Table 1.
Learning points | Clinical knowledge gaps |
---|---|
• Vascular pathology associated with STAT3-HIES can cause life threatening illness. • Hematopoietic stem cell transplantation (HSCT) does not appear to prevent this complication. |
• What is the natural history and clinical significance of vasculopathy? • Should vascular screening be offered to patients with STAT3-HIES? • Does impairment to cutaneous and lung healing extend to other (cardiac and cerebral) tissues? • What are the long-term sequelae of cerebral white matter hyperintensities in the aging STAT3-HIES patient group? • Can a molecularly-targeted approach normalize STAT3 signaling pathways across tissues? |