Treatment of Inherited Platelet Disorders: Current Status and Future Options

Publikation: Beitrag in FachzeitschriftÜbersichtsartikel (Review)BeigetragenBegutachtung

Beitragende

Abstract

Inherited platelet disorders (IPDs) comprise a heterogeneous group of entities that manifest with variable bleeding tendencies. For successful treatment, the underlying platelet disorder, bleeding severity and location, age, and sex must be considered in the broader clinical context. Previous information from the AWMF S2K guideline #086-004 (www.awmf.org) is evaluated for validity and supplemented by information of new available and future treatment options and clinical scenarios that need specific measures. Special attention is given to the treatment of menorrhagia and risk management during pregnancy in women with IPDs. Established treatment options of IPDs include local hemostatic treatment, tranexamic acid, desmopressin, platelet concentrates, and recombinant activated factor VII. Hematopoietic stem cell therapy is a curative approach for selected patients. We also provide an outlook on promising new therapies. These include autologous hematopoietic stem cell gene therapy, artificial platelets and nanoparticles, and various other procoagulant treatments that are currently tested in clinical trials in the context of hemophilia.

Details

OriginalspracheEnglisch
Seiten (von - bis)261-270
Seitenumfang10
FachzeitschriftHamostaseologie
Jahrgang43
Ausgabenummer4
PublikationsstatusVeröffentlicht - 1 Feb. 2023
Peer-Review-StatusJa

Externe IDs

PubMed 37611608

Schlagworte

ASJC Scopus Sachgebiete

Schlagwörter

  • blood platelet disorder, inherited platelet disorder, therapy, Pregnancy, Humans, Blood Platelets, Female, Hemostatics, Blood Platelet Disorders/genetics, Hemophilia A, Hematopoietic Stem Cells