title: "MY PKU": increasing self-management in patients with phenylketonuria. A randomized controlled trial.
abstract: BACKGROUND
  Phenylketonuria (PKU) is an autosomal recessive disorder of phenylalanine metabolism. The inability to convert phenylalanine (Phe) into tyrosine causes Phe to accumulate in the body. Adherence to a protein restricted diet, resulting in reduced Phe levels, is essential to prevent cognitive decline. Frequent evaluation of plasma Phe levels and, if necessary, adjustment of the diet are the mainstay of treatment. We aimed to assess whether increased self-management of PKU patients and/or their parents is feasible and safe, by providing direct online access to blood Phe values without immediate professional guidance.
  
  
  METHODS
  Thirty-eight patients aged ≥ 1 year participated in a 10 month randomized controlled trial. Patients were randomized into a study group (1) or a control group (2). Group 2 continued the usual procedure: a phone call or e-mail by a dietician in case of a deviant Phe value. Group 1 was given a personal "My PKU" web page with a graph of their recent and previous Phe values, online general information about the dietary treatment and the Dutch PKU follow-up guidelines, and a message-box to contact their dietician if necessary. Phe values were provided on "My PKU" without advice. Outcome measures were: differences in mean Phe value, percentage of values above the recommended range and Phe sample frequency, between a 10-month pre-study period and the study period in each group, and between the groups in both periods. Furthermore we assessed satisfaction of patients and/or parents with the 'My PKU' procedure of online availability.
  
  
  RESULTS
  There were no significant differences in mean Phe value, percentage of values above recommended range or in frequency of blood spot sampling for Phe determination between the pre-study period and the study period in each group, nor between the 2 groups during the periods. All patients and/or parents expressed a high level of satisfaction with the new way of disease management.
  
  
  CONCLUSIONS
  Increased self-management in PKU by providing patients and/or parents their Phe values without advice is feasible and safe and is highly appreciated.
  
  
  BACKGROUND
  The trial was registered with The Netherlands National Trial Register (NTR #1171) before recruitment of patients.
authors.affiliation: Department of Pediatrics, Academic Medical Center (AMC), University of Amsterdam, AZ Amsterdam, The Netherlands. a.m.bosch@amc.uva.nl
authors.forename: Amber E
authors.initials: AE
authors.lastname: Ten Hoedt
authors.forename: Carla Em
authors.initials: CE
authors.lastname: Hollak
authors.forename: Carolien Ca
authors.initials: CC
authors.lastname: Boelen
authors.forename: N Ada P
authors.initials: NA
authors.lastname: van der Herberg-van de Wetering
authors.forename: Nienke M
authors.initials: NM
authors.lastname: Ter Horst
authors.forename: Cora F
authors.initials: CF
authors.lastname: Jonkers
authors.forename: Frits A
authors.initials: FA
authors.lastname: Wijburg
authors.forename: Annet M
authors.initials: AM
authors.lastname: Bosch
mesh_terms: D000293:Adolescent; D000328:Adult; D002648:Child; D002675:Child, Preschool; D004032:Diet; D005260:Female; D006801:Humans; D007223:Infant; D008297:Male; D017060:Patient Satisfaction; D010649:Phenylalanine / Q000097:blood*; D010661:Phenylketonurias / Q000178:diet therapy*; D012648:Self Care; D055815:Young Adult
chemical_list: D010649:Phenylalanine