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Thalidomide for Patients with β-Thalassemia: A Multicenter Experience

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 transfus...

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Autores principales: Yang, Kun, Wu, Yi, Zhou, Yali, Long, Binbin, Lu, Qian, Zhou, Tianhong, Wang, Li, Geng, Zhili, Yin, Xiaolin
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Università Cattolica del Sacro Cuore 2020
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7202343/
https://www.ncbi.nlm.nih.gov/pubmed/32395210
http://dx.doi.org/10.4084/MJHID.2020.021
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author Yang, Kun
Wu, Yi
Zhou, Yali
Long, Binbin
Lu, Qian
Zhou, Tianhong
Wang, Li
Geng, Zhili
Yin, Xiaolin
author_facet Yang, Kun
Wu, Yi
Zhou, Yali
Long, Binbin
Lu, Qian
Zhou, Tianhong
Wang, Li
Geng, Zhili
Yin, Xiaolin
author_sort Yang, Kun
collection PubMed
description 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.
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spelling pubmed-72023432020-05-11 Thalidomide for Patients with β-Thalassemia: A Multicenter Experience Yang, Kun Wu, Yi Zhou, Yali Long, Binbin Lu, Qian Zhou, Tianhong Wang, Li Geng, Zhili Yin, Xiaolin Mediterr J Hematol Infect Dis Original Article 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. Università Cattolica del Sacro Cuore 2020-05-01 /pmc/articles/PMC7202343/ /pubmed/32395210 http://dx.doi.org/10.4084/MJHID.2020.021 Text en This is an Open Access article distributed under the terms of the Creative Commons Attribution License (https://creativecommons.org/licenses/by-nc/4.0), which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.
spellingShingle Original Article
Yang, Kun
Wu, Yi
Zhou, Yali
Long, Binbin
Lu, Qian
Zhou, Tianhong
Wang, Li
Geng, Zhili
Yin, Xiaolin
Thalidomide for Patients with β-Thalassemia: A Multicenter Experience
title Thalidomide for Patients with β-Thalassemia: A Multicenter Experience
title_full Thalidomide for Patients with β-Thalassemia: A Multicenter Experience
title_fullStr Thalidomide for Patients with β-Thalassemia: A Multicenter Experience
title_full_unstemmed Thalidomide for Patients with β-Thalassemia: A Multicenter Experience
title_short Thalidomide for Patients with β-Thalassemia: A Multicenter Experience
title_sort thalidomide for patients with β-thalassemia: a multicenter experience
topic Original Article
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7202343/
https://www.ncbi.nlm.nih.gov/pubmed/32395210
http://dx.doi.org/10.4084/MJHID.2020.021
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