Studies Find High Prevalence, Cost of Neutropenia in Pediatric Patients With Autoimmune Disease

David Bai, PharmD

Pediatric patients with autoimmune neutropenia have a higher prevalence and cost than previously suspected, according to the results of 2 studies presented at the 60th American Society of Hematology Annual Meeting & Exposition, held December 1-4 in San Diego, California.

The prevalence, cost, and clinical impact of neutropenia in pediatric patients with autoimmune disease is relatively unknown but vital for hematologists in managing this population. Although autoimmune neutropenia usually follows a benign clinical course, it is still important for hematologists to accurately assess their patients, particularly if there are alarming signs or symptoms present.

The characteristics and frequency of neutropenia in pediatric patients with autoimmune disease were described in a study of 1145 pediatric patients with primary immunodeficiency. Among these patients, 15.8% of them had neutropenia, with the smallest numbers in patients with combined immunodeficiencies with associated or syndromic features (12.01%) and the highest in patients with defects in intrinsic and innate immunity (66.67%).1

For patients who had neutropenia, most of them were classified as mild (46.4%). Moderate and severe neutropenia were present in 33.1% and 20.4% of the patients, respectively. The prevalence of neutropenia between genders was not significantly different (P = .64); however, females were more likely to have severe neutropenia than males (28% vs 16%; P = .03). Neutropenia also did not affect morbidity or mortality. Although there were minimal differences in morbidity and mortality between neutropenic and non-neutropenic patients, its limited number of complete blood count data sets did not allow investigators to make any confirmative conclusions.1

The costs of neutropenia in pediatric patients with autoimmune disease is addressed in a study of 46 patients diagnosed with autoimmune neutropenia, of which 36 (78%) had a hospital encounter for evaluation of fever, totaling 125 encounters in a 3-year period. With an estimated cost of $3771 per visit, total charges amounted to $471,375. Twenty-nine patients with severe neutropenia and isolated fever were also admitted as inpatients for observation, which could further increase total costs.2 

From these studies, investigators uncovered 3 major findings. First, the prevalence of neutropenia in patients with immunodeficiency disease a lot higher than expected. Second, from these 2 studies, there were no evidence that neutropenia affected morbidity and mortality. Third, even though autoimmune neutropenia had minimal impact on the clinical wellness of the patient, costs were high.

Based on these results, investigators concluded that future research will be needed to definitively determine the impact of neutropenia as well as to create cost-effective strategies to reduce costs while maintaining patient safety.

  1. Michniacki TF, Walkovich KJ, Sturza J, et al. Neutropenia is an under-recognized finding in pediatric primary immunodeficiency diseases: an analysis of the United States immunodeficiency network registry. Presented at: the 2018 American Society of Hematology Annual Meeting; December 3, 2018; San Diego, CA. Abstract 3685." .
  2. Kirk SE, Grimes AB, Shelke S, Despotovic JM, Powers JM. The cost of a “benign” condition: healthcare utilization and infectious outcomes in young children with autoimmune neutropenia. Presented at: the 2018 American Society of Hematology Annual Meeting; December 1, 2018; San Diego, CA. Abstract 2220."

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