Low-dose Desmopressin Research Articles

195 - Long-term safety and efficacy of low-dose desmopressin for nocturnal polyuria in men aged over eighty

195 - Long-term safety and efficacy of low-dose desmopressin for nocturnal polyuria in men aged over eighty

Investigation of the effects of intranasal desmopressin on the bleeding of the patients during open septorhinoplasty: A randomized double-blind clinical trial

ObjectivesRhinoplasty is one of the most common cosmetic surgeries in the world. Lack of adequate local homeostasis may lead to excessive bleeding during the operation, which increases the time of operation and recovery period, and the prevalence of complications. This study investigated the effects of nasal desmopressin on the quality of the surgical field and the volume of bleeding during rhinoplasty. Materials and methodsThis double-blind randomized clinical trial was performed on 120 patients aged 18–40 years who were candidates for rhinoplasty. Patients were randomly divided into three groups: low-dose desmopressin group and high-dose desmopressin group and placebo group. Hemodynamic changes and surgical field based on BOEZAART criteria, and the volume of bleeding were calculated. ResultsIn this study 115 women (95.8%) and 5 men (4.2%) participated. The mean age of patients was (27 ± 6.8). Bleeding volume in high dose desmopressin group was (21.7 cc ± 12.3), (27.7 cc ± 12.3) in low dose group, and (38.3 cc ± 12.3) in the placebo group, The difference in blood volume among the three groups was statistically significant with p < 0.005. Clean surgical field according to BOEZAART classification was marginally significant in both desmopressin groups. The differences in blood pressure, heart rate, blood and urine sodium, and hemoglobin before and after surgery between groups there not statistically significant. ConclusionBased on the results of the present study topical nasal spray desmopressin can reduce surgical field bleeding during rhinoplasty. To generalize the results to other surgeries in the ENT field it is recommended to conduct studies.

EFFICACY AND SAFETY OF INITIAL DESMOPRESSIN DOSE OF 50 μg IN ELDERLY MALE PATIENTS WITH NOCTURNAL POLYURIA

(Introduction) Low-dose desmopressin is now available for the treatment of nocturia associated with nocturnal polyuria in men, and its usefulness in a dose-dependent manner has been reported. Since side effects such as hyponatremia have reported frequently, the initial dose has been set at 25 μg in many cases considering age and other factors. In the present study, we investigated the efficacy and safety of an initial dose of 50 μg in elderly patients. (Subjects and methods) At Chikugo city hospital, 45 patients were started on desmopressin at an initial dose of 50 μg for nocturia with nocturnal polyuria. Efficacy and safety after one and four weeks were evaluated based on bladder (micturition) diary. The investigated parameters included frequency of nocturnal urination, nocturnal polyuria index, time to first nocturnal void, first nocturnal urine volume, nocturnal urine volume, International Prostate Symptom Score (IPSS), Overactive Bladder Symptom Score (OABSS), and Athens Insomnia Scale. Physical examinations were also carried out, along with blood tests. (Results) The mean age of the patients was 78.3 years, which was higher than that reported in prior studies. After one week of treatment, there was a significant decrease in the frequency of nocturnal urination and nocturnal urine volume, as well as a prolongation of the time to first nocturnal void, improvement in nocturnal polyuria index, and improvement in IPSS, IPSS-QOL, OABSS, and Athens Insomnia Scale. In terms of safety, adverse events were observed in eight patients (17.8%), and hyponatremia was observed in seven patients (15.6%), which was comparable to the findings of prior reports. (Conclusion) Good therapeutic results were obtained in elderly patients at an initial desmopressin dose of 50 μg, indicating that the drug could be safely administered to elderly patients with regular follow-ups and appropriate withdrawal and dose reductions.

Central diabetes insipidus and partial anterior pituitary dysfunction in acute myeloid leukemia.

