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Health care setting and severity, symptom burden, and complications in patients with Philadelphia-negative myeloproliferative neoplasms (MPN): a comparison between university hospitals, community hospitals, and office-based physicians

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Abstract

Philadelphia-negative myeloproliferative neoplasms (MPN) comprise a heterogeneous group of chronic hematological malignancies with significant variations in clinical characteristics. Due to the long survival and the feasibility of oral or subcutaneous therapy, these patients are frequently treated outside of larger academic centers. This analysis was performed to elucidate differences in MPN patients in three different health care settings: university hospitals (UH), community hospitals (CH), and office-based physicians (OBP). The MPN registry of the Study Alliance Leukemia is a non-interventional prospective study including adult patients with an MPN according to WHO criteria (2008). For statistical analysis, descriptive methods and tests for significant differences were used. Besides a different distribution of MPN subtypes between the settings, patients contributed by UH showed an impaired medical condition, a higher comorbidity burden, and more vascular complications. In the risk group analyses, the majority of polycythemia vera (PV) and essential thrombocythemia (ET) patients from UH were classified into the high-risk category due to previous vascular events, while for PV and ET patients in the CH and OBP settings, age was the major parameter for a high-risk categorization. Regarding MPN-directed therapy, PV patients from the UH setting were more likely to receive ruxolitinib within the framework of a clinical trial. In summary, the characteristics and management of patients differed significantly between the three health care settings with a higher burden of vascular events and comorbidities in patients contributed by UH. These differences need to be taken into account for further analyses and design of clinical trials.

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Acknowledgments

We thank all the staff of our participating centers for the registration of MPN patients and documentation of clinical data. Furthermore, we thank all the patients for their support and consent to participate in our project.

Authors’ contributions

AK and SK designed the project, collected data, performed statistical analyses, and wrote the manuscript. AK, SI, THB, and SK designed the clinical registry. SI, NG, WH, MK, DW, CM, MH, EG, JRG, UP, TG, SP, EJ, HS, GE, WEB, and THB collected data and provided scientific input and/or wrote parts of the manuscript. All authors approved the final version of the manuscript.

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Correspondence to Steffen Koschmieder.

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Ethics approval and consent to participate

All procedures performed in studies involving human participants were in accordance with the ethical standards of the institutional and/or national research committee and with the 1964 Helsinki Declaration and its later amendments or comparable ethical standards. The central ethics committee of the Medical Faculty, RWTH Aachen University (EK127/12), as well as the local ethics committees of the participating centers approved this registry. Informed consent was obtained from all individual participants included in the study.

Funding

The SAL-MPN registry had an initial funding from Novartis Pharma GmbH, Nürnberg, Germany.

Conflict of interest

AK, WH, CM, MH, EG, JRG, UP, TG, EJ, HS, GE, WEB, and THB declare that they have no conflict of interest. SI received honoraria (Pfizer, BMS) and travel grants (Roche, Mundipharma, Amgen, Hexal, Novartis, Pfizer). NG received research funding and lecture honoraria from Novartis. MK received funding from ARIAD, MBS, and Novartis. DW received research funding (Novartis, Ariad, Pfizer, AOP), a member of consultancy and advisory boards (Ariad, AOP, Baxalta, Bristol-Myers Squibb, Novartis, Pfizer, Sanofi), and received honoraria and travel grants (Ariad, AOP, Bristol-Myers Squibb, Novartis, Pfizer, Sanofi). SP received lecture honoraria and travel grants (Celgene, Novartis, Amgen, Bristol-Myers Squibb). SK received research funding (Novartis and Novartis Foundation), a member of consultancy and advisory boards (Ariad, AOP, Baxalta, Bristol-Myers Squibb, CTI, Novartis, Pfizer, Sanofi), and received honoraria and travel grants (Ariad, Alexion, AOP, Baxalta, Bristol-Myers Squibb, Celgene, CTI, Novartis, Pfizer, Safnofi, Shire).

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Kaifie, A., Isfort, S., Gattermann, N. et al. Health care setting and severity, symptom burden, and complications in patients with Philadelphia-negative myeloproliferative neoplasms (MPN): a comparison between university hospitals, community hospitals, and office-based physicians. Ann Hematol 95, 1399–1410 (2016). https://doi.org/10.1007/s00277-016-2730-y

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