Disease characteristics and prognosis of myelodysplastic syndrome presenting with isolated thrombocytopenia

Julie Waisbren*, Shira Dinner, Jessica Altman, Olga Frankfurt, Irena Helenowski, Juehua Gao, Brandon J. McMahon, Brady L. Stein

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review

14 Scopus citations


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.

Original languageEnglish (US)
Pages (from-to)44-51
Number of pages8
JournalInternational journal of hematology
Issue number1
StatePublished - Jan 1 2017


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

ASJC Scopus subject areas

  • Hematology


Dive into the research topics of 'Disease characteristics and prognosis of myelodysplastic syndrome presenting with isolated thrombocytopenia'. Together they form a unique fingerprint.

Cite this