Incorporating symptomatic assessment in therapy choice for patients with myeloproliferative neoplasms

Robyn M. Scherber, Holly L. Geyer, Ruben A. Mesa

Research output: Chapter in Book/Report/Conference proceedingChapter


Introduction Essential thrombocythemia (ET), polycythemia vera (PV), and myelofibrosis (MF) represent a unique cohort of myeloid malignancies with variable and frequently progressive symptom profiles. Both ET and PV often begin with limited symptom burdens and, in select patients, may result in a normal life expectancy. For those patients who face disease progression, the course often includes secondary complications such as thrombotic and bleeding difficulties, transformation to MF and/or acute myelogenous leukemia (AML). In contrast to ET and PV, MF is often highly symptomatic at the time of diagnosis and follows a progressive clinical course with a cumulative symptom burden that has historically proven difficult to treat. With each of these disorders comes the challenge of managing the symptoms that compromise patient quality of life, treatment options, and potentially prognosis. The timely development of myeloproliferative neoplasm (MPN)-specific patient-reported outcome (PRO) tools (i.e. Myelofibrosis Symptoms Assessment Form (MF-SAF), MPN Symptom Assessment Form (MPN-SAF), MPN-SAF Total Symptom Score (MPN-TSS) (MPN-10)) has allowed us to objectively quantify the MPN symptom burden and monitor response to therapies. In this chapter, we discuss how MPN PRO tools may be successfully incorporated into the routine assessment and management of MPN disorders. Case 1 A 46-year-old man is found at the time of a general physical examination to have thrombocytosis and is sent to you for evaluation. This individual, upon questioning, does not seem to clearly have symptoms related to an MPN. He has a hemoglobin of 13.6 g/dL, leukocyte count of 8.9 × 109/L, and platelet count of 1,100 × 109/L. A bone marrow aspirate and biopsy are performed and the patient is found to have histologic features consistent with World Health Organization (WHO) diagnosis of ET with no fibrosis, and no increase in blasts. This individual was found to be mutated for calreticulin (CALR). Your initial choice of therapy is aspirin. Additionally, you administer the MPN 10-item symptomatic questionnaire. This individual is minimally symptomatic with a score of 9 out of a possible 100. The patient is monitored with aspirin therapy alone as there are no strong cardiovascular risk factors and he is considered at low risk for ET by the International Prognostic Scoring System for ET (IPSET). The patient is followed, but now 18 months after therapy starts complaining of erythromelalgia that is unresponsive to aspirin therapy.

Original languageEnglish (US)
Title of host publicationManaging Myeloproliferative Neoplasms
Subtitle of host publicationA Case-Based Approach
PublisherCambridge University Press
Number of pages11
ISBN (Electronic)9781316017852
ISBN (Print)9781107444430
StatePublished - Jan 1 2016
Externally publishedYes

ASJC Scopus subject areas

  • General Medicine


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