SummaryCentral diabetes insipidus (CDI) is a rare manifestation of acute myeloid leukemia (AML) with unclear etiology. When present, CDI in AML has most often been described in patients with chromosome 3 or 7 aberrations and no abnormalities on brain imaging. In this case, we present a woman with newly diagnosed AML t(12;14)(p12;q13) found to have diabetes insipidus (DI) with partial anterior pituitary dysfunction and abnormal brain imaging. While in hospital, the patient developed an elevated serum sodium of 151 mmol/L with a serum osmolality of 323 mmol/kg and urine osmolality of 154 mmol/kg. On history, she reported polyuria and polydipsia for 5 months preceding hospitalization. Based on her clinical symptoms and biochemistry, she was diagnosed with DI and treated using intravenous desmopressin with good effect; sodium improved to 144 mmol/L with a serum osmolality of 302 mmol/kg and urine osmolality of 501 mmol/kg. An MRI of the brain done for the assessment of neurologic involvement revealed symmetric high-T2 signal within the hypothalamus extending into the mamillary bodies bilaterally, a partially empty sella, and loss of the pituitary bright spot. A pituitary panel was completed which suggested partial anterior pituitary dysfunction. The patient’s robust improvement with low-dose desmopressin therapy along with her imaging findings indicated a central rather than nephrogenic cause for her DI. Given the time course of her presentation with respect to her AML diagnosis, MRI findings, and investigations excluding other causes, her CDI and partial anterior pituitary dysfunction were suspected to be secondary to hypothalamic leukemic infiltration.Learning pointsLeukemic infiltration of the pituitary gland is a rare cause of central diabetes insipidus (CDI) in patients with acute myeloid leukemia (AML).Patients with AML and CDI may compensate for polyuria and prevent hypernatremia with increased water intake.AML-associated CDI can require long-term desmopressin treatment, independent of AML response to treatment.

Management of nocturia: overcoming the challenges of nocturnal polyuria.

Nocturia may be a multifactorial condition and should be regarded as a syndrome rather than a diagnosis, with many factors contributing to the clinical presentation. The effects of sleep deprivation can have a severely detrimental impact on the quality of life and productivity of the working age population, with considerable economic implications. Patients are unlikely to seek an appointment with their GP complaining of nocturia - they are more likely to complain of the effects of the condition, such as chronic tiredness, or injuries resulting from falls. The main criterion in deciding whether a patient should undergo further investigations into suspected nocturia is the degree to which the patient finds the condition bothersome. In some patients, lifestyle modifications may be an effective way to manage nocturia before medication is considered. As the only licensed product for all adults including those over 65 years of age, low dose desmopressin (Noqdirna® (as lyophilisate) Ferring Pharmaceuticals Ltd) is highly effective in the management of idiopathic nocturnal polyuria, producing improvements in clinical symptoms, sleep parameters and quality of life. Care should be administered as a joint enterprise between the patient's GP and colleagues in secondary care. This article outlines the findings of a roundtable discussion into the optimal management of patients with nocturnal polyuria.

Re: Low-Dose Desmopressin Orally Disintegrating Tablet: Suggested Clinically Meaningful Benefit in Patients with Nocturia Due to Nocturnal Polyuria.

Re: Low-Dose Desmopressin Orally Disintegrating Tablet: Suggested Clinically Meaningful Benefit in Patients with Nocturia Due to Nocturnal Polyuria.

Low-dose Desmopressin Orally Disintegrating Tablet: Suggested Clinically Meaningful Benefit in Patients with Nocturia Due to Nocturnal Polyuria

BackgroundClinical benefit has not been evaluated much in patients with nocturia. ObjectiveTo assess the clinical benefit of desmopressin orally disintegrating tablet (ODT) in women (25μg) and men (50μg) with nocturia due to nocturnal polyuria (NP). Design, setting, and patientsPatients with NP from two randomised, placebo-controlled trials in men (CS41) and women (CS40) with two or more nocturnal voids per night were included. Outcome measurements and statistical analysisChange from baseline in nocturnal voids, 33% and 50% responder status (average reduction of ≤33% and ≤50%, respectively, in the mean number of nocturnal voids vs baseline), and percentage of nights with at most one void or no voids (ie, complete response) during 3-mo treatment period were assessed for clinical benefit. Two-sided test (5% significance level) was used for all endpoints. Results and limitationsDemographics and baseline characteristics of patients in CS41 (N=230) and CS40 (N=232) were similar. A greater reduction in the mean number of nocturnal voids was seen with desmopressin ODT in men (treatment difference [TD]: −0.37 voids) compared with women (TD: −0.29 voids). For 33% and 50% responder status, TD with ODT versus placebo were 21% and 12%, respectively, in men, and 12% and 17%, respectively, in women. For the number of nights with at most one void, TDs were 11% and 13% (p<0.009 for both) for men and women, respectively. For complete response, TD was significant in men (TD: 9%, p<0.001). Limitations inherent in this analysis were evident as the data for cotreatments (baseline) and quality of life were not collected. ConclusionsA stronger treatment effect with desmopressin ODT versus placebo and the magnitude of differences are indicative of clinical benefit in patients with NP. Patient summaryWe looked at the clinical benefit of desmopressin ODT in patients with nocturnal polyuria. We conclude that clinical benefit was observed with desmopressin ODT in these patients.

