Treating Aplastic Anemia and Myelodysplastic Syndrome with Combination Therapy

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This phase II trial evaluates the effectiveness of a combination treatment involving methylprednisolone, horse anti-thymocyte globulin, cyclosporine, and filgrastim or pegfilgrastim for patients with aplastic anemia or low to intermediate-risk myelodysplastic syndrome. The study aims to assess how well this combination can improve blood counts and overall patient response. Participants will receive the treatment over a specified period, followed by regular monitoring for response and safety. The goal is to determine both the efficacy and safety of this treatment regimen.

Why it matters

Who can join: the full rules

Inclusion Criteria:

1. Patients with the diagnosis of MDS (Low, Int-1 by IPSS, or hypocellular) who are either previously treated or untreated are eligible for this trial.
2. Patients with the diagnosis of aplastic anemia who are either previously treated or untreated are eligible if they are not currently candidates for an allogeneic stem cell transplant.
3. Patients ages 18 years and older are eligible
4. Patients must have been off of cytotoxic, immunosuppressive (except steroids), or targeted therapy for at least 2 weeks prior to entering this study, and have recovered from the toxic effects of that therapy to grade 1 or less.
5. Adequate organ function as defined below:

   * liver function (bilirubin \< 2mg/dL, AST \<3 x ULN)
   * kidney function (creatinine \< 2.5 x ULN ).
6. ECOG performance status of ≤ 2.
7. Women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry and for the duration of study participation. Should a woman become pregnant or suspect she is pregnant while participating in this study, she should inform her treating physician immediately.
8. A negative urine pregnancy test is required within 1 week for all women of childbearing potential prior to enrolling on this trial.
9. Patient must have the ability to understand the requirements of the study and signed informed consent. A signed informed consent by the patient or his legally authorized representative is required prior to their enrollment on the protocol.
10. Patients should have an indication for therapy for their disease such as transfusion dependence or morbidity associated with their cytopenia(s) such as bleeding, severe fatigue, or frequent/multiple infections (eg. neutropenia).

Exclusion Criteria:

1. Pregnant women are excluded from this study. Because there is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother with the study agents, breastfeeding should be discontinued if the mother is treated on this study.
2. Known HIV infection.
3. Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements.
4. Patient with documented hypersensitivity to any of the component medications.

Where it's running

• M D Anderson Cancer Center
  Houston, Texas, United States Recruiting

Study contacts

• Principal investigator: Tapan M Kadia — M.D. Anderson Cancer Center
• Study coordinator: Tapan Kadia, MD
• Email: tkadia@mdanderson.org
• Phone: 713-563-3534

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.