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Abstract: PO1898

Ruxolitinib for Graft vs. Host Disease-Associated Nephrotic Syndrome: A Case Report

Session Information

Category: Onco-Nephrology

  • 1500 Onco-Nephrology


  • Deep, Aman, University of Pennsylvania, Philadelphia, Pennsylvania, United States
  • Vujjini, Vikas, University of Pennsylvania, Philadelphia, Pennsylvania, United States
  • Geara, Abdallah Sassine, University of Pennsylvania, Philadelphia, Pennsylvania, United States

Graft-versus-host disease (GVHD) is a serious complication of allogenic stem cell transplant in which donor T-cells attack the host antigens. We report a case in which ruxolitinib successfully treated GVHD-related nephrotic syndrome.

Case Description

A 48-years-old male known to have myelodysplastic syndrome (MDS) was referred for evaluation of proteinuria. He was diagnosed with MDS four years prior. Since his disease was persistent with azacitidine, he received allogenic stem cell transplant (SCT) about a year after the MDS diagnosis and he achieved complete remission. However, the post-transplant course was complicated by chronic GVHD which manifested mainly as non-specific interstitial pneumonia (NSIP) about three and a half-year post-transplant. NSIP was treated with high-dose oral steroid therapy, which was tapered down to a maintenance dose of 10 mg daily, and Mycophenolate Mofetil. During his course of GVHD, he had persistent mild proteinuria (UPCR less than 1 g/g of creatinine) without active urine sediment. This proteinuria was noted initially prior to NSIP diagnosis, improved while on high-dose prednisone but progressively worsened after the prednisone was tapered to 10 mg once daily. The proteinuria peaked at 2.4 g/g of creatinine with hypoalbuminemia of 2.6 g/dL at which point it was investigated with a renal biopsy. Renal biopsy showed Membranous Nephropathy with negative staining for anti-PLA2R antibody. The patient was started on ruxolitinib at a dose of 10 mg twice a day. Subsequent follow-up showed drastic reduction in proteinuria. UPCR (g/g of creatinine) of 0.96, 0.4 and 0.09 was noted at 1,2- and 5-months post-therapy initiation respectively.


Nephrotic syndrome is a rare manifestation of GVHD with membranous nephropathy histology seen in almost two thirds of patients. Traditionally treated with high-dose steroid with variable efficacy, we decided against it due to the patient-reported adverse effects from prior high-dose steroid therapy. Recent studies such as REACH2 and REACH3 trials have demonstrated that ruxolitinib, a selective Janus Kinase (JAK) 1 and 2 inhibitor, has superior efficacy than other second-line therapy options available. Hence, ruxolitinib can be considered in GVHD-associated Nephrotic syndrome especially if a steroid-sparing approach is needed.