PMID- 27350621
OWN - NLM
STAT- MEDLINE
DCOM- 20180307
LR  - 20181113
IS  - 1432-198X (Electronic)
IS  - 0931-041X (Linking)
VI  - 32
IP  - 1
DP  - 2017 Jan
TI  - Switching from immediate- to extended-release cysteamine in nephropathic 
      cystinosis patients: a retrospective real-life single-center study.
PG  - 91-97
AB  - BACKGROUND: Nephropathic cystinosis is a rare lysosomal storage disease which is 
      characterized by the accumulation of free cystine in lysosomes and subsequent 
      intracellular crystal formation of cystine throughout the body. If not treated 
      with cysteamine, a cystine-depleting agent, end-stage renal disease will develop 
      early, followed by multiple organ failure as the disease progresses. The 
      established cysteamine formulation requires a strict dosing regimen at 6-h 
      intervals. An extended release (ER) twice-daily formulation has recently been 
      developed. The aim of our study was to evaluate the implementation and outcomes 
      of this option in routine care. METHODS: All pediatric cystinosis patients' 
      records in Hannover Medical School were screened, and data on cysteamine therapy, 
      tolerability, dosing, estimated glomerular filtration rates (eGFR), white blood 
      cell cystine levels, and proton pump inhibitor (PPI) use were extracted for the 
      period January 2014 to January 2016. RESULTS: The median age of the 12 patients 
      enrolled in the study was 12.5 (range 1-18) years. At the end of the study period 
      ten of these patients received ER-cysteamine. There were no additional side 
      effects. Halitosis/bad breath was often subjectively judged as improved or 
      eliminated, and PPI use could be stopped in one of three patients. The main 
      reasons for switching to the ER formulation were difficult night-time 
      administration and uncontrolled disease. Mean eGFR values remained stable with a 
      median of 67 ml/min/1.73 m(2) before and after the transition. White blood cell 
      (WBC) cystine values remained low after the switch (1 nmol/mg protein before and 
      after transition; p = 0.64). CONCLUSIONS: In this single-center cohort, the 
      switch from IR- to ER-cysteamine was safe and effective over the short term and 
      provided advantages in terms of frequency of administration and less 
      halitosis/bad breath. The long-term benefit of this option needs to be evaluated 
      in future studies.
FAU - Ahlenstiel-Grunow, Thurid
AU  - Ahlenstiel-Grunow T
AD  - Department of Pediatric Nephrology, Hannover Medical School, Carl-Neuberg-Strasse 
      1, 30625, Hannover, Germany.
FAU - Kanzelmeyer, Nele K
AU  - Kanzelmeyer NK
AD  - Department of Pediatric Nephrology, Hannover Medical School, Carl-Neuberg-Strasse 
      1, 30625, Hannover, Germany.
FAU - Froede, Kerstin
AU  - Froede K
AD  - Department of Pediatric Nephrology, Hannover Medical School, Carl-Neuberg-Strasse 
      1, 30625, Hannover, Germany.
FAU - Kreuzer, Martin
AU  - Kreuzer M
AD  - Department of Pediatric Nephrology, Hannover Medical School, Carl-Neuberg-Strasse 
      1, 30625, Hannover, Germany.
FAU - Drube, Jens
AU  - Drube J
AD  - Department of Pediatric Nephrology, Hannover Medical School, Carl-Neuberg-Strasse 
      1, 30625, Hannover, Germany.
FAU - Lerch, Christian
AU  - Lerch C
AD  - Department of Pediatric Nephrology, Hannover Medical School, Carl-Neuberg-Strasse 
      1, 30625, Hannover, Germany.
FAU - Pape, Lars
AU  - Pape L
AD  - Department of Pediatric Nephrology, Hannover Medical School, Carl-Neuberg-Strasse 
      1, 30625, Hannover, Germany. Pape.Lars@mh-hannover.de.
LA  - eng
PT  - Journal Article
PT  - Research Support, Non-U.S. Gov't
DEP - 20160627
PL  - Germany
TA  - Pediatr Nephrol
JT  - Pediatric nephrology (Berlin, Germany)
JID - 8708728
RN  - 0 (Delayed-Action Preparations)
RN  - 0 (Renal Agents)
RN  - 48TCX9A1VT (Cystine)
RN  - 5UX2SD1KE2 (Cysteamine)
SB  - IM
CIN - Pediatr Nephrol. 2017 Jul;32(7):1281-1282. PMID: 28255804
MH  - Adolescent
MH  - Child
MH  - Child, Preschool
MH  - Cohort Studies
MH  - Cysteamine/*administration & dosage/adverse effects/*therapeutic use
MH  - Cystine/blood
MH  - Cystinosis/*drug therapy/etiology
MH  - Delayed-Action Preparations
MH  - Drug Compounding
MH  - Female
MH  - Glomerular Filtration Rate
MH  - Humans
MH  - Infant
MH  - Leukocytes/metabolism
MH  - Male
MH  - Renal Agents/*administration & dosage/adverse effects/*therapeutic use
MH  - Retrospective Studies
MH  - Treatment Outcome
OTO - NOTNLM
OT  - Cysteamine
OT  - Cystine depleting agents
OT  - Fanconi syndrome
OT  - Lysosomal storage disease
OT  - Nephropathic cystinosis
EDAT- 2016/06/29 06:00
MHDA- 2018/03/08 06:00
CRDT- 2016/06/29 06:00
PHST- 2016/04/04 00:00 [received]
PHST- 2016/05/16 00:00 [accepted]
PHST- 2016/04/28 00:00 [revised]
PHST- 2016/06/29 06:00 [pubmed]
PHST- 2018/03/08 06:00 [medline]
PHST- 2016/06/29 06:00 [entrez]
AID - 10.1007/s00467-016-3438-x [pii]
AID - 10.1007/s00467-016-3438-x [doi]
PST - ppublish
SO  - Pediatr Nephrol. 2017 Jan;32(1):91-97. doi: 10.1007/s00467-016-3438-x. Epub 2016 
      Jun 27.