Liposomal amphotericin B prophylaxis of invasive mold infections in children post allogeneic stem cell transplantation.

Background: Invasive mold infections (IMI) are a leading cause of infectious mortality in allogeneic stem cell transplant (AlloSCT) recipients. Fluconazole, the current standard for fungal prophylaxis, is ineffective against molds. We initiated a pilot study to determine the safety and activity of prophylactic liposomal amphotericin B (AMB) in preventing IMI in pediatric and adolescent AlloSCT recipients during the first 100 days.

Procedure: Fifty-one patients (57 AlloSCT) were given AMB (3 mg/kg/day) intravenously, day 0-100. Median age 6 years, 32 males, 19 females. Donors: 33 unrelated and 2 related cord blood, 13 related and 1 unrelated peripheral blood stem cell and 8 related bone marrow (BM); 30 received myeloablative and 27 reduced intensity conditioning. Graft-versus-host disease (GVHD) prophylaxis comprised tacrolimus and mycophenolate mofetil.

Results: Median follow-up is 557 days. AMB was generally well tolerated. The probability of developing >/=grade II acute GVHD and extensive chronic GVHD was 45% and 7%, respectively. Estimated 1-year OS is 62.4% for all patients with 78.8% and 26.7% for average-risk and poor-risk, respectively. The incidence of IMI was 0%.

Conclusions: These results suggest prophylactic AMB is tolerable and may prevent IMI, especially Aspergillus, during the first 100 days post AlloSCT in pediatric and adolescent patients. A randomized study is needed to determine the efficacy of this approach.