DDAVP treatment combined with FVIII clotting factor concentrates in patients with mild hemophilia A.

Completed

Conditions: clotting factor VIII deficiency
Hemophilia A
10064477
10005330

Registration Number: NL-OMON47474

Lead Sponsor: Erasmus MC, Universitair Medisch Centrum Rotterdam

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Completed

Sex: Not specified

Target Recruitment: 50

Inclusion Criteria

- Non-severe hemophilia A patients (FVIII equal to or higher than 0.01 IU/mL)
- In need of surgery or suffering from bleeding
- Age minimally 12 and maximally 70 years at study inclusion date
- Need for clotting factor concentrates; perioperatively, or around a bleeding
- Treatment duration with FVIII-concentrates of at least 48 hours
- Results of FVIII levels after a DDAVP test dose, or if test results are not
available, willingness to undergo a DDAVP test
- Male gender
- (Parental) informed consent

Exclusion Criteria

- Patients with other congenital or acquired hemostatic abnormalities
- Very low response to DDAVP after 1 hour * absolute increase in FVIII < 0.2
IU/mL
- Clinically relevant FVIII inhibiting antibodies (>0.5 BU) in medical history
or preoperatively, unless successfully treated with immunotolerance therapy
- Start of FVIII-concentrate treatment >24 hours ago
- Contraindications for DDAVP, e.g. cardiovascular disease (see the protocol,
appendix IV)
- Use of co-medication that has an interaction with DDAVP (see the protocol,
appendix IV)
- Intolerance to previous DDAVP administrations
- DDAVP not advisable due to the type of surgery/bleeding according to the
hematologist and/or surgeon

Study & Design

Study Type: Observational invasive

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>The main endpoint will be the proportion of patients within FVIII target levels<br /><br>with DDAVP and FVIII concentrate combination treatment in the first 72 hours<br /><br>after start of combination treatment, without adding off-protocol FVIII<br /><br>concentrates.</p><br>

Secondary Outcome Measures

Name	Time	Method
<p>1. A population based pharmacokinetic model<br /><br>2. Number and nature of adverse events during combined treatment<br /><br>3. Incidence & severity of bleeding<br /><br>4. Incidence of thrombosis<br /><br>5. Incidence and extent of tachyphylaxis<br /><br>6. Economical evaluation<br /><br>7. Experienced quality of care</p><br>