A Study Comparing Reduced-Intensity Allogeneic Stem Cell Transplantation with Best Supportive Therapy in Older Patients with Myelodysplastic Syndromes

Protocol
14-123
Full Title
A Multi-Center Biologic Assignment Trial Comparing Reduced Intensity Allogeneic Hematopoietic Cell Transplant to Hypomethylating Therapy or Best Supportive Care in Patients Aged 50-75 with Intermediate-2 and High Risk Myelodysplastic Syndrome (BMT CTN 1102)
Purpose

Blood transfusions and medications called “hypomethylating drugs,” such as azacitidine and 5-deoxy azacitidine, are standard therapy for myelodysplastic syndromes (MDS) in older patients. While this treatment can improve blood cell counts and extend life, it is not curative. The administration of stem cells from a matched donor, known as an allogeneic transplant, can cure MDS, but it is associated with a high risk of complications in older patients.

With a conventional allogeneic stem cell transplant, patients receive high doses of chemotherapy and radiation therapy to destroy their blood cells and prepare them for the transplant. In this study, researchers are comparing a “reduced-intensity” approach to allogeneic transplantation which uses lower doses of chemotherapy and radiation therapy. It is sometimes called a “mini-transplant,” and may make allogeneic transplantation possible for some older patients with MDS. It is being assessed in this study in patients with MDS age 50 and older.

Patients in this study who have a matched stem cell donor will undergo reduced-intensity allogeneic transplantation. Patients who do not have a match donor will receive standard MDS therapy. Researchers will compare the two groups to see how well they do and to study their quality of life.

Eligibility

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

  • Patients must have been diagnosed with intermediate-risk or high-risk MDS.
  • Prior treatment for MDS is allowed.
  • Patients must be physically well enough that they are fully ambulatory, capable of all self care, and are capable of all but physically strenuous activities. As an example, patients must be well enough that they would be able to carry out office work or light housework.
  • This study is for patients ages 50 to 75.

For more information about this study and to inquire about eligibility, please contact Dr. Hugo Castro-Malaspina at 212-639-8197 or Dr. Virginia Klimek at 212-639-6519.

Disease(s)
Hematology: Myelodysplastic Syndrome
Locations
Related Diseases