PMID- 24959339
OWN - NLM
STAT- PubMed-not-MEDLINE
DCOM- 20140624
LR  - 20211021
IS  - 2035-3006 (Print)
IS  - 2035-3006 (Electronic)
IS  - 2035-3006 (Linking)
VI  - 6
IP  - 1
DP  - 2014
TI  - Iron chelation therapy with deferasirox in the management of iron overload in 
      primary myelofibrosis.
PG  - e2014042
LID - 10.4084/MJHID.2014.042 [doi]
LID - e2014042
AB  - Deferasirox (DSX) is the principal option currently available for 
      iron-chelation-therapy (ICT), principally in the management of myelodysplastic 
      syndromes (MDS), while in primary myelofibrosis (PMF) the expertise is limited. 
      We analyzed our experience in 10 PMF with transfusion-dependent anemia, treated 
      with DSX from September 2010 to December 2013. The median dose tolerated of DSX 
      was 750 mg/day (10 mg/kg/day), with 3 transient interruption of treatment for 
      drug-related adverse events (AEs) and 3 definitive discontinuation for grade 3/4 
      AEs. According to IWG 2006 criteria, erythroid responses with DSX were observed 
      in 4/10 patients (40%), 2 of them (20%) obtaining transfusion independence. 
      Absolute changes in median serum ferritin levels (Delta ferritin) were greater in 
      hematologic responder (HR) compared with non-responder (NR) patients, already at 
      6 months of ICT respect to baseline. Our preliminary data open new insights 
      regarding the benefit of ICT not only in MDS, but also in PMF with the 
      possibility to obtain an erythroid response, overall in 40 % of patients. HR 
      patients receiving DSX seem to have a better survival and a lower incidence of 
      leukemic transformation (PMF-BP). Delta ferritin evaluation at 6 months could 
      represent a significant predictor for a different survival and PMF-BP. However, 
      the tolerability of the drug seems to be lower compared to MDS, both in terms of 
      lower median tolerated dose and for higher frequency of discontinuation for AEs. 
      The biological mechanism of action of DSX in chronic myeloproliferative setting 
      through an independent NF-kappaB inhibition could be involved, but further 
      investigations are required.
FAU - Elli, Elena Maria
AU  - Elli EM
AD  - Hematology Division, San Gerardo Hospital, Monza, Italy.
FAU - Belotti, Angelo
AU  - Belotti A
AD  - Hematology Division, San Gerardo Hospital, Monza, Italy.
FAU - Aroldi, Andrea
AU  - Aroldi A
AD  - Hematology Division, San Gerardo Hospital, Monza, Italy.
FAU - Parma, Matteo
AU  - Parma M
AD  - Hematology Division, San Gerardo Hospital, Monza, Italy.
FAU - Pioltelli, Pietro
AU  - Pioltelli P
AD  - Hematology Division, San Gerardo Hospital, Monza, Italy.
FAU - Pogliani, Enrico Maria
AU  - Pogliani EM
AD  - Hematology Division, San Gerardo Hospital, Monza, Italy.
LA  - eng
PT  - Journal Article
DEP - 20140601
PL  - Italy
TA  - Mediterr J Hematol Infect Dis
JT  - Mediterranean journal of hematology and infectious diseases
JID - 101530512
PMC - PMC4063602
EDAT- 2014/06/25 06:00
MHDA- 2014/06/25 06:01
PMCR- 2014/01/01
CRDT- 2014/06/25 06:00
PHST- 2014/03/28 00:00 [received]
PHST- 2014/05/02 00:00 [accepted]
PHST- 2014/06/25 06:00 [entrez]
PHST- 2014/06/25 06:00 [pubmed]
PHST- 2014/06/25 06:01 [medline]
PHST- 2014/01/01 00:00 [pmc-release]
AID - mjhid-6-1-e2014042 [pii]
AID - 10.4084/MJHID.2014.042 [doi]
PST - epublish
SO  - Mediterr J Hematol Infect Dis. 2014 Jun 1;6(1):e2014042. doi: 
      10.4084/MJHID.2014.042. eCollection 2014.