Peri-operative desmopressin combined with pharmacokinetic-guided factor VIII concentrate in non-severe haemophilia A patients.
Haemophilia
; 30(2): 355-366, 2024 Mar.
Article
in En
| MEDLINE
| ID: mdl-38343113
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 FVIIIC was ≤0.2 IU/mL.RESULTS:
In total 32 patients (33 procedures) were included. In the DAVID study (n = 21), of the FVIIIC 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 FVIIIC levels predictions were accurate. In the Little DAVID study (n = 12), on D0 83.3% (5/6) FVIIIC 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 FVIIIC > 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.Key words
Full text:
1
Collection:
01-internacional
Database:
MEDLINE
Main subject:
Hemostatics
/
Hemophilia A
Type of study:
Clinical_trials
/
Prognostic_studies
Limits:
Humans
Language:
En
Journal:
Haemophilia
Journal subject:
HEMATOLOGIA
Year:
2024
Document type:
Article
Affiliation country:
Country of publication: