Thalidomide induces haematologic responses in patients with β‐thalassaemia

Chen, Jiangming; Zhu, Wen; Cai, Na; Bu, Shengdi; Li, Jinyan; Huang, Lan

doi:10.1111/ejh.12955

Cited by 21 publications

(28 citation statements)

References 9 publications

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“…The reduction in splenomegaly and hypersplenism would also explain the significant elevation in leukocyte count, apparent improvements in hemoglobin level and absolute neutrophil count (non-significant), and low requirement for red blood cell transfusion that were observed in patients treated with thalidomide. Our findings regarding the possible beneficial effects of thalidomide on hemoglobin level and spleen length are in good agreement with those reported in our previous study of nine patients with b-thalassemia who were treated with thalidomide for 6 months or more (Chen et al, 2017). Two case reports have also described an increase in hemoglobin level in response to thalidomide (Aguilar-Lopez et al, 2008;Masera et al, 2010), consistent with the trends in our observations.…”

Section: Discussionsupporting

confidence: 93%

“…However, adverse reactions to thalidomide are rare in other patients. Indeed, we observed no adverse reactions to thalidomide in our previous study of patients with thalassemia ( Chen et al., 2017 ). Thus, we believe that thalidomide would be well tolerated by non-pregnant patients with hypersplenism and thrombocytopenia secondary to cirrhosis or thalassemia.…”

Section: Discussionsupporting

confidence: 48%

“…Two published case reports have described individual patients with thalassemia who responded well to thalidomide ( Aguilar-Lopez et al., 2008 ; Masera et al., 2010 ). Furthermore, our recent study of nine patients with thalassemia demonstrated that treatment with thalidomide significantly improved the hemoglobin level and reduced spleen size ( Chen et al., 2017 ). Interestingly, we also observed that the platelet levels in two of the nine patients were notably increased after therapy with thalidomide ( Chen et al., 2017 ), suggesting that thalidomide could potentially have utility in the management of thrombocytopenia in patients with hypersplenism.…”

Section: Introductionmentioning

confidence: 99%

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Thalidomide for the Treatment of Thrombocytopenia and Hypersplenism in Patients With Cirrhosis or Thalassemia

Chen¹,

Cai²,

Lai

et al. 2020

Front. Pharmacol.

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Hypersplenism and thrombocytopenia are common complications of liver cirrhosis or thalassemia, but current treatment strategies are limited. This study aimed to evaluate the efficacy and safety of thalidomide in the treatment of hypersplenism and thrombocytopenia in patients with liver cirrhosis or thalassemia. A total of 31 patients with hepatic cirrhosis (n=19) or thalassemia (n=12) diagnosed with hypersplenism and thrombocytopenia (platelet count [PLT] <100×10 9 /L) were included in this prospective cohort study between January 2015 and May 2017. Patients were treated with thalidomide (150–200 mg/d) plus conventional therapy. Spleen length, PLT, leukocyte count (WBC), absolute neutrophil count (ANC), and hemoglobin level (Hb) were measured at baseline, 3, 6, and 12 months. Any adverse events were noted. All of the 31 patients were showed a progressive increase PLT during the 12-month follow-up, and similar results were obtained when subgroup analyses were performed based on the primary disease (cirrhosis or thalassemia). WBC, ANC, and Hb also increased progressively during the 12-month follow-up. Spleen length decreased progressively during the follow-up. No serious adverse events occurred. Thalidomide is a potential treatment for thrombocytopenia caused by hypersplenism in patients with cirrhosis or thalassemia.

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Section: Discussionsupporting

confidence: 93%

Section: Discussionsupporting

confidence: 48%

Section: Introductionmentioning

confidence: 99%

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Thalidomide for the Treatment of Thrombocytopenia and Hypersplenism in Patients With Cirrhosis or Thalassemia

Chen¹,

Cai²,

Lai

et al. 2020

Front. Pharmacol.

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“…12 Moreover, thalidomide is an HbF inducer that promotes γ-globin gene expression. 13,14 A few case reports and retrospective analyses have documented significant effects of thalidomide on NTDT or TDT, [15][16][17][18][19][20] that our group subsequently confirmed in a clinical trial. 21 However, the reliability of these studies was softened by the few patients studied and short-term follow-up.…”

mentioning

confidence: 55%

Thalidomide for Patients With Thalassemia Intermedia: A Retrospective Multicenter Clinical Study

Yang¹,

Wu²,

Zhou³

et al. 2020

Mediterr J Hematol Infect Dis

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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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“…Together with its derivate pomalidomide, it also exhibits a secondary, less characterised effect in increasing γ-globin expression, suggesting a possible role in the control of IE [97]. Besides isolated reports of safety and efficacy of this approach [98,99], the few studies conducted in the last years on patients affected by TDT and NTDT reported promising results in terms of Hb rise and transfusion independence [100,101]. However, these studies involved a very limited number of patients, and high variability was observed in baseline characteristics of the study populations, including extreme conditions such as baseline Hb ≤ 4.0 g/dL in 4 out of 25 patients.…”

Section: Thalidomidementioning

confidence: 99%

Ineffective Erythropoiesis in β-Thalassaemia: Key Steps and Therapeutic Options by Drugs

Longo

Piolatto

Ferrero

et al. 2021

IJMS

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β-thalassaemia is a rare genetic condition caused by mutations in the β-globin gene that result in severe iron-loading anaemia, maintained by a detrimental state of ineffective erythropoiesis (IE). The role of multiple mechanisms involved in the pathophysiology of the disease has been recently unravelled. The unbalanced production of α-globin is a major source of oxidative stress and membrane damage in red blood cells (RBC). In addition, IE is tightly linked to iron metabolism dysregulation, and the relevance of new players of this pathway, i.e., hepcidin, erythroferrone, matriptase-2, among others, has emerged. Advances have been made in understanding the balance between proliferation and maturation of erythroid precursors and the role of specific factors in this process, such as members of the TGF-β superfamily, and their downstream effectors, or the transcription factor GATA1. The increasing understanding of IE allowed for the development of a broad set of potential therapeutic options beyond the current standard of care. Many candidates of disease-modifying drugs are currently under clinical investigation, targeting the regulation of iron metabolism, the production of foetal haemoglobin, the maturation process, or the energetic balance and membrane stability of RBC. Overall, they provide tools and evidence for multiple and synergistic approaches that are effectively moving clinical research in β-thalassaemia from bench to bedside.

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Thalidomide induces haematologic responses in patients with β‐thalassaemia

Cited by 21 publications

References 9 publications

Thalidomide for the Treatment of Thrombocytopenia and Hypersplenism in Patients With Cirrhosis or Thalassemia

Thalidomide for the Treatment of Thrombocytopenia and Hypersplenism in Patients With Cirrhosis or Thalassemia

Thalidomide for Patients With Thalassemia Intermedia: A Retrospective Multicenter Clinical Study

Ineffective Erythropoiesis in β-Thalassaemia: Key Steps and Therapeutic Options by Drugs

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