Efficacy of Low-dose PT-Cy for Prevention of GVHD in Ambulatory Allogeneic HSCT

Hospital Universitario Dr. Jose E. Gonzalez

Status and phase: Completed; Phase 2

Conditions: Graft Versus Host Disease

Treatments: Drug: Cyclophosphamide

Study type: Interventional

Funder types: Other

Identifiers

NCT07319000

HE22-0037

Details and patient eligibility

About

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.

Full 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.

Enrollment: 35 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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35 participants in 1 patient group

Low-dose post-transplant cyclophosphamide

Experimental group

Description:

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.

Treatment:

Drug: Cyclophosphamide