desmopressin / Generic mfg. - LARVOL DELTA

Velora pioneer, Phase 1/2 study to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of HMB-002 for prophylaxis in von Willebrand disease: A trial in progress (ASH 2025) - P, P1/2 | "Desmopressin provides only transient benefit and is limited by tachyphylaxis, while plasma-derived or recombinant VWF/FVIII concentrates require frequent, burdensome intravenous infusions. Documented residual VWF levels are required, and exclusion criteria include concurrent participation in interventional trials or medical conditions confounding study results. Part B requires participants to have completed the VELORA Discover observational study."

Clinical • P1/2 data • PK/PD data • Hematological Disorders • Hemophilia

The disguise of dyscrasia: Hereditary von Willebrand disease unmasked by MGUS-associated acquired VWD (ASH 2025) - "Patient received DDAVP challenge with repeat Von Willebrand panel at 0, 4, and 8 hours... This case underscores the complex interplay between MGUS and bleeding disorders, where acquired VWD may mimic or reveal underlying hereditary VWD. The patient's diagnosis of Type 1 Hereditary VWD, uncovered during evaluation for spontaneous hemorrhage and initially suspected as acquired VWD from MGUS, illustrates the importance of thorough hematologic assessment and familial testing. Recognition of hereditary bleeding disorders in the setting of monoclonal gammopathies is essential for accurate diagnosis and tailored management."

Cerebral Hemorrhage • CNS Disorders • Hematological Disorders • Hematological Malignancies • Hemophilia • Monoclonal Gammopathy • Multiple Myeloma • Musculoskeletal Diseases • Musculoskeletal Pain • Orthopedics • Subarachnoid Hemorrhage

Labile heparin response in a pre-operative cardiac surgery patient: Unmasking predisposition for post-operative coagulopathy (ASH 2025) - "High-risk patients undergoing cardiac surgery will require IV heparin prior to the procedure...The blood bank can be notified to prepare blood products such as FFP, cryoglobulin or desmopressin which can take longer to obtain in large quantities. This should lead to further evaluation into medications, toxins or biologic causes for the labile response to heparin which may mitigate adverse outcomes. Although the incidence of heparin resistance is rare, performing a clinical study in the future may determine if some of these interventions would be beneficial to patient care."

Clinical • Surgery • Addiction (Opioid and Alcohol) • Atherosclerosis • Cardiovascular • Coronary Artery Disease • Genito-urinary Cancer • Hepatology • Hypotension • Liver Failure • Solid Tumor • Testicular Cancer • Thrombocytopenia • Thrombocytopenic Purpura

Elevated aPTT in the setting of prophylactic unfractionated subcutaneous heparin administration (ASH 2025) - "Therefore, the patient was given intravenous protamine sulfate 50 mg once and received intravenous 0.6 mcg of desmopressin for concerns of uremic bleeding. Critically ill patients are predisposed to a higher baseline bleeding risk due to factors such as platelet dysfunction, coagulation factor deficiencies, more invasive procedures, and mechanical ventilation. Understanding all these factors is a crucial part of decision-making when administering anticoagulation."

Cardiovascular • Chronic Kidney Disease • CNS Disorders • Diabetes • Diabetic Nephropathy • Dyslipidemia • Heart Failure • Hematological Disorders • Hypertension • Hypotension • Metabolic Disorders • Nephrology • Renal Disease • Type 1 Diabetes Mellitus • Venous Thromboembolism

Silent hemorrhage: Unraveling the insidious rectal bleeding in hermansky-pudlak syndrome (ASH 2025) - "Anti-fibrinolytics, such as aminocaproic acid and tranexamic acid, can decrease bleeding. Perioperatively, HPS patients should receive desmopressin acetate (DDAVP), but responsiveness testing should be conducted prior, as not all affected respond...Screening patients with hypopigmentation and bleeding disorders may be beneficial and appropriate. Identification would assist in prognosis as well as prophylaxis against severe bleeding."

Dyslipidemia • Endocrine Disorders • Gastroenterology • Gastrointestinal Disorder • Gynecology • Hematological Disorders • Hemophilia • Immunology • Metabolic Disorders • Rare Diseases • Thrombosis

Risk of postpartum hemorrhage in women with von willebrand disease by treatment type: An analysis of real-world data (ASH 2025) - "Whileprophylactic therapy (e.g., desmopressin or von Willebrand factor [VWF] concentrates) is recommendedfor individuals with low VWF levels, practice varies nationally, and the effectiveness of these strategies inroutine care remains unclear... Women with VWD face elevated risks of PPH and hospitalization in the peripartum period. These findingsconfirm prior literature while quantifying risks in a contemporary U.S. population. Among treatedpatients, those receiving VWF concentrates had the highest odds of PPH, likely reflecting triage ofindividuals perceived to be at elevated hemorrhagic risk."

