The incidence and impact of thrombocytopenia in myelodysplastic syndromes

Abstract

Thrombocytopenia and platelet dysfunction contribute to hemorrhagic complications in the myelodysplastic syndromes (MDS). Reliable data regarding the frequency and consequences of thrombocytopenia in MDS are lacking. An extensive literature review indicated that the prevalence of thrombocytopenia (platelets <100 3 10 9 /L) in MDS ranged from 40% to 65%; the median frequency of thrombocytopenia prior to any MDS therapy was 65% (range, 23-93% high-risk disease. Treatment-related thrombocytopenia was observed in studies that involved azacitidine, tipifarnib, decitabine, lenalidomide, sirolimus, and combination chemotherapy with idarubicin, cytarabine, and topotecan. The reported incidence of hemorrhagic complications in the literature ranged from 3% to 53%, and the frequency of hemorrhagic deaths ranged from 14% to 24%.At MDACC, 460 patients had a coded cause of death: hemorrhage as a contributory cause of death, 20%; hemorrhage as the only cause of death, 10%. Thrombocytopenia was common in MDS, and there was an increased prevalence in higher risk IPSS categories. Many approved and investigational MDS therapies caused or exacerbated preexisting thrombocytopenia. The incidence of severe bleeding in MDS was greater than reported in current guidelines. Cancer

Subcutaneous or intravenous administration of romiplostim in thrombocytopenic patients with lower risk myelodysplastic syndromes

“…1 Unfortunately, most agents that have activity in patients with lower risk MDS target hematologic improvements along erythroid lines, leaving these patients without viable options other than platelet transfusions for treating profound or symptomatic thrombocytopenia, with the accompanying risk of alloimmunization and ultimate lack of response over time. Thrombopoietic growth factors, thus, represent promising therapies for this subpopulation of patients.…”

Revisiting Use of Growth Factors in Myelodysplastic Syndromes

“…Platelet count has direct relation with survival, and severe thrombocytopenia is associated with shorter transformation time to AML. Platelet transfusion has the risk of infection transmission, platelet alloimmunization, transfusion-related acute lung injury, and febrile nonhemolytic reactions (Kantarjian et al, 2007;Sekerers et al, 2011). Thrombopietin has a large molecule with 23% homology with erythropoietin, and its half-life is about 20 to 40 hours.…”

Disparity in perceptions of disease characteristics, treatment effectiveness, and factors influencing treatment adherence between physicians and patients with myelodysplastic syndromes

“…4,5 Most patients with MDS-even those with lower risk disease features at the time of diagnosis-will die of infectious or hemorrhagic complications related to their disease. 6,7 The only potentially curative therapy, allogeneic hematopoietic stem cell transplantation (HSCT), is currently attempted in less than 5% of patients because of the advanced age and comorbid conditions present in most patients and perceptions about the danger of HSCT. 8,9 There are currently 3 drugs approved for MDS by the US Food and Drug Administration (FDA) that offer symptomatic benefit and slow disease progression in some patients: azacitidine (AZA), decitabine (DAC), and lenalidomide (LEN).…”