{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,3,12]],"date-time":"2026-03-12T12:05:55Z","timestamp":1773317155315,"version":"3.50.1"},"reference-count":97,"publisher":"Georg Thieme Verlag KG","issue":"04","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Hamostaseologie"],"published-print":{"date-parts":[[2023,8]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:p>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\u2013004 (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.<\/jats:p>","DOI":"10.1055\/a-2080-6602","type":"journal-article","created":{"date-parts":[[2023,8,23]],"date-time":"2023-08-23T22:33:10Z","timestamp":1692829990000},"page":"261-270","source":"Crossref","is-referenced-by-count":8,"title":["Treatment of Inherited Platelet Disorders: Current Status and Future Options"],"prefix":"10.1055","volume":"43","author":[{"given":"Caroline","family":"Bargehr","sequence":"additional","affiliation":[{"name":"Department of Paediatrics 1, Medical University of Innsbruck, Innsbruck, Austria"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Ralf","family":"Kn\u00f6fler","sequence":"additional","affiliation":[{"name":"Department of Paediatric Haemostaseology, University Hospital Carl Gustav Carus Dresden, Dresden, Germany"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Werner","family":"Streif","sequence":"additional","affiliation":[{"name":"Department of Paediatrics 1, Medical University of Innsbruck, Innsbruck, Austria"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"194","published-online":{"date-parts":[[2023,8,23]]},"reference":[{"issue":"09","key":"ref1","doi-asserted-by":"crossref","first-page":"4521","DOI":"10.3390\/ijms22094521","article-title":"Inherited platelet disorders: an updated overview","volume":"22","author":"V Palma-Barqueros","year":"2021","journal-title":"Int J Mol Sci"},{"issue":"04","key":"ref2","doi-asserted-by":"crossref","first-page":"259","DOI":"10.5482\/ha12050001","article-title":"Inherited thrombocytopenias: the evolving spectrum","volume":"32","author":"C L Balduini","year":"2012","journal-title":"Hamostaseologie"},{"issue":"05","key":"ref3","doi-asserted-by":"crossref","first-page":"603","DOI":"10.1111\/j.1365-2141.2006.06343.x","article-title":"A review of inherited platelet disorders with guidelines for their management on behalf of the UKHCDO","volume":"135","author":"P HB Bolton-Maggs","year":"2006","journal-title":"Br J Haematol"},{"issue":"01","key":"ref4","doi-asserted-by":"crossref","first-page":"385","DOI":"10.1182\/asheducation-2017.1.385","article-title":"Hereditary thrombocytopenias: a growing list of disorders","volume":"2017","author":"P Noris","year":"2017","journal-title":"Hematology (Am Soc Hematol Educ Program)"},{"issue":"05","key":"ref5","doi-asserted-by":"crossref","first-page":"224","DOI":"10.1542\/pir.2018-0359","article-title":"Platelet disorders","volume":"41","author":"K M Haley","year":"2020","journal-title":"Pediatr Rev"},{"issue":"23","key":"ref6","doi-asserted-by":"crossref","first-page":"2478","DOI":"10.1182\/blood-2019-01-852350","article-title":"Simplifying the diagnosis of inherited platelet disorders? The new tools do not make it any easier","volume":"133","author":"A Greinacher","year":"2019","journal-title":"Blood"},{"issue":"06","key":"ref7","doi-asserted-by":"crossref","first-page":"1278","DOI":"10.3324\/haematol.2022.280856","article-title":"Treatment of inherited thrombocytopenias [in German]","volume":"107","author":"C L Balduini","year":"2022","journal-title":"Haematologica"},{"issue":"04","key":"ref8","doi-asserted-by":"crossref","first-page":"269","DOI":"10.5482\/HAMO-2014040001","article-title":"Therapie heredit\u00e4rer thrombozytopathien","volume":"34","author":"W Streif","year":"2014","journal-title":"Hamostaseologie"},{"issue":"01","key":"ref10","doi-asserted-by":"crossref","first-page":"47","DOI":"10.31887\/DCNS.2007.9.1\/dhalperin","article-title":"Influence of