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Patients, physicians hold different views of myeloproliferative neoplasm prognosis, treatment goals

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October 19, 2016

Patients receiving treatment for myeloproliferative neoplasms often held discordant views regarding disease prognosis, burden and treatment goals compared with their treating physicians, according to data from the MPN Landmark Study.

Hematologists and oncologists often overestimated their patients’ ability to recognize disease-related symptoms, results also showed.

Ruben A. Mesa

“Many of the therapies that we offer for patients with myeloproliferative neoplasms are clearly noncurative,” Ruben A. Mesa, MD, FACP, professor of medicine, chair of the division of hematology and medical oncology, and deputy director of Mayo Clinic Cancer Center in Phoenix, Arizona, as well as a HemOnc Today Editorial Board member, told HemOnc Today. “Their goal is improvement of symptoms, such as the reduction of thrombosis or bleeding, reduction of splenomegaly, or improvement of anemia. But, a detailed discussion of symptoms can be perceived as a time-intensive process, and in the context of a busy clinical interaction, inadequate amounts of time are often reserved for in-depth conversations.”

The MPN Landmark Study included data from 813 patients with myeloproliferative neoplasms — including myelofibrosis (n = 207), polycythemia vera (n = 380) and essential thrombocythemia (n = 226) — and 457 practicing physicians who completed subtype-specific surveys (myelofibrosis, n = 156; polycythemia vera, n = 250; essential thrombocythemia, n = 51).

Symptom assessment

The majority of physicians in each category reported using prognostic risk stratifications to classify their patients (myelofibrosis, 83%; polycythemia vera, 59%; essential thrombocythemia, 77%). In contrast, fewer patients reported that their physician stratified their disease based on a prognostic category (myelofibrosis, 54%; polycythemia vera, 17%; essential thrombocythemia, 31%).

A greater proportion of physicians than patients reported that the physician asked about the patient’s most important symptoms (myelofibrosis, 55% vs. 20%; polycythemia vera, 51% vs. 15%; essential thrombocythemia, 55% vs. 18%) or asked about a comprehensive list of symptoms (myelofibrosis, 52% vs. 35%; polycythemia vera, 54% vs. 33%; essential thrombocythemia, 65% vs. 23%).

“It is fairly rare for clinicians to use questionnaire-based symptom assessments for their patients,” Mesa said. “We feel that purely verbal, unstructured symptom assessments lead to inadequate alignment of the recognition of symptom burden between patients and physicians.”

In several domains, patients did not recognize symptoms commonly related to myeloproliferative neoplasms as being disease related. This discordance was most pronounced with regard to difficulty sleeping (myelofibrosis, 49%; polycythemia vera, 64%; essential thrombocythemia, 76%); dizziness, vertigo or lightheadedness (myelofibrosis, 40%; polycythemia vera, 37%; essential thrombocythemia, 48%); or abdominal discomfort (myelofibrosis, 23%; polycythemia vera, 54%; essential thrombocythemia, 66%).

Physicians responded that most of their patients felt anxiety about their condition (myelofibrosis, 99%; polycythemia vera, 100%; essential thrombocythemia, 98%), had difficulty focusing (myelofibrosis, 97%; polycythemia vera, 96%; essential thrombocythemia, 90%) and avoided social interactions (myelofibrosis, 92%; polycythemia vera, 83%; essential thrombocythemia, 84%).

Treatment goals, expectations

The researchers observed high levels of discordance between physicians and patients with regard to treatment goals and expectations.

The most common treatment goal among patients with myelofibrosis (42%) and polycythemia vera (25%) was to slow or delay disease progression. However, physicians treating patients with myelofibrosis reported symptom improvement as their most important goal for their patients (53%), whereas physicians treating patients with polycythemia vera identified prevention of vascular or thrombotic events as theirs (43%).

“Patients have a strong desire to delay disease progression,” Mesa said. “Ideally, physicians view this as a goal as well, but this is a point of efficacy that many therapies do not offer. This represents a frequent misperception that occurs when a patient receives a therapy in order to control thrombosis but has the expectation that it will delay disease progression.”

The largest percentages of patients and physicians in the essential thrombocythemia setting identified prevention of vascular or thrombotic events as their most important goal (patients, 35%; physicians, 57%).

Patient satisfaction reports also varied widely. Greater than one-third of patients in all subgroups reported being less than very satisfied with physician management and physician communication.

An additional subset of patients described themselves as “somewhat dissatisfied” or “very dissatisfied” with physician management (myelofibrosis, 8%; polycythemia vera, 10%; essential thrombocythemia, 12%) and physician communication (myelofibrosis, 9%; polycythemia vera, 13%; essential thrombocythemia, 16%).

“Patient education regarding current treatment goals is important,” Mesa said. “It is also helpful to clarify what is known and not known regarding disease course and disease progression. Due to the overall rarity of myeloproliferative neoplasms, there continue to be misperceptions, deficits in understanding and confusion regarding many endpoints. We hope that improved patient education can help clarify where a patient stands with this complex and chronic disease.” – by Cameron Kelsall

For more information:

Ruben A. Mesa, MD, FACP, can be reached at

Disclosure: Mesa reports a consultant role with Novartis and research funding from CTI BioPharma, Genentech, Gilead, Incyte Corporation, NS Pharma, Pfizer and Promedior. Please see the full study for a list of all other researchers’ relevant financial disclosures.

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Srdan Verstovsek

Mesa and colleagues published results from a landmark survey outlining differences in treatment goals and perceptions of symptom burden between patients with myeloproliferative neoplasms (MPNs) and their hematologist/oncologists. Their findings suggested a significant discrepancy between patient and physician perception of disease-related symptom burden and treatment goals. In particular, communication between patients and physicians needs to be improved, especially with regard to the initial prognostic assessment and the severity of MPN–related symptoms at diagnosis. These factors affect both timing of therapy and a choice of treatment for patients with MPN. Deficient communication and establishment of correct baseline assessment, therefore, may significantly affect patients’ outcomes. Disparities in patient- and physician-reported treatment goals suggest that patients and physicians have different perceptions of the problem: Physicians generally had a more realistic assessment of the achievable treatment goals.

A very surprising and worrisome finding was the fact that nearly 50% of patients with MPNs reported that they had changed their MPN physician. In addition, 75% of physicians reported a lack of compliance on the patients’ part with treatment recommendations. These findings suggest a lack of trust between patients and physicians and highlight a major need for improved communication between patients and physicians, as well as better education of patients regarding their disease and available therapies/realistic goals.

Physicians could improve communication with patients by spending more time at the initial consultation educating patients about disease prognosis and symptom burden, as well as providing a more realistic assessment of what can be achieved by current therapies. Using a questionnaire to assess MPN–related symptom burden at each visit also may also improve communication and can help both the patient and physician track specific changes in symptom burden with time. Finally, the development of new therapies that align with patients’ needs may improve trust between physicians and patients. 

Srdan Verstovsek, MD, PhD

HemOnc Today Editorial Board member

The University of Texas MD Anderson Cancer Center

Disclosure: Verstovsek reports no relevant financial disclosure.