Oral Chronic Graft-versus-host Disease
Conditions
Keywords
tacrolimus, dexamethasone, oral rinse
Brief summary
The purpose of this research study is to determine the effectiveness of topical steroid therapy (with a drug called dexamethasone) and topical tacrolimus therapy for the treatment of oral chronic Graft-Versus-Host Disease (cGVHD)
Detailed description
* Because no one knows which study option is best, participants will be randomized into one of two study groups: topical dexamethasone or topical tacrolimus. * Participants will take the medication by mouth rinse four times a day for 5 minutes at a time. After rinsing they will spit out the medication and will not be able to eat or drink for 15 minutes. * Participants will also take anti-fungal medication (fluconazole) orally once a week. * After two weeks on study treatment, participants will have a blood drawn to monitor tacrolimus levels. * After the final treatment (4 weeks), participants will have the following tests and procedures: clinical examination; questionnaire; blood tests; oral culture; optional tissue biopsy.
Interventions
DRUGTacrolimus
Tacrolimus elixir taken as an oral rinse four times a day for 4 weeks
DRUGDexamethasone
Dexamethasone elixir taken as an oral rinse 4 times a day for 4 weeks
Sponsors
Dana-Farber Cancer Institute
Boston Children's Hospital
Beth Israel Deaconess Medical Center
Massachusetts General Hospital
Brigham and Women's Hospital
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
4 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Patients with oral chronic graft-versus-host disease * Patients 4 years of age or older * Stable cGVHD medication regimen for the four weeks prior to study enrollment
Exclusion criteria
* Patients already on topical steroid or tacrolimus therapies
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| The Clinical Efficacy of Topical Steroid and Topical Tacrolimus Therapies for the Treatment of Oral cGHVD. | Participants were assessed at Baseline and 4 weeks after start of therapy | Participants were given a survey at the time of screening and 4 weeks after start of therapy. The participants self-reported three symptoms of oral cGVHD: oral sensitivity, mouth pain, and mouth dryness. Each symptom was given a score ranging from 0-10, with 0 as none and 10 as the worst. Improvement in subjective scores was defined as 3 points or further reduction from pre-treatment to post-treatment assessment. |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Dexamethasone Dexamethasone : Dexamethasone elixir taken as an oral rinse 4 times a day for 4 weeks | 28 |
| Tacrolimus Tacrolimus Arm Closed to Accrual as of January 2012
Tacrolimus : Tacrolimus elixir taken as an oral rinse four times a day for 4 weeks | 18 |
| Total | 46 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| Overall Study | Lost to Follow-up | 1 | 0 |
| Overall Study | Progressive GVHD | 0 | 1 |
Baseline characteristics
| Characteristic | Tacrolimus | Dexamethasone | Total |
|---|---|---|---|
| Age, Categorical <=18 years | 0 Participants | 0 Participants | 0 Participants |
| Age, Categorical >=65 years | 6 Participants | 6 Participants | 12 Participants |
| Age, Categorical Between 18 and 65 years | 12 Participants | 22 Participants | 34 Participants |
| Age, Continuous | 54.9 years STANDARD_DEVIATION 15.3 | 53.8 years STANDARD_DEVIATION 11.5 | 54.2 years STANDARD_DEVIATION 13 |
| Region of Enrollment United States | 18 participants | 28 participants | 46 participants |
| Sex: Female, Male Female | 6 Participants | 17 Participants | 23 Participants |
| Sex: Female, Male Male | 12 Participants | 11 Participants | 23 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | — / — | — / — |
| other Total, other adverse events | 1 / 28 | 1 / 18 |
| serious Total, serious adverse events | 0 / 28 | 1 / 18 |
Outcome results
Primary
The Clinical Efficacy of Topical Steroid and Topical Tacrolimus Therapies for the Treatment of Oral cGHVD.
Participants were given a survey at the time of screening and 4 weeks after start of therapy. The participants self-reported three symptoms of oral cGVHD: oral sensitivity, mouth pain, and mouth dryness. Each symptom was given a score ranging from 0-10, with 0 as none and 10 as the worst. Improvement in subjective scores was defined as 3 points or further reduction from pre-treatment to post-treatment assessment.
Time frame: Participants were assessed at Baseline and 4 weeks after start of therapy
Population: Of the 46 participants enrolled on the trial, 36 were deemed evaluable for response.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Dexamethasone | The Clinical Efficacy of Topical Steroid and Topical Tacrolimus Therapies for the Treatment of Oral cGHVD. | Improvement in Oral Sensitivity | 15 participants |
| Dexamethasone | The Clinical Efficacy of Topical Steroid and Topical Tacrolimus Therapies for the Treatment of Oral cGHVD. | Improvement in Mouth Pain | 11 participants |
| Dexamethasone | The Clinical Efficacy of Topical Steroid and Topical Tacrolimus Therapies for the Treatment of Oral cGHVD. | Improvement in Mouth Dryness | 11 participants |
| Dexamethasone | The Clinical Efficacy of Topical Steroid and Topical Tacrolimus Therapies for the Treatment of Oral cGHVD. | Improvement in 1 or more areas | 17 participants |
| Dexamethasone | The Clinical Efficacy of Topical Steroid and Topical Tacrolimus Therapies for the Treatment of Oral cGHVD. | Improvement in 2 or more areas | 13 participants |
| Dexamethasone | The Clinical Efficacy of Topical Steroid and Topical Tacrolimus Therapies for the Treatment of Oral cGHVD. | Improvement in all 3 areas | 7 participants |
| Tacrolimus | The Clinical Efficacy of Topical Steroid and Topical Tacrolimus Therapies for the Treatment of Oral cGHVD. | Improvement in 2 or more areas | 2 participants |
| Tacrolimus | The Clinical Efficacy of Topical Steroid and Topical Tacrolimus Therapies for the Treatment of Oral cGHVD. | Improvement in Oral Sensitivity | 3 participants |
| Tacrolimus | The Clinical Efficacy of Topical Steroid and Topical Tacrolimus Therapies for the Treatment of Oral cGHVD. | Improvement in 1 or more areas | 7 participants |
| Tacrolimus | The Clinical Efficacy of Topical Steroid and Topical Tacrolimus Therapies for the Treatment of Oral cGHVD. | Improvement in Mouth Pain | 2 participants |
| Tacrolimus | The Clinical Efficacy of Topical Steroid and Topical Tacrolimus Therapies for the Treatment of Oral cGHVD. | Improvement in all 3 areas | 1 participants |
| Tacrolimus | The Clinical Efficacy of Topical Steroid and Topical Tacrolimus Therapies for the Treatment of Oral cGHVD. | Improvement in Mouth Dryness | 5 participants |