TY - JOUR
T1 - Disease characteristics and prognosis of myelodysplastic syndrome presenting with isolated thrombocytopenia
AU - Waisbren, Julie
AU - Dinner, Shira
AU - Altman, Jessica
AU - Frankfurt, Olga
AU - Helenowski, Irena
AU - Gao, Juehua
AU - McMahon, Brandon J.
AU - Stein, Brady L.
N1 - Publisher Copyright:
© 2016, The Japanese Society of Hematology.
PY - 2017/1/1
Y1 - 2017/1/1
N2 - 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.
AB - 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.
KW - Acute Myeloid Leukemia
KW - Myelodysplastic syndrome
KW - Myeloid leukemia and dysplasia
KW - Thrombocytopenia
KW - Thrombopoietin analogs
UR - http://www.scopus.com/inward/record.url?scp=84983461389&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=84983461389&partnerID=8YFLogxK
U2 - 10.1007/s12185-016-2081-4
DO - 10.1007/s12185-016-2081-4
M3 - Article
C2 - 27558575
AN - SCOPUS:84983461389
SN - 0925-5710
VL - 105
SP - 44
EP - 51
JO - International journal of hematology
JF - International journal of hematology
IS - 1
ER -
