Hemosiderosis, Myelodysplastic Syndromes
Conditions
Keywords
MDS, Chronic transfusional hemosiderosis, Deferasirox
Brief summary
Open label, single arm study on Deferasirox treatment in MDS patients with chronic transfusional hemosiderosis. Patients receive daily oral dosis of Deferasirox in order to eliminate the quantity of iron administered during transfusions and, if needed, to reduce the overload of already present iron. After an screening phase in which patients are evaluated according to eligibility criteria, a one year treatment phase foresees monthly visits to evaluate safety and efficacy signs.
Detailed description
It has been widely shown that an appropriate chelating therapy in chronic anemias transfusion dependent can prevent the overstock of iron and can reduce the already existing overstock reducing, then, the co-morbidity and improving survival. In particular, some authors have shown in MDS affected patients undergoing intensive chelating therapy with deferoxamine haematological recovery with a reduction of the need of transfusions. With the present study, we plan to evaluate the safety and efficacy of a therapy with the new oral chelating Deferasirox in MDS patients with transfusional hemosiderosis. This is an open label, single arm study on Deferasirox treatment in MDS patients with chronic transfusional hemosiderosis. Patients will receive daily oral dosis of Deferasirox in order to eliminate the quantity of iron administered during transfusions and, if needed, to reduce the overload of already present iron. After an screening phase in which patients are evaluated according to eligibility criteria, a one year treatment phase foresees monthly visits to evaluate safety and efficacy signs.
Interventions
DRUGDeferasirox
Sponsors
Gruppo Italiano Malattie EMatologiche dell'Adulto
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Patients, both males and females, with low and intermediate I risk (IPSS score) Myelodysplastic syndrome and transfusion-induced hemosiderosis. * Age \>=18 years * Patients who never received chelation therapy or who received a therapy with Desferal after a day of wash out * Medical history of at least 20 blood transfusions (equivalent to 100 ml/kg of red cells concentrate). * Availability of data concerning blood transfusions during the 12 weeks before screening * Serum ferritin \>= 1000 µg/L at least twice (at least 2 week interval between the 2 analysis) during the year before the screening * Life expectancy \> 12 months * Availability of at least 3 complete blood counts (before transfusions) during the 12 weeks before the screening
Exclusion criteria
* Diagnosis different from MDS (i.e. myelofibrosis) * Severe renal impairment (creatinine clearance \< 60 ml/min) * ALT/AST \> 500 U/L * Active B and/or C hepatitis * Patients treated during the past 4 weeks with experimental drugs for MDS (including thalidomide, azacitidine, arsenic trioxide). These patients become eligible after a wash out of at least 4 weeks * Concomitant treatment with another iron-chelating agent
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| To evaluate the tolerability and safety profile of Deferasirox in pts with MDS with post-transfusional hemosiderosis | On a monthly basis thereafter from baseline assessment. |
Secondary
| Measure | Time frame |
|---|---|
| To evaluate Deferasirox efficacy as chelation therapy in terms of reduction of serum ferritin levels compared to basal levels | At 3, 6, 9, and 12 months from baseline assessment. |
| To evaluate the impact Deferasirox iron chelating therapy vs the normal demand of transfusions in a subgroup of pts that will not receive growth factors or chemotherapy according to their basal characteristics. | On a monthly basis thereafter from baseline assessment. |
| Quality of Life evaluation. | At 3, 6, 9, and 12 months from baseline assessment. |
| Compliance to chelating therapy evaluation. | On a monthly basis thereafter from baseline assessment. |
Countries
Italy
Outcome results
None listed