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Abstract: FR-PO1087

Significant Reduction in Proteinuria After Treatment with Tafamidis

Session Information

Category: Glomerular Diseases

  • 1203 Glomerular Diseases: Clinical, Outcomes, and Trials

Authors

  • Ferrer-Nadal, Asuncion, Hospital Son Llàtzer, Palma de Mallorca, Baleares, Spain
  • Ripoll, Tomas, Hospital Son Llatzer, Palma de Mallorca, Baleares, Spain
  • Uson, Mercedes, Hospital Son Llatzer, Palma de Mallorca, Baleares, Spain
  • Figuerola, Antoni, Hospital Son Llatzer, Palma de Mallorca, Baleares, Spain
  • Buades, Juan, Hospital Son Llatzer, Palma de Mallorca, Baleares, Spain

Group or Team Name

  • TTRhA Spanish Group
Introduction

Hereditary Transthyretin Amyloidosis (hATTR) in Mallorca represents the fifth world focus. Tafamidis (Vyndaqel, Pfizer) has been used since 2011 with promising expectations. It promotes stabilization of the TTR tetramer and slow tissue deposition. Consequently, it contributes to change the course of disease, slowing the presentation of clinical manifestations. The Portuguese group presented few cases with small reduction in proteinuria last year. We present now an interesting case of a significant decrease in heavy proteinuria.

Case Description

We display the first case presented (to our knowledge) with great proteinuria reduction from nephrotic range to mild range. This is a well documented female case of late onset Val30Met. Amyloid deposits were confirmed by positive congo red stain in a skin biopsy. Patient came into the outpatient consultation due to cardiology manifestations. Patient was studied in depth, and then neurologic, intestinal and nephrologic impairment was also found. Nephrotic range proteinuria of 4,2 g in 24h with kidney dysfunction were present. We also found an unusual dissociation among glomerular filtration rate formulas (urine and blood results). Therefore, we performed a 51Cr-EDTA renal function, and also conducted a dynamic study with 99mTc-MAG3 scintigraphy. 6 months after taking tafamidis, proteinuria was reduced to 1,6 g in 24h.

Discussion

This finding may be very promising for the future to slow the natural course of proteinuric kidney TTR amyloidosis disease. It will permit to avoid or slow progression to end-stage renal disease and renal replacement therapy.