Graft Versus Host Disease
Conditions
Brief summary
This research trial studies medical chart review in determining outcomes of second-line therapy in patients with acute graft-versus-host disease previously treated with extracorporeal photopheresis or other systemic therapies. Gathering information about second-line therapy in patients with acute graft-versus-host disease may help doctors learn more about the disease and find better treatment.
Detailed description
PRIMARY OBJECTIVES: I. To determine 6-month freedom from treatment failure for second-line therapy for acute graft versus host disease (aGVHD). II. To show that extracorporeal photopheresis (ECP) is associated with a superior 6 month (m) freedom from treatment failure (FFTF) as compared to other treatment modalities for second line therapy for aGVHD. III. To describe health care burden in patients receiving second line therapy for acute GVHD. IV. Quality of life measurement using Functional Assessment of Cancer Therapy-Bone Marrow Transplant (FACT-BMT) in patients receiving second line therapy for aGVHD. OUTLINE: Study data are collected and managed using Research Electronic Data Capture (REDCap) tools at baseline and on days 5, 28, and 56. After completion of study, patients are followed up at 6 months, and 1 and 2 years.
Interventions
OTHERmedical chart review
Ancillary studies
OTHERquality-of-life assessment
Ancillary studies
Sponsors
National Cancer Institute (NCI)
Vanderbilt-Ingram Cancer Center
Study design
Observational model
CASE_ONLY
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Enrollment on study is within 5 days (including weekends) of starting second-line therapy * aGVHD grade 2 or higher at time of enrollment; patients with late acute and recurrent aGVHD are permitted * Donor lymphocyte induced aGVHD is permitted ONLY IF donor lymphocyte infusion given for mixed chimerism and not for progression of disease and meeting all other inclusion and
Exclusion criteria
will be eligible * Corticosteroid refractory or corticosteroid dependent aGVHD * Corticosteroid refractory aGVHD is defined as worsening of aGVHD after 3 days of systemic corticosteroids (minimum dose of 1 mg/kg), or no improvement after 7 days of systemic corticosteroids (minimum dose of 1 mg/kg) * Corticosteroid dependent aGVHD is defined as recurrence of aGVHD (grade 2 or higher) during corticosteroid taper and prior to reaching 50% of initial starting dose of corticosteroids * Informed consent form
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 6-month freedom from treatment failure, defined as a patient being alive, without relapse of underlying disease, and without the addition of new systemic therapy for the treatment of aGVHD, within 6 months of starting second line therapy | 6 months | Rates of 6 m FFTF will be estimated by subtracting rates of total failures from 100%. |
| Cumulative incidence estimates of relapse | Up to 2 years | Cox regression models will be used to identify risk factors for failure. |
| Cumulative incidence estimates of non-relapse mortality | Up to 2 years | Cox regression models will be used to identify risk factors for failure. |
| Treatment change as causes of failure during second line treatment | Up to 2 years | Cox regression models will be used to identify risk factors for failure. |
| Health care burden in patients receiving second line therapy for acute GVHD | Up to 6 months | The incremental budget spend between the patients receiving treatment with ECP and patients receiving other modalities will be measured and economic methods will be used to calculate an incremental cost effectiveness ratio for relevant clinical end points. Health economic data relating to hospitalizations (length of stay in myelosuppressive unit, high-dependency unit \[step-down unit\], intensive care unit), high cost drugs, total parenteral nutrition and surgical procedures will also be collected allowing comparisons to be made between patients treated with ECP and other treatment options. |
| Quality of life measured using FACT-BMT | Up to 6 months | — |
Countries
Austria, Germany, United Kingdom, United States
Outcome results
None listed