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 |
Patient-related | |
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) |
Disease-related | |
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 |
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.”