Have you or your loved ones been diagnosed with hemophilia a?
You may be eligible to participate in a hemophilia a clinical trial.
Have you or your loved ones been diagnosed with hemophilia a? You may be eligible to participate in a hemophilia a clinical trial.
What is a clinical trial? Is participating in a clinical trial right for you? Learn more
Hemophilia A Clinical Trial in Scorrano
Have you or your loved ones been diagnosed with hemophilia a?
You may be eligible to participate in a hemophilia a clinical trial.
Have you or your loved ones been diagnosed with hemophilia a? You may be eligible to participate in a hemophilia a clinical trial.
Active not recruiting
Male & Female
All ages
The purpose of the study is to document the natural history of hemophilia A disease and long-term outcomes in terms of effectiveness, safety and quality of life in participants receiving Antihemophilic Factor (Recombinant) - Plasma/Albumin Free Method (rAHF-PFM) or Antihemophilic Factor (Recombinant) - Pegylated (rAHF-PEG) in routine clinical practice
Details for the study
Population
The study population will include male and female participants of any race and age who have a diagnosis of hemophilia A (Factor VIII (FVIII) =5%). Participants must have been prescribed rAHF-PFM or rAHF-PEG for the management of hemophilia A by the treating physician prior to the decision to enroll in the study.
Brief Title
ADVATE/ ADYNOVI Hemophilia A Outcome Database (AHEAD)
Official Title
ADVATE/ ADYNOVI Hemophilia A Outcome Database
Brief Summary
The purpose of the study is to document the natural history of hemophilia A disease and <br /> long-term outcomes in terms of effectiveness, safety and quality of life in participants <br /> receiving Antihemophilic Factor (Recombinant) - Plasma/Albumin Free Method (rAHF-PFM) or <br /> Antihemophilic Factor (Recombinant) - Pegylated (rAHF-PEG) in routine clinical practice
Treatments and/or Procedures
ADYNOVI
Antihemophilic Factor (Recombinant) Pegylated
ADVATE
Antihemophilic Factor (Recombinant) - Plasma/Albumin Free Method
Outcome Measures
Outcome measures are the tests that investigators perform to prove whether or not a treatment being tested in a clinical trial is having any effect.
Primary
Joint Health Outcomes - Assessed by Physical Exam Using Only the Pain, Bleeding, and Physical Exam Parameters of the Gilbert Scale
The World Federation of Hemophilia developed a musculoskeletal evaluation system, commonly referred to as the Gilbert test, to measure hemophilia joint health status.The Gilbert test needs to be performed in the absence of acute bleed, acute pain, and acute inflammation into the evaluated joint. Four parameters are used in each Gilbert test: pain (score: 0-3), bleeding (score: 0-3), physical exam (score: 0-12), and X-ray evaluation (score: 0-13) Scores of 0, represent no pain, no bleeding, no physical exam issues, and/or no x-ray issues. Higher scores for each of these categories represents worsening conditions.
Secondary
Quality of Life: PedHAL questionnaire - for pediatric patients
The PedHAL measures activities involving the upper extremities, basic activities involving the lower extremities and complex activities involving the lower extremities as well as an overall physical activity score for children. For participants 4-13 years of age: - PedHAL (parent version) For participants 14-17 years of age: - PedHAL (child version)
Secondary
Quality of Life: SF-10 questionnaire - for pediatric patients
The SF-10 measures generic health-related quality of life for children and is parent-completed.
Secondary
Quality of Life: EQ-5D (14 and up) questionnaire - for pediatric patients
The EQ-5D measures health utility in subjects aged 14 and up.
Secondary
Chronic pain associated with hemophilia, as measured over a period of 4 weeks on an annual basis, using the visual analog scale (VAS)
The VAS assesses the pain using a scale of 0 (no pain) to 10 (unbearable pain). During screening visit and on an annual basis, the investigators shall ask participants to rate the average level of chronic pain associated with hemophilia over the period of 4 weeks prior to visit date using the VAS.
