title: Distinct clustering of symptomatic burden among myeloproliferative neoplasm patients: retrospective assessment in 1470 patients.
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.
authors.affiliation: Division of Hospital Internal Medicine and.
authors.forename: Holly L
authors.initials: HL
authors.lastname: Geyer
authors.affiliation: Division of Hospital Internal Medicine and.
authors.forename: Robyn M
authors.initials: RM
authors.lastname: Scherber
authors.affiliation: Section of Biostatistics, Mayo Clinic, Scottsdale, AZ;
authors.forename: Amylou C
authors.initials: AC
authors.lastname: Dueck
authors.affiliation: Clinical Investigation Center, Hospital Saint-Louis, Paris, France;
authors.forename: Jean-Jacques
authors.initials: JJ
authors.lastname: Kiladjian
authors.affiliation: 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;
authors.forename: Zhijian
authors.initials: Z
authors.lastname: Xiao
authors.affiliation: Department of Hematology, Vrije Universiteit Medical Center, Amsterdam, The Netherlands;
authors.forename: Stefanie
authors.initials: S
authors.lastname: Slot
authors.affiliation: Department of Hematology, Vrije Universiteit Medical Center, Amsterdam, The Netherlands;
authors.forename: Sonja
authors.initials: S
authors.lastname: Zweegman
authors.affiliation: Fundaleu, Buenos Aires, Argentina;
authors.forename: Federico
authors.initials: F
authors.lastname: Sackmann
authors.affiliation: Department of Haematology, University Hospital La Paz, Madrid, Spain;
authors.forename: Ana Kerguelen
authors.initials: AK
authors.lastname: Fuentes
authors.affiliation: Department of Haematology, University Hospital La Paz, Madrid, Spain;
authors.forename: Dolores
authors.initials: D
authors.lastname: Hernández-Maraver
authors.affiliation: Department of Internal Medicine III, University Hospital of Ulm, Germany;
authors.forename: Konstanze
authors.initials: K
authors.lastname: Döhner
authors.affiliation: Department of Haematology, Guy's and St. Thomas National Health Service Foundation Trust, London, United Kingdom;
authors.forename: Claire N
authors.initials: CN
authors.lastname: Harrison
authors.affiliation: Department of Haematology, Guy's and St. Thomas National Health Service Foundation Trust, London, United Kingdom;
authors.forename: Deepti
authors.initials: D
authors.lastname: Radia
authors.affiliation: Unidadde Hematología, Hospital Británico, Montevideo, Uruguay;
authors.forename: Pablo
authors.initials: P
authors.lastname: Muxi
authors.affiliation: Hematology Department, Hospital del Mar, Barcelona, Spain;
authors.forename: Carlos
authors.initials: C
authors.lastname: Besses
authors.affiliation: Hematology Department, Hospital Clínic, Institut d'Investigacions Biomèdiques August Pi i Sunyer, University of Barcelona, Spain;
authors.forename: Francisco
authors.initials: F
authors.lastname: Cervantes
authors.affiliation: Internal Medicine, Nephro-Urology Hospital Organization, Uddevalla, Sweden;
authors.forename: Peter L
authors.initials: PL
authors.lastname: Johansson
authors.affiliation: Internal Medicine, Nephro-Urology Hospital Organization, Uddevalla, Sweden;
authors.forename: Bjorn
authors.initials: B
authors.lastname: Andreasson
authors.affiliation: Unit of Hematology, Azienda Ospedaliera Papa Giovanni XXIII, Bergamo, Italy;
authors.forename: Alessandro
authors.initials: A
authors.lastname: Rambaldi
authors.affiliation: Unit of Hematology, Azienda Ospedaliera Papa Giovanni XXIII, Bergamo, Italy;
authors.forename: Tiziano
authors.initials: T
authors.lastname: Barbui
authors.affiliation: Hematology Division of Hematology, Ospedale di Circolo, Varese, Italy;
authors.forename: Alessandro M
authors.initials: AM
authors.lastname: Vannucchi
authors.affiliation: Department of Hematology, Fondazione IRCCS Policlinico San Matteo, University of Pavia, Pavia, Italy;
authors.forename: Francesco
authors.initials: F
authors.lastname: Passamonti
authors.affiliation: Department of Internal Medicine, Stockholm South Hospital, Stockholm, Sweden;
authors.forename: Jan
authors.initials: J
authors.lastname: Samuelsson
authors.affiliation: Department of Hematology, University Hospital, Uppsala, Sweden; and.
authors.forename: Gunnar
authors.initials: G
authors.lastname: Birgegard
authors.affiliation: Department of Hematology/Oncology, Mayo Clinic, Scottsdale, AZ.
authors.forename: Ruben A
authors.initials: RA
authors.lastname: Mesa
mesh_terms: D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D019046:Bone Marrow Neoplasms / Q000150:complications / Q000175:diagnosis / Q000453:epidemiology*; D016000:Cluster Analysis; D005260:Female; D005843:Geography; D006801:Humans; D008297:Male; D008875:Middle Aged; D009196:Myeloproliferative Disorders / Q000150:complications / Q000175:diagnosis / Q000453:epidemiology*; D011087:Polycythemia Vera / Q000175:diagnosis / Q000453:epidemiology; D055728:Primary Myelofibrosis / Q000175:diagnosis / Q000453:epidemiology; D012189:Retrospective Studies; D012720:Severity of Illness Index; D013920:Thrombocythemia, Essential / Q000175:diagnosis / Q000453:epidemiology