Thalidomide for Patients with β-Thalassemia: A Multicenter Experience.
Efficacy
Fetal hemoglobin
Safety
Thalidomide
β-thalassemia
Journal
Mediterranean journal of hematology and infectious diseases
ISSN: 2035-3006
Titre abrégé: Mediterr J Hematol Infect Dis
Pays: Italy
ID NLM: 101530512
Informations de publication
Date de publication:
2020
2020
Historique:
received:
28
11
2019
accepted:
03
03
2020
entrez:
13
5
2020
pubmed:
13
5
2020
medline:
13
5
2020
Statut:
epublish
Résumé
This study focused on the efficacy and safety of thalidomide for patients with β-thalassemia in a multicenter trial. Patients with non-transfusion-dependent thalassemia (NTDT) or transfusion-dependent thalassemia (TDT), who were unable to pursue conventional therapy with transfusion and chelation, were recruited over 3 years in three centers in southern China. We evaluated the efficacy and safety of thalidomide in the short-term (three months) and long-term follow-up (12 and 24 months). Response to thalidomide was defined as follows: Main Responder (MaR) showing an increase in hemoglobin (Hb) level of >2.0 g/dl or free from blood transfusion and Minor Responder (MiR) achieving elevated Hb level of 1.0-2.0 g/dl or ≥50% reduction in blood transfusion frequency. The overall response rate (ORR) was 93.5%, with MaR and MiR rates accounting for 62.9% and 30.6% in short-term follow-up. For patients with NTDT, the Hb level increased from a baseline mean of 6.8±1.1 g/dl to 9.7±1.9 g/dl ( Thalidomide had significant therapeutic effects on patients with β-thalassemia with a sustained response. Peripheral neuropathy is one of the most feared complications. While these preliminary results support the potential long-term efficacy of thalidomide as a therapeutic agent for β-thalassemia, several issues need to be addressed before its application in the clinic.
Identifiants
pubmed: 32395210
doi: 10.4084/MJHID.2020.021
pii: mjhid-12-1-e2020021
pmc: PMC7202343
doi:
Types de publication
Journal Article
Langues
eng
Pagination
e2020021Déclaration de conflit d'intérêts
Competing interests: The authors declare no conflict of Interest.
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