antidepressants on hemostasis","volume":"9","author":"D Halperin","year":"2007","journal-title":"Dialogues Clin Neurosci"},{"issue":"06","key":"ref11","doi-asserted-by":"crossref","first-page":"917","DOI":"10.1111\/hae.14643","article-title":"Gynaecological management of women with inherited bleeding disorders. A UK Haemophilia Centres Doctors' Organisation Guideline","volume":"28","author":"N Curry","year":"2022","journal-title":"Haemophilia"},{"issue":"04","key":"ref12","doi-asserted-by":"crossref","first-page":"633","DOI":"10.1111\/hae.14559","article-title":"Iron deficiency anemia and bleeding management in pediatric patients with Bernard-Soulier syndrome and Glanzmann Thrombasthenia: a single-institution analysis","volume":"28","author":"A Lee","year":"2022","journal-title":"Haemophilia"},{"issue":"01","key":"ref13","doi-asserted-by":"crossref","first-page":"65","DOI":"10.1111\/hae.12002","article-title":"Dental health and oral health-related quality of life in children with congenital bleeding disorders","volume":"19","author":"K Salem","year":"2013","journal-title":"Haemophilia"},{"issue":"05","key":"ref14","doi-asserted-by":"crossref","first-page":"541","DOI":"10.1046\/j.1365-2516.2003.00819.x","article-title":"Immunization of patients with bleeding disorders","volume":"9","author":"M Makris","year":"2003","journal-title":"Haemophilia"},{"issue":"06","key":"ref15","doi-asserted-by":"crossref","first-page":"813","DOI":"10.1111\/j.1365-2141.2010.08191.x","article-title":"Modern management of severe platelet function disorders","volume":"149","author":"J Alamelu","year":"2010","journal-title":"Br J Haematol"},{"issue":"04","key":"ref16","doi-asserted-by":"crossref","first-page":"494","DOI":"10.1016\/j.transci.2018.07.010","article-title":"Inherited platelet functional disorders: general principles and practical aspects of management","volume":"57","author":"A Lee","year":"2018","journal-title":"Transfus Apheresis Sci"},{"issue":"09","key":"ref17","doi-asserted-by":"crossref","first-page":"935","DOI":"10.1002\/phar.1291","article-title":"An essential primer for understanding the role of topical hemostats, surgical sealants, and adhesives for maintaining hemostasis","volume":"33","author":"M Gabay","year":"2013","journal-title":"Pharmacotherapy"},{"issue":"01","key":"ref18","doi-asserted-by":"crossref","first-page":"40","DOI":"10.5505\/tjh.2012.07769","article-title":"The evaluation of fibrin sealants and tissue adhesives in oral surgery among patients with bleeding disorders","volume":"29","author":"G Ak","year":"2012","journal-title":"Turk J Haematol"},{"issue":"11","key":"ref19","doi-asserted-by":"crossref","first-page":"nwac162","DOI":"10.1093\/nsr\/nwac162","article-title":"Emerging hemostatic materials for non-compressible hemorrhage control","volume":"9","author":"R Dong","year":"2022","journal-title":"Natl Sci Rev"},{"key":"ref20","doi-asserted-by":"crossref","first-page":"151","DOI":"10.1016\/j.ejogrb.2019.06.003","article-title":"Uterine packing with chitosan-covered gauze compared to balloon tamponade for managing postpartum hemorrhage","volume":"240","author":"A M Dueckelmann","year":"2019","journal-title":"Eur J Obstet Gynecol Reprod Biol"},{"issue":"05","key":"ref21","doi-asserted-by":"crossref","first-page":"585","DOI":"10.2165\/11209070-000000000-00000","article-title":"Tranexamic acid: a review of its use in the treatment of hyperfibrinolysis","volume":"72","author":"P L McCormack","year":"2012","journal-title":"Drugs"},{"issue":"01","key":"ref22","doi-asserted-by":"crossref","first-page":"e1","DOI":"10.1111\/j.1365-2516.2012.02909.x","article-title":"Guidelines for the management of hemophilia","volume":"19","author":"A Srivastava","year":"2013","journal-title":"Haemophilia"},{"issue":"01","key":"ref23","doi-asserted-by":"crossref","first-page":"18","DOI":"10.1002\/ana.24558","article-title":"Tranexamic acid-associated seizures: causes and treatment","volume":"79","author":"I