Construction of a Health-related Quality of Life (HR-QOL) Questionnaire for Patients With Primary Antibody Deficiency Disease
Condition: Immune Deficiency, Antibody
MeSH terms: Primary Immunodeficiency Diseases; Deficiency Diseases; Immunologic Deficiency Syndromes; Common Variable Immunodeficiency
Study type: Observational

Summary: 
      This research is being done to construct a survey questionnaire, a quality of life tool, to
      determine the health, well-being, ability to perform daily activities, and physical, social
      and emotional functioning in participants with primary immunodeficiency disorders (PIDD).
      This quality of life (QOL) tool will help physicians understand the effects of PIDD on a
      person's health. These quality of life surveys are important tools for physicians to measure
      outcomes for satisfaction and effects of treatment.
    

Detailed description: 
      A preliminary patient and clinician-generated scale will be validated and modified during a
      multicenter scale validity study involving 85 patients from 5 sites. Feasibility surveys have
      been conducted and available sites that can be included in this study include Boston
      Children's/ Harvard, Midwest Immunology, Johns Hopkins, Children's Hospital at Baylor
      University, and the University of South Florida at All Children's Hospital Johns Hopkins
      Medicine. Patients will be enrolled at a clinic non-infusion visit. The purpose of the study
      will be explained and informed consent obtained. A case report form (CRF) of their clinical
      condition and demographics will be completed based on the information obtained by the local
      study coordinator during the interview at the time of the visit. The HR-QOL PIDD survey will
      be administered to the patient by the site PI or the study coordinator. A second survey will
      be handed to the patient to take home with them and completed at 48-120 hrs. after the clinic
      visit. The study coordinator will contact the patient during this period to ensure that the
      patient completes this second survey and returns it to the local study site. If a patient is
      on SCIg they will be asked to take the survey 3 days after the SC infusion (if weekly), and
      complete the paired survey 48-72 hrs later (before another SCIg infusion is scheduled). The
      study coordinator will obtain an interim history for the period between the paired surveys to
      ensure that, in general, the condition of the patient has not changed that could invalidate
      the validation process, and this information will be documented on the addendum case report
      form (CRF). Patient can return the surveys to the site study coordinator either by mail, fax
      or electronically by scanning the documents. Paired surveys will be returned to the central
      study site (USF St Petersburg) for data input.
    

Intervention name(s): questionnaire survey
Intervention type(s): Other
Intervention description(s): The HR-QOL PIDD survey will be administered to the patient by the site PI or the study coordinator. A second survey will be handed to the patient to take home with them and completed at 48-120 hrs. after the clinic visit. The study coordinator will contact the patient during this period to ensure that the patient completes this second survey and returns it to the local study site. A case report form (CRF) of their clinical condition and demographics will be collected at the time of informed consent.
Observational model: Cohort
Time perspective: Prospective
Sampling method: Non-Probability Sample
Study population: 
        Patients with primary antibody immune deficiency with x-linked agammaglobulinemia and
        common variable immune deficiency
      
Keywords: Immune Deficiency, antibody; Quality of Life survey