Deferoxamine in Myeloablative Allogeneic Stem Cell Transplantation for Patients With Myelodysplastic Syndromes or Acute Leukemia and Iron Overload

Condition(s) targeted: Acute Myeloid Leukemia; Acute Lymphoblastic Leukemia; Myelodysplastic Syndrome

Intervention: deferoxamine mesylate (Drug)

Phase: N/A

Status: Not yet recruiting

Sponsored by: Dana-Farber Cancer Institute

Official(s) and/or principal investigator(s):
Philippe Armand, MD, PhD, Principal Investigator, Affiliation: Dana-Farber Cancer Institute

Overall contact:
Philippe Armand, MD, PhD, Phone: 617-632-2305

The objective of this research study is to determine the safety and feasibility of chelation therapy with deferoxamine for patients with iron overload who are receiving a stem cell transplant. Patients who have iron overload prior to stem cell transplantation may have more toxicity from the transplantation procedure, and thus may benefit from an attempt at iron chelation pre- and peri-transplantation. In this study we are examining the use of deferoxamine starting 1 to 3 months prior to transplantation and continuing through the preparative regimen.

Official title: A Pilot Study of Deferoxamine Before and During Myeloablative Allogeneic Stem Cell Transplantation for Patients With Myelodysplastic Syndromes or Acute Leukemia and Iron Overload

Study design: Prevention, Non-Randomized, Open Label, Single Group Assignment, Safety/Efficacy Study

Primary outcome: Grade 3 or above toxicity attributable to treatment drug

Secondary outcome:

To estimate 1-year transplant-related mortality, relapse, disease-free and overall survival in this cohort of patients.

To measure serum hepcidin levels in patients before and after transplantation.

Minimum age: 18 Years. Maximum age: N/A. Gender(s): Both.

Criteria:

Inclusion Criteria:

- 18 years of age or older

- Histologically confirmed acute myeloid leukemia, acute lymphoblastic leukemia, or

myelodysplastic syndrome

- Planned allogeneic stem cell transplantation with myeloablative conditioning regimen;

the planned date of transplantation must be at least 4 weeks from time of enrollment

- Severe iron overload as defined by BOTH: Ferritin greater than or equal to 1500ng/ml

(at the time of donor availability) and Liver iron content estimated greater than or equal to 7mg/g dry weight by MRI (at the time of donor availability)

- Patients with a history of prior autologous transplantation will be eligible for this

study

Exclusion Criteria:

- Contraindication to magnetic resonance imaging (MRI)

- Creatinine >2. 0mg/dl or creatinine clearance <50ml/min

- Active uncontrolled bacterial or fungal infection

- History of mucormycosis

- Pre-existing clinically apparent retinal neuropathy. If patients have clinically

apparent visual loss at the time of screening, they will be excluded if either they have known retinal neuropathy or if this cannot be excluded by further testing

- Pre-existing clinically apparent sensorineural hearing loss. If patients have

auditory loss at the time of screening, they will be excluded if either they have known sensorineural hearing loss, or if this cannot be excluded by further testing

- Cardiac disease

- Pregnancy or inability or unwillingness to use contraception during the time of the

study

- Lactating patients

Philippe Armand, MD, PhD, Phone: 617-632-2305

Dana-Farber Cancer Institute, Boston, Massachusetts 02115, United States

Brigham and Women's Hospital, Boston, Massachusetts 02115, United States

Starting date: April 2008
Ending date: April 2011
Last updated: April 9, 2008