Lecker","year":"2016","journal-title":"Ann Neurol"},{"issue":"S 04","key":"ref24","doi-asserted-by":"crossref","first-page":"154","DOI":"10.1111\/j.1365-2516.2012.02856.x","article-title":"Inherited platelet disorders","volume":"18","author":"A T Nurden","year":"2012","journal-title":"Haemophilia"},{"issue":"05","key":"ref25","doi-asserted-by":"crossref","first-page":"621","DOI":"10.1111\/bjh.15409","article-title":"How we treat the platelet glycoprotein defects; Glanzmann thrombasthenia and Bernard Soulier syndrome in children and adults","volume":"182","author":"J D Grainger","year":"2018","journal-title":"Br J Haematol"},{"issue":"03","key":"ref26","doi-asserted-by":"crossref","first-page":"169","DOI":"10.1055\/s-0031-1275347","article-title":"Evaluation of desmopressin effect on primary haemostasis in pediatric patients with aspirin-like defect as hereditary thrombocytopathy","volume":"223","author":"J T Tauer","year":"2011","journal-title":"Klin Padiatr"},{"issue":"12","key":"ref27","doi-asserted-by":"crossref","first-page":"1905","DOI":"10.1182\/blood-2013-04-497123","article-title":"The effect of desmopressin on platelet function: a selective enhancement of procoagulant COAT platelets in patients with primary platelet function defects","volume":"123","author":"G Colucci","year":"2014","journal-title":"Blood"},{"issue":"02","key":"ref28","first-page":"141","volume":"10","author":"M Lozano","year":"1999"},{"issue":"S 01","key":"ref29","doi-asserted-by":"crossref","first-page":"31","DOI":"10.1111\/j.1365-2516.2007.01607.x","article-title":"Desmopressin in inherited disorders of platelet function","volume":"14","author":"A Coppola","year":"2008","journal-title":"Haemophilia"},{"issue":"18","key":"ref30","doi-asserted-by":"crossref","first-page":"1925","DOI":"10.1182\/blood.2022016558","article-title":"How I use platelet transfusions","volume":"140","author":"S J Stanworth","year":"2022","journal-title":"Blood"},{"issue":"01","key":"ref31","doi-asserted-by":"crossref","first-page":"14","DOI":"10.1080\/09537104.2021.1962837","article-title":"Neonatal platelet physiology and implications for transfusion","volume":"33","author":"F Ferrer-Mar\u00edn","year":"2022","journal-title":"Platelets"},{"issue":"10","key":"ref32","doi-asserted-by":"crossref","first-page":"797","DOI":"10.1080\/17474086.2019.1657824","article-title":"Platelet and red cell transfusions for neonates: lifesavers or Trojan horses?","volume":"12","author":"S F Fustolo-Gunnink","year":"2019","journal-title":"Expert Rev Hematol"},{"issue":"03","key":"ref33","doi-asserted-by":"crossref","first-page":"228","DOI":"10.1016\/j.tracli.2018.07.003","article-title":"Inherited platelet disorders: management of the bleeding risk","volume":"25","author":"A Dupuis","year":"2018","journal-title":"Transfus Clin Biol"},{"issue":"02","key":"ref34","doi-asserted-by":"crossref","first-page":"253","DOI":"10.1160\/TH07-09-0568","article-title":"Congenital disorders associated with platelet dysfunctions","volume":"99","author":"P Nurden","year":"2008","journal-title":"Thromb Haemost"},{"issue":"02","key":"ref35","doi-asserted-by":"crossref","first-page":"173","DOI":"10.1111\/bjh.15087","article-title":"Anti-\u03b1IIb \u03b23 immunization in Glanzmann thrombasthenia: review of literature and treatment recommendations","volume":"181","author":"M Fiore","year":"2018","journal-title":"Br J Haematol"},{"issue":"01","key":"ref36","first-page":"40","article-title":"Antibody removal therapy used successfully at delivery of a pregnant patient with Glanzmann's thrombasthenia and multiple anti-platelet antibodies","volume":"61","author":"K Ito","year":"1991","journal-title":"Vox Sang"},{"issue":"04","key":"ref37","doi-asserted-by":"crossref","first-page":"991","DOI":"10.1046\/j.1365-2141.2002.03936.x","article-title":"Protein A Sepharose immunoadsorption can restore the efficacy of platelet concentrates in patients with Glanzmann's thrombasthenia and