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DDAVP response in moderate hemophilia A
Clinical significance and future studies
As illustrated in Table 5, the use of DDAVP for moderate HA patients in clinical practice has been limited to a few cases over the last 40 years, despite the fact that this drug has been available since 1977. The study herein evinces that 40% of the patients with moderate HA in our cohort have a clinical relevant response to DDAVP for mild bleeds/injuries. Moreover, half of the patients with high inhibitor risk mutations respond to DDAVP; for this reason, it is important to always assess such patients for DDAVP responsiveness. Doing so might lead to less exposure to FVIII concentrates, which reduces risk for inhibitor devel- opment, and realizes a reduction in costs.
In order to confirm our findings, future studies should focus on the prospective inclusion of moderate HA patients. Lastly, more data on mutations are needed in order to assess the effect of F8 missense mutations on DDAVP response in these patients.
Conclusion
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In 40% of the administrations in moderate HA patients in our cohort, the use of DDAVP is adequate for treatment in case of minor bleeding or trauma; as such, it is important to consistently assess moderate patients for DDAVP respon- siveness.
Furthermore, we identified six predictors of peak FVIII:C, which, taken together, explain 65% of the variation in peak FVIII:C. Pre-DDAVP FVIII:C levels and the patients’ ability to raise VWF levels are the essential predictors for response.
Acknowledgments
Romy van Spengen, medical student in the Academic Medical Center Amsterdam, helped to construct Table 5.
Funding
This work was supported by the Access to Insight Grant from Novo Nordisk.
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