Rare diseases, Gresele (UniPg): "New drugs for platelet disorders are on the way."

(Adnkronos) – "Until recently," for platelet disorders, "innovations were in genetic diagnosis and epidemiological recognition," while there was very little therapeutic progress. "In recent years, however, there have been innovations in hemophilia, new therapeutic approaches for managing bleeding," said Paolo Gresele, professor of Internal Medicine at the University of Perugia, speaking today in Rome at the 29th National Congress of Siset – the Italian Society for the Study of Hemostasis and Thrombosis.

"Congenital platelet disorders," the expert explains, "have been largely neglected in recent years because interest has focused on more severe congenital bleeding disorders, such as hemophilia. In reality, what is emerging, and what has also emerged at this conference, is that these conditions are much more common than previously thought."

Specifically, "a new bispecific monoclonal antibody is undergoing advanced clinical trials, involving many Italian centers, which is very promising," Gresele specifies. "We hope the trial will soon offer this new therapeutic option to patients." Furthermore, "there are therapeutic innovations for certain specific bleeding manifestations. For example, it is being discovered that some of these congenital platelet dysfunction disorders present a particular vulnerability in the gastrointestinal system, with significant and recurrent bleeding, and some anti-angiogenic therapies appear to provide significant benefits to these patients."

Regarding the clinical impact of these disorders, the expert explains that "they are certainly more common than hemophilia A and von Willebrand disease, which is considered the most common inherited bleeding disorder in our community. The other important clinical development is that these disorders are associated with a sometimes severe bleeding diathesis: they therefore require diagnosis, and it is very important that they are recognized so that appropriate management and treatment can be initiated early," he concludes. 

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