A randomised comparison of oral desmopressin lyophilisate (MELT) and tablet formulations in children and adolescents with primary nocturnal enuresis

Authors


  • Clinical trial registration identifier: NCT00209261.

  • Disclosures Henri Lottmann has received honoraria from Ferring Pharmaceuticals for participating in clinical trials and symposia. He has no stock options or other equity interests. Frank Froeling, Schahnaz Alloussi, Abdul Sahib El-Radhi and Søren Rittig have no commercial relationship such as consultancies, stock ownership or other equity interests, patents received and/or pending, or any commercial relationship which might be in any way considered related to the submitted article. Schahnaz Alloussi is an Advisor of the Ferring Pharmaceuticals board of directors. Anders Riis and Bo-Eric Persson are full-time employees of Ferring Pharmaceuticals. This study was supported by a grant from Ferring Pharmaceuticals A/S.

Dr Bo-Eric Persson,
Ferring Pharmaceuticals A/S, International PharmaScience Center, Kay Fiskers Plads 11, DK-2300 Copenhagen S, Denmark
Tel.: + 45 88 33 88 34
Fax: + 45 88 33 88 80
Email: bo-eric.persson@ferring.com

Summary

Aims:  Desmopressin is a useful treatment for primary nocturnal enuresis (PNE), a common childhood condition that can persist into adolescence. This open-label, randomised, cross-over study evaluated the preference of children and adolescents with PNE for sublingual desmopressin oral lyophilisate (MELT) vs. tablet treatment, and the efficacy, safety, compliance and ease of use associated with each formulation. In total, 221 patients aged 5–15 years who were already receiving desmopressin tablets were randomised 1 : 1 to receive desmopressin treatment in the order MELT/tablet (n = 110) or tablet/MELT (n = 111) for 3 weeks each. Each formulation was administered in bioequivalent doses (0.2/0.4 mg tablets ≡ 120/240 μg MELT). Following treatment, patients were questioned regarding treatment preference. Diary card data and 100 mm Visual Analogue Scale scores were also recorded.

Results:  Overall, patients preferred the MELT formulation to the tablet (56% vs. 44%; p = 0.112). This preference was age dependent (p = 0.006); patients aged < 12 years had a statistically significant preference for desmopressin MELT (p = 0.0089). Efficacy was similar for both formulations (MELT: 1.88 ± 1.94 bedwetting episodes/week; tablet: 1.90 ± 1.85 episodes/week). Ease of use of both formulations was high. Compliance (≥ 80%) was 94.5% for MELT patients vs. 88.9% for the tablet (p = 0.059). No serious/severe adverse events were reported.

Conclusions:  There was an overall preference for the MELT, and a statistically significant preference for desmopressin MELT in children aged 5–11 years. Desmopressin MELT had similar levels of efficacy and safety at lower dosing levels than the tablet, and therefore facilitates early initiation of PNE treatment in children aged 5–6 years.

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