Predicting the Quality of Response to Specific Treatments in Patients With cGVHD, PQRST Study
Study Details
Study Description
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 initial or second-line 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.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Detailed Description
OUTLINE:
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.
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| 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
Undergo collection of blood sample
Other: Medical Chart Review
Review of medical chart
Other Names:
Other: Quality-of-Life Assessment
Ancillary studies
Other Names:
Other: Questionnaire Administration
Complete questionnaire
|
Outcome Measures
Primary Outcome Measures
- Clinical Response according to the 2014 criteria [Up to 6 months]
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 otherwise portable spirometry will be performed. Response will be assessed according to the recommendations of the 2014 NIH response measures publication or any applicable updates.
- Time to next systemic treatment [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]
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.
- Duration of treatment [Up to 3 months]
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.
- 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 [Up to 3 years]
Non-relapse mortality is defined as death in remission, and relapse is considered a competing risk.
- Patient-reported outcomes [Up to 3 years]
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.
Eligibility Criteria
Criteria
Inclusion Criteria:
-
Prior allogeneic stem cell transplant, with any graft source, donor type, and GVHD prophylaxis
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No evidence of persistent or progressive malignancy at the time of enrollment
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Agrees to be evaluated at the transplant center before initial or second-line treatment is started (may be concurrent with the enrollment visit), and later between 2-6 weeks, 3 months and 6 months after treatment is started or if a new therapy is started before 6 months
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Signed, informed consent
Exclusion Criteria:
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Inability to comply with study procedures
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Uncontrolled psychiatric disorder
-
Anticipated survival < 6 months
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Moffitt Cancer Center | Tampa | Florida | United States | 33612 |
| 2 | Dana-Farber Harvard Cancer Center | Boston | Massachusetts | United States | 02115 |
| 3 | University of Minnesota/Masonic Cancer Center | Minneapolis | Minnesota | United States | 55455 |
| 4 | Roswell Park Cancer Institute | Buffalo | New York | United States | 14263 |
| 5 | Cleveland Clinic Foundation | Cleveland | Ohio | United States | 44195 |
| 6 | Vanderbilt University/Ingram Cancer Center | Nashville | Tennessee | United States | 37232 |
| 7 | Fred Hutch/University of Washington Cancer Consortium | Seattle | Washington | United States | 98109 |
| 8 | Vancouver General Hospital/BC Cancer | Vancouver | British Columbia | Canada | V5Z 4E6 |
Sponsors and Collaborators
- Fred Hutchinson Cancer Center
- National Cancer Institute (NCI)
Investigators
- Principal Investigator: Stephanie J Lee, Fred Hutch/University of Washington Cancer Consortium
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- RG1006823
- NCI-2020-01242
- 10360
- R01CA118953