Hemophilia A, Hemophilia B
Conditions
Keywords
Hemophilia A Factor VIII, Hemophilia B Factor IX, Cross-sectional, Epidemiological, Clinical practice, United States
Brief summary
Study Design A prospective observational, cross-sectional epidemiological study in U.S. site-based clinical practice settings. 30 sites will enroll approximately 300 patients Participating patients - or their caregiver in the case of patients under the age of 18 - will be consented to participate. Physicians complete a retrospective chart review on each enrolled patient. Patients will complete a one-time study questionnaire.
Detailed description
Study recruitment was stopped due to difficulty in enrolling the targeted number of patients on March 21, 2018. Subjects currently enrolled into the study have completed the study as per protocol. There were no safety concerns involved in the decision to stop enrollment.
Interventions
DRUGHemophilia B standard half-life
Benefix
DRUGHemophilia B extended half-life
Alprolix
DRUGHemophilia A standard half-life
Xyntha and other standard half-life agents
DRUGHemophilia A extended half-life
Eloctate and Adynovate
Sponsors
Pfizer
Study design
Observational model
COHORT
Time perspective
OTHER
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
Inclusion criteria
Physician/Clinician Participants: * Must be a healthcare provider * Currently manages at least 10 hemophilia A and/or B patients Patient Participants: * Willing and able to provide informed consent * Diagnosed with hemophilia A or B * Current disease severity is either moderately severe or severe with a clotting factor level of ≤5% * If suffering from hemophilia A, must be currently taking moroctocog alfa (or another standard half-life treatment), rurioctocog alfa or efraloctocog alfa for at least six months. (If currently taking rurioctocog alfa or efraloctocog alfa, must have been switched from a standard half-life treatment and had been on that prior treatment for at least six months). * If suffering from hemophilia B, must be currently taking nonacog alfa or eftrenonacog alfa for at least six months (If currently taking eftrenonacog alfa, must have switched from nonacog alfa and had been on that prior treatment for at least six months). * Infuse at least 3 times per month
Exclusion criteria
* Female with hemophilia A or B * Mild Haemophilia A or B
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Resource Utilization Pattern | From baseline up to end of study (6 months) |
| Dosing Pattern | From baseline up to end of study (6 months) |
Countries
United States
Participant flow
Recruitment details
Out of 41 screened participants, 11 were enrolled in this study. Study got terminated afterwards due to difficulty in enrolling the targeted number of participants.
Participants by arm
| Arm | Count |
|---|---|
| Hemophilia A Cohort Participants diagnosed with Hemophilia A (disease severity either moderate: clotting factor level of less than and equal to \[\<=\]5 percent\[%\] or severe: clotting factor level of less than \[\<\]1%), and taking Xyntha or another standard half-life treatment (Adynovate or Eloctate) for at least 6 months (if taking Adynovate or Eloctate, must have been switched from a standard half-life treatment and had been on that prior treatment for at least six months and must have infused at least 3 times per month) were observed in this study for up to 6 months. | 11 |
| Hemophilia B Cohort Participants diagnosed with Hemophilia B (disease severity either moderate: clotting factor level \<=5% or severe: clotting factor level of \<1%), and taking BeneFIX or Alprolix for at least 6 months (if received Alprolix, must had switched from BeneFIX and had been on that prior treatment for at least 6 months and must have infused at least 3 times per month), were observed in this study for up to 6 months. | 0 |
| Total | 11 |
Baseline characteristics
| Characteristic | Hemophilia A Cohort | Hemophilia B Cohort | Total |
|---|---|---|---|
| Age, Categorical <=18 years | 2 Participants | 0 Participants | 2 Participants |
| Age, Categorical >=65 years | 0 Participants | 0 Participants | 0 Participants |
| Age, Categorical Between 18 and 65 years | 9 Participants | 0 Participants | 9 Participants |
| Race and Ethnicity Not Collected | — | — | 0 Participants |
| Sex: Female, Male Female | 0 Participants | 0 Participants | 0 Participants |
| Sex: Female, Male Male | 11 Participants | 0 Participants | 11 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 11 | 0 / 0 |
| other Total, other adverse events | 0 / 11 | 0 / 0 |
| serious Total, serious adverse events | 0 / 11 | 0 / 0 |
Outcome results
Primary
Dosing Pattern
Time frame: From baseline up to end of study (6 months)
Population: Data was not collected and analyzed for this outcome measure since very less number of participants were enrolled prior to study termination.
Primary
Resource Utilization Pattern
Time frame: From baseline up to end of study (6 months)
Population: Data was not collected and analyzed for this outcome measure since very less number of participants were enrolled prior to study termination.