Nocturia in men with benign prostatic hyperplasia

Nocturia, defined as nocturnal micturition with a frequency of at least once per night, is one of the most frequent lower urinary tract symptoms in men with benign prostatic hyperplasia (BPH) and often causes them to consult a physician. Nocturia is often bothersome and responsible for increased morbidity and mortality. Nocturia can be caused by increased fluid intake, increased diuresis or decreased bladder capacity, either alone or in combination. The underlying pathophysiology of nocturia can only be detected by methodical evaluation of the patient. Bladder diaries for 3 days are an essential part of the assessment. Treatment goals include reducing the nocturnal voiding frequency to less than 2 episodes per night, increasing the duration of undisturbed sleep to more than 4 hours, restoring quality of life, and reducing morbidity as well as mortality. In patients with reduced functional bladder capacity, α-blockers, 5α-reductase inhibitors, phosphodiesterase type-5 inhibitors, plant extracts or prostate operations (e. g. TURP) have shown to significantly reduce nocturnal voiding frequency. If nocturnal polyuria causes or contributes to nocturia, as shown in up to 80 % of BPH patients with nocturia, the treatment goal is to reduce urine production during the night. Low nocturnal serum concentration of the antidiuretic hormone can be treated with desmopressin to be taken at bedtime. The risk of hyponatremia is reduced with the new low-dose desmopressin formulation, which can be used even in men older than 65 years of age. Drug combinations may be useful in men with a mixed pathophysiology of nocturia.

A Danish population-based cohort study of desmopressin use in adults with nocturia or nocturnal enuresis.

This study investigated how desmopressin is prescribed to adults in Denmark. All adult users of desmopressin over an 8-year period were identified from the Danish National Prescription Registry. Adult patients with nocturia or nocturnal enuresis (NE) were identified by indication codes for "frequent nocturnal voiding" or "involuntary nocturnal voiding", respectively. Patient demographics, desmopressin formulation and dose, and concomitant medication were investigated. In all, 13 871 adults with nocturia and 2872 adults with chronic (i.e. >10 prescriptions) NE were given 102 547 and 43 712 desmopressin prescriptions, respectively. Across the entire patient cohort, 57% were women and mean patient age was 62 years. Over 40% of prescriptions were to elderly patients (≥65 years), and desmopressin use for adult enuresis increased with age. Orally disintegrating tablets were the most frequently used formulation (57%-65% of prescriptions), and a greater proportion of women than men used low-dose desmopressin (60 μg). Concomitant use of painkillers (opioids: 18%-26.7% of prescriptions; non-steroidal anti-inflammatory drugs: 14.2%-16.4% of prescriptions) and antidepressants (14.4%-18.1% of prescriptions) was common in both conditions, and 5.4%-9.2% of concomitant prescriptions were for overactive bladder medications. This study provides insights into desmopressin use among Danish adults. Nearly half the prescriptions were to patients aged ≥65 years, despite historical manufacturer recommendations that desmopressin be restricted to patients <65 years of age. NE is considered a childhood condition, but desmopressin use for adult NE increased with age. A greater proportion of desmopressin prescriptions to women than men were for the lowest dose, consistent with greater sensitivity to desmopressin in women.

Corrigendum

Antoine Kass-Iliyya and Hashim Hashim. Nocturnal Polyuria: Literature review of Definition, Pathophysiology, Investigations and Treatment. Journal of Clinical Urology 2018. Epub ahead of print 22 February 2018 (Original doi: 10.1177/2051415818756792). The following correction note has been added to this article: At time of publication, low dose desmopressin (Noqdirna) was approved for use in patients with nocturia due to nocturnal polyuria.

