CLIPPERS Responsive to Cladribine as a Durable Steroid-Sparing Agent.

OBJECTIVE: We report a case of chronic lymphocytic inflammation with pontine perivascular enhancement responsive to steroids (CLIPPERS) who achieved durable and steroid-free remission after IV cladribine. METHODS: A 25 year-old man presented with progressively worsening headaches, polydipsia, dysarthria, diplopia and vertigo, and obtundation requiring respiratory support. CSF revealed lymphocytosis, and MRI revealed a perivascular pattern of punctate enhancement involving the pons. An extensive workup for inflammatory, autoimmune, infective, and malignant explanations was unrevealing. He responded dramatically to steroids, compatible with CLIPPERS as a diagnosis of exclusion. Attempts to wean prednisone over the ensuing year resulted in 2 clinical relapses and persistent punctate enhancement. Given significant steroid side effects, steroid-sparing agents were considered. RESULTS: IV cladribine IV (0.0875 mg/kg adjusted body weight daily × 4 days at 0, 4, 8, and 16 months) was selected, given its favorable side effect profile including lower risks of malignancy and infertility and the potential for long-lasting effects. The only side effect was short-term fatigue at the time of infusion. At 20 months after cladribine initiation, he was able to wean-off prednisone altogether. Now at 33 months, he remains in clinical and MRI remission. DISCUSSION: Cladribine is a rational candidate steroid-sparing treatment for presumed neurologic autoimmune conditions such as CLIPPERS. CLASSIFICATION OF EVIDENCE: This study provides Class IV evidence that cladribine is a steroid-sparing treatment consideration in CLIPPERS.

Transplantation for autoimmune diseases in north and South America: a report of the Center for International Blood and Marrow Transplant Research.

Hematopoietic cell transplantation (HCT) is an emerging therapy for patients with severe autoimmune diseases (AID). We report data on 368 patients with AID who underwent HCT in 64 North and South American transplantation centers reported to the Center for International Blood and Marrow Transplant Research between 1996 and 2009. Most of the HCTs involved autologous grafts (n = 339); allogeneic HCT (n = 29) was done mostly in children. The most common indications for HCT were multiple sclerosis, systemic sclerosis, and systemic lupus erythematosus. The median age at transplantation was 38 years for autologous HCT and 25 years for allogeneic HCT. The corresponding times from diagnosis to HCT were 35 months and 24 months. Three-year overall survival after autologous HCT was 86% (95% confidence interval [CI], 81%-91%). Median follow-up of survivors was 31 months (range, 1-144 months). The most common causes of death were AID progression, infections, and organ failure. On multivariate analysis, the risk of death was higher in patients at centers that performed fewer than 5 autologous HCTs (relative risk, 3.5; 95% CI, 1.1-11.1; P = .03) and those that performed 5 to 15 autologous HCTs for AID during the study period (relative risk, 4.2; 95% CI, 1.5-11.7; P = .006) compared with patients at centers that performed more than 15 autologous HCTs for AID during the study period. AID is an emerging indication for HCT in the region. Collaboration of hematologists and other disease specialists with an outcomes database is important to promote optimal patient selection, analysis of the impact of prognostic variables and long-term outcomes, and development of clinical trials.