Universität zu Köln

Hackl, Agnes, Ehren, Rasmus, Kirschfink, Michael, Zipfel, Peter F., Beck, Bodo B., Weber, Lutz T. and Habbig, Sandra (2017). Successful discontinuation of eculizumab under immunosuppressive therapy in DEAP-HUS. Pediatr. Nephrol., 32 (6). S. 1081 - 1088. NEW YORK: SPRINGER. ISSN 1432-198X

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Abstract

Deficiency of complement factor H-related plasma proteins and complement factor H autoantibody-positive hemolytic uremic syndrome (DEAP-HUS), which is characterized by the deficiency of complement-factor H-related (CFHR) plasma proteins and the subsequent formation of autoantibodies against complement factor H (CFH), has been reported to have an adverse outcome in one third of patients. Therapy options include prompt removal of antibodies by plasma exchange and immunosuppressive therapy. Recently, restoration of complement control using the monoclonal antibody eculizumab has been shown to be effective as first- and as second-line therapy in cases of therapy resistance or severe side effects of the applied therapy. Here, we report a 6-year-old girl with DEAP-HUS and first-line therapy with eculizumab under immunosuppressive therapy with glucocorticoids and mycophenolate mofetil (MMF). This therapy led to a prompt and sustained clinical recovery, to a stable reduction of complement activation, and to a rapid decline in autoantibody titer. A second increase in the autoantibody titer was successfully treated with methylprednisolone and the child remained in remission. After 8.3 months of sustained complement control and 4.5 months of stable antibody suppression, eculizumab was successfully discontinued without any sign of relapse. To our knowledge, this is the first reported case of a child with DEAP-HUS treated with the combination of eculizumab and immunosuppression as first-line therapy avoiding any HUS- or therapy-related complications and resulting in prompt clinical recovery. Importantly, clinical remission is maintained after discontinuation of eculizumab under stable immunosuppression.

Item Type:	Journal Article
Creators:	Creators Email ORCID ORCID Put Code Hackl, Agnes UNSPECIFIED UNSPECIFIED UNSPECIFIED Ehren, Rasmus UNSPECIFIED UNSPECIFIED UNSPECIFIED Kirschfink, Michael UNSPECIFIED UNSPECIFIED UNSPECIFIED Zipfel, Peter F. UNSPECIFIED UNSPECIFIED UNSPECIFIED Beck, Bodo B. UNSPECIFIED UNSPECIFIED UNSPECIFIED Weber, Lutz T. UNSPECIFIED UNSPECIFIED UNSPECIFIED Habbig, Sandra UNSPECIFIED UNSPECIFIED UNSPECIFIED
URN:	urn:nbn:de:hbz:38-230523
DOI:	10.1007/s00467-017-3612-9
Journal or Publication Title:	Pediatr. Nephrol.
Volume:	32
Number:	6
Page Range:	S. 1081 - 1088
Date:	2017
Publisher:	SPRINGER
Place of Publication:	NEW YORK
ISSN:	1432-198X
Language:	English
Faculty:	Unspecified
Divisions:	Unspecified
Subjects:	no entry
Uncontrolled Keywords:	Keywords Language HEMOLYTIC-UREMIC SYNDROME; FACTOR-H ANTIBODIES; PEDIATRIC-PATIENTS; DEFICIENCY; CHILDREN Multiple languages Pediatrics; Urology & Nephrology Multiple languages
Refereed:	Yes
URI:	http://kups.ub.uni-koeln.de/id/eprint/23052