open access

Vol 5, No 2 (2014)
Review paper
Published online: 2014-09-04
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Quality of life of patients suffering from primary myelofibrosis is important parameter in assessment of treatment efficacy

Tomasz Sacha
Hematologia 2014;5(2):95-104.

open access

Vol 5, No 2 (2014)
REVIEW ARTICLES
Published online: 2014-09-04

Abstract

Myelofibrosis is a Philadelphia-negative myeloproiferative neoplasm arising from mutated abnormal hematopoietic stem cell. JAK2 mutation is detected in approximately 60% of patients. The constitutive dysregulation of the JAK-STAT and PI3K/AKT/mTOR signal transduction pathway plays a crucial role in disease pathogenesis and is present in all patients irrespective of their JAK2 mutational status. In majority of patients with primary myelofibrosis with non mutated JAK2, CALR mutations are detected. They cause cytokine-independent proliferation resulting from activation of STAT5 depended pathway. Myelofibrosis is characterized by several clinical symptoms of high intenisty that are usually highly prevalent among patients, can adversely affect quality of life and reflecting the burden of the disease could potentially limit patients overall survival. It is therefore of crucial importance to assess correctly the symptom burden among patients at the time of diagnosisand in the course of the disease. A standardised Myelofibrosis Symptom Assessment Form (MF-SAF) was developed, a new instrument measuring symptoms intensity and disease impact among patients with myelofibrosis. Its accuracy has been tested in COMFORT-1 and COMFORT-2 trials comparing ruxolitinib with placebo and the best available therapy respectively. The article presents currently used instruments for assessment of clinical symptoms and quality of life of patients with myelofibrosis and the results of analysis conducted using these tools.

Abstract

Myelofibrosis is a Philadelphia-negative myeloproiferative neoplasm arising from mutated abnormal hematopoietic stem cell. JAK2 mutation is detected in approximately 60% of patients. The constitutive dysregulation of the JAK-STAT and PI3K/AKT/mTOR signal transduction pathway plays a crucial role in disease pathogenesis and is present in all patients irrespective of their JAK2 mutational status. In majority of patients with primary myelofibrosis with non mutated JAK2, CALR mutations are detected. They cause cytokine-independent proliferation resulting from activation of STAT5 depended pathway. Myelofibrosis is characterized by several clinical symptoms of high intenisty that are usually highly prevalent among patients, can adversely affect quality of life and reflecting the burden of the disease could potentially limit patients overall survival. It is therefore of crucial importance to assess correctly the symptom burden among patients at the time of diagnosisand in the course of the disease. A standardised Myelofibrosis Symptom Assessment Form (MF-SAF) was developed, a new instrument measuring symptoms intensity and disease impact among patients with myelofibrosis. Its accuracy has been tested in COMFORT-1 and COMFORT-2 trials comparing ruxolitinib with placebo and the best available therapy respectively. The article presents currently used instruments for assessment of clinical symptoms and quality of life of patients with myelofibrosis and the results of analysis conducted using these tools.
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Keywords

myelofibrosis, quality of life, clinical symptoms, quality of life assesment methods

About this article
Title

Quality of life of patients suffering from primary myelofibrosis is important parameter in assessment of treatment efficacy

Journal

Hematology in Clinical Practice

Issue

Vol 5, No 2 (2014)

Article type

Review paper

Pages

95-104

Published online

2014-09-04

Bibliographic record

Hematologia 2014;5(2):95-104.

Keywords

myelofibrosis
quality of life
clinical symptoms
quality of life assesment methods

Authors

Tomasz Sacha

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