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Updated: Aug 23, 2026

Pre-clinical Evaluation of Tyrosine Kinase Inhibitors for Treatment of Acute Leukemia
Published on: September 18, 2013
Prospective randomized comparison of two prophylactic regimens with trimethoprim-sulfamethoxazole in leukemic
M R Rossi1, P Banfi, M Cappuccilli
1Centro di Ematologia Pediatrica, Università di Milano, Ospedale di Monza, Italy.
Insights
Continuous versus intermittent trimethoprim-sulfamethoxazole prophylaxis for children with acute hematologic malignancy showed similar efficacy and toxicity. The choice between continuous and intermittent regimens should consider cost and patient preference for optimal management of interstitial pneumonia.
Area of Science:
- Pediatric Oncology
- Infectious Disease Prevention
- Pharmacology
Background:
- Children with acute hematologic malignancy are at high risk for opportunistic infections like interstitial pneumonia.
- Prophylaxis with trimethoprim-sulfamethoxazole is commonly used to prevent these infections.
Purpose of the Study:
- To compare the efficacy and toxicity of continuous versus intermittent trimethoprim-sulfamethoxazole prophylaxis in children with acute hematologic malignancy.
- To determine if an intermittent regimen is as effective and less toxic than a continuous regimen.
Main Methods:
- A randomized trial was conducted between July 1984 and June 1986.
- Children received either continuous or intermittent (3 days/week) trimethoprim-sulfamethoxazole prophylaxis.
- Outcomes assessed included severe infections, side-effects, and compliance.
Main Results:
- The incidence of severe infections was similar between the continuous (17) and intermittent (21) groups.
- The number of reported side-effects was also comparable (continuous: 30; intermittent: 34).
- Patient compliance was similar across both treatment arms.
Conclusions:
- Neither continuous nor intermittent trimethoprim-sulfamethoxazole prophylaxis demonstrated superior efficacy or safety in this pediatric population.
- The selection of prophylaxis regimen should be guided by cost-effectiveness and individual patient/parental preferences to ensure optimal compliance.
Abstract:
Between 1 July 1984 and 30 June 1986 all children treated for acute hematologic malignancy at our center were randomized to receive continuous (group A) or intermittent (3 days/week, group B) prophylaxis with trimethoprim-sulfamethoxazole (5-25 mg/kg/day/p.o.) against interstitial pneumonia with the aim of investigating if an intermittent regimen is as effective as and less toxic than a continuous regimen. The number of severe infections (group A, 17; group B, 21) and side-effects (group A, 30; group B, 34) was similar in the two groups, and compliance was also similar. We conclude therefore that neither regimen offers advantages over the other and the decision which to use should be based on cost (where regimen B has the advantage) and the children's and parents' preferences and compliance.
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