REVIEW ARTICLE


Health-Related Quality of Life Assessment in Patients with Myelodysplastic Syndromes: Evidence from Randomized Clinical Trials



Johannes M. Giesinger1, Giorgio La Nasa2, *, Francesco Sparano3, Matthias Angermeyer4, Emanuela Morelli2, Olga Mulas2, Fabio Efficace3, Giovanni Caocci2
1 Medical University of Innsbruck, University Hospital of Psychiatry II, Innsbruck, Austria
2 Ematologia e CTMO, Ospedale Businco, ARNAS “G. Brotzu”, Cagliari, Italy
3 Italian Group for Adult Hematologic Diseases (GIMEMA), Data Center and Health Outcomes Research Unit, Rome, Italy
4 Center for Public Mental Health, University of Vienna, Gosim am Wagram, Austria


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Creative Commons License
© 2021 Giesinger et al.

open-access license: This is an open access article distributed under the terms of the Creative Commons Attribution 4.0 International Public License (CC-BY 4.0), a copy of which is available at: https://creativecommons.org/licenses/by/4.0/legalcode. This license permits unrestricted use, distribution, and reproduction in any medium, provided the original author and source are credited.

* Address correspondence to this author at the Ematologia e CTMO, Ospedale Businco, Dipartimento di Scienze Mediche e Sanità Pubblica, Università di Cagliari, Via Jenner, sn, 09124 Cagliari, Italy; Tel: +39-70-52964901; Fax: +39-70-52965317; E-mail: lanasa@tiscali.it


Abstract

Myelodysplastic syndromes (MDS) are characterized by ineffective hematopoiesis and blood cytopenia with a variable risk of progression to acute myeloid leukemia. The main goal of therapy for the large majority of patients is to improve health-related quality of life (HRQoL). Its rigorous assessment is now recommended in international MDS guidelines. Our review provides an overview of HRQoL results from randomized controlled trials (RCTs) in MDS patients. The literature search undertaken in PubMed identified 10 RCTs with HRQoL endpoints (all secondary) published between August 2008 and September 2020. These RCTs have helped to better understand the impact of therapies from the patient perspective and have generated valuable information that can be used to further support clinical decisions. However, the number of RCTs in MDS patients, including HRQoL endpoints, is still low. Given the importance of symptom relief and HRQoL improvement in the treatment of MDS patients, the assessment of the patient perspective in future RCTs is highly recommended to keep expanding the knowledge of the impact of new MDS therapies.

Keywords: Myelodysplasia, Health related quality of life, IPSS, Erythropoietin, Lenalidomide, HRQoL.