Low-dose Desmopressin and Tolterodine Combination Therapy for Treating Nocturia in Women with Overactive Bladder: A Double-blind, Randomized, Controlled Study.

Evaluation of safety and efficacy of desmopressin/tolterodine combination therapy in women. This double-blind, randomized, proof-of-concept study enrolled 106 patients (≥18 years), with overactive bladder (OAB) and nocturia, with ≥2 nocturnal voids, receiving a 3-month once-daily combination (desmopressin 25 µg, orally-disintegrating tablets [ODT]/tolterodine 4 mg [Detrol® LA]; n = 49) or monotherapy (tolterodine 4 mg/placebo ODT; n = 57). Primary endpoint was change from baseline in mean number of nocturnal voids. Secondary endpoints were change from baseline in nocturnal voided volume, time to first nocturnal void, and quality-of-life. Post-hoc exploratory analysis were performed for patients with and without baseline nocturnal polyuria (NP, n = 47 each). Overall population showed a non-significant reduction in mean number of nocturnal voids with combination versus monotherapy (full analysis set: adjusted treatment contrast [TC], -0.34; P = 0.112). Change in mean nocturnal void volume (TC, -64.16 mL; P = 0.103), mean time to first nocturnal void (TC, 18.00 min; P = 0.385) and Nocturia Impact (NI) Diary© scores were comparable. In post-hoc analysis, NP patients showed a benefit with combination versus monotherapy for nocturnal void volume (P = 0.034) and time to first nocturnal void (P = 0.045), and a non-significant improvement in NI Diary© scores. Safety profile was comparable between treatments. A single transient event of asymptomatic clinically significant hyponatremia in combination group resolved subsequently. Low-dose desmopressin could be safely combined with tolterodine for treating nocturia in women with OAB, with a significant benefit in women with NP. Further, prospective validation studies of combination therapy are warranted in mixed NP/OAB population, based on this favorable proof-of-concept finding.

MP31-01 LONG-TERM SAFETY AND EFFICACY OF SER120 1.5 MCG IN PATIENTS WITH NOCTURIA: RESULTS FROM A 2-YEAR OPEN-LABEL EXTENSION STUDY

You have accessJournal of UrologyUrodynamics/Lower Urinary Tract Dysfunction/Female Pelvic Medicine: Non-neurogenic Voiding Dysfunction I1 Apr 2017MP31-01 LONG-TERM SAFETY AND EFFICACY OF SER120 1.5 MCG IN PATIENTS WITH NOCTURIA: RESULTS FROM A 2-YEAR OPEN-LABEL EXTENSION STUDY Scott MacDiarmid, J.P. Nicandro, Maria Cheng, Steven Abrams, Seymour Fein, and Alan Wein Scott MacDiarmidScott MacDiarmid More articles by this author , J.P. NicandroJ.P. Nicandro More articles by this author , Maria ChengMaria Cheng More articles by this author , Steven AbramsSteven Abrams More articles by this author , Seymour FeinSeymour Fein More articles by this author , and Alan WeinAlan Wein More articles by this author View All Author Informationhttps://doi.org/10.1016/j.juro.2017.02.955AboutPDF ToolsAdd to favoritesDownload CitationsTrack CitationsPermissionsReprints ShareFacebookTwitterLinked InEmail INTRODUCTION AND OBJECTIVES Two randomized, placebo-controlled phase 3 trials (DB3 and DB4) demonstrated the efficacy/safety of SER120 1.5 mcg, a very low-dose desmopressin nasal spray formulation in patients with nocturia. An open-label extension (OLE) trial of the DB3 study examined the long-term safety and efficacy of SER120 1.5 mcg over a 2-year period. METHODS The DB3 study enrolled patients ≥50 years of age with ≥2 nocturic episodes/night for at least 6 months. Patients could enroll in the OLE study and receive treatment with SER120 at a lower dose and escalate to the 1.5 mcg dose for up to 126 weeks if they (1) completed the DB3 study or (2) did not participate in the DB3 study but met the DB3 study screening criteria. Patients returned for study visits every 2-8 weeks for assessments. Safety and efficacy assessments were conducted in the intent-to-treat (ITT) population (N=393) and included the incidence of low serum sodium levels, mean serum sodium levels, adverse events (AEs), duration of treatment exposure, and mean change from baseline in nocturic episodes/night. RESULTS Of the 393 patients in the ITT population, 358 received the SER120 1.5 mcg dose. At baseline, mean age was 65.7 years, and mean nocturic episodes/night was 3.3. A total of 217, 129, and 42 patients, respectively, had at least 12 months, 18 months and 24 months of exposure to the SER120 1.5 mcg dose. Significant decreases from baseline were observed in the mean nocturic episodes/night as early as week 2 (-1.3), which were sustained up to week 54 (-1.9), week 78 (-1.9), and week 110 (-2.0) (P<.0001 for all timepoints). During the 2-year treatment period, the most commonly reported AEs were nasal discomfort (24.9%), sneezing (10.2%), and rhinorrhea (9.9%), which were mostly mild or moderate in intensity. The mean serum sodium concentration at baseline was 139.7 mmol/L and remained within the normal limits over the entire treatment period. None of the patients reported hyponatremia (serum sodium levels ≤125 mmol/L) at the SER120 1.5 mcg dose for the duration of the study. CONCLUSIONS SER120 1.5 mcg was well tolerated over the long-term, with no new safety signals observed during the OLE period. A total of 217 patients reached at least 1 year of exposure with the SER120 1.5 mcg dose. Long-term treatment with SER120 1.5 mcg resulted in significant and durable improvements in nocturic episodes/night which were sustained over 2 years. © 2017FiguresReferencesRelatedDetails Volume 197Issue 4SApril 2017Page: e398 Advertisement Copyright & Permissions© 2017MetricsAuthor Information Scott MacDiarmid More articles by this author J.P. Nicandro More articles by this author Maria Cheng More articles by this author Steven Abrams More articles by this author Seymour Fein More articles by this author Alan Wein More articles by this author Expand All Advertisement Advertisement PDF downloadLoading ...

