PDSA e-News: November 30, 2018

blood cellsRetrospective study found that ITP patients with infections displayed statistically lower platelet counts than ITP patients without infections

Although the function of platelets in immune response is still under investigation, a recent retrospective study found that ITP patients that developed an infection displayed a lower platelet count. Patients with a low platelet count that developed infections also displayed a slower response to therapy. These findings support recent evidence suggesting a potential role for platelets in the inflammation process.

Qu M, Liu Q, Zhao H, et al. Low platelet count as risk factor for infections in patients with primary immune thrombocytopenia: a retrospective evaluation. Ann Hematol. 2018;97(9):1701-1706.

Principia New LogoPrincipia Biopharma Announces FDA Orphan-Drug Designation For PRN1008 For The Treatment of ITP

Principia Biopharma’s oral drug “PRN1008,” received orphan-drug designation for the treatment of ITP. PRN1008 is a reversible covalent Bruton’s Tyrosine Kinase (BTK) inhibitor; BTK is involved in B-cell activation and maturation, and partially regulates the immune response of macrophages, which eat platelets. By inhibiting BTK, the drug PRN1008 is able to block this process and thus prevents the immune response that occurs in ITP when attacking and destroying platelets. PRN1008 has been previously granted orphan-drug designation for other diseases, but with this new designation a phase 2 clinical trial on the safety and efficacy of PRN1008 in ITP patients has been approved. If found to be effective in ITP, PRN1008 could become a safe option as an oral therapy that would limit side effects seen in other more invasive immunosuppressive therapies.


Doptelet logoDova Pharmaceuticals Achieves Application Acceptance For Doptelet (Avatrombopag) To Be Used In Adult Chronic ITP patients

Dova Pharmaceuticals had recently achieved FDA approval for Doptelet (Avatrombopag) in the treatment of patients with chronic liver disease who suffer from low platelet counts and are about to undergo surgery. Doptelet, a thrombopoietin receptor agonist, acts to mimic thrombopoietin and stimulate the activation, proliferation, and maturation of platelets. The US Food and Drug Administration have now accepted an application for Doptelet to also be used in patients who suffer from chronic ITP. With the acceptance of this application, Dova hopes to gain FDA approval for the use of Doptelet in chronic ITP patients around July of next year.


ASH Guidelines on ITP Available for Public Comment

The American Society of Hematology (ASH) is seeking comment on draft clinical practice guidelines on immune thrombocytopenia (ITP). All ASH members and other stakeholders are invited to review the draft recommendations and submit comments. This includes physicians, researchers, allied professionals, industry and insurance company representatives, patients, caregivers and members of the public. Feedback received will be provided to the guideline panel for review.

(p>These guidelines were developed by ASH and supported by the University of Oklahoma Health Sciences Center using a process intended to meet the standards recommended by National Academy of Medicine. The guideline panel included multidisciplinary content experts, methodologists, and patient representatives. For more information about ASH guidelines, please visit the ASH website.

The draft recommendations and other materials are available at: www.hematology.org/Guidelines-Public-Comment. The deadline to comment is Monday December 10.

If you have any questions, please email them guidelines@hematology.org.


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