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10.1681/ASN.2015030337

http://scihub22266oqcxt.onion/10.1681/ASN.2015030337
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suck abstract from ncbi


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pmid26185201
      J+Am+Soc+Nephrol 2016 ; 27 (3 ): 952-62
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  • Patients with Fabry Disease after Enzyme Replacement Therapy Dose Reduction and Switch-2-Year Follow-Up #MMPMID26185201
  • Lenders M ; Canaan-Kühl S ; Krämer J ; Duning T ; Reiermann S ; Sommer C ; Stypmann J ; Blaschke D ; Üçeyler N ; Hense HW ; Brand SM ; Wanner C ; Weidemann F ; Brand E
  • J Am Soc Nephrol 2016[Mar]; 27 (3 ): 952-62 PMID26185201 show ga
  • Because of the shortage of agalsidase-? supply between 2009 and 2012, patients with Fabry disease either were treated with reduced doses or were switched to agalsidase-?. In this observational study, we assessed end organ damage and clinical symptoms with special focus on renal outcome after 2 years of dose-reduction and/or switch to agalsidase-?. A total of 89 adult patients with Fabry disease who had received agalsidase-? (1.0 mg/kg body wt) for >1 year were nonrandomly assigned to continue this treatment regimen (regular-dose group, n=24), to receive a reduced dose of 0.3-0.5 mg/kg and a subsequent switch to 0.2 mg/kg agalsidase-? (dose-reduction-switch group, n=28), or to directly switch to 0.2 mg/kg agalsidase-? (switch group, n=37) and were followed-up for 2 years. We assessed clinical events (death, myocardial infarction, severe arrhythmia, stroke, progression to ESRD), changes in cardiac and renal function, Fabry-related symptoms (pain, hypohidrosis, diarrhea), and disease severity scores. Determination of renal function by creatinine and cystatin C-based eGFR revealed decreasing eGFRs in the dose-reduction-switch group and the switch group. The Mainz Severity Score Index increased significantly in these two groups (P=0.02 and P<0.001, respectively), and higher frequencies of gastrointestinal pain occurred during follow-up. In conclusion, after 2 years of observation, all groups showed a stable clinical disease course with respect to serious clinical events. However, patients under agalsidase-? dose-reduction and switch or a direct switch to agalsidase-? showed a decline of renal function independent of the eGFR formula used.
  • |Abdominal Pain/chemically induced [MESH]
  • |Adult [MESH]
  • |Creatinine/blood [MESH]
  • |Cystatin C/blood [MESH]
  • |Drug Substitution/adverse effects [MESH]
  • |Enzyme Replacement Therapy/adverse effects [MESH]
  • |Fabry Disease/complications/*drug therapy/*physiopathology [MESH]
  • |Female [MESH]
  • |Follow-Up Studies [MESH]
  • |Glomerular Filtration Rate/*drug effects [MESH]
  • |Humans [MESH]
  • |Isoenzymes/*administration & dosage/adverse effects/therapeutic use [MESH]
  • |Male [MESH]
  • |Middle Aged [MESH]
  • |Renal Insufficiency, Chronic/etiology/*physiopathology [MESH]
  • |Retrospective Studies [MESH]
  • |Serum Albumin/metabolism [MESH]
  • |Severity of Illness Index [MESH]


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