537 Nocturia due to nocturnal polyuria (NP) in women with overactive bladder (OAB) may be better managed by adding a low-dose desmopressin to tolterodine therapy

537 Nocturia due to nocturnal polyuria (NP) in women with overactive bladder (OAB) may be better managed by adding a low-dose desmopressin to tolterodine therapy

The effectiveness of low-dose desmopressin in improving hypothermia-induced impairment of primary haemostasis under influence of aspirin - a randomized controlled trial.

BackgroundMild hypothermia (34–35 °C) increases perioperative blood loss. Our previous studies showed that desmopressin could have in vitro beneficial effects on hypothermia-induced primary haemostasis impairment. In this study, we investigate the in vitro effects of desmopressin on hypothermia-induced primary haemostasis impairment under the influence of aspirin in healthy volunteers.MethodsSixty healthy volunteers were randomly allocated to taking aspirin 100 mg or placebo for three days. On the sixth day blood samples were taken before and after the injection of desmopressin (1.5 microgram or 5 microgram) or normal saline subcutaneously. Measurements including Platelet Function Analyzer (PFA-100®) closure times, plasma von Willebrand Factor antigen, haemoglobin and platelet levels were made at 32 °C and 37 °C respectively.ResultsCollagen/epinephrine closure time (EPICT) was significantly prolonged by 21.13 % (95 %CI 2.34–39.74 %, p = 0.021) in aspirin group at 37 °C. While hypothermia alone prolonged both collagen/adenosine diphosphate (ADPCT) and EPICT by 17.63 % (95 %CI 13.5–20.85 %, p < 0.001) and 8.0 % (95 %CI 6.38–10.04 %, p = 0.024) respectively, addition of aspirin only further prolonged EPICT by 19.9 % (95 %CI 3.32–36.49 %, p = 0.013). In aspirin group, desmopressin 1.5 microgram and 5 microgram significantly reduced ADPCT to below baseline levels at 37 °C (p = 0.025 and <0.001 respectively), whereas reduction in EPICT was seen with desmopressin 5 microgram (p =0.008). The effect was less pronounced at 32 °C, with a significant reduction in EPICT obtained with a dosage of 5 microgram only (p = 0.011).ConclusionIt was shown that aspirin could further potentiate the hypothermia-induced closure time prolongations. Low dose desmopressin (1.5 microgram) reduced PFA-100® closure times towards baseline. A higher dosage (5 microgram) further reduced the closure times below baseline. Therefore low dose desmopressin (1.5 microgram) might have the potential to correct hypothermia-induced primary haemostasis impairment under the influence of aspirin during the perioperative period.Trial registrationClinicalTrials.gov: NCT01382134

Management of nocturia: the role of antidiuretic pharmacotherapy.

