Phase 2
N=77
Sirolimus, Cyclosporine, and Mycophenolate Mofetil in Preventing Graft-versus-Host Disease in Treating Patients With Blood Cancer Undergoing Donor Peripheral Blood Stem Cell Transplant
Adult Acute Lymphoblastic Leukemia · Adult Acute Myeloid Leukemia · Adult Diffuse Large B-Cell Lymphoma · Adult Myelodysplastic Syndrome · Adult Non-Hodgkin Lymphoma
Bottom Line
View on ClinicalTrials.gov: NCT01251575 ↗Enrolled (actual)
77
Serious AEs
0.0%
Results posted
Dec 2019
Primary outcome: Primary: Number of Patients With Grade II-IV Acute Graft Versus Host Disease (GVHD) — 15; 12 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Allogeneic Hematopoietic Stem Cell Transplantation (Procedure); Cyclosporine (Drug); Fludarabine Phosphate (Drug); Laboratory Biomarker Analysis (Other); Mycophenolate Mofetil (Drug); Peripheral Blood Stem Cell Transplantation (Procedure); Sirolimus (Drug); Total-Body Irradiation (Radiation)
- Age
- Pediatric, Adult, Older Adult
- Sex
- All
- Sponsor
- Fred Hutchinson Cancer Center
- Primary completion
- Nov 2018
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Patients With Grade II-IV Acute Graft Versus Host Disease (GVHD) |
15; 12 | — |
| SECONDARY Number of Non-Relapse Mortalities |
3 | — |
| SECONDARY Number of Patients With Grade III-IV Acute GVHD |
2 | — |
Summary
This phase II trial studies how well sirolimus, cyclosporine and mycophenolate mofetil works in preventing graft-vs-host disease (GVHD) in patients with blood cancer undergoing donor peripheral blood stem cell (PBSC) transplant. Giving chemotherapy and total-body irradiation before a donor peripheral blood stem cell transplant helps stop the growth of cancer cells. It may also stop the patient's immune system from rejecting the donor's stem cells. When the healthy stem cells from a donor are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells. Giving total-body irradiation together with sirolimus, cyclosporine, and mycophenolate mofetil before and after transplant may stop this from happening.
Eligibility Criteria
Inclusion Criteria
- Ages > 50 years with hematologic malignancies treatable by related or unrelated HCT
- Ages = 40% risk of transplant-related mortality [TRM]); this criterion can include patients with a HCT-comorbidity index (CI) score of >= 1; transplants should be approved for these inclusion criteria by the principal investigators at the collaborating centers and at Fred Hutchinson Cancer Research Center (FHCRC); all children = 5% circulating leukemic blasts (in the peripheral blood) detected by standard pathology for patients with MDS/MPS
- Central nervous system (CNS) involvement with disease refractory to intrathecal chemotherapy
- Fertile men or women unwilling to use contraceptives during and for up to 12 months following treatment
- Female patients who are pregnant or breast-feeding
- Human immunodeficiency virus (HIV) positive patients
- Patients with active non-hematologic malignancies (except non-melanoma skin cancers) or those with non-hematologic malignancies (except non-melanoma skin cancers) who have been rendered with no evidence of disease, but have a greater than 20% chance of having disease recurrence within 5 years; this exclusion does not apply to patients with non-hematologic malignancies that do not require therapy
- Fungal infections with radiological progression after receipt of amphotericin B or active triazole for greater than 1 month
- Patients with active bacterial or fungal infections unresponsive to medical therapy
- Cardiac ejection fraction 50 years of age, or history of cardiac disease or anthracycline exposure; patients with a shortening fraction 3 mg/dL, or symptomatic biliary disease
- Patients with poorly controlled hypertension on multiple antihypertensives
- Karnofsky scores < 60 or Lansky score < 50
- All patients receiving antifungal therapy voriconazole, posaconazole, or fluconazole must have sirolimus reduced according to the Standard Practice Antifungal Therapy Guidelines
- The addition of cytotoxic agents for "cytoreduction" with the exception of tyrosine kinase inhibitors (such as imatinib), cytokine therapy, hydroxyurea, low dose cytarabine, chlorambucil, or Rituxan will not be allowed within three weeks of the initiation of conditioning
- DONOR: Donor (or centers) who will exclusively donate marrow
- DONOR: Donors who are HIV-positive and/or, medical conditions that would result in increased risk for G-CSF mobilization and harvest of PBSC
- DONOR: Patients who are homozygous at the mismatched HLA class I locus or II locus, the donor is excluded if homozygous at the mismatched locus (i.e., patient is homozygous A*01:01 and donor is homozygous A*02:01); this type of mismatch is considered a two-antigen mismatch and is not allowed
Data sourced from ClinicalTrials.gov (NCT01251575). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.