Summary
Desmopressin has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Haemophilia A / B would be drug repurposing rather than first-in-human development. This does not mean it is approved for Haemophilia A / B. ClinicalTrials.gov lists 8 registered trials linking Desmopressin to Haemophilia A / B: 5 have completed; 3 are terminated, withdrawn, suspended or of unknown status. The most advanced is Phase 4 (NCT03379974). The largest enrolment is 800 participants (NCT05628558). Registration activity spans 2010 to 2022. The literature layer holds 14 publications for this pair: 2 Cochrane reviews, 1 meta-analysis, 2 systematic reviews, 4 randomised controlled trial publications and 5 clinical trial publications. Publication years run from 1986 to 2025. Because at least one synthesis-level source exists (systematic review, meta-analysis or Cochrane review), this pair has been assessed beyond single studies, although the synthesis may concern a different indication.
The RepurpOS disease-intelligence file for Hemophilia ranks 50 candidate therapeutics from Open Targets, ChEMBL, DGIdb and PubMed; only a minority carry direct clinical evidence, and this page covers one of those. Spontaneous remission context recorded for the condition: No spontaneous remission; lifelong clotting factor deficiency; joint damage progressive with each bleed without treatment; with prophylaxis, normal lifespan achievable.
Evidence table
| Evidence tier | A · Strong |
|---|---|
| Evidence score | 71.0 (trials 21.0, literature 30.0, tier 15, approved bonus 5.0) |
| Registered trials | 8 total: 0 recruiting, 0 active / not yet recruiting, 5 completed, 3 other |
| Linked publications | 14 (5 clinical trial publications, 4 randomised controlled trial publications, 2 Cochrane reviews, 2 systematic reviews, 1 meta-analysis) |
| Agent type | Drug (Small molecule) |
| Development stage (any indication) | Approved |
| Mechanism / class | Not recorded |
| Data sources | DGIdb |
| Linked via biomarker / target | F8 |
How the evidence score is calculated
- Each registered trial scores by status (recruiting / active / enrolling 3, completed 2.5, not yet recruiting 2, unknown 1, terminated / withdrawn / suspended 0.5) plus a phase bonus (phase 3-4 +2, phase 2 +1, phase 1 +0.5). The trial component is capped at 30.
- Each literature item scores by design (Cochrane review 5, meta-analysis 4, systematic review 3, RCT 3, clinical trial publication 2, curated reference 1.5, other PubMed record 1). The literature component is capped at 30.
- The existing evidence tier adds 15 (A / Strong), 10 (B / Moderate), 5 (C / Preliminary) or 0 (D / Anecdotal).
- Agents approved for any indication (max clinical phase 4) add 5, because an approved agent has an established safety profile that lowers the barrier to repurposing trials.
- The score ranks what has been studied, not what works. It does not read effect sizes or directions of effect.
Registered clinical trials
| NCT ID | Title | Status | Phase | Enrolment |
|---|---|---|---|---|
| NCT03379974 | Exercise Versus DDAVP in Patients With Mild Hemophilia A 2018 | completed | Phase 4 | 32 |
| NCT03136003 | DDAVP vs. Exercise in Patients With Mild Hemophilia A 2017 | status unknown | Phase 4 | 30 |
| NCT00994929 | Efficacy and Safety of IL-11 in DDAVP Unresponsive 2010 | completed | Phase 2 | 9 |
| NCT02506023 | Characterization of Laboratory Response to DDAVP in Adult Hemophilia A Carriers 2015 | completed | Phase 1 | 2 |
| NCT03054662 | Non Substitutive Strategies to Improve Haemophilia Care in Developing Countries. Experience in Ivory Coast. 2017 | completed | Not applicable | 142 |
| NCT06020456 | Genetic Factors of the Desmopressin Response in Carriers of Hemophilia A 2022 | completed | Not applicable | 361 |
| NCT01403740 | Turkish Acquired Haemophilia Registry 2011 | status unknown | Not applicable | 250 |
| NCT05628558 | Genetic Influence of Genetic Factors Influencing the Desmopressin's Efficacy in Mild/Moderate Hemophilia A 2020 | status unknown | Not applicable | 800 |
Published literature
- Meta-analysis DDAVP response and its determinants in bleeding disorders: a systematic review and meta-analysisLaan S, Del Castillo Alferez J, Cannegieter S et al. · Blood · 2025 · PMID 39854691
- Cochrane review Desmopressin as a Treatment in Patients With Von Willebrand Disease: A Systematic ReviewBeltran A, Jaramillo AP, Vallejo MP et al. · Cureus · 2023 · PMID 37649925
- Clinical trial publication Moderate-intensity aerobic exercise vs desmopressin in adolescent males with mild hemophilia A: a randomized trialKumar R, Dunn AL, Schneiderman JE et al. · Blood · 2022 · PMID 35839450
