Desmopressin testing in haemo-philia A patients and carriers

R. Knöfler; J. Koscielny; J. T. Tauer; B. Huhn; A. Gneuss; E. Kuhlisch; A. Hofmann; S. Petros; V. Aumann; D. Franke; K. Kentouche; G. Syrbe; K. Seeger; H. Haberland; R. Klamroth

doi:10.5482/HAMO-12-06-0012

Hämostaseologie, Inhaltsverzeichnis

Hamostaseologie 2012; 32(04): 271-275
DOI: 10.5482/HAMO-12-06-0012

Original article

Schattauer GmbH

Desmopressin testing in haemo-philia A patients and carriers

Results of a multi centre surveyDesmopressin-Testung bei Patienten mit Hämophilie A und KonduktorinnenErgebnisse einer multizentrischen Studie

Authors

R. Knöfler

¹Department of Paediatric Haematology, Oncology and Haemostaseology, University Hospital Carl Gustav Carus Dresden, Germany
J. Koscielny

²Outpatient Clinic for Coagulation Disorders and Transfusion Medicine, Charité Medical University Berlin, Germany
J. T. Tauer

¹Department of Paediatric Haematology, Oncology and Haemostaseology, University Hospital Carl Gustav Carus Dresden, Germany
B. Huhn

¹Department of Paediatric Haematology, Oncology and Haemostaseology, University Hospital Carl Gustav Carus Dresden, Germany
A. Gneuss

¹Department of Paediatric Haematology, Oncology and Haemostaseology, University Hospital Carl Gustav Carus Dresden, Germany
E. Kuhlisch

³Department of Medical Statistics, Medical Faculty of Technical University Dresden, Germany
A. Hofmann

⁴Department of Paediatrics, Municipal Hospital Chemnitz, Germany
S. Petros

⁵Department of Internal Medicine, University Hospital Leipzig, Germany
V. Aumann

⁶Department of Paediatrics, University Hospital Magdeburg, Germany
D. Franke

⁷Practice of Internal Medicine and Angiology, Magdeburg, Germany
K. Kentouche

⁸Department of Paediatric Haematology and Oncology, University Hospital Jena, Germany
G. Syrbe

⁹Haemophilia Centre, Asklepios Hospital Stadtroda, Germany
K. Seeger

¹⁰Department of Paediatric Haematology and Oncology, Charité Medical University Berlin, Germany
H. Haberland

¹¹Department of Paediatrics, Sana Hospital Lichtenberg, Berlin, Germany
R. Klamroth

¹²Department of Angiology and Clotting Disorders, Haemophilia Treatment Center, Vivantes Hospital Friedrichshain, Berlin, Germany

Abstract

Summary

Introduction: Desmopressin (DDAVP) testing (DT) in patients (pts) with haemophilia A (HA) and carriers (CHA) is up to now not standardized. This prompted us to evaluate results of DT carried out between 1996 and 2011 in centres of the Competence Network Haemor-rhagic Diatheses East. Patients and method: An increase of the factor VIII activity (FVIII) above 50% or at least the two fold of initial values within 120 min after DDAVP was defined as complete response (CR). Data from 80 patients (31 children, 49 adults) of whom 64 suffered from HA (sub-HA: n = 48; mild: n = 14; moderate: n = 2) and 16 patients CHA were evaluated. Results: In 34 patients DDAVP was given i. v. (dose range: 0.26–0.6 μg/kg body weight, mean: 0.33), in 31 intranasally (i.n. 300–600 μg) and in 15 s. c. (15–40 μg). The maximal FVIII increase was reached 60 min after DDAVP. For i. v. application the mean FVIII increase was 3.1-fold, for i. n. 2.1-fold and for s. c. 2.4-fold. A CR was de tected in 71 patients, a non-response in 9. Mild side effects such as flush, headaches or nausea were observed in 11 patients (14%). Conclusion: For desmopressin testing in patients with haemophilia A and carriers i. v. application at 0.3 μg/kg body weight and the determination of FVIII before and 60 min after desmopressin infusion is recommended.

Zusammenfassung

Einleitung: Der Desmopressin(DDAVP)-Test (DT) bei Patienten mit Hämophilie A (HA) und Konduktorinnen (KHA) ist nicht standardisiert. Das veranlasste uns, die Desmopressin-tests der Zentren des Kompetenznetzwerks Hämorrhagische Diathesen Ost von 1996 bis 2011 zu analysieren. Patienten, Methode: Ein Faktor-VIII-Aktivitätsanstieg (FVIII) auf über 50% oder um mindestens das Doppelte der Initialwerte innerhalb von 120 min nach DDAVP wurde als komplette Response (KR) gewertet. Daten von 80 Patienten (31 Kinder, 49 Erwachsene), davon 64 mit HA (Sub: n = 48; leicht: n = 14; mittelschwer: n = 2) und 16 KHA wurden evaluiert. Ergebnisse: Bei 34 Patienten wurde DDAVP i. v. gegeben (Bereich: 0,26–0,6 μg/kg Körpergewicht, Mittel: 0,33), bei 31 intranasal (i. n.; 300–600 μg) und bei 15 s. c. (15–40 μg). Der maximale FVIII-Anstieg wurde 60 min nach DDAVP erreicht. Nach i.v.-Gabe war der mittlere FVIII-Anstieg 3,1-fach, nach i. n.-Applikation 2,1-fach und nach s.c.-Gabe 2,4-fach. Ein KR lag bei 71 Patienten und ein Non-Response bei 9 vor. Leichte unerwünschte Arzneimittelwirkungen wie Flush, Kopfschmerzen oder Übelkeit traten bei 11 Patienten (14%) auf. Schlussfolgerung: Bei Patienten mit Hämophilie A und Konduktorinnen wird zur Testung i.v. DDAVP 0.3 μg/kg Körpergewicht mit FVIII- Bestimmung vor und 60 min nach Infusion empfohlen.

Keywords

Haemophilia A - desmopressin testing - DDAVP

Schlüsselwörter

Hämophilie A - Desmopressintest - DDAVP

Volltext

Referenzen

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