Strategies to manage nocturia include lifestyle modifications and treatment with alpha-blockers, antimuscarinic therapies, and antidiuretics. The concept of achieving success should not be limited to reduction of nighttime voids; it should ideally include proof of improvement of conditions generally associated with nocturia, such as falls, quality of life, and overall health. Few studies have looked specifically at parameters other than nocturnal voids, such as sleep latency, first undisturbed sleep period (FUSP), and total sleep time, including their clinical relevance to patient well-being. Lifestyle modifications, such as voiding before bedtime, limiting caffeine and alcohol, and adjusting medication timing, may be initially effective in mild cases of nocturia. Statistically significant reductions in voiding have been reported with antimuscarinic agents and alpha-blockers as initial therapy, but these reductions generally are not clinically relevant. The antidiuretic therapy desmopressin acetate, a selective vasopressin receptor 2 agonist, is effective in adults with nocturia associated with nocturnal polyuria; however, hyponatremia can occur. The newest formulation-desmopressin orally disintegrating sublingual tablet (ODST)--has greater bioavailability; thus, lower doses can be used, potentially reducing hyponatremia risk. A phase 3 study demonstrated statistically significant reductions in nocturnal voids for desmopressin ODST 50 and 100 µg versus placebo (-1.18 and -1.43 vs. -0.86; P = 0.02 and P < 0.0001, respectively) in patients with nocturia. Treatment was well-tolerated, and low-dose desmopressin ODST was associated with statistically significant increases in duration of FUSP. Development of a validated composite endpoint may help clinicians identify and compare strategies for treating nocturia.

Preliminary Experience with Low-dose Desmopressin Treatment and Urinary Arginine Vasopressin Concentration as a Response Predictor for Patients with Nocturia with Nocturnal Polyuria.

To assess whether urinary arginine vasopressin (AVP) concentrations adjusted for urinary creatinine (Cr) can predict responsiveness to low-dose desmopressin for nocturia with nocturnal polyuria (NP). Nine NP patients aged 64-84 years with ≥ 2 nocturnal voids received 2.5 µg of intranasal desmopressin for 4 weeks and were evaluated for its effectiveness. Prior to treatment, urinary AVP/Cr concentrations at first morning void and serum sodium and plasma natriuretic peptide (BNP) concentrations were measured, and all patients underwent 5% hypertonic saline infusion. Six responders to 2.5 µg desmopressin had average decreases in nocturnal frequency from 3.2 to 1.8 voids nightly and in nocturnal diuresis by 40%, without clinically significant adverse events. Non-responders remained unaffected even with dose escalation to 5.0 µg desmopressin. For responders, urinary AVP/Cr concentrations were less than 14 pg/mg · Cr, which is lower than in non-responders. Within a physiological range of plasma osmolality, plasma AVP release in response to 5% hypertonic saline infusion was less in responders than in non-responders. Blood pressure measurements and BNP concentrations were unchanged. Urinary AVP/Cr at first morning void may have potential clinical value as a predictor for responsiveness to low-dose desmopressin in nocturia with NP.

Response to low-dose desmopressin by a subcutaneous route in children with type 1 von Willebrand disease

ObjectiveThe primary objective of this study was to determine responses to low-dose desmopressin (DDAVP) by a subcutaneous route in children with type 1 VWD.MethodsThis study analyzed responses to low doses of DDAVP administered by a subcutaneous route to 14 children between the ages of 3 and 16 with type 1 VWD and a personal and familial history of bleeding. At 0 (baseline) and 1 hour (initial response) after the subcutaneous injection of DDAVP, the vital signs were assessed and blood samples were obtained for VWD panel determinations (VWF:Ag, VWF:RCo, FVIII:C levels, and Col/Epi, Col/ADP). At 4 hours (sustained response), only Col/Epi and Col/ADP were assessed.ResultsThe DDAVP mean (min–max range, μg/kg) based on the patient's weight was 0.15 (0.12–0.18). Laboratory values mean (min–max range in U/dl) baseline for VWF:RCo, VWF:Ag, and FVIII:C were 28 (20–36), 34 (25–42), and 40 (29–48), respectively. After a subcutaneous administration, the laboratory values mean (min–max range in U/dl−1) achieved for 1 hour for VWF:RCo, VWF:Ag, and FVIII:C were 109 (72–144), 132 (88–166), and 151 (96–198), respectively.PFA 100® CT (Col/Epi <134 seconds and Col/ADP <110 seconds) returned to normal values at 1 and 4 hours after a subcutaneous administration.ConclusionSubcutaneous low-dose DDAVP therapy is at least effective as 0.3 µg/kg intravenous therapy for children with type 1 VWD. This study shows that a wider use of DDAVP should be promoted, especially in developing countries.

