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Effect of deferasirox + erythropoietin vs erythropoietin on erythroid response in Low/Int‐1‐risk MDS patients: Results of the phase II KALLISTO trial
Author(s) -
Gattermann Norbert,
Coll Rosa,
Jacobasch Lutz,
Allameddine Allameddine,
Azmon Amin,
DeBonnett Laurie,
Bruederle Andreas,
Jin Jie
Publication year - 2018
Publication title -
european journal of haematology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.904
H-Index - 84
eISSN - 1600-0609
pISSN - 0902-4441
DOI - 10.1111/ejh.13096
Subject(s) - deferasirox , erythropoietin , medicine , myelodysplastic syndromes , anemia , clinical endpoint , gastroenterology , ineffective erythropoiesis , randomized controlled trial , lower risk , erythropoiesis , adverse effect , phases of clinical research , clinical trial , confidence interval , thalassemia , bone marrow
Objectives Erythropoiesis‐stimulating agents ( ESA s) remain first‐choice to treat symptomatic anemia and delay transfusion dependence in most patients with lower‐risk myelodysplastic syndromes ( MDS ) without del(5q). Deferasirox increased erythroid responses in some lower‐risk MDS patients in clinical trials, and adding low‐dose deferasirox to ESA treatment may further improve erythroid response. Methods KALLISTO ( NCT01868477 ) was a randomized, open‐label, multicenter, phase II study. Lower‐risk MDS patients received deferasirox at 10 mg/kg/d (dispersible tablets) or 7 mg/kg/d (film‐coated tablets) plus erythropoietin (n = 11), or erythropoietin alone (n = 12) for 24 weeks. The primary endpoint was the between‐group difference in erythroid response within 12 weeks. Results Erythroid response occurred in 27.3% of patients receiving deferasirox plus erythropoietin vs 41.7% of patients receiving erythropoietin alone within 12 weeks (difference 14.4%; 95% CI −24.0, 48.16). Within 24 weeks, the hematologic response rate was 27.3% with deferasirox plus erythropoietin vs 50% with erythropoietin alone, and hematologic improvement rates were 45.5% vs 100%. Deferasirox plus erythropoietin was generally well tolerated. Conclusions In this small pilot study, combining low‐dose deferasirox with erythropoietin did not improve erythroid response. It remains of interest to investigate early chelation approaches with even lower deferasirox doses plus erythropoietin in lower‐risk MDS patients before the onset of transfusion dependence.