Chronic Graft Versus Host Disease
Conditions
Brief summary
This randomized trial studies how well discontinuation or continuation of immunosuppressive therapy works in treating participants with chronic graft versus host disease. Continuation of immunosuppressive treatment may prevent graft-versus-host disease worsening.
Detailed description
PRIMARY OBJECTIVE: I. Assess feasibility of enrolling and randomizing patients with chronic graft versus host disease (GVHD) to discontinuation (standard of care) versus continuation (investigation) of immunosuppressive therapy (IST). SECONDARY OBJECTIVES: I. Assess feasibility of enrolling and randomizing patients who are not local, and evaluate the quality of data received for those patients. II. Assess whether prolonged IST decreases the need for pulses of high dose IST. III. Evaluate the effect of prolonged IST on chronic GVHD manifestations and severity, risk of relapse, infection and organ toxicity. OUTLINE: Participants are randomized to 1 of 2 arms. ARM I: Participants have their IST tapered and discontinued per the plan. ARM II: Participants continue to receive a fixed dose IST for an additional 9 months with no taper. After completion of study treatment, participants are followed up annually.
Interventions
BIOLOGICALImmunosuppressive Therapy
Discontinued IST
OTHERSurvey Administration
Ancillary studies
Sponsors
National Cancer Institute (NCI)
Fred Hutchinson Cancer Center
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
Inclusion criteria
* Prior first allogeneic stem cell transplant, with any graft source, donor type, and GVHD prophylaxis * Patients who are on one systemic immunosuppressive agent for chronic GVHD with a plan to withdraw all systemic IST; hydrocortisone or prednisone continued for treatment of adrenal insufficiency is not considered a systemic IST * No evidence of malignancy at the time of enrollment * Agree to be evaluated at the transplant center or by local provider every 3 months for 12 months after randomization * Agreement to be contacted by phone or e-mail for health status evaluation for up to 3 years * Signed, informed consent
Exclusion criteria
* Inability to comply with study procedures * Pregancy
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Feasibility of Enrolling Patients | 22.9 months | Descriptive summary of number of patients enrolled on the study (signed consent) |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Feasibility of Randomizing Patients | 22.9 months | Descriptive summary of percentage of patients randomized. |
| Compliance With Treatment | Up to 12 months after randomization | Rate of patients following study immunosuppressive therapy management based on study arm (standard taper or continuation of low dose). |
| Compliance With Data Collection | Up to 12 months after randomization | Count of surveys completed by physicians and patients |
| Graft Versus Host Disease Manifestations | Up to 12 months after randomization | New chronic graft versus host disease (GVHD) manifestations and/or worsening of existing manifestations |
| Recurrent Malignancy | Up to 12 months after randomization | Incidence of relapse of primary disease |
| Incidence of Grade >= 3 Organ Toxicity | Up to 12 months after randomization | Incidence of grade \>= 3 organ toxicity |
| Enrollment Rate of Participants Who Are Not Local | Up to 12 months after randomization | Enrollment rate of participants who are not local. Participants who are local defined as those who had all or some study visits completed at the cancer center. |
| Quality of Data of Participants Who Are Not Local | Up to 12 months after randomization | Participants who are local defined as those who had all or some study visits completed at the cancer center. |
| Incidence of Grade >= 3 Infections | Up to 12 months after randomization | Incidence of grade \>= 3 infections |
Countries
United States
Participant flow
Pre-assignment details
4 participants were not randomized. 1 could not be reached and 3 had a GVHD flare after enrollment.
