Research collected from across the U.S. Shared all over the world.

 
 

Hemophilia A Studies

Hemophilia Utilization Group Study Part VIII (HUGS VIII): Costs and Impact of Disease in People with Hemophilia A

Study Objective

To examine the cost and burden of hemophilia care, including quality of life, arthropathy, economic, and psychosocial impact on people with hemophilia A.


Inclusion criteria:

  1. Age 2 years or older;

  2. Persons with factor VIII activity level <5%;

  3. Persons who have been received the majority their hemophilia care at the HTC for both one year prior to the enrollment and current care to ensure the HTC can collect their data through the study period;

  4. For the inhibitor group, persons who have: (1) an inhibitor titer >1 Bethesda unit (BU) recorded at any HTC visit and/or (2) the participant was reported to have been receiving immune tolerance therapy at any visit;

  5. Persons who speak either English or Spanish;

  6. Persons who are willing to provide written informed consent;


Exclusion Criteria:

  1. Persons judged to be cognitively impaired as determined by the clinician;

  2. Persons who have any additional bleeding disorder.

 
 

Hemophilia B Studies

HUGS V:Hemophilia Costs and Impact of Disease Study (closed)

Study Objective

The primary objective of this proposal was to examine prospectively the cost and burden of illness hemophilia, including arthropathy, quality of life, and economic impact on patients.

Inclusion criteria for participation:

  1. Age 2 to 64 years;

  2. FIX deficiency less than or equal to 30%, with or without history of inhibitor;

  3. Receiving 90% of hemophilia care by the HTC;

  4. English or Spanish speaking;

  5. Seen at the HTC within two years prior to the initiation of the study;

  6. Willingness to provide informed consent or assent.

 

Exclusion criteria:

  1. Subjects judged to be cognitively impaired as determined by the clinician;

  2. Under age 2;

  3. Additional bleeding disorder;

  4. Failure to provide informed consent or assent.

 
 

Other Factor Deficiency Studies

 

Von Willebrand Disease

Costs of Illness and Impact of Von Willebrand Disease: A Pilot Study

Study Objectives:

The primary objective of the proposed pilot study is to prospectively examine the cost and burden of illness in patients with three major types of von Willebrand Disease (VWD). Medication treatment pattern, bleeding episodes, quality of life, and economic impact on patients will be assessed.

Providing efficient services to beneficiaries with costly, lifelong diseases like VWD requires information concerning the cost of care for the disease and identification of effective approaches to treatment for these specific patients. An understanding of the variations in approaches to treatment, outcomes of care, and variations in the cost of illness will impact individuals and their families, as well as assist in policy decisions that insure adequate provision of care to patients while maximizing patient health status.

We are proposing to collect data from both patients and physicians in order to measure patient characteristics and their clinical characteristics, which are directly or indirectly associated with the cost of care for patients with VWD at eight treatment centers in eight states. This research will be the next step in linking patients and their clinical characteristics with clinically relevant primary health outcomes with VWD, including factor utilization, quality of life and functional and work status. The data collected will also help update the cost of illness to reflect current trends in VWD care. Specifically this study will assess the economic and time burden of the disease on the patient.

Inclusion criteria for participation:

  1. Patients who have a diagnosis of VWD with type 2 (2A, 2B, 2M, 2N) OR type 3; OR

  2. Patients who have a diagnosis of VWD type 1 with VWF activity: Ag and/or RCo <30 IU/dL; OR

  3. Patients (25% of study enrollment in each participating center) who have a diagnosis of VWD type 1 with VWF activity: Ag and/or RCo ≥30 IU/dL and <50IU/dl, AND BAT positive or abnormal BS is ≥4 in adult males, ≥6 in adult females and ≥3 in children [8];

  4. Patients who have received factor concentrate, desmopressin (1-deamino-8-D-arginine vasopressin, abbreviated DDAVP) or hormones or antifibrinolytics for treatment of bleeding in the last 12 months; OR Patients who need treatment, i.e. planned surgical procedure, in the next 12 months;

  5. Patients whose VWD hematologic care directed by the VWD participating data collection centers to ensure that data collection centers can collect participant data through the study period;

  6. Patients were cared at the recruiting data collection centers at least one year prior to enrollment to this study to ensure that the clinician at the centers have provided care for the participant;

  7. Patients who are 12 years of age and older;

  8. Patients who speak or read English or Spanish;

  9. Patients who provide written informed consent/assent.

 

Exclusion criteria:

  1. Patients judged to be cognitively impaired as determined by the clinician;

  2. Patients judged to be having inhibitors or thromboembolic disease as determined by the clinician;

  3. Patients  who are using any immunosuppressive therapy or anti-platelet therapy at the enrollment time;

  4. Patients with an additional bleeding disorder;

  5. Participation in any interventional clinical trials in the past 6 months.

 
 

Sickle Cell Disease

Costs and Impact of Disease in People with Sickle Cell Disease: A Pilot Study

Specific Aims/Objectives:

  1. To develop and implement a standardized data collection tool to collect patient reported quality of life (QoL), pain, psychosocial, and disease impact in persons with sickle cell disease (SCD).

  2. To develop and implement a standardized data collection tool to collect clinical data from chart review in persons with SCD.

  3. To evaluate the quality of life, pain, psychosocial, and disease impact, and health costs from persons with SCD receiving care from two pilot sickle cell treatment centers.

Inclusion criteria:

  1. Age 2 years or older;

  2. Persons with a diagnosis of SCD;

  3. Persons who have received their SCD care at a SCD treatment center at least one year prior to the enrollment to ensure SCD treatment centers can collect the participants' data through the study period;

  4. Persons who speak either English or Spanish;

  5. Persons who provide written informed consent.

 

Exclusion Criteria:

  1. Persons judged to be cognitively impaired as determined by the clinician;

  2. Persons who have any additional blood disorder.