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.