Outcome of iron reduction therapy in ex-thalassemics.
Adolescent
Allografts
/ drug effects
Benzoates
/ administration & dosage
Child
Child, Preschool
Deferasirox
/ administration & dosage
Female
Ferritins
/ analysis
Hematopoietic Stem Cell Transplantation
/ adverse effects
Humans
Infant
Infant, Newborn
Iron
/ metabolism
Iron Chelating Agents
/ administration & dosage
Iron Overload
/ etiology
Male
Phlebotomy
/ methods
Triazoles
/ administration & dosage
beta-Thalassemia
/ drug therapy
Journal
PloS one
ISSN: 1932-6203
Titre abrégé: PLoS One
Pays: United States
ID NLM: 101285081
Informations de publication
Date de publication:
2021
2021
Historique:
received:
05
05
2020
accepted:
24
08
2020
entrez:
22
1
2021
pubmed:
23
1
2021
medline:
1
5
2021
Statut:
epublish
Résumé
There is limited data on iron reduction therapy (IRT) after successful allogeneic haematopoietic stem cell transplantation (aHSCT) for patients with thalassemia major (TM). We present the long term outcome of IRT in 149 patients with TM who underwent aHSCT during January, 2001-December, 2012. The median age was 7 years (range:1-18) and 92 (61.7%) belonged to Pesaro class 3 with a median ferritin at aHSCT of 2480ng/ml (range:866-8921). IRT was reinitiated post-aHSCT at a median of 14 months (range:5-53) post aHSCT with phlebotomy alone in 10 (6.7%) patients or iron chelation alone in 60 (40.3%) patients while 79 (53%) were treated with the combination. Reduction in serum ferritin/month [absolute quantity (ng/ml/month) was as follows: 87 (range:33-195), 130 (range:17-1012) and 147 (range:27.7-1427) in the phlebotomy, chelation and combination therapy groups, respectively (p = 0.038). With a median follow up of 80 months (range:37-182), target ferritin level of <300ng/ml was achieved in 59(40%) while a level <500ng/ml was achieved in 88 patients (59%) in a median duration of 41 months of IRT (range: 3-136). Patients in class III risk category and higher starting serum ferritin levels (>2500ng/ml) were associated with delayed responses to IRT. Our data shows that IRT may be needed for very long periods in ex-thalassaemics to achieve target ferritin levels and should therefore be carefully planned and initiated as soon as possible after aHSCT. A combination of phlebotomy and iron chelators is more effective in reducing iron overload.
Identifiants
pubmed: 33481797
doi: 10.1371/journal.pone.0238793
pii: PONE-D-20-13155
pmc: PMC7822270
doi:
Substances chimiques
Benzoates
0
Iron Chelating Agents
0
Triazoles
0
Ferritins
9007-73-2
Iron
E1UOL152H7
Deferasirox
V8G4MOF2V9
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Pagination
e0238793Déclaration de conflit d'intérêts
The authors have declared that no competing interests exist.
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