anti-glycoprotein IIb-IIIa antibodies","volume":"119","author":"I Martin","year":"2002","journal-title":"Br J Haematol"},{"issue":"03","key":"ref38","doi-asserted-by":"crossref","first-page":"332","DOI":"10.1055\/s-0040-1718373","article-title":"The use of recombinant activated factor VII in patients with Glanzmann's thrombasthenia","volume":"121","author":"M C Poon","year":"2021","journal-title":"Thromb Haemost"},{"issue":"12","key":"ref39","doi-asserted-by":"crossref","first-page":"816","DOI":"10.1007\/s00277-005-1080-y","article-title":"Use of recombinant factor VIIa in the management of severe bleeding episodes in patients with Bernard-Soulier syndrome","volume":"84","author":"M C Ozelo","year":"2005","journal-title":"Ann Hematol"},{"issue":"03","key":"ref40","doi-asserted-by":"crossref","first-page":"477","DOI":"10.1046\/j.1365-2141.2003.04286.x","article-title":"The use of recombinant factor VIIa in children with inherited platelet function disorders","volume":"121","author":"A M Almeida","year":"2003","journal-title":"Br J Haematol"},{"issue":"05","key":"ref41","doi-asserted-by":"crossref","first-page":"484","DOI":"10.1080\/09537104.2016.1237625","article-title":"PT-VWD posing diagnostic and therapeutic challenges - small case series","volume":"28","author":"A S\u00e1nchez-Luceros","year":"2017","journal-title":"Platelets"},{"issue":"02","key":"ref42","doi-asserted-by":"crossref","first-page":"199","DOI":"10.1097\/MBC.0b013e3280147fd8","article-title":"Recombinant activated factor VII for hemostatic cover of orthopedic interventions in a girl with thrombocytopenia with absent radii syndrome","volume":"18","author":"A Coppola","year":"2007","journal-title":"Blood Coagul Fibrinolysis"},{"issue":"06","key":"ref43","doi-asserted-by":"crossref","first-page":"1112","DOI":"10.3324\/haematol.2018.212845","article-title":"Thrombopoietin receptor agonists: ten years later","volume":"104","author":"W Ghanima","year":"2019","journal-title":"Haematologica"},{"key":"ref45","doi-asserted-by":"crossref","first-page":"100784","DOI":"10.1016\/j.blre.2020.100784","article-title":"Inherited thrombocytopenias: an updated guide for clinicians","volume":"48","author":"A Pecci","year":"2021","journal-title":"Blood Rev"},{"issue":"10","key":"ref46","doi-asserted-by":"crossref","first-page":"2961","DOI":"10.1182\/blood.V82.10.2961.2961","article-title":"Splenectomy and\/or bone marrow transplantation in the management of the Wiskott-Aldrich syndrome: long-term follow-up of 62 cases","volume":"82","author":"C A Mullen","year":"1993","journal-title":"Blood"},{"issue":"01","key":"ref47","doi-asserted-by":"crossref","first-page":"439","DOI":"10.1182\/blood-2007-03-076679","article-title":"Long-term outcome following hematopoietic stem-cell transplantation in Wiskott-Aldrich syndrome: collaborative study of the European Society for Immunodeficiencies and European Group for Blood and Marrow Transplantation","volume":"111","author":"H Ozsahin","year":"2008","journal-title":"Blood"},{"issue":"02","key":"ref48","doi-asserted-by":"crossref","first-page":"331","DOI":"10.1016\/j.idc.2019.01.002","article-title":"Complications of stem cell transplantation that affect infections in stem cell transplant recipients, with analogies to patients with hematologic malignancies","volume":"33","author":"S Nathan","year":"2019","journal-title":"Infect Dis Clin North Am"},{"key":"ref49","doi-asserted-by":"crossref","first-page":"449","DOI":"10.2147\/JBM.S271744","article-title":"Glanzmann thrombasthenia: perspectives from clinical practice on accurate diagnosis and optimal treatment strategies","volume":"12","author":"N Mathews","year":"2021","journal-title":"J Blood Med"},{"issue":"06","key":"ref50","doi-asserted-by":"crossref","first-page":"e521","DOI":"10.1097\/MPH.0000000000001646","article-title":"Successful use of hematopoietic stem cell transplantation for 2 pediatric cases of Glanzmann thrombasthenia and review of the