Myelodysplastic neoplasms: An overview on diagnosis, risk-stratification, molecular pathogenesis, and treatment

Marques, Fabiana Kalina; Sabino, Adriano de Paula

doi:10.1016/j.biopha.2022.113905

Cited by 8 publications

(16 citation statements)

References 74 publications

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“…11 Clinical presentation reflects the existing inadequate hematopoiesis, with profound anemia being the most prevalent feature, while neutropenia is responsible for recurrent infections. 11 Thrombocytopenia, which is observed in up to 25%, is associated with increased bleeding risk and is predictive of early death. 12 Management of MDS patients is individualized and depends mostly on clinical status, MDS subtype and prognosis, and the presence of specific mutations that pose as targets for novel treatments.…”

Section: Thrombopoietin Receptor Agonists In Myelodysplastic Neoplasmsmentioning

confidence: 99%

“…Regarding thrombocytopenia, therapeutic choices are confined. 11 Although platelet transfusions play a primary role in patients with lifelong thrombocytopenia, they have several drawbacks; since they are associated with short-term efficacy, they have limited availability, and they also bear the risk of acute reactions and alloimmunization with subsequent refractoriness to latter transfusions. 11,12 Considering that TPO-RAs effectively incite for expansion of HSCs and stimulate for bi-or tri-lineage hematopoiesis, their utility was also investigated in MDS.…”

Section: Thrombopoietin Receptor Agonists In Myelodysplastic Neoplasmsmentioning

confidence: 99%

“…11 Although platelet transfusions play a primary role in patients with lifelong thrombocytopenia, they have several drawbacks; since they are associated with short-term efficacy, they have limited availability, and they also bear the risk of acute reactions and alloimmunization with subsequent refractoriness to latter transfusions. 11,12 Considering that TPO-RAs effectively incite for expansion of HSCs and stimulate for bi-or tri-lineage hematopoiesis, their utility was also investigated in MDS. ROMI was first evaluated in a prospective phase I/II trial of patients with low-risk MDS and thrombocytopenia on supportive care only and the results showed a raise in platelet counts in most of the patients, while a durable response was observed in 46%, who remained transfusion free for 8 weeks.…”

Section: Thrombopoietin Receptor Agonists In Myelodysplastic Neoplasmsmentioning

confidence: 99%

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Novel Perspectives on Thrombopoietin Receptor Agonists Applications

Stafylidis,

Vlachopoulou,

Syriopoulou

et al. 2024

Hamostaseologie

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Second-generation thrombopoietin receptor agonists (TPO-RAs), romiplostim, eltrombopag, and avatrombopag, have been proved to be significant stimulators of megakaryopoiesis and, in the last decade, they have been incorporated in the treatment options against refractory immune thrombocytopenia in children and adults that do not respond to conventional therapy. Additionally, given their beneficial impact on hematopoiesis, they have successfully been applied in cases of non-immune thrombocytopenia, such as aplastic anemia, HCV-related thrombocytopenia, chronic liver disease, and most recently acute radiation syndrome. During the past years, a wide variety of clinical studies have been performed, in regard to the use of TPO-RAs in various thrombocytopenic settings, such as malignant hematology and hematopoietic stem cell transplantation, hereditary thrombocytopenias, and chemotherapy-treated patients with solid organ tumors. Although data indicate that TPO-RAs may be an effective and safe option for managing disease- or treatment-related thrombocytopenia in these patients, further research is needed to determine their efficacy and safety in these settings. Furthermore, recent studies have highlighted novel properties of TPO-RAs that render them as potential treatment candidates for reducing tumor burden or fighting infections. Herein, we discuss the potential novel applications of TPO-RAs and focus on data regarding their efficacy and safety in these contexts.

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Section: Thrombopoietin Receptor Agonists In Myelodysplastic Neoplasmsmentioning

confidence: 99%

Section: Thrombopoietin Receptor Agonists In Myelodysplastic Neoplasmsmentioning

confidence: 99%

Section: Thrombopoietin Receptor Agonists In Myelodysplastic Neoplasmsmentioning

confidence: 99%

See 1 more Smart Citation

Novel Perspectives on Thrombopoietin Receptor Agonists Applications

Stafylidis,

Vlachopoulou,

Syriopoulou

et al. 2024

Hamostaseologie

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“…Due to the genetic heterogeneity of MDS, the therapeutic options currently available for MDS are limited. The treatment of MDS is influenced by the disease characteristics, age of the patient, and comorbidities which vary from supportive care to hematopoietic cell transplantation [ 96 ]. As can be observed, current therapeutic options in cases diagnosed with MDS are recommended in accordance with the patient’s risk stratification, which is based on scoring systems (IPSS and IPSS-R).…”

Section: Treatment Of Myelodysplastic Neoplasmmentioning

confidence: 99%

“…As can be observed, current therapeutic options in cases diagnosed with MDS are recommended in accordance with the patient’s risk stratification, which is based on scoring systems (IPSS and IPSS-R). In patients with low-risk MDS, the aims of the treatment are represented by the improvement of cytopenias, especially the symptoms related to anemia, and the reduction in the number of required transfusions [ 96 , 97 ]. In high-risk MDS patients, the aim of the treatment is to prevent the progression of the disease and to improve survival [ 98 ].…”

Section: Treatment Of Myelodysplastic Neoplasmmentioning

confidence: 99%

The Genetic Landscape of Myelodysplastic Neoplasm Progression to Acute Myeloid Leukemia

Bănescu

Tripon

Muntean

2023

IJMS

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Myelodysplastic neoplasm (MDS) represents a heterogeneous group of myeloid disorders that originate from the hematopoietic stem and progenitor cells that lead to the development of clonal hematopoiesis. MDS was characterized by an increased risk of transformation into acute myeloid leukemia (AML). In recent years, with the aid of next-generation sequencing (NGS), an increasing number of molecular aberrations were discovered, such as recurrent mutations in FLT3, NPM1, DNMT3A, TP53, NRAS, and RUNX1 genes. During MDS progression to leukemia, the order of gene mutation acquisition is not random and is important when considering the prognostic impact. Moreover, the co-occurrence of certain gene mutations is not random; some of the combinations of gene mutations seem to have a high frequency (ASXL1 and U2AF1), while the co-occurrence of mutations in splicing factor genes is rarely observed. Recent progress in the understanding of molecular events has led to MDS transformation into AML and unraveling the genetic signature has paved the way for developing novel targeted and personalized treatments. This article reviews the genetic abnormalities that increase the risk of MDS transformation to AML, and the impact of genetic changes on evolution. Selected therapies for MDS and MDS progression to AML are also discussed.

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Myelodysplastic Syndrome

Fuchs

2024

Comprehensive Hematology and Stem Cell Research

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Myelodysplastic neoplasms: An overview on diagnosis, risk-stratification, molecular pathogenesis, and treatment

Cited by 8 publications

References 74 publications

Novel Perspectives on Thrombopoietin Receptor Agonists Applications

Novel Perspectives on Thrombopoietin Receptor Agonists Applications

The Genetic Landscape of Myelodysplastic Neoplasm Progression to Acute Myeloid Leukemia

Myelodysplastic Syndrome

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