Post Transplant Cyclophosphamide for Unrelated and Related Allogeneic Hematopoietic Stem Cell Transplantation for Hematological Malignancies
The primary rationale for the development of this research study is to find out if the use
of cyclophosphamide after a "blood" stem cell transplant is an effective treatment for
patients with blood cancers who require transplant for long-term survival but are without an
available matched-sibling donor. Historically, survival rates for patients undergoing
partially matched related or unrelated donor transplants (henceforth to be called
alternative donor transplants) have been much lower than those observed after matched
sibling stem cell transplants. Survival post alternative donor stem cell transplant has
also been affected by the requirement to remove or reduce the numbers of donor T cells
resulting in higher rates of infection, graft rejection, and relapse. One significant
limitation to conventional donor transplants with HLA matched siblings has been that over
50% of patients do not have HLA matched siblings so that increasing the safety of
alternative donor transplants could have a significant influence on the number of patients
who could safely receive transplants. Because of the historically low overall survival (OS)
after alternative donor transplants, it has become a procedure of "last resort" in many
centers unwilling to consider it unless all other options are exhausted. There fore several
centers including ours have sought to overcome problems using various strategies. The
strategy the investigators have proposed for this study (which has been used similarly by
other centers) has been to administer cyclophosphamide post the stem cell infusion
(traditionally it is given before the stem cell infusion) thereby hopefully destroying the
activated T-cells causing graft-versus host disease (GVHD) and allow T cell tolerization and
engraftment; but, not the inactivated T cells thereby hopefully preserving the anti-tumor
effects of the donor immune system. Thus, the major aim of this study will be to measure
the engraftment with this regimen and secondarily to measure incidence of GVHD and day 100
mortality. The goal is to see if in the first 3 months the use of cyclophosphamide post
stem cell infusion with alternative donors is as safe and as effective as traditional
matched transplants.
Interventional
Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment
Successful Engraftment Using Cyclophosphamide Post-Transplant
Hematopoietic engraftment will be defined as: ANC >/= 0.5x10e9/L for at least 3 days. Platelet engraftment >20,000 with no transfusions X 7 days.
Through 100 days post-transplant
No
John L Wagner, MD
Principal Investigator
Thomas Jefferson University
United States: Institutional Review Board
11D.51
NCT01349101
February 2011
February 2016
Name | Location |
---|---|
Thomas Jefferson University | Philadelphia, Pennsylvania 19107-6541 |