How to use luspatercept and erythropoiesis‐stimulating agents in low‐risk myelodysplastic syndrome DOI Creative Commons
Valeria Santini, Angela Consagra

British Journal of Haematology, Journal Year: 2025, Volume and Issue: unknown

Published: May 2, 2025

Summary Anaemia is the most common cytopenia in myelodysplastic syndrome (MDS), significantly impacting quality of life and morbidity. Erythropoiesis‐stimulating agents (ESAs) are first‐line treatment for anaemia lower risk (LR)‐MDS. The European Medicines Agency (EMA) approved epoetin alpha LR‐MDS‐related 2017, based on evidence from a unique randomized Phase 3 trial accumulated many trials, providing support to an already widely utilized therapeutic option. Luspatercept, more recently agent, ligand trap transforming growth factor beta (TGF‐β) pathway, whose activation associated with impaired terminal erythroid maturation MDS. Luspatercept facilitates late‐stage differentiation, improving transfusion‐dependent LR‐MDS, has shown activity after ESA failure MDS‐ring sideroblasts (RS) all subtypes MDS naïve patients. Due recent approval luspatercept also it become crucial determine optimal algorithm anaemic before transfusion dependence. ESAs characterized by distinct mechanisms action, their integration into strategies possible, but requires further maximize efficacy improve patient outcomes.

Language: Английский

Elderly Patients With Aplastic Anemia: Treatment Patterns and Outcomes in the Real World DOI Creative Commons
Bruno Fattizzo, Carmelo Gurnari, Sabrina Giammarco

et al.

American Journal of Hematology, Journal Year: 2025, Volume and Issue: unknown

Published: Jan. 29, 2025

ABSTRACT We retrospectively analyzed a large international cohort of 1113 patients with aplastic anemia to evaluate treatment choice and outcome in elderly as compared younger population. Overall, 319 (29%) were > 60 years old at diagnosis (60–64 ( n = 85), 106 65–69 106), 128 70 128)). Elderly showed more severe thrombocytopenia onset significantly lower overall response (complete plus partial) first‐line therapy 6 months (47% vs. 65%, p < 0.0001), irrespective modality (ATG or CyA combinations, eltrombopag, androgens); 27 (8%) received transplant second line 11 (41%) died, mainly due complications. The rate evolution MDS was greater (12% 7% AA, 0.002), whilst the AML similar (1.8 1.3%). By multivariable analysis, older age remained main factor associated mortality [HR 1.64 (95% CI 1.5–1.7), 0.001], followed by disease severity Camitta classification 2.24 1.6–3.1) for AA; HR 3.8 2.4–6) very AA], male gender [1.45 1.1–1.8), 0.001]. In this study, AA inferior even TPO‐RA era, highlighting need further optimization clinical management.

Language: Английский

Citations

1
Germline variants in acquired aplastic anemia: current knowledge and future perspectives DOI Creative Commons

Peicheng Wang,

W J Jiang,

Tianyi Lai

et al.

Haematologica, Journal Year: 2024, Volume and Issue: unknown

Published: July 11, 2024

Aplastic anemia (AA) is a disease characterized by hematopoiesis failure, bone marrow aplasia, and pancytopenia. It can be inherited or acquired. Although acquired AA believed to immune-mediated random, new evidence suggests an underlying genetic predisposition. Besides confirmed genomic mutations that contribute (such as pathogenic of TERT TERC), germline variants, often in heterozygous states, also play unignorable role the onset progression AA. These associated with failure syndromes (IBMFS) inborn errors immunity (IEI), possibly through mechanisms including gene homeostasis, DNA repair, immune injury. This article explores nuanced association between detailed clinical significance variants diagnosing management this condition. More works are encouraged better understand immunogenic whether somatic mutation participate secondary “hit” development failure.

Language: Английский

Citations

4
How to use luspatercept and erythropoiesis‐stimulating agents in low‐risk myelodysplastic syndrome DOI Creative Commons
Valeria Santini, Angela Consagra

British Journal of Haematology, Journal Year: 2025, Volume and Issue: unknown

Published: May 2, 2025

Summary Anaemia is the most common cytopenia in myelodysplastic syndrome (MDS), significantly impacting quality of life and morbidity. Erythropoiesis‐stimulating agents (ESAs) are first‐line treatment for anaemia lower risk (LR)‐MDS. The European Medicines Agency (EMA) approved epoetin alpha LR‐MDS‐related 2017, based on evidence from a unique randomized Phase 3 trial accumulated many trials, providing support to an already widely utilized therapeutic option. Luspatercept, more recently agent, ligand trap transforming growth factor beta (TGF‐β) pathway, whose activation associated with impaired terminal erythroid maturation MDS. Luspatercept facilitates late‐stage differentiation, improving transfusion‐dependent LR‐MDS, has shown activity after ESA failure MDS‐ring sideroblasts (RS) all subtypes MDS naïve patients. Due recent approval luspatercept also it become crucial determine optimal algorithm anaemic before transfusion dependence. ESAs characterized by distinct mechanisms action, their integration into strategies possible, but requires further maximize efficacy improve patient outcomes.

Language: Английский

Citations

0