GAS-Hem Feasibility Study
Condition: Hemophilia A
MeSH terms: Hemophilia A
Study type: Observational

Summary: 
      The purpose of this study is to investigate the feasibility and acceptability of the
      Standardized Goal Attainment Scaling menu for Hemophilia (GAS-Hem) as a patient reported
      outcome (PRO) measure to monitor clinical progress in participant-identified goal areas in
      individuals with hemophilia A.
    

Detailed description: 
      Hemophilia A is an X-linked recessive, congenital bleeding disorder caused by deficient or
      defective coagulation factor VIII (FVIII). The absence of FVIII leads to 'spontaneous'
      bleeding episodes (occurring primarily in joints, muscles and, less commonly, in soft
      tissues) and to excessive bleeding following trauma or injury. With proper treatment (FVIII)
      and self-care, most hemophilia patients can maintain an active, productive lifestyle. With a
      growing appreciation that a low bleed rate sometimes is only achieved, in part, by patients
      limiting their activities, there has been growing interest in understanding how hemophilia
      may still limit life despite absence of major bleeds. The current study will introduce and
      test a standardized method for individualized outcome measurement based on Goal Attainment
      Scaling (GAS), a method that allows participants to set goals to address challenges that are
      meaningful to them and monitor attainment in those areas over time. The Standardized Goal
      Attainment Scaling menu for Hemophilia (GAS-Hem) is an online instrument for setting and
      tracking personalized goals related to hemophilia. The goal menu captures common challenges
      faced by people with hemophilia, accompanied by an inventory of descriptors that can be
      personalized. The tool can be used to track the health of individuals across a wide spectrum
      of ages, life experiences and health states. This is a 12-week, multicenter, prospective,
      observational, feasibility study of up to 60 people (aged 5 and 65) with moderate to severe
      hemophilia A to investigate the feasibility and acceptability of GAS-Hem as a patient
      reported outcome (PRO) measure to monitor clinical progress in participant-identified goal
      areas. Outcomes, including GAS-Hem (primary), health-related quality of life, bleeding
      episodes (location, cause, time) and treatment (time, dose and reason for infusion) will be
      assessed at screen/baseline, with follow-up at 6 and 12 weeks. Endpoints of interest include
      participant GAS-Hem outcomes at 6 and 12 weeks, participant quality of life (QoL) outcomes at
      6 and 12 weeks, as well as participant and clinician perception of the GAS-Hem as a useful
      means of identifying and monitoring progress in relevant goal areas.
    

Intervention name(s): Factor VIII
Intervention type(s): Biological
Intervention description(s): Licensed Factor VIII products. Study is brand agnostic
Other intervention name(s): FVIII
Intervention MeSH terms: Factor VIII
Observational model: Cohort
Time perspective: Prospective
Sampling method: Non-Probability Sample
Study population: 
        Patients with moderate to severe hemophilia A in the United States or Canada.
      
Keywords: Goal setting; Feasibility; Standardized Goal Attainment Scaling menu for Hemophilia (GAS-Hem); Patient reported outcome (PRO); Goal attainment scaling (GAS)