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[Clinical use of rubomycin in neuroblastoma in children]
Insights
Rubomycin treatment showed a 68% objective response rate in children with neuroblastoma, with 41% achieving significant tumor reduction. Despite side effects like leucopenia, some patients experienced long-term remission, highlighting its potential in neuroblastoma therapy.
Area of Science:
- Pediatric Oncology
- Medical Pharmacology
Background:
- Neuroblastoma is a significant pediatric malignancy.
- Effective treatment strategies for relapsed or metastatic neuroblastoma are crucial.
Purpose of the Study:
- To evaluate the efficacy and safety of rubomycin in treating children with neuroblastoma.
- To assess the long-term outcomes of rubomycin therapy in this patient population.
Main Methods:
- Retrospective analysis of 23 children (7 months to 11 years) with neuroblastoma.
- Intravenous administration of rubomycin (0.7–1.5 mg/kg) with variable dosing schedules.
- Concurrent roentgen therapy in four patients.
Main Results:
- An objective treatment effect was observed in 68% of patients, with a pronounced effect (grades 3 and 2) in 41%.
- Common adverse events included leucopenia (87%), thrombocytopenia, and nausea/vomiting.
- Disease progression led to death in 18 patients within 4 months to 2 years, while 4 children remained disease-free for 3–6 years.
Conclusions:
- Rubomycin demonstrates significant objective efficacy in treating pediatric neuroblastoma, including relapsed and metastatic cases.
- While associated with notable toxicity, particularly leucopenia, rubomycin offers a potential for long-term remission in a subset of patients.
- Further research into optimizing rubomycin dosing and managing side effects is warranted for improved neuroblastoma treatment outcomes.
Abstract:
The results of treatment of 23 children at the age of 7 months to 11 years suffering from neuroblastoma are presented; 22 patients with tumors, relapses or metastases were subjected to the treatment and 1 child was treated prophylactically after radical operation. Four patients were subjected to roentgen therapy in addition to the treatment with rubomycin. The antibiotic was administered intravenously in doses of 0.7--1.5 mg/kg in 1--3 days or daily. The caurse dose (3--12 mg/kg) was determined by the treatment efficiency and the side reactions. The objective effect was observed in 68 per cent of the patients, including the pronounced objective effect (marks 3 and 2) in 41 per cent of the cases. Leucopenia (less than 4000 cells in 1 mm3 of the blood). thrombocytopenia, vomiting (or nousea) and changes in the ECG were registered in 20 (87 per cent), 4,9 and 2 patients respectively. When the results of the treatment were positive, repeated courses of the therapy within 1.5--2 years were carried out; 18 patients died within 4 months to 2 years after the first course of the treatment with rubomycin because of the disease development. No signs of the disease were observed in 4 children with in 3--6 years of observation.