12th Congress of the European Hematology ... - Haematologica

12 th Congress of the European Hematology Association
ulation. Aims. To evaluate the severity of iron burden in patients with
Low/Int-1-risk MDS using baseline data from two ongoing Phase II studies.
Methods. Studies US02 and US03 are evaluating the efficacy and
safety of deferasirox, and will enroll 30 and 150 patients, respectively,
with Low- or Int-1-risk MDS. Informed consent has been obtained from
all patients. Deferasirox dosing starts at 20 mg/kg/day, up to a maximum
of 30 mg/kg/day. Iron burden is being monitored by monthly serum ferritin
measurements, as well as serum iron, transferrin, transferrin saturation,
labile plasma iron (LPI) and liver iron concentration (LIC). In addition,
creatinine, calculated creatinine clearance and hematological status
are being monitored. Results. To date, US02 and US03 have enrolled 14
(aged 55-81 years) and 84 (aged 47-87 years) patients, respectively.
Enrolled patients had Low-risk (50% in US02; 28% in US03) or Int-1risk
(50% in US02; 72% in US03) MDS. A total of 51 patients (61%) in
US03 had received DFO prior to enrolment, and two had received
deferasirox. The Table 1 summarizes baseline iron parameters in patients
enrolled in both trials. At baseline, concurrent therapies included 5-azacytidine
(Vidaza; 6% of patients in US03), lenalidomide (Revlimid; 14%
in US02 and 1% in US03) and hydroxyurea (7% in US02). In US02 and
US03, baseline calculated creatinine clearance was normal (>80 mL/min)
in 46% and 49% patients, respectively; mildly abnormal (51-80 mL/min)
in 46% and 40% patients, respectively; and moderately abnormal (30'50
mL/min) in 8% and 12% patients, respectively. In US02 and US03, pretreatment
baseline cytopenias noted, in addition to anemia, included
neutropenia (