- Cochrane review Efficacy of parenteral formulations of desmopressin in the treatment of bleeding disorders: A systematic reviewSreeraman S, McKinlay S, Li A et al. · Thrombosis research · 2022 · PMID 35278886
- Systematic review Maternal and neonatal bleeding complications in relation to peripartum management in hemophilia carriers: A systematic reviewPunt MC, Waning ML, Mauser-Bunschoten EP et al. · Blood reviews · 2021 · PMID 33775466
- Systematic review Inherited Bleeding Disorders in the Obstetric PatientBannow BS, Konkle BA · Transfusion medicine reviews · 2018 · PMID 30097224
- Clinical trial publication Molecular and phenotypic determinants of the response to desmopressin in adult patients with mild hemophilia ACastaman G, Mancuso ME, Giacomelli SH et al. · Journal of thrombosis and haemostasis : JTH · 2009 · PMID 19719828
- Clinical trial publication Circumcision in hemophilia: a cost-effective method using a novel deviceKaraman MI, Zulfikar B, Caskurlu T et al. · Journal of pediatric surgery · 2004 · PMID 15486905
- Clinical trial publication Evaluation of high concentration intranasal and intravenous desmopressin in pediatric patients with mild hemophilia A or mild-to-moderate type 1 von Willebrand diseaseGill JC, Ottum M, Schwartz B · The Journal of pediatrics · 2002 · PMID 12032528
- Clinical trial publication Adverse events during use of intranasal desmopressin acetate for haemophilia A and von Willebrand disease: a case report and review of 40 patientsDunn AL, Powers JR, Ribeiro MJ et al. · Haemophilia : the official journal of the World Federation of Hemophilia · 2000 · PMID 10632735
- Randomized controlled trial [Clinical assessment of desmopressin to reduce blood loss in patients after cardiopulmonary bypass]Chuang HI, Horng YJ, Li Y et al. · Ma zui xue za zhi = Anaesthesiologica Sinica · 1993 · PMID 7968327
- Randomized controlled trial Adventures in hemostasis. Desmopressin in cardiac surgerySalzman EW, Weinstein MJ, Reilly D et al. · Archives of surgery (Chicago, Ill. : 1960) · 1993 · PMID 8431122
- Randomized controlled trial Changes in factor VIII: coagulant activity and von Willebrand factor antigen concentration after subcutaneous injection of desmopressin in dogs with mild hemophilia AMansell PD, Parry BW · Journal of veterinary internal medicine · 1991 · PMID 1920255
- Randomized controlled trial Treatment with desmopressin acetate to reduce blood loss after cardiac surgery. A double-blind randomized trialSalzman EW, Weinstein MJ, Weintraub RM et al. · The New England journal of medicine · 1986 · PMID 3517650
Mechanism and notes
No mechanism of action is recorded for this pair in the source databases.
OSMF's existing evidence tier for this pair is A (Strong), derived from the strength of the disease association recorded in DGIdb, in this case via the biomarker or target F8. The tier describes how well the drug-disease link is documented, not how well the drug works.
Frequently asked questions
Is Desmopressin approved for Haemophilia A / B?
Desmopressin has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Haemophilia A / B would be drug repurposing rather than first-in-human development. This does not mean it is approved for Haemophilia A / B. Approval status for the specific indication should always be confirmed with the relevant regulator and prescribing information.
Is Desmopressin in clinical trials for Haemophilia A / B?
ClinicalTrials.gov lists 8 registered trials linking Desmopressin to Haemophilia A / B: 5 have completed; 3 are terminated, withdrawn, suspended or of unknown status. The most advanced is Phase 4 (NCT03379974). The largest enrolment is 800 participants (NCT05628558). Registration activity spans 2010 to 2022.
What does the evidence show for Desmopressin in Haemophilia A / B?
Desmopressin has both completed registered trials and synthesis-level publications linked to Haemophilia A / B. That is the strongest profile in this database, but the summaries here do not extract effect sizes, so read the linked reviews for direction and magnitude of benefit. OSMF's existing evidence tier for this pair is A (Strong), derived from the strength of the disease association recorded in DGIdb, in this case via the biomarker or target F8. The tier describes how well the drug-disease link is documented, not how well the drug works.
Cite this page
Data: ClinicalTrials.gov, PubMed, Open Targets, ChEMBL, DGIdb and the OSMF therapeutic agent database. Last updated 2026-07-06. Page built 2026-10-07.