Treating Aplastic Anemia and Myelodysplastic Syndrome with Combination Therapy

Phase II Study of Horse Anti-Thymocyte Globulin (hATG), Cyclosporine, Methylprednisolone, and GCSF (Filgrastim or Pegfilgrastim) in Patients With Aplastic Anemia (AA), or Low/Int-1 Risk Myelodysplastic Syndrome (MDS)

This study is testing a combination of four treatments to see if it can help people with aplastic anemia or certain types of myelodysplastic syndrome improve their blood counts and overall health.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 140 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: M.D. Anderson Cancer Center (Academic / other)
• Locations: 1 site (Houston, Texas)
• Trial ID: NCT01624805 on ClinicalTrials.gov

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.

Who should consider this trial

Good fit: Ideal candidates include adults aged 18 and older diagnosed with aplastic anemia or low to intermediate-risk myelodysplastic syndrome who are not candidates for stem cell transplant.

Not a fit: Patients currently undergoing cytotoxic or immunosuppressive therapy, or those with severe organ dysfunction, may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly improve blood counts and overall health outcomes for patients with aplastic anemia and myelodysplastic syndrome.

How similar studies have performed: Other studies have shown promise with similar combination therapies, indicating potential for success in this approach.

Eligibility criteria

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 this trial is running

Houston, Texas

• 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

Conditions Aplastic Anemia; de Novo Myelodysplastic Syndrome; Myelodysplastic Syndrome; Previously Treated Myelodysplastic Syndrome