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Purpose: Our study aimed to explore real-world treatment scenarios for children and adolescents with neurotrophic tropomyosin receptor kinase (NTRK)-fused tumors, emphasizing access, responses, side effects, and outcomes., Patients and Methods: Pooled clinical data from 17 pediatric cases (11 soft-tissue sarcomas, five brain tumors, and one neuroblastoma) treated with larotrectinib and radiologic images for 14 patients were centrally reviewed. Testing for gene fusions was prompted by poor response to treatment, tumor progression, or aggressiveness., Results: Six different NTRK fusion subtypes were detected, and various payment sources for testing and medication were reported. Radiologic review revealed objective tumor responses (OR) in 11 of 14 patients: Complete responses: two; partial responses: nine; and stable disease: three cases. Grades 1 or 2 Common Terminology Criteria for Adverse Events adverse effects were reported in five patients. Regarding the entire cohort's clinical information, 15 of 17 patients remain alive (median observation time: 25 months): four with no evidence of disease and 11 alive with disease (10 without progression). One patient developed resistance to the NTRK inhibitor and died from disease progression while another patient died due to an unrelated cause., Conclusion: This real-world study confirms favorable agnostic tumor OR rates to larotrectinib in children with NTRK-fused tumors. Better coordination to facilitate access to medication remains a challenge, particularly in middle-income countries like Brazil.

Tumors of the kidney, bladder, prostate, testis, and adrenal represent a large part of the adult urologic practice, but are relatively infrequent in children. The natural history and management of these tumors in the pediatric age is different from that of the adults. As result of the successful work of several clinical trial groups in recent decades, there has been a significant improvement in their cure rates. The aim of this article is to review their most significant clinical aspects, as well as to present an update in their management.

Unlabelled: Over the last 20 years, after combining treatment of chemotherapy and radiotherapy, there has been an improvement in the survival rate of acute lymphoblastic leukemia patients, with a current cure rate of around 70%. Children with the disease have been enrolled into international treatment protocols designed to improve survival and minimize the serious irreversible late effects. Our oncology unit uses the international protocol: GBTLI LLA-85 and 90, with the drugs methotrexate, cytosine, arabinoside, dexamethasone, and radiotherapy. However, these treatments can cause gonadal damage and growth impairment., Patients and Method: The authors analyzed 20 children off therapy in order to determine the role of the various doses of radiotherapy regarding endocrinological alterations. They were divided into 3 groups according to central nervous system prophylaxis: Group A underwent chemotherapy, group B underwent chemotherapy plus radiotherapy (18 Gy), and group C underwent chemotherapy plus radiotherapy (24 Gy). Serum concentrations of LH, FSH, GH, and testosterone were determined. Imaging studies included bone age, pelvic ultrasound and scrotum, and skull magnetic resonance imaging., Results: Nine of the patients who received radiotherapy had decreased pituitary volume. There was a significant difference in the response to GH and loss of predicted final stature (Bayley-Pinneau) between the 2 irradiated groups and the group that was not irradiated, but there was no difference regarding the radiation doses used (18 or 24 Gy). The final predicted height (Bayley-Pinneau) was significantly less (P = 0.0071) in both groups treated with radiotherapy. Two girls had precocious puberty, and 1 boy with delayed puberty presented calcification of the epididymis., Conclusion: Radiotherapy was been responsible for late side effects, especially related to growth and puberty.

Objective: To evaluate quality of life in children and adolescents with acute lymphocytic leukemia (ALL) and juvenile rheumatoid arthritis (JRA)., Material and Methods: We administered the Children's Global Assessment Scale (CGAS), the Vineland Adaptative Behavior Scale (VABS) and the Autoquestionnaire qualité de vie enfant imagé (AUQEI) to a sample of 28 children with ALL, 28 children with JRA, and 28 healthy controls, aged 4 to 13 years old, who were diagnosed between 1 and 5 years previously., Results: Slight differences were found in age between patients with ALL and those with JRA. No significant differences were found in time since diagnosis or in CGAS scores. A significant difference was found in VABS global scores, as well as in VABS communication domain scores. No significant differences were found in VABS daily living skills domain scores between patients with ARJ and healthy controls. No significant differences were found among the groups in VABS socialization domain scores or in AUQEI scores., Conclusion: In our study, chronically ill children clearly performed worse in adaptative behavior development. Nevertheless, their quality of life was similar to that of healthy controls. Appropriate methods to identify pediatric patients' perception of their illnesses and treatment should be urgently developed.

