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Blood. 2014 Jun 12;123(24):3803-10. doi: 10.1182/blood-2013-09-527903. Epub 2014 Feb 19.

Distinct clustering of symptomatic burden among myeloproliferative neoplasm patients: retrospective assessment in 1470 patients.

Author information

1
Division of Hospital Internal Medicine and.
2
Section of Biostatistics, Mayo Clinic, Scottsdale, AZ;
3
Clinical Investigation Center, Hospital Saint-Louis, Paris, France;
4
Myelodysplastic Syndrome and Myeloproliferative Neoplasia Centre, Institute of Hematology and Blood Diseases Hospital and State Key Laboratory of Experimental Hematology, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin, China;
5
Department of Hematology, Vrije Universiteit Medical Center, Amsterdam, The Netherlands;
6
Fundaleu, Buenos Aires, Argentina;
7
Department of Haematology, University Hospital La Paz, Madrid, Spain;
8
Department of Internal Medicine III, University Hospital of Ulm, Germany;
9
Department of Haematology, Guy's and St. Thomas National Health Service Foundation Trust, London, United Kingdom;
10
Unidadde Hematología, Hospital Británico, Montevideo, Uruguay;
11
Hematology Department, Hospital del Mar, Barcelona, Spain;
12
Hematology Department, Hospital Clínic, Institut d'Investigacions Biomèdiques August Pi i Sunyer, University of Barcelona, Spain;
13
Internal Medicine, Nephro-Urology Hospital Organization, Uddevalla, Sweden;
14
Unit of Hematology, Azienda Ospedaliera Papa Giovanni XXIII, Bergamo, Italy;
15
Hematology Division of Hematology, Ospedale di Circolo, Varese, Italy;
16
Department of Hematology, Fondazione IRCCS Policlinico San Matteo, University of Pavia, Pavia, Italy;
17
Department of Internal Medicine, Stockholm South Hospital, Stockholm, Sweden;
18
Department of Hematology, University Hospital, Uppsala, Sweden; and.
19
Department of Hematology/Oncology, Mayo Clinic, Scottsdale, AZ.

Abstract

Symptom burden in myeloproliferative neoplasms (MPNs) is heterogeneous even among patients within the same MPN diagnosis. Using cluster analysis from prospectively gathered symptom burden data in 1470 international patients with essential thrombocythemia (ET), polycythemia vera (PV), or myelofibrosis (MF), we assessed for the presence of clusters and relationship to disease features and prognosis. In MF (4 clusters identified), clusters significantly differed by Dynamic International Prognostic Scoring System (DIPSS) risk (P < .001), leukopenia (P = .009), thrombocytopenia (P < .001), and spleen size (P = .02). Although an association existed between clusters and DIPSS risk, high symptom burden was noted in some low and intermediate-1-risk MF patients. In PV (5 clusters identified), total symptom score increased across clusters (P < .001), but clusters did not significantly differ by PV risk or the risk assessment variable of age. Among ET patients (5 clusters identified), clusters differed by gender (P = .04), anemia (P = .01), and prior hemorrhage (P = .047). Total symptom score increased across clusters (P < .001), but clusters did not significantly differ by International Prognostic Score for ET risk including the risk assessment variables. Significant symptom heterogeneity exists within each MPN subtype, sometimes independent of disease features or prognosis.

PMID:
24553173
PMCID:
PMC4067502
DOI:
10.1182/blood-2013-09-527903
[Indexed for MEDLINE]
Free PMC Article

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