Secondary
Acute pain associated with hemophilia, as measured with individual bleeding episodes, using the visual analog scale (VAS)
The VAS assesses the pain using a scale of 0 (no pain) to 10 (unbearable pain). Participants will be asked to provide ratings on level of acute pain associated with each bleeding episode using the VAS. The VAS scores will be recorded in the participant diary.
Secondary
Modalities of switching from a standard FVIII product to rAHF-PEG - 2
Difference in number of weekly doses between previous regimen and rAHFPEG
Secondary
Incidence of inhibitors after switching to rAHF-PEG
Incidence of inhibitors after switching to rAHF-PEG in the same subgroups of patients
Secondary
Quality of Life: EQ-5D questionnaire - for adult patients
The EQ-5D measures health utility in adult participants.
Secondary
Modalities of switching from a standard FVIII product to rAHF-PEG - 1
Difference in number of weekly prophylactic infusions between previous regimen and rAHF-PEG
Secondary
Quality of Life: SF-12v2 questionnaire - for adult patients
The SF-12v2 measures generic health-related quality of life for adults.
Secondary
Compliance with the dosing prescribed and its relationship with effectiveness
Evaluation of patients´ compliance to prescribed prophylactic treatment will be performed by the treating physician. Compliance will be categorized according to a 4-point table (Highly compliant, Fairly compliant, Moderately compliant, Poorly compliant)
Secondary
Annualized Bleed Rate, All Joints
The annualized bleed rate for all joints will be calculated per participant and summarized over the set of available participants with a minimum observation period of 90 days per treatment regimen.
Secondary
Annualized Bleed Rate, All Bleeds
The annualized bleed rate for all bleeds will be calculated per participant and summarized over the set of available participants.with a minimum observation period of 90 days per treatment regimen.
Secondary
Annualized bleed rate, pre-existing target joints at baseline
The annualized bleed rate for pre-existing target joints at baseline will be calculated per participant and summarized over the set of available participants with a minimum observation period of 90 days per treatment regimen.
Secondary
Quality of Life: HAL questionnaire - for adult patients
The the lHAL measures activities involving the upper extremities, basic activities involving ower extremities and complex activities involving the lower extremities as well as an overall physical activity score for adults.
Secondary
Status of joint health by X-ray by Pettersson scale
The status of joint health by X-ray by Pettersson score will be summarized for each observational year.
Secondary
Status of Joint Health by Magnetic Resonance Imaging (MRI) Scoring System- Using The Lund Scoring System (LSS)
LSS score format= A(e:s:h). Sum of values for Subchondral Cyst (score: 1-6), irregularity/erosion of Subchondral Cortex (score: 1-4), and Chondral Destruction (score: 1-6) gives value for the A component of score. e, s, h components represent effusion/hemarthrosis, hypertrophic synovial, & hemosiderin deposition (score: 0-4 for each). Max. score is 16(4:4:4). Subchondral Cyst: ≥1 bone ≥2 bones >3 cysts in ≥1 bone >3 cysts ≥2 bones Largest size >4 mm: ≥1 bone Largest size >4 mm: ≥2 bones Subchondral Cortex ≥1 bone ≥2 bones Involve > half joint surface: ≥1 bone Involve > half of joint surface: ≥2 bones Chondral Destruction ≥1 bone ≥2 bones Full thickness defect (FTD): ≥1 bone FTD: ≥2 bones FTD involves >1/3 of joint surface: ≥1 bone FTD involves >1/3 of joint surface: ≥2 bones Effusion/hemarthrosis (e): Hypertrophic synovial (s): Hemosiderin (h): (0-4 for each): 0 absent 1 equivocal 2 small 3 moderate 4 large
Secondary
Incidence of New Target Joints
The incidence of new target joints will be calculated as the total number of new target joints in all participants divided by the total number of observation days.