Purpose: The efficacy of radiation therapy (RT) combined with tamoxifen (TX) was tested in patients diagnosed with diffuse brainstem gliomas in a multicenter trial., Patients and Methods: TX was administered orally (maintenance dose: 200 mg/m(2) per day) along with conventional local RT and then continued for 52 additional weeks. Survival, tumoral radiologic response, and toxicity were evaluated. Compliance was assessed using pharmacokinetic measurements., Results: Of 29 patients, 27 completed RT (median dose, 54 Gy). Of 22 assessable patients, 11 (50%) had an objective radiologic response. The mean TX steady-state serum level was 2.44 micromol/L +/- 1.02 micromol/L. Only three patients completed the entire course of treatment without tumoral progression or significant toxicity. Common side effects included nausea and vomiting. Hepatotoxicity (five patients), neurotoxicity (two patients), venous thrombosis (one patient), bilateral ovarian cysts (two patients), and transient neutropenia (one patient) were also observed. Median survival was 10.3 months. Only four patients remain alive without tumoral progression. The 1-year survival rate (mean +/- SD) was 37.0% +/- 9.5%., Conclusion: This treatment combination produced no significant change in the overall poor prognosis of these patients. Most tumors responded initially to treatment but recurred as the study progressed. A minority of patients seemed to benefit from the extended use of TX. Generally, treatment was well tolerated, with good patient compliance, but we recommend continuous close monitoring for side effects. Based on our poor results, we recommend that alternative treatments be tested in patients with this type of tumor.

Symptomatic involvement of the gastrointestinal (GI) tract as a prominent symptom in Langerhans' cell histiocytosis (LCH) is uncommon, occurring in less than 1 to 5% of all cases, even when the disease is in its disseminated form. Up to now, there have been reports of 18 cases of LCH with GI manifestations, including our 2 cases, with diarrhea (77.7%), protein-losing enteropathy (33.3%) and bloody stool being the most frequent findings. The authors present two patients with severe diarrhea and refractory hypoalbuminemia, and with the protein-losing enteropathy documented by Cr51-labeled albumin studies. A review of the literature indicated that the presence of GI symptoms is often associated with systemic disease as well as with poor prognosis, mainly under 2 years of age. Radioisotopes are useful for documenting protein loss in several diseases with high specificity and sensitivity, and their utilization in the cases reviewed here permitted diagnoses in 6 children, as well as improved therapeutic management.

Two cases of Aspergillosis in immunocompromised children are reported. Both were caused by Aspergillus flavus. Early diagnosis and treatment led to the remission of the process. One patient had acute myeloid leukemia; the fungus was isolated from the blood. The other patient with bone marrow aplasia, presented an invasive aspergillosis of the paranasal sinuses with dissemination of fungal infection; the diagnosis was obtained by histology and culture of biopsied tissue from a palatal ulceration.

From July 1985 to February 1987, of 46 consecutive children with cancer (26 male, 20 female; median age, 4 years) with no prior history of chickenpox, the initial 30 patients were randomized either to receive or not to receive live attenuated varicella vaccine (LAVV) before chemotherapy was started and the remaining 16 patients were all immunized without randomization. Before immunization, Varicella zoster (VZ) antibodies were detected by immunofluorescence and ELISA in 11 (34%) of 32 vaccinated children and two (14%) of 14 controls, indicating previous infection. A booster effect was evident in 70% of them and no side effects were noted. Ten (28%) of 32 vaccinees were excluded from the analysis because of early death due to cancer (1-4 weeks). Seroconversion was demonstrated in ten (77%) of 13 vaccinees, with high antibody titres. Only three of them lost their antibodies 2 years after immunization, as disclosed by serological follow-up. Eight out of 13 vaccinees had household contacts with VZ and none became infected. Zoster immunoglobulin (ZIG) was never given. Among controls, seven out of 14 were exposed to VZ and four (57%) became infected. Mild side effects were observed in four (12.5%) out of 32 vaccinees (three with papulovesicular rash, 6-30 lesions, and one with a 3-day intermittent fever). Local reactions, zoster and spreading of vaccinal virus did not occur. LAVV proved to be safe and effective when administered before starting chemotherapy to children with cancer and no history of varicella.