LMs) are congenital defects, resulting from abnormal venous endothelial development. Most lesions present as superficial masses located in the head and neck region. Lymphatic malformations may cause significant complications: extensive expansion on surrounding tissues, airway obstruction, respiratory insufficiency or feeding difficulties. Treatment remains challenging; standard methods (surgery, sclerotherapy) might be ineffective. Therefore, sirolimus, due to its antitumoral and antiangiogenic effect, has been introduced an important systemic treatment option, especially for widespread, complicated, unresectable, or resistant lesions in patients with lymphatic malformations. Case series presentation: This case series illustrates the clinical presentation of three neonates with head and neck congenital lymphatic malformations, who were treated with sirolimus. Information, such as prenatal data, postnatal findings (including imaging studies), children’s general condition during hospitalization, clinical course, treatment response, safety profile of the medication, and follow-up were analysed, described and compared with data found in the literature. Conclusions.: Our findings confirm that sirolimus is a potential treatment for mixed-type lymphatic malformations, with encouraging clinical response rates and manageable side effects. Further characterisation of affected patients and lymphatic malformations, including definition of therapeutic ranges and a systematic evaluation of treatment results would allow better selection of candidates, with improved overall results. Further multicentre and ultimately randomised, studies on sirolimus treatment for lymphatic malformations, would be helpful for improved care and effective treatment options for this vulnerable pediatric population.