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Successful Pneumocystis carinii pneumonia prophylaxis using aerosolized pentamidine in children with acute leukemia
J Weinthal1, J D Frost, G Briones
1Division of Hematology/Oncology, Children's Hospital of Orange County, CA 92668.
Insights
Aerosolized pentamidine (AP) effectively prevents Pneumocystis pneumonia (PCP) in children with leukemia who cannot tolerate trimethoprim-sulfamethoxazole (TMP/SMX). This study shows AP is a well-tolerated alternative for PCP prophylaxis in pediatric leukemia patients.
Area of Science:
- Pediatric Oncology
- Infectious Disease Prophylaxis
- Hematology
Background:
- Pneumocystis pneumonia (PCP) is a significant risk for immunocompromised children, particularly those with acute leukemia.
- Trimethoprim-sulfamethoxazole (TMP/SMX) is a standard prophylaxis, but intolerance is common.
- Alternative prophylactic agents are needed for pediatric leukemia patients unable to tolerate TMP/SMX.
Purpose of the Study:
- To evaluate the safety and efficacy of aerosolized pentamidine (AP) as an alternative PCP prophylaxis.
- To assess AP's tolerability in children with acute leukemia who previously could not tolerate TMP/SMX.
Main Methods:
- A cohort of 22 children (ages 1-15) with acute leukemia received monthly AP (150-300 mg) for PCP prophylaxis.
- AP was administered following intolerance or prolonged neutropenia on TMP/SMX.
- The study spanned a 3-year period, monitoring tolerance and efficacy.
Main Results:
- Aerosolized pentamidine was well-tolerated in 22 pediatric leukemia patients over 358 treatment courses.
- AP demonstrated effectiveness in preventing PCP during the study period.
- Minimal side effects were observed, and 86% of acute lymphoblastic leukemia patients resumed full-dose chemotherapy.
Conclusions:
- Aerosolized pentamidine is a safe and effective second-line agent for PCP prophylaxis in children with acute leukemia.
- AP should be considered when TMP/SMX is not tolerated.
- Further Phase III trials are recommended to assess AP's impact on dose intensification and survival outcomes.
Purpose:
We report our experience replacing trimethoprim-sulfamethoxazole (TMP/SMX) with aerosolized pentamidine (AP) in 22 children with acute leukemia who could not tolerate TMP/SMX.
Patients And Methods:
Children (age 1 to 15 years) with acute leukemia during maintenance chemotherapy or post-bone marrow transplantation (BMT) receiving prophylaxis for Pneumocystis carinii pneumonia (PCP) with TMP/SMX received AP following prolonged neutropenia or allergy to TMP/SMX. Patients received 300 mg of AP monthly (children < 4 years received 150 mg) dissolved in 5 mL of distilled water over 20 to 30 minutes.
Results:
Over a 3-year period, 358 courses of AP were administered over 10,124 observable days. AP was adequately tolerated on a monthly basis for prophylaxis against PCP in 22 children with acute leukemia. AP was demonstrated to be effective in preventing PCP. There were minimal side effects observed during this trial. The majority of children with acute lymphoblastic leukemia (ALL; 12 of 14 [86%]) undergoing maintenance chemotherapy were able to resume full-dose therapy.
Conclusion:
AP in children is well tolerated and shows high efficacy for PCP prophylaxis in children with leukemia. We conclude that AP should be considered as second-line PCP prophylactic therapy for children with acute leukemia in instances in which TMP/SMX cannot be tolerated. Phase III trials are required to determine its effect on dose intensification and event-free survival.