literature","volume":"42","author":"B D Friend","year":"2020","journal-title":"J Pediatr Hematol Oncol"},{"issue":"07","key":"ref51","doi-asserted-by":"crossref","first-page":"971","DOI":"10.1002\/pbc.20365","article-title":"Sustained engraftment post bone marrow transplant despite anti-platelet antibodies in Glanzmann thrombasthenia","volume":"45","author":"V H Flood","year":"2005","journal-title":"Pediatr Blood Cancer"},{"issue":"04","key":"ref52","doi-asserted-by":"crossref","first-page":"635","DOI":"10.1111\/j.1365-2141.1985.tb07358.x","article-title":"Complete correction of Glanzmann's thrombasthenia by allogeneic bone-marrow transplantation","volume":"59","author":"S Bellucci","year":"1985","journal-title":"Br J Haematol"},{"issue":"03","key":"ref53","doi-asserted-by":"crossref","first-page":"327","DOI":"10.1038\/sj.bmt.1702139","article-title":"Bone marrow transplantation in severe Glanzmann's thrombasthenia with antiplatelet alloimmunization","volume":"25","author":"S Bellucci","year":"2000","journal-title":"Bone Marrow Transplant"},{"issue":"01","key":"ref54","doi-asserted-by":"crossref","first-page":"79","DOI":"10.7326\/0003-4819-138-1-200301070-00028","article-title":"Hematopoietic stem-cell transplantation for the Bernard-Soulier syndrome","volume":"138","author":"F Locatelli","year":"2003","journal-title":"Ann Intern Med"},{"issue":"04","key":"ref55","doi-asserted-by":"crossref","first-page":"647","DOI":"10.1111\/bjh.15831","article-title":"How I manage patients with Wiskott Aldrich syndrome","volume":"185","author":"E Rivers","year":"2019","journal-title":"Br J Haematol"},{"issue":"02","key":"ref56","doi-asserted-by":"crossref","first-page":"456","DOI":"10.1182\/blood-2003-05-1480","article-title":"Clinical course of patients with WASP gene mutations","volume":"103","author":"K Imai","year":"2004","journal-title":"Blood"},{"issue":"09","key":"ref57","doi-asserted-by":"crossref","first-page":"1001","DOI":"10.1001\/archneur.1984.04050200111031","article-title":"Ch\u00e9diak-Higashi syndrome. Neurologic appearance","volume":"41","author":"R E Pettit","year":"1984","journal-title":"Arch Neurol"},{"issue":"02","key":"ref58","doi-asserted-by":"crossref","first-page":"271","DOI":"10.1111\/petr.12626","article-title":"Allogeneic hematopoietic stem cell transplantation for Chediak-Higashi syndrome","volume":"20","author":"K Umeda","year":"2016","journal-title":"Pediatr Transplant"},{"issue":"01","key":"ref59","doi-asserted-by":"crossref","first-page":"132","DOI":"10.1186\/s13023-014-0132-6","article-title":"Towards the targeted management of Chediak-Higashi syndrome","volume":"9","author":"M L Lozano","year":"2014","journal-title":"Orphanet J Rare Dis"},{"issue":"07","key":"ref60","doi-asserted-by":"crossref","first-page":"411","DOI":"10.1038\/sj.bmt.1705600","article-title":"Hematopoietic cell transplantation for Chediak-Higashi syndrome","volume":"39","author":"M Eapen","year":"2007","journal-title":"Bone Marrow Transplant"},{"issue":"01","key":"ref61","doi-asserted-by":"crossref","first-page":"E31","DOI":"10.1111\/petr.12175","article-title":"Reduced intensity transplantation for congenital amegakaryocytic thrombocytopenia: report of a case and review of the literature","volume":"18","author":"G Woods","year":"2014","journal-title":"Pediatr Transplant"},{"issue":"06","key":"ref62","doi-asserted-by":"crossref","first-page":"673","DOI":"10.1055\/s-0031-1291377","article-title":"Congenital amegakaryocytic thrombocytopenia: clinical presentation, diagnosis, and treatment","volume":"37","author":"M Ballmaier","year":"2011","journal-title":"Semin Thromb Hemost"},{"key":"ref63","doi-asserted-by":"publisher","DOI":"10.3324\/haematol.2022.281392","article-title":"Defective binding of ETS1 and STAT4 due to a mutation in the promoter region of THPO as a novel mechanism of congenital amegakaryocytic thrombocytopenia","author":"V