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Bartter syndrome: benefits and side effects of long-term treatment

Maria Helena Vaisbich1, Maria Danisi Fujimura, Vera H Koch

  • 1Pediatric Nephrology Unit, Instituto da Criança, Hospital das Clínicas, Faculdade de Medicina da Universidade de São Paulo, Brazil. vaisbich@terra.com.br

Pediatric Nephrology (Berlin, Germany)
|June 19, 2004
PubMed

Insights

This study on Bartter syndrome in children highlights that while indomethacin and potassium supplementation improve growth and electrolyte balance, long-term use can cause renal and gastrointestinal issues, necessitating regular monitoring. Preliminary rofecoxib results are promising.

Area of Science:

  • Pediatric Nephrology
  • Clinical Pharmacology

Background:

  • Bartter syndrome is a rare genetic disorder affecting kidney salt reabsorption.
  • It leads to electrolyte imbalances, dehydration, and failure to thrive in children.

Purpose of the Study:

  • To evaluate the long-term efficacy and safety of indomethacin and potassium supplementation in pediatric Bartter syndrome.
  • To identify potential complications and suggest monitoring strategies.
  • To explore rofecoxib as an alternative treatment.

Main Methods:

  • Retrospective analysis of clinical and laboratory data from 12 children with Bartter syndrome.
  • Long-term follow-up assessing growth, metabolic status, electrolyte balance, and adverse events.
  • Endoscopic evaluation for gastrointestinal complications.
  • Preliminary assessment of rofecoxib treatment.

Main Results:

  • Indomethacin and potassium supplementation improved growth velocity and metabolic/electrolyte balance in most patients.
  • Long-term complications included persistent hypokalemia, renal issues (nephrocalcinosis, cysts, decreased GFR), and gastrointestinal problems (ulcers, gastritis).
  • Rofecoxib showed satisfactory preliminary results as an alternative therapy.

Conclusions:

  • Long-term pharmacological therapy for Bartter syndrome requires careful monitoring for renal and gastrointestinal side effects.
  • Regular surveillance of renal function and gastrointestinal endoscopy are crucial.
  • Rofecoxib may be a viable alternative to indomethacin.

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