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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...
Autores principales: | , , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Università Cattolica del Sacro Cuore
2020
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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. |
format | Online Article Text |
id | pubmed-7202343 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2020 |
publisher | Università Cattolica del Sacro Cuore |
record_format | MEDLINE/PubMed |
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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