Treatment of recurrent CNS disease post-bone marrow transplant in familial HLH

CNS involvement in Hemophagocytic Lymphohistiocytosis (HLH) has been reported in 63-73% of children at diagnosis [Haddad et al. (1997); Blood 89: 794-800; Horne et al. (2008); Br J Haematol 140: 327-335]. Patients can present with neurologic symptoms, abnormal CSF cytology, abnormal neuro-imaging, or a combination of these findings. CNS involvement is usually associated with a poor prognosis and increased mortality. The 3 year overall survival is 44% in patients with CNS involvement compared to 67% in patients without CNS involvement at diagnosis [Horne et al. (2008); Br J Haematol 140: 327-335]. We describe a treatment strategy employing systemic dexamethasone to control CNS disease in a patient with familial HLH and persistent CNS disease post Bone Marrow Transplant.