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Thalidomide for Patients with β-Thalassemia: A Multicenter Experience.

Abstract：

Objective:This study focused on the efficacy and safety of thalidomide for patients with β-thalassemia in a multicenter trial. Methods: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. Results: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 (P<0.001). Elevated Hb was mainly attributable to increased fetal hemoglobin (HbF) levels. Among patients with TDT, while an increase in the average Hb concentration was observed, there was a significant drop in yearly transfusions from 20.7±7.7 to 5.8±6.8 blood units per year (P<0.001). The response of patients in both categories was sustained even after an average follow up of 14.6±9.6 months (3-37 months). Minimal side-effects were documented throughout, except peripheral neurotoxicity in one patient. Logistic regression analysis identified the ratio of HbF at baseline (P=0.038, OR=1.111, 95% CI: 1.006-1.226) as an independent risk factor for the primary response to thalidomide. Conclusion: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.

journal_name

Mediterr J Hematol Infect Dis

journal_title

Mediterranean journal of hematology and infectious diseases

authors

Yang K,Wu Y,Zhou Y,Long B,Lu Q,Zhou T,Wang L,Geng Z,Yin X

doi

10.4084/MJHID.2020.021

subject

Has Abstract

pub_date

2020-05-01 00:00:00

pages

e2020021

issue

issn

2035-3006

pii

mjhid-12-1-e2020021

journal_volume

pub_type

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Thalidomide for Patients with β-Thalassemia: A Multicenter Experience.