Efficacy of Low-dose Post-transplant Cyclophosphamide for Prevention of Graft-versus-host Disease in Ambulatory Allogeneic Hematopoietic Stem Cell Transplantation
Trial Snapshot
- Phase
- Phase 2
- Status
- Completed
- Sponsor
- Enrollment
- 35
- Locations
- 1
- Primary Endpoint
- Efficacy of Low-dose PT-Cy
Study Overview
Brief Summary
Low-dose post-transplant cyclophosphamide have demonstrated therapeutic efficacy in allogeneic stem cell transplants which is comparable with the standard dose and also facilitates early hematological recovery.
Detailed Description
Graft-versus-host disease (GVHD) is the most important complication that occurs in hematopoietic stem cell transplantation (HSCT). GVHD prophylaxis based on the use of post-transplant cyclophosphamide (PT-Cy) has proven to be a practical, easily and accessible agent that allows both identical and haploidentical transplants to be performed with lower incidences of this disease.
Low-dose cyclophosphamide have already demonstrated therapeutic efficacy in these types of transplants which is comparable with the standard dose and also facilitates early hematological recovery that can in turn reduce risks of infection, hospital stay and total costs for the patient.
The investigators will conduct a phase 2, non-randomized, single center, non-comparative clinical trial to demonstrate the effectiveness of of low-dose PT-Cy which is accessible to a population with limited resources while maintaining acceptable efficacy and safety to prevent GVHD.
Study Design
- Study Type
- Interventional
- Allocation
- Na
- Intervention Model
- Single Group
- Primary Purpose
- Treatment
- Masking
- None
Eligibility Criteria
- Ages
- 18 Years to — (Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •Age >18 years.
- •Both genders.
- •Diagnosis of any blood disease in need of an allogeneic bone marrow transplant (aplastic anemia, acute myeloid leukemia, chronic granulocytic leukemia, acute lymphoblastic leukemia, myelodysplastic syndrome, chronic myelomonocytic leukemia, Hodgkin lymphoma, and Non-Hodgkin lymphoma).
- •Patients who have compatible related donors to perform an identical or haploidentical allogeneic transplant.
- •Patients with functional status 0 to 2 using the ECOG scale.
Exclusion Criteria
- •Poor functional status (ECOG>2).
- •Organic dysfunction (Marshall score ≥2).
- •Pregnancy
- •Heart failure (NYHA III or IV).
- •Renal failure (GFR <30 ml/min/1.72m2).
- •History of ventricular arrhythmias or uncontrolled arrhythmias.
- •Acute myocardial infarction, unstable angina, or stable angina in the last six months.
- •Uncontrolled active infection.
- •Liver disease (Child-Pugh C).
- •Patients not approved by the local transplant committee for any other reason.
Arms & Interventions
Low-dose post-transplant cyclophosphamide
Low dose post-transplant cyclophosphamide therapy will be administered as follows:
Allogeneic stem cell transplant:
Cyclophosphamide 30/mg/kg/day for 2 continuous days.
Haploidentical stem cell transplant:
Cyclophosphamide 40/mg/kg/day for 2 continuous days.
Intervention: Cyclophosphamide (Drug)
Outcomes
Primary Outcomes
Efficacy of Low-dose PT-Cy
Time Frame: 6 months
Low-dose PT-Cy (30 and 40 mg/kg/day/2 days) is effective in achieving an incidence of grade III/IV (Glucksberg grade) acute GVHD and moderate to severe chronic GVHD equal to or below historical controls of 20 and 30%, respectively.
Secondary Outcomes
No secondary outcomes reported
Investigators
Cesar Homero Gutierrez-Aguirre
Professor of Hematology Service
Hospital Universitario Dr. Jose E. Gonzalez