THALIDOMIDE FOR PATIENTS WITH THALASSEMIA INTERMEDIA: A RETROSPECTIVE MULTICENTER CLINICAL STUDY

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Kun Yang
Yi Wu
Yali Zhou
Tianhong Zhou
Li Wang
Zhili Geng
Xiaolin Yin
(*) Corresponding Author:

Abstract

Objective: This study focused on the efficacy and safety of thalidomide for patients with thalassemia intermedia (TI) in a multicenter trial.


Methods?Clinical and laboratory data of 62 patients subjected to thalidomide therapy in four centers were retrospectively analyzed. 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 Hb level of >2.0 g/dl or removal from blood transfusion and Minor Responder (MiR) achieving elevated hemoglobin (Hb) level of 1.0-2.0 g/dl or ?50% reduction in blood transfusion frequency.


Results?The overall response rate (ORR) of 62 patients with TI was 93.5% (58/62), with MaR and MiR rates accounting for 62.9% (39/62) and 30.6% (19/62) in short-term follow-up and 66.1% (41/62) and 27.4% (17/62) in long-term follow-up, respectively. The clinical response during long-term follow-up was maintained and the Hb level remained stable during the observation period. The response was still observed in patients with dose reduction despite a slight decrease in Hb level. However, Hb decreased rapidly to the baseline level after drug discontinuation. No effect of thalidomide on spleen size in nonsplenectomized patients was evident. Minimal side-effects were documented throughout, except peripheral neurotoxicity in one patient. Nevertheless, the mean serum ferritin (SF) level was significantly increased after treatment.


Conclusion: Thalidomide had significant therapeutic effects on patients with TI, and the response was sustained with acceptable short-term and long-term adverse reactions. While these preliminary results support the potential long-term efficacy and safety of thalidomide as a therapeutic agent for TI, several issues need to be addressed before its application in the clinic.


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Author Biographies

Yi Wu, The 923rd Hospital of the Joint Logistics Support Force of the Peoples Liberation Army

Department of Hematology

Yali Zhou, The 923rd Hospital of the Joint Logistics Support Force of the Peoples Liberation Army

Department of Hematology

Tianhong Zhou, The 923rd Hospital of the Joint Logistics Support Force of the Peoples Liberation Army,

Department of Hematology

Li Wang, The 923rd Hospital of the Joint Logistics Support Force of the Peoples Liberation Army,

The 923rd Hospital of the Joint Logistics Support Force of the Peoples Liberation Army,

Zhili Geng, The 923rd Hospital of the Joint Logistics Support Force of the Peoples Liberation Army

Department of Hematology

Xiaolin Yin, The 923rd Hospital of the Joint Logistics Support Force of the Peoples Liberation Army,

Department of Hematology