A Phase III Study of Alpha 1-Antitrypsin plus Corticosteroids versus Corticosteroids Alone to Treat High-Risk Acute Graft-versus-Host Disease after an Allogeneic Stem Cell Transplant


Full Title

A Randomized, Double-Blind, Placebo-Controlled Multicenter Phase III Trial of Alpha 1 - Antitrypsin (AAT) Combined with Corticosteroids vs Corticosteroids Alone for the Treatment of High Risk Acute Graft-versus-Host Disease (GVHD) Following Allogeneic Hematopoietic Stem Cell Transplant (NMDP IRB) (BMT CTN 1705)


One potential complication of receiving a stem cell transplant from another person (“allogeneic transplant”) is graft-versus-host disease (GVHD). GVHD is caused when cells from the donor view the recipient’s cells as foreign, resulting in symptoms such as rash, diarrhea, nausea, vomiting, and liver dysfunction, among others. Corticosteroid drugs are usually used to treat GVHD, but they don’t always work well and have numerous side effects.

In this study, researchers want to see if adding a drug called alpha 1-antitrypsin (AAT) to corticosteroid treatment is more effective than corticosteroids alone to treat acute GVHD. AAT is a natural protein in the body that may be able to decrease inflammation, protect tissues in the body from from attacks by the donated cells, and treat GVHD better than steroids alone. In this study, patients with high-risk acute GVHD will be randomly assigned to receive corticosteroids with either AAT or a placebo.

Who Can Join

To be eligible for this study, patients must meet several criteria, including but not limited to the following:

  • Patients must have newly diagnosed high-risk acute GVHD following an allogeneic stem cell transplant.
  • This study is for patients age 18 and older.

For more information about this study and to inquire about eligibility, please contact Dr. Doris Ponce at 646-608-3739.