Patient case: 49-year-old female with Gastrointestinal stromal tumor. Genetic variant: KIT Exon 9 (A502_Y503dup). Other conditions: None.

Clinical trial: Efficacy of Ferric Carboxymaltose in Patients With Gastrointestinal Stromal Tumor (GIST) Receiving Systemic Therapy

Summary: The goal of this clinical research study is to compare Injectafer® (ferric carboxymaltose) with an iron supplement to learn which may be more effective in improving red blood cell counts in patients who have iron-deficiency anemia (a low red blood cell count) because of a gastrointestinal stromal tumor (GIST) and/or systemic therapy.
  The safety of ferric carboxymaltose will also be studied.

Detailed description: Study Groups and Study Drug Administration:
  If participant is found to be eligible to take part in this study, they will be randomly assigned (as in the flip of a coin) to 1 of 2 study groups. This is done because no one knows if one group is better, the same, or worse than the other.
  -  If participant is in Group A, they will receive ferric carboxymaltose injection by vein over about 15 minutes. Participant will receive 2 injections about 7 days apart (for example, on Days 0 [the day participant is assigned to a study group] and 7).
  -  If participant is in Group B, they will take iron supplements by mouth every day. This is considered standard of care for iron deficiency anemia and the study staff will discuss with participant which iron supplements they will take and their risks.
  Participant and the study staff will know to which group they are assigned.
  Length of Study:
  Participant may receive up to 2 injections of ferric carboxymaltose (if participant is in Group A) or up to 3 months of oral iron supplements (if participant is in Group B). Participant will no longer be able to take the study drug if intolerable side effects occur or if they are unable to follow study directions.
  Participation on the study will be over after participant has completed the Week 24 visit.
  Study Visits:
  Baseline (within 1 week after participant has been assigned to a study group):
  -  Participant will have a physical exam.
  -  Participant will complete a questionnaire about their health. It should take about 5 minutes to complete.
  -  Blood (about 1 tablespoon) will be drawn for routine tests and to test the level of iron in participant's blood.
  One (1) time every week during Months 1-3, blood (about 1 tablespoon) will be drawn for routine tests.
  During Weeks 4, 8, 12, and 24:
  -  Participant will have a physical exam.
  -  Participant will complete the same questionnaire they did at baseline.
  -  Blood (about 1 tablespoon) will be drawn for routine tests and to test the level of iron in participant's blood.
  During Weeks 16 and 20, blood (less than 1 tablespoon) will be drawn to test the level of iron in participant's blood.
  This is an investigational study. Ferric carboxymaltose is FDA approved and commercially available to treat iron deficiency anemia; however, it is considered investigational to use in patients who have cancer-related or systemic therapy-related anemia.
  Up to 50 participants will take part in this study. All will be enrolled at MD Anderson.

Eligibility: Inclusion Criteria:
  1. GIST patients with IDA planned to start or are receiving systemic therapy with TKIs.
  2. Evidence of iron deficiency anemia including, Hgb < 11 g/dL, but > 8 g/dL; and transferrin saturation (TSAT) < 20%.
  3. No H/O allergic reaction to iron therapy.
  4. No clinical signs active of bleeding.
  5. Adequate hematologic (ANC > 1500/mm^3, platelet count > 100,000/mm^3), renal (serum creatinine < 1.5mg/dL), and hepatic (serum bilirubin count < 1.5 x normal and serum glutamic-oxaloacetic transaminase (SGOT) or serum glutamic pyruvic transaminase (SGPT) < 3 x normal) functions.
  6. Patients must have Eastern Cooperative Oncology Group (ECOG) performance status of 0 - 2.
  7. Signed informed consent to the study.
  8. Male and Females of child bearing potential must use acceptable methods of birth control which include oral contraceptives, spermicide with either a condom, diaphragm or cervical cap, use of an intrauterine device (IUD) or abstinence.
  9. Patients are required to read and understand English to comply with protocol requirements.
  10. Age >=18 years old.
  11. Life expectancy of at least 6 months.
  Exclusion Criteria:
  1. Pregnant or lactating women.
  2. Patients with any co-morbid condition which renders patients at high risk of treatment complication.
  3. Patient has uncontrolled angina, congestive heart failure (New York Heart Association > class II or known ejection fraction < 40%), uncontrolled cardiac arrhythmia or hypertension, or acute myocardial infarction within 3 months.
  4. Patient has an active seizure disorder. (Patients with a previous history of seizure disorders will be eligible for the study, if they have had no evidence of seizure activity, and they have been free of antiseizure medication for the previous 5 years).
  5. Psychological, social, familial, or geographical reasons that would prevent scheduled visits and follow-up.
  6. Prior surgery or radiotherapy (RT) within 2 weeks of study entry.
  7. Known hypersensitivity reaction to any component of ferric carboxymaltose.
  8. Any anemia treatment within 4 weeks before inclusion (oral iron, IV iron, or erythropoiesis-stimulating agents), or transfusion of PRBCs in 2 weeks.
  9. Hemochromatosis or other iron storage disorders.
  10. Known positive hepatitis with evidence of active disease.
  11. Patients with overt bleeding.
  12. Ferritin >/= 800 ng/mL.

Relevance to patient case: not relevant