Clinical • Real-world • Real-world evidence • Genetic Disorders • Hematological Disorders • Hemophilia • Obesity • Postpartum Hemorrhage

A 10-year retrospective review of the effectiveness and safety of tranexamic acid in women with inherited bleeding disorders in the peripartum setting (ASH 2025) - "31% patients also received an additional hemostatic agent (17% received DDAVP, 15% receivedfactor concentrate -VWF:FVIII, FXI, FVIII, rFVIIa and FIX). This data suggests that TXA is associated with very low risk of thrombosis when used asprophylactic treatment for PPH among patients with inherited bleeding disorders in the puerperium.Under 1% of patients over 10 years experienced a thrombotic event and had two or more risk factors forVTE. In contrast, at least 17% had PPH despite provision of preventative peripartum hemostatic therapythat almost always included TXA. This suggests that on balance, TXA should be provided to patients withinherited bleeding disorders with the goal of PPH prevention but caution should be used in those with 2or more risk factors for thrombosis."

Retrospective data • Review • Cardiovascular • Hematological Disorders • Hemophilia • Hemophilia A • Hemophilia B • Obesity • Postpartum Hemorrhage • Pulmonary Embolism • Rare Diseases • Respiratory Diseases • Varicose Veins

A retrospective analysis of the safety and effectiveness of recombinant von willebrand factor in children with von willebrand disease undergoing surgery (ASH 2025) - P3 | "There were no reports of perioperative FVIII,tranexamic acid, desmopressin, anemia treatment, or need for transfusion. Results from this study suggest that off-label use of rVWF is effective for the perioperativemanagement of surgical bleeding in pediatric individuals with VWD undergoing major and minorprocedures, including those with severe disease. Results from this study suggest that off-label use of rVWF is effective for the perioperativemanagement of surgical bleeding in pediatric individuals with VWD undergoing major and minorprocedures, including those with severe disease. No new safety events were observed. These results lendfurther support for the positive benefit-risk profile for rVWF in a pediatric population."

Retrospective data • Surgery • Cardiovascular • Hematological Disorders • Hemophilia • Immunology • Rare Diseases • Thrombosis

Blood group o and a lower endogenous thrombin potential are associated with increased risk for future bleeding after hemostatic challenges in bleeding disorder of unknown cause (ASH 2025) - "In thisstudy, we investigated the association between prolonged bleeding or bleeding-related complicationsafter surgery or dental extractions and relevant coagulation-related laboratory parameters (hemoglobin,platelet count, fibrinogen, von Willebrand factor antigen and activity, C-reactive protein, thrombin andplasmin generation assays (all parameters) and native rotational thromboelastometry (clotting time, clotformation time, mean clot firmness, maximum lysis and area under the curve, AUC), measured at studyinclusion, by calculating univariate logistic regression models adjusted for age, sex, blood group O andthe use of bleeding prophylaxis, such as desmopressin or tranexamic acid.Results. Importantly, we couldshow an association between the endogenous thrombin generation potential, measured using acommercially available thrombin generation assay, and bleeding after hemostatic challenges in patientsafter the BDUC diagnosis. Lastly, our data highlights the importance..."

Hematological Disorders • CRP

Evaluation of a newly formulated intranasal desmopressin versus intranasal and subcutaneous desmopressin in individuals with type 1 von Willebrand disease and mild Hemophilia A (ASH 2025) - "Thediscontinuation of intranasal desmopressin acetate (Stimate) production in 2020 reduced treatmentoptions for these patients. Using the ASH ISTH NHF WFH criteria for desmopressin responsiveness and a non-inferiority margin of 15%, newly formulated intranasal desmopressin is non-inferior to the discontinuedintranasal desmopressin acetate or subcutaneous desmopressin in type 1 VWD. These findings supportthe use of newly formulated desmopressin to manage bleeding in type 1 VWD and mild hemophilia A.Expanded availability of newly formulated intranasal desmopressin may improve access to safe andefficacious treatment worldwide."

Clinical • Cardiovascular • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases • Thrombosis

DP11 A case of Langerhans cell histiocytosis masquerading as lymphocytic hypophysitis and panhypopituitarism. (PubMed, Br J Dermatol) - "The patient was initiated on replacement therapy for panhypopituitarism, including hydrocortisone, desmopressin, thyroxine and gonadotropin. Treatment with 12 cycles of single-agent cytarabine achieved complete remission. However, during follow-up, the patient experienced a limited recurrence of cutaneous lesions, which was managed effectively with cycles of high-dose methotrexate...The complexity of this rare condition emphasizes the need for a multidisciplinary approach to manage its aggressive course. Early diagnosis and treatment are crucial, especially in cases affecting high-risk end organs."

