Abstract | INTRODUCTION: AIMS/METHODS: KAMPER is an ongoing, observational, multicentre registry with the primary objective of providing information over 15 years on long-term safety of sapropterin dihydrochloride treatment in patients with HPA. Here we report initial data on characteristics from patients recruited by the time of the third interim analysis and results at 1 year. RESULTS: Overall, 325 patients from 55 sites in seven European countries were included in the analysis: 296 (91.1%) patients with PAH deficiency (median [Q1, Q3] age, 10.3 [7.2, 15.0] years) and 29 (8.9%) with BH4 deficiency (12.8 [6.6, 18.9] years). Fifty-nine patients (18.2%) were aged ≥18 years; 4 patients were pregnant. No elderly patients (aged ≥65 years) or patients with renal or hepatic insufficiency were enroled in the study. Twelve-month data were available for 164 patients with PAH deficiency and 16 with BH4 deficiency. No new safety concerns were identified as of May 2013. CONCLUSIONS:
|
Authors | Friedrich K Trefz, Ania C Muntau, Florian B Lagler, Flavie Moreau, Jan Alm, Alberto Burlina, Frank Rutsch, Amaya Bélanger-Quintana, François Feillet, KAMPER investigators |
Journal | JIMD reports
(JIMD Rep)
Vol. 23
Pg. 35-43
( 2015)
ISSN: 2192-8304 [Print] United States |
PMID | 25822821
(Publication Type: Journal Article)
|