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A Randomized Trial of Antithymocyte Globulin Versus Cyclosporine to Treat the Cytopenia of Myelodysplastic Syndrome

Phase 2
Not Enrolling
Hematologic Diseases, Myelodysplastic Syndromes

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Trial Information

A Randomized Trial of Antithymocyte Globulin Versus Cyclosporine to Treat the Cytopenia of Myelodysplastic Syndrome

Approximately 40% of the patients with myelodysplastic syndrome (MDS) die as a consequence
of their cytopenia. As in aplastic anemia, the cytopenia of MDS may be partly due to
cytotoxic T cell activity. Immunosuppressive therapy may therefore reverse the cytopenia.
In a phase II pilot study of anti-thymocyte globulin (ATG) to treat myelodysplastic syndrome
(MDS); 41% of patients (61% of patients with refractory anemia) have responded in terms of
transfusion independence. Recently, Jonasova et al [32] reported a 82% substantial
hematological response rate in 18 patients with MDS of the refractory anemia (RA) subtype
treated with cyclosporine alone. Just over 50% of the patients in this series had MDS of
the hypocellular type. Cyclosporine alone if indeed efficacious would be a powerful
therapeutic option that could be readily used by hematologists in the community to treat
patients with MDS. This efficacy needs to be proven in a larger study which includes
patients with the other subtypes of MDS and more patients with the non-hypocellular forms of
MDS (which constitute approximately 70% of the cases in the community). As MDS is a
heterogeneous group of disorders, a randomized comparison with the other immunomodulating
intervention of proven benefit, ATG, is appropriate. In this randomized study patients with
MDS will receive either ATG alone or cyclosporine alone.

Inclusion Criteria

MDS of RA & RAEB sub-types.

No current treatment and off other treatments for at least two weeks.

ECOG performance status less than or equal to 2.

No MDS of FAB sub-group' refractory anemia with ring sideroblasts' (RARS).

No transforming to acute leukemia (FAB sub-group RAEB-Tie.; greater than 20% blasts in
marrow aspirate).

No hypoplastic marrow without one major or two minor criteria as outlined in table 3 of
the appendix.

No one being treatment with growth factors or cyclosporine within four weeks prior to
entry to protocol.

No previous treatment with ATG.

No ECOG performance status of greater than 2.

No active uncontrolled infection.

No women which current pregnancy, or unwilling to take oral contraceptives if of
childbearing potential.

No patients for whom bone marrow transplant is indicated as primary therapy.

No one age less than 18 years.

Must be able to give informed consent.

No HIV positive patients.

No active malignant disease (excluding basal cell carcinoma).

No one with serum creatine greater than 2mg/dl.

No patients who are moribund or patients with concurrent hepatic, renal, cardiac,
metabolic, or any disease of such severity that death within 7-10 days is likely.

Type of Study:


Study Design:

Endpoint Classification: Safety/Efficacy Study, Primary Purpose: Treatment


United States: Federal Government

Study ID:




Start Date:

December 1998

Completion Date:

May 2000

Related Keywords:

  • Hematologic Diseases
  • Myelodysplastic Syndromes
  • Anemia
  • Immunosuppressive
  • Phase II Trial
  • Pre-Leukemia
  • Therapy
  • Myelodysplastic Syndrome
  • Hematologic Diseases
  • Myelodysplastic Syndromes
  • Preleukemia



Warren G. Magnuson Clinical Center (CC) Bethesda, Maryland  20892