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ATG 2.5 mg/kg/day via IV will be given for 4 doses. Each participant will receive only one cycle of therapy. The daily infusion will be administered over at least 6 hours and slowed as necessary to minimize infusion-related symptoms.

Antithymocyte globulin (ATG)

All participants will be pre-treated with prednisone (1 mg/kg/day by mouth) 2 days prior to the first ATG does and continuing for 14 days after the final dose to prevent serum sickness

Troy Overfield

Ronald Paquette, MD

Tera Uliano, RN

Alan List, MD

Robin Heggeland, RN

Jaroslaw P. Maciejewski, MD, PhD

Lynn Ruiz

Thomas P. Loughran, Jr., MD

Myelodysplastic syndrome (MDS) is a rare, potentially serious bone marrow disease. Currently available treatments for MDS have been only somewhat beneficial. The purpose of this study is to determine the effects of the medication antithymocyte globulin (ATG) in adults with MDS and to determine which individuals with MDS are most likely to benefit from treatment with ATG.

In people with MDS, the bone marrow stops making healthy blood cells and instead produces poorly functioning, malformed, and immature blood cells. This can lead to anemia resulting from too few healthy red blood cells, infection resulting from too few healthy white blood cells, and bleeding resulting from too few healthy platelets. The exact cause of MDS remains unknown, but it may be caused by abnormal autoimmune activity in which activated T cells, a type of white blood cell, prevent normal bone marrow production. ATG, a medication that inhibits immune function, can restore normal blood production in some people with MDS, but it is not known how this happens and why it does not happen in all MDS patients. The purpose of this study is to examine the effects of ATG in adults with MDS and to determine which individuals with MDS are most likely to benefit from treatment with ATG.

Based on disease severity and likely disease progression, participants will be separated into either a high-risk group or a low-risk group. Participants will be hospitalized for a 4-day period during which they will receive daily infusions of ATG. Oral prednisone will be given 2 days before hospitalization, throughout hospitalization, and then for 14 days after hospitalization to limit the side effects of ATG. Antihistamines and acetaminophen will also be given during hospitalization to reduce the chances of an allergic reaction to ATG. After discharge, all participants will attend monthly study visits that will include blood collection, review of disease symptoms, and evaluation of medication response. At Week 16, participants in the high-risk group will undergo additional blood collection, a bone marrow biopsy, and a thorough evaluation of disease progression and the effects of MDS on daily living abilities. Participants in the low-risk group will undergo these same procedures at Week 24. Follow-up for all participants may last up to 2 years.

Effects of Antithymocyte Globulin in Adults With Myelodysplastic Syndrome

Mechanism and Response of Thymoglobulin in Patients With Myelodysplastic Syndrome (MDS)

Rare Diseases Clinical Research Network

Office of Rare Diseases (ORD)

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization	Link
Effects of Antithymocyte Globulin in Adults With Myelodysplastic Syndrome	NCT00466843	Myelodysplastic...	Antithymocyte g... Prednisone	18 Years -	Office of Rare Diseases (ORD)
Effects of Antithymocyte Globulin in Adults With Myelodysplastic Syndrome	NCT00466843	Myelodysplastic...	Antithymocyte g... Prednisone	18 Years -	Office of Rare Diseases (ORD)

Spots Global Cancer Trial Database for abnormal hematopoiesis