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Publications

Journal Article Ahead of print

PubMed: 41605691 DOI: 10.1507/endocrj.EJ25-0431

Use, efficacy, and safety of desmopressin for congenital nephrogenic diabetes insipidus in children: a nationwide survey.

Endocr J

Authors: Kento Ikegawa, Masanobu Fujimoto, Kohei Aoyama, Hirofumi Nakayama, Takuo Kubota, Keisuke Nagasaki, Setsuko Naganuma, Makoto Ono, Shinji Higuchi, Haruo Mizuno, Susumu Kanzaki, Yoshihiko Morikawa, Masataka Honda, Naoko Amano, Yukihiro Hasegawa

Affiliations

  • Division of Endocrinology and Metabolism, Tokyo Metropolitan Children's Medical Center, Tokyo, Japan.
  • Clinical Research Support Center, Tokyo Metropolitan Children's Medical Center, Tokyo, Japan.
  • Division of Pediatrics and Perinatology, Faculty of Medicine, Tottori University, Tottori, Japan.
  • Department of Pediatrics and Neonatology, Nagoya City University Graduate School of Medical Sciences, Aichi, Japan.
  • Department of Pediatrics, Graduate School of Medicine, The University of Osaka, Osaka, Japan.
  • Division of Pediatrics, Department of Homeostatic Regulation and Development, Niigata University Graduate School of Medical and Dental Sciences, Niigata, Japan.
  • Department of Pediatrics, Kumamoto Rosai Hospital, Kumamoto, Japan.
  • Department of Pediatrics, Chiba Kaihin Municipal Hospital, Chiba, Japan.
  • Division of Pediatric Endocrinology, Metabolism, and Nephrology, Osaka City General Hospital, Osaka, Japan.
  • Department of Pediatrics, Fujita Health University School of Medicine, Aichi, Japan.
  • Asahigawaso Rehabilitation and Medical Center, Okayama, Japan.
  • Research Promotion Center, Tokyo Metropolitan Hospital Organization, Tokyo, Japan.
  • Department of Pediatrics, Keio University School of Medicine, Tokyo, Japan.
  • Department of Pediatrics, Tama-Hokubu Medical Center, Tokyo, Japan.

Abstract

Congenital nephrogenic diabetes insipidus (CNDI) is characterized by resistance of the distal nephrons and collecting ducts to arginine vasopressin (AVP). High doses of 1-deamino-8-D-arginine vasopressin (DDAVP), a V2-receptor-selective agonist, are effective in some cases. The present study aimed to demonstrate the use, efficacy, and safety of DDAVP and the characteristics of patients who responded to this treatment. The present, retrospective, multicentric, observational survey of patients with CNDI receiving DDAVP was based on a previous, nationwide survey conducted by the Japanese Society for Pediatric Endocrinology (JSPE) and collected data on the use (formulation, dosage, and treatment duration), efficacy (change in urine output and height SDS), and safety of DDAVP. In the initial survey, 43 of 123 JSPE council members (35.0%) observed the patients. The secondary survey of 13 patients found DDAVP to be effective in five patients (38.5%), as evidenced by a 12.6-31.6% decrease in urine output. The maximum urine osmolality on a water deprivation test and urine osmolality after vasopressin injection were lower in patients who were unresponsive to DDAVP than in those who were responsive to the drug (106 vs. 206 mOsm/H 2O/kg, 140 vs. 525 mOsm/H 2O/kg). The AVPR2 variants identified in the DDAVP-responsive group were p.Ala37Pro, p.Leu44Phe, p.Arg104Cys, and p.Tyr128Ser. DDAVP was effective against CNDI with residual V2R function. The water deprivation test with vasopressin injection and genetic testing may be useful for predicting responsiveness to DDAVP.

MeSH

Diabetes Mellitus, Arginine Vasopressin, Diabetes Insipidus, Nephrogenic, Endocrinology, Injections, Deamino Arginine Vasopressin, Safety, Arginine, Therapeutics, Water Deprivation, Diabetes Insipidus, Child