Participants by arm
| Arm | Count |
|---|---|
| Arm I (Discontinued IST) Participants have their IST tapered and discontinued per the plan. | 9 |
| Arm II (Continued IST) Participants continue to receive a fixed dose IST for an additional 9 months with no taper. | 8 |
| Total | 17 |
Baseline characteristics
| Characteristic | Arm I (Discontinued IST) | Arm II (Continued IST) | Total |
|---|---|---|---|
| Age, Categorical <=18 years | 0 Participants | 0 Participants | 0 Participants |
| Age, Categorical >=65 years | 5 Participants | 4 Participants | 9 Participants |
| Age, Categorical Between 18 and 65 years | 4 Participants | 4 Participants | 8 Participants |
| Ethnicity (NIH/OMB) Hispanic or Latino | 0 Participants | 0 Participants | 0 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 9 Participants | 8 Participants | 17 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 1 Participants | 1 Participants | 2 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants | 1 Participants | 1 Participants |
| Race (NIH/OMB) More than one race | 0 Participants | 1 Participants | 1 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) White | 8 Participants | 5 Participants | 13 Participants |
| Region of Enrollment United States | 9 participants | 8 participants | 17 participants |
| Sex: Female, Male Female | 0 Participants | 0 Participants | 0 Participants |
| Sex: Female, Male Male | 9 Participants | 8 Participants | 17 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 9 | 0 / 8 |
| other Total, other adverse events | 8 / 9 | 6 / 8 |
| serious Total, serious adverse events | 0 / 9 | 2 / 8 |
Outcome results
Primary
Feasibility of Enrolling Patients
Descriptive summary of number of patients enrolled on the study (signed consent)
Time frame: 22.9 months
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Total Number of Participants | Feasibility of Enrolling Patients | 21 Participants |
Secondary
Compliance With Data Collection
Count of surveys completed by physicians and patients
Time frame: Up to 12 months after randomization
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Total Number of Participants | Compliance With Data Collection | Total surveys collected | 48 surveys |
| Total Number of Participants | Compliance With Data Collection | Patient surveys missed | 3 surveys |
| Total Number of Participants | Compliance With Data Collection | Physician surveys missed | 5 surveys |
| Arm II (Continued IST) | Compliance With Data Collection | Total surveys collected | 39 surveys |
| Arm II (Continued IST) | Compliance With Data Collection | Patient surveys missed | 4 surveys |
| Arm II (Continued IST) | Compliance With Data Collection | Physician surveys missed | 6 surveys |
Secondary
Compliance With Treatment
Rate of patients following study immunosuppressive therapy management based on study arm (standard taper or continuation of low dose).
Time frame: Up to 12 months after randomization
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Total Number of Participants | Compliance With Treatment | 7 Participants |
| Arm II (Continued IST) | Compliance With Treatment | 8 Participants |
Secondary
Enrollment Rate of Participants Who Are Not Local
Enrollment rate of participants who are not local. Participants who are local defined as those who had all or some study visits completed at the cancer center.
Time frame: Up to 12 months after randomization
Population: All participants were local, no data were collected for this outcome measure.
Secondary
Feasibility of Randomizing Patients
Descriptive summary of percentage of patients randomized.
Time frame: 22.9 months
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Total Number of Participants | Feasibility of Randomizing Patients | 17 Participants |
Secondary
Graft Versus Host Disease Manifestations
New chronic graft versus host disease (GVHD) manifestations and/or worsening of existing manifestations
Time frame: Up to 12 months after randomization
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Total Number of Participants | Graft Versus Host Disease Manifestations | 2 Participants |
| Arm II (Continued IST) | Graft Versus Host Disease Manifestations | 0 Participants |
Secondary
Incidence of Grade >= 3 Infections
Incidence of grade \>= 3 infections
Time frame: Up to 12 months after randomization
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Total Number of Participants | Incidence of Grade >= 3 Infections | 1 Participants |
| Arm II (Continued IST) | Incidence of Grade >= 3 Infections | 0 Participants |
Secondary
Incidence of Grade >= 3 Organ Toxicity
Incidence of grade \>= 3 organ toxicity
Time frame: Up to 12 months after randomization
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Total Number of Participants | Incidence of Grade >= 3 Organ Toxicity | 0 Participants |
| Arm II (Continued IST) | Incidence of Grade >= 3 Organ Toxicity | 0 Participants |
Secondary
Quality of Data of Participants Who Are Not Local
Participants who are local defined as those who had all or some study visits completed at the cancer center.
Time frame: Up to 12 months after randomization
Population: All participants were local, no data were collected for this outcome measure.
Secondary
Recurrent Malignancy
Incidence of relapse of primary disease
Time frame: Up to 12 months after randomization
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Total Number of Participants | Recurrent Malignancy | 0 Participants |
| Arm II (Continued IST) | Recurrent Malignancy | 0 Participants |