Capaci","year":"2022","journal-title":"Haematologica"},{"issue":"07","key":"ref64","doi-asserted-by":"crossref","first-page":"875","DOI":"10.1182\/blood-2017-02-768036","article-title":"Bone marrow failure unresponsive to bone marrow transplant is caused by mutations in thrombopoietin","volume":"130","author":"A Seo","year":"2017","journal-title":"Blood"},{"issue":"06","key":"ref65","doi-asserted-by":"crossref","first-page":"586","DOI":"10.1182\/bloodadvances.2018016501","article-title":"MECOM-associated syndrome: a heterogeneous inherited bone marrow failure syndrome with amegakaryocytic thrombocytopenia","volume":"2","author":"M Germeshausen","year":"2018","journal-title":"Blood Adv"},{"issue":"04","key":"ref66","doi-asserted-by":"crossref","first-page":"598","DOI":"10.1007\/s12185-022-03505-7","article-title":"Reduced-intensity conditioning is effective for allogeneic hematopoietic stem cell transplantation in infants with MECOM-associated syndrome","volume":"117","author":"M Irie","year":"2023","journal-title":"Int J Hematol"},{"issue":"20","key":"ref67","doi-asserted-by":"crossref","first-page":"2391","DOI":"10.1182\/blood-2016-03-643544","article-title":"The 2016 revision to the World Health Organization classification of myeloid neoplasms and acute leukemia","volume":"127","author":"D A Arber","year":"2016","journal-title":"Blood"},{"issue":"05","key":"ref68","doi-asserted-by":"crossref","first-page":"598","DOI":"10.1053\/j.seminoncol.2016.10.003","article-title":"Genetic predisposition to leukemia and other hematologic malignancies","volume":"43","author":"S Feurstein","year":"2016","journal-title":"Semin Oncol"},{"issue":"04","key":"ref69","doi-asserted-by":"crossref","first-page":"306","DOI":"10.1053\/j.seminhematol.2014.08.001","article-title":"Inherited predisposition to acute myeloid leukemia","volume":"51","author":"L A Godley","year":"2014","journal-title":"Semin Hematol"},{"issue":"S 01","key":"ref70","doi-asserted-by":"crossref","first-page":"131","DOI":"10.1111\/ijlh.12999","article-title":"Inherited thrombocytopenia and platelet disorders with germline predisposition to myeloid neoplasia","volume":"41","author":"P Galera","year":"2019","journal-title":"Int J Lab Hematol"},{"issue":"01","key":"ref71","doi-asserted-by":"crossref","first-page":"28","DOI":"10.3109\/10428194.2012.701738","article-title":"Proposal for the clinical detection and management of patients and their family members with familial myelodysplastic syndrome\/acute leukemia predisposition syndromes","volume":"54","author":"J E Churpek","year":"2013","journal-title":"Leuk Lymphoma"},{"key":"ref72","doi-asserted-by":"crossref","first-page":"73","DOI":"10.1007\/s11010-020-03834-3","article-title":"Gene therapy: a double-edged sword with great powers","volume":"474","author":"R Tang","year":"2020","journal-title":"Mol Cell Biochem"},{"issue":"254","key":"ref73","first-page":"1","article-title":"Gene therapy for Wiskott-Aldrich syndrome - long-term efficacy and genotoxicity","volume":"6","author":"C J Braun","year":"2014","journal-title":"Sci Transl Med"},{"key":"ref74","doi-asserted-by":"crossref","first-page":"1233151","DOI":"10.1126\/science.1233151","article-title":"Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome","volume":"341","author":"A Aiuti","year":"2013","journal-title":"Science"},{"issue":"01","key":"ref75","doi-asserted-by":"crossref","first-page":"71","DOI":"10.1038\/s41591-021-01641-x","article-title":"Long-term safety and efficacy of lentiviral hematopoietic stem\/progenitor cell gene therapy for Wiskott-Aldrich syndrome","volume":"28","author":"A Magnani","year":"2022","journal-title":"Nat Med"},{"issue":"02","key":"ref76","doi-asserted-by":"crossref","first-page":"161","DOI":"10.1055\/s-0041-1722862","article-title":"Gene therapy for inherited bleeding disorders","volume":"47","author":"V R Arruda","year":"2021","journal-title":"Semin Thromb Hemost"},{"issue":"05","key":"ref77","doi-asserted-by":"crossref","first-page":"e239","DOI":"10.1016\/S2352-3026(19)30021-3","article-title":"Lentiviral