Overview of clinical and experimental VWD treatment strategies
| Treatment status . | Advantages . | Disadvantages . |
|---|---|---|
| Current | ||
| Desmopressin | Relatively inexpensive, no blood-borne contaminations, easy availability, self-administration | Short-term effect, tachyphylaxis, variable response, mild side effects |
| VWF/FVIII concentrates | Effective in nonresponders to DDAVP | Short-term effect, higher costs, risk of contamination, variable quality |
| Investigational | ||
| Interleukin-11 | DDAVP surrogate/complement, sustained effect | Minor to moderate side effects, increase in platelet count and fibrinogen levels |
| Future | ||
| Recombinant VWF | Standardized product, no risk of blood-borne contamination | Short-term effect, no FVIII for acute needs |
| Gene therapy | Potential of lifelong cure, increase in patient's quality of life | Safety and ethical issues |
| Treatment status . | Advantages . | Disadvantages . |
|---|---|---|
| Current | ||
| Desmopressin | Relatively inexpensive, no blood-borne contaminations, easy availability, self-administration | Short-term effect, tachyphylaxis, variable response, mild side effects |
| VWF/FVIII concentrates | Effective in nonresponders to DDAVP | Short-term effect, higher costs, risk of contamination, variable quality |
| Investigational | ||
| Interleukin-11 | DDAVP surrogate/complement, sustained effect | Minor to moderate side effects, increase in platelet count and fibrinogen levels |
| Future | ||
| Recombinant VWF | Standardized product, no risk of blood-borne contamination | Short-term effect, no FVIII for acute needs |
| Gene therapy | Potential of lifelong cure, increase in patient's quality of life | Safety and ethical issues |