Johannes Hofer1,2,3, Magdalena Riedl Khursigara4,5, Markus Perl6, Thomas Giner4, Alejandra Rosales4, Gerard Cortina4, Siegfied Waldegger4, Therese Jungraithmayr4,7, Reinhard Würzner8. 1. Institute of Neurology of Senses and Language, Hospital of St John of God, Linz, Austria. Johannes.Hofer@bblinz.at. 2. Research Institute of Developmental Medicine, Johannes Kepler University Linz, Linz, Austria. Johannes.Hofer@bblinz.at. 3. Department of Pediatrics I, Medical University of Innsbruck, Innsbruck, Austria. Johannes.Hofer@bblinz.at. 4. Department of Pediatrics I, Medical University of Innsbruck, Innsbruck, Austria. 5. Department of Pediatrics, The Hospital for Sick Children, University of Toronto, Toronto, Ontario, Canada. 6. Department of Pediatrics III, Medical University of Innsbruck, Innsbruck, Austria. 7. Department of Pediatric Nephrology, Hospital Memmingen, Memmingen, Germany. 8. Institute of Hygiene & Medical Microbiology, Medical University of Innsbruck, Innsbruck, Austria. reinhard.wuerzner@i-med.ac.at.
Abstract
BACKGROUND: The complement factor H antibody (CFH-Ab)-associated hemolytic uremic syndrome (HUS) forms a distinct subgroup within the complement-mediated HUS disease spectrum. The autoimmune nature of this HUS subgroup implies the potential benefit of a targeted immunosuppressive therapy. Data on long-term outcome are scarce. METHODS: This observational study evaluates the clinical outcome of 19 pediatric CFH-Ab HUS patients from disease onset until their 5-year follow-up. RESULTS: All but one relapse occurred during the first 2 years, and patients who had no relapse within the first 6 months were relapse-free until the end of the observation period. Kidney function at disease onset determines long-term kidney function: all individuals with normal kidney function at disease onset had normal kidney function after 5 years, and all patients with reduced kidney function at onset had impaired kidney function at the last follow-up. Level of CFH-Ab titer at disease onset was not correlated with a higher risk of recurrences or worse long-term outcome after 5 years. Resolution of CFH-Ab titers after 5 years was common. CONCLUSIONS: CFH-Ab HUS patients have a varied overall long-term course. Early relapses are common, making close surveillance during the first years essential, regardless of the initial CFH-Ab titer.
BACKGROUND: The complement factor H antibody (CFH-Ab)-associated hemolytic uremic syndrome (HUS) forms a distinct subgroup within the complement-mediated HUS disease spectrum. The autoimmune nature of this HUS subgroup implies the potential benefit of a targeted immunosuppressive therapy. Data on long-term outcome are scarce. METHODS: This observational study evaluates the clinical outcome of 19 pediatric CFH-Ab HUS patients from disease onset until their 5-year follow-up. RESULTS: All but one relapse occurred during the first 2 years, and patients who had no relapse within the first 6 months were relapse-free until the end of the observation period. Kidney function at disease onset determines long-term kidney function: all individuals with normal kidney function at disease onset had normal kidney function after 5 years, and all patients with reduced kidney function at onset had impaired kidney function at the last follow-up. Level of CFH-Ab titer at disease onset was not correlated with a higher risk of recurrences or worse long-term outcome after 5 years. Resolution of CFH-Ab titers after 5 years was common. CONCLUSIONS: CFH-Ab HUS patients have a varied overall long-term course. Early relapses are common, making close surveillance during the first years essential, regardless of the initial CFH-Ab titer.
Authors: Marie-Agnès Dragon-Durey; Sidharth Kumar Sethi; Arvind Bagga; Caroline Blanc; Jacques Blouin; Bruno Ranchin; Jean-Luc André; Nobuaki Takagi; Hae Il Cheong; Pankaj Hari; Moglie Le Quintrec; Patrick Niaudet; Chantal Loirat; Wolf Herman Fridman; Véronique Frémeaux-Bacchi Journal: J Am Soc Nephrol Date: 2010-11-04 Impact factor: 10.121
Authors: Johannes Hofer; Andreas R Janecke; L B Zimmerhackl; Magdalena Riedl; Alejandra Rosales; Thomas Giner; Gerard Cortina; Carola J Haindl; Barbara Petzelberger; Miriam Pawlik; Verena Jeller; Udo Vester; Bettina Gadner; Michael van Husen; Michael L Moritz; Reinhard Würzner; Therese Jungraithmayr Journal: Clin J Am Soc Nephrol Date: 2012-12-14 Impact factor: 8.237