Disease characteristics and prognosis of myelodysplastic syndrome presenting with isolated thrombocytopenia

作者：Wai**ren Julie^*; Dinner Shira; Altman Jessica; Frankfurt Olga; Helenowski Irena; Gao Juehua; McMahon Brandon J; Stein Brady L

来源：International Journal of Hematology, 2017, 105(1): 44-51.

DOI：10.1007/s12185-016-2081-4

• Thrombocytopenia
• Myelodysplastic syndrome
• Acute Myeloid Leukemia
• Thrombopoietin analogs
• Myeloid leukemia and dysplasia

Patients with myelodysplastic syndrome (MDS) who present with isolated thrombocytopenia (TCP) constitute a poorly described subgroup. The aim of the present study was to retrospectively evaluate disease characteristics and prognosis in patients with MDS and isolated TCP at a tertiary care center. Fifty patients (12 %) had isolated thrombocytopenia as the first presentation of MDS. Patients had varying MDS sub-classifications and cytogenetic profiles. The most common IPSS-R risk score was low (n = 24), although half of the patients had either IPSS-R intermediate (n = 18), high or very high risk disease (n = 7). Leukemic transformation occurred in 10 patients and there were 14 deaths (28 %) amongst all IPSS-R risk scores. Therapeutic agents used in this patient subgroup included hypomethylating agents and thrombopoietin receptor agonists. Overall, MDS with isolated TCP did not appear to have an inherently indolent course, as has been suggested previously. Future studies are needed to improve risk stratification, identify relevant contributors to disease pathogenesis, and better define treatment modalities.

• 出版日期2017-1
• 单位西北大学