Erlotinib Study for Myelodysplastic Syndrome (MDS)
Condition: Myelodysplastic Syndrome
MeSH terms: Preleukemia; Myelodysplastic Syndromes; Syndrome
Intervention: Erlotinib
Intervention description: Participants took erlotinib at least 1 hour before, or 2 hours after they ate a meal or snack. Participants were advised to take erlotinib at around the same time every day.
Intervention MeSH terms: Erlotinib Hydrochloride
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 39.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No
Keywords: MDS; blood diseases; bone marrow; hematopoietic; leukemia

Summary:

      The purpose of this research study is to find out what effects, good and/or bad, erlotinib
      has on the patient and their myelodysplastic syndrome. Erlotinib has been approved by the
      Food and Drug Administration (FDA) to treat non-small cell lung cancer; however, erlotinib
      use in this study is considered investigational as the FDA has not approved it for the
      treatment of myelodysplastic syndrome.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Patients must have an established diagnosis of myelodysplastic syndrome (MDS) and have
             either: Low or intermediate 1 risk disease by International Prognostic Scoring System
             (IPSS) for MDS with symptomatic anemia (defined as hemoglobin less than 10.0 g/dl) or
             transfusion dependent anemia (defined as requiring ≥ 4 units of red blood cells (RBCs)
             administered with a pretreatment hemoglobin value of ≤ 9 g/dL in the 8 weeks prior to
             Day 1 of treatment in this study). Patients with anemia must have no response to at
             least to 6 weeks trial of erythroid stimulating agents (ESA) [erythropoietin/
             darbepoetin]. Patients with serum erythropoietin levels more than 500 mU/ ml on
             diagnosis are eligible to the study without erythropoietin/darbepoetin prior
             treatment. Patients who do not meet anemia criteria are still eligible if they had
             thrombocytopenia with two or more platelet counts < 50 x 10^9/L or a significant
             clinical hemorrhage requiring platelet transfusions or if they had neutropenia with an
             absolute neutrophil count (ANC) < 1 x 10^9/L; Intermediate-2 or high risk MDS by IPSS.

          -  Patients ≥ 60 years with Acute Myeloid Leukemia (AML) by WHO classification and
             myeloblasts percentage 20-30% (RAEB-t by MDS French-American-British (FAB)
             classification) are eligible for the study if deemed not suitable for induction
             chemotherapy or declined that option.

          -  All prior treatment must have been discontinued 28 days prior to Day 1 of treatment in
             this study except (ESA) and colony stimulating factors where it should be stopped 14
             days prior to start therapy on study, and hydroxyurea should be stopped 2 days before.

          -  Prior bone marrow or stem cell transplant is allowed.

          -  Secondary or therapy related MDS patients are eligible.

          -  Patients with chronic myelomonocytic leukemia (CMML) are eligible.

          -  Patients must have a performance status of 0 - 2 by Zubrod performance status
             criteria.

          -  Pretreatment pathology materials must be available for morphologic review. Collection
             of blood and marrow specimens for pathology review must be completed within 28 days
             prior to registration.

          -  No other prior malignancy is allowed except for the following: adequately treated
             basal cell or squamous cell skin cancer, in situ cervical cancer, adequately treated
             Stage I or II cancer from which the patient is currently in complete remission, or any
             other cancer from which the patient has been disease-free for at least 2 years.

          -  In calculating days of tests and measurements, the day a test or measurement is done
             is considered Day 0. Therefore, if a test is done on a Monday, the Monday four weeks
             later would be considered Day 28. This allows for efficient patient scheduling without
             exceeding the guidelines. If Day 28 or 60 falls on a weekend or holiday, the limit may
             be extended to the next working day.

          -  All patients must be informed of the investigational nature of this study and must
             sign and give written consent in accordance with institutional and federal guidelines.

        Exclusion Criteria:

          -  Patients must not have received prior remission induction chemotherapy as treatment
             for MDS.

          -  Patients must not be pregnant or nursing because of the potential risks of the drugs
             used in this study. Women/men of reproductive potential may not participate unless
             they have agreed to use an effective contraceptive method.

          -  Patients who are known HIV positive are not eligible for this study.