title: Bleeding and surgery in children with Glanzmann thrombasthenia with and without the use of recombinant factor VII a.
abstract: BACKGROUND
  An inherited deficiency of platelet glycoprotein II b/III a (GP II b/III a), Glanzmann thrombasthenia, can lead to excessive bleeding and require platelet transfusion to secure hemostasis. Antibodies to GP II b/III a or HLA may platelet transfusion render ineffective to stop bleeding or to cover surgery. Recombinant factor VII a has been introduced as therapeutic alternative and has been suggested to be effective. PATIENTS AND AIMS OF THE STUDY: In a retrospective evaluation, bleeding episodes and surgery in six patients treated with antifibrinolytics and with and without the additional use of rFVII a were analysed to achieve informations for treatment indication and efficacy.
  
  
  RESULTS
  Nineteen mucosal and subcutaneous bleeding episodes, two dental surgeries and seven joint bleeds occurred. In 11 mild to moderate mucocutaneous bleeds treated without rFVII a, seven stopped within 48 hours, three stopped until the fourth day; one showed recurrence. Three bleeds were treated with rFVII a and responded within 24 hours. One severe bleed treated without rFVII a did not stop until the 8 (th) day after cautery. In 4 severe bleeds treated with rFVII a, one stopped within 24 hours, one showed recurrence, one was treated with platelet transfusion concurrently and one did not respond to rFVII a. Clinical signs persisted in one conservatively treated elbow joint bleed, whereas in two episodes treated with rFVII a, the bleeding responded within 5 and 7 days and in four episodes in at least 4 days. Two dental surgeries showed no recurrence after rFVII a over 18 or 36 hours.
  
  
  CONCLUSIONS
  In severe mucocutaneous bleeding episodes or joint bleeding rFVII a is of some benefit whereas in surgeries like teeth extraction, prophylactically administered rFVII a seems effective to avoid bleeding. In mild to moderate mucocutaneous bleeding events, antifibrinolytics and local measures were sufficient in most cases and the additional use of rFVII a does not seem to be necessary. Further information is needed to elaborate clear indications for the rational use of rFVII a in bleeding episodes in patients with Glanzmann thrombasthenia compared to standardized baseline treatment. This information may generate a prospective multicenter study to provide clear advice with respect to bleeding site, severity and duration.
authors.affiliation: Department of Pediatric Oncology, Hematology and Immunology, Heinrich-Heine-University Düsseldorf. hennewig@med.uni-duesseldorf.de
authors.forename: U
authors.initials: U
authors.lastname: Hennewig
authors.forename: H-J
authors.initials: HJ
authors.lastname: Laws
authors.forename: S
authors.initials: S
authors.lastname: Eisert
authors.forename: U
authors.initials: U
authors.lastname: Göbel
mesh_terms: D000293:Adolescent; D000933:Antifibrinolytic Agents / Q000627:therapeutic use; D001780:Blood Coagulation Tests; D002648:Child; D002675:Child, Preschool; D004305:Dose-Response Relationship, Drug; D004334:Drug Administration Schedule; D015942:Factor VIIa / Q000627:therapeutic use*; D005260:Female; D006395:Hemarthrosis / Q000097:blood / Q000188:drug therapy; D006470:Hemorrhage / Q000097:blood / Q000188:drug therapy* / Q000209:etiology; D006801:Humans; D008297:Male; D006472:Oral Hemorrhage / Q000097:blood / Q000188:drug therapy; D017713:Platelet Transfusion; D019106:Postoperative Hemorrhage / Q000097:blood / Q000188:drug therapy*; D011994:Recombinant Proteins / Q000627:therapeutic use; D012008:Recurrence; D012189:Retrospective Studies; D013915:Thrombasthenia / Q000097:blood / Q000188:drug therapy*; D014081:Tooth Extraction
chemical_list: D000933:Antifibrinolytic Agents; D011994:Recombinant Proteins; D015942:Factor VIIa