Hematopoietic stem cell transplantation in treating CNS-FHL: A treatment game changer?


  • This study shows that hematopoietic stem cell transplantation (HCT) is well tolerated and effective in treating CNS-isolated inflammation associated with familial hemophagocytic lymphohistiocytosis (FHL) gene mutations (CNS-FHL) (Class IV evidence).

Why this matters

    HCT has the ability to correct the underlying genetic etiology versus controlling inflammatory disease through immunosuppression, and is therefore likely to have a profound impact, and markedly reduce patient morbidity and mortality.