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

Efficacy of Low-dose Post-transplant Cyclophosphamide for Prevention of Graft-versus-host Disease in Ambulatory Allogeneic Hematopoietic Stem Cell Transplantation

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.

Study Overview

• Status: Completed
• Conditions: Graft Versus Host Disease
• Intervention / Treatment: Drug: Cyclophosphamide

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 Type: Interventional
• Enrollment (Actual): 35
• Phase: Phase 2

Contacts and Locations

Study Locations

• Mexico
  • Monterrey, Mexico, 64460
    • Hospital Universitario de Monterrey

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: 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.	Drug: Cyclophosphamide; low-dose post-transplant cyclophosphamide

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Efficacy of Low-dose PT-Cy	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.	6 months

Collaborators and Investigators

• Sponsor: Hospital Universitario Dr. Jose E. Gonzalez

Publications and helpful links

General Publications

• Sugita J, Kamimura T, Ishikawa T, Ota S, Eto T, Kuroha T, Miyazaki Y, Kumagai H, Matsuo K, Akashi K, Taniguchi S, Harada M, Teshima T. Reduced dose of posttransplant cyclophosphamide in HLA-haploidentical peripheral blood stem cell transplantation. Bone Marrow Transplant. 2021 Mar;56(3):596-604. doi: 10.1038/s41409-020-01065-0. Epub 2020 Sep 24.

Study record dates

Study Major Dates

• Study Start (Actual): January 1, 2023
• Primary Completion (Actual): December 31, 2023
• Study Completion (Actual): December 31, 2023

Study Registration Dates

• First Submitted: September 8, 2025
• First Submitted That Met QC Criteria: December 19, 2025
• First Posted (Actual): January 6, 2026

Study Record Updates

• Last Update Posted (Actual): January 6, 2026
• Last Update Submitted That Met QC Criteria: December 19, 2025
• Last Verified: December 1, 2025

More Information

Terms related to this study

• Keywords: Cyclophosphamide; Transplants
• Additional Relevant MeSH Terms: Immune System Diseases; Graft vs Host Disease; Organic Chemicals; Hydrocarbons; Phosphoramide Mustards; Nitrogen Mustard Compounds; Mustard Compounds; Hydrocarbons, Halogenated; Phosphoramides; Organophosphorus Compounds; Cyclophosphamide
• Other Study ID Numbers: HE22-0037

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No