Ann Woolfrey

Hematopoietic cell transplant (HCT) is the only curative therapy for many life-threatening hematologic diseases, and theoretically could cure other chronic hematologic and immunologic disorders. The research projects under Dr. Woolfrey’s direction are aimed toward improving the effectiveness of hematopoietic cell transplants, particularly for patients with nonmalignant disorders. These studies explore the use of lower dose (nonmyeloablative) conditioning regimens to reduce the short and long term toxicities associated with HCT, the use of HCT to restore normal immunity in patients with HIV or refractory autoimmune diseases, and the use of alternative donors, including unrelated and mismatched related donors.

Clinical Studies
1. Nonmyeloablative conditioning regimen using low-dose total body irradiation with or without fludarabine for treatment of immune deficiencies, metabolic storage diseases, hemaglobinopathies, and other inherited disorders. The primary goal of each study is to determine whether establishment of stable mixed chimerism using a low-toxicity regimen results in amelioration of the disease, as an alternative to standard transplant procedures.

2. Nonmyeloablative conditioning regimen using low-dose total body irradiation and fludarabine for treatment of HIV. The primary goal is to determine whether immunity can be reconstituted in HIV patients using a non-toxic regimen for HCT. A second goal is to establish a method for allogeneic transplant that is tolerated by patients with HIV for cure of life-threatening malignancies.

3. High-dose immunoablative therapy followed by autologous HCT for treatment of refractory autoimmune disorders. The primary goal is to determine whether immune ablation followed by rescue with purified autologous stem cells could result in restoration of immunity without recurrence of the autoreactive T cells. Patients eligible include children with juvenile rheumatoid arthritis, systemic lupus erythematosus, systemic sclerosis, or dermatomyositis.

4. Use of haploidentical donors for HCT in the treatment of life-threatening hematologic diseases in children. The goal of these studies has been to determine the outcome of transplants for patients who undergo HCT using haploidentical donors.

Hematology
Oncology
Pediatrics
Medicine
Bone Marrrow Transplantation
Hematopoietic Cell Transplantation
Immunology

Immune Reconstitution
Tolerance
HIV
Juvenile Rheumatoid Arthritis