Tocilizumab for the treatment of TAFRO syndrome: a systematic literature review.

TAFRO syndrome is a newly recognized disease entity characterized by thrombocytopenia, anasarca, fever, reticulin myelofibrosis, renal dysfunction, and organomegaly. The objective of this study was to investigate the effectiveness of tocilizumab, an anti-interleukin-6 receptor antibody, in patients with TAFRO syndrome. We performed a systematic literature review from inception to July 5, 2020, for articles reporting tocilizumab administration for the treatment of TAFRO syndrome. We identified 31 patients with TAFRO syndrome treated with tocilizumab. The mean age was 49.8 years, and 61.3% of the patients were male. The mean observation period was 12.6 months. Tocilizumab was used at the standard intravenous dose (8 mg/kg) weekly or every 2 weeks in combination with other immunosuppressive drugs, such as glucocorticoids, rituximab, cyclosporine, or cyclophosphamide, in most of the patients. Eighteen patients (58.1%) received tocilizumab as a first-line treatment, while it was a second-line or a third-line treatment for 13 patients with insufficient responses to the prior treatments. Sixteen patients (51.6%) obtained complete response to tocilizumab treatment, whereas 15 patients showed only partial or no response. Detailed factors of ineffectiveness included persistent thrombocytopenia (n = 7), persistent anasarca (n = 5), persistent renal dysfunction (n = 2), and persistent fever (n = 2). A total of four patients (12.9%) succumbed to the disease, while the remaining twenty-seven patients survived. Two patients achieved drug-free remission at last visit, and disease remission was maintained with tocilizumab monotherapy in five patients. No new safety signal was reported. Tocilizumab was effective in ~ 50% of the patients, suggesting it could serve as a treatment choice for TAFRO syndrome. Poor clinical response to tocilizumab observed in other patients highlights the need for the additional therapeutic treatment options.