Methylprednisolone, Horse Anti-Thymocyte Globulin, Cyclosporine, Filgrastim, and/or Pegfilgrastim or Pegfilgrastim Biosimilar in Treating Patients With Aplastic Anemia or Low or Intermediate-Risk Myelodysplastic Syndrome
Methylprednisolone, Horse Anti-Thymocyte Globulin, Cyclosporine, Filgrastim, and/or Pegfilgrastim or Pegfilgrastim Biosimilar in Treating Patients With Aplastic Anemia or Low or Intermediate-Risk Myelodysplastic Syndrome
This phase II trial studies methylprednisolone, horse anti-thymocyte globulin, cyclosporine,
 filgrastim, and/or pegfilgrastim or pegfilgrastim biosimilar in treating patients with
 aplastic anemia or low or intermediate-risk myelodysplastic syndrome. Horse anti-thymocyte
 globulin is made from horse blood and targets immune cells known as T-lymphocytes. Since
 T-lymphocytes are believed to be involved in causing low blood counts in aplastic anemia and
 in some cases of myelodysplastic syndromes, killing these cells may help treat the disease.
 Methylprednisolone and cyclosporine work to suppress immune cells called lymphocytes. This
 may help to improve low blood counts in aplastic anemia and myelodysplastic syndromes.
 Filgrastim and pegfilgrastim are designed to cause white blood cells to grow. This may help
 to fight infections and help improve the white blood cell count. Giving methylprednisolone
 and horse anti-thymocyte globulin together with cyclosporine, filgrastim, and/or
 pegfilgrastim may be an effective treatment for patients with aplastic anemia or
 myelodysplastic syndrome.
PRIMARY OBJECTIVES:

 I. To evaluate the efficacy of the combination of hATG (horse anti-thymocyte globulin),
 methylprednisolone, cyclosporine, and GCSF (filgrastim) in achieving response (complete
 response [CR], partial response [PR], or hematologic improvement [HI]) in patients with
 aplastic anemia, or myelodysplastic syndromes (MDS).

 SECONDARY OBJECTIVES:

 I. To assess the safety, tolerability, and toxicities of the combination of hATG,
 methylprednisolone, cyclosporine, and GCSF in patients with aplastic anemia, or MDS. II. To
 assess time to response, response duration, and overall survival of patients with aplastic
 anemia, or MDS being treated with the combination of hATG, methylprednisolone, cyclosporine,
 and GCSF.

 OUTLINE:

 Patients receive methylprednisolone intravenously (IV) over 10 minutes on days 1-4 and IV or
 orally (PO) with taper over days 5-30. Patients also receive horse anti-thymocyte globulin IV
 over 8 hours daily on days 1-4, cyclosporine PO twice daily (BID) on days 1-180, and
 pegfilgrastim or pegfilgrastim biosimilar subcutaneously (SC) on day 5 and/or filgrastim SC
 beginning on day 5 and continuing until absolute neutrophil count recovers. Treatment
 continues for up to 6 months in the absence of disease progression or unacceptable toxicity.

 After completion of study treatment, patients are followed up every 6-12 months.
Inclusion Criteria:

 -  Patients with the diagnosis of MDS (low, int-1 by International Prognostic Scoring
 System [IPSS], or hypocellular) who are either previously treated or untreated are
 eligible for this trial

 -  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

 -  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

 -  Bilirubin < 2 mg/dL

 -  Aspartate aminotransferase (AST) < 3 x upper limit of normal (ULN)

 -  Creatinine < 2.5 x ULN

 -  Eastern Cooperative Oncology Group (ECOG) performance status of =< 2

 -  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

 -  A negative urine pregnancy test is required within 1 week for all women of
 childbearing potential prior to enrolling on this trial

 -  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

 -  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 (e.g. neutropenia)

 Exclusion Criteria:

 -  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

 -  Known human immunodeficiency virus (HIV) infection

 -  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

 -  Patient with documented hypersensitivity to any of the component medications