Secondary
Overall effectiveness assessment for prophylaxis therapy
Excellent: Same or lower breakthrough bleed rate (BBR) within last 12 months (M) compared with prior prophylaxis; if participant did not receive prior prophylaxis with rAHF-PFM, rAHF-PEG or other Factor VIII (FVIII), same or better than expected outcome according to investigator's expectation Good: Minor increase in BBR within last 12M compared with prior prophylaxis; if participant did not receive prophylaxis with rAHF-PFM, rAHF-PEG or other FVIII, slightly less than expected outcome according to investigator's expectation Fair: Moderate increase in BBR in last 12M compared with prior prophylaxis; if participant did not receive prophylaxis with rAHF-PFM, rAHF-PEG or other FVIII, somewhat less than expected outcome according to investigator's expectation Poor: Significant increase in BBR in the 12M compared with prior prophylaxis; if participant did not receive prophylaxis with rAHF-PFM, rAHF-PEG or other FVIII, little to no benefit according to investigator's expectation
Secondary
Overall effectiveness assessment for on-demand treatment
Excellent: Bleed episodes typically respond to same or fewer number of infusion and same or lower dose as compared with previous on-demand treatment or investigator's expectation Good: Most bleed episodes typically respond to same number of infusion and dose but some require more infusions or higher dose as compared with previous on-demand treatment or investigator's expectation Fair: Bleed episodes typically require more infusions and/or higher dose than expected as compared with previous on-demand treatment or investigator's expectation Poor: Bleed episodes routinely fail to respond to same number of infusion and dose and require additional or different factor concentrate for hemostatic control as compared with previous on-demand treatment or investigator's expectation
Secondary
Global effectiveness assessment for on-demand treatment
Excellent: Full relief of pain and cessation of bleeding as evidenced by objective signs (e.g., swelling, tenderness, irritability, inconsolability, and decreased range of motion in the case of musculoskeletal hemorrhage) within approximately 8 hours of a single infusion. No additional infusion is required for the control of bleeding. Administration of further infusions to maintain hemostasis would not affect this scoring. Good: Definite pain relief and/or improvement in signs of bleeding within approximately 8 hours after the infusion. Possibly requires more than 1 infusion for complete resolution. Fair: Probable or slight relief of pain and slight improvement in signs of bleeding within approximately 8 hours after the infusion. Requires more than 1 infusion for complete resolution. Poor: No improvement or condition worsens.
Secondary
Number of rAHF-PFM or rAHF-PEG units required for bleed cessation
Antihemophilic Factor (Recombinant) - Plasma/Albumin Free Method (rAHF-PFM) Antihemophilic Factor (Recombinant) - Pegylated (rAHF-PEG)
Secondary
Number of rAHF-PFM or rAHF-PEG infusions required for bleed cessation
Antihemophilic Factor (Recombinant) - Plasma/Albumin Free Method (rAHF-PFM) Antihemophilic Factor (Recombinant) - Pegylated (rAHF-PEG)
Secondary
Status of joint health using the Hemophilia Joint Health Score (HJHS)
The International Prophylaxis Study Group (IPSG) developed a scoring system for musculoskeletal evaluation, the HJHS, optimized for use in children with no or minimal joint disease. The HJHS includes the following parameters: swelling, duration of swelling, muscle atrophy, joint pain, crepitus on motion, flexion loss, extension loss, strength and global gait.
Study Criteria
Inclusion Criteria: - Participant has hemophilia A {FVIII lesser than or equal to (<=)5%} - Participant is prescribed Antihemophilic Factor (Recombinant) - Plasma/Albumin Free Method (rAHF-PFM) or Antihemophilic Factor (Recombinant) - Pegylated (rAHF-PEG) by the treating physician - Participant or participant's legally authorized representative provides informed consent Exclusion Criteria: - Participant has known hypersensitivity to the active substance or any of the excipients - Participant has known allergic reaction to mouse or hamster proteins - Participant has participated in another clinical study involving an investigational product (IP) or device within 30 days prior to study enrollment or is scheduled to participate in another clinical study involving another FVIII concentrate or device during the course of this study