Low-Intensity Conditioning Prior to Bone Marrow Transplantation for Patients with Dyskeratosis Congenita/Telomere Disease


Full Title

Radiation- and Alkylator-Free Hematopoietic Cell Transplantation for Bone Marrow Failure Due to Dyskeratosis Congenita / Telomere disease


Dyskeratosis congenital/telomere disease (DC/TD) is a rare disease that can cause bone marrow failure. The only cure is transplantation of bone marrow from a healthy donor. Typically, before the transplant, the patient’s diseased bone marrow is destroyed with intensive chemotherapy and radiation therapy. This “conditioning” regimen can cause serious side effects, especially in patients with DC/TD.

The purpose of this study is to see if a low-intensity conditioning regimen is effective prior to bone marrow transplantation in patients with DC/TD and if it causes fewer serious side effects. The conditioning regimen does not use radiation or chemotherapy drugs from the “alkylator” family (which would cause serious side effects in patients with DC/TD). It uses two chemotherapy drugs (alemtuzumab and fludarabine) in a regimen that is less intensive than the traditional approach.


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

  • Patients must have bone marrow failure due to DC/TD.
  • Patients must have a matched related or unrelated bone marrow donor.
  • This study is for patients 30 days to 65 years of age.

For more information about this study and to inquire about eligibility, please contact Dr. Farid Boulad at 212-639-6684.





Disease Status

Newly Diagnosed & Relapsed/Refractory