Construction of a Health-related Quality of Life (HR-QOL) Questionnaire for Patients With Primary Antibody Deficiency Disease

NCT02542228 | Completed

• 1 other identifier: 6119806000
• Study Type: observational
• Target: 76
• Locations: 0 countries
• Sites: N/A

Brief 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.

Conditions

Immune Deficiency, Antibody

Keywords

Immune Deficiency, antibody; Quality of Life survey

Outcome Measures

Primary Outcomes (1)

• Develop and validate a disease specific Quality of Life Questionnaire for patients with primary antibody immune deficiency disease (PIDD)

  A 28 item questionnaire will be administered to patients with antibody immune deficiency using a 5 point Likert type scale. The questions were generated by clinical immunologist, infusion nurses with feedback from patients. The questions cover all of the SF-36 QOL instrument except body pain. Paired questionnaires were collected 48 to 120 hrs apart from each patient. Rasch analysis will be used to analysis the performance of the questionnaire for disordered items, lack of discrimination between item questions, overlap of questions, and the spread of the 5 point scale that will lead to item reduction. The primary outcome is to generate a validated HR-QOL questionnaire for patients with primary antibody deficiency that will be useful for clinical care and potentially for clinical trials.

  one year

Interventions

questionnaire survey OTHER

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.

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Patients with primary antibody immune deficiency with x-linked agammaglobulinemia and common variable immune deficiency

You may qualify if:

• Subjects with documented x-linked agammaglobulinemia, or common variable immunodeficiency, autosomal recessive agammaglobulinemia
• Subjects must be able to read and speak English
• Willing to sign consent and follow study schedule.
• Subjects 18 years to 65 years of age.
• Subject must be receiving Ig treatment for diagnosis.

You may not qualify if:

• Subject or guardian unwilling to sign consent or adhere to study schedule.
• Pregnancy,
• Severe concurrent medical conditions which would prevent treatment or assessment, including significant hematological, renal, lung or liver dysfunction or malignancies
• Presence of any other medical condition, which in the opinion of the investigator might interfere with performance or interpretation of this study
• Can not read or understand English

Sponsors & Collaborators

• University of South Florida: lead
• University of Virginia: collaborator
• Boston Children's Hospital: collaborator
• Baylor College of Medicine: collaborator
• Johns Hopkins University: collaborator
• Midwest Immunology Clinic: collaborator
• CSL Behring: collaborator

Related Publications (1)

• Ballow M, Conaway MR, Sriaroon P, Rachid RA, Seeborg FO, Duff CM, Bonilla FA, Younger MEM, Shapiro R, Burns TM. Construction and validation of a novel disease-specific quality-of-life instrument for patients with primary antibody deficiency disease (PADQOL-16). J Allergy Clin Immunol. 2017 Jun;139(6):2007-2010.e8. doi: 10.1016/j.jaci.2016.11.029. Epub 2017 Jan 5. No abstract available.

  PMID: 28065678 DERIVED

MeSH Terms

Conditions

Immunologic Deficiency Syndromes

Interventions

Surveys and Questionnaires

Condition Hierarchy (Ancestors)

Immune System Diseases

Intervention Hierarchy (Ancestors)

Data Collection; Epidemiologic Methods; Investigative Techniques; Health Care Evaluation Mechanisms; Quality of Health Care; Health Care Quality, Access, and Evaluation; Public Health; Environment and Public Health

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 31, 2015
• First Posted: September 4, 2015
• Study Start: March 1, 2014
• Primary Completion: January 1, 2016
• Study Completion: September 1, 2016
• Last Updated: January 13, 2017

Record last verified: 2017-01