Myelodysplastic Syndromes (MDS) Presenting with Isolated Thrombocytopenia: Characteristics, Outcomes, and Clinical Presentation Differences from Immune Thrombocytopenic Purpura (ITP)

Autor: Vassiliki Pappa, Vasileios Papadopoulos, Flora N. Kontopidou, Eleftheria Hatzimichael, Alexandra Kourakli, Ioannis Adamopoulos, Panagiotis Zikos, Stamatis Karakatsanis, Athanasios Galanopoulos, Konstantina Papathanasiou, Argiris Symeonidis, Panagiotis T. Diamantopoulos, Sotirios G. Papageorgiou, Dimitris Tsokanas, Kotsianidis Ioannis, Menelaos Papoutselis, Helen A. Papadaki, Nora-Athina Viniou, Emily Stavroulaki, Anna Vardi, Maria Dimou, Konstantinos Liapis, Christina Misidou, Epameinondas Koumpis, Maria Ximeri, Charalampos Pontikoglou, George Vrachiolias, Theodoros P. Vassilakopoulos, Eleni Bouronikou, Nikolaos Charchalakis, Aikaterini Megalakaki, Panayiotis Panayiotidis
Rok vydání: 2021
Předmět:
Zdroj: Blood. 138:1535-1535
ISSN: 1528-0020
0006-4971
DOI: 10.1182/blood-2021-149630
Popis: Introduction: Less than 5% of patients with MDS present with thrombocytopenia as an isolated abnormality (MDS-IT). There have been few systematic studies on MDS-IT and data regarding its course and prognosis are conflicting. Previous studies have defined MDS-IT based on the IPSS thresholds (Hb ≥10 g/dL; ANC ≥1.8×10 9/L; PLT Methods: We identified patients who had PLT 13 g/dL (men) or >12 g/dL (women), and ANC ≥1.8 ×10 9/L, registered in the Hellenic National Registry of Myelodysplastic and Hypoplastic Syndromes which includes 2792 patients (analysis cut-off date; July 7, 2016). Patients were divided into 4 groups: group 1 had PLT 149-100 ×10 9/L; group 2, 99-50 ×10 9/L; group 3, Results: A total of 77 patients (45 men; 32 women) with MDS-IT were identified (2.9% of total MDS cohort). Of these, 28.6% were classified in group 1; 49.4% in group 2; 14.3% in group 3; and 7.8% in group 4. Median PLT count was 87 ×10 9/L (12-139 ×10 9/L), WBC count 4.6 ×10 9/L, and Hb 13.6 g/dL. Bone marrow (BM) blasts ranged from 0-9% (median, 2%). Median follow-up was 51.0 months (41.6-60.4), during which 15 (19.5%) patients died. AML developed in 9 patients (11.7%). Histologically, MDS with multilineage dysplasia (MLD) was seen in 77.6% whereas MDS with excess blasts (EB) and MDS with single lineage dysplasia (SLD) comprised 10.7% and 11.9% of cases, respectively. Most patients (73.5%) had lower-risk MDS on the IPSS-R (i.e. IPSS-R ≤3.5). Of the 59 patients with cytogenetic data, 83.1% had favorable, 13.5% intermediate, and 3.4% poor risk cytogenetics. Most (40) had a normal karyotype followed by isolated del(20q) (6). All patients with del(20q) showed a characteristic set of clinical features: age >60 years, blasts 0-3%, bilineage (erythroid/megakaryocytic) dysplasia, and increased reticulin fibrosis. There were no significant differences between any of the 4 PLT groups regarding age, sex, IPSS-R, cytogenetics, BM blasts, and histology. Median OS was 109 months (95% CI 103-115) and LFS 108 months (101-115). Our results showed no significant difference in OS (P=0.891) and LFS (P=0.871) between the 4 PLT groups. As compared with total MDS cohort, MDS-IT occurred at younger age (64.7 vs. 72.4 years, P In comparing MDS-IT with ITP, the median age at diagnosis was 66.0 years for MDS-IT and 49.0 years for ITP (P80 years. Its incidence reached a peak between the ages of 70-79 years, whereas ITP occurred at a more constant level over time (Figure 1B). Women predominated in ITP and men in MDS-IT (P=0.007). Overall, ITP was associated with more marked thrombocytopenia than MDS-IT (15.0 ×10 9/L vs. 87.0 ×10 9/L) (P Conclusions: In one of the largest reported series, we conclude that MDS-IT is associated with MDS-MLD, favorable cytogenetics, lower-risk IPSS-R, high survival rate, and a low risk of AML evolution. Our data suggest that the superior prognosis in MDS-IT than general MDS may have intrinsic genomic underpinnings as survival curves remained unchanged after correcting for age, sex, blasts and IPSS-R. Importantly, no significant differences in OS and LFS were noted between the 4 PLT subgroups, suggesting that the degree of thrombocytopenia does not correlate with mortality in MDS-IT. From the diagnostic standpoint, age 80 years and PLT Figure 1 Figure 1. Disclosures Viniou: Sandoz: Research Funding; Takeda: Research Funding; Novartis: Honoraria, Research Funding; Sanofi: Research Funding; Janssen: Honoraria, Research Funding; Pfizer: Research Funding; Abbvie: Research Funding; Bristol-Myers Squibb: Honoraria, Research Funding; Roche: Research Funding; Astellas: Research Funding; Celgene: Research Funding. Vassilakopoulos: Dr. Reddy's: Research Funding; Amgen: Honoraria, Research Funding; GlaxoSmithKline: Honoraria, Other: Travel; AbbVie: Consultancy, Honoraria; Integris: Honoraria; Pfizer: Research Funding; Roche: Consultancy, Honoraria, Other: Travel; Takeda: Consultancy, Honoraria, Other: Travel, Research Funding; Genesis Pharma: Consultancy, Honoraria, Other: Travel; Merck: Honoraria, Research Funding; Novartis: Consultancy, Honoraria; Karyopharm: Research Funding; AstraZeneca: Honoraria. Hatzimichael: Roche: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees; Amgen: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees; MSD: Consultancy, Honoraria; Gilead: Honoraria; Janssen Cilag: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees; Genesis: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees; Abbvie: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees; Takeda: Honoraria; Pharmathen- Innovis: Honoraria; GSK: Honoraria; Bristol Myersr Squibb: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees; Novartis: Consultancy, Membership on an entity's Board of Directors or advisory committees; Celgene: Consultancy, Membership on an entity's Board of Directors or advisory committees; Pfizer: Consultancy, Membership on an entity's Board of Directors or advisory committees. Symeonidis: Amgen: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding; Sanofi/Genzyme: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding; Demo: Research Funding; MSD: Consultancy, Research Funding; Janssen: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding; WinMedica: Research Funding; Astellas: Consultancy, Research Funding; Gilead: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding; Sanofi: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees; BMS: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding; Roche: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding; Takeda: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding; GSK: Research Funding; Novartis: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding; AbbVie: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding; GenesisPharma: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding; Pfizer: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding.
Databáze: OpenAIRE
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