Chelation efficacy and erythroid response during deferasirox treatment in patients with myeloproliferative neoplasms in fibrotic phase.
At present, very few data are available on deferasirox (DFX) in the treatment of patients with Philadelphia-negative myeloproliferative neoplasms in fibrotic phase (FP-MPN) and transfusion dependence. To address this issue, a retrospective analysis of 28 patients (22 male and 6 female) with FP-MPN and iron overload secondary to transfusion dependence was performed, based on patients enrolled in the database of our regional cooperative group who received treatment with DFX. DFX was started after a median interval from diagnosis of 12.8 months (IR 7.1-43.1) with median ferritin values of 1415 ng/mL (IR 1168-1768). Extra-hematological toxicity was reported in 16 of 28 patients (57.1%), but only two patients discontinued treatment due to toxicity. Among 26 patients evaluable for response (≥6 months of treatment), after a median treatment period of 15.4 months (IR 8.1-22.3), 11 patients (42.3%) achieved a stable and consistent reduction in ferritin levels <1000 ng/mL. As for hematological improvement, 6 of 26 patients (23%) showed a persistent (>3 months) rise of Hb levels >1.5 g/dL, with disappearance of transfusion dependence in four cases. Treatment with DFX is feasible and effective in FP-MPN with iron overload. Moreover, in this setting, an erythroid response can occur in a significant proportion of patients.
Department of Cellular Biotechnologies and Hematology, University "Sapienza", Rome, Italy.
Roberto
R
Latagliata
Department of Cellular Biotechnologies and Hematology, University "Sapienza", Rome, Italy.
Chiara
C
Montagna
Hematology, Sant'Andrea Hospital, Rome, Italy.
Raffaele
R
Porrini
Hematology, University Tor Vergata, Rome, Italy.
Ambra
A
Di Veroli
Hematology, Sandro Pertini Hospital, Rome, Italy.
Sabrina Crescenzi
SC
Leonetti
Hematology, Sant'Eugenio Hospital, Rome, Italy.
Pasquale
P
Niscola
Hematology, Santa Maria Goretti Hospital, Latina, Italy.
Fabrizio
F
Ciccone
Hematology, Regina Elena - IFO Hospital, Rome, Italy.
Antonio
A
Spadea
Department of Cellular Biotechnologies and Hematology, University "Sapienza", Rome, Italy.
Massimo
M
Breccia
Hematology, University Tor Vergata, Rome, Italy.
Luca
L
Maurillo
Hematology, Polo Universitario Pontino, Latina, Italy.
Angela
A
Rago
Hematology, San Camillo Hospital, Rome, Italy.
Francesca
F
Spirito
Hematology, San Giovanni Hospital, Rome, Italy.
Michele
M
Cedrone
Hematology, University "Campus Biomedico", Rome, Italy.
Marianna
M
De Muro
Hematology, Belcolle Hospital, Viterbo, Italy.
Marco
M
Montanaro
Hematology, Nuovo Regina Margherita Hospital, Rome, Italy.
Alessandro
A
Andriani
Hematology, Sandro Pertini Hospital, Rome, Italy.
Antonino
A
Bagnato
Hematology, Sant'Andrea Hospital, Rome, Italy.
Enrico
E
Montefusco
Department of Cellular Biotechnologies and Hematology, University "Sapienza", Rome, Italy.
Giuliana
G
Alimena
D000368:Aged; D001565:Benzoates / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D015913:Chelation Therapy; D000077588:Deferasirox; D004909:Erythrocyte Indices; D004920:Erythropoiesis / Q000187:drug effects*; D005260:Female; D005355:Fibrosis; D005500:Follow-Up Studies; D006801:Humans; D007502:Iron Chelating Agents / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D019190:Iron Overload / Q000097:blood / Q000175:diagnosis / Q000188:drug therapy* / Q000209:etiology*; D008297:Male; D008875:Middle Aged; D009196:Myeloproliferative Disorders / Q000150:complications* / Q000401:mortality / Q000473:pathology* / Q000628:therapy; D012189:Retrospective Studies; D065227:Transfusion Reaction; D016896:Treatment Outcome; D014230:Triazoles / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*
D001565:Benzoates; D007502:Iron Chelating Agents; D014230:Triazoles; D000077588:Deferasirox
chelation treatment; deferasirox; hematological improvement; myeloproliferative neoplasms