PMID- 30558524
OWN - NLM
STAT- MEDLINE
DCOM- 20190111
LR  - 20190111
IS  - 1607-8454 (Electronic)
IS  - 1024-5332 (Linking)
VI  - 24
IP  - 1
DP  - 2019 Dec
TI  - International sentinel site surveillance of patients with transfusional 
      hemosiderosis treated with deferasirox in actual practice setting.
PG  - 238-246
LID - 10.1080/16078454.2018.1558758 [doi]
AB  - OBJECTIVE: The study evaluates the long-term deferasirox treatment of adult and 
      pediatric patients with chronic transfusional iron overload in clinical practice. 
      METHODS: In this non-interventional study, patients were observed for up to 3 
      years from initiation of deferasirox treatment both prospectively and 
      retrospectively for up to 1 year prior to enrollment. The primary end points were 
      the proportion of patients with >/=1 notable increase in serum creatinine (SCr), 
      and >/=1 notable increase in alanine aminotransferase (ALT). RESULTS: Overall, 120 
      patients were enrolled and 51 completed the study, with a limited 3-year dropout 
      rate of 12.5% due to adverse events (AEs). Increase in SCr > 33% above baseline 
      and the age-adjusted ULN (upper limit of normal) was observed in 14 patients 
      (95%CI, 7.1-19.2). The ALT levels >5 x ULN was observed in 1 patient. Most 
      frequent AEs reported during treatment with deferasirox include gastrointestinal 
      disturbances. CONCLUSIONS: The long-term treatment with deferasirox was 
      manageable in most transfusion-dependent patients with no unexpected safety 
      findings. Regular monitoring and an adjusted deferasirox dosing strategy per 
      local labels allowed continued iron chelation treatment and control of 
      transfusional iron in the majority of patients on study.
FAU - El-Beshlawy, Amal
AU  - El-Beshlawy A
AD  - a Cairo University , Cairo , Egypt.
FAU - Inusa, Baba
AU  - Inusa B
AD  - b Evelina Children's Hospital, Guy's and St Thomas' Hospital NHS Trust , London , 
      UK.
FAU - Beneitez Pastor, David
AU  - Beneitez Pastor D
AUID- ORCID: 0000-0002-6541-6600
AD  - c Hematology Department , Hospital Universitari Vall d'Hebron , Barcelona , 
      Spain.
FAU - Xicoy, Blanca
AU  - Xicoy B
AD  - d ICO-Badalona-Hospital Germans Trias i Pujol, Josep Carreras Leukemia Research 
      Institute , Barcelona , Spain.
FAU - Soledada Duran Nieto, Maria
AU  - Soledada Duran Nieto M
AD  - e Departamento de Hematologica , Hospital Materno Infantil de Jaen , Jaen , 
      Spain.
FAU - Bruederle, Andreas
AU  - Bruederle A
AD  - f Novartis Pharma AG , Basel , Switzerland.
FAU - Azmon, Amin
AU  - Azmon A
AD  - f Novartis Pharma AG , Basel , Switzerland.
FAU - Gilotti, Geralyn
AU  - Gilotti G
AD  - g Novartis Pharmaceuticals Corporation , East Hanover , NJ , USA.
FAU - Elalfy, Mohsen
AU  - Elalfy M
AD  - h Ain Shams University , Cairo , Egypt.
LA  - eng
PT  - Clinical Trial
PT  - Journal Article
PT  - Multicenter Study
PL  - England
TA  - Hematology
JT  - Hematology (Amsterdam, Netherlands)
JID - 9708388
RN  - V8G4MOF2V9 (Deferasirox)
SB  - IM
MH  - Adolescent
MH  - Adult
MH  - Aged
MH  - *Blood Transfusion
MH  - Deferasirox/*administration & dosage
MH  - Female
MH  - Hemosiderosis/blood/*drug therapy/etiology
MH  - Humans
MH  - Iron Overload/blood/*drug therapy/etiology
MH  - Male
MH  - Middle Aged
MH  - Retrospective Studies
MH  - Transfusion Reaction/blood/*drug therapy
OTO - NOTNLM
OT  - Deferasirox
OT  - iron chelation therapy
OT  - iron overload
OT  - safety
EDAT- 2018/12/19 06:00
MHDA- 2019/01/12 06:00
CRDT- 2018/12/19 06:00
PHST- 2018/12/19 06:00 [entrez]
PHST- 2018/12/19 06:00 [pubmed]
PHST- 2019/01/12 06:00 [medline]
AID - 10.1080/16078454.2018.1558758 [doi]
PST - ppublish
SO  - Hematology. 2019 Dec;24(1):238-246. doi: 10.1080/16078454.2018.1558758.