haemopoietic stem\/progenitor cell gene therapy for treatment of Wiskott-Aldrich syndrome: interim results of a non-randomised, open-label, phase 1\/2 clinical study","volume":"6","author":"F Ferrua","year":"2019","journal-title":"Lancet Haematol"},{"issue":"11","key":"ref78","doi-asserted-by":"crossref","first-page":"1327","DOI":"10.1182\/blood-2017-04-777136","article-title":"Gene therapy for Wiskott-Aldrich syndrome in a severely affected adult","volume":"130","author":"E C Morris","year":"2017","journal-title":"Blood"},{"issue":"08","key":"ref79","doi-asserted-by":"crossref","first-page":"2671","DOI":"10.1182\/blood-2004-12-4619","article-title":"Therapeutic expression of the platelet-specific integrin, alphaIIbbeta3, in a murine model for Glanzmann thrombasthenia","volume":"106","author":"J Fang","year":"2005","journal-title":"Blood"},{"issue":"23","key":"ref80","doi-asserted-by":"crossref","first-page":"9583","DOI":"10.1073\/pnas.1016394108","article-title":"Platelet gene therapy improves hemostatic function for integrin alphaIIbbeta3-deficient dogs","volume":"108","author":"J Fang","year":"2011","journal-title":"Proc Natl Acad Sci U S A"},{"issue":"01","key":"ref81","doi-asserted-by":"crossref","first-page":"61","DOI":"10.1097\/01.AOG.0000148889.15061.fb","article-title":"Age and the prevalence of bleeding disorders in women with menorrhagia","volume":"105","author":"C S Philipp","year":"2005","journal-title":"Obstet Gynecol"},{"issue":"02","key":"ref82","doi-asserted-by":"crossref","first-page":"122","DOI":"10.1016\/j.jpag.2018.11.005","article-title":"Bleeding disorders in adolescents with heavy menstrual bleeding: the Queensland Statewide Paediatric and Adolescent Gynaecology Service","volume":"32","author":"B O'Brien","year":"2019","journal-title":"J Pediatr Adolesc Gynecol"},{"issue":"10","key":"ref83","doi-asserted-by":"crossref","first-page":"2759","DOI":"10.1111\/jth.14974","article-title":"Standardizing care to manage bleeding disorders in adolescents with heavy menses - a joint project from the ISTH pediatric\/neonatal and women's health SSCs","volume":"18","author":"A Zia","year":"2020","journal-title":"J Thromb Haemost"},{"issue":"02","key":"ref84","doi-asserted-by":"crossref","first-page":"128","DOI":"10.1056\/NEJMoa1204724","article-title":"Levonorgestrel intrauterine system versus medical therapy for menorrhagia","volume":"368","author":"J Gupta","year":"2013","journal-title":"N Engl J Med"},{"issue":"17","key":"ref85","doi-asserted-by":"crossref","first-page":"2632","DOI":"10.1182\/blood.2021011595","article-title":"How I manage pregnancy in women with Glanzmann thrombasthenia","volume":"139","author":"M Fiore","year":"2022","journal-title":"Blood"},{"issue":"04","key":"ref86","doi-asserted-by":"crossref","first-page":"584","DOI":"10.1111\/j.1365-2516.2009.02137.x","article-title":"Bernard Soulier syndrome in pregnancy: a systematic review","volume":"16","author":"P Peitsidis","year":"2010","journal-title":"Haemophilia"},{"issue":"05","key":"ref87","doi-asserted-by":"crossref","first-page":"e858","DOI":"10.1111\/j.1365-2516.2011.02516.x","article-title":"A systematic review of the management and outcomes of pregnancy in Glanzmann thrombasthenia","volume":"17","author":"S Siddiq","year":"2011","journal-title":"Haemophilia"},{"issue":"12","key":"ref88","doi-asserted-by":"crossref","first-page":"e27376","DOI":"10.1002\/pbc.27376","article-title":"From thrombasthenia to next generation thrombocytopenia: neonatal alloimmune thrombocytopenia induced by maternal Glanzmann thrombasthenia","volume":"65","author":"A A Barg","year":"2018","journal-title":"Pediatr Blood Cancer"},{"issue":"22","key":"ref89","doi-asserted-by":"crossref","first-page":"2398","DOI":"10.1182\/blood.2022017296","article-title":"iPLAT1: the first-in-human clinical trial of iPSC-derived platelets as a phase 1 autologous transfusion study","volume":"140","author":"N