Updated Recommendations for Selecting HCT Candidates in Patients with MDS

In this report from the European Society for Blood and Marrow Transplantation (EBMT), an international panel of experts present HCT treatment decision recommendations for patients with MDS.

Recommendations were classified as either patient-related or disease-related, and focus on determining which factors can best identify HCT candidates. The most relevant disease-related factors are highlighted in the table below, which shows the prognostic risk factors relevant for HCT eligibility and their corresponding measurement tools.

 Prognostic risk factors Measurement tools
 Age (chronological) Calendar, IPSS-R
 Performance status (functional ability) Karnofsky status ≥80%
 Frailty (reduced physical fitness)  Specific tools have to be tested in HCT
 Comorbidities HCT-specific Comorbidity Index (HCT-CI) 
 Percentage of marrow blasts IPSS(-R), WPSS, WHO
 Cytogenetic risk groups IPSS(-R), WPSS, CP
 Severity of cytopenias IPSS(-R), WPSS
 Marrow fibrosis WHO criteria
 Transfusions burden WPSS
 Flow cytometry  ELN flow cytometry score
 Molecular mutations No specific tools yet 
 Disease status (after non-HCT treatment interventions)  
 ESA failure High Epo levels, high transfusion intensity 
 Lenalidomide failure Absence of 5q- 
 HMA failure HMA-therapy-specific risk score 
 ICT MDS-specific risk score 
Table abbreviations: CI=comorbidity index, ELN=European Leukemia Net, ESA=erythropoietin-stimulating agent, Epo=erythropoietin, HMA=hypomethylating agents, ICT=intensive chemotherapy, WHO=World Health Organization, WPSS=WHO classification-based Prognostic Scoring System, CPSS=CMML-specific prognostic scoring system, IPSS-R=International Prognostic Scoring System-Revised

Clinical factors most likely to determine response to treatment modalities include intensive chemotherapy (ICT), hypomethylating agents (HMA), and immunomodulatory agents, such as lenalidomide, and hematopoietic growth factors.

The panel concluded that the most relevant clinical tools to determine HCT eligibility are the IPSS-R and HCT co-morbidity index. “Fit patients with higher-risk IPSS-R and those with lower-risk IPSS-R with poor-risk genetic features, profound cytopenias, and high transfusion burden are candidates for HCT.”

de Witte T, et al. Blood

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