Predicting the Quality of Response to Specific Treatments in Patients With cGVHD, PQRST Study

NCT04431479 | Recruiting

• 4 other identifiers: RG1006823NCI-2020-0124210360R01CA118953
• Study Type: observational
• Target: 300
• Locations: 2 countries
• Sites: 10

Brief Summary

This trial collects clinical data and blood samples to predict the quality of response to specific treatments in patients with chronic graft-versus-host disease (cGVHD) who are about to start a new therapy. Collecting and analyzing clinical data and blood samples from patients with cGVHD before and after treatment initiation may help doctors identify changes that may predict treatment response.

Conditions

Chronic Graft Versus Host Disease

Outcome Measures

Primary Outcomes (6)

• Clinical Response according to the 2014 criteria

  At the assessments, the 9 provider-reported National Institute of Health (NIH) organ severity scores (skin, eye, mouth, esophagus, upper gastrointestinal \[GI\], lower GI, liver, lung, and joint manifestations) will be collected reflecting disease activity in the past week. All scored items are single 4-7 point Likert scales. Based on past work, we anticipate it will take approximately 8 minutes to conduct the physical exam and record the relevant results. Pulmonary function testing results will be collected if available. Response will be assessed according to the recommendations of the 2014 NIH response measures publication or any applicable updates.

  Up to 6 months

• Time to next systemic treatment

  Any addition of another systemic cGVHD treatment for medical reasons will be considered a failure, whether added because of a new or worsening manifestation of cGVHD, used as a "steroid sparing agent," or substituted due to toxicity.

  From the start of the index medication until the addition of another systemic chronic graft versus host disease (cGVHD) treatment with death and treated recurrent malignancy considered competing events, assessed up to 3 years

• Duration of treatment

  Duration of treatment is defined as the time until discontinuation of therapeutic systemic immunosuppression (adrenal replacement and topical/local therapies are allowed) without resumption for at least 3 months.

  Up to 3 months

• Survival

  From the start of the index medication to death with patients lost to follow up or alive at the conclusion of the study censored, assessed up to 3 years

• Non-relapse mortality

  Non-relapse mortality is defined as death in remission, and relapse is considered a competing risk.

  Up to 3 years

• Patient-reported outcomes

  Will be assessed using Lee symptom scale and Patient Reported Outcomes Measurement Information System (PROMIS). The summary score of the Lee Symptom Scale and the PROMIS Global will be calculated according to the instructions of the developers. For analyses assessing change in quality of life, improvement or worsening of the Summary symptom score by 6 points or more or the PROMIS Physical or Mental Functioning scales by 5 points or more compared to baseline will be considered a clinically significant change.

  Up to 3 years

Study Arms (1)

Observational (questionnaire, biospecimen, chart review)

Patients complete questionnaires over 10 minutes about physical symptoms, activity level, and emotional well-being and have their medical records reviewed at baseline, 1, 3, and 6 months after starting index treatment, and at start of a new systemic treatment. Patients also undergo collection of blood samples over 1-2 minutes at baseline and at 1 month after starting index treatment, or at a treatment change visit if new therapy has not started.

Procedure: Biospecimen Collection; Other: Medical Chart Review; Other: Quality-of-Life Assessment; Other: Questionnaire Administration

Interventions

Biospecimen Collection PROCEDURE

Undergo collection of blood sample

Observational (questionnaire, biospecimen, chart review)

Medical Chart Review OTHER

Review of medical chart

Also known as: Chart Review

Observational (questionnaire, biospecimen, chart review)

Quality-of-Life Assessment OTHER

Ancillary studies

Also known as: Quality of Life Assessment

Observational (questionnaire, biospecimen, chart review)

Questionnaire Administration OTHER

Complete questionnaire

Observational (questionnaire, biospecimen, chart review)

Eligibility Criteria

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

Study Population

Patients with chronic graft-versus-host disease (GVHD) who are starting a new therapy for GVHD

You may qualify if:

• Adults age 18 or older
• Prior allogeneic stem cell transplant, with any graft source, donor type, and GVHD prophylaxis
• No evidence of persistent or progressive malignancy at the time of enrollment
• Agrees to be evaluated at the transplant center before a new line of treatment is started (may be concurrent with the enrollment visit), and later between 2-6 weeks, 3 months and 6 months after index treatment is started or if an additional new therapy is started before 6 months
• Signed, informed consent

You may not qualify if:

• Inability to comply with study procedures
• Uncontrolled psychiatric disorder
• Anticipated survival \< 6 months

Sponsors & Collaborators

• Fred Hutchinson Cancer Center: lead
• National Cancer Institute (NCI): collaborator

Study Sites (10)

University of Florida

Gainesville, Florida, 32610, United States

COMPLETED

Moffitt Cancer Center

Tampa, Florida, 33612, United States

RECRUITING

Dana-Farber Harvard Cancer Center

Boston, Massachusetts, 02115, United States

RECRUITING

University of Minnesota/Masonic Cancer Center

Minneapolis, Minnesota, 55455, United States

COMPLETED

Roswell Park Cancer Institute

Buffalo, New York, 14263, United States

COMPLETED

Cleveland Clinic Foundation

Cleveland, Ohio, 44195, United States

RECRUITING

UPMC Hillman Cancer Center

Pittsburgh, Pennsylvania, 15232, United States

COMPLETED

Vanderbilt University/Ingram Cancer Center

Nashville, Tennessee, 37232, United States

COMPLETED

Fred Hutch/University of Washington Cancer Consortium

Seattle, Washington, 98109, United States

RECRUITING

Vancouver General Hospital/BC Cancer

Vancouver, British Columbia, V5Z 4E6, Canada

COMPLETED

Related Publications (1)

• Hamilton BK, Onstad L, Carpenter PA, Pidala J, El Jurdi N, Farhadfar N, Kitko CL, Lee CJ, Mehta R, Chen GL, Cutler C, Lee SJ. Study Protocol: Predicting the Quality of Response to Specific Treatments (PQRST) in Chronic Graft-versus-Host Disease. Contemp Clin Trials. 2024 Oct;145:107637. doi: 10.1016/j.cct.2024.107637. Epub 2024 Jul 20.

  PMID: 39038701 DERIVED

Biospecimen

Retention: SAMPLES WITH DNA

Blood

MeSH Terms

Conditions

Bronchiolitis Obliterans Syndrome

Condition Hierarchy (Ancestors)

Organizing Pneumonia; Bronchiolitis Obliterans; Bronchiolitis; Bronchitis; Bronchial Diseases; Respiratory Tract Diseases; Lung Diseases, Obstructive; Lung Diseases; Graft vs Host Disease; Immune System Diseases

Study Officials

• Stephanie J. Lee

  Fred Hutch/University of Washington Cancer Consortium

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Gaby Desatnik

CONTACT

206-667-1356; cgvhd@fredhutch.org

Study Design

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

Study Record Dates

• First Submitted: June 11, 2020
• First Posted: June 16, 2020
• Study Start: June 29, 2020
• Primary Completion (Estimated): August 2, 2026
• Study Completion (Estimated): August 2, 2026
• Last Updated: November 19, 2025

Record last verified: 2025-11

Data Sharing

• IPD Sharing: Will not share

Locations

CA (1); US (9)