Efficacy and Safety of Low Dose Desmopressin Orally Disintegrating Tablet in Women with Nocturia: Results of a Multicenter, Randomized, Double-Blind, Placebo Controlled, Parallel Group Study

Previous studies suggest a lower dose of desmopressin orally disintegrating tablet may be effective in females compared to males with nocturia. We confirm the efficacy and safety of 25 μg desmopressin orally disintegrating tablet compared to placebo in female patients. In this 3-month, randomized, double-blind, parallel group study 25 μg desmopressin once daily was compared to placebo in women with nocturia (2 or more nocturnal voids). The co-primary efficacy end points were change from baseline in mean number of nocturnal voids and proportion of patients achieving at least a 33% reduction from baseline in the mean number of nocturnal voids (33% responders). The full analysis set comprised 261 patients (age range 19 to 87 years). Desmopressin significantly reduced the mean number of nocturnal voids and increased the odds of a 33% or greater response compared to placebo during 3 months, assessed by longitudinal analysis (-0.22, p = 0.028 and OR 1.85, p = 0.006, respectively). Desmopressin increased the mean time to first nocturnal void by 49 minutes compared to placebo at 3 months (p = 0.003). The response to desmopressin was seen by week 1 of treatment and was sustained throughout the trial. Significant increases in health related quality of life and sleep quality were observed compared to placebo. Desmopressin was well tolerated. Serum sodium levels remained greater than 125 mmol/L throughout the trial and 3 transient decreases to less than 130 mmol/L were recorded. At a dose of 25 μg, desmopressin orally disintegrating tablet is an effective and well tolerated treatment for women with nocturia. Treatment provides rapid and sustained improvement in nocturia and quality of life.

Efficacy and Safety of Low Dose Desmopressin Orally Disintegrating Tablet in Men with Nocturia: Results of a Multicenter, Randomized, Double-Blind, Placebo Controlled, Parallel Group Study

We investigated the efficacy and safety of 50 and 75 μg desmopressin orally disintegrating tablets in men with nocturia (2 or more nocturnal voids). In this 3-month, randomized, double-blind, parallel study 50 and 75 μg desmopressin were compared with placebo. The co-primary efficacy end points were changes from baseline in mean number of nocturnal voids and proportions of patients achieving at least a 33% reduction from baseline in nocturnal voids (33% responders) during a 3-month treatment period. The full analysis set comprised 385 men (age range 20 to 87 years). The 50 and 75 μg doses significantly reduced the number of nocturnal voids (-0.37, p <0.0001 and -0.41, p = 0.0003, respectively) and increased the odds of a 33% or greater response (OR 1.98, p = 0.0009 and OR 2.04, p = 0.0004, respectively) compared with placebo during 3 months. Desmopressin 50 and 75 μg increased the time to first void from baseline by approximately 40 minutes compared to placebo (p = 0.006 and p = 0.003, respectively). The response to desmopressin was seen by 1 week of treatment and was sustained. Significant increases in health related quality of life and sleep quality were observed compared to placebo. Desmopressin was well tolerated as only 2 subjects (age 74 and 79 years) on 50 μg had a serum sodium level of less than 130 mmol/L (vs 9 subjects on 75 μg). Desmopressin (orally disintegrating tablet) is an effective and well tolerated treatment for men with nocturia. Treatment with 50 μg desmopressin, the minimum effective dose, provided sustained improvement of nocturia throughout the study and meaningful benefits to patients with an improved safety profile.

Low-dose desmopressin improves hypothermia-induced impairment of primary haemostasis in healthy volunteers

Low-dose desmopressin improves hypothermia-induced impairment of primary haemostasis in healthy volunteers