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

Abstract

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.

Magnetic Resonance Imaging Quantification of the Liver Iron Burden and Volume Changes Following Treatment With Thalidomide in Patients With Transfusion-Dependent ß-Thalassemia

“…Consistent with previous studies, our study illustrated that hemoglobin and erythrocyte count significantly increased during 12 months of thalidomide therapy, and the median annual red blood cell transfusion rate significantly decreased [from baseline at 36.0 U (range 2.0-96.0 U) to the 12th month at 2.0 U (range 0-23.0 U)]. A prospective cohort study (Chen et al, 2020) reported that the platelet count progressively increased during 12 months of thalidomide therapy, and the results of our study showed that although the platelet count did not significantly change, it tended to increase during the 12-months follow up.…”

“…In ß-thalassemia major, to compensate for anemia, an increasing rate of extramedullary hemopoiesis would occur, leading to increased production of abnormal red blood cells and their clearance; hence, hypersplenism and increases in spleen size are also observed (Kolnagou et al, 2013). Chen et al (2020) reported that all 31 patients (hepatic cirrhosis, n = 19 and thalassemia, n = 12) who received thalidomide treatment showed progressive decrease in spleen length during a 12-months follow up. In addition, increase in liver size could also be observed in patients with ß-thalassemia major; Fischer et al (1999) revealed increase in iron concentration of 0.6-11.0 mg/g in the liver and in the size of the liver of approximately 18% per 1 mg/g liver in patients with ßthalassemia major.…”

“…Li et al (2018) reported that thalidomide had a significant effect in patients with thalassemia, whether transfusion-independent or transfusion-dependent. Furthermore, a recent study (Chen et al, 2017) of nine patients with thalassemia showed that treatment with thalidomide significantly improved the hemoglobin level and reduced spleen size, and another study (Chen et al, 2020) suggested that thalidomide could potentially be useful for the management of thrombocytopenia in patients with hypersplenism. A multicenter study (Yang et al, 2020) reported that thalidomide had significant therapeutic effects in patients with ß-thalassemia with a sustained response.…”

Iron and platelets: A subtle, under‐recognized relationship

“…Chronic acquired iron overload is mainly the result of multiple transfusions and/or ineffective erythropoiesis as occurs in ß-thalassemias 98,99 and myelodysplastic syndrome (MDS) 100 or following therapeutic ablation in bone marrow transplantation. 2 Due to platelet changes, whether quantitative [101][102][103] and/or qualitative, 99,[104][105][106] which can develop as part of the underlying hematological diseases (or as a result of their medications), it is difficult to attribute platelet modifications to iron overload alone. One way to approach this issue is to evaluate the impact of iron chelation treatment on thrombocytes.…”

Clinical Performance Study of a New Fully Automated Red Blood Cell Permeability Fragility Analyzer

“…Thalassemia is a group of genetic diseases caused by the reduction of hemoglobin α or β -chain synthesis [ 1 ]. It is one of the most common single-gene genetic diseases in humans.…”