Evaluation of Desmopressin Effect on Primary Haemostasis in Pediatric Patients with Aspirin-Like Defect as Hereditary Thrombocytopathy

Abstract

Objectives: Despite about 3 decades of clinical experience with the therapy of inherited thrombocytopathies (HTP) with desmopressin (DDAVP) the mechanisms of haemostatic effects of DDAVP in these diseases remain unclear. Therefore platelet function diagnostics was carried out in whole blood (WB) from children with aspirin-like defect as one of the clinically mild forms of HTP after DDAVP administration.

Design and methods: 11 children (age range: 3–16 years) were treated with DDAVP i. v. (0.3 μg/kg as short infusion). Before, after 120, and 240 min of DDAVP administration the following parameters were measured: platelet aggregation (PA) and ATP release induced by ADP, collagen, ristocetin and thrombin; PFA-100^® closure times (CT), factor VIII activity (FVIII:C), Von Willebrand factor antigen (VWF:Ag), collagen binding activity (VWF:CB) and blood count.

Results: PA, ATP release and blood count were not influenced by DDAVP administration. PFA-100^® CTs were markedly reduced at 120 and 240 min after DDAVP, respectively. FVIII:C, VWF:Ag and VWF:CB were increased after 120 min.

Conclusion: The DDAVP-induced improvement of primary haemostasis in patients with aspirin-like defect is mainly due to the marked increase of the VWF. For the evaluation of the clinical effect of DDAVP administration in patients with aspirin-like defect the investigation of a larger group of patients is needed.

Zusammenfassung

Einleitung: Obwohl seit 3 Jahrzehnten klinische Erfahrungen mit dem Einsatz von Desmopressin (DDAVP) bei Patienten mit hereditären Thrombozytopathien (HTP) bestehen, sind die hämostatischen DDAVP-Effekte bei diesen Erkrankungen unbekannt. Daher wurden Thrombozytenfunktionsuntersuchungen im Vollblut (VB) bei Kindern mit Aspirin-like-Defekt als einer der klinisch leichten Formen einer HTP nach DDAVP-Gabe durchgeführt.

Patienten und Methoden: 11 Kinder (3–16 Jahre) wurden vor, 120 und 240 min nach i. v. Gabe von DDAVP (0,3 μg/kg als Kurzinfusion) untersucht. Folgende Parameter wurden bestimmt: Vollblutaggregation (VBA) und ATP-Freisetzung induziert durch ADP, Kollagen, Ristocetin und Thrombin; PFA-100^® Verschlusszeiten (VZ), Faktor-VIII-Aktivität (FVIII:C), Von-Willebrand-Faktor-Antigen (VWF:Ag), Kollagenbindungsaktivität (VWF:CB) und Blutbildparameter (BP).

Ergebnisse: VBA, ATP-Freisetzung und BP wurden durch DDAVP nicht relevant beeinflusst. Die VZ waren 120 und 240 min nach DDAVP deutlich verkürzt. FVIII:C, VWF:Ag und VWF:CB zeigten nach 120 min erhöhte Werte.

Schlussfolgerung: Die DDAVP-induzierte Verbesserung der primären Hämostase bei Patienten mit Aspirin-like-Defekt ist vor allem auf den deutlichen Anstieg des VWF zurückführen. Für die Beurteilung des klinischen Effektes der DDAVP-Gabe bei Patienten mit Aspirin-like-Defekt ist die Untersuchung einer größeren Patientengruppe erforderlich.

Key words

desmopressin - platelet function - whole blood - thrombocytopathies

Schlüsselwörter

Desmopressin - Plättchenfunktion - Vollblut - Thrombozytopathien

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