Journal • Endocrine Disorders • Hematological Disorders • Langerhans Cell Histiocytosis • Metabolic Disorders • Rare Diseases • CCND1 • CD1a • CD20 • CD68 • TNFRSF8 • TP63

Strategies to reduce hyponatraemia risk in desmopressin ODT therapy. (PubMed, Glob Epidemiol) - No abstract available

Journal

Haemostasis and beyond: The expanding role of desmopressin in intensive care. (PubMed, World J Crit Care Med) - "However, despite its mechanistic appeal and broad pharmacologic utility, the full scope of desmopressin's applications in the intensive care unit (ICU) remains underrecognized. This review aims to provide a comprehensive examination of desmopressin's pharmacological characteristics, evidence-based indications in critically ill patients, therapeutic efficacy, safety profile, and practical considerations for dosing in the ICU setting."

Journal • Review • Cardiovascular • Cerebral Hemorrhage • CNS Disorders • Critical care • Hematological Disorders • Hemophilia • Hemophilia A • Metabolic Disorders • Mood Disorders • Rare Diseases

Safety and efficacy of vibegron in pediatric patients with treatment-resistant nocturnal enuresis: a multicenter retrospective study. (PubMed, Transl Pediatr) - "Although enuresis alarms and desmopressin are the first-line treatments recommended by clinical guidelines, 20-40% of affected children demonstrate inadequate response and require additional therapeutic interventions. Add-on strategies-particularly triple therapy-were more effective than monotherapy switching, strengthening the incorporation of vibegron as part of multimodal treatment strategies. Given the retrospective nature of the study, prospective randomized trials are warranted to confirm these findings and optimize the treatment protocols."

Journal • Retrospective data • Pediatrics • Urinary Incontinence • Urology

Arginine Vasopressin Deficiency and Oxytocin Deficiency in the Endocrine Clinic. (PubMed, J Clin Endocrinol Metab) - "Treatment relies on desmopressin and careful education to prevent both dehydration and hyponatremia...Diagnosis remains challenging due to unreliable basal OXT levels and limited stimulation tests; novel approaches, including 3,4-methylenedioxymethamphetamine (MDMA) challenge and neurophysin I as a surrogate marker, are under investigation. Preliminary studies suggest intranasal OXT may improve socio-emotional outcomes, but robust evidence from randomized controlled trials is needed."

Journal • Cardiovascular • Heart Failure • Immunology • Metabolic Disorders • Oncology • Psychiatry • OXT

Webb-Dattani syndrome in a 17-year-old girl. (PubMed, Endocrinol Diabetes Metab Case Rep) - "Management included hormone replacement with hydrocortisone, levothyroxine, and desmopressin, as well as fluid regulation and supportive care. Long-term survival, although rare, is possible with coordinated multidisciplinary care addressing endocrine, neurological, renal, and infectious complications. This case underscores the importance of individualized endocrine replacement therapy and caregiver education in managing complex syndromic conditions such as WEDAS."

Journal • Developmental Disorders • Endocrine Disorders • Growth Hormone Deficiency • Infectious Disease • Metabolic Disorders

Giant Sellar Meningocele: Intact Pituitary Function and New-onset Arginine Vasopressin Deficiency After Surgery. (PubMed, JCEM Case Rep) - "Within 24 hours, he developed arginine vasopressin (AVP) deficiency requiring long-term desmopressin therapy, which persisted beyond 6 months, fulfilling the criteria for permanent AVP deficiency...This case underscores that adult sellar meningoceles can present with intact anterior pituitary function but still carry a risk of isolated permanent AVP deficiency after surgical repair. To our knowledge, this is among the largest adult sellar meningoceles reported with preserved anterior pituitary function at presentation and subsequent permanent isolated AVP deficiency, emphasizing the need for careful perioperative counseling and long-term endocrine follow-up."

Journal • Endocrine Cancer • Metabolic Disorders • Ophthalmology • Pain

Ultrasound-Guided Jugular Vein Access for Inferior Petrosal Sinus Sampling: A Safe and Feasible Technique. (PubMed, Cureus) - "Despite normal urinary free cortisol (UFC) levels (likely influenced by renal dysfunction), clinical suspicion prompted further testing, which revealed an inverted cortisol rhythm and lack of suppression on low-dose dexamethasone...A central-to-peripheral ACTH gradient >2 at baseline and >3 after desmopressin confirmed a pituitary source...This case demonstrates the feasibility and safety of combining femoral and direct jugular access to complete IPSS when conventional approaches are limited. This is the first reported case of IPSS performed using a hybrid right femoral and left ultrasound-guided jugular approach, offering a practical alternative when femoral access is not feasible and reinforcing the diagnostic value of IPSS in challenging cases."

