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Intravenous Pentamidine Is Safe and Effective as Primary Pneumocystis Pneumonia Prophylaxis in Children and Adolescents Undergoing Hematopoietic Stem Cell Transplantation

DeMasi, James M. RN, CPNP*; Cox, Jennifer A. MD; Leonard, David PhD; Koh, Andrew Y. MD*‡; Aquino, Victor M. MD*‡

The Pediatric Infectious Disease Journal: September 2013 - Volume 32 - Issue 9 - p 933–936
doi: 10.1097/INF.0b013e318292f560
Original Studies

Background: Pneumocystis carinii pneumonia (PCP) is a potentially life-threatening but preventable infection that may occur after hematopoietic stem cell transplantation (HSCT). Intravenous pentamidine has been used in the prevention of PCP in the post-transplant period, although there are few trials published in the literature evaluating its safety and efficacy.

Methods: We retrospectively reviewed the medical records of children who underwent HSCT from January 1, 2005, to October 1, 2011, who received intravenous pentamidine as first-line PCP prophylaxis initiated at admission. Demographic, clinical, microbiologic, management and outcome data were collected.

Results: One hundred sixty-seven consecutive HSCTs in 137 pediatric patients were given intravenous pentamidine before myeloablation and then every 28 days until the subject was at least a minimum 30 days post-HSCT, had stable neutrophil engraftment (absolute neutrophil count >1000/mm2 for 3 days without growth factor support) and for allogeneic patients, no evidence of active graft versus host disease and weaning on their immunosuppressive therapy. No cases of PCP were seen in this cohort. Ten (7%) had a grade I side effect of nausea/vomiting requiring slower infusion time and 2 (2%) had a grade IV reaction with anaphylaxis (rash) and hypotension with 1 child requiring transfer to the intensive care unit.

Conclusions: Intravenous pentamidine was safe and effective for the prevention of PCP in pediatric HSCT patients. Given the potential neutropenic effects of trimethoprim-sulfamethoxazole, compliance with drug administration and inferior efficacy of other PCP prophylactic medications, intravenous pentamidine should be considered as first-line therapy for the prevention of PCP in children undergoing HSCT.

From the *Children’s Medical Center Dallas, Dallas, TX; Huntsville Hospital for Women and Children, Huntsville, AL; and University of Texas Southwestern Medical Center, Dallas, TX.

Accepted for publication March 19, 2013.

This work was supported by the Children’s Cancer Fund of Dallas; Wipe Out Kids Cancer and Children’s Medical Center of Dallas Foundation. The authors have no other funding or conflicts of interest to disclose.

Address for correspondence: Victor M. Aquino, MD, Associate Professor of Pediatrics, University of Texas Southwestern Medical Center Dallas, 5323 Harry Hines Blvd, Dallas, TX 75290-9063. E-mail:

© 2013 by Lippincott Williams & Wilkins, Inc.