A Survey of Provider Practices for Diagnosis and Treatment of Pediatric Moderate Aplastic Anemia From the North American Pediatric Aplastic Anemia Consortium.

Pediatric moderate aplastic anemia (MAA) lacks defined diagnostic criteria and a clear standard of care due to limited understanding of its pathophysiology and natural history. To understand current diagnostic and management practices for pediatric patients with MAA, a survey of the North American Pediatric Aplastic Anemia Consortium (NAPAAC) was conducted among 104 providers across 57 institutions. The survey results show that the approach to MAA remains inconsistent. The survey demonstrates broad variability regarding the working definition, diagnostic work-up, and therapeutic management of children with MAA. The diagnostic work-up and treatment options for children with MAA are largely driven by management guidelines for pediatric severe aplastic anemia (SAA). Treatment triggers and preferred therapy types varied widely among respondents. Curated next-generation sequencing panels and whole exome/whole genome sequencing were included by only 55% and 9% of respondents, respectively, suggesting the need to more broadly consider inherited bone marrow failure syndromes in the differential diagnosis of these patients. Based on the most commonly reported practices across NAPAAC institutions, we have included a proposed diagnostic and management algorithm in this manuscript. Effective, risk-adapted treatment for children with MAA requires a better understanding of the biology, natural history, and treatment outcomes in this heterogeneous population.

Duke Scholars

Author Jennifer Ann Rothman Pediatrics, Hematology-Oncology