erlotinib study for myelodysplastic syndrome (mds)

Conditions: Myelodysplastic Syndrome

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.

screening period: informed consent, physical examination, medical history report, blood tests, pregnancy test (if applicable), list of current medications, description of symptoms, chest x-ray, ecg, bone marrow aspirate/biopsy within 4 weeks of study start. weeks 2,6,10 and 14: blood tests. weeks 4 and 12: blood tests, physical exam, patients will answer question about how they are feeling and if there are any changes to medication they have taken. weeks 8 and 16: blood tests, physical exam, patients will answer question about how they are feeling and if there are any changes to medication they have taken, bone marrow aspirate/biopsy (if physician has determined the patient has had a clinical response or partial response to treatment. after week 16 (if responding to treatment): have a bone marrow aspirate/biopsy (will be repeated at time of relapse, i.e., more than 50% increase in the percentage of myeloblasts \[leukemia cells\] or drop in blood counts after they improved or requiring regular blood transfusions after not requiring them for at least 8 weeks, or after 1 year in study). after the patient has stopped taking erlotinib: periodic follow-up on patients' status.

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.

Keywords: mds blood diseases bone marrow hematopoietic leukemia