Effective Treatment with Ruxolitinib and Ropeginterferon Alfa-2b for Refractory TAFRO-like Syndrome.
Key claim: Combination therapy with ruxolitinib and ropeginterferon alfa-2b successfully resolved refractory TAFRO-like syndrome in a polycythemia vera patient with JAK2 V617F mutation.
Abstract
Owing to the lack of appropriate treatment, TAFRO syndrome often presents with multiple organ dysfunction and fatality. The Janus kinase (JAK)/signal transducers and activators of transcription (STAT) pathway has recently been shown to play an important role in the pathogenesis of inflammation in TAFRO syndrome, and inhibitors of the JAK/STAT pathway may be effective as therapeutic agents for TAFRO syndrome. We herein report the successful treatment using combination therapy with ruxolitinib and ropeginterferon alfa-2b of a case of TAFRO-like syndrome with a long history of polycythemia vera with JAK2 V617F refractory to several treatments.
Why this matters
TAFRO-like syndrome is a rare, poorly-characterized hyperinflammatory complication that has not been well-documented in the PV natural-history literature, and this case extends the recognized phenotypic spectrum of JAK2 V617F-driven disease. The successful combination of ruxolitinib (JAK1/2 inhibition of cytokine storm) with ropeginterferon alfa-2b (disease-modifying clonal suppression) suggests a mechanistically rational salvage strategy that pairs two PV-established agents for an off-label refractory indication, potentially informing management of other inflammatory complications in PV.