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Monosomal karyotype improves IPSS-R stratification in MDS and AML patients treated with Azacitidine
Thomas Cluzeau1, Nicolas Mounier, Jean-Michel Karsenti
1Centre Méditerranéen de médecine moléculaire, INSERM U1065, CHU Nice, Hôpital Archet 1, Service Hématologie Clinique, 151 Route Saint Antoine de Ginestière, Nice, France. cluzeau.thomas@gmail.com
Abstract:
IPSS-R classifies cytogenetic abnormalities into five prognostic groups for survival. Monosomal karyotype (MK) is not a subgroup of IPSS-R. Additional prognostic information from MK in poor and very poor karyotype has been recently shown. The aim of our study was to determine the prognostic value of IPSS-R and MK for response and survival in AZA-treated high-risk MDS and AML with 20-30% of blasts patients. The study population included 154 patients who were classified according to IPSS-R. IPSS-R was not predictive of response (intermediate, 64%; poor, 44%; very poor, 56%; P = 0.28) or survival (intermediate, 25 months; poor, 12 months; very poor, 11 months; P = 0.14). Twenty-one patients (15%) presented with MK and had a median OS of 9 months. Patients with a very high IPSS-R score without MK had a median OS of 15 months, while patients with a high IPSS-R score without MK had a median OS of 13 months (P = 0.18). We reclassified patients into the following three groups to include MK status: very high (MK only; OS median: 9 months), high (very high IPSS-R without MK and high IPSS-R without MK; OS median: 14 months) and intermediate (OS median: 25 months). As in recent publication including MK prognostic, we confirmed that this classification was predictive for survival in AZA treated patients (P = 0.008). IPSS-R failed to discriminate between the prognostic subgroups. Stratification with MK has value in the prognosis of our cohort of AZA-treated patients.
Insights
The International Prognostic Scoring System-Revised (IPSS-R) alone does not predict outcomes in patients with high-risk myelodysplastic syndromes (MDS) treated with azacitidine (AZA). Incorporating monosomal karyotype (MK) status improves prognostic accuracy for survival in this AZA-treated cohort.
Area of Science:
- Hematology
- Oncology
- Genetics
Background:
- The International Prognostic Scoring System-Revised (IPSS-R) is a standard tool for classifying myelodysplastic syndromes (MDS) based on cytogenetic abnormalities.
- Monosomal karyotype (MK) is an independent adverse prognostic factor in MDS, but its integration with IPSS-R requires further investigation, especially in patients receiving specific treatments.
- Azacitidine (AZA) is a hypomethylating agent used to treat high-risk MDS and acute myeloid leukemia (AML) with low blast counts.
Purpose of the Study:
- To evaluate the prognostic value of IPSS-R and MK for treatment response and survival in patients with high-risk MDS and AML (20-30% blasts) treated with AZA.
- To determine if combining IPSS-R with MK status can improve prognostic stratification compared to IPSS-R alone in this patient population.
Main Methods:
- A cohort of 154 patients with high-risk MDS/AML receiving AZA was retrospectively analyzed.
- Patients were initially classified according to IPSS-R.
- MK status was assessed, and a novel classification incorporating both IPSS-R and MK was developed and validated for survival prediction.
Main Results:
- IPSS-R alone was not predictive of response or survival in AZA-treated patients (P=0.28 for response, P=0.14 for survival).
- Patients with MK (15% of the cohort) had a significantly shorter median overall survival (OS) of 9 months.
- A reclassification incorporating MK status (very high [MK only], high [IPSS-R high/very high without MK], intermediate) significantly predicted survival (P=0.008), outperforming IPSS-R alone.
Conclusions:
- IPSS-R alone is insufficient for predicting outcomes in AZA-treated high-risk MDS/AML patients.
- Incorporating monosomal karyotype status into prognostic models significantly improves survival prediction in this cohort.
- Stratification using a combined IPSS-R and MK classification offers valuable prognostic information for guiding treatment decisions in AZA-treated MDS/AML.
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