deamino-arginine-vasopressin and Genetic-Diseases--Inborn

To investigate predictive factors for the outcome of treatment of primary monosymptomatic nocturnal enuresis (PMNE) with desmopressin.. Data from a large open multicentre study were analysed. The study comprised 399 children with PMNE who were recruited for long-term desmopressin treatment. Before treatment a history was taken and the children observed for 4 weeks. After a 6-week dose-titration period with desmopressin, the children were classified into four groups depending on the response rate.. The children who improved during desmopressin treatment were older, had fewer wet nights during the observation period and had only one wet episode during the night, mostly after midnight. Many of them did not require the maximum dose of desmopressin to become dry. No hereditary factor for the response to desmopressin was found.. Those most likely to be permanently dry with desmopressin treatment are older children who respond to 20 microg desmopressin and who do not wet frequently.

Topics: Child; Deamino Arginine Vasopressin; Enuresis; Female; Genetic Diseases, Inborn; Humans; Male; Predictive Value of Tests; Renal Agents; Treatment Outcome

Tissue plasminogen activator (t-PA) and von Willebrand factor (vWF) are both released by vascular endothelial cells after the infusion of DDAVP. Such release has not been observed in patients with severe von Willebrand's disease (vWD). In the present work we demonstrate that the degree of simultaneous DDAVP-induced release of t-PA and vWF, in patients with vWD, is strictly related to the platelet vWF content. Twelve patients with type I, and three patients with type III vWD were studied. The type I vWD group included three patients with reduced platelet vWF content (platelet-low) and nine patients with normal levels (platelet-normal). In all patients studied the plasma t-PA levels were within the normal range. No significant change in either t-PA or vWF was observed after DDAVP in the patients with undetectable levels of platelet vWF (type III vWD). A mild increase was found in those patients with type I platelet-low, while in type I platelet-normal vWD the response was similar to that observed in normal subjects. The release of the two molecules appeared, therefore, to be linked to platelet vWF content and the rates of increase in both t-PA and vWF were similar in each group of patients studied. Since platelets are regarded as a tissue compartment of vWF our findings seem to suggest that the presence of vWF and its release from endothelial cells is required for a normal concomitant release of t-PA. In contrast, post-DDAVP release of vWF seems to be independent from that of t-PA since it was normal in a patient with congenital deficiency of t-PA release.

Topics: Adolescent; Adult; Antigens; Blood Platelets; Deamino Arginine Vasopressin; Factor VIII; Female; Genetic Diseases, Inborn; Humans; Male; Middle Aged; Tissue Plasminogen Activator; von Willebrand Diseases; von Willebrand Factor