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Clinical effectiveness and safety of erythropoietin‐stimulating agents for the treatment of low‐ and intermediate‐1−risk myelodysplastic syndrome: a systematic literature review
Author(s) -
Park Sophie,
Greenberg Peter,
Yucel Aylin,
Farmer Caroline,
O'Neill Frank,
De Oliveira Brandao Cisio,
Fenaux Pierre
Publication year - 2019
Publication title -
british journal of haematology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.907
H-Index - 186
eISSN - 1365-2141
pISSN - 0007-1048
DOI - 10.1111/bjh.15707
Subject(s) - medicine , darbepoetin alfa , epoetin alfa , myelodysplastic syndromes , anemia , erythropoietin , intensive care medicine , clinical trial , systematic review , oncology , medline , bone marrow , political science , law
Summary Many patients with lower‐risk myelodysplastic syndrome ( MDS ) experience anaemia, which has negative consequences. Erythropoiesis‐stimulating agents ( ESA s) and their biosimilars are used to treat anaemia in MDS and, currently, epoetin alfa and darbepoetin alfa are commonly used and recommended by clinical guidelines. To better understand the evidence available on the use of ESA s for anaemia in lower‐risk MDS , we conducted a systematic literature review to identify randomized and nonrandomized prospective studies reporting on clinical efficacy/effectiveness, patient‐reported quality of life (QoL), and safety. We extended our review to include retrospective studies for darbepoetin alfa specifically and to ascertain the feasibility of completing an indirect network meta‐analysis comparing epoetin and darbepoetin alfa. Overall, 53 articles reporting on 35 studies were included. The studies indicated a clinical benefit of ESA s, with benefits observed across key clinical outcomes. ESA s showed consistent improvement in erythroid response rates ( ESA ‐naïve, 45–73%; previous ESA exposure, 25–75%) and duration of response. Comparative studies demonstrated similar progression to acute myeloid leukaemia and several showed improved overall survival and QoL. Limited safety concerns were identified. This analysis confirmed ESA therapy should be the foremost first‐line treatment of anaemia in most patients with lower‐risk MDS who lack the 5q deletion.