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- Title
Peri‐operative desmopressin combined with pharmacokinetic‐guided factor VIII concentrate in non‐severe haemophilia A patients.
- Authors
Romano, Lorenzo G. R.; Schütte, Lisette M.; van Hest, Reinier M.; Meijer, Karina; Laros‐van Gorkom, Britta A. P.; Nieuwenhuizen, Laurens; Eikenboom, Jeroen; Heubel‐Moenen, Floor C. J. I.; Uitslager, Nanda; Coppens, Michiel; Fijnvandraat, Karin; Driessens, Mariëtte H. E.; Polinder, Suzanne; Cnossen, Marjon H.; Leebeek, Frank W. G.; Mathôt, Ron A. A.; Kruip, Marieke J. H. A.
- Abstract
Introduction: Non‐severe haemophilia A patient can be treated with desmopressin or factor VIII (FVIII) concentrate. Combining both may reduce factor consumption, but its feasibility and safety has never been investigated. Aim: We assessed the feasibility and safety of combination treatment in nonsevere haemophilia A patients. Methods: Non‐severe, desmopressin responsive, haemophilia A patients were included in one of two studies investigating peri‐operative combination treatment. In the single‐arm DAVID study intravenous desmopressin (0.3 μg/kg) once‐a‐day was, after sampling, immediately followed by PK‐guided FVIII concentrate, for maximally three consecutive days. The Little DAVID study was a randomized trial in patients undergoing a minor medical procedure, whom received either PK‐guided combination treatment (intervention arm) or PK‐guided FVIII concentrate only (standard arm) up to 2 days. Dose predictions were considered accurate if the absolute difference between predicted and measured FVIII:C was ≤0.2 IU/mL. Results: In total 32 patients (33 procedures) were included. In the DAVID study (n = 21), of the FVIII:C trough levels 73.7% (14/19) were predicted accurately on day 1 (D1), 76.5% (13/17) on D2. On D0, 61.9% (13/21) of peak FVIII:C levels predictions were accurate. In the Little DAVID study (n = 12), on D0 83.3% (5/6) FVIII:C peak levels for both study arms were predicted accurately. Combination treatment reduced preoperative FVIII concentrate use by 47% versus FVIII monotherapy. Desmopressin side effects were mild and transient. Two bleeds occurred, both despite FVIII:C > 1.00 IU/mL. Conclusion: Peri‐operative combination treatment with desmopressin and PK‐guided FVIII concentrate dosing in nonsevere haemophilia A is feasible, safe and reduces FVIII consumption.
- Subjects
HEMOPHILIACS; BLOOD coagulation factor VIII; DESMOPRESSIN; HEMOPHILIA treatment; HEMOPHILIA
- Publication
Haemophilia, 2024, Vol 30, Issue 2, p355
- ISSN
1351-8216
- Publication type
Article
- DOI
10.1111/hae.14946