Sugimoto","year":"2022","journal-title":"Blood"},{"issue":"22","key":"ref90","doi-asserted-by":"crossref","first-page":"2314","DOI":"10.1182\/blood.2022018291","article-title":"Engineered platelets for clinical application: a step closer","volume":"140","author":"L Xia","year":"2022","journal-title":"Blood"},{"issue":"01","key":"ref91","doi-asserted-by":"crossref","first-page":"49","DOI":"10.1016\/j.stemcr.2019.11.011","article-title":"iPSC-derived platelets depleted of HLA Class I are inert to anti-HLA Class I and natural killer cell immunity","volume":"14","author":"D Suzuki","year":"2020","journal-title":"Stem Cell Reports"},{"key":"ref92","doi-asserted-by":"crossref","first-page":"47","DOI":"10.1038\/nature15218","article-title":"Nanotechnology: platelet mimicry","volume":"526","author":"O C Farokhzad","year":"2015","journal-title":"Nature"},{"issue":"10","key":"ref93","doi-asserted-by":"crossref","first-page":"16292","DOI":"10.1021\/acsnano.2c05306","article-title":"Platelet-inspired intravenous nanomedicine for injury-targeted direct delivery of thrombin to augment hemostasis in coagulopathies","volume":"16","author":"A Girish","year":"2022","journal-title":"ACS Nano"},{"issue":"07","key":"ref94","doi-asserted-by":"crossref","first-page":"1535","DOI":"10.1111\/jth.15734","article-title":"Platelet-inspired nanomedicine in hemostasis thrombosis and thromboinflammation","volume":"20","author":"S Raghunathan","year":"2022","journal-title":"J Thromb Haemost"},{"issue":"07","key":"ref95","doi-asserted-by":"crossref","first-page":"690","DOI":"10.1080\/09537104.2018.1475633","article-title":"Advancement of platelet-inspired nanomedicine","volume":"29","author":"S Wang","year":"2018","journal-title":"Platelets"},{"issue":"01","key":"ref97","doi-asserted-by":"crossref","first-page":"279","DOI":"10.1182\/bloodadvances.2019001140","article-title":"Concizumab: a novel anti-TFPI therapeutic for hemophilia","volume":"5","author":"A D Shapiro","year":"2021","journal-title":"Blood Adv"},{"issue":"02","key":"ref98","doi-asserted-by":"crossref","first-page":"240","DOI":"10.1111\/bjh.18495","article-title":"Long-term safety and efficacy of the anti-tissue factor pathway inhibitor marstacimab in participants with severe haemophilia: Phase II study results","volume":"200","author":"J Mahlangu","year":"2023","journal-title":"Br J Haematol"},{"issue":"05","key":"ref99","doi-asserted-by":"crossref","first-page":"702","DOI":"10.1111\/hae.14595","article-title":"Befovacimab, an anti-tissue factor pathway inhibitor antibody: Early termination of the multiple-dose, dose-escalating Phase 2 study due to thrombosis","volume":"28","author":"M E Mancuso","year":"2022","journal-title":"Haemophilia"},{"issue":"06","key":"ref100","doi-asserted-by":"crossref","first-page":"1436","DOI":"10.1111\/jth.15270","article-title":"Targeting of antithrombin in hemophilia A or B with investigational siRNA therapeutic fitusiran - results of the phase 1 inhibitor cohort","volume":"19","author":"K J Pasi","year":"2021","journal-title":"J Thromb Haemost"}],"container-title":["H\u00e4mostaseologie"],"original-title":[],"language":"de","link":[{"URL":"http:\/\/www.thieme-connect.de\/products\/ejournals\/pdf\/10.1055\/a-2080-6602.pdf","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2023,12,19]],"date-time":"2023-12-19T17:13:55Z","timestamp":1703006035000},"score":1,"resource":{"primary":{"URL":"http:\/\/www.thieme-connect.de\/DOI\/DOI?10.1055\/a-2080-6602"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2023,8]]},"references-count":97,"journal-issue":{"issue":"04","published-online":{"date-parts":[[2023,8,23]]},"published-print":{"date-parts":[[2023,8]]}},"URL":"https:\/\/doi.org\/10.1055\/a-2080-6602","archive":["Portico","CLOCKSS"],"relation":{},"ISSN":["0720-9355","2567-5761"],"issn-type":[{"value":"0720-9355","type":"print"},{"value":"2567-5761","type":"electronic"}],"subject":[],"published":{"date-parts":[[2023,8]]}}}