‘We cannot paint them all with the same brush’: the need for a better definition of patients with myelodysplastic syndromes for clinical trial design

Gurnari, Carmelo; Visconte, Valeria

doi:10.1111/bjh.17909

Cited by 5 publications

(2 citation statements)

References 13 publications

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“…This includes disentangling the role of specific genetic mutations along a complex structure of gene–gene interactions [ 66 , 67 ]. Indeed, almost all MDS/AML patients harbor ≥1 mutation at the time of diagnosis, a situation even more complex at later stages whereby clonal evolution mechanisms may further complicate the molecular architecture [ 66 , 68 ]. This adds on to the extensive subclonal diversification and complexity underlying the mechanisms of resistance of primary and secondary HMA failure.…”

Section: Management Of Patients After Hma Failurementioning

confidence: 99%

What’s Next after Hypomethylating Agents Failure in Myeloid Neoplasms? A Rational Approach

Awada

Gurnari

Xie

et al. 2023

Cancers

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Hypomethylating agents (HMA) such as azacitidine and decitabine are a mainstay in the current management of patients with myelodysplastic syndromes/neoplasms (MDS) and acute myeloid leukemia (AML) as either single agents or in multidrug combinations. Resistance to HMA is not uncommon, and it can result due to several tumor cellular adaptations. Several clinical and genomic factors have been identified as predictors of HMA resistance. However, the management of MDS/AML patients after the failure of HMA remains challenging in the absence of standardized guidelines. Indeed, this is an area of active research with several potential therapeutic agents currently under development, some of which have demonstrated therapeutic potential in early clinical trials, especially in cases with particular mutational characteristics. Here, we review the latest findings and give a rational approach for such a challenging scenario.

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Section: Management Of Patients After Hma Failurementioning

confidence: 99%

What’s Next after Hypomethylating Agents Failure in Myeloid Neoplasms? A Rational Approach

Awada

Gurnari

Xie

et al. 2023

Cancers

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“…Nevertheless, in a study on long-term outcomes of higher-risk MDS patients treated with HMA and not undergone subsequent HSCT, only 4% of cases were alive at 5 years from treatment start, regardless of the type of HMA used [72]. Therefore, participation of patients not eligible for HSCT in clinical trials is strongly recommended, especially after HMA failure [73,74]. Of note is that no difference in rates of CR, ORR and survival has been shown between the two drugs AZA and DEC, and this finding has been demonstrated not only in MDS but also in AML patients unfit for intensive chemotherapy [75,76].…”

Section: Treatment Of Higher-risk Mdsmentioning

confidence: 99%

How I Manage Transplant Ineligible Patients with Myelodysplastic Neoplasms

2022

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Myelodysplastic neoplasms, formerly known as myelodysplastic syndromes (MDS), represent a group of clonal disorders characterized by a high degree of clinical and molecular heterogeneity, and an invariable tendency to progress to acute myeloid leukemia. MDS typically present in the elderly with cytopenias of different degrees and bone marrow dysplasia, the hallmarks of the disease. Allogeneic hematopoietic stem cell transplant is the sole curative approach to date. Nonetheless, given the disease’s demographics, only a minority of patients can benefit from this procedure. Currently used prognostic schemes such as the Revised International Prognostic Scoring System (R-IPSS), and most recently the molecular IPSS (IPSS-M), guide clinical management by dividing MDS into two big categories: lower- and higher-risk cases, based on a cut-off score of 3.5. The main clinical problem of the lower-risk group is represented by the management of cytopenias, whereas the prevention of secondary leukemia progression is the goal for the latter. Herein, we discuss the non-transplant treatment of MDS, focusing on current practice and available therapeutic options, while also presenting new investigational agents potentially entering the MDS therapeutic arsenal in the near future.

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When to use which molecular prognostic scoring system in the management of patients with MDS?

Kewan,

Bewersdorf,

Gurnari

et al. 2023

Best Practice & Research Clinical Haematology

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‘We cannot paint them all with the same brush’: the need for a better definition of patients with myelodysplastic syndromes for clinical trial design

Cited by 5 publications

References 13 publications

What’s Next after Hypomethylating Agents Failure in Myeloid Neoplasms? A Rational Approach

What’s Next after Hypomethylating Agents Failure in Myeloid Neoplasms? A Rational Approach

How I Manage Transplant Ineligible Patients with Myelodysplastic Neoplasms

When to use which molecular prognostic scoring system in the management of patients with MDS?

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