Journal • Cardiovascular • Chronic Kidney Disease • Cushing’s Disease • Diabetes • Endocrine Disorders • Genetic Disorders • Hypertension • Metabolic Disorders • Nephrology • Obesity • Pituitary Gland Carcinoma • Renal Disease

Buckle up! Managing surgery in patients with bleeding disorder of unknown cause. (PubMed, Hematology Am Soc Hematol Educ Program) - "Extrapolating from the management of phenotypically similar bleeding disorders, a therapeutic approach may include antifibrinolytic agents (tranexamic acid), desmopressin, and/or platelet transfusion. Education of both patients and providers is central throughout the hemostatic planning process. Primary research in BDUC remains limited; future multisite observational and therapeutic clinical trials in BDUC may help us better understand multifactorial bleeding risk and further define the role of hemostatic therapies."

Journal • Review • Hematological Disorders

Pregnancy management for patients with bleeding disorders. (PubMed, Hematology Am Soc Hematol Educ Program) - "Hemostatic therapies include antifibrinolytic agents, desmopressin, factor concentrates, and blood components such as cryoprecipitate, plasma, and platelets. Antifibrinolytics such as tranexamic acid have the most robust evidence for PPH management, though factor concentrates are now routinely utilized in certain circumstances...We favor selecting the mode of delivery based upon maternal indications whenever possible. Post partum, therapies may be continued for days or sometimes weeks after delivery, as pwBD are at high risk for delayed PPH."

Journal • Review • Anesthesia • Hematological Disorders • Hemophilia • Obstetrics • Postpartum Hemorrhage • Rare Diseases

Treatment and outcome of a boy with lgG4-related hypophysitis caused by SARS-CoV-2 re-infection. (PubMed, Front Endocrinol (Lausanne)) - "Treatment with glucocorticoids and desmopressin led to significant resolution of symptoms and normalization of pituitary morphology...We successfully controlled clinical symptoms, but further follow-up observations are needed to assess recovery. Although the role of SARS-CoV-2 infection in this patient's condition is only suggestive, other reports have described a relationship between SARS-CoV-2 infection and lgG4-related hypophysitis."

Journal • Endocrine Disorders • Growth Hormone Deficiency • Infectious Disease • Inflammation • Metabolic Disorders • Novel Coronavirus Disease • Respiratory Diseases • Urinary Incontinence

The Role of Desmopressin in Kidney Biopsies: A Narrative Review. (PubMed, Can J Kidney Health Dis) - "More studies are still needed, but we believe desmopressin can be justified to reduce bleeding complications of kidney biopsy in the setting of chronic kidney disease (CKD). Trials involved small sample sizes, single-center data, and were largely observational in nature."

Journal • Review • Chronic Kidney Disease • Nephrology • Renal Disease

Use of recombinant activated factor VII to stop post-kidney biopsy bleeding in a child. (PubMed, Pediatr Nephrol) - "Following a kidney biopsy, the patient experienced a large, expanding hematoma with active bleeding that did not improve despite pre-biopsy desmopressin, 90 min of pressure to the biopsy site, and platelet transfusions. Recombinant activated FVIIa was administered, and the bleeding immediately stopped. This case supports the use of recombinant FVIIa in post-kidney biopsy hemorrhage when bleeding is not controlled using conventional strategies."

Journal • Acute Kidney Injury • ANCA Vasculitis • Cardiovascular • Hematological Disorders • Hypertension • Nephrology • Pediatrics • Renal Disease • Thrombocytopenia • Vasculitis

Antiangiogenic Treatment of Patients with Hereditary Hemorrhagic Telangiectasia: Experience of a Hungarian Center. (PubMed, J Clin Med) - "Seven patients were administered desmopressin, a synthetic analog of antidiuretic hormone (ADH), based on our previous experience in reducing bleeding in von Willebrand disease. Antiangiogenic therapy with thalidomide (50 mg oral tablets) was used in 24 patients, while bevacizumab was administered to 5 patients...Frequent hospitalization places a significant burden on the healthcare system. Thus, we have used treatment options for HHT patients that primarily act by inhibiting VEGF, and these treatment modalities have yielded successful results in our hands."

Journal • Cardiovascular • CNS Disorders • Hematological Disorders • Hemophilia • Otorhinolaryngology • Rare Diseases • ACVRL1 • ALK1 • ENG

An Unusual Case of Refractory Seizures. (PubMed, J Assoc Physicians India) - "The patient was further treated for syndrome of inappropriate antidiuretic hormone secretion (SIADH)-induced hyponatremia with fluid restriction and desmopressin. Inadequate response seen after IVIg infusion prompted further immunosuppression with rituximab. Satisfactory control of the seizure activity was attained after the second rituximab dose."

Journal • Brain Cancer • Cardiovascular • CNS Disorders • Diabetes • Epilepsy • Glioma • Heart Failure • Hypoglycemia • Immunology • Infectious Disease • Metabolic Disorders • Oncology • Solid Tumor