Post-Transplant Cyclophosphamide after Matched Sibling and Unrelated Donor Hematopoietic Stem Cell Transplantation in Pediatric Patients with Acute Myeloid Leukemia

Abstract

Non-relapse mortality due to GVHD and infections represents a major source of morbidity and mortality in pediatric HSCT recipients. Post-transplant cyclophosphamide (PTCy) has emerged as an effective and safe GVHD prophylaxis strategy, with improved GVHD and relapse-free survival in matched (related and unrelated) and mismatched haploidentical HSCT adult recipients. However, there are no published data in pediatric patients with acute myeloid leukemia who received matched-donor HSCT with PTCy. We demonstrate, in this case series, that the use of PTCy in this population is potentially safe, effective in preventing acute GVHD, does not impair engraftment, is associated with reduced non-relapse mortality, and does not hinder immune reconstitution post HSCT.

Keywords: immune reconstitution; matched-donor transplant; pediatric acute myeloid leukemia; post-transplant cyclophosphamide.

Figure 1

Computed tomography (CT) chest imaging of an 11-year-old patient with relapsed AML is shown. CT chest imaging with bilateral ground-glass opacities and pulmonary nodules (seen inside the black circle) consistent with pulmonary toxoplasmosis. The CT chest was performed 3 months prior to HSCT.

Figure 2

Computed tomography (CT) chest imaging of a 6-year-old Hispanic patient with relapsed AML. (A) Pre-HSCT CT chest indicates a 0.8 × 0.6 cm right upper lobe pulmonary nodule (arrow), a 0.6 cm right lower lobe lung nodule, and 0.4 cm left upper lobe pulmonary nodule. (B) Post-HSCT CT chest indicates resolving post-infectious nodules with no evidence of active infection.