Abstract
Background : Wiskott Aldrich syndrome (WAS) is a rare disease and
hematopoietic stem cell transplant (HCT) is considered the treatment
modality of choice for WAS. Methods : We conducted a cross-sectional
analysis on the Kids’ inpatient database and compared hospitalization
rates, complications and healthcare utilizations in the transplant and
non-transplant arms. Results. : Of the 396 patients with WAS admitted
between 2006-2012, 114 underwent transplant and 269 did not. The
non-transplant arm included older children, female patients and more
African Americans. Death rates, income and payer source were similar in
both arms, however the total charge for each admission was higher in the
transplant arm. ED visits were similar but elective admissions were more
in the transplant arm. Length of stay was prolonged in the transplant
arm. When comparing morbidities, lymphomas, ulcerative colitis and
autoimmune complications of WAS were seen only in the non-transplant
arm. Conclusions : Our study shows that transplant is the largest
contributor to healthcare utilization in WAS patients. We noted a change
in practice moving away from splenectomy in WAS patients. We identified
healthcare disparities based on race and socioeconomic status and found
that only a few centers across the nation manage WAS, suggesting need
for wider resources for this very rare disease.