Phase II Study of Horse Anti-Thymocyte Globulin (hATG), Cyclosporine, Methylprednisone, and GCSF (Filgrastim or Pegfilgrastim) in Patients With Aplastic Anemia (AA), or Low/Int-1 Risk Myelodysplastic Syndrome (MDS)
Study Drug Administration:
If you are found to be eligible to take part in this study, you will be admitted to the
hospital. On the first day, before starting the hATG, a small amount of diluted hATG will
be injected under your skin to make sure that you are not allergic to it. If you have a
reaction, more testing may be done. If the reaction is severe, you will be taken off study.
On Days 1-4 you will receive methylprednisolone by vein over about 10 minutes. You will
receive hATG by vein over 8 hours.
If you are able to tolerate ATG without any significant side effects, you should be out of
the hospital in about 4-5 days.
On Days 1-180 you will take cyclosporine by mouth 2 times a day at about the same time
On Day 5 (or starting on Day 5) you will receive filgrastim or pegfilgrastim by an injection
under your skin. Your doctor will decide which drug you will receive. If you receive
pegfilgrastim, you will receive it one time on Day 5. If you receive filgrastim, you will
receive it starting on Day 5. You will continue to receive it until your blood counts
You will be given standard drugs to help decrease the risk of side effects. You may be
given a drug called eltrombopag to help increase your platelet counts if you have low
platelets or complications related to low platelets. You may ask the study staff for
information about how the drugs are given and their risks.
One time weekly for the first 4-6 weeks and then 1 time a month during Months 2-6
- Your complete medical history will be recorded.
- You will have a physical exam, including measurement of your height, weight, and vital
signs (blood pressure, heart rate, breathing rate, and temperature).
- Blood (about 2 tablespoons) will be drawn for routine tests.
At the end of Month 3 and then when the doctor thinks it is needed, you will have a bone
marrow aspirate/biopsy to check the status of the disease.
Length of Treatment:
You may continue taking the study drug for up to 6 months. You will no longer be able to
take the study drug if the disease gets worse, if intolerable side effects occur, or if you
are unable to follow study directions.
Your participation on the study will be over once you have completed the follow-up visits.
When you are off treatment, every 6-12 months you will be called by a member of the study
staff. You will be asked about any side effects you may be having. The phone calls will
take about 5-10 minutes.
This is an investigational study. hATG, cyclosporine, methylprednisolone, and
filgrastim/pegfilgrastim are all FDA approved and commercially available for use in patients
with AA and MDS. The use of filgrastim/pegfilgrastim with this drug combination is
Up to 100 patients will take part in this study. All will be enrolled at MD Anderson.
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Achievement of Response
Achievement of response defined: In aplastic anemia (AA) patients, response rate measured by complete response (CR) or partial response (PR); in myelodysplastic syndrome (MDS) patients, the response rate is measured by CR, PR, or hematologic improvement (HI). Criteria for HI per the Modified International Working Group Response Criteria in MDS. Patients eligible for trial and receive any dose of hATG and cyclosporine, included in estimating response rates and counted as treatment failures if response cannot be assessed for any reason.
Tapan Kadia, MD
UT MD Anderson Cancer Center
United States: Institutional Review Board
|UT MD Anderson Cancer Center||Houston, Texas 77030|