title: Disease characteristics and prognosis of myelodysplastic syndrome presenting with isolated thrombocytopenia.
abstract: 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.
authors: Department of Internal Medicine, Northwestern University Feinberg School of Medicine, 251 E Huron St, Chicago, IL, 60611, USA. Julie.waisbren@northwestern.edu.
authors: Julie
authors: J
authors: Waisbren
authors: Division of Hematology/Oncology, Department of Medicine, Northwestern University Feinberg School of Medicine, 645 N. Michigan Ave, Suit 1020, Chicago, IL, 60611, USA.
authors: Shira
authors: S
authors: Dinner
authors: Division of Hematology/Oncology, Robert H. Lurie Medical Research Center, Northwestern University Feinberg School of Medicine, 303 E Superior Room 5-111, Chicago, IL, 60611, USA.
authors: Jessica
authors: J
authors: Altman
authors: Division of Hematology/Oncology, Northwestern University Feniberg School of Medicine, NMH/Arkes Family Pavilion Suite 850, 676 N.Saint Clair, Chicago, IL, 60611, USA.
authors: Olga
authors: O
authors: Frankfurt
authors: Department of Preventative Medicine, Northwestern University Feinberg School of Medicine, 680 N. Lake Shore Drive, Suit 1400, Chicago, IL, 60611, USA.
authors: Irena
authors: I
authors: Helenowski
authors: Department of Pathology, Northwestern University Feinberg School of Medicine, NMH/Feinberg Room 7-209A, 251 E Huron St, Chicago, IL, 60611, USA.
authors: Juehua
authors: J
authors: Gao
authors: Division of Hematology/Oncology, Department of Medicine, Northwestern University Feinberg School of Medicine, 645 N. Michigan Ave, Suit 1020, Chicago, IL, 60611, USA.
authors: Brandon J
authors: BJ
authors: McMahon
authors: Division of Hematology/Oncology, Department of Medicine, Northwestern University Feinberg School of Medicine, 645 N. Michigan Ave, Suit 1020, Chicago, IL, 60611, USA.
authors: Brady L
authors: BL
authors: Stein
mesh_terms: D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D000964:Antimetabolites, Antineoplastic / Q000627:therapeutic use; D001374:Azacitidine / Q000031:analogs & derivatives / Q000627:therapeutic use; D001565:Benzoates / Q000627:therapeutic use; D000077209:Decitabine; D005260:Female; D006801:Humans; D006834:Hydrazines / Q000627:therapeutic use; D015470:Leukemia, Myeloid, Acute / Q000209:etiology*; D008297:Male; D008875:Middle Aged; D009190:Myelodysplastic Syndromes / Q000150:complications* / Q000175:diagnosis / Q000188:drug therapy; D011379:Prognosis; D011720:Pyrazoles / Q000627:therapeutic use; D011961:Receptors, Fc / Q000627:therapeutic use; D053628:Receptors, Thrombopoietin / Q000819:agonists; D011993:Recombinant Fusion Proteins / Q000627:therapeutic use; D012189:Retrospective Studies; D012306:Risk; D013921:Thrombocytopenia / Q000150:complications* / Q000175:diagnosis / Q000188:drug therapy; D013926:Thrombopoietin / Q000627:therapeutic use
chemical_list: D000964:Antimetabolites, Antineoplastic; D001565:Benzoates; D006834:Hydrazines; D011720:Pyrazoles; D011961:Receptors, Fc; D053628:Receptors, Thrombopoietin; D011993:Recombinant Fusion Proteins; D000077209:Decitabine; D013926:Thrombopoietin; C488777:romiplostim; D001374:Azacitidine; C520809:eltrombopag
keywords: Acute Myeloid Leukemia; Myelodysplastic syndrome; Myeloid leukemia and dysplasia; Thrombocytopenia; Thrombopoietin analogs