Established and novel agents for myelodysplastic syndromes

Abstract

The myelodysplastic syndromes (MDS) are the most commonly diagnosed myeloid malignancy, with Ͼ15 000 new cases identified in the United States yearly. Prognostic scoring systems supplant a formal staging approach and, in general, divide patients into those with lower-risk and those with higher-risk MDS. Although treatment goals for patients with lower-risk disease focus on minimizing transfusions and optimizing quality of life, in higher-risk MDS, the goal is to delay transformation to acute leukemia and to prolong survival. In lower-risk patients, isolated cytopenias are treated with erythropoiesis-stimulating agents or growth factors such as thrombopoietin mimetics. For patients with the del(5q) cytogenetic abnormality or those who fail these initial approaches, lenalidomide may be tried, as can experimental agents. Lower-risk patients with multiple cytopenias may be treated with immunosuppressive drugs or low-dose hypomethylating agents. For patients with higher-risk disease, hypomethylating agents are the preferred initial treatment approach, with evaluation for hematopoietic cell transplantation at diagnosis. Several novel agents are being developed for MDS patients who have failed hypomethylating drugs. Learning Objective• To describe appropriate therapies for lower-and higher-risk myelodysplastic syndromes and novel agents being developed

A systematic classification of megakaryocytic dysplasia and its impact on prognosis for patients with myelodysplastic syndromes

“…Median follow-up was 22 months (range 1–180 months). Subjects with lower-risk MDS fall into the international prognostic scoring system-revised (IPSS-R) categories of very low-, low-, and intermediate-risk groups and those with higher-risk MDS into the high- and very high-risk groups [ 20 ].…”

Myelodysplastic Syndromes

“…47,48 The goals of MDS therapy depend on prognostic assessment and the individual patient profile. For lower-risk patients, common goals of therapy include symptom control and quality of life, improvement of hematopoiesis, and delay of disease progression.…”

Nowotwory mieloproliferacyjne i zespoły mielodysplastyczne — postępy w diagnostyce i terapii

“…Należy jednak brać pod uwagę istotną hepato-i nefrotoksyczność leku [35,44]. Próby zastosowania eltrombopagu i romiplostymu u chorych na MDS i małopłytkowość wykazały ich skuteczność w zakresie zwiększenia liczby płytek i obniżenia ryzyka krwawienia, natomiast romiplostym powodował zwiększenie liczby blastów, co spowodowało przerwanie próby klinicznej [45,46].…”