Open-Label Single-Arm Pilot Study of Deferasirox (Exjade®) in Adult Allogeneic Hematopoietic Stem Cell Transplant Recipients With Transfusional Iron Overload
- Confirmed diagnosis of iron overload, defined as serum ferritin > 1,000 ng/mL and
liver iron concentration ≥ 5 mg iron/g on tissue proton transverse relaxation rates
Magnetic Resonance Imaging (MRI)
- Underwent prior allogeneic hematopoietic stem cell transplantation (HSCT) using
either myeloablative or reduced-intensity conditioning at least 12 months ago
- No evidence of relapse or progression of the primary disease for which allogeneic
HSCT was performed
- Patients who have become red-cell transfusion independent (i.e., no red cell
transfusions within the past 3 months) as well as patients who require red cell
transfusions are eligible
- Meets one of the following criteria:
- Ineligible for phlebotomy (hemoglobin < 11 g/dL, poor intravenous access, or
unable to undergo phlebotomy every 4 weeks)
- Have failed treatment with phlebotomy (serum ferritin > 50% of baseline after 3
months of phlebotomy)
- Refused phlebotomy
- ECOG performance status of 0-2
- Life expectancy ≥ 6 months
- Adequate renal function defined as serum creatinine < or = 1.6 mg/dL and creatinine
clearance of > or = 60 ml/min calculated using the Crockcroft-Gault formula on 2
occasions within 30 days of enrollment
- Sexually active men and women must use an effective method of contraception.
Alternatively, women must have undergone clinically documented total hysterectomy
and/or oophorectomy, or tubal ligation or be postmenopausal.
- Must be able to give written informed consent.
- Prior therapy with deferoxamine allowed provided it was completed ≥ 12 months ago
- Contraindication for performing MRI or inability to undergo MRI because of
claustrophobia or weight (>350 pounds).
- Inability to take medications orally.
- Uncontrolled bacterial, viral, or fungal infection
- ANC ≥ 1,000/mm³
- Hemoglobin ≥ 8.0 g/dL
- Platelet count ≥ 50,000/mm³
- Aspartate aminotransferance (AST) and alanine aminotransferase (ALT) ≤ 5 times the
upper limit of normal
- Less than 4 weeks since prior and no concurrent systemic investigational drug
- Less than 7 days since prior and no concurrent topical investigational drug.
Concurrent non-investigational medications needed to treat concomitant medical
conditions are allowed, with the exception of other chelating agents. Concurrent
growth factors such as epoetin alfa, darbepoetin alfa, filgrastim (G-CSF), and
sargramostim (GM-CSF) allowed. Concurrent irradiated packed red-cell and platelet
transfusions allowed as clinically indicated. Concurrent low-doses of vitamin C
supplements (≤ 200 mg/day) allowed.
- Concurrent iron supplements or multivitamins with iron.
- Aluminum-containing antacid therapies may not be taken simultaneously with
deferasirox, but may be taken 2 hours before or after administration of deferasirox
- On dialysis or status post-renal transplantation
- Pregnant or nursing